moderna-ir-v2

Statement on the Safety of Spikevax

Fri, 12 Sep 2025 22:29:28 GMT

Cambridge, MA – September 12, 2025 — The safety of Spikevax is rigorously monitored by Moderna, the U.S. FDA, and regulators in more than 90 countries. Multiple, overlapping safety monitoring systems are in place that work to detect and evaluate any new or evolving safety considerations. With more than one billion doses distributed globally, these systems – including in national health systems across Europe, the United Kingdom, Canada, Australia, and the U.S. - have not reported any new or undisclosed safety concerns in children or in pregnant women.

Moderna is not aware of any deaths in the last year or pertinent new information from prior years. Published, peer-reviewed research by multiple governments, independent investigators and by Moderna has demonstrated the safety profile of Spikevax.

Additional context:

• COVID-19 vaccination has not been shown to cause miscarriage, stillbirth, preterm birth, or birth defects. Importantly, vaccination protects pregnant individuals from severe COVID-19, which is linked to higher rates of maternal complications and mortality. Similarly, global pediatric surveillance data continues to demonstrate the safety profile of Spikevax in pediatric populations.

Moderna Recognizes the Success of Operation Warp Speed and Affirms Its Ongoing Commitment to Transparency

Wed, 03 Sep 2025 16:43:41 GMT

Cambridge, MA – September 3, 2025 — Moderna is committed to transparency and welcomes the opportunity to highlight the extensive research regarding our COVID-19 vaccine, which has been published in hundreds of peer-reviewed publications and shared with regulators.

Moderna is a research-driven, American company and we are immensely proud of the role we played in the historic achievements of Operation Warp Speed. As President Trump said at the time , “we are the most exceptional nation in the history of the world… American companies were the first to produce a verifiably safe and effective vaccine.” These vaccines helped end the COVID-19 pandemic, re-open economies and save millions of lives.

Since 2021, the real-world effectiveness and safety of the vaccines have been confirmed by governments and health systems around the world. Moderna itself has conducted vaccine effectiveness studies every year, in more than 5 million people to-date. These studies consistently show that the Moderna COVID-19 vaccine confers a high degree of protection against hospitalizations among the most vulnerable. We have also actively monitored the safety of the more than one billion doses administered to-date, and have always transparently shared those results with regulators and with the public, including through peer-reviewed publications and presentations. We will continue to do so as part of our commitment to transparency.

The promise of mRNA is immense. The same technology that helped turn the tide of a global pandemic is now paving the way for breakthroughs in cancer, autoimmune and rare diseases. To help ensure Americans have clear and accessible information, Moderna will be compiling previously disclosed information on our website at the link here .

Forward Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding: the efficacy, safety and tolerability of Moderna's COVID vaccines; the potential for Moderna’s platform to address rare diseases and cancer; and Moderna’s publication of vaccine data. The forward-looking statements in this press release are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Moderna's control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include, among others, those risks and uncertainties described under the heading "Risk Factors" in Moderna's Annual Report on Form 10-K for the fiscal year ended December 31, 2024, and in subsequent filings made by Moderna with the U.S. Securities and Exchange Commission, which are available on the SEC's website at www.sec.gov. Except as required by law, Moderna disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Moderna's current expectations and speak only as of the date of this press release.

IR Insights: Recapping our Second Quarter of 2025 Earnings Report

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Fri, 01 Aug 2025 13:58:35 GMT

Earlier today, Moderna reported financial results and provided business updates for the second quarter of 2025.

Stéphane Bancel, CEO, Stephen Hoge, President, and Jamey Mock, CFO, sat down for an episode of IR Insights to provide more insight into our financial performance and upcoming late-stage pipeline milestones.

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Moderna Announces an Organizational Restructuring

Thu, 31 Jul 2025 11:05:08 GMT

Cambridge, MA — July 31, 2025 — Moderna today announced an organizational restructuring that will reduce its global workforce by approximately 10 percent. This action is part of the Company’s previously stated plan to decrease estimated GAAP operating expenses by $1.4 to $1.7 billion by 2027 and better align its cost structure and capabilities with current business conditions while also sustaining investment in its mRNA pipeline. Affected employees will receive severance, equity vesting and comprehensive transition support. Moderna anticipates a total headcount of under 5,000 employees by year-end.

A related message from CEO Stéphane Bancel was emailed to all Moderna employees on July 31, 2025. Click here to read more.

A Difficult But Necessary Step Forward

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Thu, 31 Jul 2025 11:05:01 GMT

Moderna has added a new blog to its website.

Moderna announced an organizational restructuring as part of the Company’s previously stated plan to decrease GAAP operating expenses and better align its cost structure and capabilities with current business conditions.

A related message from CEO Stéphane Bancel was emailed to all Moderna employees on July 31, 2025. Click here to read more >

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IR Insights: Recapping our First Quarter of 2025 Earnings Report

Thu, 01 May 2025 19:24:13 GMT

Moderna has added a new blog to its website

Earlier today, Moderna reported financial results and provided business updates for the first quarter of 2025.

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IR Insights: Recapping our Fourth Quarter and Fiscal Year 2024 Earnings Report

Fri, 14 Feb 2025 13:33:31 GMT

Moderna has added a new blog to its website

Earlier today, Moderna reported financial results and provided business updates for the fourth quarter and fiscal year of 2024.

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IR Insights: Recapping Business and Pipeline Updates from the 43rd Annual J.P. Morgan Healthcare Conference

Mon, 13 Jan 2025 16:00:03 GMT

Moderna has added a new blog to its website Earlier today, Moderna presented an update on Moderna’s business and pipeline of mRNA development programs at the 43rd Annual J.P. Morgan Healthcare Conference. Stéphane Bancel, CEO, and Jamey Mock, CFO, sat down for an episode of IR Insights to give an overview of key highlights from the presentation. Click here to read the blog and watch the video

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Moderna – 2024 Shareholder Letter from CEO Stéphane Bancel

Mon, 06 Jan 2025 13:28:26 GMT

Moderna has added its 2024 Shareholder Letter from CEO Stéphane Bancel to its website.

As we reflect on 2024, it is clear that the past year brought formidable challenges for Moderna along with significant advances, particularly for our development pipeline. With a continuous learning mindset, we analyzed these outcomes and adapted our business strategy based on our successes and challenges. We are confident that with our industry-leading mRNA platform, technology, resources and team, we are well-positioned to execute this strategy and continue building a future where Moderna leads in healthcare innovation, delivering on our Mission for patients in 2025 and beyond.

Click here to read the full 2024 shareholder letter from Moderna CEO Stéphane Bancel

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Moderna – The Hidden Cost of COVID: Long COVID's Impact on the Workforce

Fri, 20 Dec 2024 16:33:13 GMT

Moderna has added a new blog to its website

Long COVID is rippling across businesses, increasing illness in the workforce, reducing productivity and driving up healthcare costs. Employers are on the front lines of this challenge and are key to the solution. By prioritizing prevention with updated COVID-19 vaccinations and implementing supportive policies, employers can protect their workforce and mitigate the economic toll of Long COVID. These actions aren’t just good for employees—they’re strategic investments in productivity, resilience and the bottom line.

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Moderna’s Commitment to Patient Access in the United States

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Thu, 12 Dec 2024 15:09:39 GMT

Moderna’s vaccines have protected the lives of hundreds of millions of people around the world from COVID-19 and have dramatically lessened the burden of the pandemic to society.

As the public health emergency ends, the United States government will no longer be providing vaccines at no cost. Moderna remains committed to ensuring that people in the United States will have access to our COVID-19 vaccines regardless of ability to pay.

Moderna’s COVID-19 vaccines will continue to be available at no cost for insured people whether they receive them at their doctors’ offices or local pharmacies. For uninsured or underinsured people, Moderna’s patient assistance program* will provide COVID-19 vaccines at no cost.

Everyone in the United States will have access to Moderna’s COVID-19 vaccine regardless of their ability to pay.

*Available after the expiry of the COVID-19 Public Health Emergency on May 11, 2023.

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IR Insights: Recapping our Third Quarter 2024 Earnings Report

Thu, 07 Nov 2024 16:30:05 GMT

Moderna has added a new blog to its website

Earlier today, Moderna reported financial results and provided business updates for the third quarter of 2024. Members of our executive team sat down to provide more insight into our financial performance and upcoming late-stage pipeline milestones. Watch our latest IR Insights video below to hear more from our CEO Stéphane Bancel, President Stephen Hoge, and CFO Jamey Mock.

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Celebrating U.S. FDA Approval of Our Updated COVID-19 Vaccine

Thu, 22 Aug 2024 16:25:02 GMT

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COVID-19 Summer Surge: Three Things to Know

Thu, 15 Aug 2024 16:21:30 GMT

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IR Insights: Recapping our Second Quarter 2024 Earnings Report

Thu, 01 Aug 2024 16:19:58 GMT

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Understanding Long COVID’s Impact on the Workforce

Wed, 31 Jul 2024 16:10:50 GMT

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Impacting Human Health: Reflecting on Moderna’s Third Annual ESG Report

Tue, 25 Jun 2024 16:09:09 GMT

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Marking CMV Awareness Month in June

Mon, 24 Jun 2024 16:07:35 GMT

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The Potential Impact of Combination Vaccines on Public Health

Mon, 10 Jun 2024 16:06:04 GMT

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Moderna Licenses COVID-19-related Intellectual Property to a Pharmaceutical Company in Japan

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Sun, 28 Apr 2024 14:18:23 GMT

Moderna non-exclusively out-licenses COVID-19-related patents for the territory of Japan
Moderna to receive upfront payment and low double-digit royalty on net sales of the company's COVID-19 product

Moderna has entered a non-exclusive out-licensing agreement with a pharmaceutical company based in Japan for mRNA COVID-19-related intellectual property for the territory of Japan.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding: Moderna’s licensing of its intellectual property and terms thereof. The forward-looking statements in this press release are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Moderna's control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include, among others, those risks and uncertainties described under the heading "Risk Factors" in Moderna's Annual Report on Form 10-K for the fiscal year ended December 31, 2023, filed with the U.S. Securities and Exchange Commission (SEC), and in subsequent filings made by Moderna with the SEC, which are available on the SEC's website at www.sec.gov. Except as required by law, Moderna disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Moderna's current expectations and speak only as of the date of this press release.

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Statement on Kenya Manufacturing Facility

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Thu, 11 Apr 2024 14:34:38 GMT

Moderna has paused its efforts to build an mRNA manufacturing facility in Kenya while it determines future demand for mRNA vaccines on the African continent. The demand in Africa for COVID-19 vaccines has declined since the pandemic and is insufficient to support the viability of the factory planned in Kenya. Moderna has not received any vaccine orders for Africa since 2022 and has faced the cancellation of previous orders, resulting in more than $1 billion in losses and write-downs. Despite these challenges, Moderna is committed to ensuring equitable access and meeting emerging demands from African nations for its COVID-19 vaccine through its global manufacturing network.

Moderna is actively working on the development of public health vaccines, including those for diseases that predominantly affect the African continent, such as HIV and malaria. These initiatives are part of our broader commitment to help address global health challenges through our innovative mRNA technology. However, these investigational vaccines are at an early development stage. Given this, and in alignment with our strategic planning, Moderna believes it is prudent to pause its efforts to build an mRNA manufacturing facility in Kenya. This approach will allow Moderna to better align its infrastructure investments with the evolving healthcare needs and vaccine demand in Africa.

Moderna appreciates the strong support received from the governments of the United States and Kenya on this journey. We look forward to a continuing dialogue about future options to support the Kenyan healthcare ecosystem, including as we advance other mRNA vaccines and therapeutics of importance to the African continent through our pipeline.

Forward-Looking Statement Disclaimer

This statement contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding: Moderna’s plans for meeting the evolving healthcare needs and vaccine demand in Africa, and ensuring equitable access; and Moderna’s development of public health vaccines to help address global health challenges. The forward-looking statements in this post are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties and other factors, many of which are beyond Moderna’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties and other factors include, among others, those risks and uncertainties described under the heading “Risk Factors” in Moderna’s Annual Report on Form 10-K for the fiscal year ended December 31, 2023, filed with the U.S. Securities and Exchange Commission (SEC), and in subsequent filings made by Moderna with the SEC, which are available on the SEC’s website at www.sec.gov . Except as required by law, Moderna disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this presentation in the event of new information, future developments or otherwise. These forward-looking statements are based on Moderna’s current expectations and speak only as of the date of this statement.

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IR Insights: Recapping our Fifth Annual Vaccines Day

Wed, 27 Mar 2024 15:57:55 GMT

Earlier today, Moderna hosted its fifth annual Vaccines Day, where our leadership shared key updates on the Company’s growing infectious disease portfolio. Members of our executive team sat down to dive into key clinical updates across Moderna’s respiratory, latent and other vaccine portfolios, as well as our financial and commercial vaccine strategy. Watch our latest IR Insights video to hear more from Stéphane Bancel, CEO, Dr. Stephen Hoge, President, Jamey Mock, CFO and Dr. Jackie Miller, SVP and Head of Development for Infectious Diseases.

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Long COVID Awareness Day: Amplifying the Patient Voice

Fri, 15 Mar 2024 15:55:49 GMT

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Statement on RSVVW Abstract on mRNA-1345

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Thu, 08 Feb 2024 14:29:52 GMT

At an upcoming presentation at the RSVVW’24 conference, Moderna will share follow-up data from its ongoing ConquerRSV Phase 3 study of its investigational RSV vaccine candidate, mRNA-1345.

As previously published in the New England Journal of Medicine, the primary analysis from this trial demonstrated vaccine efficacy (VE) of 83.7% (95.88% CI: 66.1%, 92.2%; p<0.0001) against RSV lower respiratory tract disease (RSV-LRTD) as defined by two or more symptoms and VE of 82.4% (96.36% CI: 34.8%, 95.3%; p=0.0078) in RSV-LRTD as defined by three or more symptoms with a median follow-up of 3.7 months. Solicited adverse reactions were mostly mild to moderate (grade 1 or grade 2, resolving within 1 to 2 days). The most frequently reported were injection site pain, fatigue, headache, myalgia, and arthralgia. No safety concerns were identified. No cases of Guillain-Barre Syndrome (GBS) have been reported with mRNA-1345 in the Phase 3 RSV trial.

The upcoming presentation includes follow-up data through a data cut off of April 30, 2023, with a median follow-up duration of 8.6 months, with a range of 15 days to 530 days, and including subjects from the Northern and Southern Hemispheres. In this supplemental analysis, mRNA-1345 maintained durable efficacy, with sustained VE of 63.3% (95% CI: 48.7%, 73.7%) against RSV-LRTD including two or more symptoms. VE was 74.6% (95% CI, 50.7, 86.9) against RSV-LRTD with ≥2 symptoms, including shortness of breath, and VE was 63.0% (95% CI, 37.3%, 78.2%) against RSV LRTD including three or more symptoms. The stringent statistical criterion of the study, a lower bound on the 95% CI of >20%, continued to be met for both endpoints.

Comparisons across seasons are challenging with respiratory viruses, particularly in the last several years. For example, the 2022-2023 RSV season in the United States was characterized by higher incidence and hospitalization rates, which at their peak were approximately four-fold higher than in the prior season, with a longer duration of sustained transmission, as demonstrated by CDC data.[1] The 2021-2022 season was shortened significantly by countermeasures that were taken against the Omicron variant during the COVID pandemic.

In the absence of head-to-head clinical trials, comparative conclusions regarding the safety and efficacy of mRNA-1345 relative to other RSV vaccines cannot be made. These trials differed in study populations, geographic locations, infection surveillance methods, and case definitions used for RSV.

Moderna is anticipating approvals of mRNA-1345 in the first half of 2024. The Company is encouraged by the strong competitive profile for its RSV vaccine, with robust efficacy data meeting all pre-specified statistical criteria, a well-established safety and tolerability profile, and as the only pre-filled syringe product available at the time of launch.

The full RSVVW’24 presentation will be available on February 15, 2024 on the Scientific & Medical Meetings section of Moderna’s investor website. The abstract from the upcoming presentation is available on page 88, at the following link: https://resvinet.org/wp-content/uploads/2024/02/Abstract-Booklet-08Feb24.pdf.

Forward-Looking Statement Disclaimer

This statement contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including regarding the safety, efficacy and competitive profile for Moderna’s RSV vaccine candidate, mRNA-1345. The forward-looking statements here are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond the Company’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include those other risks and uncertainties described under the heading “Risk Factors” in the Company’s most recent Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (“SEC”) and in subsequent filings made by the Company with the SEC, which are available on the SEC’s website at www.sec.gov. Except as required by law, the Company disclaims any intention or responsibility for updating or revising any forward-looking statements contained here in the event of new information, future developments or otherwise. These forward-looking statements are based on the Company’s current expectations and speak only as of the date hereof.

[1] See https://www.cdc.gov/rsv/research/rsv-net/dashboard.html

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Respiratory Syncytial Virus (RSV): What You Need to Know

Wed, 07 Feb 2024 15:53:31 GMT

Moderna has added a new blog to its website

Cases of RSV are on the rise again this winter, and as RSV becomes a seasonal concern, it’s critical we understand its burden and who is most at risk. Read our latest blog to learn more about RSV and what Moderna is doing to address it.

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Initiation of the tender procedure for mRNA COVID-19 vaccines by the Health Emergency Preparedness and Response Authority (HERA) of the European Commission (EC)

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Tue, 16 Jan 2024 14:24:54 GMT

Initiation of the tender procedure for mRNA COVID-19 vaccines by the Health Emergency Preparedness and Response Authority (HERA) of the European Commission (EC)

Moderna welcomes the initiation of a tendering procedure for mRNA COVID-19 vaccines by the Health Emergency Preparedness and Response Authority (HERA) of the European Commission (EC) and intends to participate in this process. HERA was set up as a service of the EC to strengthen Europe's ability to prevent, detect, and rapidly respond to cross-border health emergencies.

As one of two mRNA COVID-19 vaccines approved for use in Europe alongside BioNTech-Pfizer’s vaccine, which has an agreement with the European Commission for the provision of COVID-19 vaccines until 2026, the initiated tendering procedure will allow Member States to secure access to a diversified portfolio of mRNA-based COVID-19 vaccines in 2024 and beyond.

A diversified supply of mRNA COVID-19 vaccines is vital for Europe and public health worldwide. mRNA technology has shown itself to be a proven platform for COVID-19 vaccine development through the pandemic. It has the necessary flexibility to mitigate the risk posed by future variants of concern and to facilitate respective vaccine updates.

Moderna has consistently shown its ability to develop, manufacture, and supply COVID-19 vaccines against circulating variants. Moderna’s updated COVID-19 vaccine for the 2023 vaccination season continues to provide an immune response against currently circulating strains of COVID-19, including JN.1.

It is vital that healthcare professionals and the general public have a choice in addressing their vaccination needs in line with the latest scientific and real-world evidence. This is vital for vulnerable populations, such as the elderly and immunocompromised, and to help bolster the readiness of healthcare systems across the European Union.

Key stakeholders in Europe, such as the European Medicines Agency, have expressed the need for a diversified portfolio of COVID-19 vaccines. As we look towards the 2024 vaccination season and associated preparation, it will be important to allow adequate time to prepare the capacity required for manufacturers to manufacture and supply COVID-19 vaccines for this year’s season, taking into account expected recommendations from global public health authorities and regulators on the potential need for variant updated vaccines.

Forward-Looking Statement Disclaimer

This statement contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including regarding: the potential tender process for COVID-19 vaccines by the HERA, the terms of the potential tender; the benefits of a diversified supply of mRNA COVID-19 vaccines for Europe; the strengths of Moderna’s platform; and the potential for Moderna to secure additional sales of COVID-19 vaccines to Member States. The forward-looking statements here are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond the Company’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include those other risks and uncertainties described under the heading “Risk Factors” in the Company’s most recent Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (“SEC”) and in subsequent filings made by the Company with the SEC, which are available on the SEC’s website at www.sec.gov. Except as required by law, the Company disclaims any intention or responsibility for updating or revising any forward-looking statements contained here in the event of new information, future developments or otherwise. These forward-looking statements are based on the Company’s current expectations and speak only as of the date hereof.

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Moderna 2023 Shareholder Letter

Tue, 02 Jan 2024 15:45:28 GMT

Moderna has added its 2023 Shareholder Letter from CEO Stéphane Bancel to its website

As we reflect on the past year, our commitment to leveraging mRNA to deliver transformative medicines for patients has never been stronger. We have the platform, technology, resources and the team to establish a new era of medicine. In his annual shareholder letter, Moderna CEO Stéphane Bancel provides updates on our journey to advance our Mission to deliver the greatest possible impact to people through mRNA medicines.

Click here to read the full 2023 Shareholder Letter from Moderna CEO Stéphane Bancel

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How Moderna is Helping Advance the Fight Against Cancer

Mon, 18 Dec 2023 18:08:10 GMT

Last week, we announced follow-up data from our phase 2b study evaluating our investigational individualized neoantigen therapy in combination with KEYTRUDA, Merck’s anti-PD-1 therapy, in patients with high-risk melanoma following complete resection. Watch the next installment of our IR Insights video series to hear more from Dr. Stephen Hoge, Moderna’s president, and Dr. Kyle Holen, our Head of Development in Oncology and Therapeutics.

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Moderna Statement on Anticipated 2023 COVID-19 Sales

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Mon, 16 Oct 2023 14:43:45 GMT

Moderna remains comfortable that its guided wide range of $6 billion to $8 billion for anticipated revenues from sale of its COVID-19 vaccine for the full year 2023, as provided in its second quarter earnings release dated August 3, 2023, reflects the uncertainty of U.S. vaccination rates.

As noted previously, if the U.S. market for COVID-19 vaccines is approximately 50 million administered doses, Moderna still expects to be in the bottom half of the disclosed range; if the U.S. market is approximately 100 million administered doses, Moderna still expects to be in the top half of the disclosed range. Moderna believes it is still too early in the U.S. vaccination season to accurately project where vaccination rates will land for the full year.

Moderna anticipates that it will have improved visibility about the expected U.S. market size after seeing full vaccination trends through October 2023. Moderna will provide an update at its earnings call on November 2, 2023.

Forward-Looking Statement Disclaimer

This statement contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including regarding: the anticipated range of potential revenues from the sale of Moderna’s COVID-19 vaccine for 2023; the factors impacting COVID-19 vaccine revenues; and Moderna’s future visibility into revenues based on vaccination trends. The forward-looking statements here are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond the Company’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include those other risks and uncertainties described under the heading “Risk Factors” in the Company’s most recent Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (“SEC”) and in subsequent filings made by the Company with the SEC, which are available on the SEC’s website at www.sec.gov. Except as required by law, the Company disclaims any intention or responsibility for updating or revising any forward-looking statements contained here in the event of new information, future developments or otherwise. These forward-looking statements are based on the Company’s current expectations and speak only as of the date hereof.

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R&D Day Reflections with CFO Jamey Mock

Mon, 25 Sep 2023 18:09:48 GMT

Earlier this month, we hosted our seventh annual Research & Development Day at NASDAQ, where we showcased our expansion of the field of mRNA medicine to analysts and investors. This included several significant clinical updates across oncology, rare and infectious diseases and details on Moderna’s business aspirations for the years to come.

Jamey Mock, Chief Financial Officer of Moderna, reflects on our ambitious business goals outlined during R&D Day – including how we’ll get there, Moderna’s investment priories, and the financial and clinical promise of our mRNA platform.

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Moderna’s efforts to ensure significant supply of COVID-19 vaccines reaches pharmacies and points of care in the U.S.

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Thu, 21 Sep 2023 14:49:11 GMT

Immediately after receiving U.S. FDA approval, Moderna shipped millions of updated COVID-19 vaccines to our distributors across the United States. We are working to support these distributors to ensure that the significant supply of vaccines we have already provided to them continues to rapidly reach pharmacies and other points of care. Additional supply is being shipped daily as we continue to see high demand. Moderna continues to have an ample supply of updated COVID-19 vaccine available, with an additional 6 million doses available as of today.

Forward-Looking Statements

This statement contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding: Moderna’s efforts to ensure significant supply of COVID-19 vaccines reaches pharmacies and points of care, demand for COVID-19 vaccines, and the sufficiency of Moderna's supply of vaccines. The forward-looking statements here are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Moderna's control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include those other risks and uncertainties described under the heading "Risk Factors" in Moderna's Annual Report on Form 10-K for the year ended December 31, 2022, filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent filings made by Moderna with the SEC, which are available on the SEC's website at www.sec.gov. Except as required by law, Moderna disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Moderna's current expectations and speak only as of the date of this press release.

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Moderna Ramps Down Mrna Drug Substance Production For Its Covid-19 Vaccine At Lonza

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Tue, 19 Sep 2023 15:04:48 GMT

Moderna will ramp down production of mRNA drug substance for its COVID-19 vaccine at Lonza’s site in Visp, Switzerland, in Q3 2023. This is part of the Company’s plan to right-size its manufacturing footprint in line with the resizing of the COVID-19 vaccine market due to the move from a pandemic to an endemic market. Moderna expects to absorb the demand currently supported by Lonza at its internal manufacturing site in Norwood, MA for 2024 – 2025. In 2025, Moderna expects additional capacity at its new mRNA manufacturing facilities in the UK, Canada, and Australia when completed. Moderna continues to maintain external drug substance capacity at Rovi in Spain.

Moderna is grateful for Lonza’s support in helping to scale up and meet the manufacturing demands during a global public health emergency. Lonza’s activities in Visp have been crucial in enabling Moderna to deliver its vaccine worldwide, including Switzerland.

At R&D Day, Moderna announced that it continues to expect 2023 COVID-19 sales of $6 billion to $8 billion, dependent on U.S. vaccination rates. While 2023 cost of sales remain elevated, the Company is currently resizing its manufacturing footprint and supply base to accelerate gross margin expansion towards its longer-term target of 75-80%.

Forward-Looking Statements

This statement contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding: Moderna’s plans to ramp down manufacturing at Lonza’s site in Visp, Switzerland; Moderna’s future manufacturing of COVID-19 vaccines; Moderna’s anticipated COVID-19 vaccine sales for 2023, the factors impacting potential sales; and Moderna’s cost of sales and expected margins for 2023. The forward-looking statements here are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Moderna's control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include those other risks and uncertainties described under the heading "Risk Factors" in Moderna's Annual Report on Form 10-K for the year ended December 31, 2022, filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent filings made by Moderna with the SEC, which are available on the SEC's website at www.sec.gov. Except as required by law, Moderna disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Moderna's current expectations and speak only as of the date of this press release.

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Our Continued Innovation at Work: Celebrating U.S. FDA Approval of Our Updated COVID-19 Vaccine

Mon, 11 Sep 2023 18:11:29 GMT

I am pleased to announce that the U.S. Food and Drug Administration (FDA) has approved our supplemental Biologics License Application (sBLA) for Spikevax® (COVID-19 Vaccine, mRNA) for individuals 12 years of age and older, which helps protect against the most dominant variants as recommended by the June 2023 FDA VRBPAC. This timely approval helps ensure everyone will have the tools they need to do their part in protecting themselves and slowing the spread of the virus as we head into the fall and winter months in the U.S.

Read Stéphane Bancel's full blog post here

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3 Things to Know About the 2023-2024 COVID-19 Season

Wed, 06 Sep 2023 18:13:28 GMT

It has been more than three years since the pandemic first changed life as we know it, and while we’re no longer wiping down groceries and staying six feet apart, one thing remains clear: COVID-19 isn’t going away anytime soon. According to the CDC, COVID-19 is on the rise again as of late August – Wastewater monitoring suggests a recent rise in infections in parts of the U.S., and hospitalizations have increased 24% in a two-week period.¹ The urge to put COVID-19 behind us is something we can all relate to. But, in the face of rapidly circulating new variants, we know better than to ignore what’s around the corner as we head into the fall and winter seasons.

Read Moderna’s full blog post to learn how to navigate the upcoming season

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Building the Next Generation of Moderna: Meet Our Spring Co-Ops By: Tracey Franklin, Chief Human Resources Officer

Fri, 28 Jul 2023 18:15:05 GMT

By: Tracey Franklin, Chief Human Resources Officer

As we continue to build our company, we are committed to accelerating the next generation of leaders. Our people are truly amazing, and it’s a privilege to support such a diverse community of culture carriers and to inspire careers in STEM. The Moderna co-op program is a powerful example of how we are building the future of Moderna.

Read Tracey Franklin’s full blog post and learn more about how Moderna is building their next generation

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Taking Action and Raising Awareness: The Impact of Cytomegalovirus (CMV)

Wed, 28 Jun 2023 18:22:14 GMT

By: Patricia Gauthier, VP, Global Franchise Leader – Latent Viruses

CMV is a latent virus that is often mistaken for a common cold. More than half of U.S. adults over the age of 40 have been infected at some point in their lives, but pregnant women can unknowingly pass the infection to their unborn babies, resulting in devastating complications. ¹

Congenital CMV is the most common infectious cause of birth defects and developmental disabilities in the U.S., impacting approximately one in 200 babies – more common than Down syndrome, fetal alcohol syndrome, and spina bifida. One in five babies with born with CMV will have severe, life-altering health problems, such as vision impairment, hearing loss, or decreased muscle strength and coordination. ^2,3

Read Patricia Gauthier’s full blog post and learn from families impacted by CMV

References

1. https://www.cdc.gov/cmv/clinical/overview.html

2. https://www.cdc.gov/cmv/congenital-infection.html

3. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3539797/#:~:text=This%20corresponds%20to%20approximately%2040%2C000,syndrome%2C%20and%20spina%20bifida2 .

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Impacting Human Health: Reflecting on our ESG Progress

Wed, 14 Jun 2023 18:23:20 GMT

By: Shannon Thyme Klinger, Chief Legal Officer

Earlier this year, we evolved our mission to better reflect our belief that Moderna’s mRNA medicines will continue to have a profound impact on human health. We are working to deliver on our commitments to tackle some of the biggest challenges to public health, which include unequal access to quality healthcare and medicines, inequalities affecting vulnerable communities around the world, and the increasing negative effects of climate change.

Our annual Environmental, Social and Governance (ESG) Report outlines our corporate social responsibility (CSR) strategy and progress aligned to five guiding focus areas for ongoing and future efforts.

Read Shannon Thyme Klinger’s full blog post here

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Exploring One Medicine for One Patient: Individualized Neoantigen Therapies

Mon, 05 Jun 2023 18:24:36 GMT

By: Michelle Brown, M.D., Ph.D., Program Leader, Oncology

Each person’s cancer is different because cancer cells have genetic mutations that make them unique, which can make the cancer difficult to treat. That's why we, in collaboration with Merck, are researching mRNA medicines that are designed specifically for an individual patient. Individualized neoantigen therapies (INTs) are an innovative approach that tailor treatment to each patient's individual cancer.

This week, we are participating in the American Society for Clinical Oncology (ASCO) Annual Meeting, where distant metastasis-free survival (DMFS) results from the Phase 2b study of our investigational mRNA individualized neoantigen therapy, mRNA-4157 (V940), in combination with KEYTRUDA® (pembrolizumab), Merck’s anti-PD-1 therapy, for the adjuvant treatment of patients with high-risk stage III/IV melanoma following complete resection, will be presented. The ASCO Annual Meeting is the world’s largest oncology conference, and we are honored to share these new data with the global oncology community.

We are incredibly grateful to the participants in our clinical trials and the investigators and clinical trial staff who are helping to advance this important cancer research.

Read Michelle’s full blog post

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Leveraging 13 Years of Investment in mRNA Innovation for Future Pandemic Preparedness

Wed, 24 May 2023 16:20:53 GMT

By: Hamilton Bennett, Senior Director of Vaccine Access and Partnerships

At Moderna, we know what it means to push past possible – especially when talking about time to response. Just two days after the first complete sequence of the COVID-19 virus was made publicly available online in January of 2020, Moderna's COVID-19 vaccine candidate was under development. Today, it’s been approved in more than 70 countries. While I am proud of the impact Moderna had in ending this pandemic, we must not lose sight of the millions of lives lost over the last three years. Unfortunately, COVID-19 will not be our last pandemic, but the lessons that can be learned will help us better prepare for the next one.

Read Hamilton’s full blog post

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University of Minnesota Commencement Remarks to the Class of 2023

Mon, 15 May 2023 16:17:23 GMT

By: Stéphane Bancel, Chief Executive Officer

This past weekend, I was honored to deliver a commencement speech  to the graduating Class of 2023 at the University of Minnesota. In the speech, I shared my experience on three topics: Making the world a better place, obsessing about learning and people.

I believe these are the key ingredients that led a kid from Marseille, France, who sat in the 1995 University of Minnesota graduating class, to have the opportunity to build a company like Moderna.

As I shared with the graduates, I care deeply about making the world a better place. About helping people and their loved ones to prevent diseases, to diagnose diseases and to treat diseases.

To accomplish such an impact, it requires a village, but more importantly, it requires people who are Mission driven. People who care. People who can collaborate because it is NOT about them, but it is about the Mission. And it requires that all of us keep learning so we can do it better each day, because each day matters.

Congratulations to all the 2023 graduates. You have a unique opportunity to make the world a better place. How you will decide to do it, is up to you.

Multimedia Files:

University of Minnesota Commencement Remarks to the Class of 2023

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Statement Regarding Section 1498 Motion

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Mon, 23 May 2022 15:33:03 GMT

The claims brought by Alnylam are unfounded because Moderna’s COVID-19 vaccine does not infringe any valid patents. Moderna quickly responded when the pandemic struck, swiftly developing, manufacturing, and providing doses of its COVID-19 vaccine to people around the world.

At this preliminary stage of the litigation, we have moved to dismiss Alnylam’s claims relating to the sale of COVID-19 vaccine doses to the U.S. Government because Alnylam has sued the wrong party in the wrong court. Under Federal law, claims against U.S. Government-contracted suppliers must proceed against the Government in the U.S. Court of Federal Claims. This law provides an important statutory protection for authorized government suppliers and played a critical role in encouraging companies, including Moderna, to step up and help the Government fight the COVID-19 pandemic.

Ultimately, however, Moderna’s lipids do not resemble Alnylam’s work. Any assertion that the Alnylam patent covers Moderna’s COVID-19 vaccine is specious, and, as a result, Alnylam’s claims will fail even if Alnylam complies with the statutory requirements and refiles in the Court of Federal Claims against the U.S. Government.

Forward Looking Statements

This statement contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including regarding: the Company’s rights to its intellectual property related to its COVID-19 vaccine; litigation related to that intellectual property; and the appropriate counterparty and venue in any suit related to the infringement of intellectual property where the ultimate customer is the U.S. Government. The forward-looking statements here are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Moderna's control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include those other risks and uncertainties described under the heading "Risk Factors" in Moderna's most recent Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent filings made by Moderna with the SEC, which are available on the SEC's website at www.sec.gov. Except as required by law, Moderna disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Moderna's current expectations and speak only as of the date hereof.

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Departure of Jorge Gomez and Continuation of David Meline as Chief Financial Officer

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Wed, 11 May 2022 15:34:26 GMT

On Wednesday, May 11, Moderna announced that its recently appointed Chief Financial Officer, Jorge Gomez, has departed the Company, effective immediately. This announcement follows the May 10 public disclosure by Mr. Gomez’s former employer, Dentsply Sirona Inc., of an internal investigation into its financial reporting practices.

Moderna’s recently retired Chief Financial Officer, David Meline, will continue in his role of Chief Financial Officer (CFO) as of May 11 while Moderna reopens its search for a new CFO.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding: the resignation and appointment of the Company’s Chief Financial Officer. In some cases, forward-looking statements can be identified by terminology such as “will,” “may,” “should,” “could,” “expects,” “intends,” “plans,” “aims,” “anticipates,” “believes,” “estimates,” “predicts,” “potential,” “continue,” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. The forward-looking statements in this press release are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Moderna’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include, among others, those risks and uncertainties described under the heading “Risk Factors” in Moderna’s most recent Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent filings made by Moderna with the SEC, which are available on the SEC’s website at www.sec.gov. Except as required by law, Moderna disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Moderna’s current expectations and speak only as of the date hereof.

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Statement Regarding Section 1498 Motion

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Fri, 06 May 2022 16:28:06 GMT

The claims brought by Genevant and Arbutus are unfounded because Moderna’s COVID-19 vaccine does not infringe any valid patents. Moderna quickly responded when the pandemic struck, swiftly developing, manufacturing, and providing doses of its COVID-19 vaccine to people around the world.

At this preliminary stage of the litigation, we have moved to dismiss plaintiffs’ claims relating to the sale of COVID-19 vaccine doses to the U.S. Government because plaintiffs have sued the wrong party in the wrong court. Under Federal law, claims against U.S. Government-contracted suppliers must proceed against the Government in the U.S. Court of Federal Claims. This law provides an important statutory protection for authorized government suppliers and played a critical role in encouraging companies, including Moderna, to step up and help the Government fight the COVID-19 pandemic.

Ultimately, however, given that Moderna’s vaccine does not infringe any valid patents, plaintiffs’ claims will fail even if Arbutus and Genevant comply with the statutory requirements and refile in the Court of Federal Claims against the U.S. Government.

Forward Looking Statements

This statement contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including regarding: the Company’s rights to its intellectual property related to its COVID-19 vaccine; litigation related to that intellectual property; and the ability of claimants to bring claims against the Company or the U.S. Government for alleged patent infringement. The forward-looking statements here are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Moderna's control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include those other risks and uncertainties described under the heading "Risk Factors" in Moderna's most recent Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent filings made by Moderna with the SEC, which are available on the SEC's website at www.sec.gov. Except as required by law, Moderna disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Moderna's current expectations and speak only as of the date hereof.

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Global Access to COVID-19 Vaccines

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Thu, 28 Apr 2022 18:44:39 GMT

Today, at the Moderna 2022 Annual Meeting of Shareholders, shareholders voted against the shareholder proposal requesting that we commission a report assessing the feasibility of transferring our intellectual property related to our COVID-19 vaccine (76% voted against; 24% voted for). We share the concern that the proponents and others have for ensuring equitable access to COVID-19 vaccines. Since 2020, we have taken several actions to ensure that we are able to meet demand for our vaccine in low- and middle-income countries. We are committed to continuing to address those needs and to help address long-term issues that have historically led to a gap in vaccine access. Today’s vote indicates that the significant majority of our shareholders are supportive of the approach we have taken, and we will continue to address issues related to vaccine access and communicate with shareholders and others on this topic.

Expanding Our Manufacturing Capacity

From the earliest days of the COVID-19 pandemic, the team at Moderna has worked tirelessly to help combat it. Through these efforts, hundreds of millions of individuals have been vaccinated around the world. Throughout, our goal has been to produce as much of our vaccine as possible, to protect as many people as possible across the globe.

When it became clear that there would be significant demand and need for mRNA COVID-19 vaccines early in 2021, the Moderna Board of Directors responded swiftly, authorizing additional investments to significantly expand our manufacturing capacity, both internal and external, with a goal of addressing the need for vaccines, particularly in low- and middle-income countries.

The Company partnered with more than half a dozen established manufacturers to ramp up production around the globe, including Lonza, Baxter, Catalent, Recipharm, Rovi, Samsung, Thermo Fisher and others. Our efforts, and those of our manufacturing partners, have produced significant results, and this year, we anticipate that our capacity will be more than sufficient to meet demand. This is no small feat for a company that had not produced a commercial product prior to December 2020.

Committing Vaccines to COVAX and the African Union

Beginning in the summer of 2020, the Moderna team was engaged with Gavi, the Vaccine Alliance, on behalf of the COVAX Facility, hoping to secure a commitment from them to procure a significant number of Moderna COVID-19 vaccines. An agreement was not reached until April 2021, though we were pleased to commit up to 500 million doses to COVAX – a number that was subsequently increased to 650 million doses. Similarly, we were proud to reach an agreement with the African Union to supply 110 million doses, which we were prepared to start delivering as early as the fourth quarter of 2021. In each case, we offered these vaccines at our lowest price, and in the latest agreements the price for each of these organizations was $7 per 100 µg dose.

Despite our efforts, ultimately COVAX and the African Union deferred or declined hundreds of millions of doses of Moderna’s vaccine. While we were prepared to deliver tens of millions of doses to the African Union in December 2021, they asked us to delay delivery, noting that they did not have the means of distributing them. They also declined to exercise an option for 60 million doses that were available to them in the second quarter of this year.

Similarly, COVAX has declined options for over 320 million doses that Moderna was prepared to deliver in 2022, noting that they have ample access to vaccines. And even for those doses where COVAX submitted a firm order covering the first and second quarters, they have asked to defer delivery. As a result, Moderna is incurring significant costs as it winds down relationships with outside manufacturers who were engaged to produce these declined doses.

Asking Moderna to transfer intellectual property to local manufacturers—as the Oxfam America proposal suggested—when hundreds of millions of doses are being declined and already operating manufacturing plants are being idled will do nothing to accelerate the end of the pandemic. It would also require diverting Moderna personnel already engaged in manufacturing with other partners, or who are working on other initiatives, including the company’s Global Health strategy as described below.

We Are Committed to Helping Close the “Last Mile”

We recognize that closing the “last mile” on the distribution of vaccines remains a significant hurdle to closing the access gap. While Moderna cannot solve this problem alone, we are open to working with those working to solve this issue on the ground to help us ensure that doses are getting where they are needed.

Our Commitment to Closing the Access Gap and Advancing Global Health

Looking forward, Moderna is also committed to closing the gap in vaccine access that we have seen in the current COVID-19 pandemic. There are multiple prongs to this strategy:

We are committed to ensuring that our intellectual property, or concerns about enforcement of our intellectual property, do not pose a barrier to access. In October 2020, we were the first and only company to pledge not to enforce our patents for those making vaccines to fight Covid-19 during the pandemic. We have since announced that Moderna will never enforce its COVID-19 intellectual property rights in the Gavi-eligible AMC-92 countries, or against manufacturers who are producing COVID-19 vaccines for distribution in those markets.
We will internally fund and build an mRNA manufacturing plant in Kenya , capable of producing up to 500 million doses per year (at the 50 µg dose) so that Africa has its own local source of vaccines, and we can start to build an ecosystem of individuals with the knowledge of how to manufacture this new generation of medicines.
We are pursuing a technological solution to closing the access gap, working on a next generation version of our COVID-19 vaccine, mRNA-1283, that will be refrigerator stable so that countries in the developing world do not need to maintain rigorous cold chain storage requirements that are needed for current mRNA vaccines.
We have updated our approach to contracting, allowing us discretion to reprioritize deliveries to those countries that have the greatest need, rather than tying the timing for deliveries to the timing of a customer’s commitment.
Our Global Health strategy , announced in March 2022, is focused on the development of mRNA vaccines against the 15 highest priority pathogens that have been identified by the WHO and the Coalition for Epidemic Preparedness Innovations (CEPI), like tuberculosis, malaria and HIV, which pose a significant threat to the developing world. This includes preparedness against Disease X, which could be the next pathogen to cause a pandemic.
Our mRNA Access initiative is opening Moderna's preclinical manufacturing capabilities and research and development expertise to global partners, to together explore the possibility of mRNA to tackle the world’s greatest global public health threats.

We share with the global public health community as well as our shareholders, a binding commitment to seeing an end to the current COVID-19 pandemic, and we will continue to take all appropriate actions to address and combat the pandemic as quickly as possible.

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Moderna Recall Notification of Lot #000190A

Fri, 08 Apr 2022 18:50:19 GMT

Moderna and ROVI Pharma Industrial Services, S.A.U. today announced a recall of one lot (lot #000190A) of the Moderna COVID-19 vaccine (Spikevax®). The lot is being recalled due to a foreign body being found in one vial in the lot manufactured at the Company’s contract manufacturing site, ROVI, in Spain. The impacted vial was punctured and was not administered.

Moderna, through its marketing authorization holder, Moderna Biotech Spain, S.L., and ROVI, were alerted to this issue through a product complaint from a vaccination center in Malaga, Spain. The vial was returned for forensic assessment and investigation. This lot, which consisted of 764,900 doses, was distributed in Norway, Poland, Portugal, Spain, and Sweden from 13-14 January 2022.

Moderna conducted a cumulative search of its global safety database, and no safety concerns were reported in individuals who received the Moderna COVID-19 vaccine from this lot. To date, no safety or efficacy issues have been identified.

More than 900 million doses of the Moderna COVID-19 vaccine have been administered to date worldwide. Moderna does not believe that this poses a risk to other vials in the lot and does not believe that this affects the significant benefit/risk profile of the vaccine.

Given the Company’s priority to assure quality, and out of an abundance of caution, this lot is being recalled. Moderna is proactively communicating with health authorities as the investigation proceeds. Moderna and Rovi remain committed to working transparently and expeditiously with regulators to address any potential concerns.

Direct consignees or healthcare professionals that have impacted product with this lot number should contact 000190AAction@modernatx.com with any inquiries regarding recalled product.

Forward Looking Statements

This statement contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including regarding: the Company’s recall of one lot of its COVID-19 vaccine (mRNA-1273); the lack of safety concerns or reports related administration of vaccines from this lot; the risks associated with administration of the vaccine; and the steps the Company will take to coordinate with regulators. The forward-looking statements here are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Moderna's control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include those other risks and uncertainties described under the heading "Risk Factors" in Moderna's most recent Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent filings made by Moderna with the SEC, which are available on the SEC's website at www.sec.gov. Except as required by law, Moderna disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Moderna's current expectations and speak only as of the date hereof.

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Moderna’s Updated Patent Pledge

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Mon, 07 Mar 2022 19:54:40 GMT

Moderna is pioneering the development of messenger RNA (mRNA) vaccines and therapeutics. From Moderna’s inception in 2010, we saw the potential of this new class of medicines to make a significant difference in patients’ lives. Since its founding, Moderna has invested billions of dollars into research and development to make mRNA medicines a reality. When the COVID-19 pandemic struck, Moderna was an R&D-focused company with many medicines in various stages of preclinical and clinical development. Based on our experience, we believed our mRNA technology could make a difference in combatting the pandemic. Since 2020, we have mobilized our existing mRNA platform to develop, test and manufacture more than 825 million doses of an authorized, safe and effective COVID-19 vaccine, approximately 25% of which have been delivered to low- and middle-income countries.

As the pandemic surged in October 2020, we voluntarily committed that, “while the pandemic continues, Moderna will not enforce our COVID-19 related patents against those making vaccines intended to combat the pandemic.” At that time, as a biotech company still working to develop its first commercial products, we understood that our portfolio of intellectual property was – and still is – an important asset ¹ that allowed us to attract investment. Such private investment made our mRNA technology possible. Further, that very intellectual property and associated rights protect and enhance our ability to continue to develop innovative medicines. Nevertheless, we felt and continue to believe that we have a special obligation to remove any perceived impediments created by our intellectual property rights so that the world could be vaccinated during the pandemic. That is why we have also licensed our patents to several manufacturing partners and raised more than $1.9 billion in private capital to scale up our manufacturing capacity so that we can now make billions of doses of our vaccine each year.

To underscore our commitment to low-and middle-income countries, Moderna is now updating our patent pledge to never enforce our patents for COVID-19 vaccines against companies manufacturing in or for the 92 low- and middle-income countries in the Gavi COVAX Advance Market Commitment (AMC), provided that the manufactured vaccines are solely for use in the AMC 92 countries.

This commitment builds on Moderna’s efforts to ensure equitable access across the world, including through our agreement with COVAX to provide up to 650 million doses of our vaccine through 2022 to low- and middle-income countries at our lowest-tiered price, with tens of millions of additional doses committed directly to the African Union. Moderna has also announced plans to establish a state-of-the-art mRNA manufacturing facility in Kenya, investing up to $500 million to produce up to 500 million doses each year for the African continent.

In non-AMC 92 countries, vaccine supply is no longer a barrier to access. In these countries, the Company expects those using Moderna-patented technologies will respect the Company’s intellectual property. Moderna remains willing to license its technology for COVID-19 vaccines to manufacturers in these countries on commercially reasonable terms. Doing so enables Moderna to continue to invest in research to develop new vaccines, prepare for the next pandemic, and meet other pressing areas of unmet medical need.

Moderna is proud of what our team of more than 3,000 employees has accomplished since the pandemic began, and every single one of us is dedicated to increasing supply of our vaccine and combatting COVID-19 globally.

¹ A summary of our intellectual property can be found here . A selection of representative issued U.S. patents relevant to our mRNA-1273 vaccine against COVID-19 is available here .

Forward-Looking Statements

This statement contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including regarding: the Company’s development of its COVID-19 vaccine; the total manufacturing capacity for the Company’s COVID-19 vaccine; the Company’s commitment to not enforce its intellectual property rights against companies in the Gavi COVAX Advance Market Committement countries manufacturing COVID-19 vaccines for use in those countries; agreements to provide COVID-19 vaccines to COVAX and the African Union; the Company’s plans to invest in manufacturing capacity in Kenya and the scale of production anticipated from that facility; and the Company’s approach to intellectual property enforcement outside the GAVI COVAX AMC 92 countries. The forward-looking statements here are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Moderna's control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include those other risks and uncertainties described under the heading "Risk Factors" in Moderna's most recent Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent filings made by Moderna with the SEC, which are available on the SEC's website at www.sec.gov. Except as required by law, Moderna disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Moderna's current expectations and speak only as of the date hereof.

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Statement on COVID-19 Vaccine Clinical Studies in Children & Adolescents

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Wed, 12 Jan 2022 19:59:31 GMT

The U.S. FDA requested additional participants at a given dose level in pediatric cohorts after the VRBPAC review of requirements for pediatric EUA on June 10, 2021, and Moderna complied at that time. Outside the United States, Moderna has received regulatory authorizations in Europe, UK, Australia, and Canada for adolescents 12-17 years of age, and has submitted applications for 6-11 years of age.

In early December, Moderna also decided to evaluate the potential of lower doses to meet regulatory guidance for immunogenicity in children 6-11 years of age and in adolescents 12-17 years of age in our ongoing clinical trials. The Company is also evaluating a booster dose in adolescents 12-17 years of age. The Company is in the process of implementing those various protocol amendments.

We expect to report data in children 2-5 years of age in March. If the data is supportive and subject to regulatory consultation, Moderna may proceed with regulatory filings for children 2-5 years of age thereafter.

This post contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including regarding: the Company’s development of a vaccine against COVID-19 (mRNA-1273); the Company’s evaluation of different doses for mRNA-1273 in adolescent and pediatric populations; the evaluation of booster doses of mRNA-1273 in adolescents; and the timing for anticipated results in pediatric trials for mRNA-1273 and subsequent regulatory filings. The forwardlooking statements in this press release are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Moderna’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include those other risks and uncertainties described under the heading “Risk Factors” in Moderna’s most recent Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent filings made by Moderna with the SEC, which are available on the SEC’s website at www.sec.gov. Except as required by law, Moderna disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this post in the event of new information, future developments or otherwise. These forward-looking statements are based on Moderna’s current expectations and speak only as of the date hereof.

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Statement on Intellectual Property

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Fri, 17 Dec 2021 22:13:03 GMT

Moderna has decided at this time not to pursue issuance of the U.S. patent application for the mRNA sequence of the Moderna COVID-19 vaccine that had been allowed by the U.S. Patent and Trademark Office earlier this year. Moderna informed the National Institutes of Health (NIH) on December 10 th . The Company has filed a continuation application, which could enable some or all of these claims to later issue.

Moderna has taken this action to allow more time for discussions with the NIH. Moderna believes that its scientists invented the specific mRNA sequence at the heart of the patent in question. The Company acknowledges that NIH feels equally strongly that its scientists should be listed as co-inventors for their contemporaneous work on the protein sequence.

Moderna decided that issuance of the mRNA sequence patent in the current environment could interfere with further discussions aimed at an amicable resolution with the NIH. Further, the Company would like to avoid any distraction to the important public-private efforts ongoing to address emerging SARS-CoV-2 variants, including Omicron. Moderna continues to be grateful to the NIH and its scientists for their substantial contributions to developing the Moderna COVID-19 vaccine.

This decision has no impact on Moderna’s other intellectual property, including its pending and granted COVID-19 vaccine patents around the world.

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Our Global Commitment to Vaccine Access

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Fri, 08 Oct 2021 22:28:38 GMT

Letter from Stéphane Bancel

When the COVID-19 pandemic struck, Moderna was an R&D-focused company that had yet to commercialize a product. But we believed our messenger RNA (mRNA) technology could make a difference, and within a very short time, we were able to advance our technology from the realm of scientific possibility, to authorized product and commercial production at unprecedented speed and scale. I want to thank our employees, our partners and the participants in our clinical trials who have been the heroes of our fight to end this pandemic.

From the beginning our goal has been to help protect as many people as possible around the globe. To date, more than 250 million people have been vaccinated globally with the Moderna COVID-19 vaccine. However, we recognize that access to vaccines continues to be a challenge in many parts of the world. So, we remain focused on implementing a comprehensive and always evolving strategy to ensure that low-income countries get access to our vaccine. Our strategy now has five pillars:

First, to support global access of vaccines, in October 2020 and before we had our Phase 3 data, we announced that we would not enforce our COVID-19 related patents during the pandemic. As a small company, we are still limited in our capacity to help and focused on scaling our manufacturing, but we never wanted our patents to be a barrier to others bringing forward mRNA vaccines.

Second, we committed to support the COVAX Facility for low-income countries, announcing an agreement in May 2021 to supply up to 500 million doses of our COVID-19 vaccine from the fourth quarter of 2021 through 2022.

Third, we have worked closely with the U.S. Government and others that have purchased more vaccine than they need today to facilitate donation through COVAX or bilaterally, providing the regulatory, supply chain and pharmacovigilance support necessary to allow more than 50 million doses of our vaccine to be distributed by COVAX through September 2021.

Fourth, yesterday, we announced our plan to build a state-of-the-art mRNA facility in Africa with the goal of producing up to 500 million doses of vaccines each year. While this will take longer to deliver, we believe we must start to extend Moderna’s impact in Africa by equipping the continent with our technology.

Fifth, we are currently investing to expand our capacity to deliver another 1 billion doses to low-income countries in 2022, out of a total targeted capacity of between 2 billion to 3 billion doses for 2022. Given that fewer than 1 billion people live in OECD countries, this was done solely to provide more doses to low-income countries.

A year ago, we had the ambitious goal of producing up to 1 billion doses at our own facility, supplemented by partnerships. We had a lot to prove to ourselves and others, and few might have predicted how far we have come today. But we recognize that our work is not done. We are committed to doubling our manufacturing and expanding supply even further until our vaccine is no longer needed in low-income countries. Our responsibility during the pandemic is to push ourselves to our limits to expand availability of our vaccine, and to make investments in the future to prevent the limitations we face today from such a pandemic ever happening again.

We believe our strategy is comprehensive, but if more is needed and we are unable to deliver more without undermining our current commitments, we will continue to add pillars to our strategy until this pandemic is over for all.

Sincerely,

Stéphane Bancel
Chief Executive Officer
Moderna Inc.

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Statement on Myocarditis and Pericarditis

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Thu, 07 Oct 2021 22:16:34 GMT

Moderna is aware of the decision of Health Authorities in Sweden and Finland to pause the vaccination of individuals under the age of 30.

At Moderna, the safety of the people who use our products is paramount. We are committed to helping to end the ongoing COVID-19 pandemic, that to date has affected millions of people around the world, and claimed the lives of over 4 million of them.

Moderna is aware of the very rare occurrence of myocarditis and/or pericarditis following administration of mRNA vaccines against COVID-19. These are typically mild cases and individuals tend to recover within a short time following standard treatment and rest. The risk of myocarditis is substantially increased for those who contract COVID-19, and vaccination is the best way to protect against this. A recent large analysis of 6.2 million people from the US Vaccine Safety Datalink (VSD) following mRNA COVID-19 vaccination did not identify an increased safety signal for either mRNA vaccine. Similarly, a study of over 2.3 million individuals conducted in the Kaiser Permanente network identified 15 cases of myocarditis in individuals receiving a mRNA vaccine, for an observed incidence of 5.8 cases per million people following the second dose of vaccine.

The Moderna COVID-19 Vaccine (Spikevax) has demonstrated substantial benefit. The protection afforded by the Moderna COVID-19 vaccine is highly robust and has been confirmed in the real-world setting as well as large, randomized trials including the Phase 3 COVE study. We continue to closely monitor the safety of all our products, both in clinical practice and in clinical trials.

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Joint Statement from Moderna and Takeda

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Fri, 01 Oct 2021 22:22:24 GMT

STATEMENT REGARDING Moderna COVID-19 Vaccine Recall Investigation Report – October 2021

Takeda and Moderna today published a report of the investigation prompted by the observation of foreign particles in unpunctured vials from a single lot of Moderna’s COVID-19 vaccine distributed in Japan by Takeda. The lot was suspended on August 26, 2021, JST and voluntarily recalled on September 2, 2021, JST. Two other lots manufactured in the same series were included in the suspension and voluntary recall as a precautionary measure.

The investigation report is intended to provide a clear view into what happened, when, and how it has been addressed. Specifically, it provides details on the particle analysis, root cause analysis and health risk assessment conducted by Moderna (the vaccine developer and manufacturer), ROVI Pharma Industrial Services, S.A. (Moderna’s European contract manufacturing organization), and Takeda (the Japan Marketing Authorization Holder and authorized distributor). In addition, the report provides information based on a draft for-cause audit of ROVI, conducted on-site jointly by Moderna and Takeda.

In summary, the root cause analysis, particle analysis and health risk assessment established the following:

The rare presence of 316L stainless steel particles – observed in one of the recalled lots – presented no undue risk to patient safety and did not adversely affect the benefit/risk profile of the product.
The most probable cause of the particles identified in one of the recalled lots is related to friction between two pieces of metal installed in the stoppering module of the production line.
This was the result of incorrect assembly and was due to human error specific to visually misjudging the required 1mm gap between the star-wheel and the stopper.

The investigation conclusion has confirmed the scope of the event, and corrective actions—including improvements to standard operating procedures at the changeover, and the utilization of a new precision tool—will help mitigate the risk of this issue reoccurring. These actions will be directly overseen and confirmed by Moderna, in collaboration with Takeda. A list of corrective and preventive actions following the root cause analysis and for-cause audit are provided in the investigation report.

The Moderna COVID-19 vaccine has a well-established safety and efficacy profile. To date, more than 200 million doses of the Moderna COVID-19 vaccine have been administered to more than 110 million individuals in 45 countries, representing a critical component of the global fight against COVID-19. Takeda and Moderna’s top priority is to continue to support the MHLW’s efforts to help fight this ongoing pandemic and bring this vaccine to everyone who can benefit from it.

The investigation report can be read here .

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Joint Statement from Moderna and Takeda on the Investigation of Suspended Lots of Moderna’s COVID-19 Vaccine in Japan

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Wed, 01 Sep 2021 22:04:53 GMT

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Joint Statement from Moderna and Takeda

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Sat, 28 Aug 2021 21:28:30 GMT

Takeda and Moderna were notified on August 28 JST, of the deaths of two individuals who both recently received the Moderna COVID-19 Vaccine for Intramuscular Injection in Japan. This is a tragic event, and the loss of life is something that we take very seriously. We offer our sincerest condolences to their loved ones.

At this time, we do not have any evidence that these deaths are caused by the Moderna COVID-19 vaccine, and it is important to conduct a formal investigation to determine whether there is any connection.

Takeda, the authorized distributor, and Moderna, the manufacturer, are working with the Ministry of Health, Labour and Welfare (MHLW) to investigate the two deaths. The investigation is being conducted with the greatest sense of urgency, transparency and integrity and is of the highest priority. Takeda and Moderna will keep the public informed as we learn more.

On August 26 JST, Takeda announced the decision to suspend the use of three lots of Moderna’s COVID-19 Vaccine in Japan in alignment with MHLW following reports from vaccination sites of a foreign substance found in vials. The complaints that prompted the suspension were isolated to one specific lot, but a total of three lots manufactured in the same series were included in the suspension by MHLW out of an abundance of caution. To date, we have no product quality complaints related to particulate matter in the lot related to the unfortunate passing of the two individuals.

Takeda has requested that Moderna and Moderna’s European contract manufacturing organization, urgently conduct a thorough investigation to determine the nature of the foreign substance. We are aware of unofficial reports that have provided initial indication of the type of particle matter in the vials. These reports are inconclusive and it is important to rely on a formal investigation before determining the precise nature of the particle. Moderna is in the process of conducting that investigation and the vials have been sent to a qualified lab for analysis and initial findings will be available early next week.

To date, more than 200 million doses of the Moderna COVID-19 vaccine have been administered to more than 110 million individuals in 45 countries, representing a critical component of our global fight against COVID-19.

For additional updates and resources about the COVID-19 vaccine program in Japan please go to the official COVID-19 information center .

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COVID-19 Vaccination Requirement for All Workers in the U.S.

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Fri, 20 Aug 2021 20:40:08 GMT

Moderna will require COVID-19 vaccination for all workers in the United States, effective October 1, 2021. Requests for medical or religious exemptions will be considered on an individual basis. All Moderna workers are still required to follow local health authority and Moderna health and safety protocols when on-site.

In alignment with public health strategies designed to slow the spread of COVID-19, the Company previously implemented heightened guidelines, including mask wearing, as well as COVID-19 testing to help ensure the safety of its workers and business continuation. The Company also made its COVID-19 vaccine available to its workers in the U.S. under Emergency Use Authorization starting in December 2020.

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Statement on CVST or Thrombotic Events

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Tue, 13 Apr 2021 20:17:11 GMT

A comprehensive assessment of the totality of the available safety data for mRNA-1273 after over 64.5 million doses administered globally does not suggest an association with cerebral venous sinus thrombosis (CVST) or thrombotic events.

Analyses performed using data through March 22, 2021. Number of vaccinations was derived from the U.S. Centers for Disease Control and Prevention (CDC) website, European Centre for Disease Prevention and Control (ECDC) website, and inferred for other countries based on distribution and the proportion of doses distributed administered in EX-U.S. settings.

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Statement on California Department of Public Health (CDPH) Report

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Wed, 20 Jan 2021 20:03:35 GMT

Update January 20, 2021: The California Department of Public Health (CDPH) announced that it is advising providers that they can immediately resume the administration of lot 041L20A of the Moderna COVID-19 Vaccine, following an in-depth review of possible allergic reactions reported by some recipients. According to California State Epidemiologist Dr. Erica Pan in a statement issued by CDPH, “We convened the Western States Scientific Safety Review Workgroup and additional allergy and immunology specialists to examine the evidence collected. We had further discussions with the County of San Diego Department of Public Health, the FDA, CDC and manufacturer, and found no scientific basis to continue the pause. Providers that paused vaccine administration from Moderna Lot 41L20A can immediately resume. These findings should continue to give Californians confidence that vaccines are safe and effective, and that the systems put in place to ensure vaccine safety are rigorous and science-based. Members of my family who have qualified to receive the vaccine as health care workers or because of their age have already received the COVID-19 vaccine, and I encourage every Californian to get the vaccine when it's their turn."

January 19, 2021: Moderna acknowledges receiving a report from the California Department of Public Health (CDPH) that several individuals at one vaccination center in San Diego were treated for possible allergic reactions after vaccination from one lot of Moderna’s COVID-19 Vaccine. The Company is fully cooperating with CDPH in investigating these reported adverse events. Consistent with the statement from CDPH, at this point Moderna is unaware of comparable clusters of adverse events from other vaccination centers which may have administered vaccines from the same lot, or from other Moderna lots.

Moderna confirmed that a total of 1,272,200 doses were produced in batch number 041L20A, with nearly a million doses (964,900) already distributed to approximately 1,700 vaccination sites in 37 states. According to CDPH, that includes more than 330,000 doses from this lot distributed to 287 providers across the state of California. A total of 307,300 doses remain in storage and not yet distributed.

While Moderna said it does not know how many doses may have ended up in arms of people, it did report that the lot was shipped between January 4th and January 8th, and thus it expects that a significant portion of the distributed doses have been already used. This investigation is still ongoing and Moderna is working closely with FDA and CDC to understand the clinical cases and whether the broad pause in use of the lot is warranted.

Moderna said a detailed review of the manufacturing intermediates and final drug product was performed including raw materials, batch records, release and characterization testing results, and Moderna shipping and storage records. The review confirmed that all criteria for product release of lot 041L20A were met.

Forward-Looking Statements

This statement contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding: potential patient reactions to the Moderna COVID-19 Vaccine, the underlying cause of those reactions, and the process for assessing those reactions with the CDPH, FDA and CDC. In some cases, forward-looking statements can be identified by terminology such as “will,” “may,” “should,” “could”, “expects,” “intends,” “plans,” “aims,” “anticipates,” “believes,” “estimates,” “predicts,” “potential,” “continue,” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. The forward-looking statements in this statement are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include those risks and uncertainties described under the heading “Risk Factors” in Moderna’s most recent Quarterly Report on Form 10-Q filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent filings made by Moderna with the SEC, which are available on the SEC’s website at www.sec.gov. Except as required by law, Moderna disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this statement in the event of new information, future developments or otherwise. These forward-looking statements are based on Moderna’s current expectations and speak only as of the date hereof.

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Moderna’s Statement on Phase 3 Study of COVID-19 Vaccine Protocol Update

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Tue, 29 Dec 2020 22:58:27 GMT

Moderna has posted an updated protocol for its Phase 3 study of the Moderna COVID-19 Vaccine. The protocol reflects inclusion of Amendment 6 from Dec. 23, 2020. The purpose of this amendment is to inform all ongoing study participants of the availability of and eligibility criteria of any COVID-19 vaccine made available under an Emergency Use Authorization (EUA) and to offer participants who originally received placebo in this study the potential benefit of vaccination against COVID-19, given that the primary efficacy endpoint for the Moderna COVID-19 Vaccine was met per the protocol-defined interim analysis. The protocol is available here .

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Statement on Moderna Plan for Worker Vaccination

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Tue, 29 Dec 2020 22:56:12 GMT

To provide an additional layer of COVID-19 protection as Moderna workers conduct essential services in developing, manufacturing and delivering a COVID-19 vaccine, Moderna is making its vaccine available to its workers, contractors and board members in the U.S. under Emergency Use Authorization. The program will extend to adult household members of our team to reduce the risk of absenteeism and disruption due to a COVID-19 infection in an adult household member. Participation in the program is confidential and entirely voluntary.

All costs for the program are being paid for by Moderna, including the supply of vaccine and its administration. The very small quantities of vaccine to be used are separate and distinct from those committed by Moderna to the U.S. Government.

For transparency, the Company notified appropriate health authorities about the program in advance and is participating in all relevant monitoring and reporting obligations under the Emergency Use Authorization.

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Moderna’s Commitment to Vaccines and Therapeutics Access

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Wed, 23 Dec 2020 22:52:14 GMT

Moderna is committed to working on multiple levels to optimize the impact of mRNA vaccines and therapeutics. Our philosophy on pricing and access reflects a few basic principles:

Moderna is committed to developing a broad portfolio of vaccines and therapeutic solutions to address epidemiological challenges worldwide.
Moderna will invest in R&D in areas of unmet need.
Moderna will work to include communities that have historically been under-represented in clinical research in our development programs, as well as those that are disproportionately impacted by the respective diseases.
Moderna aims to provide effective and affordable vaccines and therapeutics to all populations.
Moderna will price its products through differential pricing frameworks.
Moderna is committed to participating in key public-private partnerships such as Gavi, the Vaccine Alliance.
Gavi-eligible countries will get Moderna’s lowest prices, and Moderna commits to an annual independent third-party audit on this commitment.

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Statement on Variants of the SARS-CoV-2 Virus

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Wed, 23 Dec 2020 22:48:44 GMT

The Moderna COVID-19 Vaccine expresses the full-length Spike protein of the SARS-COV-2 virus, allowing for the generation of neutralizing antibody responses to multiple domains of the protein. The full-length Spike protein is 1,273 amino acids long, so while recent variants involve multiple mutations, for instance up to 8 amino acid changes in the spike protein of the B1.1.7 strain, these represent less than a 1% difference from the spike protein encoded by Moderna’s vaccine.

While we plan to run tests to confirm the activity of the vaccine against any strain, the broad range of potential neutralizing antibodies made possible by the Moderna COVID-19 Vaccine provide confidence that our vaccine will also be effective at inducing neutralizing antibodies against them.

We have already tested sera from animals and humans vaccinated with the Moderna COVID-19 Vaccine against a number of previous variants of the SARS-CoV-2 virus that have emerged since the first outbreak of the pandemic and found our vaccine to remain equally effective.

Based on the data to date, Moderna expects that the vaccine-induced immunity from the Moderna COVID-19 Vaccine would be protective against the variants of the SARS-CoV-2 virus recently described in the UK. We will be performing additional tests of the vaccine in the coming weeks to confirm this expectation.

We continue to test the Moderna COVID-19 Vaccine against new SARS-CoV-2 variants, and our expectation is that the vaccine’s effectiveness will hold against them as well.

Forward-Looking Statements

This statement contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding: Moderna’s expectations regarding the ability of the Moderna COVID-19 Vaccine to protect and provide immunity against variant strains of the SARS-CoV-2 virus, and Moderna’s ongoing test of the effectiveness of the Moderna COVID-19 Vaccine against these strains. In some cases, forward-looking statements can be identified by terminology such as “will,” “may,” “should,” “could”, “expects,” “intends,” “plans,” “aims,” “anticipates,” “believes,” “estimates,” “predicts,” “potential,” “continue,” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. The forward-looking statements in this statement are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include those risks and uncertainties described under the heading “Risk Factors” in Moderna’s most recent Quarterly Report on Form 10-Q filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent filings made by Moderna with the SEC, which are available on the SEC’s website at www.sec.gov. Except as required by law, Moderna disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this statement in the event of new information, future developments or otherwise. These forward-looking statements are based on Moderna’s current expectations and speak only as of the date hereof.

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Statement on Cyberattack on the European Medicines Agency

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Mon, 14 Dec 2020 22:45:13 GMT

Today, Moderna was informed by the European Medicines Agency (EMA) that in connection with a recent compromise of EMA computer systems, certain documents exchanged in the context of the pre-submission discussions related to Moderna’s COVID-19 vaccine candidate, mRNA-1273, were unlawfully accessed. Moderna’s submission to the EMA did not include any information identifying individual study participants and, thus, there is no information at this time that any study participants have been identified in any way. Moderna has not identified any indications of compromise of its own data or systems in conjunction with this incident. We await further information from EMA’s continuing investigation as Moderna continues to focus on bringing an effective and safe COVID-19 vaccine to the public.

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Statement by Moderna on Intellectual Property Matters during the COVID-19 Pandemic

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Thu, 08 Oct 2020 22:40:41 GMT

Moderna is a pioneer in the development of messenger RNA (mRNA) vaccines and therapeutics. From its inception in 2010, Moderna saw the potential of this new class of medicines to make a significant difference in patients’ lives. With the support of our investors we have invested billions of dollars into research and development to make mRNA medicines a reality. One of the exciting discoveries advanced by Moderna was the combination of mRNA and lipid nanoparticles (LNPs) to make vaccines, and the demonstration of this potential in human clinical trials for eleven different infectious disease vaccines since 2015. Those discoveries and the expertise we developed have uniquely positioned Moderna to respond to the COVID-19 pandemic quickly. Information on our work toward a COVID-19 vaccine can be found here .

As a company committed to innovation, Moderna recognizes that intellectual property rights play an important role in encouraging investment in research. Our portfolio of intellectual property is an important asset that will protect and enhance our ability to continue to invest in innovative medicines. A summary of our intellectual property can be found here. A selection of representative issued US patents relevant to our mRNA-1273 vaccine against COVID-19 is available here.

Beyond Moderna’s vaccine, there are other COVID-19 vaccines in development that may use Moderna-patented technologies. We feel a special obligation under the current circumstances to use our resources to bring this pandemic to an end as quickly as possible. Accordingly, while the pandemic continues, Moderna will not enforce our COVID-19 related patents against those making vaccines intended to combat the pandemic. Further, to eliminate any perceived IP barriers to vaccine development during the pandemic period, upon request we are also willing to license our intellectual property for COVID-19 vaccines to others for the post pandemic period.

Moderna is proud that its mRNA technology is poised to be used to help end the current pandemic.

Forward-Looking Statements

This statement contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including regarding Moderna's stance with respect to enforcement and licensing of its intellectual property rights during and following the COVID-19 pandemic. In some cases, forward-looking statements can be identified by terminology such as “will,” “may,” “should,” “could”, “expects,” “intends,” “plans,” “aims,” “anticipates,” “believes,” “estimates,” “predicts,” “potential,” “continue,” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. The forward-looking statements in this statement are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include those risks and uncertainties described under the heading “Risk Factors” in Moderna’s most recent Quarterly Report on Form 10-Q filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent filings made by Moderna with the SEC, which are available on the SEC’s website at www.sec.gov. Except as required by law, Moderna disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this statement in the event of new information, future developments or otherwise. These forward-looking statements are based on Moderna’s current expectations and speak only as of the date hereof.

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Statement from Moderna on Patent Trial and Appeal Board (PTAB) Ruling

brian.balbirnie@issuerdirect.com (Brian Balbirnie) — Fri, 24 Jul 2020 22:35:10 GMT

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jul. 24, 2020-- Moderna, Inc. (Nasdaq: MRNA), a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, today released a statement on the July 23 rd U.S. Patent Trial and Appeal Board (PTAB) ruling:

The recently issued Patent Trial and Appeal Board ruling on the 8,058,069 patent relates to Moderna’s challenge to certain legacy patents held by Arbutus, commenced well before the development of mRNA-1273. These actions were taken by Moderna in response to the longstanding aggressive posture taken by Arbutus and its predecessor company against many developers of nucleic acid-based therapeutics. Through its actions, Moderna successfully overturned one legacy patent held by Arbutus and invalidated the broadest claims of a second one. Moderna’s continued development of its proprietary LNP formulation technology and manufacturing processes have advanced well beyond the technology described in these legacy Arbutus patents. Our improved proprietary LNP formula, used to manufacture mRNA-1273, is not covered by the Arbutus patents. Moderna is not aware of any significant intellectual property impediments for any products we intend to commercialize, including mRNA-1273.

Forward Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including regarding Moderna’s proprietary LNP formulation technology and manufacturing processes and Moderna’s awareness of any significant intellectual property impediments for any products it intends to commercialize. In some cases, forward-looking statements can be identified by terminology such as “will,” “may,” “should,” “could”, “expects,” “intends,” “plans,” “aims,” “anticipates,” “believes,” “estimates,” “predicts,” “potential,” “continue,” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. The forward-looking statements in this press release are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Moderna’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include, among others: the fact that there has never been a commercial product utilizing mRNA technology approved for use; the fact that the rapid response technology in use by Moderna is still being developed and implemented; the fact that the safety and efficacy of mRNA-1273 has not yet been established; potential adverse impacts due to the global COVID-19 pandemic such as delays in regulatory review, manufacturing and clinical trials, supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy; and those other risks and uncertainties described under the heading “Risk Factors” in Moderna’s most recent Quarterly Report on Form 10-Q filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent filings made by Moderna with the SEC, which are available on the SEC’s website at www.sec.gov. Except as required by law, Moderna disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Moderna’s current expectations and speak only as of the date hereof.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200724005460/en/

Moderna Contacts

Media:
Colleen Hussey
Senior Manager, Corporate Communications
203-470-5620
Colleen.Hussey@modernatx.com

Investors:
Lavina Talukdar
Head of Investor Relations
617-209-5834
Lavina.Talukdar@modernatx.com

Source: Moderna, Inc.

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Moderna Named A Top Employer By Science For The Fourth Consecutive Year

Thu, 25 Oct 2018 21:50:38 GMT

CAMBRIDGE, Mass., October 25, 2018 — Moderna, Inc., a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, today announced it has been named one of the global biopharmaceutical industry’s top employers in Science and Science Careers’ 2018 Top Employers Survey. This is the fourth consecutive year Moderna has been selected and this year the company was ranked 4th. The annual employee survey evaluates companies in biotechnology, biopharmaceutical, pharmaceutical and other related industries in categories such as leadership in innovation, alignment between work culture and values and commitment to social responsibility.

“It is an honor to be recognized as a top employer by Science and to be included amongst this group of industry leaders,” said Stéphane Bancel, CEO at Moderna. “As a mission-driven company, we work to attract exceptional team members who are passionate about delivering on the potential of our mRNA science for patients. Our R&D leadership has built an organization where professional and growth opportunities are matched by a commitment to continuous innovation and scientific excellence, and have fostered our company’s culture of respect for the individual, community and citizenship, and where employee feedback is a core component of decision making.”

Moderna earlier this month introduced a range of enhanced benefits, including 16 weeks of paid parental leave and 16 weeks of caregiver leave, and a sabbatical program that offers four weeks of paid time off after five years of full-time employment with the company. These programs complement Moderna’s existing benefits which include extensive healthcare coverage, paid time off for volunteering and subsidized childcare.

The Science and Science Careers’ 2018 survey findings are based on 8,015 completed surveys from readers of Science and other survey invitees. Survey respondents came from North America (63%), Europe (24%), and Asia/Pacific Rim (9%); 93% work in biotechnology, biopharmaceutical, and pharmaceutical companies. The complete feature can be found online on the ScienceCareers.org website and in the October 26, 2018 print issue of Science.

About Moderna

Moderna pioneers the discovery and development of messenger RNA (mRNA) therapeutics and vaccines, an entirely new class of medicines that directs the body’s cells to produce intracellular or secreted proteins that can have a therapeutic or preventive benefit for both patients and healthy individuals. With its platform, Moderna is creating mRNA medicines for a wide range of diseases and conditions, in many cases by addressing currently undruggable targets or underserved areas of medical need. Moderna is developing its innovative mRNA medicines for infectious diseases, immuno-oncology, rare diseases and cardiovascular diseases, through solely controlled programs and collaborations with strategic partners.

Headquartered in Cambridge, Mass., privately held Moderna currently has strategic relationships with AstraZeneca, Plc. (AZ), Merck, Inc (MRK) and Vertex Pharmaceuticals (VRTX), as well as the Defense Advanced Research Projects Agency (DARPA), an agency of the U.S. Department of Defense; the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS); and the Bill & Melinda Gates Foundation. Moderna has been ranked a top biopharma industry employer by Science for the past four years, and in 2017 was selected as a “Top Place to Work” by the Boston Globe. To learn more, visit www.modernatx.com .

Contacts

Moderna

Investors:
Lorence Kim
617-209-5849
lorence.kim@modernatx.com

Media:
Jason Glashow
617-674-5648
jason.glashow@modernatx.com

VIEW ALL NEWS

Newly Published Data in Nature Medicine Show Potential of mRNA to Produce Therapeutic and Protective Protein Levels in Pre-Clinical Models of Acute Intermittent Porphyria

Mon, 08 Oct 2018 22:07:14 GMT

Research Demonstrates Effective Restoration of Defective Liver Enzyme

CAMBRIDGE, Mass., October 8, 2018 — Moderna, Inc., a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, today announced new pre-clinical research published in the journal Nature Medicine that further demonstrates the utility of its mRNA platform to express therapeutic levels of protein in liver tissue to potentially treat patients with rare metabolic disorders.

The paper, published in collaboration with researchers from the Center for Applied Medical Research (CIMA) at the University of Navarra, Pamplona, Spain, reported the therapeutic potential of mRNA in reconstituting a functional enzyme in pre-clinical models of acute intermittent porphyria (AIP). AIP is an inherited, rare metabolic disorder that is caused by a deficiency in the body’s ability to metabolize porphyrin precursors. People with the disease typically experience discrete episodes or “attacks” that can be life-threatening, with nausea and severe gastrointestinal and/or neuropathic pain.

“In these pre-clinical models, we’ve shown that mRNA has the potential to restore the enzymatic deficiency inside liver cells responsible for AIP and normalize markers of the disease during attacks,” said Paolo Martini, Ph.D., Chief Scientific Officer, Rare Diseases. “These data further support our ongoing efforts in rare metabolic diseases where mRNA can allow a particular tissue to produce therapeutic levels of a functional protein.”

In the study, a single administration of mRNA encoding for human porphobilinogen deaminase (hPBGD), delivered in proprietary lipid nanoparticles, led to expression of hPBGD protein in mouse liver tissue in as little as two hours, with maintained activity throughout the entire duration of an induced attack. PBGD activity and porphyrin precursor levels were also shown to normalize. Protection against key hallmarks of the disease, including mitochondrial dysfunction, hypertension, pain and motor impairment, were also observed in mice that received hPBGD mRNA. The research also showed safety and sustained levels of PBGD expression after repeat dosing of hPBGD mRNA in non-human primates.

“Today’s treatments for AIP can help manage patient symptoms, but there remains a real need to address the underlying cause of the disease,” said Antonio Fontanellas, Ph.D., a porphyria researcher in the hepatology program at CIMA and a senior author on the paper. “This pre-clinical research on an mRNA approach for the treatment of AIP is encouraging because in the models it suggests that a single dose can quickly restore enzyme PBGD activity for the duration of a typical attack and that dosing can be repeated to prevent new crises.”

About CIMA

The Center for Applied Medical Research (CIMA) is the University of Navarra's biomedical research institute. Its mission is to carry out translational research to a high standard of excellence, based on novel biological knowledge and aimed at finding therapeutic solutions to patients' needs.

CIMA collaborates with other research institutions, governments, biotech and pharmaceutical companies to get the essential synergy to reach patients with new therapeutic and diagnostic solutions.

About Moderna Therapeutics

Moderna pioneers the discovery and development of messenger RNA (mRNA) therapeutics and vaccines, an entirely new class of medicines that directs the body’s cells to produce intracellular or secreted proteins that can have a therapeutic or preventive benefit for both patients and healthy individuals. With its breakthrough platform, Moderna is creating mRNA medicines for a wide range of diseases and conditions, in many cases by addressing currently undruggable targets or underserved areas of medical need. Moderna is developing its innovative mRNA medicines for infectious diseases, immuno-oncology, rare diseases, and cardiovascular diseases, through solely controlled programs and collaborations with strategic partners.

Headquartered in Cambridge, Mass., privately held Moderna currently has strategic relationships with AstraZeneca, Plc. (AZ), Merck, Inc (MRK) and Vertex Pharmaceuticals (VRTX), as well as the Defense Advanced Research Projects Agency (DARPA), an agency of the U.S. Department of Defense; the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS); and the Bill & Melinda Gates Foundation. In 2017 Moderna was ranked a top biopharma industry employer by Science Magazine and a Top Place to Work by the Boston Globe . To learn more, visit www.modernatx.com .

Contacts

Moderna

Investors:
Lorence Kim
617-209-5849
lorence.kim@modernatx.com

Media:
Jason Glashow
617-674-5648
jason.glashow@modernatx.com

VIEW ALL NEWS

Dr. Wellington Sun Joins Moderna as Head, Vaccine Strategy and Regulatory Affairs

Wed, 05 Sep 2018 21:54:26 GMT

CAMBRIDGE, Mass., September 05, 2018 — Moderna, Inc., a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, today announced that Dr. Wellington Sun will join the company as its Head, Vaccine Strategy and Regulatory Affairs, starting later this month. Dr. Sun comes to Moderna from the U.S. Food and Drug Administration (FDA), where he was Director of the Division of Vaccines and Related Product Applications at the Center for Biologics Evaluation and Research (CBER). Dr. Sun will report to Dr. Tal Zaks, M.D., Chief Medical Officer of Moderna.

“We are delighted to welcome Wellington to Moderna. As an infectious disease specialist and with extensive experience in vaccine research, regulatory affairs and clinical trial design, he is uniquely qualified to shape our development strategy as we work to advance our broad portfolio of mRNA-based development candidates,” said Dr. Zaks.

Moderna has 21 mRNA development candidates in its pipeline with 10 in clinical development. This includes nine infectious disease vaccine programs, with seven currently in Phase 1 trials.

“I look forward to bringing my clinical, scientific and regulatory experience to help realize the potential of mRNA technology in preventing intractable infectious diseases through the development of safe and effective vaccines,” said Dr. Sun. “I am excited to work with the team here to identify new development candidates while progressing the portfolio of existing clinical programs.”

During his decade-long tenure at the FDA, Dr. Sun oversaw approvals of 28 original infectious disease vaccine applications and more than 100 supplement applications for new indications. While there, he worked closely with partners including the Centers for Disease Control and Prevention (CDC), the National Institutes of Health and the Biomedical Advanced Research and Development Authority, on infectious disease vaccine issues and policies. He also worked with the World Health Organization and global regulatory agencies to address clinical trial and regulatory issues related to emerging infectious diseases such as the H1N1 pandemic and Ebola and Zika epidemics.

Dr. Sun joined the FDA from the CDC, where he led field and laboratory research on Dengue fever and West Nile virus, and was principal investigator of the first-ever controlled human infection trial in subjects previously vaccinated with tetravalent dengue vaccines. Prior to the CDC, he served for 27 years in the U.S. Army Medical Corps, working as both a clinician and then as a vaccine researcher at the Walter Reed Army Institute of Research. While there he led the Department of Defense respiratory adenovirus and flavivirus basic and clinical research programs.

Dr. Sun holds an M.D. from Uniformed Services University of the Health Sciences and a B.A. from Harvard University. He is board certified in both Internal Medicine and Infectious Disease.

About Moderna Therapeutics

Moderna pioneers the discovery and development of messenger RNA (mRNA) therapeutics and vaccines, an entirely new class of medicines that directs the body’s cells to produce intracellular or secreted proteins that can have a therapeutic or preventive benefit for both patients and healthy individuals. With its breakthrough platform, Moderna is creating mRNA medicines for a wide range of diseases and conditions, in many cases by addressing currently undruggable targets or underserved areas of medical need. Moderna is developing its innovative mRNA medicines for infectious diseases, immuno-oncology, rare diseases, and cardiovascular diseases, through solely controlled programs and collaborations with strategic partners.

Headquartered in Cambridge, Mass., privately held Moderna currently has strategic relationships with AstraZeneca, Plc. (AZ), Merck, Inc (MRK) and Vertex Pharmaceuticals (VRTX), as well as the Defense Advanced Research Projects Agency (DARPA), an agency of the U.S. Department of Defense; the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS); and the Bill & Melinda Gates Foundation. In 2017 Moderna was ranked a top biopharma industry employer by Science Magazine and a Top Place to Work by the Boston Globe . To learn more, visit www.modernatx.com .

Contacts

Moderna

Investors:
Lorence Kim
617-209-5849
lorence.kim@modernatx.com

Media:
Jason Glashow
617-674-5648
jason.glashow@modernatx.com

VIEW ALL NEWS

Moderna Opens New Manufacturing Site in Norwood, MA

Tue, 17 Jul 2018 21:47:22 GMT

200,000 Square Foot State-of-the-Art Plant Built to Support Development of mRNA Medicines

NORWOOD, Mass., July 17, 2018 — Moderna Therapeutics, a clinical-stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, today announced the opening of its state-of-the-art manufacturing facility in Norwood, Massachusetts.

The digitally-enabled and environmentally sustainable 200,000 square foot clinical development manufacturing plant was built to advance Moderna’s pipeline of mRNA-based medicines. Designed to Current Good Manufacturing Practices (cGMP) specifications, the site gives the company the capacity to develop materials for preclinical toxicology studies as well as Phase 1 and 2 clinical development programs, and to manufacture, test and run fill/finish operations for its portfolio of mRNA development candidates.

Today, Moderna has 21 programs in its mRNA pipeline, including potential treatments for different forms of cancer, rare diseases, infectious diseases and heart failure.

The new site also houses the company’s Personalized Cancer Vaccine (PCV) Unit for individualized supply batches of mRNA for potential personalized cancer vaccines. By making this direct investment in PCV next-generation manufacturing, the company can reduce the time it takes to manufacture a dedicated cancer vaccine for individual patients in clinical trials.

“The Norwood site is core to our long-term strategy, enabling us to leverage the potential of our mRNA platform, control our supply chain, and provide the necessary scale and flexibility to support the development of high-quality mRNA medicines for patients in the decades ahead,” said Stephane Bancel, CEO of Moderna. “Opening this new manufacturing facility in Massachusetts was also important as it enables close collaboration with our development and research teams in Cambridge as we advance our mRNA platform and science. We are excited to continue to work with state and local partners, leverage the remarkable innovation and talent from the Massachusetts life sciences and technology sectors, and create new biopharma manufacturing roles as we work to develop new medicines for people with serious and life-threatening diseases.”

“Our administration has reinforced our commitment to life sciences, health care and the digital health ecosystem by investing over $500 million for strategic investments to support companies in innovation and technology,” said Massachusetts Governor Charlie Baker. “Thanks to companies like Moderna, Massachusetts continues to be a global leader in the industry and we welcome their growth and investment in Norwood to support the manufacturing market here in the Commonwealth.”

Norwood Site Highlights

Workforce

More than 200 employees will work at the Norwood facility in a wide range of roles including production operations, manufacturing science and technology, quality control, engineering services, supply chain management and process development. Moderna has hired more than 150 people (full time and contractors) for Norwood-based positions since the end of 2016, and plans to add close to 50 additional manufacturing roles in 2018.

Digital Integration

The digitally-enabled site connects information systems, robotics and machinery to enable the continuous exchange of data and provide information on all manufacturing activities. Using advanced algorithms and analytics, over 7,000 events are monitored in real-time to help increase efficiency, ensure quality and maintain data integrity. Moderna has also created a paperless manufacturing environment, eliminating many manual and redundant processes.

Environmental Sustainability

As part of Moderna’s commitment to sustainability , the manufacturing facility was designed to achieve Leadership in Energy and Environmental Design (LEED) certification. Moderna is limiting energy use with advanced energy metering, LED lighting, and systems designed to reduce water usage by up to 25 percent. The site will have charging stations for nearly two dozen electric vehicles, and deploy air quality and efficient waste management systems. Next year, the Company will also add solar panels to provide renewable energy to the building.

“Our investment in Norwood brings us even closer to the industrialization of our mRNA platform,” said Juan Andres, Senior Vice President of Manufacturing for Moderna. “Norwood will allow us to own the end-to-end production of materials, run multiple programs simultaneously, and produce more than 100 cGMP batches annually in an automated and digitally-integrated environment. We believe this provides us with an important advantage as we work to develop multiple pipeline candidates for patients and to scale capacity to meet individual program demands.”

About Moderna Therapeutics

Moderna is a leader in the discovery and development of messenger RNA (mRNA) therapeutics and vaccines, an entirely new class of medicines that directs the body’s cells to produce intracellular or secreted proteins that can have a therapeutic or preventive benefit for both patients and healthy individuals. With its breakthrough platform, Moderna is creating mRNA medicines for a wide range of diseases and conditions, in many cases by addressing currently undruggable targets or underserved areas of medical need. Moderna is developing its innovative mRNA medicines for infectious diseases, immuno-oncology, rare diseases, and cardiovascular diseases, through solely controlled programs and collaborations with strategic partners.

Headquartered in Cambridge, Mass., privately held Moderna currently has strategic relationships with AstraZeneca, Plc. (AZ), Merck, Inc (MRK) and Vertex Pharmaceuticals (VRTX), as well as the Defense Advanced Research Projects Agency (DARPA), an agency of the U.S. Department of Defense; the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS); and the Bill & Melinda Gates Foundation. In 2017 Moderna was ranked a top biopharma industry employer by Science Magazine and a Top Place to Work by the Boston Globe . To learn more, visit www.modernatx.com .

Moderna Contacts

Investors:
Lorence Kim
617-209-5849
lorence.kim@modernatx.com

Media:
Jason Glashow
617-674-5648
jason.glashow@modernatx.com

VIEW ALL NEWS

Moderna Provides Mid-Year Corporate Update

Wed, 11 Jul 2018 21:40:08 GMT

CAMBRIDGE, Mass., July 11, 2018 — Moderna Therapeutics, a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create transformative medicines for patients, today provided updates on its portfolio of development candidates and progress on its 2018 priorities.

Moderna now has 21 mRNA development candidates in its pipeline with 10 in clinical development, including one program in Phase 2 and nine programs in Phase 1.

The Company remains focused on executing against its pre-clinical and clinical development pipeline; introducing new rare disease and prophylactic vaccine candidates in areas of unmet need; continuing investment in its mRNA development platform; and opening the company’s new GMP clinical development manufacturing plant.

Key updates from the first half of the year include:

A Phase 1 study of MRK-V171, an undisclosed infectious disease candidate being co-developed with Merck, met its primary and secondary objectives.
Initiation of a Phase 1 study for NCI-4650, the second personalized cancer vaccine program to enter clinical testing. The program is being sponsored by the National Cancer Institute (NCI).
Completion of IND-enabling toxicology studies for mRNA-1944, a Chikungunya antibody. Sponsored by the U.S. Defense Advanced Research Projects Agency (DARPA), Moderna plans to conduct a Phase 1 randomized, placebo-controlled, dose-ranging study in healthy adults.
A new development candidate, mRNA-3283, for Phenylketonuria (PKU).
A new development candidate, mRNA-3630, for Fabry disease.
Publication of data in Molecular Therapy , which show the first example of using lipid nanoparticles to repeat dose mRNA in non-human primates safely and at therapeutically relevant levels.
Completion of the move of nearly 200 employees to a new clinical development cGMP plant in Norwood, Mass. The 200,000-sq. ft. facility, designed to produce materials for pre-clinical, Phase 1 and Phase 2 programs, will be officially opened next week.

“The first half of this year has been about execution on our strategic priorities including progressing pre-clinical and clinical programs across our portfolio, generating new rare disease development candidates, advancing our mRNA platform technology and delivery science, and completing construction of our new manufacturing facility,” said Moderna CEO Stephane Bancel. “Our strategic partnerships continue to be productive, and we were pleased to recently add a fourth collaboration with Merck, focusing on mRNA cancer vaccines, to our ongoing joint development efforts. Lastly, we closed an important funding round earlier this year, with those proceeds enabling us to further invest in research, our platform, and the acceleration of our programs as we work to bring new medicines to patients.”

As of today, more than 700 subjects have been dosed in Moderna’s internally developed and collaboration programs. Moderna's full pipeline can be found here.

Clinical and Development Pipeline Updates

Moderna’s pipeline of development candidates are in the following modalities: prophylactic vaccines, cancer vaccines, intra-tumoral immuno-oncology, local regenerative therapeutics, and systemic therapeutics. Following is a full summary of program updates from the first half of 2018 by modality.

Prophylactic Vaccines

Moderna has nine infectious disease vaccine programs, with seven completed or in ongoing Phase 1 trials. Four of these vaccines have met their primary safety and secondary immunogenicity endpoints in Phase 1 studies. The Company is developing commercial vaccines – including increasingly complex vaccines with multiple antigens – and public health vaccines aimed at helping prevent future epidemics and pandemics.

Commercial Vaccines

MRK-V171, an undisclosed infectious disease vaccine candidate: This Phase 1 study, which was run by Moderna as part of the collaboration with Merck, achieved its primary and secondary objectives.
MRK-V213, an undisclosed infectious disease vaccine candidate: Led by Merck, MRK-V213 is an mRNA infectious disease vaccine development candidate that is being evaluated in investigational new drug (IND)-enabling toxicology studies.
Cytomegalovirus (CMV) vaccine (mRNA-1647): Moderna continues to enroll its CMV vaccine trial. The Phase 1 study is randomized, placebo-controlled, and dose-ranging with the goal of evaluating the safety and immunogenicity of mRNA-1647, a vaccine against the pentamer and gB complexes of CMV, in healthy adults. The Phase 1 program includes the parallel evaluation of mRNA-1443, a vaccine against the pp65 T-cell antigen of CMV.
Human metapneumovirus (HMPV) and human parainfluenza virus 3 (PIV3) vaccine (mRNA-1653): Moderna is now in the dose selection period of its Phase 1 clinical trial for this HMPV/PIV3 vaccine. The study is randomized, placebo-controlled, and dose-ranging with the goal of evaluating the safety and immunogenicity of mRNA-1653 in healthy adults.
Recent Publications:
Vaccine : Multi-antigenic, pentamer-based, human cytomegalovirus mRNA vaccines that elicit potent humoral and cell-mediated immunity. (Feb. 20, 2018).

Public Health Vaccines

H10N8 influenza vaccine (mRNA-1440) and H7N9 influenza vaccine (mRNA-1851): Moderna has completed a Phase 1 study of mRNA-1440 in human subjects and is also now completing a Phase 1 study of mRNA-1851 against H7N9. Both programs have met their primary safety and secondary immunogenicity endpoints. Future development of both programs is expected to continue with government or other grant funding.
Chikungunya vaccine (mRNA-1388): The Phase 1 study for mRNA-1388 has completed enrollment and met its primary safety and secondary immunogenicity endpoints. Subjects in the study continue to be followed. This trial is randomized, placebo-controlled and dose ranging with the goal of evaluating the safety and immunogenicity of mRNA-1388 in healthy adults in a non-endemic region. The pre-clinical work, IND-enabling studies, and Phase 1 clinical study were supported in part by DARPA.
Zika vaccines (mRNA-1325 and mRNA-1893): Moderna is continuing efforts with the U.S. Biomedical Advanced Research and Development Authority (BARDA), within the Office of the Assistant Secretary for Preparedness and Response at the U.S. Department of Health and Human Services, to develop a Zika vaccine. Its first candidate, mRNA-1325, has completed enrollment in a Phase 1 study to evaluate safety and immunogenicity in healthy adults. While the vaccine was well tolerated, the immunogenicity endpoint at doses up to 100µg has not been met. Despite a safety profile that could have warranted additional dose escalation, current development efforts are focused on a next-generation vaccine, mRNA-1893, that contains an engineered sequence shown to be 20 times more potent in non-human primate Zika challenge studies.

Cancer Vaccines

Moderna is collaborating with Merck on two cancer vaccine programs, one of which is in Phase 1 while an IND was authorized by the U.S. Food and Drug Administration (FDA) for the second. In addition, Moderna is also supplying an mRNA personalized cancer vaccine for an NCI investigator-initiated Phase 1 trial. This modality leverages the potential of cells to use mRNA for making antigens from mutated proteins that are specific to tumors that are presented and recognized by T cells, thus immunizing or vaccinating a patient against their cancer.

Personalized cancer vaccine (mRNA-4157): Led by Moderna as part of the collaboration with Merck, the Phase 1 study is currently in the dose-escalation phase of the protocol, enrolling patients with resected solid tumors in the adjuvant setting as a monotherapy and patients with locally advanced or metastatic solid tumors that are unresectable in combination with Merck’s KEYTRUDA®.
KRAS vaccine (mRNA-5671): In May, Moderna and Merck announced the expansion of an existing collaboration to develop and commercialize novel personalized mRNA cancer vaccines. As part of that agreement, Merck will now advance mRNA-5671, an mRNA cancer vaccine for the KRAS oncogene, into human studies. Moderna has transferred the IND to Merck. The open-label, multi-center, dose-escalation and dose-expansion Phase 1 study is designed to evaluate the safety and tolerability of mRNA 5671 both as a monotherapy and in combination with KEYTRUDA.
Personalized cancer vaccine (NCI-4650): NCI-4650 uses Moderna’s personalized vaccine platform and technology to manufacture individualized vaccines against known immunogenic neoantigens that are identified by the NCI via ex vivo analysis of each patient’s tumor cells and neoantigens predicted for each patient by its bioinformatics algorithm. NCI-4650 is currently being studied in a Phase 1, investigator-initiated trial involving up to 12 patients with advanced metastatic disease. The study is being sponsored by the NCI.

Intra-Tumoral Immuno-Oncology (I/O)

Moderna currently has three intra-tumoral immuno-oncology (I/O) programs. One is in Phase 1 and two have completed IND-enabling toxicology studies and are advancing toward clinical studies. These programs are focused on driving anti-cancer T cell responses with mRNA therapies injected directly into tumors.

OX40L (mRNA-2416): Moderna’s open-label, multi-center, Phase 1 study of repeated intra-tumoral injections of mRNA-2416 in patients with advanced relapsed/refractory solid tumor malignancies and lymphomas continues to dose escalate. The primary objectives of this study include safety and tolerability of escalating doses of mRNA-2416 intratumorally and/or defining the maximum tolerated dose and a recommended dose for expansion.
OX40L, IL23, and IL36γ (mRNA-2752): This development candidate comprises three mRNAs encoding for OX40L, IL23, and IL36γ. mRNA-2752 completed GLP toxicology studies and Moderna is now preparing an IND. The Phase 1 study is designed to be an open-label, multi-center study of intra-tumoral injections of mRNA-2752 alone or in combination with checkpoint inhibitors. The primary objectives of this study will be the safety and tolerability of mRNA-2752.
IL-12 program (MEDI1191): This program, in partnership with AstraZeneca, has completed IND-enabling toxicology studies. Upon successful completion of pre-clinical development by Moderna, AstraZeneca will fund and lead the clinical development for MEDI1191, as well as lead potential regulatory filings. Moderna will be responsible for clinical supply.

Local Regenerative Therapeutics

Localized production of proteins has the potential to be used as a regenerative medicine for damaged tissues. Moderna’s most advanced program is exploring this application in cardiology in collaboration with AstraZeneca.

VEGF-A (AZD8601): AstraZeneca is continuing the Phase 2a study of AZD8601 for VEGF-A for ischemic heart disease. The primary goal of this trial is to assess the safety and tolerability of AZD8601 following epicardial injection in patients undergoing coronary artery bypass grafting (CABG) surgery with moderately impaired systolic function.
Recent Publication:
Molecular Therapy : Biocompatible, Purified VEGF-A mRNA Improves Cardiac Function after Intracardiac Injection 1 Week Post-Myocardial Infarction in Swine. (June 15, 2018)

Systemic Therapeutics

Moderna has six programs in its systemic therapeutics modality, with one having completed and a second having started IND-enabling toxicology studies. In this modality, mRNA is either delivered systemically to create proteins that are secreted outside the cell with the aim of producing pharmaceutically active therapeutic proteins with effects across the human body – or intracellularly within target organs as a therapeutic approach for diseases caused by a missing or defective protein.

Systemic Therapeutics (Secreted)

Chikungunya antibody (mRNA-1944): mRNA-1944, a program to develop a passive immunity approach to prevent Chikungunya infection, successfully completed GLP toxicology studies. Moderna is now preparing an IND. A Phase 1 randomized, placebo-controlled, dose-ranging study in healthy adults has been designed to evaluate the safety and tolerability of escalating doses of mRNA-1944. This program is sponsored by DARPA.
Relaxin (AZD7970): Partnered with AstraZeneca, AZD7970 is advancing in pre-clinical development for the treatment of heart failure. Under the collaboration, AstraZeneca will sponsor the Phase 1 trial to assess safety, tolerability, and duration of systemic exposure to the Relaxin protein.
Fabry disease (mRNA-3630): Individuals with Fabry disease have a deficiency in the α-GAL enzyme resulting in a reduced or complete inability to metabolize glycosphingolipids in lysosomes. The annual incidence is reported to be 1:80,000 live births. This new development candidate aims to instruct cells to produce α-GLA both locally in multiple affected tissues, and to secrete it into circulation from organs such as the liver for delivery to distal tissues.

Systemic Therapeutics (Intracellular)

MMA (mRNA-3704): mRNA-3704 is advancing in pre-clinical development and is now in IND-enabling toxicology studies. Moderna plans to conduct an open-label, multi-center, dose-escalating Phase 1/2 study of multiple ascending doses of mRNA-3704 in patients with MMA. In June, mRNA-3704 received Rare Pediatric Disease Designation by the FDA and earlier this year was granted Orphan Drug Designation by both the FDA and the European Medicines Agency.
Propionic Acidemia (PA) (mRNA-3927): Moderna continues to progress the preclinical development of mRNA-3927 for the treatment of patients with PA.
MMA and PA Natural History Study: Moderna recently enrolled the first patients in a global natural history study of MMA and PA (MaP Study) designed to identify and correlate clinical and biomarker endpoints for these disorders. This is a global, multi-center, non-interventional study for patients with confirmed diagnosis of MMA due to methylmalonyl-CoA mutase (MUT) deficiency or PA. Up to 60 MMA patients and up to 60 PA patients in the U.S. and Europe will be followed prospectively for 1-3 years. Retrospective data will be collected as available.
Phenylketonuria (PKU) (mRNA-3283): Individuals with PKU have a PAH protein deficiency resulting in a reduced or complete inability to metabolize the essential amino acid phenylalanine into tyrosine. The disease affects approximately 50,000 patients globally, and if untreated can lead to severe neurocognitive deficits. This new development candidate encodes an intracellular enzyme to treat PKU. Moderna plans to conduct a Phase 1/2 open-label, dose-escalation clinical trial with single ascending dose and subsequent multiple ascending dose arms.

Q4 2017 and Recent Business/Financial Updates

Continued strong cash position: Moderna maintained a strong cash position through the first half of 2018, bolstered by an aggregate $560 million new private equity “G” round announced in February. That round included both existing and new investors, including Abu Dhabi Investment Authority, BB Biotech AG, Julius Baer, EDBI and Sequoia Capital China. In May, Merck made a $125 million equity investment in Moderna in a newly priced “H” round in connection with the expansion of its mRNA cancer vaccine collaboration. As of June 30, 2018, the company had approximately $1.35 billion in cash.
Norwood Clinical Development Manufacturing: Moderna’s new facility, which is scheduled to officially open on July 17, is designed to simultaneously support multiple Phase 1 and Phase 2 clinical trials and pre-clinical programs, and to scale capacity and the capability to support new medicines enabled by Moderna’s mRNA platform.
Workforce: As of June 30, Moderna had 645 full-time employees, having started the year with 600.

Board of Directors and Management Updates

Paul Sagan : Mr. Sagan joined the company’s Board of Directors in June. He was the CEO of Akamai from 2005 – 2013 and remains a director there. Mr. Sagan is also a director of VMware, Inc. and Catalina Labs, a privately-held company. He replaces Dr. John D. Mendlein on the Board. After five years serving on Moderna’s board, Dr. Mendlein joined the Company earlier this year as President, Corporate and Product Strategy and is a member of Moderna’s Executive Committee.
Megan Pace : Ms. Pace joined Moderna in April as the company’s Chief Corporate Affairs Officer and as a member of the company’s executive committee. Prior to Moderna, she served as Senior Vice President, Strategic Operations and PKR Program Executive at Agios; Senior Vice President, Corporate Communications at Vertex; and Senior Director, Public Affairs at Genentech.
Lori Henderson : Ms. Henderson joined Moderna in April as General Counsel and Corporate Secretary, and is a member of the company’s executive committee. She joins Moderna from Albany Molecular Research (AMRI), a pharmaceutical contract research and manufacturing organization, where she was Senior Vice President, General Counsel, Corporate Secretary, and Head of Business Development. Prior to AMRI, Ms. Henderson was General Counsel, Corporate Secretary, and Chief Administrative Officer at Rand Worldwide and prior to that, General Counsel, Corporate Secretary, and Chief Administrative Officer of Moldflow Corporation.

About Moderna Therapeutics

Headquartered in Cambridge, Mass., privately held Moderna currently has strategic relationships with AstraZeneca, Plc. (AZ), Merck, Inc (MRK) and Vertex Pharmaceuticals (VRTX), as well as the Defense Advanced Research Projects Agency (DARPA), an agency of the U.S. Department of Defense; the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS); and the Bill & Melinda Gates Foundation. In 2017 Moderna was ranked a top biopharma industry employer by Science Magazine and a Top Place to Work by the Boston Globe . To learn more, visit www.modernatx.com .

Moderna Contacts

Media:
Jason Glashow
617-674-5648
jason.glashow@modernatx.com

Investors:
Lorence Kim
617-209-5849
lorence.kim@modernatx.com

VIEW ALL NEWS

Former Akamai CEO Paul Sagan Joins Moderna’s Board of Directors

Thu, 14 Jun 2018 21:29:29 GMT

CAMBRIDGE, Mass., June 14, 2018 — Moderna Therapeutics, a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, today announced that Paul Sagan has joined the company’s Board of Directors. Mr. Sagan brings a wealth of experience as an independent corporate director in addition to being the former CEO of Akamai Technologies, Inc., from 2005 - 2013, where he remains a member of the company’s board.

“We are focused on continuously strengthening our board with leaders from across industries who understand the complexity of building pioneering companies and translating breakthrough innovations into long-term benefit for individuals and society,” said Noubar Afeyan, co-Founder and Chairman of Moderna Therapeutics and Chief Executive Officer of Flagship Pioneering. “Paul is that kind of visionary leader and I am excited he has joined Moderna’s board, bringing his insights as a technology executive to a life sciences company that leverages digital drug design and automation throughout the enterprise.”

“Throughout my career, I have sought to work with companies that innovate to solve seemingly intractable challenges, and do so in ways that change traditional business paradigms,” said Mr. Sagan. “Moderna has these qualities, and I am looking forward to helping the board and management team advance their vision of creating mRNA medicines to change the way many diseases may be treated in the future.”

Today Moderna has 10 programs in clinical development and 19 development candidates in its pipeline overall, including potential new medicines that span infectious disease vaccines, cancer vaccines, intratumoral immuno-oncology, local regenerative therapeutics, and systemic therapeutics.

With the addition of Mr. Sagan, Dr. John Mendlein will now come off Moderna’s board of directors. Dr. Mendlein joined Moderna’s Executive Committee at the beginning of 2018 in the role of President, Corporate and Product Strategy. He had served on the board since 2012.

“I want to thank John for his many contributions to our board and his ongoing commitment to our vision and science. We are thrilled that he will continue to be part of our leadership team,” said Moderna’s CEO Stephane Bancel. “I now look forward to working with Paul and benefitting from his guidance and experience leading hyper-growth companies. Throughout his career, Paul has demonstrated a vision and passion for new technology and developing markets for those technologies, and I am pleased to welcome him to our team as we work to bring important new medicines to patients.”

In addition to serving on the Akamai board, Mr. Sagan is the Lead Director of VMware, Inc. and he previously served as a director of iRobot Corp., and as a board member of Digitas Inc., Dow Jones & Company, and EMC Corp. before they were acquired.

Mr. Sagan began his career as a television journalist and his work was recognized with three Emmy Awards for broadcast news in New York City. Later he joined Time Warner to co-found three businesses: NY1, the 24-hour cable news network; Roadrunner, the world’s first consumer broadband service; and Pathfinder, one of the first internet advertising platforms. He joined internet pioneer Akamai as COO in 1998 and later served as president and CEO.

Mr. Sagan is a managing director at General Catalyst, and he is a graduate of Northwestern University, where he is a life trustee. He also serves as Chairman of the Board of Directors of ProPublica, and as Chair of the Massachusetts Board of Elementary and Secondary Education.

About Moderna Therapeutics

Headquartered in Cambridge, Mass., privately held Moderna currently has strategic relationships with AstraZeneca, Plc. (AZ), Merck, Inc (MRK) and Vertex Pharmaceuticals (VRTX), as well as the Defense Advanced Research Projects Agency (DARPA), an agency of the U.S. Department of Defense; the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS); and the Bill & Melinda Gates Foundation. In 2017 Moderna was ranked a top biopharma industry employer by Science Magazine and a Top Place to Work by the Boston Globe . To learn more, visit www.modernatx.com .

Moderna Contacts

Investors:
Lorence Kim
617-209-5849
lorence.kim@modernatx.com

Media:
Jason Glashow
617-674-5648
jason.glashow@modernatx.com

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Moderna and Merck Expand mRNA Cancer Vaccines Collaboration

Thu, 03 May 2018 21:26:32 GMT

Expansion Includes the Joint Development of Moderna’s KRAS Oncogene Program and Other Potential mRNA Cancer Vaccines; Merck to Make Equity Investment in Moderna

CAMBRIDGE, Mass. and KENILWORTH N.J., May 3, 2018 — Moderna Therapeutics and Merck (NYSE:MRK), known as MSD outside the United States and Canada, today announced an expansion of their 2016 collaboration to develop and commercialize novel personalized messenger RNA (mRNA) cancer vaccines to now include shared antigen mRNA cancer vaccines including mRNA-5671, Moderna’s mRNA KRAS cancer vaccine.

Moderna developed mRNA-5671 starting in 2017. The two companies will now advance jointly mRNA-5671 in human studies, and plan to conduct combination studies with additional immuno-oncology therapies.

“Augmentation of immune responses offers great promise in cancer therapy, as our work with the PD-1-specific antibody KEYTRUDA has shown,” said Dr. Roger M. Perlmutter, President, Merck Research Laboratories. “We now look forward to expanding our exploration of mRNA cancer vaccines, working in concert with our colleagues at Moderna.”

Under the expanded agreement, Merck will be responsible for clinical development of mRNA-5671 and associated costs while Moderna will be responsible for clinical supply and associated costs. Following the completion of human proof-of-concept (hPOC) studies, Merck may opt-in on further development and commercialization of mRNA-5671 upon payment of an undisclosed fee to Moderna. Following opt-in, the parties will share equally the global net profits and costs associated with mRNA-5671. As part of this agreement, the parties may also initiate and collaborate on other shared antigen mRNA cancer vaccines programs. In addition, Merck will make a $125 million investment in Moderna in newly priced series H preferred equity. Moderna closed a series G round earlier this year.

“We are excited to build upon our productive relationship with Merck and to rapidly advance our novel mRNA-based KRAS cancer vaccine into the clinic,” said Stéphane Bancel, Moderna’s Chief Executive Officer. “While KRAS has long been a challenging target, we believe our mRNA platform offers a novel approach designed to generate and specifically present KRAS mutations to the immune system, potentially allowing the patient’s own immune system to attack and eradicate cancers that harbor these mutations.”

KRAS is one of the most frequently mutated oncogenes in human cancer, occurring in approximately 90 percent of pancreatic cancers and 30 percent of non-small cell lung cancer (NSCLC), where they are associated with worse outcomes. Presentation of epitopes containing KRAS mutations to the immune system may elicit an anti-tumor response. mRNA-5671 encodes for the four most commonly found KRAS mutations, and is designed to target most of the KRAS mutations that occur in NSCLC, colorectal cancer and pancreatic cancer.

The Moderna KRAS mRNA program utilizes tumor sequencing to identify suitable patients with specific mutations in KRAS in order to personalize their therapy, and complements the other personalized mRNA cancer vaccines in the collaboration.

About the Updated Collaboration

This is the fourth collaboration between Merck and Moderna in the past three years. The oncology alliance builds upon an initial June 2016 agreement to jointly develop personalized mRNA cancer vaccines, combining Merck’s established leadership in immuno-oncology with Moderna’s pioneering mRNA vaccine platform and GMP manufacturing capabilities. Under the 2016 personal cancer vaccine (PCV) agreement, Merck made an upfront cash payment to Moderna of $200 million to discover and develop individually tailored cancer vaccines for patients across a spectrum of cancers through proof of concept.

In November 2017, the companies announced a key milestone with the first-in-human dosing of mRNA-4157, an mRNA PCV. The Phase 1 open-label, dose escalation, multicenter study in the United States (KEYNOTE-603) will assess the safety, tolerability and immunogenicity of mRNA-4157 alone in subjects with resected solid tumors and in combination with KEYTRUDA® (pembrolizumab), an anti-PD-1 therapy, in subjects with unresectable solid tumors.

About KEYTRUDA ^® (pembrolizumab) Injection 100mg

KEYTRUDA is an anti-PD-1 therapy that works by increasing the ability of the body’s immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

Merck has the industry’s largest immuno-oncology clinical research program, which currently involves more than 750 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient’s likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.

KEYTRUDA® (pembrolizumab) Indications and Dosing

Melanoma

KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma at a fixed dose of 200 mg every three weeks until disease progression or unacceptable toxicity.

Lung Cancer

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have high PD-L1 expression [tumor proportion score (TPS) ≥50%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.

KEYTRUDA, as a single agent, is also indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS ≥1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.

KEYTRUDA, in combination with pemetrexed and carboplatin, is indicated for the first-line treatment of patients with metastatic nonsquamous NSCLC. This indication is approved under accelerated approval based on tumor response rate and progression-free survival. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

In metastatic NSCLC, KEYTRUDA is administered at a fixed dose of 200 mg every three weeks until disease progression, unacceptable toxicity, or up to 24 months in patients without disease progression.

When administering KEYTRUDA in combination with chemotherapy, KEYTRUDA should be administered prior to chemotherapy when given on the same day. See also the Prescribing Information for pemetrexed and carboplatin.

Head and Neck Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) with disease progression on or after platinum-containing chemotherapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. In HNSCC, KEYTRUDA is administered at a fixed dose of 200 mg every three weeks until disease progression, unacceptable toxicity, or up to 24 months in patients without disease progression.

Classical Hodgkin Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory classical Hodgkin lymphoma (cHL), or who have relapsed after three or more prior lines of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. In adults with cHL, KEYTRUDA is administered at a fixed dose of 200 mg every three weeks until disease progression or unacceptable toxicity, or up to 24 months in patients without disease progression. In pediatric patients with cHL, KEYTRUDA is administered at a dose of 2 mg/kg (up to a maximum of 200 mg) every three weeks until disease progression or unacceptable toxicity, or up to 24 months in patients without disease progression.

Urothelial Carcinoma

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma who are not eligible for cisplatin-containing chemotherapy. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

KEYTRUDA is also indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

In locally advanced or metastatic urothelial carcinoma, KEYTRUDA is administered at a fixed dose of 200 mg every three weeks until disease progression or unacceptable toxicity, or up to 24 months in patients without disease progression.

Microsatellite Instability-High (MSI-H) Cancer

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR)

solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options, or
colorectal cancer that has progressed following treatment with fluoropyrimidine, oxaliplatin, and irinotecan.

This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.

In adult patients with MSI-H cancer, KEYTRUDA is administered at a fixed dose of 200 mg every three weeks until disease progression, unacceptable toxicity, or up to 24 months in patients without disease progression. In children with MSI-H cancer, KEYTRUDA is administered at a dose of 2 mg/kg (up to a maximum of 200 mg) every three weeks until disease progression or unacceptable toxicity, or up to 24 months in patients without disease progression.

Gastric Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 [Combined Positive Score (CPS) ≥1] as determined by an FDA-approved test, with disease progression on or after two or more prior lines of therapy including fluoropyrimidine- and platinum-containing chemotherapy and if appropriate, HER2/neu-targeted therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The recommended dose of KEYTRUDA is 200 mg every three weeks until disease progression, unacceptable toxicity, or up to 24 months in patients without disease progression.

Selected Important Safety Information for KEYTRUDA ^®

KEYTRUDA can cause immune-mediated pneumonitis, including fatal cases. Pneumonitis occurred in 94 (3.4%) of 2799 patients receiving KEYTRUDA, including Grade 1 (0.8%), 2 (1.3%), 3 (0.9%), 4 (0.3%), and 5 (0.1%) pneumonitis, and occurred more frequently in patients with a history of prior thoracic radiation (6.9%) compared to those without (2.9%). Monitor patients for signs and symptoms of pneumonitis. Evaluate suspected pneumonitis with radiographic imaging. Administer corticosteroids for Grade 2 or greater pneumonitis. Withhold KEYTRUDA for Grade 2; permanently discontinue KEYTRUDA for Grade 3 or 4 or recurrent Grade 2 pneumonitis.

KEYTRUDA can cause immune-mediated colitis. Colitis occurred in 48 (1.7%) of 2799 patients receiving KEYTRUDA, including Grade 2 (0.4%), 3 (1.1%), and 4 (<0.1%) colitis. Monitor patients for signs and symptoms of colitis. Administer corticosteroids for Grade 2 or greater colitis. Withhold KEYTRUDA for Grade 2 or 3; permanently discontinue KEYTRUDA for Grade 4 colitis.

KEYTRUDA can cause immune-mediated hepatitis. Hepatitis occurred in 19 (0.7%) of 2799 patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.4%), and 4 (<0.1%) hepatitis. Monitor patients for changes in liver function. Administer corticosteroids for Grade 2 or greater hepatitis and, based on severity of liver enzyme elevations, withhold or discontinue KEYTRUDA.

KEYTRUDA can cause hypophysitis. Hypophysitis occurred in 17 (0.6%) of 2799 patients receiving KEYTRUDA, including Grade 2 (0.2%), 3 (0.3%), and 4 (<0.1%) hypophysitis. Monitor patients for signs and symptoms of hypophysitis (including hypopituitarism and adrenal insufficiency). Administer corticosteroids and hormone replacement as clinically indicated. Withhold KEYTRUDA for Grade 2; withhold or discontinue for Grade 3 or 4 hypophysitis.

KEYTRUDA can cause thyroid disorders, including hyperthyroidism, hypothyroidism, and thyroiditis. Hyperthyroidism occurred in 96 (3.4%) of 2799 patients receiving KEYTRUDA, including Grade 2 (0.8%) and 3 (0.1%) hyperthyroidism. Hypothyroidism occurred in 237 (8.5%) of 2799 patients receiving KEYTRUDA, including Grade 2 (6.2%) and 3 (0.1%) hypothyroidism. The incidence of new or worsening hypothyroidism was higher in patients with HNSCC, occurring in 28 (15%) of 192 patients with HNSCC, including Grade 3 (0.5%) hypothyroidism. Thyroiditis occurred in 16 (0.6%) of 2799 patients receiving KEYTRUDA, including Grade 2 (0.3%) thyroiditis. Monitor patients for changes in thyroid function (at the start of treatment, periodically during treatment, and as indicated based on clinical evaluation) and for clinical signs and symptoms of thyroid disorders. Administer replacement hormones for hypothyroidism and manage hyperthyroidism with thionamides and beta-blockers as appropriate. Withhold or discontinue KEYTRUDA for Grade 3 or 4 hyperthyroidism.

KEYTRUDA can cause type 1 diabetes mellitus, including diabetic ketoacidosis, which have been reported in 6 (0.2%) of 2799 patients. Monitor patients for hyperglycemia or other signs and symptoms of diabetes. Administer insulin for type 1 diabetes, and withhold KEYTRUDA and administer antihyperglycemics in patients with severe hyperglycemia.

KEYTRUDA can cause immune-mediated nephritis. Nephritis occurred in 9 (0.3%) of 2799 patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.1%), and 4 (<0.1%) nephritis. Monitor patients for changes in renal function. Administer corticosteroids for Grade 2 or greater nephritis. Withhold KEYTRUDA for Grade 2; permanently discontinue KEYTRUDA for Grade 3 or 4 nephritis.

Immune-mediated rashes, including Stevens-Johnson syndrome (SJS), toxic epidermal necrolysis (TEN) (some cases with fatal outcome), exfoliative dermatitis, and bullous pemphigoid, can occur. Monitor patients for suspected severe skin reactions and based on the severity of the adverse reaction, withhold or permanently discontinue KEYTRUDA and administer corticosteroids. For signs or symptoms of SJS or TEN, withhold KEYTRUDA and refer the patient for specialized care for assessment and treatment. If SJS or TEN is confirmed, permanently discontinue KEYTRUDA.

KEYTRUDA can cause other clinically important immune-mediated adverse reactions. These immune-mediated reactions may occur in any organ system. For suspected immune-mediated adverse reactions, ensure adequate evaluation to confirm etiology or exclude other causes. Based on the severity of the adverse reaction, withhold KEYTRUDA and administer corticosteroids. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Based on limited data from clinical studies in patients whose immune-related adverse reactions could not be controlled with corticosteroid use, administration of other systemic immunosuppressants can be considered. Resume KEYTRUDA when the adverse reaction remains at Grade 1 or less following corticosteroid taper. Permanently discontinue KEYTRUDA for any Grade 3 immune-mediated adverse reaction that recurs and for any life-threatening immune-mediated adverse reaction.

The following clinically significant immune-mediated adverse reactions occurred in less than 1% (unless otherwise indicated) of 2799 patients: arthritis (1.5%), uveitis, myositis, Guillain-Barré syndrome, myasthenia gravis, vasculitis, pancreatitis, hemolytic anemia, and partial seizures arising in a patient with inflammatory foci in brain parenchyma. In addition, myelitis and myocarditis were reported in other clinical trials, including classical Hodgkin lymphoma, and postmarketing use.

Solid organ transplant rejection has been reported in postmarketing use of KEYTRUDA. Treatment with KEYTRUDA may increase the risk of rejection in solid organ transplant recipients. Consider the benefit of treatment with KEYTRUDA vs the risk of possible organ rejection in these patients.

KEYTRUDA can cause severe or life-threatening infusion-related reactions, including hypersensitivity and anaphylaxis, which have been reported in 6 (0.2%) of 2799 patients. Monitor patients for signs and symptoms of infusion-related reactions, including rigors, chills, wheezing, pruritus, flushing, rash, hypotension, hypoxemia, and fever. For Grade 3 or 4 reactions, stop infusion and permanently discontinue KEYTRUDA.

Immune-mediated complications, including fatal events, occurred in patients who underwent allogeneic hematopoietic stem cell transplantation (HSCT) after being treated with KEYTRUDA. Of 23 patients with cHL who proceeded to allogeneic HSCT after treatment with KEYTRUDA on any trial, 6 patients (26%) developed graft-versus-host disease (GVHD), one of which was fatal, and 2 patients (9%) developed severe hepatic veno-occlusive disease (VOD) after reduced-intensity conditioning, one of which was fatal. Cases of fatal hyperacute GVHD after allogeneic HSCT have also been reported in patients with lymphoma who received a PD-1 receptor–blocking antibody before transplantation.

These complications may occur despite intervening therapy between PD-1 blockade and allogeneic HSCT. Follow patients closely for early evidence of transplant-related complications such as hyperacute GVHD, severe (Grade 3 to 4) acute GVHD, steroid-requiring febrile syndrome, hepatic VOD, and other immune-mediated adverse reactions, and intervene promptly.

In clinical trials in patients with multiple myeloma, the addition of KEYTRUDA to a thalidomide analogue plus dexamethasone resulted in increased mortality. Treatment of these patients with a PD-1 or PD-L1 blocking antibody in this combination is not recommended outside of controlled clinical trials.

Based on its mechanism of action, KEYTRUDA can cause fetal harm when administered to a pregnant woman. If used during pregnancy, or if the patient becomes pregnant during treatment, apprise the patient of the potential hazard to a fetus. Advise females of reproductive potential to use highly effective contraception during treatment and for 4 months after the last dose of KEYTRUDA.

In KEYNOTE-006, KEYTRUDA was discontinued due to adverse reactions in 9% of 555 patients with advanced melanoma; adverse reactions leading to discontinuation in more than one patient were colitis (1.4%), autoimmune hepatitis (0.7%), allergic reaction (0.4%), polyneuropathy (0.4%), and cardiac failure (0.4%). Adverse reactions leading to interruption of KEYTRUDA occurred in 21% of patients; the most common (≥1%) was diarrhea (2.5%). The most common adverse reactions with KEYTRUDA vs ipilimumab were fatigue (28% vs 28%), diarrhea (26% with KEYTRUDA), rash (24% vs 23%), and nausea (21% with KEYTRUDA). Corresponding incidence rates are listed for ipilimumab only for those adverse reactions that occurred at the same or lower rate than with KEYTRUDA.

In KEYNOTE-010, KEYTRUDA monotherapy was discontinued due to adverse reactions in 8% of 682 patients with metastatic NSCLC. The most common adverse event resulting in permanent discontinuation of KEYTRUDA was pneumonitis (1.8%). Adverse reactions leading to interruption of KEYTRUDA occurred in 23% of patients; the most common (≥1%) were diarrhea (1%), fatigue (1.3%), pneumonia (1%), liver enzyme elevation (1.2%), decreased appetite (1.3%), and pneumonitis (1%). The most common adverse reactions (occurring in at least 20% of patients and at a higher incidence than with docetaxel) were decreased appetite (25% vs 23%), dyspnea (23% vs 20%), and nausea (20% vs 18%).

In KEYNOTE-021(G1), when KEYTRUDA was administered in combination with carboplatin and pemetrexed (carbo/pem) in advanced nonsquamous NSCLC, KEYTRUDA was discontinued in 10% of 59 patients. The most common adverse reaction resulting in discontinuation of KEYTRUDA (≥2%) was acute kidney injury (3.4%). Adverse reactions leading to interruption of KEYTRUDA occurred in 39% of patients; the most common (≥2%) were fatigue (8%), neutrophil count decreased (8%), anemia (5%), dyspnea (3.4%), and pneumonitis (3.4%). The most common adverse reactions (≥20%) with KEYTRUDA compared to carbo/pem alone were fatigue (71% vs 50%), nausea (68% vs 56%), constipation (51% vs 37%), rash (42% vs 21%), vomiting (39% vs 27%), dyspnea (39% vs 21%), diarrhea (37% vs 23%), decreased appetite (31% vs 23%), headache (31% vs 16%), cough (24% vs 18%), dizziness (24% vs 16%), insomnia (24% vs 15%), pruritus (24% vs 4.8%), peripheral edema (22% vs 18%), dysgeusia (20% vs 11%), alopecia (20% vs 3.2%), upper respiratory tract infection (20% vs 3.2%), and arthralgia (15% vs 24%). This study was not designed to demonstrate a statistically significant difference in adverse reaction rates for KEYTRUDA as compared to carbo/pem alone for any specified adverse reaction.

In KEYNOTE-012, KEYTRUDA was discontinued due to adverse reactions in 17% of 192 patients with HNSCC. Serious adverse reactions occurred in 45% of patients. The most frequent serious adverse reactions reported in at least 2% of patients were pneumonia, dyspnea, confusional state, vomiting, pleural effusion, and respiratory failure. The most common adverse reactions (reported in at least 20% of patients) were fatigue, decreased appetite, and dyspnea. Adverse reactions occurring in patients with HNSCC were generally similar to those occurring in patients with melanoma or NSCLC, with the exception of increased incidences of facial edema (10% all Grades; 2.1% Grades 3 or 4) and new or worsening hypothyroidism.

In KEYNOTE-087, KEYTRUDA was discontinued due to adverse reactions in 5% of 210 patients with cHL, and treatment was interrupted due to adverse reactions in 26% of patients. Fifteen percent (15%) of patients had an adverse reaction requiring systemic corticosteroid therapy. Serious adverse reactions occurred in 16% of patients. The most frequent serious adverse reactions (³1%) included pneumonia, pneumonitis, pyrexia, dyspnea, GVHD, and herpes zoster. Two patients died from causes other than disease progression; one from GVHD after subsequent allogeneic HSCT and one from septic shock. The most common adverse reactions (occurring in ³20% of patients) were fatigue (26%), pyrexia (24%), cough (24%), musculoskeletal pain (21%), diarrhea (20%), and rash (20%).

In KEYNOTE-052, KEYTRUDA was discontinued due to adverse reactions in 11% of 370 patients with locally advanced or metastatic urothelial carcinoma. The most common adverse reactions (in ³20% of patients) were fatigue (38%), musculoskeletal pain (24%), decreased appetite (22%), constipation (21%), rash (21%), and diarrhea (20%). Eighteen patients (5%) died from causes other than disease progression. Five patients (1.4%) who were treated with KEYTRUDA experienced sepsis which led to death, and 3 patients (0.8%) experienced pneumonia which led to death. Adverse reactions leading to interruption of KEYTRUDA occurred in 22% of patients; the most common (≥1%) were liver enzyme increase, diarrhea, urinary tract infection, acute kidney injury, fatigue, joint pain, and pneumonia. Serious adverse reactions occurred in 42% of patients, the most frequent (≥2%) of which were urinary tract infection, hematuria, acute kidney injury, pneumonia, and urosepsis.

In KEYNOTE-045, KEYTRUDA was discontinued due to adverse reactions in 8% of 266 patients with locally advanced or metastatic urothelial carcinoma. The most common adverse reaction resulting in permanent discontinuation of KEYTRUDA was pneumonitis (1.9%). Adverse reactions leading to interruption of KEYTRUDA occurred in 20% of patients; the most common (≥1%) were urinary tract infection (1.5%), diarrhea (1.5%), and colitis (1.1%). The most common adverse reactions (³20%) in patients who received KEYTRUDA vs those who received chemotherapy were fatigue (38% vs 56%), musculoskeletal pain (32% vs 27%), pruritus (23% vs 6%), decreased appetite (21% vs 21%), nausea (21% vs 29%), and rash (20% vs 13%). Serious adverse reactions occurred in 39% of KEYTRUDA-treated patients, the most frequent (≥2%) of which were urinary tract infection, pneumonia, anemia, and pneumonitis.

It is not known whether KEYTRUDA is excreted in human milk. Because many drugs are excreted in human milk, instruct women to discontinue nursing during treatment with KEYTRUDA and for 4 months after the final dose.

There is limited experience in pediatric patients. In a study, 40 pediatric patients (16 children aged 2 years to younger than 12 years and 24 adolescents aged 12 years to 18 years) with advanced melanoma, lymphoma, or PD-L1–positive advanced, relapsed, or refractory solid tumors were administered KEYTRUDA 2 mg/kg every 3 weeks. Patients received KEYTRUDA for a median of 3 doses (range 1–17 doses), with 34 patients (85%) receiving KEYTRUDA for 2 doses or more. The safety profile in these pediatric patients was similar to that seen in adults treated with KEYTRUDA. Toxicities that occurred at a higher rate (³15% difference) in these patients when compared to adults under 65 years of age were fatigue (45%), vomiting (38%), abdominal pain (28%), hypertransaminasemia (28%), and hyponatremia (18%).

About Merck

For more than a century, Merck, a leading global biopharmaceutical company known as MSD outside of the United States and Canada, has been inventing for life, bringing forward medicines and vaccines for many of the world’s most challenging diseases. Through our prescription medicines, vaccines, biologic therapies and animal health products, we work with customers and operate in more than 140 countries to deliver innovative health solutions. We also demonstrate our commitment to increasing access to health care through far-reaching policies, programs and partnerships. Today, Merck continues to be at the forefront of research to advance the prevention and treatment of diseases that threaten people and communities around the world - including cancer, cardio-metabolic diseases, emerging animal diseases, Alzheimer’s disease and infectious diseases including HIV and Ebola. For more information, visit www.merck.com and connect with us on Twitter , Facebook , Instagram , YouTube and LinkedIn .

Media:
Pamela Eisele, 267-305-3558
Ian McConnell, 973- 901-5722

Investors:
Teri Loxam, 908-740-1986
Michael DeCarbo, 908-740-1807

About Moderna Therapeutics

Moderna pioneers the discovery and development of messenger RNA (mRNA) therapeutics and vaccines, an entirely new class of medicines that directs the body’s cells to produce intracellular or secreted proteins that can have a therapeutic or preventive benefit for both patients and healthy individuals. With its breakthrough platform, Moderna is creating mRNA medicines for a wide range of diseases and conditions, in many cases by addressing currently undruggable targets or underserved areas of medical need. Moderna is developing its innovative mRNA medicines for infectious diseases, immuno-oncology, rare diseases, and cardiovascular diseases, through solely controlled programs and collaborations with strategic partners.

Headquartered in Cambridge, Mass., privately held Moderna currently has strategic relationships with AstraZeneca, Plc. (AZ), Merck (MRK) and Vertex Pharmaceuticals (VRTX), as well as the Defense Advanced Research Projects Agency (DARPA), an agency of the U.S. Department of Defense; the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS); and the Bill & Melinda Gates Foundation. In 2017, Moderna was ranked a top biopharma industry employer by Science Magazine and a Top Places to Work by the Boston Globe . To learn more, visit www.modernatx.com .

Moderna Contacts

Media:
Jason Glashow
617-674-5648
jason.glashow@modernatx.com

Investors:
Lorence Kim
617-209-5849
lorence.kim@modernatx.com

Forward-looking Statement of Merck & Co., Inc., Kenilworth, N.J., USA

This news release of Merck & Co., Inc., Kenilworth, N.J., USA (the “company”) includes “forward-looking statements” within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. These statements are based upon the current beliefs and expectations of the company’s management and are subject to significant risks and uncertainties. There can be no guarantees with respect to pipeline products that the products will receive the necessary regulatory approvals or that they will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements.

Risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and health care legislation in the United States and internationally; global trends toward health care cost containment; technological advances, new products and patents attained by competitors; challenges inherent in new product development, including obtaining regulatory approval; the company’s ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of the company’s patents and other protections for innovative products; and the exposure to litigation, including patent litigation, and/or regulatory actions.

The company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise. Additional factors that could cause results to differ materially from those described in the forward-looking statements can be found in the company’s 2017 Annual Report on Form 10-K and the company’s other filings with the Securities and Exchange Commission (SEC) available at the SEC’s Internet site ( www.sec.gov ).

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Lori Henderson Joins Moderna as General Counsel and Corporate Secretary

Tue, 24 Apr 2018 21:13:53 GMT

CAMBRIDGE, Mass., April 24, 2018 — Moderna Therapeutics, a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, today announced that Lori Henderson has joined the company as General Counsel. She will serve on Moderna’s Executive Committee and as Corporate Secretary, reporting to Dr. John Mendlein, President, Corporate and Product Strategy.

“Lori brings a valuable blend of corporate, life sciences, and technology experience that is important to Moderna as we mature and drive development of programs across our mRNA pipeline, create partnerships with key industry collaborators, and continue to advance our technology platform and core infrastructure. Her regulatory, governance, corporate development, and biopharmaceutical industry experience, as well as her background working with executive leadership and corporate boards, will be a tremendous asset as we continue to evolve our organization moving forward,” said Dr. Mendlein.

Ms. Henderson has nearly 30 years as a corporate attorney in both law firm and general counsel roles guiding global public and private companies. She joins Moderna from Albany Molecular Research (AMRI), a pharmaceutical contract research and manufacturing organization, where she was Senior Vice President, General Counsel, Corporate Secretary, and Head of Business Development. While at AMRI, which was recently acquired by private equity firms, she led all legal and business development functions, and was instrumental in the overall growth of the organization, the acquisition of multiple companies, ongoing regulatory and compliance matters, and engagement with pharmaceutical and licensing partners.

Prior to AMRI, Ms. Henderson was General Counsel, Corporate Secretary, and Chief Administrative Officer at Rand Worldwide, a technology sales and services organization, and prior to that, General Counsel, Corporate Secretary, and Chief Administrative Officer of Moldflow Corporation (which was sold to Autodesk). Ms. Henderson started her career in the corporate practice of the Boston law firm Goodwin Procter.

“It is a privilege to join Moderna and its leadership team at such an exciting time in the evolution of the company,” said Henderson. “I look forward to helping build an organization that supports the long-term demands of Moderna’s evolving business, its science and development programs, and efforts to bring mRNA medicines to patients where there is an urgent need for new therapies and vaccines.”

Ms. Henderson received her Juris Doctorate from The George Washington University Law School and received her B.A. from Gordon College.

About Moderna Therapeutics

Moderna pioneers the discovery and development of messenger RNA (mRNA) therapeutics and vaccines, an entirely new class of medicines that directs the body’s cells to produce intracellular or secreted proteins that can have a therapeutic or preventive benefit for both patients and healthy individuals. With its breakthrough platform, Moderna is creating mRNA medicines for a wide range of diseases and conditions, in many cases by addressing currently undruggable targets or underserved areas of medical need. Moderna is developing its innovative mRNA medicines for infectious diseases, immuno-oncology, rare diseases, and cardiovascular diseases, through solely controlled programs and collaborations with strategic partners.

Headquartered in Cambridge, Mass., privately held Moderna currently has strategic relationships with AstraZeneca, Plc. (AZ), Merck, Inc (MRK) and Vertex Pharmaceuticals (VRTX), as well as the Defense Advanced Research Projects Agency (DARPA), an agency of the U.S. Department of Defense; the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS); and the Bill & Melinda Gates Foundation. In 2017, Moderna was ranked a top biopharma industry employer by Science Magazine and a Top Places to Work by the Boston Globe . To learn more, visit www.modernatx.com .

Moderna Contacts

Investors:
Lorence Kim
617-209-5849
lorence.kim@modernatx.com

Media:
Jason Glashow
617-674-5648
jason.glashow@modernatx.com

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Megan Pace Joins Moderna as Chief Corporate Affairs Officer

Wed, 04 Apr 2018 20:51:33 GMT

CAMBRIDGE, Mass., April 4, 2018 — Moderna Therapeutics, a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, today announced that Megan Pace has joined Moderna as Chief Corporate Affairs Officer. She will serve on Moderna’s Executive Committee and report to Chief Executive Officer Stéphane Bancel.

“I am thrilled to welcome Megan to Moderna. As a rapidly growing organization, we will benefit from her extensive public affairs experience and prior leadership roles at biotech companies that have made the successful transition from research and development to their first product launches,” said Stéphane Bancel, Chief Executive Officer. “Megan has a deep understanding of the complexity of drug development and commercialization globally, and I look forward to having her on the Moderna Executive Committee.”

“I am excited to join Moderna at an important time in the Company’s efforts to bring potentially transformative mRNA medicines through the pipeline and to patients,” said Pace. “I look forward to developing relationships with key stakeholders and increasing awareness of the work being done by Moderna scientists and clinicians in pursuit of this new class of vaccines and therapies.”

Ms. Pace has nearly 20 years of communications, government affairs, patient advocacy, investor relations, program leadership and corporate planning experience in the biotech sector, including prior executive team roles at Agios Pharmaceuticals and Vertex Pharmaceuticals. Prior to Moderna, she served as Senior Vice President, Strategic Operations and PKR Program Executive at Agios; Senior Vice President, Corporate Communications at Vertex; and Senior Director, Public Affairs at Genentech. She also worked on the communications and government affairs teams at Eli Lilly and Company, as well as at Porter Novelli, a global public relations firm.

Ms. Pace holds a B.A. from the College of Charleston. She is a board member of the Boomer Esiason Foundation and the Make-a-Wish Foundation of Massachusetts and Rhode Island.

About Moderna Therapeutics

Moderna pioneers the discovery and development of messenger RNA (mRNA) therapeutics and vaccines, an entirely new class of medicines that directs the body’s cells to produce intracellular or secreted proteins that can have a therapeutic or preventive benefit for both patients and healthy individuals. With its breakthrough platform, Moderna is creating mRNA medicines for a wide range of diseases and conditions, in many cases by addressing currently undruggable targets or underserved areas of medical need. Moderna is developing its innovative mRNA medicines for infectious diseases, immuno-oncology, rare diseases, and cardiovascular diseases, through solely controlled programs and collaborations with strategic partners.

Headquartered in Cambridge, Mass., privately held Moderna currently has strategic relationships with AstraZeneca, Plc. (AZ), Merck, Inc (MRK) and Vertex Pharmaceuticals (VRTX), as well as the Defense Advanced Research Projects Agency (DARPA), an agency of the U.S. Department of Defense; the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS); and the Bill & Melinda Gates Foundation. In 2017, Moderna was ranked a top biopharma industry employer by Science Magazine and a Top Places to Work by the Boston Globe . To learn more, visit www.modernatx.com .

Moderna Contacts

Investors:
Lorence Kim
617-209-5849
lorence.kim@modernatx.com

Media:
media@modernatx.com

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Newly Published Data Highlight Moderna’s mRNA Platform in Advancing Prophylactic Vaccine for Cytomegalovirus (CMV) Infections

Tue, 20 Feb 2018 20:47:07 GMT

Paper in the Journal Vaccine Shows Versatility of Platform for Encoding and Delivering Complex CMV Antigens, including Six mRNAs for CMV Glycoprotein gB and Pentamer Protein

CAMBRIDGE, Mass., February 20, 2018 — Moderna Therapeutics, a clinical stage biotechnology company that is pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for high unmet medical needs in patients, today announced the publication of new data in the scientific journal Vaccine that demonstrate the versatility of its mRNA vaccine platform in developing novel prophylactic vaccines to potentially prevent human cytomegalovirus (CMV) infections and associated disease burdens, both for pregnant women and in transplant patients.

Human cytomegalovirus (CMV) is the leading cause of infection in newborns, and can lead to deafness, microcephaly (small, not fully developed heads) and severe disabilities, including vision loss and mental deficiencies, among other serious complications. It is also the most frequent viral disease in transplant recipients, often leading to transplant failure. At this time, there are no approved vaccines for CMV. Moderna currently is conducting a Phase 1 study in CMV in the U.S. with its clinical development candidate mRNA-1647.

The newly published data showed that Moderna’s mRNA encoding CMV glycoproteins gB and pentameric complex (PC), encapsulated in its delivery system, produced potent and durable neutralizing antibody titers in immunized mice and non-human primates. The mRNA delivered six mRNAs -- including the five subunits encoding the pentamer protein, which then correctly assembled within the cell to make the pentamer protein.

“Using mRNA, we have shown the ability to induce very precise copies of even complex antigens in-vivo, that in turn induce robust immune responses in mouse and non-human primate models. This gives us real hope that we will eventually see similar results in humans,” said Dr. Mike Watson, SVP Vaccine Partnerships & Health Impact. “These data highlight the ability of the platform to effectively, rapidly and precisely induce complex antigens that have proved so challenging to other vaccine approaches. For CMV, these platform characteristics give us the opportunity to potentially tailor the vaccine to target different CMV-related diseases, such as those associated with congenital CMV infections and transplant-associated CMV infections.”

The two dominant glycoproteins of CMV, gB and PC, play different roles in the infection of different cells. When brought together in a multi-valent vaccine, it may be possible to neutralize infection across a wide variety of cells, including fibroblasts, endothelial, epithelial and myeloid cells – with the majority of neutralizing antibodies in CMV seropositive individuals directed against PC. Moderna researchers also developed a pp65 mRNA/LNP that was used with gB and PC to broaden T cell responses. Neutralizing antibodies against envelope glycoproteins and cellular responses targeting a variety of viral proteins, particularly pp65, are believed to help control CMV infection and re-activation.

Moderna began dosing patients in November 2017 to assess the safety, tolerability and immunogenicity of mRNA-1647. Additional clinical trial information can be found here .

Notes for Editors

“Multi-antigenic human cytomegalovirus mRNA vaccines that elicit potent humoral and cell-mediated immunity,” by Dr. Shinu John, Ph.D; Dr. Olga Yuzhakov, Ph.D; Angela Woods; Jessica Deterling; Dr. Kimberly Hassett, PhD; Dr. Christine A Shaw, Ph.D; Dr. Giuseppe Ciaramella, Ph.D. DOI: 10.1016/j.vaccine.2018.01.029.

Copies of this paper are available to credentialed journalists upon request; please contact Elsevier's Newsroom at newsroom@elsevier.com or +31 20 485 2492.

About Vaccine

Vaccine is the pre-eminent journal for those interested in vaccines and vaccination. It is the official journal of The Edward Jenner Society and The Japanese Society for Vaccinology, and is published by Elsevier. www.journals.elsevier.com/vaccine .

About Moderna’s mRNA Prophylactic Vaccines

Messenger RNA (mRNA) plays a fundamental role in human biology, directing protein production in cells. When used as a drug, mRNA can direct cells to produce therapeutic proteins (mRNA therapeutics) to fight disease or antigenic proteins (mRNA vaccines) to prevent disease.

Moderna’s mRNA vaccines encode for viral antigenic proteins associated with viruses. The mRNA directs cells to produce and express the proteins, much like a native infection would do, but without the ability to cause disease.

As a result, the immune system recognizes the antigenic proteins as foreign to the body and produces antibodies that have the potential to neutralize the virus and to prevent infections in the event the vaccinated person is exposed to the actual virus in the future.

About Moderna Therapeutics

Moderna pioneers the discovery and development of messenger RNA (mRNA) therapeutics and vaccines, an entirely new class of medicines that directs the body’s cells to produce intracellular or secreted proteins that can have a therapeutic or preventive benefit for both patients and healthy individuals. With its breakthrough platform, Moderna is creating mRNA medicines for a wide range of diseases and conditions, in many cases by addressing currently undruggable targets or underserved areas of medical need. Moderna is developing its innovative mRNA medicines for infectious diseases, immuno-oncology, rare diseases, and cardiovascular diseases, through solely controlled programs and collaborations with strategic partners.

Headquartered in Cambridge, Mass., privately held Moderna currently has strategic relationships with AstraZeneca, Plc. (AZ), Merck, Inc (MRK) and Vertex Pharmaceuticals (VRTX), as well as the Defense Advanced Research Projects Agency (DARPA), an agency of the U.S. Department of Defense; the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS); and the Bill & Melinda Gates Foundation. In 2017, Moderna was ranked a top biopharma industry employer by Science Magazine and a Top Places to Work by the Boston Globe . To learn more, visit www.modernatx.com .

Moderna Contacts

Investors:
Lorence Kim
617-209-5849
lorence.kim@modernatx.com

Media:
media@modernatx.com

VIEW ALL NEWS

Moderna Closes $500 Million Equity Financing

Thu, 01 Feb 2018 20:42:35 GMT

CAMBRIDGE, Mass., February 1, 2018 — Moderna Therapeutics, a clinical stage biotechnology company that is pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for high unmet medical needs in patients, today announced that it has raised $500 million in a new funding round. This latest equity financing includes support from new U.S. and international institutional investors, and existing institutional investors.

New investors include a wholly-owned subsidiary of the Abu Dhabi Investment Authority (ADIA), BB Biotech AG, Julius Baer, Singapore-based EDBI and Sequoia Capital China. Existing investors that also participated in this round include Fidelity Management & Research Company, Pictet, Viking Global Investors, ArrowMark Partners and Alexandria Venture Investments.

The funding will help drive the company’s continued execution of its rapidly growing pipeline of 19 development candidates – including 10 programs in clinical trials. Proceeds will also be used to further Moderna’s investment in its research engine and other 2018/2019 priorities including:

Innovative drug discovery in rare disease and prophylactic vaccines, while initiating several phase two clinical trials;
Continued investment in mRNA science and technology to further advance the company’s mRNA development and delivery platform; and
Investment in the company’s manufacturing infrastructure and digital backbone.

“We continue to execute across the organization and against our goal of rapidly building a pipeline of mRNA medicines, which we have demonstrated by moving 10 candidates into the clinic over the past 24 months. We are delighted by the strong support from new U.S. and global investors in our business, science and vision for the future, and the continued and expanded support from existing investors,” said Stéphane Bancel, Chief Executive Officer of Moderna. “This past year we have demonstrated an ability to scale our platform and introduce new development candidates across multiple disease modalities, including promising opportunities in rare disease, immuno-oncology, and cardiovascular and infectious diseases.”

With the completion of this funding round, Moderna has nearly $1.4 billion in cash on its balance sheet in addition to its potential access to up to nearly $250 million in grants from the Bill & Melinda Gates Foundation, the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS), and the Defense Advanced Research Projects Agency (DARPA).

Placement agents for this financing round were Goldman Sachs & Co. LLC, JP Morgan Securities LLC and Morgan Stanley & Co. LLC. Goodwin Procter provided legal counsel.

About Moderna Therapeutics

Moderna pioneers the discovery and development of messenger RNA (mRNA) therapeutics and vaccines, an entirely new class of medicines that directs the body’s cells to produce intracellular or secreted proteins that can have a therapeutic or preventive benefit for both patients and healthy individuals. With its breakthrough platform, Moderna is creating mRNA medicines for a wide range of diseases and conditions, in many cases by addressing currently undruggable targets or underserved areas of medical need. Moderna is developing its innovative mRNA medicines for infectious diseases, immuno-oncology, rare diseases, and cardiovascular diseases, through solely controlled programs and collaborations with strategic partners.

Headquartered in Cambridge, Mass., privately held Moderna currently has strategic relationships with AstraZeneca, Plc. (AZ), Merck, Inc (MRK) and Vertex Pharmaceuticals (VRTX), as well as the Defense Advanced Research Projects Agency (DARPA), an agency of the U.S. Department of Defense; the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS); and the Bill & Melinda Gates Foundation. In 2017 Moderna was ranked a top biopharma industry employer by Science Magazine and a Top Places to Work by the Boston Globe . To learn more, visit www.modernatx.com .

Moderna Contacts

Investors:
Lorence Kim
617-209-5849
lorence.kim@modernatx.com

Media:
media@modernatx.com

VIEW ALL NEWS

Moderna Announces an Array of Clinical Advances and Outlines 2018 Priorities; 19 Development Candidates, including 10 Clinical Programs, Highlight Productivity of mRNA Platform

Mon, 08 Jan 2018 20:39:58 GMT

Clinical advances include: planned phase 2 study for a VEGF mRNA therapeutic; a new cancer vaccine IND application; and initiation of two phase 1 studies of prophylactic vaccines for infectious diseases

Three new development candidates target liver expression of therapeutic human proteins and first antibody

2018 pipeline strategy focuses on new rare disease development candidates and advancing additional prophylactic vaccines development candidates in areas of high unmet need

Cambridge, Mass., January 8, 2018 — Moderna Therapeutics, a clinical stage biotechnology company that is pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for high unmet medical needs in patients, today announced important advances in its mRNA development pipeline, demonstrating the increasing productivity of its platform, including its first mRNA program to enter phase 2, new infectious disease vaccine and oncology programs entered into Phase 1 clinical studies, and the ongoing expansion of its pipeline with several new development candidates (DCs). A leader in mRNA science and development, Moderna continues to make notable progress across its broad, diverse pipeline, which now includes 19 mRNA drug candidates spanning infectious diseases, immuno-oncology, rare diseases and cardiovascular diseases.

Moderna’s Chief Executive Officer Stéphane Bancel detailed company strategy and progress today at the 36th Annual J.P. Morgan Healthcare Conference in San Francisco.

The company today announced several new advances including:

A Phase 2a study of mRNA AZD8601, a localized mRNA therapeutic encoding for vascular endothelial growth factor, VEGF-A, being developed in partnership with AstraZeneca. Information on the clinical study, including design and target indication, will be detailed in the coming weeks. Led by AstraZeneca, this will be Moderna’s first phase 2 study.
A new development candidate, mRNA-3927, for a rare disease within the liver modality. mRNA-3927 directs liver expression of a deficient enzyme in patients with propionic acidemia (PA), a serious and potentially life-threatening rare disease, which is part of a family of disorders known as organic acidemias. There are no approved therapies or ongoing clinical trials for PA. In September, Moderna announced its first rare disease DC, mRNA-3704, to treat methylmalonic acidemia, or MMA, another serious and often deadly organic acidemia.
The filing of an investigational new drug (IND) application for mRNA-5671, a KRAS cancer vaccine. KRAS is one of the most frequently mutated oncogenes in human cancer (approximately 30 percent of all cases). mRNA-5671 encodes for the four most commonly found KRAS mutations, which will cover most of the mutations that occur in non-small cell lung cancer, colorectal cancer and pancreatic cancer.
The initiation of two phase 1 prophylactic vaccine studies for mRNA-1647, a cytomegalovirus (CMV) vaccine, and mRNA-1653, a human metapneumovirus and parainfluenza virus type 3 (HMPV+PIV3) combination vaccine. CMV is the most common cause of newborn disability and the most frequent viral disease in transplant recipients, often leading to transplant failure. mRNA-1647 is made of 6 mRNAs, one coding for the herpesvirus glycoprotein (gB) antigen and 5 coding for the pentamer. HMPV and PIV3 are the second and third most common causes, respectively, of lower respiratory hospitalizations in children, behind respiratory syncytial virus (RSV). Currently, there are no approved vaccines for CMV, HMPV or PIV3.
A new development candidate, mRNA-1944, which directs liver expression of an antibody that can potentially neutralize chikungunya virus circulating in the blood. Moderna has a Phase 1 study underway for a prophylactic vaccine, mRNA-1388, to prevent infection from the chikungunya virus. An antibody approach would be more desirable in certain settings, such as in immuno-compromised populations, when rapid post-exposure treatment or prophylaxis is warranted, or when protection is needed only for short periods of time. This program is sponsored by DARPA.

“We are proud of the progress we have made over the past year as we continue to see real development pipeline momentum and productivity from our platform, and continue to deliver to the clinic important advances in mRNA science. We have achieved critical milestones in R&D, having gone from four clinical programs at the beginning of the year to now having 10 medicines in human testing, and our intention is to continue to rapidly advance our pipeline with an array of new development programs,” said Bancel. “2016 was the year of mRNA vaccines in the clinic. 2017 was the year of several mRNA therapeutics in the clinic. In 2018, we will continue to evolve our pipeline of mRNA therapeutics, specifically focusing on discovering new rare disease drug candidates, while remaining committed to advancing new vaccine development candidates to address serious unmet needs. We also will continue to work toward a summer 2018 opening and rolling scale-up of our GMP clinical mRNA manufacturing facility, which is a cornerstone of our long-term strategy to move multiple development programs simultaneously into and through phase 1, phase 2, and phase 3 clinical studies.”

As of today, nearly 700 subjects have been dosed across Moderna’s internally developed and partnered clinical programs with AstraZeneca and Merck. Moderna's full pipeline can be found here.

2018 Strategic Priorities

During today’s presentation, Mr. Bancel outlined Moderna’s key strategic priorities for 2018 which include:

Effective execution of the development pipeline by continuing to advance programs through clinical study and by moving additional development programs into the clinic;
Emphasis on the discovery of new rare disease development candidates and new prophylactic vaccines to address high unmet medical needs;
Continued investment in the evolution of the company’s mRNA platform, including exploring new modalities to expand the application of its technology in new therapeutic areas;
Completion of construction of the company’s 200,000 square foot GMP mRNA clinical manufacturing facility in Norwood, Mass., with an anticipated opening in the summer of 2018 and subsequent rolling scale-up of the facility.

Detailed Q4 2017 Clinical and Development Program Updates

Moderna’s pipeline spans five modalities: prophylactic vaccines, therapeutic vaccines, intratumoral immuno-oncology therapeutics, localized therapeutics and liver therapeutics. Following are advances from across modalities since the company’s September business update:

Commercial Prophylactic Vaccines

Initiation of Phase 1 study of mRNA-1647, a cytomegalovirus (CMV) vaccine: The Phase 1, placebo-controlled multi-center study of mRNA-1647 began dosing patients in November 2017, and will assess safety, tolerability and immunogenicity. [ clinicaltrials.gov listing ]. A complex vaccine, mRNA-1647 consists of six mRNAs, including five proteins (gH, gL, UL128, UL130 and UL131A) designed to express the pentamer complex, and another CMV antigen, the herpesvirus glycoprotein (gB) protein. CMV is the most common cause of newborn disability, leading to deafness, microcephaly (small, not fully developed heads and severe disabilities), vision loss and mental deficiencies, among other serious complications. It is also the most frequent viral disease in transplant recipients, often leading to transplant failure. Currently, there is no approved vaccine for CMV.
Initiation of Phase 1 study of mRNA-1653, a combination human metapneumovirus and parainfluenza virus type 3 (HMPV+PIV3) vaccine: The placebo-controlled, multi-site Phase 1 study of mRNA-1653 began dosing patients in December 2017 and will assess for safety, tolerability, and immunogenicity. HMPV and PIV3 typically cause mild respiratory illness, but can become severe in young children, the elderly and other immunocompromised adults. HMPV and PIV3 are the second and third most common causes, respectively, of lower respiratory hospitalizations in children, behind RSV. Currently, there is no approved vaccine for either HMPV or PIV3.
Publications: in September, Moderna announced a publication in the August issue of Molecular Therapy that provides mechanistic insights about its mRNA prophylactic vaccines. The research, led by Professor Karin Loré, Ph.D., and her group at the Karolinska Institutet in Stockholm, Sweden, characterizes how Moderna’s vaccines target key antigen-presenting cells, leading to both B cell and T cell activation, which yields a potent immune response. Two additional papers based on Dr. Lore’s work offer additional insights into the method of action of Moderna’s vaccine technology. A paper published in November 2017 in Frontiers in Immunology demonstrates that Moderna vaccine technology is able to stimulate a type of B cell that makes high-quality, antigen-specific antibodies consistent with high seroconversion rates in humans. A second paper published in November in the Journal of Immunology, shows that Moderna’s vaccine technology produces a desirable kinetic immune activation and subsequent suppression by myeloid derived suppressor cells (MDSCs), which are major regulators of T- cell responses.

Moderna also continues to advance vaccines in collaboration with government agencies and non-government organizations to address major public health issues. The company is furthering its efforts through its current contract with the Biomedical Advanced Research and Development Agency (BARDA) – part of the Office of the Assistant Secretary for Preparedness and Response at the U.S. Department of Health and Human Services – to develop an mRNA Zika vaccine, now including a head-to-head comparison of two potential mRNA candidates (mRNA 1325 and mRNA 1893) through Phase 1, after which it will determine the best candidate for further clinical development to BLA submission for licensure.

Therapeutic Vaccines

Initiation of Phase 1 study of personalized cancer vaccine (PCV), mRNA-4157 (KEYNOTE-603): In November, Moderna announced that it has initiated dosing for a Phase 1 study of its PCV. The Phase 1 open-label, dose escalation, multicenter study in the United States will assess the safety, tolerability and immunogenicity of mRNA-4157 alone in subjects with resected solid tumors and in combination with KEYTRUDA® (pembrolizumab), an anti-PD-1 therapy, marketed by Merck (known as MSD outside the U.S. and Canada) in subjects with unresectable solid tumors. Moderna has a strategic collaboration with Merck to develop PCVs in collaboration with KEYTRUDA®. Moderna first identifies neoepitopes present in a patient’s tumor and then creates an mRNA-based PCV encoding for approximately 20 neoepitopes. When injected into the patient, the mRNA-based PCV directs the patient’s cells to express the selected neoepitopes. In turn, this may help the patient’s immune system better recognize cancer cells as foreign and eradicate them. mRNA-4157 also has the potential to enhance clinical outcomes associated with checkpoint inhibitor therapies. Leveraging its rapid cycle time, small-batch manufacturing technique and digital infrastructure, Moderna plans to manufacture and supply each individually tailored and manufactured PCV to patients within weeks.
Investigational new drug (IND) application filed for mRNA-5671, a KRAS cancer vaccine: KRAS is one of the most frequently mutated oncogenes in human cancer (approximately 30% of all cases). KRAS mutations are found principally in non-small cell lung cancer (NSCLC), colorectal cancer and pancreatic cancer, and are associated with worse outcomes. Hotspots of KRAS mutations are found in different tumor types and can serve as tumor rejection epitopes. Presentation of these epitopes to the immune system may elicit a robust anti-tumor response. mRNA-5671 encodes for the four most commonly found KRAS mutations, which will cover most of the mutations that occur in NSCLC, colorectal cancer and pancreatic cancer.
National Cancer Institute (NCI) to study mRNA-based PCV: In collaboration with Moderna, the Surgery Branch of the NCI’s Center for Cancer Research plans to sponsor a Phase 1/2 study to investigate the safety and immunogenicity of mRNA-based PCVs for patients under the direction of Steven A. Rosenberg, M.D., Ph.D., Chief of Surgery, NCI. As part of this collaboration, Moderna will manufacture mRNA-based personalized cancer vaccines (NCI-4650) for up to 12 patients with advanced-stage, metastatic cancers.

Localized Therapeutics

Phase 2a study of mRNA AZD-8601: Phase 2a study of mRNA AZD8601: Dosing of patients is anticipated for early in the first quarter of 2018 for the Phase 2a study of mRNA AZD8601, a localized mRNA therapeutic encoding for vascular endothelial growth factor, VEGF-A. The mRNA AZD8601 program is led by AstraZeneca. Data from a Phase 1 randomized, double-blind, placebo-controlled, single ascending dose study that assessed the safety, tolerability and pharmacokinetics (PK) of mRNA AZD8601 after single dose administration in male patients with Type 2 diabetes mellitus are expected to be presented at a scientific congress and published in 2018.

Liver Therapeutics

Naming of pre-clinical development candidate mRNA-3927, encoding an intracellular enzyme to treat Propionic Acidemia (PA): PA is a rare, autosomal recessive organic acidemia/aciduria caused by a mitochondrial enzyme deficiency in propionyl-CoA carboxylase (PCC) due to mutations in PCCA (PA type I) or PCCB (PA type II). mRNA-3927 combines mRNA-encoded proteins for both the PCCA and PCCB enzyme components with the goal of addressing all PA subtypes. PA is a natural follow-on to the MMA program, as both are organic acidemias with defective enzymes along the same metabolic pathway. PA is a rare disease with no approved therapy. The disorder typically impacts newborn children, and patients with PA often present acutely with metabolic acidosis, cardiac arrhythmias and hyperammonemia causing severe central nervous system dysfunction.
Naming of pre-clinical development candidate mRNA-1944, encoding an antibody against the chikungunya virus: mRNA-1944 encodes for an antibody that can neutralize the chikungunya virus circulating in the blood. Moderna has a Phase 1 study underway for a prophylactic mRNA vaccine (mRNA-1388) to prevent infection from chikungunya virus. However, in certain situations, such as in immuno-compromised populations, when rapid post-exposure treatment or prophylaxis is warranted, or when protection is needed only for short periods (three to six months), an antibody approach is desirable. Chikungunya typically causes mild fever and transient joint pain. In approximately 15 percent of infected patients, it can cause long-term, severe arthritis. Chikungunya historically has been limited to warmer climates in Asia and Africa, but recent cases have been identified in the Americas and Europe. There is no approved vaccine or treatment for chikungunya.
Collaboration for mRNA AZD-7970, encoding the secreted protein relaxin to treat heart failure: In November, Moderna and AstraZeneca announced a new strategic agreement to co-develop and co-commercialize mRNA AZD-7970, which is designed to instruct cells in the body to produce and express relaxin, a secreted protein with systemic effect. Heart failure occurs when the heart is weakened and cannot pump enough blood to meet the body’s needs. Biologic functions for relaxin suggest that expression of the hormone may directly impact underlying conditions that exacerbate heart failure, leading to the regrowth of heart tissue, controlling inflammation, reordering the extracellular matrix, improving renal function, and relieving hepatic portal pressure.
Publications: In December, Moderna announced the publication of preclinical data supporting its first rare disease development program, mRNA-3704, a therapeutic for methylmalonic acidemia (MMA), a serious and often life-threatening organic acidemia disorder. The data, published in the journal Cell Reports, demonstrate that intravenous (IV) administration of an mRNA therapeutic encoding for human methylmalonyl-CoA mutase (hMUT), the enzyme most frequently mutated in MMA, enabled liver expression of hMUT in MMA mouse models, leading to a significant reduction in methylmalonic acid and complete survival of treated mice versus control group with a dramatic improvement in weight gain. Repeat IV dosing did not increase markers of liver toxicity or inflammation. The study was conducted in partnership with researchers at the Medical Genomics and Metabolic Genetics Branch of the National Human Genome Research Institute at the National Institutes of Health.

Q4 2017 AND RECENT BUSINESS/FINANCIAL UPDATES

Board of Directors and Organizational Updates

John Mendlein joined Moderna as President, Corporate and Product Strategy. Earlier this month, Moderna announced that John Mendlein, Ph.D., joined the company as President, Corporate and Product Strategy. In this role, Dr. Mendlein will be responsible for corporate strategy, product advancement and strategy, partnering and product protection. He will serve on Moderna’s Executive Committee and report to Chief Executive Officer Stéphane Bancel. Dr. Mendlein has helped start and lead numerous innovative life sciences companies. He is Vice Chairman of the Board and a founder of Fate Therapeutics, Inc., and holds board positions with Editas Medicine, Inc., and Axcella Health, Inc. He also serves on the Biotechnology Industry Organization (“BIO”) emerging companies board. Dr. Mendlein previously served as the Chief Executive Officer of aTyr Pharma, and Fate Therapeutics, as well as Adnexus Therapeutics, Inc., (acquired by BMS). Before that, he served as Chairman and Chief Executive Officer of Affinium Pharmaceuticals, Ltd. (acquired by Debiopharm Group), and as a board member, General Counsel and Chief Knowledge Officer at Aurora Bioscience Corporation (acquired by Vertex Pharmaceuticals).
Stephen Berenson appointed to Board of Directors. In October, Moderna announced that Stephen Berenson was joining the company’s Board of Directors. Mr. Berenson, who joined Flagship Pioneering in June of 2017, previously served for 12 years as the Vice Chairman of Investment Banking at J.P. Morgan, and focused on providing high-touch strategic advice and complex transaction execution to leading companies across all industries globally. In total, Mr. Berenson spent more than 33 years with J.P. Morgan as an investment banker, where he worked across all major geographies, product areas and industry groups. He played key roles in building J.P. Morgan's M&A, equities and technology investment banking businesses.
Continued growth across organization: In 2016, Moderna expanded its headcount from approximately 500 to nearly 600 team members.
Continued strong cash position: Moderna maintained a strong cash position in 2017. As of December 31, 2017, the company had approximately $910 million in cash, as compared to $1.306 billion in cash as of December 31, 2016.
2017 cash inflows : From reimbursement, product milestones and investment income was approximately $55 million.
Significant investments in the business: Moderna’s cash operating expense and capital expenditures in 2017 totaled approximately $455 million.

About Moderna Therapeutics

Moderna pioneers the discovery and development of messenger RNA (mRNA) therapeutics and vaccines, an entirely new class of medicines that directs the body’s cells to produce intracellular or secreted proteins that can have a therapeutic or preventive benefit for both patients and healthy individuals. With its breakthrough platform, Moderna is creating mRNA medicines for a wide range of diseases and conditions, in many cases by addressing currently undruggable targets or underserved areas of medical need. Moderna is developing its innovative mRNA medicines for infectious diseases, immuno-oncology, rare diseases, and cardiovascular diseases, through solely controlled programs and collaborations with strategic partners.

Headquartered in Cambridge, Mass., privately held Moderna currently has strategic relationships with AstraZeneca, Plc. (AZ), Merck, Inc (MRK) and Vertex Pharmaceuticals (VRTX), as well as the Defense Advanced Research Projects Agency (DARPA), an agency of the U.S. Department of Defense; the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS); and the Bill & Melinda Gates Foundation. In 2017 Moderna was ranked a top biopharma industry employer by Science Magazine and a Top Places to Work by the Boston Globe . To learn more, visit www.modernatx.com .

Moderna Contacts

Investors:
Lorence Kim
617-209-5849
lorence.kim@modernatx.com

Media:
media@modernatx.com

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Dr. John Mendlein Joins Moderna as President, Corporate and Product Strategy

Wed, 03 Jan 2018 20:32:00 GMT

Biotechnology Industry Veteran to Lead Company’s Strategic Growth Initiatives and Help Drive Advancement of mRNA Drug Candidates

Cambridge, Mass., January 3, 2018 — Moderna Therapeutics, a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, today announced that John Mendlein, Ph.D., a current board member and the former CEO of multiple biotechnology companies, has joined Moderna as President, Corporate and Product Strategy. In this role, Dr. Mendlein will be responsible for corporate strategy, product advancement and strategy, partnering and product protection. He will serve on Moderna’s Executive Committee and report to Chief Executive Officer Stéphane Bancel. He will also continue to serve on Moderna’s Board of Directors where he has been a member since 2012.

“I have had the privilege of working closely with John for almost six years and I am delighted to now bring him into our company where he will contribute to our mission with a unique set of capabilities and experiences, including his scientific expertise and his insights from having run several successful biotech companies,” said Stéphane Bancel, Chief Executive Officer. “We will benefit from his deep product and platform experience ranging from platform development, to the successful transition of product candidates into the clinic, to product and platform protection. John will be a great addition to our executive team, and I am thankful that he decided to deploy his many talents during this important time in the evolution of our organization.”

“Moderna represents a rare and vast opportunity to change the lives of patients and medicine from vaccines to immuno-oncology to rare diseases to newly emerging areas of its mRNA product engine,” said Dr. Mendlein. “Over the past six years, under Stéphane’s leadership, I witnessed as a board member one of the most remarkable enterprise transformations as the team here took a captivating idea from our Chairman Dr. Noubar Afeyan, applied world-class science, and created an unrivaled product engine that generates high-impact product opportunities for patients at a scale and speed not previously achieved by our industry. The implications of Moderna’s ability to program life with mRNA are both profound and impactful. I feel very lucky and privileged to join the Moderna team at this exciting time and to work with Stéphane and the team to benefit patients and shareholders alike.”

Dr. Mendlein has helped start and lead numerous innovative life sciences companies. He is Vice Chairman of the Board and a founder of Fate Therapeutics, Inc., and holds board positions with Editas Medicine, Inc., and Axcella Health, Inc. He also serves on the Biotechnology Industry Organization (“BIO”) emerging companies board. Dr. Mendlein previously served as the Chief Executive Officer of aTyr Pharma, and Fate Therapeutics, as well as Adnexus Therapeutics, Inc., (acquired by BMS). Before that, he served as Chairman and Chief Executive Officer of Affinium Pharmaceuticals, Ltd. (acquired by Debiopharm Group), and as a board member, General Counsel and Chief Knowledge Officer at Aurora Bioscience Corporation (acquired by Vertex Pharmaceuticals).

Dr. Mendlein holds a Ph.D. in physiology and biophysics from the University of California, Los Angeles, a J.D. from the University of California, Hastings College of the Law, and a B.S. in biology from the University of Miami. Dr. Mendlein is the co-author or co-inventor of over 210 publications and published patents.

About Moderna Therapeutics

Moderna pioneers the discovery and development of messenger RNA (mRNA) therapeutics and vaccines, an entirely new class of medicines that directs the body’s cells to produce intracellular or secreted proteins that can have a therapeutic or preventive benefit for both patients and healthy individuals. With its breakthrough platform, Moderna is creating mRNA medicines for a wide range of diseases and conditions, in many cases by addressing currently undruggable targets or underserved areas of medical need. Moderna is developing its innovative mRNA medicines for infectious diseases, immuno-oncology, rare diseases, and cardiovascular diseases, through solely controlled programs and collaborations with strategic partners.

Headquartered in Cambridge, Mass., privately held Moderna currently has strategic relationships with AstraZeneca, Plc. (AZ), Merck, Inc (MRK) and Vertex Pharmaceuticals (VRTX), as well as the Defense Advanced Research Projects Agency (DARPA), an agency of the U.S. Department of Defense; the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS); and the Bill & Melinda Gates Foundation. In 2017 Moderna was ranked a top biopharma industry employer by Science Magazine and a Top Places to Work by the Boston Globe . To learn more, visit www.modernatx.com .

Moderna Contacts

Investors:
Lorence Kim
617-209-5849
lorence.kim@modernatx.com

Media:
media@modernatx.com

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Moderna to Provide Corporate and Pipeline Updates and Outline Strategic Priorities at 2018 J.P. Morgan Healthcare Conference

Tue, 02 Jan 2018 20:28:54 GMT

Cambridge, Mass., January 2, 2018 —Moderna Therapeutics, a clinical stage biotechnology company that is pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for high unmet medical needs in patients, announced today that CEO Stéphane Bancel will provide a detailed strategic update and share new advances in the company’s rapidly expanding pipeline of mRNA development candidates, at this year’s 36 ^th annual J.P. Morgan Healthcare Conference.

Mr. Bancel’s presentation will take place on Monday, January 8, 2018 at 2:30 p.m. PT in Elizabethan Room C/D at The Westin St. Francis Hotel in San Francisco.

About Moderna Therapeutics

Moderna pioneers the discovery and development of messenger RNA (mRNA) therapeutics and vaccines, an entirely new class of medicines that directs the body’s cells to produce intracellular or secreted proteins that can have a therapeutic or preventive benefit for both patients and healthy individuals. With its breakthrough platform, Moderna is creating mRNA medicines for a wide range of diseases and conditions, in many cases by addressing currently undruggable targets or underserved areas of medical need. Moderna is developing its innovative mRNA medicines for infectious diseases, immuno-oncology, rare diseases, and cardiovascular diseases, through solely controlled programs and collaborations with strategic partners.

Headquartered in Cambridge, Mass., privately held Moderna currently has strategic relationships with AstraZeneca, Plc. (AZ), Merck, Inc (MRK) and Vertex Pharmaceuticals (VRTX), as well as the Defense Advanced Research Projects Agency (DARPA), an agency of the U.S. Department of Defense; the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS); and the Bill & Melinda Gates Foundation. In 2017 Moderna was ranked a top biopharma industry employer by Science Magazine and a Top Places to Work by the Boston Globe . To learn more, visit www.modernatx.com .

Moderna Contacts

Investors:
Lorence Kim
617-209-5849
lorence.kim@modernatx.com

Media:
media@modernatx.com

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Moderna Publishes Preclinical Data in Cell Reports on mRNA Therapeutic to Treat Rare Liver Disease Methylmalonic Acidemia (MMA), A Disorder of Organic Acid Metabolism

Tue, 19 Dec 2017 17:50:16 GMT

Repeat systemic dosing enabled liver expression of functional hMUT enzyme, significantly improving survival and weight gain in MMA mice

No increased markers of liver toxicity or inflammation

Moderna continuing to advance MMA development candidate, mRNA-3704, toward clinical study

Cambridge, Mass., December 19, 2017 —Moderna Therapeutics, a clinical stage biotechnology company that is pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, today announced the publication of preclinical data supporting its first rare liver disease development program, an mRNA therapeutic for methylmalonic acidemia (MMA), a serious and often life-threatening organic acidemia.

The data, published online today in the journal Cell Reports, demonstrate that intravenous (IV) administration of an mRNA therapeutic encoding for human methylmalonyl-CoA mutase (hMUT), the enzyme most frequently mutated in MMA, enabled liver expression of MUT in MMA mouse models, leading to a significant reduction in methylmalonic acid, a substantial improvement in weight gain, and the complete survival of the full cohort of treated mice versus control group (see illustration below). Repeat IV dosing did not increase markers of liver toxicity or inflammation. The study was conducted in partnership with researchers in the Medical Genomics and Metabolic Genetics Branch of the National Human Genome Research Institute at the National Institutes of Health.

“Methylmalonic acidemia is a significant unmet need, and many children experience severe and often life-threatening metabolic crises due to a mutated (MUT) enzyme. The only effective treatment option today is liver and/or kidney transplantation. In contrast, our mRNA-based approach directly introduces into the body’s liver cells the genetic instructions they need to produce a functional MUT enzyme,” said Paolo Martini, Ph.D., Moderna’s Chief Scientific Officer, Rare Diseases, and the lead author on the publication. “These data give us an important early demonstration of our drug’s ability to instigate MUT protein expression from within liver cells, which lowers methylmalonic acid levels and significantly improves outcomes in a preclinical setting. They also show our capability to systemically repeat dosing to the liver with a favorable safety profile.”

Dr. Martini continued, “We are continuing to progress our MMA therapeutic development candidate, mRNA-3704, toward clinical study, and are driven by the acute and urgent need for an effective treatment option for these patients.”

About the Study Findings

In the study, intravenous (IV) administration of an mRNA therapeutic encoding for human methylmalonyl-CoA mutase (hMUT), the enzyme most frequently mutated in MMA, was evaluated in two mouse models of methylmalonic acidemia (MMA) developed by Dr. Charles P. Venditti, M.D., Ph.D., Senior Investigator, Medical Genomics and Metabolic Genetics Branch, and Head, Organic Acid Research Section, National Human Genome Research Institute, National Institutes of Health, and his team. Dr. Venditti is also a corresponding author on the paper. The models recapitulated the more severe mut° subtype, where there is no liver enzyme activity, or the partial deficiency type of MMA, mut-, where there is some liver enzyme activity. The hMUT mRNA utilized one of Moderna’s novel, proprietary delivery technologies, N2GL, which enables the delivery of mRNA drugs to hepatic cells and the ability to repeat dose. Both Moderna and NIH researchers conducted research for the study.

MUT plays an important role in metabolism. When MUT is deficient, toxic metabolites accumulate in the blood and urine, leading to a build-up of methylmalonic acid. This, in turn, causes severe and even deadly complications for patients, primarily children.

The researchers first evaluated a single IV bolus injection of hMUT mRNA. In both the mut° and mut-subtypes, a single IV bolus injection increased liver expression of a functional MUT enzyme, leading to a 75% and 85% reduction in plasma methylmalonic acid concentrations, respectively. The researchers observed acute metabolic correction after hMUT mRNA treatment, with plasma methylmalonic acid as well as methylmalonic acid in various tissues, including liver, heart, kidney, skeletal muscle and brain, acutely reduced.

The researchers also evaluated whether weekly IV injections of hMUT mRNA in the more severe mut° subtype could improve survival and correct biochemical and growth abnormalities that are a hallmark of the disorder. A cohort of mice receiving weekly IV injections of hMUT mRNA (n=7) for five weeks with an additional ten-day washout period were compared to cohorts of mice receiving control mRNA (n=6) or no treatment (phosphate buffered saline) (n=6). All of the mice treated with hMUT mRNA survived the entire duration of the study. In contrast, high mortality was observed in both the control and untreated cohorts, with only one control mouse surviving the entire duration of the study and no untreated mice surviving. Additionally, the treated mice thrived, with 40% more weight gain compared to the sole surviving mouse in the control group. Plasma methylmalonic acid levels in treated mice significantly decreased, by 62-89%, from base levels. The sole surviving mouse in the control group showed no metabolic response.

Repeat dosing of hMUT mRNA over the five-week treatment period did not increase markers of liver toxicity or inflammation and did not generate anti-drug antibodies against hMUT.

About Methylmalonic Acidemia and mRNA-3704

Methylmalonic acidemia, or MMA, is a rare, autosomal recessive organic acidemia/aciduria, most commonly (approximately 60% of cases) caused by a deficiency of the enzyme methylmalonic CoA mutase (MUT), due to a defective or missing MUT protein.

MMA is primarily a pediatric disease with onset in early infancy. The majority of patients with MMA have no functional MUT enzyme, which disrupts the metabolic pathway and leads to a build-up of toxic methylmalonic acid in the blood and urine. This acid accumulation causes, on average, three life-threatening metabolic crises per year. As a result, MMA is associated with significant mortality and morbidity, and there are no approved therapies. Standard of care includes dietary and palliative measures. Currently, liver and/or kidney transplant is the only effective treatment.

Moderna is developing an mRNA therapeutic, mRNA-3704, which directs cells in the liver to produce and express a functional MUT enzyme, restoring the metabolic pathway and reducing toxic acid build-up.

About Moderna Therapeutics

Headquartered in Cambridge, Mass., privately held Moderna currently has strategic agreements with AstraZeneca, Merck, and Vertex Pharmaceuticals, as well as the Defense Advanced Research Projects Agency (DARPA), an agency of the U.S. Department of Defense; the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS); and the Bill & Melinda Gates Foundation. To learn more, visit www.modernatx.com .

Moderna Contacts

Investors:
Lorence Kim
617-209-5849
lorence.kim@modernatx.com

Media:
media@modernatx.com

VIEW ALL NEWS

Moderna Announces First-in-Human Dosing for Phase 1 Study (KEYNOTE-603) of mRNA-4157, a Personalized Cancer Vaccine, for the Treatment of Solid Tumors

Wed, 15 Nov 2017 17:46:34 GMT

- Novel cancer vaccine encodes 20 neoepitopes on a single mRNA molecule to elicit a completely individualized immune response

-Moderna and Merck collaborating to evaluate mRNA-4157 in combination with KEYTRUDA® (pembrolizumab)

November 15, 2017; Cambridge, Mass. —Moderna Therapeutics, a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, announced today that it has started dosing patients in a Phase 1 study of mRNA-4157, an mRNA-based personalized cancer vaccine. The Phase 1 open-label, dose escalation, multicenter study in the United States (KEYNOTE-603) will assess the safety, tolerability and immunogenicity of mRNA-4157 alone in subjects with resected solid tumors and in combination with KEYTRUDA® (pembrolizumab), an anti-PD-1 therapy, marketed by Merck (known as MSD outside the U.S. and Canada) in subjects with unresectable solid tumors.

The first-in-human dosing of mRNA-4157 marks a key milestone in the strategic collaboration between Moderna and Merck to advance the novel mRNA-based personalized cancer vaccine in combination with KEYTRUDA for the treatment of multiple types of cancer.

“When we combine the potential for robust T-cell response stimulated by our mRNA vaccine, which encodes for 20 patient-specific mutations, with Merck’s checkpoint inhibitor, Keytruda, we have a unique opportunity to make a transformative difference for patients with cancer,” said Tal Zaks, M.D., Ph.D., Chief Medical Officer at Moderna. “Having now successfully designed, manufactured and dosed a completely customized personalized cancer vaccine, we look forward to progressing the Phase 1 clinical study and gathering important human data on mRNA-4157 in the months ahead.”

KEYNOTE-603 is expected to enroll up to 90 patients across multiple clinical study sites in the United States. Part A of the study will assess the safety, tolerability and immunogenicity of mRNA-4157 alone in subjects with resected solid tumors (in the adjuvant setting). Part B of the study will evaluate mRNA-4157 in combination with KEYTRUDA in subjects with unresectable solid tumors. The ClinicalTrials.gov Identifier for the mRNA-4157 study is: NCT03313778. A link to the ClinicalTrials.gov listing for the study can be found here .

“Our goal in this important collaboration is to deliver personalized vaccines to patients suffering from malignant disease, with the hope that this stimulus will generate a tumor-specific immune response in the presence of Keytruda, our approved immune-stimulatory cancer therapy,” said Roger M. Perlmutter, M.D., Ph.D., President, Merck Research Laboratories. “This trial leverages advances in genomics, advanced data analytics, and immunology to permit the generation of personalized cancer vaccines, a potentially transformative approach to cancer treatment. We are hopeful that this collaboration with Moderna, now entering clinical trials, will yield tangible benefits for cancer patients.”

“Checkpoint inhibitors and other immuno-oncology therapies are continuing to revolutionize how we treat cancer. However, despite the strong and durable responses we see in some patients, many other patients’ disease continues to progress,” said Howard A. “Skip” Burris III, MD, President, Clinical Operations & Chief Medical Officer at Sarah Cannon , and a Principal Investigator of the mRNA-4157 Phase 1 study. “An individualized medicine designed to help each patient’s immune system better recognize cancer as foreign and attack it would be a critical addition to oncologists’ treatment arsenal, potentially helping many more patients respond more effectively to treatment.”

About mRNA-4157

Moderna is creating an individualized, mRNA-based personalized cancer vaccine to deliver one medicine for one patient at a time. Through next-generation sequencing, Moderna identifies mutations found on a patient’s cancer cells, called neoepitopes. Neoepitopes can help the immune system distinguish cancer cells from normal cells. Using algorithms developed by its in-house bioinformatics team, Moderna predicts 20 neoepitopes present on the patient’s cancer that should elicit the strongest immune response, based on unique characteristics of the patient’s immune system and particular mutations. Moderna then creates a vaccine that encodes for each of these mutations and loads them onto a single mRNA molecule.

Once injected into the patient, the vaccine should direct the patient’s cells to express the selected neoepitopes. In turn, this may help the patient’s immune system better recognize cancer cells as foreign and destroy them. Leveraging its rapid cycle time, small-batch manufacturing technique and digital infrastructure, Moderna plans to manufacture and supply each individually manufactured personalized cancer vaccine to patients within weeks.

mRNA-4157 also has the potential to enhance clinical outcomes associated with checkpoint inhibitor therapies. In 2016, Moderna and Merck formed a collaboration to develop mRNA-4157 in combination with Merck’s anti-PD-1 therapy, KEYTRUDA.

About the Moderna and Merck Collaboration to Advance mRNA-4157

Under the terms of the agreement announced in June 2016, Merck made an upfront cash payment to Moderna of $200 million, which Moderna is using to lead all research and development efforts through proof of concept. The development program will entail multiple studies in several types of cancer and include the evaluation of mRNA-4157 in combination with KEYTRUDA. Moderna is also utilizing the upfront payment to fund a portion of the ongoing build-out of its GMP mRNA clinical manufacturing facility in Norwood, Mass., for the purposes of personalized cancer vaccine manufacturing.

Following human proof of concept studies, Merck has the right to elect to make an additional undisclosed payment to Moderna. If exercised, the two companies will then equally share cost and profits under a worldwide collaboration for the development of mRNA-4157. Moderna will have the right to elect to co-promote mRNA-4157 in the U.S. The agreement entails exclusivity around combinations with KEYTRUDA. Moderna and Merck will each have the ability to combine mRNA-4157 with other immuno-oncology (non-PD-1) agents.

About Moderna Therapeutics

Headquartered in Cambridge, Mass., privately held Moderna currently has strategic agreements with AstraZeneca, Merck and Vertex Pharmaceuticals, as well as the Defense Advanced Research Projects Agency (DARPA), an agency of the U.S. Department of Defense; the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS); and the Bill & Melinda Gates Foundation. To learn more, visit www.modernatx.com .

KEYTRUDA® is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, N.J., USA

Moderna Contacts

Media:
Liz Melone
617-674-5648
liz.melone@modernatx.com

Investors:
Lorence Kim
617-209-5849
lorence.kim@modernatx.com

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Moderna Announces New Collaboration with AstraZeneca to Co-Develop and Co-Commercialize Relaxin mRNA Therapeutic for Heart Failure

Wed, 01 Nov 2017 17:44:10 GMT

CAMBRIDGE, Mass., November, 1, 2017 — Moderna Therapeutics today announced a new license and collaboration with AstraZeneca to co-develop and co-commercialize a messenger RNA (mRNA) therapeutic encoding for Relaxin. The companies will advance the new Relaxin development candidate, AZD7970, toward the clinic as an investigational treatment for heart failure.

Heart failure occurs when the heart is weakened and cannot pump enough blood to meet the body’s needs. AZD7970 is being developed to instruct cells in the body to produce and express Relaxin, a secreted protein with systemic effect. Biologic functions for Relaxin suggest that expression of the hormone may directly impact underlying conditions that exacerbate heart failure, leading to the regrowth of heart tissue, controlling inflammation, reordering the extracellular matrix, improving renal function, and relieving hepatic portal pressure. AZD7970 utilizes one of Moderna’s proprietary formulations, N2GL, which enables systemic, repeat dosing of mRNA therapeutics.

“AstraZeneca has been a valued biopharma partner to Moderna since 2013 and is one of our largest shareholders. Together, we are excited to progress this Relaxin mRNA therapeutic toward the clinic, after significant efforts at Moderna that have advanced the program through discovery,” said Stéphane Bancel, Chief Executive Officer, Moderna. “We could not have chosen a more supportive and strategic partner than AstraZeneca to help advance transformative mRNA medicines for high unmet needs in the cardiovascular space.”

Approximately 5.7 million adults in the U.S. alone have heart failure. About half of those who develop heart failure die within five years of being diagnosed. A global health burden, heart failure impacts approximately 26 million people around the world.

“At AstraZeneca, we are committed to harnessing breakthrough science and technology to advance innovative treatments for heart failure, and as part of that commitment, we are pleased to enter into a new collaboration with Moderna to pursue a novel Relaxin programme,” said Menelas Pangalos, Executive Vice President of AstraZeneca’s Innovative Medicines and Early Development (IMED) Biotech Unit. “mRNA therapeutics have the potential to help address the unmet need and poor outcomes associated with heart failure and, ultimately, to become a transformational treatment for patients.”

Under the terms of the new agreement between the companies, Moderna will fund and be responsible for preclinical development of AZD7970, including the conduct of Good Laboratory Practice (GLP) toxicology studies. AstraZeneca will be responsible for early clinical development of AZD7970, and Moderna and AstraZeneca will share the costs of late-stage clinical development. Moderna and AstraZeneca will co-commercialize AZD7970 in the U.S. under a 50:50 profit sharing arrangement. AstraZeneca will lead ex-U.S. commercialization efforts, with Moderna receiving tiered royalties up to substantial double digits on ex-U.S. sales.

This marks the second strategic agreement between Moderna and AstraZeneca to advance mRNA therapeutics in the cardiometabolic space. The lead program from the first strategic agreement, which was announced in 2013, is AZD8601, a localized mRNA therapeutic that encodes for vascular endothelial growth factor (VEGF-A). AZD8601 has successfully completed a Phase 1 study, and AstraZeneca has submitted a Clinical Trial Application (CTA) in Europe to initiate a Phase 2a study of AZD8601 in heart failure patients undergoing cardiac bypass grafting (CABG) surgery.

About Moderna’s Pipeline

Moderna’s pipeline comprises 17 mRNA development candidates (DCs), including both internally developed and partnered programs across five modalities: prophylactic vaccines, therapeutic vaccines, intratumoral immuno-oncology therapeutics, localized therapeutics and liver therapeutics. Clinical trials are underway for seven of these DCs, with the remaining ten DCs advancing toward the clinic.

About Moderna Therapeutics

Moderna is a clinical stage pioneer of messenger RNA (mRNA) therapeutics and vaccines, an entirely new drug technology that directs the body’s cells to produce intracellular or secreted proteins. With its breakthrough platform, Moderna is developing mRNA vaccines and therapeutics as a new class of medicines for a wide range of diseases and conditions, in many cases by addressing currently undruggable targets. Moderna is developing its innovative mRNA medicines for infectious diseases, cancer (immuno-oncology), rare liver diseases, cardiovascular diseases and pulmonary diseases, through proprietary development and collaborations with strategic partners. Headquartered in Cambridge, Mass., privately held Moderna currently has strategic agreements with AstraZeneca, Merck and Vertex Pharmaceuticals, as well as the Defense Advanced Research Projects Agency (DARPA), an agency of the U.S. Department of Defense; the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS); and the Bill & Melinda Gates Foundation. To learn more, visit www.modernatx.com .

Moderna Contacts

Investors:
Lorence Kim, 617-209-5849
Lorence.kim@modernatx.com

Media:
Liz Melone, 617-256-6622
liz.melone@modernatx.com

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Moderna Named One of Global Biopharmaceutical Industry’s Top Employers in Science Magazine’s Annual Survey

Thu, 19 Oct 2017 17:33:03 GMT

Cambridge, Mass., Oct. 19, 2017 —Moderna Therapeutics, a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, today announced that it has been named one of the global biopharmaceutical industry’s top employers in Science’s and Science Careers’ 2017 Top Employers Survey . The annual survey polled employees in the biotechnology, biopharmaceutical, pharmaceutical, and related industries across the globe to determine the 20 best employers in these industries. This is the third consecutive year that Moderna has been named to this prestigious list, which includes some of the most notable companies in the industry. Moderna ranked sixth on the list this year.

“We are very proud to once again be named a top employer in the biopharma industry and be included among this prestigious group of companies,” said Stéphane Bancel, CEO at Moderna. “We have made significant strides this year to advance our mission to deliver on the promise of mRNA science for patients. This progress is all due to the contributions and commitment of our remarkable team and their unparalleled ability to be bold, creative, agile and undaunted. We continue to attract world-class talent to Moderna, and we take very seriously our responsibility to create an environment where our employees can develop and grow in their careers and contribute meaningfully every day. We’ll continue to push the boundaries of innovation while striving to be an exemplary employer and a model corporate citizen.”

Moderna’s medicines use mRNA, a fundamental component of human biology, to direct cells in the body to make and express proteins to prevent and fight disease. The company’s development pipeline currently consists of 16 mRNA medicines across several therapeutic areas including infectious diseases, cancer/immuno-oncology, cardiovascular disease and rare liver diseases. Clinical studies are underway for seven of these development candidates, with the remaining nine advancing toward the clinic. In addition, Moderna is progressing multiple research programs toward development.

Science and Science Careers’ web-based survey includes results from 6,950 responses from readers of Science, as well as other survey invitees. The survey took place in the spring of 2017. Survey participants were located in North America, Europe and Asia/Pacific Rim, with 94 percent working in biotechnology, biopharmaceutical and pharmaceutical companies. Rankings were determined by an independent research firm that analyzed survey responses rating companies based on various characteristics including a research-driven environment, easy adaptation to change and financial strength.

The complete feature can be found at www.sciencecareers.org/TopEmployers2017 . The rankings also will appear in the October 20, 2017, print issue of Science.

About Moderna Therapeutics

Moderna Contacts

Media:
Liz Melone
617-674-5648
liz.melone@modernatx.com

Investors:
Lorence Kim
617-209-5849
lorence.kim@modernatx.com

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Stephen Berenson Joins Moderna’s Board of Directors

Tue, 10 Oct 2017 17:30:52 GMT

Flagship Pioneering Executive Partner and Former J.P. Morgan Vice Chairman of Investment Banking Brings Key Expertise to Moderna’s Board

Cambridge, Mass., October 10, 2017— Moderna Therapeutics, a clinical stage biotechnology company that is pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, announced today that Stephen Berenson has been appointed to the company’s Board of Directors, and will also serve on the company’s Audit Committee. Mr. Berenson joined Flagship Pioneering as Executive Partner in June of this year. He previously served for twelve years as Vice Chairman of Investment Banking at J.P. Morgan, where he worked for 33 years.

“We continue to assemble a Board that is particularly suited to help guide Moderna through its fast-paced growth and bright future as a high-tech enterprise advancing mRNA medicines,” said Noubar Afeyan, Ph.D., Co-Founder and Chairman, Moderna Therapeutics, and Chief Executive Officer of Flagship Pioneering. “The insights Stephen gained during his long tenure at J.P. Morgan, one of the world’s leading financial institutions, will be invaluable to Moderna as we continue to develop the company’s long-term strategy and work to address unmet needs across a spectrum of therapeutic areas.”

“I have had the opportunity to get to know Moderna well over the last six months, including as a guest to its Board,” said Mr. Berenson. “In my more than thirty years as an investment banker, I gained a strong understanding of the characteristics of market-leading organizations. Not only does Moderna embody all those characteristics, but the scope of the human health problems it aims to address and the scale of its business opportunity make it one of the most interesting companies I’ve ever had the opportunity to work with. I’ve been tremendously impressed with how methodically Stephane and the team are executing on the vision they set six years ago, and am honored to serve on its Board of Directors.”

Moderna’s development pipeline now consists of 16 mRNA medicines across infectious diseases, cancer/immuno-oncology, cardiovascular disease and rare liver diseases. Clinical studies are underway for seven of those development candidates, with the remaining nine advancing toward the clinic. Moderna continues to advance several programs through research and toward development.

“Having spent time with Stephen over the past year and, of course, knowing his reputation as one of investment banking’s most well-respected leaders, I greatly appreciate and deeply value his strategic skills and his vast experience financing world-class companies, especially in the technology space,” said Stéphane Bancel, Chief Executive Officer at Moderna. “I am thrilled to welcome Stephen to our Board. He will be a wonderful addition to our team. I look forward to working closely with him as we build this unique company and maximize the impact we can have for patients through our mRNA medicines.”

As Executive Partner at Flagship, Mr. Berenson serves on Flagship's Investment and Management Committees. He is focused on capital formation at the fund and portfolio company levels and assists the firm's management teams in growing and realizing value. Mr. Berenson is also responsible for driving strategic and operational improvements across Flagship and its 35 companies, helping to deepen relationships with critical external partners and serve on the Boards of Directors of select portfolio companies.

Prior to joining Flagship, Mr. Berenson spent more than 33 years with J.P. Morgan as an investment banker, where he worked across all major geographies, product areas and industry groups. Mr. Berenson played key roles in building J.P. Morgan's M&A, equities and technology investment banking businesses. During his last twelve years at J.P. Morgan, he was vice chairman of investment banking and focused on providing high-touch strategic advice and complex transaction execution to leading companies across all industries globally. He was co-founder of J.P. Morgan's Global Strategic Advisory Council and co-founder of the firm's Board Initiative. During Mr. Berenson's leadership of the Board Initiative, more than 1,000 independent directors of public companies attended at least one of J.P. Morgan's board events to discuss, debate and share best practices on the key issues they faced in the fulfillment of their duties.

Mr. Berenson contributed significantly to J.P. Morgan's culture through his deep support of recruiting, training and development, as well as mentoring. In addition, he helped to build and maintain a robust control environment through his participation on the firm's Equity Underwriting and Reputational Risk Committees.

Mr. Berenson holds an S.B. in mathematics from the Massachusetts Institute of Technology. He is a member of the Board of Trustees of the Mahaiwe Performing Arts Center in Great Barrington, Massachusetts.

About Moderna Therapeutics

Moderna Contacts

Media:
Liz Melone
617-674-5648
liz.melone@modernatx.com

Investors:
Lorence Kim
617-209-5849
lorence.kim@modernatx.com

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Moderna Announces Publication in Molecular Therapy Characterizing Potent Immune Response Generated by Its mRNA Prophylactic Vaccines

Tue, 26 Sep 2017 17:28:37 GMT

Research led by Karolinska Institutet describes how Moderna’s vaccines target key antigen-presenting cells, leading to both B cell and T cell responses

Cambridge, Mass., September 26, 2017— Moderna Therapeutics, a clinical stage biotechnology company that is pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, announced a new publication in Molecular Therapy that provides mechanistic insights about its mRNA prophylactic vaccines. The research, led by Professor Karin Loré, Ph.D., and her group at the Karolinska Institutet in Stockholm, Sweden, characterizes how Moderna’s vaccines target key antigen-presenting cells, leading to both B cell and T cell activation, which yields a potent immune response.

The study utilized a research version of Moderna’s influenza H10N8 vaccine, which encodes for the viral antigenic protein hemagglutinin (HA) encapsulated in lipid nanoparticles (LNPs). In the study, the H10N8 vaccine induced protective titers of HA antibody, as well as CD4+ T cell responses, after intramuscular or intradermal injection into non-human primates (NHPs). Two types of cells, monocytes at the injection site, and dendritic cells in the draining lymph nodes, primarily expressed and presented HA to the immune system and upregulated key co-stimulatory receptors, CD80 and CD86. The vaccine also led to the upregulation of type 1 interferon (IFN)-inducible genes, including MXI and CXCL10. This innate immune activation resulted in the priming of H10-specific CD4+ T cells in the lymph nodes. The vaccine’s ability to induce both innate immunity and antigen production by antigen-presenting cells generates a potent vaccine-specific immune response.

“There is a growing body of data demonstrating the robust immune response generated by mRNA vaccines. We designed this study to shed light on the mechanistic underpinnings of this novel and hugely promising vaccine technology,” said Professor Karin Loré, Ph.D., principal investigator and corresponding author on the paper. “These data increase our understanding of how mRNA vaccines target key antigen-presenting cells to generate vaccine-specific T cell and B cell responses. We now know which cells are translating the mRNA, and we understand the cellular activation of target cells. We look forward to seeing the continued progress of mRNA vaccines in the clinic as a novel approach for addressing persistent and emerging unmet needs in the infectious disease space.”

A Phase 1 clinical study of Moderna’s H10N8 vaccine (mRNA-1440) in healthy volunteers has been completed, with subjects continuing to be followed for safety monitoring. In April 2017, Moderna published positive interim data from a 100 µg intramuscular (IM) cohort from the Phase 1 study demonstrating mRNA-1440 induced high levels of immunogenicity, and was safe and well tolerated.

“We are very pleased to have collaborated with Dr. Loré and her highly respected immunology research team at Karolinska on this important study. As pioneers of mRNA vaccines, it’s incumbent upon us to advance a deeper understanding of the fundamental mechanisms by which they are working to generate immunity and prevent infectious diseases,” said Giuseppe Ciaramella, Ph.D., Chief Scientific Officer, Infectious Diseases at Moderna, and a senior author on the paper. “Given the software-like nature of our medicines, these findings should translate across our vaccine platform. We look forward to publishing additional mechanism of action insights as we continue to advance our ambitious pipeline of prophylactic vaccines.”

Moderna’s development pipeline currently comprises 16 development candidates (DCs) across infectious diseases, immuno-oncology, cardiovascular diseases and rare liver diseases. Nine of these DCs are prophylactic vaccines – including monovalent, multivalent and multi-pathogen vaccines. There are Phase 1 studies currently underway for five of these vaccines.

About Moderna’s mRNA Prophylactic Vaccines

About Moderna Therapeutics

Moderna Contacts

Media:
Liz Melone
617-256-6622
liz.melone@modernatx.com

Investors:
Lorence Kim
617-209-5849
lorence.kim@modernatx.com

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Moderna Announces Pipeline and Corporate Update

Thu, 14 Sep 2017 17:26:08 GMT

Phase 1 studies underway for seven mRNA medicines

Moderna achieves key milestones advancing mRNA therapeutics for cardiovascular diseases, cancer and rare liver diseases

Pipeline comprises 16 development candidates, including first therapeutic for rare liver disease methylmalonic acidemia (MMA)

Clinical Study Highlights:

Successful completion of AstraZeneca’s Phase 1 study of first mRNA therapeutic, mRNA AZD-8601 (VEGF-A); Phase 2a study preparations underway
Initiation of Phase 1 study of mRNA-2416, Moderna’s first immuno-oncology therapeutic, targeting intratumoral expression of membrane-bound OX40L
Initiation of DARPA-supported Phase 1 study of mRNA-1388, a Chikungunya vaccine; fifth prophylactic vaccine to enter clinical study

New Preclinical Development Candidate Nominations:

mRNA-3704, first therapeutic for severe, life-threatening rare liver disease, methylmalonic acidemia (MMA)
mRNA-5671, a cancer vaccine for the treatment of KRAS-mutated cancers of the lung, colorectum and pancreas
mRNA-2752, an intratumoral immuno-oncology triple combination mRNA encoding for OX40L+IL23+IL36γ
mRNA MRK-1278, second prophylactic vaccine under Merck collaboration
mRNA-1893 replaces mRNA-1706 as backup Zika vaccine

CAMBRIDGE, Mass., September 14, 2017 — Moderna Therapeutics, a clinical stage biotechnology company that is pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, provided an update today, noting continued progress of clinical programs and the introduction of several new development candidates (DCs) across its broad, diverse development pipeline. Moderna announced the update in conjunction with an Investor R&D Day hosted by its management team this morning in New York.

A Phase 1 study of mRNA AZD-8601, the first-ever mRNA therapeutic to be evaluated in a clinical study, has been successfully completed. mRNA AZD-8601 is anticipated to move into Phase 2 development. mRNA AZD-8601 is being developed by Moderna’s partner AstraZeneca to express a local and transient surge of vascular endothelial growth factor-A (VEGF-A) as a potential treatment for cardiovascular diseases. The Phase 1 randomized, double-blind, placebo-controlled, single ascending dose study, which assessed the safety, tolerability and pharmacokinetics (PK) of mRNA-AZD-8601 after single dose administration in male patients with Type 2 diabetes mellitus was successfully completed. The study met its primary endpoint of safety and tolerability and also demonstrated proof of mechanism as measured by expression of VEGF-A protein in the skin (protein PK).

AstraZeneca has submitted a Clinical Trial Application (CTA) in Europe to initiate a Phase 2a study of mRNA AZD-8601 in heart failure patients undergoing coronary artery bypass grafting (CABG) surgery.

Moderna initiated a Phase 1 study of mRNA-2416, an intratumoral (iTu) immuno-oncology (I-O) therapeutic that encodes for the membrane expression of the co-stimulatory protein OX40 Ligand, or OX40L, to potentially enhance T-cell attack against tumors. This is Moderna’s first I-O therapeutic to enter clinical study.

Moderna also unveiled its first clinical development candidate (DC) that utilizes a novel modality, liver expression of therapeutic proteins. Utilizing this modality, the company will advance toward the clinic an mRNA DC, mRNA-3704, for methylmalonic acidemia (MMA), a serious and often life-threatening rare liver disease for which there are no approved therapies.

In total, Moderna announced the addition of four new DCs to its development pipeline today. The company also announced that it has replaced its preclinical stage Zika vaccine DC, mRNA-1706, with mRNA-1893, a Zika vaccine with an enhanced mRNA construct anticipated to augment immunogenicity. Moderna’s development pipeline now comprises 16 mRNA therapeutics and vaccines spanning infectious diseases, cancer (I-O), cardiovascular diseases and rare liver diseases. Approximately 460 subjects have been dosed to date across clinical studies for seven mRNA vaccines and therapeutics.

“2017 is a major inflection point for Moderna, as we’ve made significant progress advancing mRNA therapeutics for unmet needs across several disease areas,” said Stéphane Bancel, Chief Executive Officer of Moderna. “In the cardiovascular space, our partner AstraZeneca successfully completed a Phase 1 study for mRNA-AZD-8601, a VEGF-A therapeutic, and planning is underway for a Phase 2a study. In addition, we have initiated first-in-human dosing in a Phase 1 study of mRNA-2416, our first immuno-oncology candidate, encoding OX40 ligand. We also have progressed the expression of therapeutic proteins in the liver as a development stage modality. As such, we are now able to advance chronically dosed mRNA therapeutics to development for rare liver diseases. I’m very thankful to the Moderna team for their commitment, dedication and continued progress to deliver on our mission.”

Mr. Bancel added, “This past May we experienced the extremely sad, untimely loss of Henri Termeer, a Moderna board member, and a dear friend and mentor to so many of us at Moderna and across our industry. Henri was tirelessly dedicated to helping patients, particularly those impacted by rare diseases. We hope to honor his remarkable legacy by delivering on the promise of mRNA science for patients.”

Clinical and Development Program Updates

mRNA is a fundamental component of human biology, giving cells the instructions they need to make proteins that carry out every function of the body. Moderna is using mRNA as a drug to direct cells in the body to produce proteins to fight or prevent disease. Moderna combines elements of its mRNA platform into distinct approaches, called modalities, to address diseases. A modality is a technological solution set that Moderna can deploy to create a family of medicines for different diseases.

Moderna’s pipeline now comprises 16 mRNA DCs across five modalities: prophylactic vaccines, therapeutic vaccines, intratumoral immuno-oncology therapeutics, localized therapeutics and liver therapeutics. Moderna also has an mRNA lung therapeutics modality that is currently in the research stage, in which Vertex and Moderna are researching potential mRNA therapeutics for cystic fibrosis.

“We are very pleased with the progress our team has made advancing our proprietary, next generation formulations, including N2GL,” said Stephen Hoge, President of Moderna. “Based on our preclinical data we believe these represent a dramatic step change in performance, which we will submit for publication in the coming months. Combining formulations like N2GL with other platform improvements has allowed us to progress mRNA medicines for devastating rare metabolic diseases into development, the first of which is methylmalonic acidemia, or MMA.”

mRNA Prophylactic Vaccines Modality

Prophylactic Vaccines — Clinical Development Updates

Enrollment completed for Phase 1 studies of mRNA-1440 (influenza A virus subtype H10N8 vaccine) and mRNA-1851 (influenza A virus subtype H7N9 vaccine): The ongoing Phase 1 studies for mRNA-1440 (H10N8 mRNA vaccine) and mRNA-1851 (H7N9 mRNA vaccine) have completed enrollment, with 201 and 156 healthy volunteers enrolled, respectively. Both studies remain active, with subjects continuing to be followed for safety monitoring.
Moderna published interim data from the mRNA-1440 Phase 1 study in April 2017 in the journal Molecular Therapy, and plans to publish complete findings from both the mRNA-1440 and mRNA-1851 Phase 1 studies in 2018.
As previously described , these first two programs were strategically selected with the goal of quickly assessing both the safety and efficacy of Moderna’s mRNA vaccine platform in humans. H10N8 and H7N9 both represent influenza strains with pandemic potential. Currently, there is no approved vaccine for either strain of the virus.
Phase 1 study of mRNA MRK-1777 continues to progress: A Phase 1 randomized, placebo-controlled, dose-ranging study of mRNA MRK-1777, an mRNA prophylactic vaccine program for an undisclosed target, continues to enroll healthy volunteers. mRNA MRK-1777 is the first named DC under the existing collaboration between Merck and Moderna to discover and develop mRNA-based vaccines against viral diseases. Moderna is conducting the Phase 1 study of mRNA MRK-1777.
Phase 1 study of Chikungunya vaccine, mRNA-1388, initiated: In August, Moderna began enrolling healthy volunteers in the US for a Phase 1 study of mRNA-1388, an mRNA Chikungunya prophylactic vaccine. The randomized, placebo-controlled, dose-ranging study will evaluate the safety and immunogenicity of mRNA-1388 in healthy adults.
Chikungunya typically causes mild fever and transient joint pain. In approximately 15 percent of infected patients, it can cause long-term, severe arthritis. Chikungunya historically has been limited to warmer climates in Asia and Africa, but recent cases have been identified in the Americas and Europe. Currently, there is no approved vaccine for Chikungunya.
Clinical development of mRNA-1388 is supported by an award from the Defense Advanced Research Projects Agency ( DARPA ), an agency of the U.S. Department of Defense.
Phase 1/2 study of Zika mRNA vaccine, mRNA-1325, continues to progress: Moderna continues to enroll healthy subjects in a Phase 1/2 study of mRNA-1325, a Zika mRNA vaccine. The randomized, placebo-controlled, dose-ranging study is underway in the US.
Children born to mothers infected with Zika can develop microcephaly, a severe disease characterized by abnormally small heads and severe neurologic disabilities. Zika infection is also strongly associated with Guillain-Barré Syndrome (GBS), an autoimmune disease that attacks the peripheral nervous system, leading to rapidly progressive and potentially life-threatening muscle weakness. GBS can lead to death caused by respiratory arrest if a patient is not ventilated. Currently, there are no treatment options or approved vaccines for the Zika virus or congenital Zika syndrome.
Moderna received a funding award of up to $125 million from the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS), to accelerate development of its Zika mRNA vaccine.

Prophylactic Vaccines — Preclinical Development Updates

Additional mRNA prophylactic vaccine DCs continue to advance: Moderna continued to make progress advancing three additional mRNA prophylactic vaccine DCs toward the clinic, all of which utilize one of the company’s proprietary, novel formulations, V1GL:

mRNA-1893, a Zika mRNA vaccine, replaced mRNA-1706, a Zika mRNA vaccine previously announced by Moderna. mRNA-1706 contained the same active pharmaceutical ingredient (API) as Moderna’s mRNA-1325 Zika mRNA vaccine, but utilized Moderna’s V1GL formulation. Based on experience with other mRNA-encoded antigens, the leader sequence of the Zika antigen in mRNA-1325, which encodes a signal peptide that instructs the cell to secrete the protein, was from an Immunoglobulin E, or IgE, protein (a type of antibody).
In a paper published in Cell on Moderna’s Zika mRNA vaccine, researchers found that using a leader sequence from the Japanese encephalitis virus (JEV) led to improved immunogenicity and superior protection versus IgE in viral challenge models.
Following further investigation, Moderna has decided to replace mRNA-1706 with a new DC, mRNA-1893, that uses the JEV signal peptide. mRNA-1893 also utilizes Moderna’s V1GL formulation. While GLP toxicology studies had been successfully completed for mRNA-1706, given mRNA-1893 has a unique API, Moderna will now proceed with conducting GLP toxicology studies on this new, enhanced Zika mRNA vaccine.
mRNA-1647, a cytomegalovirus (CMV) mRNA vaccine , which consists of six mRNAs, including five proteins (gH, gL, UL128, UL130 and UL131A) designed to express the Pentamer complex, and another CMV antigen, the herpesvirus glycoprotein (gB) protein. GLP toxicology studies have been successfully completed for mRNA-1647.
CMV is the most common cause of newborn disability, leading to deafness, microcephaly (small, not fully developed heads and severe disabilities), vision loss and mental deficiencies, among other serious complications. It is also the most frequent viral disease in transplant recipients, often leading to transplant failure. Currently, there is no approved vaccine for CMV.
mRNA-1653, a combination human metapneumovirus (HMPV) and parainfluenza virus (PIV3) vaccine. GLP toxicology studies have been successfully completed for mRNA-1653. HMPV and PIV3 typically cause mild respiratory illness, but can become severe in young children, the elderly and other immunocompromised adults. HMPV and PIV3 are the second and third most common causes, respectively, of lower respiratory hospitalizations in children, behind RSV. Currently, there is no approved vaccine for either HMPV or PIV3.

Second Merck-partnered infectious disease mRNA vaccine DC named, mRNA MRK-V213 : Merck named its second DC, mRNA MRK-V213, under its existing collaboration with Moderna to discover and develop mRNA-based vaccines against viral diseases. mRNA MRK-V213 is for an undisclosed infectious disease indication.

Prophylactic Vaccines Publications

First human proof-of-concept data published in Molecular Therapy: In April 2017, Moderna published in the journal Molecular Therapy positive interim data from a 100 µg intramuscular (IM) cohort from its ongoing Phase 1 study of mRNA-1440, an influenza A virus subtype H10N8 mRNA prophylactic vaccine. 31 subjects were enrolled in the cohort, 23 of whom received mRNA-1440 and 8 of whom received placebo. mRNA-1440 demonstrated strong efficacy based Hemagglutination Inhibition Assay (HAI) and Microneutralization Assay (MN) titers, two measures of immunogenicity in response to vaccination, and was safe and well-tolerated. 100% (n = 23) and 87% (n = 20) achieved titers consistent with protection at day 43 as measured by HAI titers and MN titers, respectively, compared to 0% in the placebo arm.
These were the first-ever published data demonstrating an mRNA vaccine’s ability to elicit robust prophylactic immunity in humans, as well as the first human proof-of-concept data from Moderna’s mRNA platform.
First Zika mRNA vaccine preclinical data published in Cell: In February, the first pre-clinical data for Moderna’s Zika mRNA vaccine were published in Cell. The data demonstrated that an mRNA vaccine encoding for Zika prM-E protected against Zika virus in three different mouse strains after two doses: prime plus boost. Extraordinarily high titers of neutralizing antibodies were produced, achieving sterilizing immunity. In addition, a fusion loop mutant vaccine reduced production of potentially disease-enhancing anti-Dengue antibodies. The research was led by Moderna scientists and researchers at the Washington University School of Medicine.
Preclinical Data Showing Zika mRNA Vaccine Prevents In Utero Transmission Published in Cell: In July, data were published in Cell demonstrating that Moderna’s Zika mRNA vaccine prevented Zika virus transmission from pregnant mice to their fetuses. The findings also demonstrated that Moderna’s Zika mRNA vaccine protected the placenta and fetus from Zika virus-induced injury. In the study, Moderna’s Zika mRNA vaccine was evaluated in addition to a live-attenuated vaccine candidate developed by the University of Texas Medical Branch (UTMB). The research was conducted by scientists from the National Institute of Allergy and Infectious Diseases (NIAID) , part of the National Institutes of Health (NIH), Washington University School of Medicine and UTMB.

mRNA Therapeutic Vaccines Modality

Moderna’s mRNA therapeutic vaccines utilize one of the company’s proprietary formulations, V1GL.

Therapeutic Vaccines — Clinical Development Updates

IND for mRNA-based personalized cancer vaccine (PCV), mRNA-4157, filed and deemed safe to proceed to clinic: Moderna filed an IND with the FDA for mRNA-4157, its mRNA-based PCV, and has received a safe to proceed notification from the FDA.
Moderna, under its existing collaboration with Merck to develop personalized cancer vaccines (PCVs), will evaluate mRNA-4157 in combination with Merck’s anti-PD-1 therapy, KEYTRUDA® (pembrolizumab). Under the IND, Moderna will identify neoepitopes present in a patient’s tumor and then create an mRNA-based PCV encoding for approximately 20 neoepitopes. When injected into the patient, the mRNA-based PCV will direct the patient’s cells to express the selected neoepitopes. In turn, this may help the patient’s immune system better recognize cancer cells as foreign and eradicate them. mRNA-4157 also has the potential to enhance clinical outcomes associated with checkpoint inhibitor therapies. Leveraging its rapid cycle time, small-batch manufacturing technique and digital infrastructure, Moderna plans to manufacture and supply each individually tailored and manufactured PCV to patients within weeks.
National Cancer Institute (NCI) to sponsor and conduct study of mRNA-based PCV: In collaboration with Moderna, the Surgery Branch of the NCI’s Center for Cancer Research will sponsor and conduct a Phase 1/2 study to investigate the safety and immunogenicity of mRNA-based PCVs for patients with advanced-stage, metastatic cancers under the direction of Steven A. Rosenberg, M.D., Ph.D., Chief of Surgery, NCI. NCI will supply Moderna with amino acid sequences of confirmed and predicted neoepitopes for up to 12 patients. Based on these sequences, Moderna will manufacture an mRNA-based personalized cancer vaccine (NCI-4650) for each patient.

Therapeutic Vaccines — Preclinical Development Updates

New KRAS cancer vaccine DC named, mRNA-5671: Moderna has named a novel cancer vaccine DC that encodes for KRAS epitopes. KRAS is one of the most frequently mutated oncogenes in human cancer (approximately 30% of all cases). KRAS mutations are found principally in non-small cell lung cancer (NSCLC), colorectal cancer and pancreatic cancer, and are associated with worse outcomes. Hotspots of KRAS mutations are found in different tumor types and can serve as tumor rejection epitopes. Presentation of these epitopes to the immune system may elicit a robust anti-tumor response.
For this vaccine, Moderna plans to include an mRNA that encodes for the four most commonly found KRAS mutations, which will cover most of the mutations that occur in NSCLC, colorectal cancer and pancreatic cancer. Moderna has successfully completed GLP toxicology studies for mRNA-5671.

mRNA Intratumoral (iTu) Immuno-Oncology (I-O) Modality

Moderna’s intratumoral (iTu) immuno-oncology (I-O) therapeutics utilize one of the company’s proprietary formulations, N1GL.

iTu I-O Therapeutics — Clinical Development Updates

Phase 1 study of mRNA-2416 (encoding for OX40 Ligand, or OX40L) initiated: In August, Moderna began dosing for a Phase 1 study of mRNA-2416, an iTu I-O therapeutic targeting OX40L. This marks the first I-O program from Moderna’s pipeline to move into clinical study.
The Phase 1 open-label, multicenter, dose escalation study of mRNA-2416 is designed to determine the safety and tolerability of escalating iTu doses of mRNA-2416 in patients with relapsed/refractory solid tumor malignancies or lymphoma, and define the maximum tolerated dose (MTD) and recommended dose for expansion (RDE) and schedule for iTu injections of mRNA-2416. The study will enroll patients up to 10 sites in the US.
OX40L is a powerful co-stimulatory protein that enhances the expansion, function and survival of T cells to mount an attack against cancer cells. When mRNA-2416 is delivered directly into a tumor, cells in the tumor express the OX40L protein as a membrane-bound homotrimer on their surface, which, in turn, may lead to a stronger T cell attack against the tumor. mRNA-2416 may elicit an abscopal effect in metastatic cancer, in which localized injection into one tumor would lead not only to shrinking of that tumor but also shrinking of tumors elsewhere in the body.

iTu I-O Therapeutics — Preclinical Development Updates

iTu triple combination I-O therapeutic, mRNA-2752, nominated: mRNA-2752 is a triple combination I-O therapeutic that leverages the multi-target activation enabled by Moderna’s mRNA technology to enhance immune response against tumors. Specifically, mRNA-2752 comprises three mRNAs that express OX40L, Interleukin 23 (IL23) and Interleukin 36 γ (IL36γ). OX40L is a powerful co-stimulatory protein that enhances the expansion, function and survival of T cells to mount an attack against cancer cells. IL23 and IL36γ are secreted proteins that have established roles in mediating immune responses and have been implicated in driving various inflammatory diseases.
The three mRNAs are co-formulated and injected directly into a tumor. iTu delivery may enable delivery of targets locally that are too toxic systemically. mRNA-2752 may also elicit an abscopal effect in metastatic cancer. In addition, based on pre-clinical models, Moderna anticipates synergies combining mRNA-2752 with checkpoint inhibitors.
In April, Moderna gave oral and poster presentations at the American Association for Cancer Research (AACR) Annual Meeting in Washington, D.C., demonstrating that the triple combination of OX40L, IL23 and IL36γ and had potent and durable anti-tumor activity in multiple preclinical models.
GLP toxicology studies for mRNA IL12 iTu I-O, mRNA-2095, ongoing: GLP toxicology studies continue for mRNA-2905, an iTu I-O mRNA therapeutic that encodes for IL12, a powerful cytokine that activates the immune system after being released from cells. When mRNA-2905, a partnered program with AstraZeneca, is delivered directly into a tumor, cells in the tumor express IL12 at a high concentration in the local microenvironment, which, in turn, may lead to a stronger T cell attack against the tumor. By expressing IL12 locally, systemic side effects that previously have been seen from delivery of the IL12 protein into the blood may be more manageable. Moderna is also investigating the potential of mRNA-2905 to elicit an abscopal effect in metastatic cancer. Combining mRNA-2905 with a checkpoint inhibitor may improve outcomes from cancer therapy.
mRNA-2905 is being developed through a collaboration Moderna announced in 2016 with AstraZeneca to discover, co-develop and co-commercialize immuno-oncology mRNA therapeutics.

mRNA Localized Therapeutics Modality

Localized Therapeutics — Clinical Development Update

Phase 1 study of mRNA AZD-8601 completed enrollment; CTA filed for Phase 2a Study: A Phase 1 study for mRNA AZD-8601, an mRNA localized therapeutic being developed by Moderna’s partner AstraZeneca that encodes for vascular endothelial growth factor-A (VEGF-A), has been successfully completed. The Phase 1 randomized, double-blind, placebo-controlled, single ascending dose study assessed the safety, tolerability and pharmacokinetics (PK) of mRNA AZD-8601 after single dose administration in male patients with Type 2 diabetes mellitus. The study met its endpoint of safety and tolerability and also demonstrated proof of mechanism as measured by expression of VEGF-A protein in the skin (protein PK).
When directed via local tissue injection, VEGF-A mRNA may potentially lead to the creation of more blood vessels and improved blood supply. mRNA AZD-8601 could one day provide a unique regenerative treatment option for patients with heart failure or after a heart attack, as well as for diabetic wound healing and other ischemic vascular diseases.
AstraZeneca has submitted a Clinical Trial Application (CTA) in Europe to initiate a Phase 2a study of mRNA AZD-8601 in heart failure patients undergoing coronary artery bypass grafting (CABG) surgery.

“The potential impact of harnessing mRNA as a therapeutic to address human disease has long been understood, but the possibility of translating this potential into reality had remained elusive given the complexities of using mRNA as a drug,” said Tal Zaks, Chief Medical Officer at Moderna. “We’ve reached an important milestone with the successful completion of the Phase 1 study of mRNA-AZD-8601. And the Phase 2a study of mRNA-AZD-8601 in patients undergoing coronary artery bypass grafting surgery will provide further insight into the potential of VEGF-A mRNA to provide a unique regenerative treatment option for patients with cardiovascular disease. Seeing the first mRNA therapeutic program move to Phase 2 study will be an important step for the entire mRNA field.”

mRNA Liver Therapeutics Modality

Moderna’s liver therapeutics utilize one of the company’s proprietary formulations, N2GL.

New liver therapeutics modality advanced from research to development stage: Moderna advanced liver therapeutics from a research modality to a development modality through technology advancements that are believed to enable intravenous (IV) chronic dosing of mRNA therapeutics that will express protein in the liver. This may enable Moderna to address many serious diseases that are undruggable with existing drug development approaches. This is Moderna’s fifth modality to advance to the development stage.
Many diseases, including many rare genetic diseases, are caused by defects or deficits in proteins expressed by liver cells. By delivering mRNA drugs via IV to the liver, and directing liver cells to express functional proteins, Moderna can potentially stimulate production of therapeutic proteins in ways that cannot be achieved with other current technologies.

Liver Therapeutics — Preclinical Development Updates

First liver therapeutic DC nominated, mRNA-3704 for methylmalonic acidemia (MMA), a rare liver disease: MMA is a rare, autosomal recessive organic acidemia/aciduria, most commonly (approximately 60% of cases) caused by a deficiency of the enzyme methylmalonic CoA mutase, or MUT, due to a defective or missing MUT gene.
MMA is primarily a pediatric disease with onset in early infancy. The majority of patients with MMA have no functional MUT enzyme, leading to, on average, three life-threatening metabolic crises per year. As a result, MMA is associated with significant mortality and morbidity, and there are no approved therapies. Standard of care includes dietary and palliative measures. Currently, liver and/or kidney transplant is the only effective treatment.
mRNA-3704 is being developed as a chronically dosed mRNA therapeutic that directs cells in the liver to produce and express the MUT enzyme, restoring the metabolic pathway to reduce toxic metabolite build-up.

Year-to-Date Business Updates and Highlights

Board of Directors Updates

Israel Ruiz appointed to Board of Directors: In February, Moderna announced that Israel Ruiz, Executive Vice President and Treasurer of the Massachusetts Institute of Technology (MIT), was appointed to its Board of Directors. Mr. Ruiz also serves as Audit Committee Chair. As MIT’s Chief Financial Officer, a Trustee of the MIT Corporation and a member of its Executive Committee, Mr. Ruiz is the chief steward of over $17 billion of financial assets, $3.4 billion in operating revenues and is responsible for administering MIT’s $5 billion capital plan through 2030. In addition, Mr. Ruiz currently serves as Audit Committee Chair on the Board of Directors of Fortive (NYSE: FTV), a diversified industrial growth company with 24,000 global employees and more than $6 billion in annual revenue. In 2015, Fortive was spun out from Danaher (NYSE: DHR), a Fortune 150 company and global science and technology innovator.
Moncef Slaoui, Ph.D., appointed to Board of Directors: In July, Moncef Slaoui was appointed to Moderna’s Board of Directors. While at GlaxoSmithKline PLC (GSK), Dr. Slaoui led that company’s global pharmaceutical R&D activities from 2006 through to 2015, most recently serving as its Chairman of Global Vaccines. Dr. Slaoui also served on GSK’s Board of Directors for 11 years. In conjunction with Dr. Slaoui’s appointment to Moderna’s Board of Directors, Lee Babiss, Ph.D., stepped down from his role as a Director.

Management Team Updates

Melissa Moore, Ph.D., elected to National Academy of Sciences: In May, Melissa J. Moore, Ph.D, Chief Scientific Officer of Moderna’s mRNA research platform, was elected to the National Academy of Sciences (NAS). Established in 1863, the NAS provides independent and objective advice to the US government and other organizations on matters related to science and technology. Election to membership in the NAS is widely considered one of the highest honors a scientist can receive. The NAS membership totals approximately 2,250 members and nearly 440 foreign associates, of whom approximately 200 have received Nobel prizes.
Juan Andres named Senior Vice President of Late Stage Technical Development and Manufacturing: In August, Moderna announced that Juan Andres joined Moderna as Senior Vice President of Late Stage Technical Development and Manufacturing. Mr. Andres previously served as Global Head Technical Operations (Manufacturing and Supply) at Novartis. Mr. Andres will be responsible for the scale-up of Moderna’s manufacturing, quality and operations efforts as it continues to advance its growing pipeline of mRNA medicines and prepares to bring its state-of-the-art Good Manufacturing Practice (GMP) mRNA clinical manufacturing facility online in 2018. In addition, he will lead efforts aimed at preparing Moderna for Phase 3 and commercial capabilities and capacity. Steve Harbin, who previously served as Senior Vice President of Manufacturing and Operations at Moderna, has transitioned to new roles as Chief of Staff and Chief Sustainability Officer.
Saqib Islam, Chief Business Officer, and James Kasinger, General Counsel and Secretary, depart Moderna: Mr. Islam and Mr. Kasinger have decided to pursue other opportunities. Mr. Islam will be departing the company in mid-September. Mr. Kasinger left Moderna earlier this year. A search to identify successors is underway.

Partner Updates

Moderna and Alexion terminate partnership: In July, Alexion announced its plans to conclude its partnership contract with Moderna, one of several actions undertaken by the company to reshape its R&D strategy. The ten product options that were part of the strategic agreement the two companies formed in 2014 were terminated, and the rights to development treatments for those rare diseases reverted to Moderna. While Alexion’s previously announced mRNA program in Crigler-Najjar syndrome type 1 (CN1) remains an area of research interest for Moderna, the company’s initial rare liver disease program in MMA is the current priority for development.

Operational Updates

R&D organizational update: Moderna has decided to move from its venture-based R&D model to a therapeutic area R&D model. Research/Preclinical Development will report to Moderna’s President Stephen Hoge, MD, and Clinical Development will report to Tal Zaks, MD, PhD, Moderna’s Chief Medical Officer. Research and development teams will be aligned around three core matrixed therapeutic areas – infectious diseases, immuno-oncology and rare liver diseases. As Moderna advances parallel opportunities for mRNA vaccines and therapeutics into and through the clinic, a therapeutic area R&D model will enable Moderna to more effectively share clinical learnings across therapeutic areas. In addition, the continued evolution of Moderna’s platform coupled with the rapid cycle time in drug discovery will allow tighter integration between therapeutic area discovery teams and the platform research teams.
Moderna is not eliminating any headcount as part of the organizational update. Moderna’s venture names and branding (Valera, Elpidera, Onkaido and Caperna) are being dissolved, with the entire organization moving forward under the corporate “Moderna” entity and brand.
Continued progress on Norwood GMP manufacturing facility build-out: To support and manage the breadth of simultaneous clinical studies Moderna anticipates in the coming years, the company is building a 200,000 square foot GMP mRNA clinical manufacturing facility in Norwood, Mass. The build-out continues to progress according to plan, and Moderna anticipates beginning operations at the Norwood facility in mid-2018.
The fully integrated facility will enable the manufacture, quality, control and supply of clinical grade mRNA therapies and vaccines for GLP toxicology studies as well as Phase 1 and Phase 2 clinical studies. Moderna will carry out all manufacturing activities at the site—from raw material production to APIs, formulation, filling and finish.
Continued growth across organization: Since January 2017, Moderna added approximately 50 new team members and now has more than 550 team members.

Financials as of June 30, 2017

Maintained strong cash position: Moderna has maintained a strong cash position in 2017. As of June 30, 2017, the company had $1.098 billion in cash, as compared to $1.307 billion in cash as of December 31, 2016. This affords Moderna several years of runway to support its continued growth and pipeline acceleration.
Continued significant investments in the business: Moderna’s gross investment in the business in the first half of 2017 totaled $212 million in operating expense and capital expenditures. Net of reimbursements and product milestones, $183 million of cash was used for operating expense and capital expenditures.

About Moderna Therapeutics

Moderna Contacts

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Liz Melone
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Industry Veteran Juan Andres Joins Moderna as Senior Vice President of Late Stage Technical Development and Manufacturing

Thu, 03 Aug 2017 17:19:12 GMT

Former Global Head Technical Operations (Manufacturing and Supply Chain) with Novartis Will Lead Moderna’s Manufacturing and Operations Scale-Up

CAMBRIDGE, Mass., August 3, 2017 — Moderna Therapeutics, a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, today announced that Juan Andres, former Global Head Technical Operations (Manufacturing and Supply) at Novartis, has joined the company as Senior Vice President of Late Stage Technical Development and Manufacturing (beyond human proof of concept, or hPOC). Mr. Andres will be responsible for the scale-up of Moderna’s manufacturing and quality efforts as it continues to advance its growing pipeline of mRNA medicines and prepares to bring its state-of-the-art Good Manufacturing Practice (GMP) mRNA clinical manufacturing facility online in 2018. In addition, he will lead efforts aimed at preparing Moderna for Phase 3 development and commercialization. Mr. Andres will work closely with Moderna President Stephen Hoge, who will continue to lead Early Stage Technical Development (pre-hPOC), to ensure successful transition between early and late development activities. Mr. Andres will be a member of Moderna’s Executive Committee and report to the company’s Chief Executive Officer Stéphane Bancel.

“Having had the privilege to work with Juan while we were at Lilly together, I know first-hand his abilities and effectiveness leading a complex global pharma development and manufacturing organization,” said Stéphane Bancel, Chief Executive Officer at Moderna. “Juan would be a hugely valuable addition to any number of biopharma companies’ executive committees around the globe. So, I feel truly honored that he chose to join Moderna. With the pace of our progress and the scale of our ambitions, ensuring we can deliver consistently high quality GMP mRNA is a critical enabler of Moderna’s long-term vision. With Juan as part of our team, we have now significantly enhanced our ability to execute our vision and deliver on our mission. I look forward to working with him again.”

“I’ve spent a great deal of time with Stéphane and the Moderna team in recent months, and I have gotten an ‘under the hood’ look at the science, technology platform, advancing R&D programs, and the sophisticated infrastructure that is being built to accelerate Moderna’s mRNA research efforts and drive its early development programs,” said Juan Andres, Moderna’s incoming Senior Vice President of Late Stage Technical Development and Manufacturing. “I look forward to leveraging the expertise and skill set I’ve accumulated working at established large pharma organizations to help Moderna build its drug development organization. The novelty of what Moderna has set out to accomplish, coupled with the fact that I know and trust Stéphane’s leadership instincts and abilities, convinced me that I could not pass up this tremendous opportunity.”

Mr. Andres was previously the Global Head of Technical Operations (Manufacturing and Supply Chain) for over 25,000 Novartis employees across all of its divisions. In this role, he had responsibility for all of Novartis’ manufacturing sites, and related support functions and technologies worldwide, overseeing small molecules drug substance and drug product, as well as biologics drug substance, drug product and technical development. Mr. Andres’ previous roles at Novartis included Group Novartis Quality Head, Global Head of Technical Research and Development, and Global Pharmaceutical Operations Head. Prior to Novartis, Mr. Andres worked at Eli Lilly & Co. for 18 years in a variety of manufacturing, production and quality roles in the United States, Puerto Rico, UK and Spain, most recently serving as Vice President, Pharmaceutical Manufacturing. He holds an undergraduate degree in pharmacy from Alcala de Henares University in Madrid and completed an advanced development program at the London Business School.

Steve Harbin, who previously served as Senior Vice President of Manufacturing and Operations at Moderna, is transitioning to new roles as Chief of Staff and Chief Sustainability Officer.

“I have worked with Steve for the past 17 years at three different companies now. In each role he has held, because of his broad expertise, his strong leadership skills, and his ability to successfully tackle any project, he has proven to be an invaluable asset to the respective companies and an incredible partner to me,” said Mr. Bancel. “Steve has been leading our corporate sustainability efforts to date and will now be able to focus more intently on these initiatives as they continue to expand and permeate across our organization. As Chief of Staff, he’ll continue to work closely with me on critical projects related to our scale-up and advancement as a development stage company.”

Moderna announced its first 12 mRNA development programs in January 2017, including eight prophylactic vaccines, a personalized cancer vaccine, two immuno-oncology therapeutics, and a therapeutic for cardiometabolic diseases. Five of these programs are in clinical study in the United States, Europe and Australia, and the remaining seven programs are advancing toward the clinic.

To support and manage the breadth of simultaneous clinical studies Moderna foresees in the coming months and years, the company is building a 200,000 square foot GMP mRNA clinical manufacturing facility in Norwood, Mass. The fully integrated facility will enable the manufacture, quality, control and supply of clinical grade mRNA therapies and vaccines for Good Laboratory Practices (GLP) toxicology studies as well as Phase 1 and Phase 2 clinical studies. At the site, Moderna will carry out all manufacturing activities—from raw material production to active pharmaceutical ingredients (APIs), formulation, filling and finish. Moderna plans to open the facility in 2018.

About Moderna Therapeutics

Moderna Contacts

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Liz Melone
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Moncef M. Slaoui, Ph.D. Joins Moderna’s Board of Directors

Thu, 27 Jul 2017 17:16:20 GMT

Former Chairman of Global Vaccines and Director at GlaxoSmithKline PLC to Add Expertise to Support Moderna’s Continued Build of Its Drug Development Organization

CAMBRIDGE, Mass., July 27, 2017 — Moderna Therapeutics, a clinical stage biotechnology company that is pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, announced today that Moncef M. Slaoui, Ph.D., has joined its Board of Directors. Dr. Slaoui served in several senior research & development (R&D) roles with GlaxoSmithKline PLC (GSK) during his time with the company, most recently as its Chairman of Global Vaccines. While at GSK, Dr. Slaoui also served on its Board of Directors for 11 years and led its global pharmaceutical R&D activities from 2006 through to 2015. In conjunction with Dr. Slaoui’s appointment to Moderna’s board, Lee Babiss, Ph.D., will be stepping down from his role as a director.

“We are assembling a board that can support the next phase of Moderna’s growth and development as a high-tech enterprise leveraging the digital nature of mRNA to advance a broad array of breakthrough biopharmaceutical products. Given his various leadership roles with GSK and his experience as a member of GSK’s board, Moncef brings significant vaccine and therapeutic development experience, in addition to experience managing complex pipelines from a large biopharma perspective,” said Noubar Afeyan, Ph.D., Co-Founder and Chairman, Moderna Therapeutics, and CEO of Flagship Pioneering. “As we welcome Moncef, I’d also like to thank Lee Babiss for the role he has played on the board for the last four years, giving important guidance and insights as we developed the mRNA platform and our early product candidates.”

“It is quite rare to see a biopharma company achieve the state of growth and pipeline expansion that Moderna has in such a short period of time – a result of the novel characteristics of its mRNA platform, and the unique business model and infrastructure Moderna has created to exploit this technology,” said Moncef M. Slaoui, Ph.D. “With many programs simultaneously advancing through development, Moderna will confront a unique set of challenges as it scales in the years ahead. I look forward to working with the team to navigate this uncharted territory, and support the strategic planning already underway as Moderna works to deliver mRNA medicines and vaccines to address serious unmet medical needs in the U.S. and globally.”

“We are thrilled to welcome Moncef to the Moderna board. I’ve had the privilege of spending a great deal of time with and getting to know him over the past year. Being able to leverage Moncef’s experience, skills and insights will be invaluable as we continue to evolve Moderna into a large development organization with 12 mRNA therapeutic and vaccine development candidates announced to date and several more to follow in the near term,” said Stéphane Bancel, Chief Executive Officer at Moderna.

Dr. Slaoui served as the Chairman of Global Vaccines at GSK since 2009. His previous roles at GSK included Chairman of Global Research and Development and Vaccines, Chairman of Research and Development, and Senior Vice President of Worldwide Business Development and External Alliances. Dr. Slaoui was Chairman of GSK’s Pipeline Investment Board (PIB) between 2006 and 2015 and served as a Director of GSK from 2006 to 2017. He currently serves as Chairman of the Board of Galvani, a bioelectronics-dedicated biotech co-owned by GSK and Verily, an Alphabet company. Dr. Slaoui previously was a member of the Advisory Board of the Agency for Science, Technology and Research, or A*STAR, in Singapore, and a member of the National Institutes of Health’s (NIH) Advisory Committee to the Director. He currently serves as a member of the Advisory Board to the Qatar Foundation.

Dr. Slaoui was a Professor of Immunology at the University of Mons, Belgium. He has authored more than 100 scientific papers and presentations. Dr. Slaoui earned a Ph.D. in Molecular Biology and Immunology from the University Libre de Bruxelles, Belgium and completed postdoctoral studies at Harvard Medical School and Tufts University School of Medicine, Boston.

About Moderna Therapeutics

Moderna Contacts

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Liz Melone
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Moderna Provides Update on mRNA Rare Disease Research and Development Strategy

Thu, 27 Jul 2017 17:14:14 GMT

CAMBRIDGE, Mass., July 27, 2017 — Moderna Therapeutics, a clinical stage biotechnology company that is pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, provided an update today on its rare disease Research & Development (R&D) strategy, issuing the following statement related to Alexion Pharmaceuticals, Inc.’s (Nasdaq: ALXN) R&D realignment announcement.

Alexion announced today that it plans to conclude its partnership contract with Moderna, one of several actions Alexion announced today as it reshapes its Research & Development (R&D) strategy. Specifically related to Moderna, Alexion is discontinuing existing preclinical programs that fall outside of the company’s complement franchise, including mRNA therapies, in order to align its portfolio with its redefined R&D strategy.

In 2014, Moderna had limited capacity and nascent capabilities for rare disease R&D efforts and decided to enter into a strategic agreement with Alexion to advance mRNA therapeutics for rare diseases. Under the terms of the agreement, Alexion made an upfront payment to Moderna of $100 million to purchase ten product options to discover, develop and commercialize treatments for certain rare diseases utilizing Moderna’s mRNA technology. With today’s announcement, the ten product options are terminated, and the rights to develop treatments for those rare diseases revert to Moderna.

As previously described, Moderna has acquired extensive knowledge in rare disease biology and regulatory affairs over the last few years and has built a dedicated, internal rare disease R&D organization that is advancing multiple mRNA research programs for rare diseases.

Stéphane Bancel, Chief Executive Officer at Moderna said, “We are thankful for our partnership with Alexion. We understand their decision to narrow their focus as part of a new R&D strategy. Moderna remains as committed as ever to advancing first-in-class mRNA therapeutics for patients impacted by serious and life-threatening rare diseases. Over the past few years, we have built a robust R&D infrastructure, through which we are successfully advancing many internal mRNA programs across multiple therapeutic areas. Our drug discovery and research efforts in rare diseases are a strategic focus. We have continued to make important progress on this front, with several programs for rare metabolic diseases progressing toward the development stage.” Bancel added, “We look forward to providing a detailed corporate update across our broad portfolio of internal and partnered mRNA R&D programs in the fall.”

Robert Langer, Sc.D., Academic Co-Founder of Moderna, Chair of the Board Science and Technology Platform Committee, and Institute Professor at MIT, added, "Moderna has made significant progress over the past 18 months advancing breakthrough technologies for its rare disease mRNA therapeutics that will move into development soon. The potential of this technology to bring forth effective treatments for many patients who currently have no treatment options is quite profound – and perhaps broader than we initially foresaw just a few years ago."

About Moderna Therapeutics

Moderna Contacts

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Publication in Cell Demonstrates Moderna’s Zika mRNA Vaccine Prevents In Utero Transmission of Zika Virus in Mice, Protects Against Zika-Related Congenital Damage

Thu, 13 Jul 2017 17:12:16 GMT

First Study to Show Protection from Zika Virus during Pregnancy

CAMBRIDGE, Mass., July 13, 2017 — Moderna Therapeutics, a clinical stage biotechnology company that is pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, today announced new data demonstrating that its Zika mRNA vaccine prevented Zika virus transmission from pregnant mice to their fetuses. The findings, which were published today in Cell, also demonstrated that Moderna’s Zika mRNA vaccine protected the placenta and fetus from Zika virus-induced injury.

In the study, Moderna’s Zika mRNA vaccine was evaluated in addition to a live-attenuated vaccine candidate developed by the University of Texas Medical Branch (UTMB). The research was conducted by scientists from the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), and Washington University School of Medicine and UTMB.

Children born to mothers infected with Zika can develop microcephaly, a severe disease characterized by abnormally small heads and severe neurologic disabilities. Zika infection is also strongly associated with Guillain-Barré Syndrome (GBS), an autoimmune disease that attacks the peripheral nervous system, leading to rapidly progressive and potentially life-threatening muscle weakness. GBS can lead to death caused by respiratory arrest if a patient is not ventilated. There are no treatment options or approved vaccines for the Zika virus or congenital Zika syndrome. This is the first study to establish vaccine protection from the Zika virus during pregnancy.

“We’re highly encouraged by these preclinical findings demonstrating the ability of our mRNA vaccine to provide robust prevention of maternal transmission of Zika and protection against congenital defects,” said Giuseppe Ciaramella, Ph.D., Chief Scientific Officer of Moderna’s infectious disease-focused venture, Valera, and an author on the paper. “The threat to pregnant women and women who may be planning on getting pregnant remains a serious concern in certain regions of the U.S. and abroad. We look forward to further study of our Zika mRNA vaccine to prevent Zika infections, with the ultimate goal of improving outcomes for mothers, their children, and families in the U.S. and globally.”

About the Zika mRNA Vaccine Findings

The study was designed to evaluate protection of fetuses during pregnancy in mice. Researchers gave a cohort of non-pregnant female mice (n=20) a 10 µg intramuscular (IM) injection of the Zika mRNA vaccine followed by a boost at 28 days. An additional cohort of non-pregnant mice (n=20) received placebo injections at the same time points. At day 49, the mice that received the mRNA vaccine produced high levels of neutralizing antibodies against Zika virus in their blood compared to placebo.

Both cohorts were then mated and infected with the Zika virus. After seven days, most fetuses in the vaccinated mice showed no evidence of having Zika virus transmitted to them from their pregnant mothers compared to placebo. In addition, vaccinated mice had significantly lower levels of Zika virus RNA in maternal, placental and fetal tissues compared to placebo-injected mice, resulting in protection against damage to the placenta and fetus. Specifically,

Placenta and fetal heads from the placebo cohort showed high levels of viral RNA levels while corresponding tissues in mice immunized with Moderna’s Zika mRNA vaccine showed marked virological protection (placenta, 200-fold mean reduction; fetal head, 13,000-fold mean reduction).
10 of 19 (53%) placentas and 11 of 19 (58%) fetal heads from mice who received Moderna’s Zika mRNA vaccine had viral RNA levels at the limit of detection of the assay, suggesting virtually complete protection, and the remainder had substantially lower levels than those detected in samples from mice in the placebo cohort.
Only three of 19 (16%) of placentas and 0 fetal heads from maternal mice immunized with Moderna’s mRNA Zika vaccine were positive for the infectious virus compared to 21 of 23 (91%) of placentas and 10 of 23 (43%) fetal heads from placebo-vaccinated maternal mice.

The researchers repeated the experiment in order to determine the effects on fetal viability at birth, again comparing maternal mice who were vaccinated with Moderna’s mRNA vaccine (n=14) and maternal mice who received placebo (n=14). None of the mice in the placebo group delivered pups at term due to extensive placental injury and fetal demise. In contrast, 100% of the fetuses from mice who received Moderna’s mRNA vaccine were born without signs of damage, and the heads of newborn pups of mothers in this treatment group showed no measurable levels of viral RNA.

“These are very promising findings and, as the first study to demonstrate protection from Zika in the pregnancy setting, are an important development in our efforts to combat Zika virus,” said Michael Diamond, M.D., Ph.D., Professor, Departments of Medicine, Molecular Microbiology, Pathology & Immunology, and Associate Director, Center for Human Immunology and Immunotherapy Program at Washington University School of Medicine, and a lead author on the Cell paper. “This type of collaboration, fusing the expertise of academia, government and industry, will be critical in order to speed advancement of novel vaccines like the mRNA vaccine and live-attenuated vaccine involved in this study.”

Moderna’s Zika mRNA vaccine, mRNA-1325, is currently in Phase 1/2 clinical study in healthy volunteers. The company’s pipeline includes seven additional mRNA prophylactic vaccines, all of which address infectious diseases for which there currently are no approved vaccines.

In February 2017, a paper published in Cell demonstrated that Moderna’s mRNA vaccine protected mice against Zika. In April 2017, Moderna published human data for its mRNA vaccine technology in Molecular Therapy, which showed that its first prophylactic vaccine candidate, mRNA-1440 -- an mRNA prophylactic vaccine against avian H10N8 influenza – induced high levels of immunogenicity and was safe and well tolerated.

About Moderna’s Zika mRNA Vaccine

Moderna’s Zika mRNA vaccine encodes for viral antigenic proteins (Zika virus prM and E) associated with the Zika virus. The mRNA directs cells to produce and express the proteins on the cell surface, much like a native infections would do, but without the ability to cause disease. This is because no other viral proteins are present to enable the production of an infectious Zika virus.

As a result, the immune system recognizes the antigenic proteins as foreign to the body and produces antibodies that have the potential to neutralize the Zika virus, and prevent infections in the event the vaccinated person is exposed to the actual virus in the future.

In 2016, Moderna received a funding award of up to $125 million from the Biomedical Advanced Research and Development Authority ( BARDA ), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS), to accelerate development of its Zika mRNA vaccine. Moderna’s preclinical work for mRNA-1325 was funded through a grant from the Defense Advanced Research Projects Agency ( DARPA ).

About Moderna Therapeutics

Headquartered in Cambridge, Mass., privately held Moderna currently has strategic agreements with AstraZeneca, Merck, Alexion Pharmaceuticals and Vertex Pharmaceuticals, as well as the Defense Advanced Research Projects Agency (DARPA), an agency of the U.S. Department of Defense; the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS); and the Bill & Melinda Gates Foundation. To learn more, visit www.modernatx.com .

Moderna Contacts

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Melissa J. Moore, Ph.D., Chief Scientific Officer, mRNA Research Platform at Moderna, Elected to National Academy of Sciences

Wed, 03 May 2017 17:08:45 GMT

CAMBRIDGE, Mass., May 3, 2017 — Moderna Therapeutics, a clinical stage biotechnology company that is pioneering messenger RNA (mRNA) Therapeutics™ to create a new generation of transformative medicines for patients, today announced that Melissa J. Moore, Ph.D, Chief Scientific Officer of Moderna’s mRNA research platform, has been elected to the National Academy of Sciences (NAS). Dr. Moore is among 84 new members and 21 foreign associates elected to the NAS yesterday in recognition of their distinguished and continued achievements in original research.

“I’m incredibly honored and humbled to have been elected to the National Academy of Sciences. I truly believe there has been no better, more exciting time to be a scientist than today,” said Dr. Moore. “Scientific research is all about collaboration. I’ve been fortunate to have had the opportunity to learn from and work side by side with inspiring mentors and colleagues throughout my career, most recently at the University of Massachusetts Medical School and currently at Moderna Therapeutics. I look forward to contributing to the mission of the NAS, and working with my fellow NAS members and associates as we consider complex problems facing our country and the world today and contemplate how science and technology can help advance thoughtful public policy decisions.”

The National Academy of Sciences is a private, non-profit society of distinguished scholars. Established in 1863, the NAS provides independent and objective advice to the United States government and other organizations on matters related to science and technology. Election to membership in the NAS is widely considered one of the highest honors a scientist can receive. The NAS membership totals approximately 2,250 members and nearly 440 foreign associates, of whom approximately 200 have received Nobel prizes.

“Melissa is a remarkable scientist and a wonderful leader,” said Stéphane Bancel, Chief Executive Officer of Moderna. “Her curiosity and passion to always ask ‘why’, her ability to bring individuals and teams together to tackle challenges and arrive at creative solutions, and her attention to detail and high bar for excellence, already have had a significant impact across our organization and on the science that we do at Moderna. We are thrilled for her for achieving this amazing, well-deserved recognition as a newly elected member of the National Academy of Sciences.”

Dr. Moore was appointed Chief Scientific Officer of Moderna’s mRNA research platform in September 2016 after serving as a member of the company’s Scientific Advisory Board. Before joining Moderna, she served as Professor of Biochemistry & Molecular Pharmacology at the University of Massachusetts Medical School (UMMS) and Investigator at the Howard Hughes Medical Institute (HHMI). She also was a founding Co-Director of the RNA Therapeutics Institute (RTI) at UMMS. Her research at UMMS and HHMI encompassed an array of topics related to the role of RNA and RNA-protein (RNP) complexes in gene expression.

About Moderna Therapeutics

Moderna is a clinical stage pioneer of messenger RNA (mRNA) Therapeutics™, an entirely new drug technology that directs the body’s cells to produce intracellular or secreted proteins. With its breakthrough platform, Moderna is developing mRNA vaccines and therapeutics as a new class of medicines for a wide range of diseases and conditions, in many cases by addressing currently undruggable targets. Moderna is developing its innovative mRNA medicines for infectious diseases, cancer (immuno-oncology), rare diseases, cardiovascular disease and pulmonary disease, through proprietary development and collaborations with strategic partners.

Moderna Contacts

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Moderna Announces Positive Interim Phase 1 Clinical Data Demonstrating First mRNA Vaccine Candidate, mRNA-1440, Induces High Levels of Immunogenicity

Thu, 27 Apr 2017 02:04:43 GMT

100% of subjects in 100 µg intramuscular (IM) cohort achieved HAI titers that are accepted correlate of seroprotection against seasonal flu

mRNA-1440 safe and well tolerated; tolerability consistent with approved vaccines

Data published in the journal Molecular Therapy; First human proof-of-concept data from Moderna’s novel mRNA platform

CAMBRIDGE, Mass., April 27, 2017 — Moderna Therapeutics, a clinical stage biotechnology company that is pioneering messenger RNA (mRNA) Therapeutics™ to create a new generation of transformative medicines for patients, today announced positive interim data from an ongoing Phase 1 study of mRNA-1440, an mRNA infectious disease vaccine against avian H10N8 influenza, demonstrating mRNA-1440 induced high levels of immunogenicity, and was safe and well tolerated. The findings were published online today in the journal Molecular Therapy.

In a 100 µg intramuscular (IM) cohort of 31 subjects (23 of whom received mRNA-1440 and eight of whom received placebo), mRNA-1440 demonstrated strong efficacy based on Hemagglutination Inhibition Assay (HAI) and Microneutralization Assay (MN) titers, two measures of immunogenicity in response to vaccination. 100% (n = 23) and 87% (n = 20) achieved titers consistent with protection at day 43 as measured by HAI titers and MN titers, respectively, compared to 0% in the placebo arm. The majority of adverse events were mild to moderate; three subjects who received mRNA-1440 (13%) experienced severe adverse events, which included injection-site reactions and common cold-like symptoms. These events were considered expected, and were manageable and reversible. There were no serious adverse events. Overall, mRNA-1440 was safe and well tolerated, and demonstrated a safety profile consistent with that of approved vaccines.

“We’re highly encouraged by these data; they provide early evidence that our mRNA vaccine technology is safely and effectively directing the body’s cells to produce and express viral antigenic proteins and elicit high levels of immunity that are expected to protect against viral infection,” said Tal Zaks, M.D., Ph.D., Chief Medical Officer of Moderna. “Furthermore, they provide important validation of our core mRNA platform, as we continue to advance our development pipeline , tackling more complex vaccines including personalized cancer vaccines, and moving our mRNA therapeutics into clinical development.”

These are the first human proof-of-concept data from Moderna’s mRNA technology platform. In addition, they are the first-ever published data demonstrating a prophylactic mRNA vaccine’s ability to elicit robust immunity in humans. The publication also included preclinical data demonstrating that mRNA-1440 and Moderna’s second mRNA vaccine program, mRNA-1851 against avian H7N9 influenza, both generated strong protective immunity in mice, ferrets and non-human primates, demonstrating that Moderna’s platform technology translated across species from small animals to large animals to humans.

“These human proof-of-concept data mark a crucial milestone for Moderna as we work to advance the potential of mRNA vaccines and therapeutics to translate into clinical benefit across a breadth of diseases and unmet needs. Importantly, these published data also represent continued progress for the emerging field of mRNA science as a whole, as we explore the viability and expansive promise of this entirely new class of medicines,” said Stéphane Bancel, Chief Executive Officer of Moderna. “We look forward to completing this study and sharing the full data results in 2018, and to reporting other preclinical and clinical milestones related to our development pipeline this year.”

About the Study Design

Moderna is conducting a Phase 1 randomized, double-blind, placebo-controlled, dose-escalating study to evaluate the safety and immunogenicity of mRNA-1440 against H10N8 influenza in healthy adult subjects. H10N8 is a subtype of the Influenza A virus, for which there are no approved vaccines. The primary endpoint of the study was safety and tolerability, with immunogenicity measured by Hemagglutination Inhibition Assay (HAI) and Microneutralization Assay (MN), as additional exploratory endpoints. The Phase 1 study was designed to evaluate two doses of mRNA (prime + boost) at Day 1 and Day 22 in healthy volunteers. HAI titers and MN titers were measured at Day 43.

The study, which began dosing healthy volunteers in December 2015, and is being run by PAREXEL (NASDAQ: PRXL) at its Berlin, Germany site, has completed enrollment, with 201 healthy volunteers enrolled. The study remains active, with subjects continuing to be followed. Upon study completion and data analysis in 2018, Moderna plans to publish full study findings.

About the Interim Results

The interim results announced and published today were obtained 43 days post-vaccination of 31 subjects, 23 of whom received mRNA-1440 at 100 µg intramuscularly and 8 of whom received placebo.

Immunogenicity Findings

100% of subjects who received mRNA-1440 (n = 23) achieved a 1:40 HAI titer, compared to 0% of placebo subjects.
78% (n = 23) had a 4x HAI increase from baseline, compared to 0% of placebo subjects.
HAI titers of 1:40 or an HAI increase of 4x from baseline are expected to be protective in seasonal flu vaccines.
87% of subjects who received mRNA-1440 (n = 20) achieved a 1:20 MN titer, compared to 0% of placebo subjects.
87% (n = 20) achieved a 4x MN increase from baseline, compared to 0% of placebo subjects.
MN titers of 1:20 or an MN increase of 4x from baseline are expected to be protective.

Safety Findings

The vaccine was safe and well tolerated, with tolerability consistent with other approved vaccines.
The majority of adverse events (AEs) were mild (107/163 events; 66%) or moderate (52/163 events; 32%), using the FDA Center for Biologics Evaluation and Research (CBER) AE severity scale. ^[1]
The majority of AEs included injection-site pain, myalgia, headache, fatigue and chills/common cold-like symptoms.
Four events (accounting for 2.5% of all events) that occurred in 3 subjects (13% of subjects) were categorized as severe and included injection-site erythema (redness of the skin, 1.2%), injection-site induration (hardening, 0.6%), and chills/common cold-like symptoms (0.6%). These were expected and reversible.
No serious adverse events were observed.

Currently there are four Phase 1 mRNA vaccine studies and one Phase 1 mRNA therapeutic study underway utilizing Moderna’s mRNA platform technology. To date, more than 450 healthy volunteers have been dosed across these initial five clinical programs. Seven additional development candidates, including mRNA vaccines and mRNA therapeutics, are advancing toward clinical development. Moderna is advancing its development pipeline and research programs through proprietary development and collaborations with strategic partners, including AstraZeneca (NYSE: AZN), Merck (NYSE: MRK), Alexion Pharmaceuticals (NASDAQ: ALXN) and Vertex Pharmaceuticals (NASDAQ: VRTX).

ADDITIONAL DETAILS

About the Hemagglutination Inhibition Assay (HAI) and Microneutralization Assay (MN)

HAI measures the amount of antibodies specific for hemagglutinin (HA) that are generated by a subject in response to receiving a vaccine. HA is a viral antigenic protein. By binding to HA, antibodies inhibit HA binding to erythrocytes (red blood cells) and, thus, inhibit the formation of an insoluble red aggregate at the bottom of a test vessel. An HAI titer measures the maximum dilution in which binding is inhibited. HAI titers of at least 40 (1:40 diluted) are associated with at least a 50% reduction in risk for seasonal influenza; 1:40 is used by the U.S. Food and Drug Administration (FDA) as the approval endpoint for seasonal flu vaccines.

MN is a functional assay that measures whether a subject’s serum prevents viral infection in vitro. MN titers measure the maximum dilution in which viral infection is inhibited. An MN titer of at least 20 (1:20 dilution) is thought to be clinically meaningful.

mRNA-1440 and mRNA-1851 – Enabling Rapid Assessment of Platform Safety and Efficacy

Moderna selected mRNA vaccines against H10N8 (mRNA-1440) and H7N9 (mRNA-1851), two Influenza A strains with pandemic potential, as its first development candidates (DCs) in order to rapidly assess the safety and efficacy of its mRNA platform in humans.

Because the H10N8 and H7N9 strains are not circulating in the general population where the trials are taking place (the U.S. and Germany), Moderna is able to study the efficacy of its vaccine technology in naïve patient populations. Therefore, the protective antibodies present in subjects’ blood after treatment with mRNA-1440 and mRNA-1851 are likely attributed to Moderna’s vaccines and not to active immunity as a result of previous exposure to the virus.

In addition, studying these influenza strains is allowing Moderna to measure vaccine efficacy against a well-understood endpoint, HAI titers. HAI is used by the FDA and the World Health Organization (WHO) to measure how well antibodies are predicted to inactivate an influenza virus. Vaccines demonstrating titers of 1:40 are considered effective in reducing the risk for influenza infection and are, thus, approved as seasonal flu vaccines.

About mRNA-1440 – Influenza A Virus Subtype H10N8 Vaccine

Influenza A subtype H10N8 has infected three people in China in 2013, resulting in two deaths. If H10N8 were to become a pandemic, there is no approved vaccine. A Phase 1 study of healthy volunteers conducted in Europe has completed enrollment, with a total of 201 subjects enrolled. The study remains active, with subjects continuing to be followed. Moderna plans to publish complete findings in 2018 upon completion of the study and full data analysis.

About mRNA-1851 – Influenza A Virus Subtype H7N9 Vaccine

Influenza A subtype H7N9 has a high potential of becoming a pandemic. As of April 12, 2017, nearly 1,440 cases have been confirmed and 545 deaths since February 2013. ^[2] There is no approved vaccine against this strain. A Phase 1 study of healthy volunteers is underway in the U.S. with 156 healthy volunteers dosed.

How mRNA-1440 and mRNA-1851 Work

Each day, trillions of mRNA deliver the genetic code the body’s cells need to produce proteins. When used as a drug, mRNA can direct cells to produce therapeutic proteins (mRNA therapeutics) to fight disease or antigenic proteins (mRNA vaccines) to prevent disease.

mRNA-1440 and mRNA-1851 are mRNA vaccines that encode for the viral protein hemagglutinin as the antigen. The vaccines deliver mRNA encoding for hemagglutinin to the body’s cells, directing them to produce and express this viral antigenic protein transiently on the cell’s surface, much like a native infection would do, but without the ability to cause disease. This is because no other viral proteins are present to enable the production of an infectious virus.

As a result, the immune system recognizes the hemagglutinin as foreign to the body and produces antibodies that have the potential to neutralize the H10N8 or H7N9 virus, and prevent infections in the event the vaccinated person is exposed to the actual virus in the future.

Moderna’s mRNA vaccines are delivered via intramuscular (IM) injection, similar to traditional vaccines. Unlike DNA vaccines, mRNA vaccines do not require electroporation or other delivery devices, which can be painful.

[1] https://www.fda.gov/biologicsbloodvaccines/guidancecomplianceregulatoryinformation/guidances/vaccines/ucm074775.htm
[2] Source: http://www.fao.org/ag/againfo/programmes/en/empres/h7n9/situation_update.html

About Moderna Therapeutics

Moderna Contacts

Media:
Liz Melone
617-256-6622
liz.melone@modernatx.com

Investors:
Lorence Kim
617-209-5849
lorence.kim@modernatx.com

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Moderna Appoints Israel Ruiz to Its Board of Directors

Wed, 22 Feb 2017 01:56:33 GMT

CAMBRIDGE, Mass., February, 22, 2017 — Moderna Therapeutics, a clinical stage biotechnology company that is pioneering messenger RNA (mRNA) Therapeutics™ to create a new generation of transformative medicines for patients, announced today the appointment of Israel Ruiz, Executive Vice President and Treasurer of the Massachusetts Institute of Technology (MIT), to the company’s Board of Directors, where he will serve as Audit Committee Chair.

Mr. Ruiz serves as MIT’s Chief Financial Officer and, as a Trustee of the MIT Corporation and a member of its Executive Committee, is the chief steward of over $17 billion of financial assets, $3.4 billion in operating revenues and is responsible for administering MIT’s $5 billion capital plan through 2030. In addition, Mr. Ruiz currently serves as Audit Committee Chair on the Board of Directors of Fortive (NYSE: FTV), a diversified industrial growth company with 24,000 global employees and more than $6 billion in annual revenue. In 2015, Fortive was spun out from Danaher (NYSE: DHR), a Fortune 150 company and global science and technology innovator.

“We are delighted to welcome Israel to Moderna’s Board of Directors. His progressive leadership at MIT has helped ensure the continued, unparalleled contributions of the Institute on a regional, national and global scale, through education, innovation and entrepreneurship,” said Noubar Afeyan, Ph.D., cofounder and Chairman, Moderna Therapeutics, and CEO of Flagship Pioneering. “We look forward to leveraging Israel’s insights as Moderna evolves into a leading, clinical stage biotechnology company by harnessing the promise of messenger RNA science to improve lives.”

With a strong understanding of MIT’s innovation ecosystem and future technology trajectories, Mr. Ruiz was instrumental in leading the re-zoning efforts of Kendall Square in Cambridge, Mass. in 2013 to enable mixed-use development and accelerate the process of moving ideas from lab to market. Mr. Ruiz continues to actively co-lead the development of the process through its complex execution phase, expected to last beyond 2020.

“A critical component of Moderna’s success to date has been our ability to tap into a breadth of viewpoints and guidance from leading experts across the corporate and academic worlds. To that end, we are thrilled that Israel is joining the Moderna board,” said Stéphane Bancel, Chief Executive Officer at Moderna. “Israel’s proven track record at MIT, one of the world’s leading incubators of innovation, as well as his active role in advancing Kendall Square as a world-class innovation hub, afford him a unique perspective on how Moderna can drive innovation on behalf of patients, and also advance change through broader contributions to society. In addition, Israel’s experience as Audit Committee Chair on Fortive’s Board of Directors well positions him to help us continue to ensure Moderna’s financial strength and establish a framework for long-term success.”

“At MIT, we are driven to bring knowledge to bear on the world’s great challenges, and I see this same spirit of intellect and impact embodied by the Moderna team. They are working with rigor and intensity to drive breakthroughs in both science and technology with the goal of delivering a new class of medicines to address significant medical challenges and unmet needs around the world,” said Mr. Ruiz. “I am extremely honored to join Moderna’s Board, and look forward to lending my support to help further advance the mission and vision of this unique and immensely exciting company.”

At MIT, Mr. Ruiz is responsible for financial and debt strategy development, budget and capital planning, and the integrity of financial information. His other areas of responsibility include human resources, information systems, campus facilities, security and safety, compliance, government relations, international support, sustainability and medical.

Prior to becoming Executive Vice President and Treasurer in 2011, Mr. Ruiz held several roles of increasing responsibility at MIT, most recently serving as Vice President of Finance. Involved since the early 2000s with digital education, Mr. Ruiz was instrumental in launching a group to evaluate e-learning opportunities in 2009-2010 in response to the global financial crisis. The work of this group ultimately led MIT to launching its online efforts, MITx in 2011 and edX in 2012, in partnership with Harvard University. In 2014, Mr. Ruiz co-led the Task Force that published the “Future of MIT Education” outlining the tremendous opportunities that digital learning technologies bring to residential education and to the global market for education. Mr. Ruiz previously held management and engineering roles at Hewlett-Packard and Nissan Automotive.

Mr. Ruiz serves on the MIT-related Board of Directors of edX (an MIT and Harvard on-line learning initiative), MIT Endicott House and MIT Technology Review. He is a director of Fortive (NYSE: FTV). He is also a director of the Governing Board of the Eliot Innovation School and very active in the Boston Public Schools.

Mr. Ruiz holds a master’s degree from the MIT Sloan School of Management and a six-year degree in industrial and mechanical engineering from the Polytechnic University of Catalonia, in his native Barcelona.

About Moderna Therapeutics

Moderna is a clinical stage pioneer of messenger RNA Therapeutics™, an entirely new in vivo drug technology that directs the body’s cells to produces human proteins, antibodies and novel protein constructs, which are in turn secreted or active intracellularly. With its breakthrough platform, Moderna is developing mRNA vaccines and therapeutics to address currently undruggable targets and deliver a new class of medicines for a wide range of diseases and conditions. Moderna is developing its innovative mRNA medicines for infectious diseases, cancer (immunooncology), rare diseases, cardiovascular disease and pulmonary disease, through its ecosystem of internal ventures and strategic partners.

Founded by Flagship Pioneering, Cambridge-based Moderna is privately held and has strategic agreements with AstraZeneca, Merck, Alexion Pharmaceuticals and Vertex Pharmaceuticals, as well as the Defense Advanced Research Projects Agency (DARPA), an agency of the U.S. Department of Defense; the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS); and the Bill & Melinda Gates Foundation. To learn more, visit www.modernatx.com .

Moderna Contacts

Investors:
Maren Winnick, 617-674-5297
maren.winnick@modernatx.com
or

Media:
Liz Melone, 617-256-6622
liz.melone@modernatx.com

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Moderna Provides Pipeline and Full-Year Corporate Update

Mon, 09 Jan 2017 01:47:13 GMT

Highlights Breadth, Depth and Velocity of Development Pipeline

12 mRNA development candidates across three therapeutic areas - infectious diseases, immuno-oncology and cardiovascular disease
Five clinical studies underway; 332 subjects dosed to date

CAMBRIDGE, Mass., January 9, 2017 – Moderna Therapeutics, a clinical stage biotechnology company that is pioneering messenger RNA (mRNA) Therapeutics™ to create a new generation of transformative medicines for patients, provided a business update today at the 35 ^th Annual J.P. Morgan Healthcare Conference in San Francisco, Calif. Moderna’s Chief Executive Officer, Stéphane Bancel, highlighted the company’s current development pipeline, which includes 12 mRNA development candidates (DCs), including vaccines and therapeutics across three therapeutic areas: infectious diseases, immuno-oncology and cardiovascular disease. Clinical studies for five of the DCs are now underway in the U.S., Europe and Australia. Among these is Moderna’s Zika mRNA vaccine, mRNA-1325, which the company progressed from idea to first-in-human study in 12 months. Moderna has filed two additional investigational new drug (IND) applications with the U.S. Food & Drug Administration (FDA); one of these INDs is now open and the other was filed in late December 2016.

“With clinical studies underway for five medicines, 332 healthy subjects dosed thus far, and seven additional development candidates advancing to the clinic, we have rapidly pivoted from a discovery company to a development company with a pipeline of unusual breadth and depth. Moderna is at an inflection point,” said Stéphane Bancel, Chief Executive Officer of Moderna. “We’ve invested heavily in our mRNA platform, research engine and early development engine to build the world’s leading mRNA company. With this infrastructure in place, we are now able to advance high-quality mRNA medicines with a breadth, speed and scale not common in our industry. Among our 2016 highlights, we are particularly proud that we were able to move our Zika mRNA vaccine candidate from initial concept to clinical study in just 12 months in response to the urgent need for a safe and effective Zika vaccine. I want to thank the Moderna team and our partners for their significant achievements over the past year to advance the promise of mRNA medicines for patients.”

A live webcast of the presentation can be accessed in the Newsroom section of Moderna’s website at modernatx.com . A replay of the webcast will be archived on Moderna’s website for at least 30 days following the presentation.

Moderna Development Pipeline

As of Jan. 9, 2017

MODERNA’S mRNA DEVELOPMENT PIPELINE – OVERVIEW AND DC HIGHLIGHTS

Modality-Centric Approach

mRNA is a fundamental component of human biology and, used as a drug, directs cells in the body to produce proteins to fight or prevent disease. Moderna combines elements of its mRNA platform into distinct approaches, called modalities, to address diseases. Moderna is advancing multiple modalities, which are technological solution sets that can be deployed to create a family of medicines for different diseases within one therapeutic area, and often across therapeutic areas.

Moderna’s current DCs utilize two of the company’s modalities: vaccines and localized therapeutics. The vaccines modality is being applied to advance mRNA-based viral vaccines for multiple infectious diseases, as well as mRNA-based personalized cancer vaccines. Both the viral vaccines and personalized cancer vaccines are delivered via intramuscular (IM) injection. The localized therapeutics modality is being applied to advance mRNA-based immuno-oncology therapeutics, delivered via intratumoral (iTu) injection, as well as mRNA-based therapeutics for cardiovascular disease and other ischemic vascular diseases.

Other modalities Moderna is pursuing in discovery include intravenous (IV) systemic therapeutics, IV liver therapeutics and inhaled pulmonary therapeutics. Moderna’s development pipeline includes the use of in-licensed delivery technologies as well as proprietary, next-generation delivery technologies.

Development Candidates (DCs) - By Modality and Therapeutic Application

Vaccines Modality (IM Injection)

Therapeutic Application #1 - Infectious Diseases/Viral Vaccines

mRNA-1440 and mRNA-1851 – Enabling rapid assessment of platform safety and efficacy

Moderna strategically selected its first two DCs with the goal of quickly assessing both the safety and efficacy of its mRNA platform in humans. These two DCs target influenza strains with pandemic potential: mRNA-1440 for influenza A subtype H10N8 and mRNA-1851 for influenza A subtype H7N9.

Because these strains are not circulating in the general population where the trials are taking place (the U.S. and Germany), Moderna is able to study the efficacy of its vaccine technology in naïve patient populations. Therefore, antibodies present in subjects’ blood after treatment with mRNA-1440 and mRNA-1851 are likely attributed to Moderna’s vaccines and not to active immunity as a result of previous exposure to the virus. To strengthen the quality of its clinical research, Moderna has conducted these trials with 25 percent of healthy subjects getting placebo.

In addition, studying these influenza strains is allowing Moderna to measure vaccine efficacy against a well-understood endpoint, the hemagglutination inhibition assay, or HAI. HAI is used by FDA and World Health Organization (WHO) to measure how well antibodies bind to and inactivate an influenza virus. Vaccines demonstrating titers of 1:40 are considered effective in reducing the risk for influenza infection and are, thus, approved as seasonal flu vaccines.

mRNA-1440 – Influenza A virus subtype H10N8 vaccine: Influenza A subtype H10N8 has infected three people in China in 2013, resulting in two deaths. If H10N8 were to become a pandemic, there is no approved vaccine. A Phase 1 study of healthy volunteers conducted in Europe has completed enrollment, with a total of 201 subjects enrolled. The study remains active, with subjects continuing to be followed. Moderna plans to publish topline study findings in 2017 and complete findings in 2018 upon completion of the study and full data analysis.
mRNA-1851 – Influenza A virus subtype H7N9 vaccine: Influenza A subtype H7N9 has a high potential of becoming a pandemic. More than 600 cases have been reported to date in China, with a mortality rate of approximately one in three people infected. There is no approved vaccine against this strain. A Phase 1 study of healthy volunteers is underway in the U.S., with 104 healthy volunteers dosed to date.

mRNA MRK-1777 – Viral vaccine for undisclosed indication: This viral vaccine is a Merck-partnered program. Under the terms of the collaboration and license agreement Moderna announced with Merck in 2015, Moderna is conducting a Phase 1 study of mRNA MRK-1777 in healthy volunteers, which is underway in Australia.

mRNA-1388 – Chikungunya virus vaccine: Chikungunya typically causes mild fever and transient joint pain. In approximately 15 percent of infected patients, it can cause long-term, severe arthritis. Chikungunya historically has been limited to warmer climates in Asia and Africa, but recent cases have been identified in the Americas and Europe. There is no approved vaccine for Chikungunya. Development of mRNA-1388 is funded through an award from the Defense Advanced Research Projects Agency (DARPA), an agency of the U.S. Department of Defense. An IND application for mRNA-1388 has been filed with the FDA.

mRNA-1325 – Zika virus vaccine: The Zika virus is a rapidly emerging pandemic with potential long-term public health implications. Zika is primarily transmitted by mosquitos, but can also be transmitted sexually. Children born to mothers infected with Zika can develop microcephaly, a severe disease characterized by small, not fully developed heads and severe disabilities. Recent data shows that 42 percent of Zika-infected pregnancies result in structural brain damage to the baby. Zika is also thought to cause the autoimmune condition Guillain-Barré syndrome in adults. There is no treatment or approved vaccine for Zika.

In response to the urgent global threat Zika presents, Moderna advanced mRNA-1325 from concept to first-in-human study in 12 months. A Phase 1/2 study is now enrolling healthy volunteers in the U.S.

In September 2016, Moderna announced a funding award of up to $125 million from the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS), to accelerate development of its Zika mRNA vaccine. To date, BARDA has granted $52 million of the award to Moderna to support its Phase 1 clinical study, toxicology studies, vaccine formulation and manufacturing. The agreement includes additional funding options up to $73 million to support Phase 2 and Phase 3 clinical studies.

mRNA-1706 – Zika virus vaccine (proprietary formulation): Moderna is advancing a second version of its Zika mRNA vaccine that contains the same active pharmaceutical ingredient (API) as the mRNA-1325 Zika mRNA vaccine, but utilizes one of the company’s next generation, novel formulations, V1GL. Good Laboratory Practice (GLP) toxicology studies are currently underway for mRNA-1706.

mRNA-1647 – Cytomegalovirus (CMV) vaccine: CMV leads to severe disease in two populations: newborns and transplant patients. CMV is the most common cause of newborn disability, leading to deafness, microcephaly (small, not fully developed heads and severe disabilities), vision loss and mental deficiencies, among other serious complications. It is also the most frequent viral disease in transplant recipients, often leading to transplant failure. There is no approved vaccine for CMV.

The majority of neutralizing antibodies the body produces to fight CMV infection are against the CMV Pentamer complex, which consists of five proteins (gH, gL, UL128, UL130 and UL131A). Producing the CMV Pentamer recombinantly has proven very difficult. There has been no success to date developing a CMV vaccine. Moderna’s mRNA platform has afforded the company the ability to rationally design a CMV vaccine that is capable of expressing the CMV Pentamer; the five components of the Pentamer are designed to act as a single antigen. mRNA-1647 combines six mRNAs to express the CMV Pentamer and another CMV antigen, the herpesvirus glycoprotein (gB) protein.

mRNA-1653 – Human Metapneumovirus (HMPV) and Parainfluenza virus (PIV3) vaccine: Most children have been infected at least once with both HMPV and PIV3 by age five. These viruses typically cause mild respiratory illness, but can become severe in young children, the elderly and other immunocompromised adults. HMPV and PIV3 are the second and third most common causes, respectively, of lower respiratory hospitalizations in children, behind RSV. There is no approved vaccine for either HMPV or PIV3. mRNA-1653 combines mRNAs encoding for viral antigens associated with both HMPV and PIV3.

Vaccines Modality (IM Injection)

Therapeutic Application #2 – Personalized Cancer Vaccines

mRNA-4157 – Personalized Cancer Vaccines: Moderna, in partnership with Merck, is developing an mRNA-based personalized cancer vaccine to prime the immune system to recognize cancer cells and mount a strong, tailored response to each individual patient’s cancer. Moderna will identify neoantigens present in each patient’s specific tumor and will create a personalized vaccine encoding for approximately 20 unique neoantigens. When injected into the body, the mRNA directs cells to produce and express these neoantigens. In turn, this activates the immune system to better recognize and destroy the cancer cells. Moderna’s mRNA-based personalized cancer vaccine has the potential to be synergistic with checkpoint inhibitor therapies, including its partner Merck’s anti-PD-1 therapy, KEYTRUDA® (pembrolizumab).

Leveraging its rapid cycle time, small-batch manufacturing technique and digital infrastructure, Moderna plans to manufacture and supply its personalized cancer vaccines tailored to individual patients within weeks.

Localized Therapeutics Modality

Therapeutic Application #1 – Immuno-Oncology Therapeutics (Intratumoral, or iTu, Injection)

mRNA-2416 – OX40L Immunotherapy: OX40 Ligand, or OX40L, is a powerful co-stimulatory protein that enhances the expansion, function and survival of T cells to mount an attack against cancer cells. Moderna is investigating the potential effect of iTu injection of mRNA encoding for the OX40L protein into a tumor. When mRNA-2416 is delivered directly into a tumor, cells in the tumor express the OX40 ligand protein on their surface, which, in turn, may lead to a stronger T cell attack against the tumor. Additionally, Moderna is investigating the potential for mRNA-2416 to elicit an abscopal effect in metastatic cancer, in which localized injection into one tumor would lead not only to shrinking of that tumor but also shrinking of tumors elsewhere in the body. Combining mRNA-2416 with a checkpoint inhibitor may improve outcomes from cancer therapy.

An IND for mRNA-2416 has been filed with the FDA.

mRNA-2905 – IL-12 Immunotherapy: Interleukin 12, or IL-12, is a powerful cytokine that activates the immune system after being released from cells. Moderna, in partnership with AstraZeneca, is investigating the potential effect of iTu injection of mRNA encoding for the IL-12 protein. When mRNA-2905 is delivered directly into a tumor, cells in the tumor express IL-12 at a high concentration in the local microenvironment, which, in turn, may lead to a stronger T cell attack against the tumor. By expressing IL-12 locally, systemic side effects that previously have been seen from delivery of IL-12 protein into the blood may be more manageable. Moderna is also investigating the potential of mRNA-2905 to elicit an abscopal effect in metastatic cancer, in which localized injection into one tumor would lead not only to shrinking of that tumor but also shrinking of tumors elsewhere in the body. Combining mRNA-2905 with a checkpoint inhibitor may improve outcomes from cancer therapy.

mRNA-2905 is being developed through a collaboration Moderna announced in early 2016 with AstraZeneca to discover, co-develop and co-commercialize immuno-oncology mRNA therapeutics. Under the terms of the agreement, Moderna is leading discovery efforts and preclinical development, and AstraZeneca will oversee early clinical development (led by MedImmune). GLP toxicology studies are currently underway for mRNA-2905. Moderna and AstraZeneca will share the costs of late-stage clinical development. The two companies will co-commercialize resulting products in the U.S. under a 50:50 profit sharing arrangement.

Localized Therapeutics Modality

Therapeutic Application #2 – Cardiovascular Therapeutics (Intracardiac Injection)

mRNA AZD-8601 – VEGF-A: mRNA AZD-8601 is an investigational mRNA-based therapy being developed by AstraZeneca that encodes for vascular endothelial growth factor-A (VEGF-A). Using mRNA to initiate a strong, local and transient surge of VEGF-A expression could help overcome challenges associated with previous approaches to regulate this protein in tissues. When directed via local tissue injection, VEGF-A mRNA may potentially lead to the creation of more blood vessels and improved blood supply. mRNA AZD-8601 could one day provide a unique regenerative treatment option for patients with heart failure or after a heart attack, as well as for diabetic wound healing and other ischemic vascular diseases.

A Phase 1 safety study is currently enrolling patients in Europe. This study is a randomized, single-blind, placebo-controlled, single ascending dose study in male patients with Type 2 diabetes mellitus, performed at a single study center. This study is an essential first step to proving the clinical value of mRNA VEGF-A expression in cardiometabolic diseases.

2016 BUSINESS UPDATES AND HIGHLIGHTS

2016 Partnerships

Immuno-Oncology Collaboration with AstraZeneca: In January, Moderna announced a new collaboration with AstraZeneca to discover, co-develop and co-commercialize immuno-oncology mRNA therapeutic candidates. The collaboration is in addition to the exclusive agreement announced by the companies in 2013 to develop mRNA therapeutics for the treatment of cardiovascular, metabolic and renal diseases as well as selected targets in oncology.
Inclusion of New Infectious Disease Vaccine Program with Merck: In January, Moderna announced that Merck licensed a vaccine program against an undisclosed viral target, including mRNA 1566 and a set of related novel vaccine candidates, as part of the ongoing collaboration between the companies. The inclusion of this new program, which was not part of the original collaboration agreement, follows the rapid progress made in the first year of the collaboration.
Global Health Partnership with the Bill & Melinda Gates Foundation: Moderna also announced in January a partnership with the Bill & Melinda Gates Foundation to advance the development of a novel, affordable combination of mRNA-based antibody therapeutics to help prevent human immunodeficiency virus (HIV) infection. The global health partnership has the potential for follow-on projects to develop additional mRNA-based projects for various infectious diseases.
Personalized Cancer Vaccines Collaboration with Merck: In June, Moderna announced a new strategic collaboration with Merck to advance novel mRNA-based personalized cancer vaccines with KEYTRUDA® (pembrolizumab) for the treatment of multiple types of cancer. The collaboration will leverage Moderna’s rapid cycle time, small-batch manufacturing and digital infrastructure to supply vaccines tailored to individual patients within weeks. Under the terms of the agreement, Merck made an upfront cash payment to Moderna of $200 million, which Moderna is using to lead all research and development efforts through proof of concept.
Research Collaboration with Vertex in Cystic Fibrosis: In July, Moderna announced an exclusive research collaboration and licensing agreement with Vertex Pharmaceuticals to discover and develop mRNA therapeutics for the treatment of cystic fibrosis (CF). The three-year collaboration will focus on the use of mRNA therapeutics, administered via pulmonary delivery, to treat the underlying cause of CF by enabling cells in the lungs to produce functional copies of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which is known to be defective in people with CF.
BARDA Funding Award for Zika mRNA Vaccine: In September, Moderna announced that it had received a BARDA funding award of up to $125 million for mRNA-1325, an investigational Zika vaccine. To date, BARDA has granted $52 million of the award to Moderna to support its Phase 1 clinical study, toxicology studies, vaccine formulation and manufacturing. The agreement includes options for additional funding up to $73 million to support Phase 2 and Phase 3 clinical studies.

2016 Infrastructure Investments and Achievements

Build-out of Good Manufacturing Practices (GMP) mRNA Clinical Manufacturing Facility: In September, Moderna announced the build-out of a state-of-the-art GMP clinical manufacturing facility to support its growing number of clinical programs. Moderna is making an initial investment of $110 million to build out the 200,000-square-foot facility, located in Norwood, Mass. The facility will enable the manufacture, quality, control and supply of clinical grade mRNA therapies and vaccines for GLP toxicology studies as well as Phase 1 and Phase 2 clinical studies. At the site, which is expected to open in mid-2018, Moderna will carry out fully integrated manufacturing activities—from raw material production to active pharmaceutical ingredients (APIs), formulation, filling and finish.
Continued Expansion of Internal Expertise:
Key Leadership Hires: In October, Moderna welcomed two key senior additions. Bolstering its scientific team, the company appointed Melissa Moore, Ph.D., as Chief Scientific Officer of its mRNA Research Platform. Previously a member of Moderna’s Scientific Advisory Board, Dr. Moore joined Moderna from the University of Massachusetts Medical School (UMMS), where she served as Professor of Biochemistry & Molecular Pharmacology, Eleanor Eustis Farrington Chair in Cancer Research and Investigator at the Howard Hughes Medical Institute (HHMI). In addition, Moderna appointed Annie Seibold Drapeau as Chief Human Resources Officer. Most recently an Operating Partner at Bain Capital Private Equity, Ms. Drapeau is leading Moderna’s talent and organizational strategy to support its continued growth and advancement of its mRNA pipeline.
Growth across the Organization: In 2016, Moderna expanded its headcount from approximately 325 to more than 500 team members.
Ranked Third Top Employer in Biopharma Industry by Science: For the second consecutive year, Moderna was named among the industry’s best employers by Science and Science Careers’ annual Top Employer survey. Moderna ranked #3 this year, moving up four spots from the 2015 Top Employer survey. The survey polls employees across the globe in biotechnology, biopharmaceutical, pharmaceutical and related industries to rate companies on various key characteristics to arrive at a list of the 20 best employers.
Recognized by The Boston Globe in its 2016 Top Places to Work Feature: Moderna was recognized by The Boston Globe as one of the top employers in Massachusetts in its annual Top Places to Work feature . Among the hundreds of life sciences companies in Mass., Moderna was only one of six companies from the pharmaceutical / biopharmaceutical and life science categories included in this year’s Top Places to Work list.
Transitioned to SAP for Finance Business Processes: At the end of 2016, Moderna took another important step toward becoming a fully digital biotech company with the implementation of SAP for finance business processes and materials receiving. The roll-out of a highly integrated enterprise resource planning (ERP) solution is a critical component of preparing for the launch of Moderna’s Norwood, Mass., GMP clinical manufacturing facility. The deployment of SAP will enable Moderna to scale as a company, efficiently and in an integrated fashion, as it advances its mission to deliver a new generation of transformative medicines for patients.

2016 Financials

Strengthened Balance Sheet with New Equity Financing: In September, Moderna announced the close of a $474 million equity financing, which included strong support from existing institutional investors, pharmaceutical partners and new institutional investors from the U.S., Europe and Asia.
Granted First Two Tranches of BARDA Funding for Zika mRNA Vaccine: In September 2016, Moderna announced a funding award of up to $125 million from BARDA to accelerate development of its Zika mRNA vaccine. To date, BARDA has granted $52 million of the $125 million award to Moderna to support its Phase 1 clinical study, toxicology studies, vaccine formulation and manufacturing. This includes the granting of an initial $8 million in September and in December the granting of a second, tranche of $44 million.
Over $1 Billion in Cash Inflows and Available Grants in 2016: In addition to the $474 million equity financing, Moderna received $36 million in reimbursement and product milestones from its collaborators. Also, upfront payments from new collaborations signed in 2016, plus a technical milestone from an existing collaboration, brought in $290 million. When considered with the $225 million in potentially available funding from grants and awards from foundations and government agencies, Moderna accessed over $1 billion of cash and available grants during the year.
Strong Cash Position Affords Several Years of Runway: As of December 31, 2016, Moderna had $1.307 billion in cash, as compared to $802 million as of December 31, 2015. This affords Moderna several years of runway to support its continued growth and pipeline acceleration.
Investments in the Business: Moderna’s gross cash investment in the business totaled approximately $300 million in operating expense and capital expenditures. Net of reimbursements and product milestones, approximately $260 million of cash was used for operating expense and capital expenditures.

“In 2017, we will remain focused on progressing our current development candidates to and through the clinic; discovering and bringing forth additional mRNA medicines as new development candidates; and continuing to invest heavily in our mRNA platform as well as the build-out of our GMP clinical manufacturing facility in Norwood, Mass. We also look forward to begin publishing data on our clinical programs as well as key insights related to our platform,” said Stéphane Bancel, Moderna’s Chief Executive Officer. “And we will continue to invest in building our team and working diligently to ensure that our employees continue to feel inspired and empowered every day to innovate and drive impact for patients.”

About Moderna Therapeutics

Moderna is a clinical stage pioneer of messenger RNA Therapeutics™, an entirely new in vivo drug technology that directs the body’s cells to produces human proteins, antibodies and entirely novel protein constructs, which are in turn secreted or active intracellularly. With its breakthrough platform, Moderna is developing mRNA vaccines and therapeutics to address currently undruggable targets and deliver a new class of medicines for a wide range of diseases and conditions. Moderna is developing and plans to commercialize its innovative mRNA medicines for infectious diseases, cancer (immunooncology), rare diseases, cardiovascular disease and pulmonary disease, through its ecosystem of internal ventures and strategic partners.

Headquartered in Cambridge, Mass., privately held Moderna currently has strategic agreements with AstraZeneca , Merck , Alexion Pharmaceuticals and Vertex Pharmaceuticals , as well as the Defense Advanced Research Projects Agency ( DARPA ), an agency of the U.S. Department of Defense; the Biomedical Advanced Research and Development Authority ( BARDA ), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS); and the Bill & Melinda Gates Foundation . To learn more, visit www.modernatx.com .

Moderna Contacts

Investors:
Maren Winnick
617-674-5297
maren.winnick@modernatx.com

Media:
Liz Melone
617-256-6622
liz.melone@modernatx.com

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Moderna Joins the Human Vaccines Project to Help Advance Fundamental Understanding of the Immune System

Wed, 04 Jan 2017 01:33:24 GMT

Public-Private Consortium Collaborating to Generate New Immunological Insights, Accelerate Development of Vaccines and Immunotherapies

CAMBRIDGE, Mass., January 4, 2017 — Moderna Therapeutics, a clinical stage biotechnology company pioneering messenger RNA (mRNA) Therapeutics™ to create a new generation of transformative medicines for patients, announced today that it will join the Human Vaccines Project, a non-profit public-private partnership focused on decoding the human immune system to accelerate the development of vaccines and immunotherapies against major infectious diseases and cancer. Moderna will join the global, cross-sector consortium of academic research centers, biopharmaceutical companies, governments and non-profit organizations in sharing knowledge and resources to generate key insights about immunological protection, and address primary scientific hurdles to developing new vaccines and immunotherapies.

“We are proud to support the important efforts of the Human Vaccines Project to unlock basic understanding of the immune system and translate this knowledge to accelerate infectious disease vaccines and cancer immunotherapies,” said Michael Watson, President of Valera, Moderna’s infectious disease-focused venture. “Collaborating with biopharma, academic, non-profit and government organizations has been a key focus of Moderna’s strategy to advance the promise of mRNA science for patients. We look forward to contributing to this consortium in kind, helping advance knowledge about human immunity that, ultimately, could help people around the world.”

Moderna currently has four mRNA-based infectious disease vaccines in clinical study and another four infectious disease vaccines advancing toward the clinic. The company is also developing an mRNA-based personalized cancer vaccine.

The Human Vaccines Project is a decade-long effort aimed at decoding the human immune system by harnessing recent technological advances in genomics, bioinformatics and systems biology. The Project has created a network of leading university and academic research centers that serve as its scientific hubs. These hubs work collaboratively to develop and execute the Project’s scientific plan, comprising 1.) the Human Immunome Program focused on defining the parts or components of the immune system, and 2.) the Rules of Immunogenicity Program, which seeks to define the rules of immunological protection. The involvement of Moderna and other biopharmaceutical companies will help promote the rapid translation of research breakthroughs generated by the Project into potential new products.

“We are honored to have Moderna join the Human Vaccines Project’s efforts to address the immunologic challenges impeding development of new and improved vaccines and immunotherapies for major infectious diseases and cancers,” said Wayne C. Koff, President and CEO, Human Vaccines Project. By harnessing recent technological advances from biomedical, computational, and engineering sciences, including Moderna’s transformative mRNA platform, the Project offers the potential to decode the human immune system, accelerate product development, and usher in a new era of global disease prevention.”

About the Human Vaccines Project

The Human Vaccines Project is a non-profit public-private partnership with the mission to accelerate the development of vaccines and immunotherapies against major infectious diseases and cancers by decoding the human immune system. The Project has a growing list of partners and financial supporters including: Vanderbilt University Medical Center, the J. Craig Venter Institute, the La Jolla Institute, The Scripps Research Institute, UC San Diego, Aeras, Boehringer Ingelheim, Crucell/Janssen, GSK, Pfizer, MedImmune, Regeneron, Sanofi Pasteur, the Robert Wood Johnson Foundation and the John D. and Catherine T. MacArthur Foundation. The Project brings together leading academic research centers, industrial partners, nonprofits and governments to address the primary scientific barriers to developing new vaccines and immunotherapies, and has been endorsed by 35 of the world’s leading vaccine scientists. www.humanvaccinesproject.org

About Moderna Therapeutics

Moderna is a clinical stage pioneer of messenger RNA Therapeutics™ , an entirely new in vivo drug technology that directs the body’s cells to produces human proteins, antibodies and entirely novel protein constructs, which are in turn secreted or active intracellularly. With its breakthrough platform, Moderna is developing mRNA vaccines and therapeutics to address currently undruggable targets and deliver a new class of medicines for a wide range of diseases and conditions. Moderna is developing and plans to commercialize its innovative mRNA medicines for infectious diseases, cancer (immunooncology), rare diseases, cardiovascular disease and pulmonary disease, through its ecosystem of internal ventures and strategic partners.

Headquartered in Cambridge, Mass., privately held Moderna currently has strategic agreements with AstraZeneca , Merck , Alexion Pharmaceuticals and Vertex Pharmaceuticals , as well as the Defense Advanced Research Projects Agency ( DARPA ), an agency of the U.S. Department of Defense; the Biomedical Advanced Research and Development Authority ( BARDA ), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS); and the Bill & Melinda Gates Foundation . To learn more, visit www.modernatx.com .

Moderna Contacts

Investors:
Maren Winnick
617-674-5297
maren.winnick@modernatx.com

Media:
Liz Melone
617-256-6622
liz.melone@modernatx.com

VIEW ALL NEWS

Moderna to Highlight its mRNA Development Candidate Pipeline at 2017 J.P. Morgan Healthcare Conference

Tue, 03 Jan 2017 01:30:03 GMT

CAMBRIDGE, Mass., January 3, 2017 — Moderna Therapeutics, a clinical stage biotechnology company pioneering messenger RNA (mRNA) Therapeutics ^™ to create a new generation of transformative medicines for patients, announced today that Stéphane Bancel, Chief Executive Officer, will present the company’s advancing pipeline, which currently includes 12 mRNA development candidates, at the 35th Annual J.P. Morgan Healthcare Conference.

Mr. Bancel’s presentation will take place on Monday, January 9, 2017 at 2:30 p.m. PT in Elizabethan Room C/D at The Westin St. Francis Hotel in San Francisco.

About Moderna Therapeutics

Moderna Therapeutics is a clinical stage pioneer of messenger RNA Therapeutics™, an entirely new in vivo drug technology that produces human proteins, antibodies and entirely novel protein constructs inside patient cells, which are in turn secreted or active intracellularly. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of diseases and conditions. Moderna is developing and plans to commercialize its innovative mRNA medicines through its own ventures and its strategic relationships with established pharmaceutical and biotech companies. Its current ventures include Valera (infectious diseases), Onkaido (immuno-oncology), Caperna (personalized cancer vaccines) and Elpidera (rare diseases). Founded by Flagship VentureLabs®, Cambridge-based Moderna is privately held and currently has strategic agreements with AstraZeneca , Alexion Pharmaceuticals , Merck and Vertex Pharmaceuticals . To learn more, visit www.modernatx.com .

Moderna Contacts

Investors:
Maren Winnick
617-674-5297
maren.winnick@modernatx.com

Media:
Liz Melone
617-256-6622 liz.melone@modernatx.com

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Moderna Therapeutics Named the No. 3 Top Employer by Science Magazine

Thu, 27 Oct 2016 19:45:18 GMT

Cambridge, Mass., October 27, 2016 — Moderna Therapeutics, a clinical stage biotechnology company pioneering messenger RNA (mRNA) Therapeutics™ to create a new generation of transformative medicines for patients, today announced that Science and Science Careers has recognized Moderna as one of the biopharmaceutical industry’s top employers for the second consecutive year. Rankings are determined by Science and Science Careers’ annual Top Employer Survey, which polls employees across the globe in biotechnology, biopharmaceutical, pharmaceutical and related industries to rate companies on various key characteristics to arrive at a list of the 20 best employers. Moderna ranked #3 this year, moving up four spots from the 2015 Top Employer survey.

“We are very proud to be recognized as a top employer in the biopharmaceutical industry by Science for the second year in a row. Doing great science is necessary to create great medicines. A company cannot do great science without the right work environment and culture,” said Stéphane Bancel, CEO at Moderna. “We are very grateful to have such an exceptional group of scientists and industry professionals here at Moderna. This team is so committed to deliver on the promise of mRNA science for patients. They thrive on challenges and creative problem-solving, embody the spirt of collaboration, and share an unwavering focus on quality, integrity and respect. This team is relentless. We look forward to growing our team and continuing to hire best-in-class industry professionals who share our goal of addressing unmet medical needs by advancing mRNA medicines through scientific innovation.”

Ranked among a field of some of the industry’s most notable companies, Moderna was exemplary in three of the key driving characteristics of top employers, as identified by Science and Science Careers, including Innovative Leader in the Industry, Treats Employees with Respect and Has Clear Vision.

The rankings will appear in the October 28, 2016, print issue of Science, and are currently available online at ScienceCareers.org.

Science and Science Careers’ web-based survey includes results from 5,984 responses from readers of Science, as well as other survey invitees. The survey took place in the spring of 2016. Survey participants came from North America, Europe and Asia/Pacific Rim, with 93 percent working in biotechnology, biopharmaceutical and pharmaceutical companies. Rankings were determined by an independent research firm that analyzed survey responses rating companies based on 23 characteristics including a research-driven environment, easy adaptation to change and financial strength.

About Moderna Therapeutics

Moderna is a clinical stage pioneer of messenger RNA Therapeutics™ , an entirely new in vivo drug technology that produces human proteins, antibodies and entirely novel protein constructs inside patient cells, which are in turn secreted or active intracellularly. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of diseases and conditions. Moderna is developing and plans to commercialize its innovative mRNA drugs through its own ventures and its strategic relationships with established pharmaceutical and biotech companies. Its current ventures are: Onkaido , focused on oncology, Valera , focused on infectious diseases, Elpidera , focused on rare diseases, and Caperna , focused on personalized cancer vaccines. Founded by Flagship VentureLabs™ , Cambridge-based Moderna is privately held and currently has strategic agreements with AstraZeneca , Alexion Pharmaceuticals , Merck and Vertex Pharmaceuticals . To learn more, visit www.modernatx.com .

Moderna Contacts

Investors:
Maren Winnick
617-674-5297
maren.winnick@modernatx.com

Media:
Liz Melone
617-256-6622
liz.melone@modernatx.com

VIEW ALL NEWS

Moderna Appoints Annie Seibold Drapeau as Chief Human Resources Officer

Thu, 06 Oct 2016 19:43:25 GMT

Bain Capital operating partner and former human resources tech executive to lead Moderna’s talent and organizational strategy to support company’s continued growth and pipeline advancement

CAMBRIDGE, Mass., October 6, 2016 — Moderna Therapeutics, a clinical stage biotechnology company pioneering messenger RNA (mRNA) Therapeutics™ to create a new generation of transformative medicines for patients, announced today the appointment of Annie Seibold Drapeau as Chief Human Resources Officer. In her new role, Ms. Drapeau will lead Moderna’s talent and organizational strategy to support its continued growth and advancement of its mRNA drug pipeline. She currently serves as an Operating Partner at Bain Capital Private Equity, where she partners with companies across the private equity portfolio on a range of talent and organizational capability initiatives. Ms. Drapeau will join Moderna in late October and be a member of the company’s executive committee, reporting to Moderna CEO Stéphane Bancel.

“We are thrilled to welcome Annie to lead human resources; her role will be critical to Moderna’s future and success,” said Mr. Bancel. “Annie is a highly respected thought leader in building strong organizations across various sectors. She is well positioned to help us tailor an organizational and talent strategy that aligns with our unique culture, business model and our vision to impact many lives around the world with a new class of mRNA medicines. I had the chance to spend a lot of time with Annie throughout the course of the interview process, and I am looking forward to having her on the Moderna executive committee.”

“I’m very excited to join the Moderna team, particularly at this critical juncture for the company,” said Ms. Drapeau. “As Moderna continues its evolution as a clinical stage company with a fully integrated manufacturing facility to be built in Norwood, I’ll be focused on working across the organization to ensure the necessary capabilities are in place to support the company’s growth. It’s clear that Moderna’s success has been driven by its incredibly talented and passionate people. As Moderna continues to make amazing progress, I look forward to partnering with my colleagues to ensure we preserve and nurture this unique culture that inspires people to do exciting and truly transformational work each day.”

Prior to joining Bain Capital Private Equity, Ms. Drapeau served as Executive Vice President, Strategy and Talent at Iron Mountain, a $3 billion data storage and protection business with nearly 20,000 employees worldwide. She had responsibility for corporate strategy, human resources and the company’s portfolio of emerging business opportunities. She also led the company’s diversity and sustainability efforts. Before joining Iron Mountain, Ms. Drapeau held leadership roles in human resources, strategy and finance at the Center for Leading Organizations, VistaPrint, Digitas, PepsiCo and JPMorgan. She has been published in the Harvard Business Review on the topic of trust and leadership, and also led a session on that topic at the Annual Meeting of the World Economic Forum in Davos. Ms. Drapeau holds a bachelor’s in chemical engineering from Bucknell University and an MBA from the Amos Tuck School at Dartmouth College. She chairs the Boston Advisory Board of Directors for the Posse Foundation, sits on the Alumni Association Board of Directors at Bucknell University and is a Trustee of the Nashoba Brooks School.

Earlier this month, Moderna announced plans to build a 200,000 square foot, fully integrated GMP mRNA clinical manufacturing facility in Norwood, Mass., to support and manage its anticipated breadth of clinical studies across its portfolio. Set to open in early 2018, the facility will employ approximately 100 of Moderna’s current 460 team members. In addition, Moderna plans to hire more than 100 new employees for the site.

About Moderna Therapeutics

Moderna Contacts

Investors:
Maren Winnick
617-674-5297
maren.winnick@modernatx.com

Media:
Liz Melone
617-256-6622
liz.melone@modernatx.com

VIEW ALL NEWS

Moderna to Build State-of-the-Art Fully Integrated GMP mRNA Clinical Manufacturing Facility in Norwood, Mass.

Wed, 21 Sep 2016 19:41:13 GMT

Key component of Moderna’s early development engine, designed to simultaneously advance many mRNA medicines across the company’s broad therapeutic portfolio

Initial investment of $110M to build out 200,000 square foot facility; Will employ more than 200 employees

Fully integrated site from raw material to API, formulation, filling and finish

Annual capacity of 40 GMP clinical mRNA lots at facility opening; Ability to scale to more than 100 mRNA lots annually

CAMBRIDGE, Mass., September 21, 2016 — Moderna Therapeutics, a clinical stage biotechnology company pioneering messenger RNA (mRNA) Therapeutics™ to create a new generation of transformative medicines for patients, announced today that it has signed a long-term lease (15 years plus renewal options) to build a state-of-the-art Good Manufacturing Practices (GMP) clinical manufacturing facility in Norwood, Mass.

Moderna is committed to developing a broad array of medicines leveraging its leading mRNA platform, and to delivering on the promise of mRNA science for patients as quickly as possible. Moderna and its partners are simultaneously advancing mRNA drugs for many diseases toward the clinic, and the company anticipates conducting many clinical studies across its portfolio in parallel.

To support and manage this anticipated breadth of clinical studies, Moderna’s 200,000 square foot Norwood facility will enable the manufacture, quality, control and supply of clinical grade mRNA therapies and vaccines for Good Laboratory Practices (GLP) toxicology studies as well as Phase 1 and Phase 2 clinical studies. At the site, Moderna will carry out all manufacturing activities—from raw material production to active pharmaceutical ingredients (APIs), formulation, filling and finish. Moderna will begin initial build-out of the new facility in October 2016. The company plans to open the facility by early 2018.

“We are thrilled to announce the site selection for our GMP manufacturing facility, as this is a critical component of the early development engine we are building to advance many mRNA programs in parallel in human clinical studies,” said Stéphane Bancel, CEO of Moderna. “With our development pipeline expanding, we need to scale up from our current Cambridge-based GMP clinical supply manufacturing facility. Our Norwood facility will enable us to deliver our mRNA therapies and vaccines quickly, which will support rapid progression from development candidate nomination to human proof-of-concept. I’m very grateful to our team and our partners for enabling us to progress to this exciting next phase in Moderna’s history. With the addition of this new facility, we are moving a significant step closer to delivering novel mRNA medicines for patients.”

Moderna’s Norwood clinical supply facility will host GMP manufacturing and quality control; the company’s Personalized Cancer Vaccines unit; preclinical technical operations and testing; and general administrative functions to support these teams. At the facility’s opening, the annual GMP manufacturing capacity will be 40 GMP mRNA lots and is expected to scale up to over 100 GMP mRNA clinical scale lots annually in the future. Approximately 100 of Moderna’s current 460 team members will move from the company’s three existing locations in Cambridge, Mass., to the Norwood facility. In addition, Moderna plans to hire more than 100 new employees for the Norwood site.

“With the addition of this facility, we are pleased to expand our presence as a driver of innovation in Massachusetts. We appreciate the support we’ve received at both the state and local levels, and we look forward to creating new jobs and continuing to contribute to the life sciences community and the economy here in Massachusetts,” said Mr. Bancel.

“This is quite an ambitious project and will result in a truly unique facility,” said Steve Harbin, Senior Vice President, Manufacturing and Operations. “We are designing the Norwood site to accommodate a broad range of GMP manufacturing needs that reflect the diversity of our pipeline—from small-scale, rapid cycle-time manufacturing of personalized cancer vaccines to larger scale Phase 2 clinical study supply across a number of therapeutic areas. This will be among the most integrated end-to-end process facilities I have built and seen in my 25 years working in the pharmaceutical industry.”

In addition to the Norwood facility, other key components of Moderna’s early development engine include its collaborations with leading research organizations Charles River Laboratories, to support GLP toxicology studies, and Pharmaceutical Product Development, LLC (PPD) to support Phase 1 and Phase 2 studies. The company is also building a highly automated and digital enterprise to seamlessly integrate and orchestrate cloud-based IT systems to manage and industrialize the complex planning and execution of its mRNA pipeline scale-up at every stage of development. In addition, Moderna has amassed deep institutional expertise in the U.S. and global regulatory landscape. The ability to share and apply learnings from ongoing regulatory interactions across its ecosystem of internal therapeutically focused ventures and external partners generates a network effect that benefits Moderna and its partners, helping to advance programs through regulatory processes.

About Moderna Therapeutics

Moderna Contacts

Investors:
Maren Winnick
617-674-5297
maren.winnick@modernatx.com

Media:
Liz Melone
617-256-6622
liz.melone@modernatx.com

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Moderna Appoints Melissa J. Moore, Ph.D., as Chief Scientific Officer of Moderna’s mRNA Research Platform

Tue, 13 Sep 2016 19:35:57 GMT

Renowned UMass Medical School Professor and Founding Co-Director of the RNA Therapeutics Institute (RTI) to Lead Moderna’s mRNA Biology Research

Dr. Moore to Oversee Moderna’s Efforts to Further Advance Its Leading mRNA Platform to Promote Continued Pipeline Expansion and Deliver a New Class of Medicines for Patients

CAMBRIDGE, Mass., September 13, 2016 — Moderna Therapeutics, a clinical stage biotechnology company pioneering messenger RNA (mRNA) Therapeutics™ to create a new generation of transformative medicines for patients, today announced that Melissa J. Moore, Ph.D., has been appointed Chief Scientific Officer (CSO) of Moderna’s mRNA research platform. Responsible for leading all mRNA biology research at Moderna, Dr. Moore will play a critical leadership role as the company enters a new growth phase, and will help guide Moderna towards its vision of delivering on the promise of mRNA science for patients. Dr. Moore will assume her new role at Moderna as of October 3, 2016.

Currently a member of Moderna’s Scientific Advisory Board, Dr. Moore joins the company from the University of Massachusetts Medical School (UMMS), where she is currently Professor of Biochemistry & Molecular Pharmacology, Eleanor Eustis Farrington Chair in Cancer Research and Investigator at the Howard Hughes Medical Institute (HHMI). In addition, Dr. Moore was a founding Co-Director of the RNA Therapeutics Institute (RTI) at UMMS. The RTI's mission is to accelerate the translation of basic research discoveries into medicines. She was also instrumental in the creation of the Massachusetts Therapeutic and Entrepreneurship Realization Initiative (MassTERi), a faculty-led program intended to facilitate the translation of UMMS discoveries into drugs, products, technologies and companies. Upon joining Moderna, Dr. Moore will resign from HHMI, but retain her academic affiliation with UMMS as a part-time faculty member.

"We are excited and honored to have Melissa join our team,” said Stéphane Bancel, CEO of Moderna. “As we enter a new growth phase for Moderna, we continue to challenge ourselves to deliver on the broadest possible array of mRNA medicines. Messenger RNA biology is, of course, a foundational backbone of our company. Melissa is an exceptional scientist whose knowledge and expertise in this area will have an enormous impact on Moderna. And, importantly, she shares our vision and commitment to help patients and improve lives. We could not be more thrilled."

Dr. Moore’s research at UMMS and HHMI has encompassed a broad array of topics related to the role of RNA and RNA-protein (RNP) complexes in gene expression and touched on many human diseases including cancer, neurodegeneration and preeclampsia. Prior to UMMS, she spent 13 years as a professor at Brandeis University. She began working on RNA during her postdoctoral training with Phillip Sharp, Ph.D., Institute Professor, Koch Institute for Integrative Cancer Research at the Massachusetts Institute of Technology (MIT), where she also received her Ph.D. in Biological Chemistry. Dr. Moore also holds a B.S. in Chemistry and Biology from the College of William and Mary.

“As a member of Moderna’s Scientific Advisory Board, I've had a front row seat to experience their groundbreaking progress first-hand,” said Dr. Moore. “And through my work at the RTI and with MassTERi, I've come to appreciate the power of academic-industry collaboration and entrepreneurship. My new role at Moderna is a natural next step in my own progression as a basic researcher passionate about translating our ever-increasing knowledge of RNA biology into products that improve the human condition. I look forward to working with the incredibly talented Moderna team and building stronger connections and collaborations between Moderna and academia, as we work to advance the promise of mRNA therapeutics as a completely new modality for creating the medicines of the future.”

“As we prepare for the next stage of our growth, we have been looking for an exceptional scientific leader with expertise across molecular and cellular biology, biochemistry, and bioinformatics of mRNA to direct our basic science research efforts. But, just as important, we have been looking for someone who could mentor and inspire Moderna’s large and growing scientific team,” said Stephen Hoge, M.D., President of Moderna. “We feel incredibly fortunate to have found both in Melissa. Under her leadership we expect to accomplish great things on behalf of patients in the years ahead.”

About Moderna Therapeutics

Moderna Contacts

Investors:
Maren Winnick
617-674-5297
maren.winnick@modernatx.com

Media:
Liz Melone
617-256-6622
liz.melone@modernatx.com

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Moderna Announces Funding Award from BARDA for $8 Million with Potential of up to $125 Million to Accelerate Development of Zika Messenger RNA (mRNA) Vaccine

Wed, 07 Sep 2016 19:30:55 GMT

Company plans to file IND by end of 2016

CAMBRIDGE, Mass., September 7, 2016 — Moderna Therapeutics, a clinical stage biotechnology company pioneering messenger RNA (mRNA) Therapeutics™ to create a new generation of transformative medicines for patients, today announced a funding award of $8 million with the potential of up to $125 million from the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response ( ASPR ) within the U.S. Department of Health and Human Services (HHS), to accelerate development of a novel Zika mRNA vaccine.

Under the terms of the agreement, the initial $8 million award will support a Phase 1 clinical study, toxicology studies, vaccine formulation and manufacturing. The agreement includes options for additional funding up to $117 million to support Phase 2 and Phase 3 clinical studies, as well as large-scale manufacturing.

Moderna plans to file an investigational new drug (IND) application with the U.S. Food & Drug Administration (FDA) by the end of 2016. The company’s preclinical work for its Zika vaccine has been funded through a grant from the Defense Advanced Research Projects Agency (DARPA). Development efforts are being led by Valera, Moderna’s infectious disease-focused venture.

“We believe our mRNA vaccine technology offers potential advantages in efficacy, speed of development, and production scalability and reliability, which may position Moderna as a leader in preparing for and responding to infectious disease threats, such as Zika, that place millions of people at risk around the world,” said Stéphane Bancel, CEO of Moderna. “We feel a tremendous sense of responsibility to advance our Zika mRNA vaccine as quickly as possible, and we are thankful to BARDA for its commitment to support and help expedite our development efforts. We plan to initiate a Phase 1 study within the next several months.”

Moderna has two additional Phase 1 mRNA infectious disease vaccine studies currently underway in the US and Europe and has dosed approximately 250 healthy human volunteers to date. The company expects to publish clinical data on its first Phase 1 study in 2017. Additional areas of therapeutic focus for Moderna currently include rare diseases, immuno-oncology and cardiovascular disease, among others.

“With two mRNA infectious disease vaccines already advancing through clinical studies and a growing pipeline of vaccines, all based on the same underlying mRNA vaccine technology, we’re in the fortunate position of being able to rapidly apply learnings to inform our Zika vaccine development program,” said Michael Watson, President of Valera. “It’s clear the world needs novel, innovative approaches to address both known and future infectious disease threats. We hope to be at the forefront of advancing this innovation.”

About Moderna’s mRNA Vaccine Technology

Vaccines work by mimicking an infection from a known pathogen, such as a virus, without causing disease. They teach the immune system to recognize antigens, which are parts of pathogens. Current vaccines introduce antigens to the body as weakened or inactivated pathogens or as selected antigens produced outside the body. Moderna’s approach more closely mimics nature by delivering mRNA to the body’s cells, which, in turn, produce antigenic proteins as if the body was infected by a virus. These antigenic proteins are identified and remembered by the immune system. When a person is exposed to the pathogen in the future, the body is able to recognize it as foreign and mounts an immune response, including production of antibodies that can help to destroy the pathogen.

About Moderna Therapeutics

Moderna Contacts

Investors:
Maren Winnick
617-674-5297
maren.winnick@modernatx.com

Media:
Liz Melone
617-256-6622
liz.melone@modernatx.com

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Moderna Enters New Growth Phase with Closing of $474 Million Equity Financing and its First BARDA Grant for up to $125 Million for Zika mRNA Vaccine

Wed, 07 Sep 2016 19:24:37 GMT

CAMBRIDGE, Mass., September 7, 2016 — Moderna Therapeutics, a clinical stage biotech company pioneering mRNA Therapeutics™ to create a new generation of transformative medicines for patients, announced today that it has raised $474 million. This latest equity financing includes strong support from existing institutional investors and world-class strategic pharmaceutical partners as well as participation of new institutional investors from the United States, Europe and Asia.

With this financing completed, Moderna currently has $1.4 billion in cash on its balance sheet and the potential for over $230 million of additional available funds from grants committed by the Bill & Melinda Gates Foundation, the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS), and the Defense Advanced Research Projects Agency (DARPA).

“Moderna is, yet again in its short five years, accelerating its expansion and entering a new growth phase,” said Stéphane Bancel, CEO of Moderna. “Over the last nine months, our team has started two clinical trials, dosed around 250 healthy volunteers, filed an additional two INDs, moved into personalized cancer vaccine development with Merck and into lung mRNA drug discovery with Vertex, raised a new large equity financing of $474 million and closed our first BARDA grant for up to $125 million to advance a Zika mRNA vaccine. We are pivoting toward a new chapter in our company’s history, and we feel privileged to deploy our substantial capital resources to advance our mission to deliver on the promise of mRNA therapeutics as an entire new class of medicines.”

Moderna plans to use proceeds from the financing to support:

Continued growth of its clinical pipeline and accelerated advancement of development candidates into clinical studies;
Construction of a new Good Manufacturing Practice (GMP) manufacturing facility in anticipation of increasing clinical activities across therapeutic areas;
Sustained investment in its mRNA technology platform at the pace of $100 million per year; and
Continued expansion of its ecosystem into new therapeutic areas and modalities.

“We feel a tremendous sense of responsibility to work at a rapid pace to simultaneously advance mRNA medicines to address a broad array of significant unmet needs – from diseases that impact millions of people to ultra-rare diseases that impact a very few,” added Mr. Bancel. “We believe the capital we have raised to date, from a diverse syndicate of blue-chip investors as well as highly respected partners, is critical to deliver on Moderna’s unique, and potentially immense, opportunity to help patients.”

About Moderna Therapeutics

Moderna Contacts

Investors:
Maren Winnick
617-674-5297
maren.winnick@modernatx.com

Media:
Liz Melone
617-256-6622
liz.melone@modernatx.com

VIEW ALL NEWS

Moderna Therapeutics Provides Mid-Year Corporate Update, Announces Continued Clinical Development Progress and Pipeline Acceleration

Tue, 26 Jul 2016 19:20:38 GMT

Eleven mRNA-based development candidates nominated to date across ventures and partners

Two Phase 1 studies underway for mRNA-based infectious disease vaccines
CTA filed for Phase 1 study of AZD8601, AstraZeneca’s investigational mRNA-based Therapeutic that encodes for VEGF-A
Eight candidates advancing through IND-enabling studies

Several additional clinical studies planned to initiate in 2016

CAMBRIDGE, Mass., July 26, 2016 – Moderna Therapeutics, a clinical stage biotechnology company that is pioneering messenger RNA (mRNA) Therapeutics™ to create a new generation of transformative medicines for patients, today announced a mid-year corporate update, highlighting continued clinical development progress and acceleration of its pipeline. A Phase 1 study for mRNA 1851 is now underway in the U.S.; this is Moderna’s second clinical program. A Phase 1 study for mRNA 1440 continues enrollment in Europe. mRNA 1440 and mRNA 1851 are infectious disease vaccine candidates for undisclosed targets and indications that were developed utilizing Moderna’s proprietary mRNA vaccine technology. More than 200 healthy volunteers have been dosed to date across the two ongoing studies.

Today, Moderna and AstraZeneca announced the filing of a Clinical Trial Application (CTA) in Europe for a Phase 1 study of AZD8601, an investigational mRNA-based therapy that encodes for vascular endothelial growth factor-A (VEGF-A). AZD8601 is part of the collaboration announced between Moderna and AstraZeneca in 2013 to discover, develop and commercialize mRNA Therapeutics to treat serious cardiovascular, metabolic and renal diseases as well as cancer. AZD8601 is the first program from the collaboration to progress towards clinical trials. AZD8601 could one day provide a unique regenerative treatment option for patients with heart failure, diabetic wound healing and other ischemic vascular diseases.

Eight additional nominated mRNA development candidates are advancing through IND-enabling studies across a pipeline composed of Moderna’s internally led programs and partnered programs. Several of these candidates are on track to enter the clinic in 2016.

“In the first half of 2016, our team continued to make significant strides to advance our goal to deliver a new generation of transformative medicines for patients across a broad spectrum of diseases. In the five years since Moderna’s founding, we have initiated two clinical trials, filed a CTA for a third program and moved another eight development candidates into IND-enabling studies. We have amassed an incredibly talented and dedicated team and forged key partnerships with notable biopharma leaders who are dedicated to advancing mRNA Therapeutics to meet serious unmet medical needs,” said Stéphane Bancel, CEO of Moderna. “We look forward to a busy and productive second half of 2016 as we work to progress several additional candidates to the clinic.”

There are more than 90 discovery programs advancing across Moderna’s ecosystem of internal ventures and external partners. Current therapeutic areas of focus include infectious diseases, rare diseases, oncology, immuno-oncology and cardiovascular disease, among others.

1H 2016 Highlights

Expansion of Ecosystem to Advance mRNA Therapeutics for Unmet Needs

In 1H 2016, Moderna continued to expand its ecosystem and collaborate with key strategic partners to advance mRNA research and broaden the potential reach and impact of mRNA Therapeutics across various disease areas. Highlights include:

Immuno-Oncology Collaboration with AstraZeneca. In January, Moderna announced a new collaboration with AstraZeneca to discover, co-develop and co-commercialize immuno-oncology mRNA therapeutic candidates. The collaboration is in addition to the exclusive agreement announced by the companies in 2013 to develop mRNA Therapeutics for the treatment of cardiovascular, metabolic and renal diseases as well as selected targets in oncology.
Inclusion of New Infectious Disease Vaccine Program with Merck. In January, Moderna announced that Merck licensed a vaccine program against an undisclosed viral target, including mRNA 1566 and a set of related novel vaccine candidates, as part of the ongoing collaboration between the companies. The inclusion of this new program, which was not part of the original collaboration agreement, follows the rapid progress made in the first year of the collaboration.
Global Health Partnership with the Bill & Melinda Gates Foundation. Moderna also announced in January a partnership with the Bill & Melinda Gates Foundation to advance the development of a novel, affordable combination of mRNA-based antibody therapeutics to help prevent human immunodeficiency virus (HIV) infection. The global health partnership has the potential for follow-on projects to develop additional mRNA-based projects for various infectious diseases.

Strong Financial Position Affords Several Years of Runway

As of June 30, Moderna had approximately $1 billion in cash, pro forma for the cash payments connected with the Merck and Vertex collaborations. This affords Moderna several years of runway to support its continued growth and pipeline acceleration.

Moderna also reported the following financial results for 1H 2016:

GAAP revenues of $36 million
Cash inflows of approximately $80 million, consisting of milestone payments and reimbursements
Investments of $135 million to continue to advance its platform and pipeline

Establishment of Internal GMP Manufacturing Capabilities and Laboratory Expansion

As part of its efforts to support the accelerated output of its mRNA research and enable simultaneous clinical trials across various therapeutic areas, Moderna has expanded both its manufacturing capacity and its overall footprint in Cambridge, Mass. Highlights since January include:

Establishing a wholly owned and validated GMP manufacturing facility in Cambridge, Mass., that is producing clinical-grade product.
Increasing the square footage of office, lab and manufacturing space from 115,000 sq. ft. at the beginning of 2016 to 200,000 sq. ft. as of June 2016.

Growth of the Moderna Team

To support its clinical progress and discovery engine, Moderna has continued to grow its internal team. Highlights include:

Increasing employee headcount from 320 in January to 440 by the end of June.
Appointing Saqib Islam, former Executive Vice President and Chief Strategy and Portfolio Officer of Alexion Pharmaceuticals, as Moderna’s Chief Business Officer.
Appointing Dr. Michael Watson, MB ChB, MRCP, AFPM, former Global Head of Vaccination Policy and Advocacy at Sanofi Pasteur, as President of Valera, Moderna’s infectious disease-focused venture company.

Q2 2016 and Recent Highlights

Personalized Cancer Vaccines Collaboration with Merck. In June, Moderna announced a new strategic collaboration with Merck to advance novel mRNA-based personalized cancer vaccines with KEYTRUDA® (pembrolizumab) for the treatment of multiple types of cancer. The collaboration will leverage Moderna’s rapid cycle time, small-batch manufacturing to supply vaccines tailored to individual patients within weeks. Clinical study of the personalized cancer vaccine is anticipated to begin in 2017.
Research Collaboration with Vertex in Cystic Fibrosis. In July, Moderna announced an exclusive research collaboration and licensing agreement with Vertex Pharmaceuticals to discover and develop mRNA Therapeutics for the treatment of cystic fibrosis. The three-year collaboration will focus on the use of mRNA therapies, administered via pulmonary delivery, to treat the underlying cause of CF by enabling cells in the lungs to produce functional copies of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which is known to be defective in people with CF.

About Moderna Therapeutics

Moderna Contacts

Investors:
Maren Winnick
617-674-5297
maren.winnick@modernatx.com

Media:
Liz Melone
617-256-6622
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AstraZeneca and Moderna Announce Filing of First Clinical Trial Application in Messenger RNA Therapeutics™ Collaboration

Tue, 26 Jul 2016 19:17:03 GMT

CAMBRIDGE, Mass., and LONDON, UK, July 26, 2016 — AstraZeneca and Moderna Therapeutics today announced that AstraZeneca has filed a Clinical Trial Application (CTA) with the Paul Ehrlich Institute and the German Federal Ministry of Health to initiate a Phase 1 clinical trial of AZD8601. The program is part of a collaboration between AstraZeneca and Moderna to discover, develop and commercialize messenger RNA (mRNA) Therapeutics™ to treat serious cardiovascular, metabolic and renal diseases as well as cancer. It marks the first program resulting from the collaboration to progress towards clinical trials.

AZD8601 is an investigational mRNA-based therapy that encodes for vascular endothelial growth factor-A (VEGF-A). Its discovery and development has also involved collaboration with Kenneth Chien, M.D., Ph.D., Professor of Cellular and Molecular Biology and Medicine and his team at the Integrated Cardio-Metabolic Centre at Karolinska Institutet in Stockholm, Sweden.

Marcus Schindler, Vice President, Innovative Medicines & Early Development at AstraZeneca said, “Developing regenerative therapies for the treatment of cardiometabolic disease is a hugely exciting and innovative area. We believe that using mRNA Therapeutics to initiate a strong, local and transient surge of VEGF-A expression could help us overcome challenges associated with previous approaches to regulate this protein in tissues. AZD8601 could one day provide a unique regenerative treatment option for patients with heart failure, diabetic wound healing and other ischemic vascular diseases.”

Stéphane Bancel, Chief Executive Officer, Moderna said, “This marks a significant milestone for both Moderna and AstraZeneca as our first partnered mRNA program reaches the clinic. It is a validation of our shared vision to harness the potential of mRNA Therapeutics to address serious unmet needs with the goal of improving patients’ lives. This has been a highly collaborative partnership since its inception, and I want to recognize the tremendous work of all involved, including the AstraZeneca team, my Moderna colleagues and Dr. Chien and his team. Together, I believe we will continue to make significant strides that will push new boundaries in the treatment of cardiovascular and metabolic diseases.”

Dr. Chien said, “It has been rewarding working as an integrated academic partner with AstraZeneca in its joint work with Moderna to generate a complete package of strong pre-clinical data over the past three years since our initial publication in mouse studies, and we are thrilled that the clinical trial application has been filed.”

About AZD8601

mRNA is responsible for carrying genetic instructions transcribed from DNA, which cells then translate to produce proteins. Proteins are responsible for directing the body’s biological functions. Moderna’s pioneering mRNA Therapeutics are designed to trigger the cellular machinery to produce specific proteins. In this application, AZD8601 may enable the delivery of genetic instructions to spur the production of VEGF-A.

The AZD8601 program is built upon a decade of pioneering research on heart stem cells in cardiovascular development conducted by Dr. Chien, including the finding that VEGF-A can act as a cell fate switch for cardiac progenitors. ^i ii

i Lui KO, Zangi L, Silva EA, Bu L, Sahara M, Li RA, Mooney DJ, Chien KR. Driving vascular endothelial cell fate of human multipotent Isl1+ heart progenitors with VEGF modified mRNA. Cell Res. 2013 Oct;23(10):1172-86.

ii Zangi L, Lui KO, von Gise A, Ma Q, Ebina W, Ptaszek LM, Später D, Xu H, Tabebordbar M, Gorbatov R, Sena B, Nahrendorf M, Briscoe DM, Li RA, Wagers AJ, Rossi DJ, Pu WT, Chien KR. Modified mRNA directs the fate of heart progenitor cells and induces vascular regeneration after myocardial infarction. Nat Biotechnol. 2013 Oct;31(10):898-907.

About AstraZeneca

AstraZeneca is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialization of prescription medicines, primarily for the treatment of diseases in three therapy areas – Respiratory and Autoimmunity, Cardiovascular and Metabolic Diseases, and Oncology. The company is also active in inflammation, infection and neuroscience through numerous collaborations. AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. For more information please visit: www.astrazeneca.com .

About Moderna Therapeutics

Moderna Contacts

Investors:
Maren Winnick
617-674-5297
maren.winnick@modernatx.com

Media:
Liz Melone
617-256-6622
liz.melone@modernatx.com

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Vertex and Moderna Establish Exclusive Collaboration to Discover and Develop mRNA Therapeutics™ for Cystic Fibrosis

Wed, 06 Jul 2016 19:13:53 GMT

Collaboration to explore use of mRNA Therapeutics to treat the underlying cause of CF by enabling cells to produce functional CFTR proteins in the lungs

Moderna to receive $40 million upfront, made up of a $20 million cash payment and a $20 million convertible note investment, with potential for up to additional $275 million in milestones plus royalty payments

BOSTON AND CAMBRIDGE, MASS – July 6, 2016 — Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and Moderna Therapeutics today announced that the two companies have entered into an exclusive research collaboration and licensing agreement aimed at the discovery and development of messenger Ribonucleic Acid (mRNA) Therapeutics™ for the treatment of cystic fibrosis (CF). The three-year collaboration will focus on the use of mRNA therapies to treat the underlying cause of CF by enabling cells in the lungs to produce functional copies of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which is known to be defective in people with CF. Through the collaboration, the companies will explore the potential utilization of pulmonary mRNA delivery.

“We are excited to begin this collaboration with Moderna to further broaden our pipeline and support our goal of treating the underlying cause of CF for all people with this disease. The collaboration with Moderna underscores our commitment to invest in and pioneer multiple innovative approaches to expand and enhance the future treatment of CF,” said David Altshuler, M.D., Ph.D., Vertex’s Executive Vice President, Global Research and Chief Scientific Officer. “Moderna’s messenger RNA Therapeutics represent a promising new approach that could provide functional CFTR proteins to the lungs of people with CF, which would complement our existing approach of using CFTR modulators to treat CF.”

“Vertex’s deep scientific expertise and commitment to creating the best treatment regimens for cystic fibrosis patients make them an ideal partner to advance mRNA Therapeutics for this disease,” said Stéphane Bancel, Chief Executive Officer of Moderna. “This collaboration is illustrative of our consistent strategy to partner with world class therapeutic experts in order to maximize the potential of leveraging mRNA as a novel therapeutic approach across a spectrum of serious diseases. We’re particularly excited to explore the potential of pulmonary delivery as a new modality to bring transformative mRNA medicines to patients and address unmet needs.”

Under the terms of the collaboration, Vertex and Moderna will conduct exclusive research, development and commercialization activities to advance mRNA Therapeutics that aim to enable cells of people with CF to produce functional CFTR proteins in the lungs. Moderna will lead discovery efforts, leveraging its leading mRNA platform technology and mRNA delivery expertise along with Vertex’s scientific experience in CF biology and the functional understanding of CFTR, as well as the company’s proprietary assay platform that utilizes human bronchial epithelial (HBE) cells of multiple different CF gene mutations from people with CF. Vertex will lead all preclinical, development and commercialization activities associated with the advancement of mRNA Therapeutics that result from this collaboration and will fund all expenses related to the collaboration.

Vertex will pay Moderna $20 million in cash as part of its upfront commitment to the collaboration. Vertex will also make a $20 million investment in Moderna in the form of a convertible note that will convert to equity. The investment will provide Vertex with an ownership stake in Moderna. Vertex will also pay Moderna future development and regulatory milestones of up to $275 million, including $220 million in approval and reimbursement milestones, as well as tiered royalty payments on future sales.

About Moderna’s mRNA Therapeutics™

mRNA is responsible for carrying genetic instructions transcribed from DNA, which cells then translate to produce proteins that, when defective or missing, can underlie certain diseases, including CF. Moderna’s pioneering mRNA Therapeutics™ are designed to trigger the cellular machinery to utilize their natural processes to produce specific functional proteins. Through this collaboration, mRNA Therapeutics may be developed to enable the delivery of correct genetic instructions into cells in the lungs, which may trigger the cells to produce functional CTFR protein. This mRNA-based approach could be applicable to any person with CF regardless of a person’s specific CFTR mutations. Moderna currently has two Phase 1 clinical studies underway of mRNA vaccines for the prevention of infectious diseases. The company has over 90 discovery programs advancing across its ecosystem of therapeutically focused ventures and external partners that span rare diseases, infectious diseases, cancer and cardiovascular disease, among others.

Vertex’s CF Research and Development Pipeline

Vertex’s strategy in CF is to increase the number of people eligible for treatment with its two currently approved CF medicines and to develop new medicines that have the potential to treat all people with the disease in the future. Vertex’s two approved CF medicines, known as CFTR modulators, are designed to treat the defective CFTR protein in people with specific mutations in the CF gene. Beyond its two approved medicines, Vertex is also advancing multiple other potential medicines for the treatment of CF, including additional CFTR modulators as well as other collaborative approaches, including sodium channel inhibition, gene editing and, as announced today, mRNA-based therapies. The most advanced approach is the development of an additional two-drug combination of CFTR modulators, which is currently being evaluated in a broad Phase 3 development program. The company plans to advance a three-drug combination of CFTR modulators into Phase 2 development in people with CF in the second half of 2016.

About Cystic Fibrosis

Cystic fibrosis is a rare, life-shortening genetic disease affecting approximately 75,000 people in North America, Europe and Australia. Today, the median predicted age of survival for a person with CF is between 34 and 47 years, but the median age of death remains in the mid-20s.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are more than 1,900 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic, or genotyping test, lead to CF by creating non-working or too few CFTR protein at the cell surface. The defective function or absence of CFTR proteins in people with CF results in poor flow of salt and water into and out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.

About Vertex

Vertex is a global biotechnology company that aims to discover, develop and commercialize innovative medicines so people with serious diseases can lead better lives. In addition to our clinical development programs focused on cystic fibrosis, Vertex has more than a dozen ongoing research programs aimed at other serious and life-threatening diseases.

Founded in 1989 in Cambridge, Mass., Vertex today has research and development sites and commercial offices in the United States, Europe, Canada and Australia. For six years in a row, Science magazine has named Vertex one of its Top Employers in the life sciences. For additional information and the latest updates from the company, please visit www.vrtx.com .

Special Note Regarding Forward-looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, Dr. Altshuler's statements in the second paragraph of the press release, Mr. Bancel’s statements in the third paragraph of the press release and the information provided regarding the future development of mRNA therapies to treat the underlying cause of CF. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that data may not support further development of the mRNA therapies subject to the collaboration due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com . Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

Vertex Pharmaceuticals Incorporated

Investors:
Michael Partridge: 617-341-6108
or
Eric Rojas: 617-961-7205
or
Zach Barber: 617-341-6992

Media:
US: 617-341-6992 
Europe & Australia: +44 20 3204 5275
mediainfo@vrtx.com

About Moderna Therapeutics

Moderna Contacts

Investors:
Maren Winnick
617-674-5297
maren.winnick@modernatx.com

Media:
Liz Melone
617-256-6622
liz.melone@modernatx.com

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Merck and Moderna Announce Strategic Collaboration to Advance Novel mRNA-Based Personalized Cancer Vaccines with KEYTRUDA® (pembrolizumab) for the Treatment of Multiple Types of Cancer

Wed, 29 Jun 2016 19:05:57 GMT

Collaboration Combines Merck’s Leadership in Immuno-Oncology with Moderna’s Pioneering mRNA Vaccine Technology and Rapid Cycle Time, Small-Batch GMP Manufacturing Capabilities

Kenilworth, N.J. and Cambridge, Mass., June 29, 2016 — Merck (NYSE:MRK), known as MSD outside the United States and Canada, and Moderna Therapeutics today announced a strategic collaboration and license agreement to develop and commercialize novel messenger RNA (mRNA)-based personalized cancer vaccines. The collaboration will combine Merck’s established leadership in immuno-oncology with Moderna’s pioneering mRNA vaccine technology and GMP manufacturing capabilities to advance individually tailored cancer vaccines for patients across a spectrum of cancers.

Moderna and Merck will develop personalized cancer vaccines that utilize Moderna’s mRNA vaccine technology to encode a patient’s specific neoantigens, unique mutations present in that specific patient’s tumor. When injected into a patient, the vaccine will be designed to elicit a specific immune response that will recognize and destroy cancer cells. The companies believe that the mRNA-based personalized cancer vaccines’ ability to specifically activate an individual patient’s immune system has the potential to be synergistic with checkpoint inhibitor therapies, including Merck’s anti-PD-1 therapy, KEYTRUDA® (pembrolizumab). In addition, Moderna has developed a rapid cycle time, small-batch manufacturing technique that will uniquely allow the company to supply vaccines tailored to individual patients within weeks.

Under the terms of the agreement, Merck will make an upfront cash payment to Moderna of $200 million, which Moderna will use to lead all research and development efforts through proof of concept. The development program will entail multiple studies in several types of cancer and include the evaluation of mRNA-based personalized cancer vaccines in combination with Merck’s KEYTRUDA. Moderna will also utilize the upfront payment to fund a portion of the build-out of a GMP manufacturing facility in suburban Boston for the purpose of personalized cancer vaccine manufacturing.

Following human proof of concept studies, Merck has the right to elect to make an additional undisclosed payment to Moderna. If exercised, the two companies will then equally share cost and profits under a worldwide collaboration for the development of personalized cancer vaccines. Moderna will have the right to elect to co-promote the personalized cancer vaccines in the U.S. The agreement entails exclusivity around combinations with KEYTRUDA. Moderna and Merck will each have the ability to combine mRNA-based personalized cancer vaccines with other (non-PD-1) agents.

“Combining immunotherapy with vaccine technology may be a new path toward improving outcomes for patients,” said Dr. Roger Perlmutter, president, Merck Research Laboratories. “While the area of personalized cancer vaccine research has faced challenges in the past, there have been many recent advances, and we believe that working with Moderna to combine an immuno-oncology approach, using KEYTRUDA, with mRNA-based personalized cancer vaccines may have the potential to transform the treatment of cancer.”

“Our team has made significant progress since beginning our work in personalized cancer vaccines just last year. Through this collaboration with Merck, we are now well-positioned to accelerate research and development with a goal of entering the clinic in 2017, as well as to apply our unique GMP manufacturing capabilities to support the rapid production of these highly individualized vaccines,” said Stéphane Bancel, chief executive officer of Moderna. “We value our continued collaboration with Merck, and we look forward to working together to harness the potential of personalized cancer vaccines and immuno-oncology to bring a new treatment paradigm to patients.”

Merck and Moderna have an existing collaboration and license agreement focused on the discovery and development of mRNA-based infectious disease vaccines and passive immunity treatments. Moderna is also advancing its own pipeline of infectious disease vaccine candidates and currently has two phase 1 studies underway in Europe and the U.S.

About KEYTRUDA® (pembrolizumab) Injection 100 mg

KEYTRUDA is a humanized monoclonal antibody that works by increasing the ability of the body’s immune system to help detect and fight tumor cells. KEYTRUDA blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.

KEYTRUDA is also indicated for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 as determined by an FDA-approved test with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA. This indication is approved under accelerated approval based on tumor response rate and durability of response. An improvement in survival or disease-related symptoms has not yet been established. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

KEYTRUDA is administered at a dose of 2 mg/kg as an intravenous infusion over 30 minutes every three weeks for the approved indications.

Selected Important Safety Information for KEYTRUDA® (pembrolizumab)

Immune-mediated pneumonitis, including fatal cases, occurred in patients receiving KEYTRUDA. Pneumonitis occurred in 32 (2.0%) of 1567 patients with melanoma, including Grade 1 (0.8%), 2 (0.8%), and 3 (0.4%) pneumonitis. Pneumonitis occurred in 19 (3.5%) of 550 patients with NSCLC, including Grade 2 (1.1%), 3 (1.3%), 4 (0.4%), or 5 (0.2%) pneumonitis and more frequently in patients with a history of asthma/chronic obstructive pulmonary disease (5.4%) or prior thoracic radiation (6.0%). Monitor patients for signs and symptoms of pneumonitis. Evaluate suspected pneumonitis with radiographic imaging. Administer corticosteroids for Grade 2 or greater pneumonitis. Withhold KEYTRUDA for Grade 2; permanently discontinue KEYTRUDA for Grade 3 or 4 or recurrent Grade 2 pneumonitis.

Immune-mediated colitis occurred in 31 (2%) of 1567 patients with melanoma, including Grade 2 (0.5%), 3 (1.1%), and 4 (0.1%) colitis. Immune-mediated colitis occurred in 4 (0.7%) of 550 patients with NSCLC, including Grade 2 (0.2%) or 3 (0.4%) colitis. Monitor patients for signs and symptoms of colitis. Administer corticosteroids for Grade 2 or greater colitis. Withhold KEYTRUDA (pembrolizumab) for Grade 2 or 3; permanently discontinue KEYTRUDA for Grade 4 colitis.

Immune-mediated hepatitis occurred in patients receiving KEYTRUDA. Hepatitis occurred in 16 (1%) of 1567 patients with melanoma, including Grade 2 (0.1%), 3 (0.7%), and 4 (0.1%) hepatitis. Monitor patients for changes in liver function. Administer corticosteroids for Grade 2 or greater hepatitis and, based on severity of liver enzyme elevations, withhold or discontinue KEYTRUDA.

Hypophysitis occurred in 13 (0.8%) of 1567 patients with melanoma, including Grade 2 (0.3%), 3 (0.3%), and 4 (0.1%) hypophysitis. Hypophysitis occurred in 1 (0.2 %) of 550 patients with NSCLC, which was Grade 3 in severity. Monitor patients for signs and symptoms of hypophysitis (including hypopituitarism and adrenal insufficiency). Administer corticosteroids and hormone replacement as clinically indicated. Withhold KEYTRUDA for Grade 2; withhold or discontinue for Grade 3 or 4 hypophysitis.

Hyperthyroidism occurred in 51 (3.3%) of 1567 patients with melanoma, including Grade 2 (0.6%) and 3 (0.1%) hyperthyroidism. Hypothyroidism occurred in 127 (8.1%) of 1567 patients with melanoma, including Grade 3 (0.1%) hypothyroidism. Hyperthyroidism occurred in 10 (1.8%) of 550 patients with NSCLC, including Grade 2 (0.7%) or 3 (0.3%) hyperthyroidism. Hypothyroidism occurred in 38 (6.9%) of 550 patients with NSCLC, including Grade 2 (5.5%) or 3 (0.2%) hypothyroidism. Thyroid disorders can occur at any time during treatment. Monitor patients for changes in thyroid function (at the start of treatment, periodically during treatment, and as indicated based on clinical evaluation) and for clinical signs and symptoms of thyroid disorders. Administer replacement hormones for hypothyroidism and manage hyperthyroidism with thionamides and beta-blockers as appropriate. Withhold or discontinue KEYTRUDA for Grade 3 or 4 hyperthyroidism.

Type 1 diabetes mellitus, including diabetic ketoacidosis, occurred in 3 (0.1%) of 2117 patients. Monitor patients for hyperglycemia or other signs and symptoms of diabetes. Administer insulin for type 1 diabetes, and withhold KEYTRUDA and administer anti-hyperglycemics in patients with severe hyperglycemia.

Immune-mediated nephritis occurred in patients receiving KEYTRUDA. Nephritis occurred in 7 (0.4%) of 1567 patients with melanoma including, Grade 2 (0.2%), 3 (0.2%), and 4 (0.1%) nephritis. Monitor patients for changes in renal function. Administer corticosteroids for Grade 2 or greater nephritis. Withhold KEYTRUDA (pembrolizumab) for Grade 2; permanently discontinue KEYTRUDA for Grade 3 or 4 nephritis.

Other clinically important immune-mediated adverse reactions can occur. For suspected immune-mediated adverse reactions, ensure adequate evaluation to confirm etiology or exclude other causes. Based on the severity of the adverse reaction, withhold KEYTRUDA and administer corticosteroids. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Based on limited data from clinical studies in patients whose immune-related adverse reactions could not be controlled with corticosteroid use, administration of other systemic immunosuppressants can be considered. Resume KEYTRUDA when the adverse reaction remains at Grade 1 or less following corticosteroid taper. Permanently discontinue KEYTRUDA for any Grade 3 immune-mediated adverse reaction that recurs and for any life-threatening immune-mediated adverse reaction.

The following clinically significant, immune-mediated adverse reactions occurred in less than 1% (unless otherwise indicated) of 1567 patients with melanoma: arthritis (1.6%), exfoliative dermatitis, bullous pemphigoid, uveitis, myositis, Guillain-Barré syndrome, myasthenia gravis, vasculitis, pancreatitis, hemolytic anemia, and partial seizures arising in a patient with inflammatory foci in brain parenchyma. The following clinically significant, immune-mediated adverse reactions occurred in less than 1% of 550 patients with NSCLC: rash, vasculitis, hemolytic anemia, serum sickness, and myasthenia gravis.

Severe and life-threatening infusion-related reactions have been reported in 3 (0.1%) of 2117 patients. Monitor patients for signs and symptoms of infusion related reactions including rigors, chills, wheezing, pruritus, flushing, rash, hypotension, hypoxemia, and fever. For Grade 3 or 4 reactions, stop infusion and permanently discontinue KEYTRUDA.

In Trial 6, KEYTRUDA was discontinued due to adverse reactions in 9% of 555 patients with advanced melanoma; adverse reactions leading to discontinuation in more than one patient were colitis (1.4%), autoimmune hepatitis (0.7%), allergic reaction (0.4%), polyneuropathy (0.4%), and cardiac failure (0.4%). Adverse reactions leading to interruption of KEYTRUDA occurred in 21% of patients; the most common (≥1%) was diarrhea (2.5%). The most common adverse reactions with KEYTRUDA vs ipilimumab were fatigue (28% vs 28%), diarrhea (26% with KEYTRUDA), rash (24% vs 23%), and nausea (21% with KEYTRUDA). Corresponding incidence rates are listed for ipilimumab only for those adverse reactions that occurred at the same or lower rate than with KEYTRUDA (pembrolizumab).

In Trial 2, KEYTRUDA was discontinued due to adverse reactions in 12% of 357 patients with advanced melanoma; the most common (≥1%) were general physical health deterioration (1%), asthenia (1%), dyspnea (1%), pneumonitis (1%), and generalized edema (1%). Adverse reactions leading to interruption of KEYTRUDA occurred in 14% of patients; the most common (≥1%) were dyspnea (1%), diarrhea (1%), and maculo-papular rash (1%). The most common adverse reactions with KEYTRUDA vs chemotherapy were fatigue (43% with KEYTRUDA), pruritus (28% vs 8%), rash (24% vs 8%), constipation (22% vs 20%), nausea (22% with KEYTRUDA), diarrhea (20% vs 20%), and decreased appetite (20% with KEYTRUDA). Corresponding incidence rates are listed for chemotherapy only for those adverse reactions that occurred at the same or lower rate than with KEYTRUDA.

KEYTRUDA was discontinued due to adverse reactions in 14% of 550 patients with NSCLC. Serious adverse reactions occurred in 38% of patients. The most frequent serious adverse reactions reported at least 2% of patients were pleural effusion, pneumonia, dyspnea, pulmonary embolism, and pneumonitis. The most common adverse reactions (reported in at least 20% of patients) were fatigue (44%), cough (29%), decreased appetite (25%), and dyspnea (23%).

No formal pharmacokinetic drug interaction studies have been conducted with KEYTRUDA.

Safety and effectiveness of KEYTRUDA have not been established in pediatric patients.

Merck’s Focus on Cancer

Our goal is to translate breakthrough science into innovative oncology medicines to help people with cancer worldwide. At Merck Oncology, helping people fight cancer is our passion and supporting accessibility to our cancer medicines is our commitment. Our focus is on pursuing research in immuno-oncology and we are accelerating every step in the journey – from lab to clinic – to potentially bring new hope to people with cancer.

As part of our focus on cancer, Merck is committed to exploring the potential of immuno-oncology with one of the fastest-growing development programs in the industry. We are currently executing an expansive research program that includes more than 300 clinical trials evaluating our anti-PD-1 therapy across more than 30 tumor types. We also continue to strengthen our immuno-oncology portfolio through strategic acquisitions and are prioritizing the development of several promising immunotherapeutic candidates with the potential to improve the treatment of advanced cancers.

For more information about our oncology clinical trials, visit www.merck.com/clinicaltrials .

About Merck

For 125 years, Merck has been a global health care leader working to help the world be well. Merck is known as MSD outside the United States and Canada. Through our prescription medicines, vaccines, biologic therapies, and animal health products, we work with customers and operate in more than 140 countries to deliver innovative health solutions. We also demonstrate our commitment to increasing access to health care through far-reaching policies, programs and partnerships. For more information, visit www.merck.com and connect with us on Twitter , Facebook , YouTube and LinkedIn .

Merck Media Contacts:

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(267) 305-3558

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About Moderna Therapeutics

Moderna Contacts

Investors:
Maren Winnick
617-674-5297
maren.winnick@modernatx.com

Media:
Liz Melone
617-256-6622
liz.melone@modernatx.com

Forward-Looking Statement of Merck & Co., Inc., Kenilworth, N.J., USA

The company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise. Additional factors that could cause results to differ materially from those described in the forward-looking statements can be found in the company’s 2015 Annual Report on Form 10-K and the company’s other filings with the Securities and Exchange Commission (SEC) available at the SEC’s Internet site ( www.sec.gov ).

# # #

Please see Prescribing Information for KEYTRUDA (pembrolizumab) at: http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_pi.pdf and Patient Information/Medication Guide for KEYTRUDA at: http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_mg.pdf .

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Moderna Therapeutics and Charles River Laboratories Announce Strategic Collaboration to Scale Moderna’s Nonclinical Development Efforts for Novel mRNA Therapeutics

Mon, 06 Jun 2016 19:01:52 GMT

Partnership will support Moderna’s expanding pipeline and help accelerate discovery programs and development candidate progress into the clinic

CAMBRIDGE, Mass. and WILMINGTON, Mass., June 6, 2016 — Moderna Therapeutics, a clinical stage pioneer in the development of messenger RNA (mRNA) Therapeutics™ and Charles River Laboratories International, Inc. (NYSE: CRL), a leading early-stage contract research organization (CRO), today announced a strategic collaboration to support Moderna’s nonclinical discovery and development efforts. Charles River will be a key partner as Moderna continues to grow and advance its pipeline spanning multiple drug modalities and therapeutic areas, conducting nonclinical activities to progress development candidates through investigational new drug (IND)-enabling studies and into the clinic.

“With ten programs in development across our internal efforts and external collaborations, Moderna continues to generate development candidates and discovery programs at an accelerated pace through our unique mRNA therapeutics research engine. This strategic relationship will help enable the scale, efficiency and speed needed to support the full breadth of discovery programs underway and to continue advancing our development candidates,” said Stéphane Bancel, Chief Executive Officer of Moderna. “In particular, this collaboration will allow us to accelerate GLP toxicology study timelines, which will be instrumental as we continue to progress our development candidates into the clinic. Charles River’s expertise across discovery and nonclinical development activities, combined with their familiarity with our novel platform, make them an excellent partner for Moderna. The ability to work with Charles River in its Massachusetts facility will enhance the collaboration, given its proximity to our operations in Cambridge.”

Moderna’s pipeline is composed of a series of novel drug modalities, each representing a distinct application of the company’s proprietary core expression mRNA platform to encode proteins that achieve a therapeutic benefit. Moderna’s current modalities include infectious disease vaccines, personalized cancer vaccines, rare disease-associated intracellular/transmembrane liver proteins, intratumoral cancer therapy, and secreted antibodies and proteins. Moderna is leveraging these modalities to advance drugs across a broad spectrum of therapeutic areas via its wholly owned ventures as well as a growing ecosystem of partners.

For nearly 70 years, Charles River has been in the business of providing the research models required in research and development of new drugs, devices and therapies. Over this time, the company has expanded upon its core competency of in vivo biology to develop a diverse portfolio of discovery and safety assessment services, both Good Laboratory Practice (GLP) and non-GLP, which is able to support clients from target identification through preclinical development. Utilizing Charles River’s broad portfolio of products and services, which can be tailored to specific research requirements, enables clients to create a more flexible drug development model which reduces their costs, enhances their productivity and effectiveness, and increases speed to market.

“We are very pleased to enter into this strategic relationship with Moderna,” said James C. Foster, Chairman, President and Chief Executive Officer of Charles River Laboratories. “We look forward to employing our unique portfolio and extensive scientific expertise to support Moderna’s nonclinical discovery and development needs and advance its mRNA platform.”

About Charles River

Charles River provides essential products and services to help pharmaceutical and biotechnology companies, government agencies and leading academic institutions around the globe accelerate their research and drug development efforts. Our dedicated employees are focused on providing clients with exactly what they need to improve and expedite the discovery, early-stage development and safe manufacture of new therapies for the patients who need them. To learn more about our unique portfolio and breadth of services, visit www.criver.com .

Charles River Contacts

Investors:

Susan E. Hardy
781-222-6190
susan.hardy@crl.com

Media:
Amy Cianciaruso
781-222-6168
amy.cianciaruso@crl.com

About Moderna Therapeutics

Moderna is a clinical stage pioneer of messenger RNA Therapeutics™ , an entirely new in vivo drug technology that produces human proteins, antibodies and entirely novel protein constructs inside patient cells, which are in turn secreted or active intracellularly. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of diseases and conditions. Moderna is developing and plans to commercialize its innovative mRNA drugs through its own ventures and its strategic relationships with established pharmaceutical and biotech companies. Its current ventures are: Onkaido , focused on oncology, Valera , focused on infectious diseases, Elpidera , focused on rare diseases, and Caperna , focused on personalized cancer vaccines. Founded by Flagship VentureLabs™ , Cambridge-based Moderna is privately held and currently has strategic agreements with AstraZeneca , Alexion Pharmaceuticals , and Merck . To learn more, visit www.modernatx.com .

Moderna Contact

Investors:
Maren Winnick
617-674-5297
maren.winnick@modernatx.com

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Moderna Announces Appointment of Dr. Michael Watson as President of Valera

Mon, 04 Apr 2016 18:58:01 GMT

Former Global Head of Vaccination Policy and Advocacy at Sanofi Pasteur Will Lead Moderna’s Infectious Disease-Focused Venture Company

CAMBRIDGE, Mass., April 4, 2016 — Moderna Therapeutics, a clinical stage pioneer in the development of messenger RNA (mRNA) Therapeutics™ , announced today that Dr. Michael Watson, MB ChB, MRCP, AFPM, has been appointed President of Valera , one of Moderna’s four wholly owned venture companies. Valera is focused on the development and commercialization of mRNA-based vaccines and therapeutics for the prevention and treatment of viral, bacterial and parasitic infectious diseases.

“We are thrilled to have Mike assume the helm of Valera, particularly at this critical inflection point for the company, as we continue to progress our clinical stage infectious disease pipeline,” said Stéphane Bancel, chief executive officer (CEO) of Moderna. “Mike brings deep experience and expertise in global vaccine development and availability. Under his leadership, we will implement the strategies necessary to support development and potential commercialization of our mRNA therapeutics, which could address serious infectious disease needs in the U.S. and globally.”

Dr. Watson is a trained physician in internal medicine and infectious disease, bringing a twenty-year career in vaccine work to his new role with Moderna. Previously, Dr. Watson was Global Head of Vaccination Policy and Advocacy at Sanofi Pasteur. He has also held positions including Head of R&D for Acambis, UK Medical Director of Aventis Pasteur MSD as well as Head of Clinical and Epidemiology for SPMSD in France. His broad experience developing and licensing vaccines for conditions from smallpox, to encephalitis, to influenza and more has involved collaboration with key organizations such as the World Health Organization (WHO), Bill & Melinda Gates Foundation, GAVI (The Vaccine Alliance) and other government and non-governmental organizations. He has served as Chair of the Vaccines Committee and the Pandemic Influenza Preparedness Group of the International Federation of Pharmaceutical Manufacturers Association, (IFPMA) and is a member of the Board of Vaccines Europe and a board member of The Foundation for Innovative New Vaccines, FIND.

“The potential for mRNA therapeutics to meaningfully address some of the world’s most pressing infectious disease concerns is truly exciting. With this technology, I believe we have the opportunity to address serious unmet needs in infectious disease at an industry-leading pace. Moderna’s technology, coupled with its goal to deliver transformative medicines to millions of patients, makes this an extraordinary opportunity,” said Dr. Watson. “I am eager and proud to join Valera and work with this outstanding team and Moderna’s esteemed infectious disease-focused partners.”

Across its ecosystem of wholly owned ventures and external partners, Moderna is advancing a pipeline of more than 90 discovery programs, which span a broad spectrum of therapeutic areas. In addition to infectious diseases, these currently include rare diseases, oncology, immuno-oncology and cardiovascular disease, among others.

In January, Moderna announced that a Phase I study was underway in Europe for mRNA 1440, an infectious disease vaccine in development for an undisclosed target and indication. Moderna also announced a partnership with the Bill & Melinda Gates Foundation to advance the development of a novel, affordable combination of mRNA-based antibody therapeutics to help prevent human immunodeficiency virus (HIV) infection. The global health partnership may also include additional mRNA-based development projects for various infectious diseases. Moderna also has a license and collaboration agreement with Merck for the discovery and development of vaccines and passive immunity treatments against viral diseases using modified mRNA.

About Moderna Therapeutics

Moderna is a clinical stage pioneer of messenger RNA Therapeutics™ , an entirely new in vivo drug technology that produces human proteins, antibodies and entirely novel protein constructs inside patient cells, which are in turn secreted or active intracellularly. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of diseases and conditions. Moderna is developing and plans to commercialize its innovative mRNA drugs through its own ventures and its strategic relationships with established pharmaceutical and biotech companies. Its current ventures are: Onkaido , focused on oncology, Valera , focused on infectious diseases, Elpidera , focused on rare diseases, and Caperna , focused on personalized cancer vaccines. Founded by Flagship VentureLabs™ , Cambridge-based Moderna is privately held and currently has strategic agreements with AstraZeneca , Alexion Pharmaceuticals , and Merck . To learn more, visit www.modernatx.com .

Moderna Contacts

Investors:
Maren Winnick
617-674-5297
maren.winnick@modernatx.com

Media:
Liz Melone
617-256-6622
liz.melone@modernatx.com

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Moderna Therapeutics Appoints Saqib Islam as Chief Business Officer

Mon, 28 Mar 2016 18:55:08 GMT

Seasoned Industry Leader to Join Moderna’s Executive Team

CAMBRIDGE, Mass., March 28, 2016 — Moderna Therapeutics, a clinical stage pioneer in the development of messenger RNA (mRNA) Therapeutics™, announced today that Saqib Islam has been appointed Chief Business Officer, effective May 9th. As a member of the executive leadership team, among Mr. Islam’s areas of focus will be oversight of Moderna’s global strategic planning, corporate development and business development functions. Each of these functions will be critical to Moderna as the company progresses its pipeline and seeks to accelerate its commercial potential.

Mr. Islam joins Moderna from Alexion Pharmaceuticals, Inc., where he served as Executive Vice President, Chief Strategy and Portfolio Officer. In that role, he led Alexion’s Corporate Strategy, Corporate Development, Business Development, Enterprise Risk Management, Competitive Intelligence and Corporate Project Management functions and had primary responsibility for executing Alexion’s corporate growth strategies and contributing to its assessment and management of global operations. Previously, Mr. Islam worked for over 20 years in international business management with a focus on business development, strategic decision-making and planning, and capital markets.

“We are thrilled to welcome Saqib to our team; his expertise, intellect and drive for excellence are a perfect fit for Moderna. From his prior vantage point at Alexion, both as a partner and an investor, Saqib has had an in-depth view under the Moderna hood and knows of the unprecedented potential of our company,” said Stéphane Bancel, Chief Executive Officer at Moderna. “Our research engine is enabling us to amass a pipeline the scale of which has not previously been imagined. With his excellent business acumen and innovative thinking, Saqib will guide Moderna in architecting and implementing the business development and global strategic activities we will need to accommodate and maximize this potential.”

Mr. Islam’s extensive background in the healthcare banking sector includes previous positions of increasing responsibility in the investment banking divisions of Merrill Lynch, Morgan Stanley and Credit Suisse Securities. Earlier in his career, Mr. Islam provided strategic analysis and counseled clients across a broad set of industry segments for The Boston Consulting Group. He received a bachelor’s degree from McGill University and a J.D. from Columbia Law School.

“Already a clinical stage company, Moderna has extraordinary opportunities ahead of it, with its breakthrough science, accelerated discovery engine, and unique business model, and a demonstrated organizational agility with respect to advancing its pipeline,” said Mr. Islam. “I look forward to working closely with Stéphane and the leadership team as we evaluate these opportunities and evolve Moderna’s internal development and external partnering strategies to support the creation and commercialization of a new generation of transformative medicines for patients.”

Moderna’s pipeline is composed of a series of novel drug modalities, each representing a distinct application of the company’s proprietary core expression mRNA platform to encode proteins that achieve a therapeutic benefit. Moderna’s current modalities include infectious disease vaccines, personalized cancer vaccines, intracellular/transmembrane liver proteins, intratumoral cancer therapy, and secreted antibodies and proteins. Moderna is leveraging these modalities to advance drugs across a broad spectrum of therapeutic areas via its wholly owned ventures, as well as a growing number of partnering companies and organizations.

About Moderna Therapeutics

Moderna is a clinical stage pioneer of messenger RNA Therapeutics™ , an entirely new in vivo drug technology that produces human proteins, antibodies and entirely novel protein constructs inside patient cells, which are in turn secreted or active intracellularly. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of diseases and conditions. Moderna is developing and plans to commercialize its innovative mRNA drugs through its own ventures and its strategic relationships with established pharmaceutical and biotech companies. Its current ventures are: Onkaido , focused on oncology, Valera , focused on infectious diseases, Elpidera , focused on rare diseases, and Caperna , focused on personalized cancer vaccines. Founded by Flagship VentureLabs™ , Cambridge-based Moderna is privately held and currently has strategic agreements with AstraZeneca , Alexion Pharmaceuticals , and Merck . To learn more, visit www.modernatx.com .

Moderna Contacts

Investors:
Maren Winnick
617-674-5297
maren.winnick@modernatx.com

Media:
Liz Melone
617-256-6622
liz.melone@modernatx.com

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Moderna Therapeutics, Through Valera, its Infectious Disease Venture, Announces Initial Grant of up to $20 Million to Advance mRNA-Based Antibody Combination to Help Prevent HIV Infection

Tue, 12 Jan 2016 18:53:23 GMT

Bill & Melinda Gates Foundation to support development of HIV mRNA antibody program

Global health partnership may include additional mRNA-based development projects for various infectious diseases with funding up to a total of $100 Million

CAMBRIDGE, Mass., January 12, 2016 — Moderna Therapeutics, a pioneer in the development of messenger RNA (mRNA) Therapeutics™, today announced a partnership with the Bill & Melinda Gates Foundation to advance the development of a novel, affordable combination of mRNA-based antibody therapeutics to help prevent human immunodeficiency virus (HIV) infection. The development efforts will be led by Valera, Moderna’s infectious disease-focused venture.

The Gates Foundation has committed up to $20 million in grant funding to support Moderna’s evaluation of the antibody combination in a preclinical setting as well as the conduct of a first-in-human Phase 1 clinical study. Through an agreement between the two organizations, there may be potential for follow-on projects of up to $100 million total (including the HIV antibody project) to support the development of additional mRNA-based projects for various infectious diseases.

“Moderna Therapeutics’ research has considerable potential for the development of an effective prevention intervention for HIV, and potentially other infectious diseases that disproportionately affect the world’s poorest people,” said Trevor Mundel, president of Global Health at the Bill & Melinda Gates Foundation. “We look forward to collaborating with Moderna on the advancement of new tools to fight HIV.”

“We are honored that the Bill & Melinda Gates Foundation has selected Moderna as a partner, recognizing the tremendous potential of our mRNA drug platform to address some of the world’s most widespread and urgent health concerns,” said Stéphane Bancel, CEO of Moderna. “The foundation’s mission to help all people lead healthy and productive lives is well aligned with Moderna’s mission to deliver on the promise of transformative mRNA science to bring new medicines to patients. We are excited to initiate the first project with the foundation to develop an antibody-combination prevention modality to prevent HIV infection. Working together, I believe we will have a profound impact on millions of lives.”

Since Valera’s launch in January 2015, the venture has focused exclusively on utilizing Moderna’s platform to advance vaccines and therapeutics for the prevention and treatment of viral, bacterial and parasitic infectious diseases. Efforts at the venture already have yielded substantial results, including the demonstration of preclinical efficacy of Moderna’s mRNA-based vaccines in multiple viral disease models.

On January 11, 2016, Moderna announced that a Phase I study is currently underway in Europe for mRNA 1440, an infectious disease vaccine being developed by Valera for an undisclosed target and indication. The company also announced that an investigational new drug (IND) application has been filed with the U.S. Food and Drug Administration (FDA) for a second, undisclosed infectious disease vaccine, mRNA 1850, with a Phase I study anticipated to commence in Q1 2016.

Valera will apply its mRNA vaccine platform and Moderna’s drug platform to its programs of focus for the Bill & Melinda Gates Foundation partnership.

About Valera, a Moderna Venture

Valera, a Moderna venture, is focused exclusively on the advancement of vaccines and therapeutics for the prevention and treatment of infectious diseases. Valera is leveraging Moderna’s messenger RNA Therapeutics ^™ platform, an entirely new in vivo drug technology that produces human proteins, antibodies and entirely novel protein constructs inside patient cells, which are in turn secreted or active intracellularly. For more information please visit www.modernatx.com/ventures .

About Moderna Therapeutics

Moderna is a clinical stage pioneer of messenger RNA Therapeutics™ , an entirely new in vivo drug technology that produces human proteins, antibodies and entirely novel protein constructs inside patient cells, which are in turn secreted or active intracellularly. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of diseases and conditions. Moderna is developing and plans to commercialize its innovative mRNA drugs through its own ventures and its strategic relationships with established pharmaceutical and biotech companies. Its current ventures are: Onkaido , focused on oncology, Valera , focused on infectious diseases, Elpidera , focused on rare diseases, and Caperna , focused on personalized cancer vaccines. Founded by Flagship VentureLabs™ , Cambridge-based Moderna is privately held and currently has strategic agreements with AstraZeneca , Alexion Pharmaceuticals , and Merck .To learn more, visit www.modernatx.com .

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Moderna Licenses New Vaccine Candidates Against a New Viral Target to Merck

Mon, 11 Jan 2016 18:51:34 GMT

Ongoing collaboration between Moderna and Merck is yielding rapid progress; First vaccine candidate anticipated to enter the clinic in 2016

CAMBRIDGE, Mass., January 11, 2016 — Moderna Therapeutics, a pioneer in the development of messenger RNA (mRNA) Therapeutics ^™ , today announced that Merck, known as MSD outside the United States and Canada, has licensed a vaccine program against an undisclosed viral target, including mRNA 1566 and a set of related novel vaccine candidates, as part of the ongoing collaboration between the companies to discover and develop vaccines and passive immunity treatments against viral diseases using modified messenger RNA (mRNA). The inclusion of this new program, which was not part of the original collaboration agreement, follows the rapid progress made in the first year of the collaboration. Moderna’s contribution is led by Valera, its infectious disease-focused venture.

“Since signing our first agreement in January 2015, the Merck and Moderna teams have accomplished something quite unprecedented in drug development. In just ten months, they were able to move an idea for a new vaccine from concept clear through to the nomination of a vaccine candidate that entered pre-clinical development in November 2015. That innovative vaccine candidate is anticipated to enter the clinic in 2016,” said Stéphane Bancel, Chief Executive Officer (CEO) of Moderna. “I am very proud of the work of the teams over the past year. It is rewarding to see the remarkable success the collaboration has yielded thus far, as well as Merck’s desire to pursue an additional strategically important target and develop more medicines together.”

Under the terms of the amendment, Moderna has licensed a vaccine program against an undisclosed viral target, including mRNA 1566 and a set of related novel vaccine candidates to Merck. mRNA 1566 has shown promising results in early development efforts undertaken by the Valera team. Moderna will receive an upfront payment for this program and will be eligible to receive development, regulatory and commercial milestone payments related to the new target, as well as tiered royalties on net sales of resulting products. Consistent with the existing collaboration, Merck will lead the development of the candidate and commercialization of any products resulting from the agreement, while Moderna will design and synthesize the mRNA vaccine candidates directed against selected targets. As part of the initial three-year research agreement announced in January 2015, Merck made an upfront cash payment of $50 million and a $50 million equity investment to utilize granted licenses to commercialize five product candidates.

“The results generated by our initial efforts through this collaboration provide strong evidence for deploying Moderna’s messenger RNA technology against important viral targets,” said Daria Hazuda, vice president, Discovery, Infectious Disease and Vaccines, Merck Research Laboratories. “We are excited to add this new program to the focus of our agreement, and look forward to our continued work with the team.”

Moderna’s pipeline is composed of a series of novel drug modalities, each representing a distinct approach to using the company’s novel mRNA expression platform to encode proteins that achieve a therapeutic benefit. Moderna’s current modalities include infectious disease vaccines, personalized cancer vaccines, intracellular/transmembrane proteins, intratumoral cancer therapy, and secreted antibodies and proteins. Moderna is leveraging these modalities to advance drugs across a broad spectrum of therapeutic areas via its therapeutically focused ventures and its strategic partnerships. In addition to Valera, Moderna’s ventures also include Elpidera, focused on rare diseases; Onkaido, focused on oncology; and Caperna, focused on personalized cancer vaccines.

About Valera, a Moderna Venture

About Moderna Therapeutics

Moderna Therapeutics is a clinical stage pioneer of messenger RNA Therapeutics™, an entirely new in vivo drug technology that produces human proteins, antibodies and entirely novel protein constructs inside patient cells, which are in turn secreted or active intracellularly. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of diseases and conditions. Moderna is developing and plans to commercialize its innovative mRNA medicines through its own ventures and its strategic relationships with established pharmaceutical and biotech companies. Its current ventures include Valera (infectious diseases), Onkaido (immuno-oncology), Caperna (personalized cancer vaccines) and Elpidera (rare diseases). Founded by Flagship VentureLabs®, Cambridge-based Moderna is privately held and currently has strategic agreements with AstraZeneca , Alexion Pharmaceuticals and Merck . To learn more, visit www.modernatx.com .

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Moderna Therapeutics Announces Transition to a Clinical Stage Company, Provides Business Update and Outlines 2016 Strategic Priorities

Mon, 11 Jan 2016 18:49:36 GMT

First Phase I study underway in Europe for mRNA 1440; IND for second program, mRNA 1851, filed with the U.S. FDA

Ecosystem of ventures and partners saw significant growth in 2015, propelling rapid expansion of pipeline spanning multiple mRNA-based drug modalities and therapeutic areas

2016 goals focused on advancing multiple clinical trials, fueling continuing early exploration of mRNA modalities and extending investment in core mRNA platform and ventures

CAMBRIDGE, Mass., January 11, 2016 — Moderna Therapeutics, a pioneer in the development of messenger RNA (mRNA) Therapeutics™, today announced it is now a clinical stage company with its first Phase I study for mRNA 1440 currently underway in Europe. Moderna also announced it has filed an investigational new drug (IND) application with the U.S. Food and Drug Administration (FDA) for mRNA 1851, its second clinical program, with a Phase I study anticipated to begin in Q1 2016. mRNA 1440 and mRNA 1851 are infectious disease vaccines for undisclosed targets and indications. Moderna’s Chief Executive Officer, Stéphane Bancel, made the announcement during a presentation today at the J.P. Morgan 34th Annual Healthcare Conference in San Francisco, Calif. Mr. Bancel highlighted the company’s broad accomplishments in 2015 and outlined key focus areas for 2016.

“2015 was a year of tremendous growth and progress for Moderna. With the introduction of three new ventures and the advancement of key external collaborations, we have established a unique infrastructure that is enabling us to amass a pipeline that covers an unprecedented number of drug modalities and therapeutic targets,” said Stéphane Bancel, CEO of Moderna. “Advancing to the clinic certainly was the most gratifying milestone. We are now a major step closer to delivering on the promise of transformative mRNA science to bring breakthrough medicines to patients across a vast array of diseases, including infectious diseases, rare diseases, cancer and cardiovascular diseases, among others. I am deeply grateful to our wonderful team who has made all of these successes happen.”

2015 and Recent Highlights

Expansion of the Moderna Ecosystem

In 2015, Moderna executed on its strategic plan to rapidly expand its ecosystem, which allows for simultaneous progression of both internal development programs as well as programs through key external partnerships. Highlights included:

Launching three new therapeutic area-focused ventures: Valera , focused on infectious diseases; Elpidera , focused on rare diseases; and Caperna , focused on personalized cancer vaccines. The new ventures joined Onkaido , the company’s first venture focused on oncology, which launched in 2014.
Announcing a three-year license and collaboration agreement with Merck to develop messenger mRNA-based antiviral vaccines and passive immunity therapies for four undisclosed viruses. Merck is expected to enter the clinic with its first mRNA Therapeutics program in 2016.
Accelerating ongoing relationship with existing partners AstraZeneca and Alexion to advance mRNA research programs for cardiovascular and rare diseases, respectively, with both companies expected to be in position to advance their first mRNA Therapeutics programs to the clinic in 2016.
Extending existing grant from the Defense Advanced Research Projects Agency (DARPA) to finance preclinical toxicology studies and the Phase I study of mRNA 1388 expected to begin in 2016. This extension builds on the original grant announced in 2013 to develop antibody-producing drugs to protect against a wide range of known and unknown emerging infectious diseases and engineered biological threats.

Moderna announced today a new collaboration with AstraZeneca to discover, co-develop and co-commercialize immuno-oncology mRNA therapeutic candidates. The collaboration is in addition to the exclusive agreement announced by the companies in 2013 to develop mRNA Therapeutics for the treatment of cardiovascular, metabolic and renal diseases as well as selected targets in oncology.

Moderna also announced today that Merck has licensed a vaccine program against an undisclosed viral target, including mRNA 1566 and a set of related novel vaccine candidates, as part of the ongoing collaboration between the companies. The inclusion of this new program, which was not part of the original collaboration agreement, follows the rapid progress made in the first year of the collaboration.

Becoming a Clinical Stage Company

In 2015, Moderna became a clinical stage company with the initiation of its first Phase I study for mRNA 1440 and submission of a regulatory filing for a second clinical program, mRNA 1851. Highlights included:

Initiating first clinical trial; 12 healthy volunteers already dosed in ongoing Phase I study for mRNA 1440.
Filing an IND with the U.S. FDA to advance a second program, mRNA 1851, to the clinic, with the Phase 1 study anticipated to begin in Q1 2016.

To support its evolution as a clinical stage company, in January 2016, Moderna announced a strategic collaboration with Pharmaceutical Products Development, LLC (PPD). PPD’s support will include investigational new drug (IND) strategic planning, as well as clinical trial design and execution.

Rapid Pipeline Advancement

Across its ecosystem of ventures and partner collaborators, Moderna continued to accelerate its pipeline, with multiple research programs rapidly advancing across modalities and therapeutic areas.

Robust Financial Position Enabled by Landmark Funding

In 2015, Moderna announced $500 million in new equity financing to support the expansion of its platform and pipeline. Since the company’s founding in 2011 by Flagship VentureLabs™, Moderna has secured $1.2 billion in funding through financing activities and commercial partnerships. Additional highlights included:

Starting and ending the year with approximately $800 million of cash, while investing approximately $175 million to build Moderna’s business.

Growth of Internal Expertise and Infrastructure

To help guide its rapid evolution as a clinical stage company, Moderna made key additions to its senior leadership team and Board of Directors, significantly grew its employee base and enhanced its infrastructure and capabilities in 2015. Highlights included:

Appointing to its board of directors, Elizabeth (Betsy) Nabel, M.D., President of Harvard-affiliated Brigham and Women’s Health Care.
Appointing Tal Zaks, M.D., former Senior Vice President and Head of Sanofi Global Oncology, as Moderna’s Chief Medical Officer.
Appointing Marcello Damiami, technology and life sciences sectors IT expert from Motorola and bioMerieux, as Chief Digital Officer.
Appointing Jeff Hrkach, formerly Chief Technology Officer of BIND Therapeutics, as Head of Delivery Innovation.
Expanding headcount from 192 to 326 team members.
Signing a lease at 500 Technology Square in Cambridge, Mass., to accommodate the company’s research and development expansion, which will bring Moderna’s total footprint in Cambridge to more than 170,000 square feet this year.

2016 Business Objectives: Sustained, Cross-Ecosystem Growth Trajectory

Mr. Bancel mapped out key areas of focus for Moderna in the year ahead as the company continues to progress as a clinical stage, patient-facing enterprise, including:

Moving several more drugs into the clinic in 2016 across both internal and external development programs in various therapeutic areas of high unmet need.
Advancing several additional discovery programs to start preclinical toxicology studies in 2016, with the aim of initiating clinical studies in 2017.
Building a high-quality, scalable clinical development engine to match the accelerated output of Moderna’s mRNA research, enabling multiple drugs to enter the clinic and be studied in clinical trials in parallel, over the next 24 months.
Investing approximately $350 million in Moderna’s venture programs as well as its core mRNA platform to continue to build the industry’s leading mRNA operating system.
Growing Moderna’s team to 500 members by end of 2016.

“It’s remarkable to consider all that we have been able to accomplish since the company’s inception just over four years ago,” said Mr. Bancel. “Our success to date speaks to the validity and superiority of our novel mRNA approach, the vision and dedication of our team and the continued support and commitment of our partners and investors. I believe this trajectory will define Moderna in 2016 and for many years to come.”

About Moderna Therapeutics

Moderna is a clinical stage pioneer of messenger RNA Therapeutics™ , an entirely new in vivo drug technology that produces human proteins, antibodies and entirely novel protein constructs inside patient cells, which are in turn secreted or active intracellularly. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of diseases and conditions. Moderna is developing and plans to commercialize its innovative mRNA drugs through its own ventures and its strategic relationships with established pharmaceutical and biotech companies. Its current ventures are: Onkaido , focused on oncology, Valera , focused on infectious diseases, Elpidera , focused on rare diseases, and Caperna , focused on personalized cancer vaccines. Founded by Flagship VentureLabs™ , Cambridge-based Moderna is privately held and currently has strategic agreements with AstraZeneca , Alexion Pharmaceuticals and Merck . To learn more, visit www.modernatx.com .

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AstraZeneca and Moderna Therapeutics Announce New Collaboration to Co-Develop and Co-Commercialize Immuno-Oncology mRNA Therapeutics™

Mon, 11 Jan 2016 18:46:51 GMT

Moderna to lead preclinical development; AstraZeneca to lead clinical development; Moderna to co-commercialize and share profits on resulting products in the US

CAMBRIDGE, Mass., and LONDON, UK, January 11, 2016 — AstraZeneca, along with its global biologics research and development arm, MedImmune, and Moderna Therapeutics today announced a new collaboration to discover, co-develop and co-commercialize messenger RNA (mRNA) therapeutic candidates for the treatment of a range of cancers. The collaboration is in addition to the agreement announced by the companies in 2013 to develop mRNA Therapeutics™ for the treatment of cardiovascular, metabolic and renal diseases as well as selected targets in oncology.

The collaboration will combine MedImmune’s protein engineering and cancer biology expertise with Moderna’s mRNA platform. mRNA-based therapies are an innovative treatment approach that enables the body to produce therapeutic protein in vivo, opening up new treatment options for a wide range of diseases that cannot be addressed today using existing technologies.

Under the terms of the new agreement, AstraZeneca and Moderna, a pioneer of mRNA Therapeutics™, have agreed to collaborate on two specific immuno-oncology programs, based on promising pre-clinical data, including pharmacology in tumor models. Moderna will fund and be responsible for discovery and preclinical development of product candidates, with the aim of delivering one Investigational New Drug (IND) application-ready molecule for each of the two programs. Moderna’s efforts will be led by its oncology-focused venture, Onkaido. AstraZeneca will be responsible for early clinical development, led by MedImmune, and Moderna and AstraZeneca will share the costs of late-stage clinical development. The two companies will co-commercialize resulting products in the US under a 50:50 profit sharing arrangement. AstraZeneca will lead ex-US commercialization efforts, with Moderna receiving tiered royalties up to substantial double digits on ex-US sales.

Pascal Soriot, Chief Executive Officer, AstraZeneca, said: “We’re pleased to be expanding our relationship with Moderna with this new collaboration, to advance the potential of pioneering messenger RNA technology in developing game-changing new treatments for cancer patients.”

“Since our companies’ original strategic agreement in March 2013, Moderna’s relationship with AstraZeneca has been very fruitful. This new agreement with AstraZeneca demonstrates the effectiveness of our existing relationship and the power of our mRNA technology,” said Stéphane Bancel, Chief Executive Officer of Moderna. “We’re gratified to deepen our relationship with AstraZeneca and MedImmune with this major initiative, and we look forward to getting underway immediately with our new joint immuno-oncology programs.”

Under the companies’ original strategic agreement, AstraZeneca holds exclusive access to select any target of its choice in cardiometabolic diseases, as well as select targets in oncology, over a period of up to five years for subsequent development in mRNA. Several projects are progressing towards clinical development under the arrangement, and a first-in-human study is expected to commence in late 2016.

With its novel technology to enable mRNA as a drug, Moderna is building a fully scaled drug discovery and development platform centered on the rapid and low-cost design, delivery and production of mRNA drug candidates. Moderna is also advancing an innovative business model built on the decentralization of drug development activities. With an ecosystem of in-house concept development, ventures focused on therapeutic areas and a cluster of major pharma and biotech partners, Moderna is enabling more than 90 discovery and preclinical programs today across oncology, infectious diseases, rare diseases and cardiovascular diseases. Moderna’s pipeline also covers a broad expanse of novel drug modalities, each representing a distinct approach to using the company’s novel mRNA expression platform to encode proteins that achieve a therapeutic benefit.

About MedImmune

MedImmune is the global biologics research and development arm of AstraZeneca, a global, innovation-driven biopharmaceutical business that focuses on the discovery, development and commercialization of small molecule and biologic prescription medicines. MedImmune is pioneering innovative research and exploring novel pathways across key therapeutic areas, including respiratory, inflammation and autoimmunity; cardiovascular and metabolic disease; oncology; neuroscience; and infection and vaccines. The MedImmune headquarters is located in Gaithersburg, Md., one of AstraZeneca’s three global R&D centers, with additional sites in Cambridge, UK and Mountain View, CA. For more information, please visit www.medimmune.com .

About AstraZeneca in Oncology

Oncology is a therapy area in which AstraZeneca has deep-rooted heritage. It will be potentially transformational for the company’s future, becoming the sixth growth platform. Our vision is to help patients by redefining the cancer treatment paradigm and one day eliminate cancer as a cause of death. By 2020, we are aiming to bring at least six new cancer medicines to patients.

Our broad pipeline of next-generation medicines is focused on four main disease areas – lung, ovarian, breast and haematological cancers. These are being targeted through four key platforms – immuno-oncology, the genetic drivers of cancer and resistance, DNA damage repair and antibody drug conjugates – with a strong focus on combinations. Our recently announced investment in Acerta Pharma also adds the potentially transformational BTK inhibitor class of treatments to our portfolio, subject to closure in the first quarter of 2016, strengthening further our focus on targeted therapies.

About AstraZeneca

AstraZeneca is a global, innovation-driven biopharmaceutical business that focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of diseases in three main therapy areas - respiratory, inflammation, autoimmune disease, cardiovascular and metabolic disease and oncology – as well as in infection and neuroscience. AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. For more information please visit: www.astrazeneca.com

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Moderna Therapeutics to Present at 2016 J.P. Morgan Healthcare Conference

Mon, 04 Jan 2016 18:43:08 GMT

CAMBRIDGE, Mass., January 4, 2016 — Moderna Therapeutics, a pioneer in the development of messenger RNA (mRNA) Therapeutics™, announced today that Stéphane Bancel, Chief Executive Officer, will present a company overview and pipeline update at the 34th Annual J.P. Morgan Healthcare Conference.

Mr. Bancel will focus on Moderna’s progress to accelerate drug discovery and expand the company’s clinical development pipeline across an expansive number of drug modalities and therapeutic areas. Moderna’s proprietary approach utilizes mRNA to instruct native cellular machinery to make proteins in vivo, from native proteins to antibodies and other entirely novel protein constructs that can have therapeutic activity inside and outside of cells.

Moderna’s pipeline includes programs being pursued by the company’s four ventures:

Valera - Infectious Diseases
Elpidera - Rare Diseases
Onkaido - Immuno-Oncology
Caperna – Personalized Oncology Vaccines

as well as programs being advanced with its partners: Astra-Zeneca (NYSE:AZN), Alexion (NASDAQ:ALXN), Merck (NYSE:MRK) and DARPA.

Mr. Bancel’s presentation will take place on Monday, January 11, 2016 at 2:00 p.m. PT in Elizabethan C/D at the Westin St. Francis Hotel in San Francisco.

For more information on Moderna Therapeutics, including how mRNA works, partnership opportunities and job opportunities, visit www.modernatx.com .

About Moderna Therapeutics

Moderna Therapeutics is a clinical stage pioneer of messenger RNA Therapeutics™ , an entirely new in vivo drug technology that produces human proteins, antibodies and entirely novel protein constructs inside patient cells, which are in turn secreted or active intracellularly. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of diseases and conditions. The company currently plans to develop and commercialize its innovative mRNA drugs through a combination of strategic relationships as well as newly formed ventures, including Onkaido Therapeutics LLC , focused on oncology drug development, Valera LLC , focused on infectious diseases, Elpidera LLC , focused on rare diseases, and Caperna LLC , focused on personalized cancer vaccines. Founded by Flagship VentureLabs® , Cambridge-based Moderna is privately held and currently has strategic agreements with AstraZeneca , Alexion Pharmaceuticals , and Merck . To learn more, visit www.modernatx.com .

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Dr. Elizabeth Nabel Joins Moderna Therapeutics’ Board of Directors

Tue, 15 Dec 2015 20:15:45 GMT

CAMBRIDGE, Mass., December 15, 2016 — Moderna Therapeutics, a pioneer in the development of messenger RNA (mRNA) Therapeutics™, announced today that Elizabeth (Betsy) Nabel, M.D., President of Harvard-affiliated Brigham and Women’s Health Care (BWHC) has been appointed to the company’s Board of Directors.

Dr. Nabel is a distinguished cardiologist and biomedical researcher and a Professor of Medicine at Harvard Medical School. Since assuming the helm of BWHC in 2010, she has helped advance a new model of medicine for the institution by strategically focusing on innovation in healthcare, research and discovery, and in personalized and precision medicine. Among the many initiatives she has pioneered, under her guidance and oversight, BWHC launched a new translational research and clinical facility and a $1 billion campaign to advance healthcare innovation. Dr. Nabel is also the inaugural Chief Health and Medical Advisor to the National Football League (NFL), where she provides strategic input on the NFL’s ongoing medical, health and scientific efforts.

“We are thrilled and honored to have Dr. Nabel join Moderna’s Board of Directors. At this critical phase of the company’s evolution, as Moderna expands its clinical development pipeline, Betsy will bring critical insights from her extensive experience as a renowned clinician, researcher and global healthcare leader,” said Noubar Afeyan, Ph.D., co-founder and Chairman, Moderna Therapeutics, and Managing Partner and CEO, Flagship Ventures.

“Betsy’s pioneering expertise in translational research is well-aligned with our rapid progress of our mRNA therapeutics from scientific concept to real-world medical solutions for a broad spectrum of diseases and unmet patient needs,” said Stéphane Bancel, Moderna’s Chief Executive Officer. “As we advance toward 2016, a year that will hold new, clinical milestones for our company, we look forward to leveraging Betsy’s many contributions.”

Dr. Nabel previously served as the Director of the National Institute of Health’s National Heart, Lung and Blood Institute (NHLBI) where she leveraged the $3 billion research portfolio to establish pioneering scientific programs in genomics, stem cells and translational research. One of Dr. Nabel’s signature advocacy efforts was the Red Dress Heart Truth campaign, which raises heart awareness in women through unprecedented industry partnerships. A long-time champion for global health, while at NHLBI, Dr. Nabel established Centers of Excellence in developing countries to combat cardiovascular and lung diseases. At BWHC she helped create a national teaching hospital in Haiti and is advancing training for clinicians in under-resourced countries. An accomplished physician-scientist, Dr. Nabel’s work on the molecular genetics of cardiovascular diseases has yielded 17 patents and resulted in more than 250 scientific publications. Among a series of career-spanning accolades, Dr. Nabel has been elected to the American Academy of the Arts and Sciences, The National Academy of Medicine, the Association of American Physicians and the American Society of Clinical Investigation. She is also a Fellow of the American Association for the Advancement of Science.

“I am honored to join this distinguished Board of Directors at this exciting time,” said Dr. Nabel. “There is great potential for mRNA therapeutics to transform the future of medicine and expand the boundaries of science to improve the lives of patients.”

About Moderna Therapeutics

Moderna is pioneering messenger RNA Therapeutics™ , an entirely new in vivo drug technology that produces human proteins, antibodies and entirely novel protein constructs inside patient cells, which are in turn secreted or active intracellularly. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of disease conditions. The company currently plans to develop and commercialize its innovative mRNA drugs through a combination of strategic relationships as well as newly formed ventures, including Onkaido Therapeutics LLC , focused on oncology drug development, Valera LLC , focused on infectious diseases, Elpidera LLC , focused on rare diseases, and Caperna LLC , focused on personalized cancer vaccines. Founded by Flagship VentureLabs ^® , Cambridge-based Moderna is privately held and currently has strategic agreements with AstraZeneca , Alexion Pharmaceuticals and Merck . www.modernatx.com .

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Moderna Launches Caperna LLC, Its Fourth Venture, to Develop Personalized Cancer Vaccines

Thu, 22 Oct 2015 20:38:18 GMT

Caperna developing personalized cancer vaccines in combination with other cancer immunotherapies. Moderna finalizing the development of rapid cycle-time, small-batch GMP capabilities to support the development of personalized therapies.

CAMBRIDGE, Mass., October 22, 2015 — Moderna Therapeutics, a pioneer in the development of messenger RNA (mRNA) Therapeutics™, today announced the launch of Caperna LLC, the fourth Moderna venture, which will focus exclusively on the advancement of personalized cancer vaccines.

Caperna will apply Moderna’s mRNA vaccine technology to the field of cancer vaccines, building on advances in recent years in cancer immunotherapy, including the availability of checkpoint inhibitors and the ability to rapidly determine individual patient mutations. Caperna will develop personalized cancer vaccines that encode a patient’s specific neoepitopes, utilizing Moderna’s unique infrastructure to manufacture within weeks small batches of vaccines tailored to each patient.

Substantial efforts at Moderna’s infectious disease venture Valera have demonstrated preclinical efficacy of Moderna’s mRNA-based vaccines in multiple viral disease models, which has led to the nomination of several viral vaccine development candidates. These advancements have spurred the formation of Caperna and the pursuit of vaccine development for the treatment of cancer.

Tal Zaks, M.D., Ph.D., Chief Medical Officer of Moderna, who was formerly Senior Vice President and Head of Global Oncology at Sanofi, will serve as Interim President of Caperna. Nicholas Valiante, Ph.D., who recently joined Moderna from Novartis Vaccines where he was Global Head of Immunology & Immunotherapy Research, will serve as Vice President and Head of Personalized Vaccine Sciences.

“With such a potent vaccine platform and a manufacturing process that lends itself directly to rapid production of patient-specific therapies, we believe Moderna’s mRNA approach will offer distinct advantages in the development of new cancer therapies,” said Dr. Zaks. “We expect our ability to specifically activate and direct the immune system will synergize with checkpoint inhibitor therapies like PD-1 antibodies.”

“What is perhaps most exciting about our approach and our core protein expression platform is its potential to leverage a broad spectrum of leading-edge drug modalities,” said Stéphane Bancel, Chief Executive Officer of Moderna. “Caperna is an important example of Moderna’s unique ability to rapidly translate our ever-evolving understanding of mRNA into new therapeutic opportunities that have the potential to impact and change numerous lives. Caperna will be backed by Moderna’s $840 million of cash on hand.”

Onkaido , Moderna’s initial oncology venture, remains focused on the development of mRNA-based therapeutics for oncology in areas outside of the personalized cancer vaccine effort at Caperna.

About Caperna LLC, a Moderna Venture

Caperna LLC , the fourth venture company formed by Moderna, is focused exclusively on the advancement of personalized cancer vaccines. Caperna is leveraging Moderna’s messenger RNA Therapeutics™ platform, an entirely new in vivo drug technology that produces proteins natively, to develop novel, personalized cancer vaccines for the treatment of cancer. For more information please visit www.modernatx.com/ventures .

About Moderna Therapeutics

Moderna is a clinical stage pioneer of messenger RNA Therapeutics™ , an entirely new in vivo drug technology that produces human proteins, antibodies and entirely novel protein constructs inside patient cells, which are in turn secreted or active intracellularly. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of diseases and conditions. The company currently plans to develop and commercialize its innovative mRNA drugs through a combination of strategic relationships as well as newly formed ventures, including Onkaido Therapeutics LLC, focused on oncology drug development, Valera LLC , focused on infectious diseases, Elpidera LLC , focused on rare diseases, and Caperna LLC , focused on personalized cancer vaccines. Founded by Flagship VentureLabs™ , Cambridge-based Moderna is privately held and currently has strategic agreements with AstraZeneca , Alexion Pharmaceuticals and Merck . To learn more, visit www.modernatx.com .

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Moderna Launches Third Venture Company Elpidera for Rare Diseases

Tue, 12 May 2015 20:35:35 GMT

Greg Licholai, M.D., hired as president to lead the venture to advance novel messenger RNA therapies

CAMBRIDGE, Mass., May 12, 2015 — Moderna Therapeutics, a pioneer in the development of messenger RNA (mRNA) Therapeutics™, today announced the launch of Elpidera LLC, a new Moderna venture focused exclusively on the advancement of mRNA-based medicines for the treatment of rare diseases. The third in a series of venture companies created by Moderna, Elpidera will be led by Dr. Greg Licholai, an industry leader with vast experience in drug development, rare disease and R&D innovation. Elpidera, which is derived from the Greek word elpida meaning “hope,” will leverage Moderna’s mRNA platform to create novel therapies to address diseases in small patient populations with severe unmet medical needs.

Moderna signed a strategic agreement with Alexion in January 2014 allowing Alexion to have 10 product options in rare diseases. Elpidera will advance programs that are independent from the Alexion programs and proprietary to Moderna, while also supporting Alexion in its efforts to leverage the Moderna technology platform through the existing agreement.

“The creation of new venture teams is critical to advancing our decentralized drug development business strategy at Moderna. We are pleased to announce Elpidera as our second venture company launched in 2015 after launching Valera, our venture company focused in infectious diseases,” said Stéphane Bancel, chief executive officer of Moderna. “Ventures are the other leg of our strategy to add to the efforts of our pharma and biotech partners. In total, we are driving more than 50 preclinical mRNA programs across cardiovascular, infectious diseases, oncology, and rare diseases.”

Dr. Greg Licholai will serve as president of Elpidera. He has more than 25 years of experience in biotechnology leadership, in both consulting and entrepreneurial capacities. Prior to Moderna, he was senior vice president for real-world and late-stage research at Quintiles. Before that he was a partner-level consultant at McKinsey & Company, where he led a business line devoted to big data analytics for new drug launches. Dr. Licholai also has extensive experience as an executive at multiple biotechnology companies. He was chief operating officer of Proteostasis, a private biotechnology company, and earlier, was one of the first senior executives at rare disease biotech Amicus Therapeutics, helping put three products into human clinical trials for orphan indications and take the company public in 2007. He was also co-founder of Immunome, a biotech firm focused on a novel antibody platform. Dr. Licholai received his M.D. from Yale Medical School and his M.B.A from Harvard Business School.

“I am excited to join Elpidera and leverage Moderna’s mRNA platform to discover new treatments for patients living with rare, genetic diseases,” said Dr. Licholai. “As it has demonstrated in other therapeutic areas, mRNA Therapeutics hold great potential to serve rare patient populations in a way never before seen. Our approach offers hope in a novel and powerful platform to potentially affect the many thousands of underserved patients suffering from hundreds of diseases with extraordinarily high unmet medical needs. I am pleased to bring my broad array of experience leading innovative biotech companies and background in rare diseases to the team, and am committed to the company’s unique vision to make a real difference – for patients and the industry.”

Mr. Bancel added, “Greg is a superb leader with extensive biotechnology and orphan disease experience, and we are happy to have him on board to generate new medicines and advance the pipeline.”

Moderna is creating first-in-class in vivo medicines called mRNA Therapeutics™ designed to directly utilize the body’s natural processes to enable the in vivo production of therapeutic proteins. The company has demonstrated the ability to direct the production of both intracellular proteins, which remain within the cells, and secreted proteins, which are released into the bloodstream and act to restore function elsewhere in the body. The range of potential applications make mRNA Therapeutics a powerful tool to address the more than 7,000 rare diseases identified to date, only a small fraction of which are adequately addressed by existing therapies.

In addition to strategic agreements with industry collaborators AstraZeneca (cardiovascular/oncology), Alexion Pharmaceuticals (rare diseases) and Merck (infectious diseases), Moderna is creating a series of venture teams focused in distinct therapeutic areas, enabling the rapid advancement of new mRNA medicines across different modalities and therapeutic areas at an unprecedented pace and scale. This model has enabled Moderna to simultaneously scale up to more than 50 drug development programs across multiple therapeutic areas.

For more information on Moderna, its venture companies and disruptive business model, please visit www.modernatx.com or follow on Twitter @moderna_tx .

About Elpidera LLC, a Moderna Venture

Elpidera LLC , the third venture company formed by Moderna, is focused exclusively on the advancement of messenger RNA (mRNA) Therapeutics™ for the treatment of rare diseases. Elpidera, which is derived from the Greek word elpida meaning "hope," is leveraging Moderna’s mRNA platform, an entirely new in vivo drug technology that produces human proteins, antibodies and entirely novel protein constructs inside patient cells, which are in turn secreted or active intracellularly. For more information please visit www.modernatx.com/ventures .

About Messenger RNA Therapeutics ^TM

Moderna is creating first-in-class in vivo medicines called mRNA Therapeutics™ . mRNA Therapeutics™ are designed to directly utilize the body’s natural processes to enable the in vivo production of both intracellular proteins, which remain within the cells, and secreted proteins, which are released into the bloodstream and act to restore function elsewhere in the body. This is a quantum change in the way protein therapeutics are traditionally produced and used, and has the potential to transform the treatment of a broad range of diseases.

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Moderna #1 on CNBC Disruptor 50 List

Tue, 12 May 2015 20:33:30 GMT

CAMBRIDGE, Mass., May 12, 2015 — Moderna Therapeutics, a pioneer in the development of messenger RNA (mRNA) Therapeutics™, today announced it has been named the top Disruptor on the third-annual CNBC Disruptor 50 list. For the second year in a row, CNBC recognizes Moderna as one of the country’s most ambitious and innovative companies changing the economy and overall business landscape.

With its novel technology to enable mRNA as a drug, Moderna is building a fully scaled drug discovery and development platform centered on the rapid and low-cost design, delivery and production of mRNA drug candidates. Moderna is also advancing an innovative business model built on the decentralization of drug development activities. With an ecosystem of in-house concept development, ventures focused on therapeutic areas and a cluster of major pharma and biotech partners, Moderna is enabling more than 50 preclinical programs today across cardiovascular, infectious diseases, oncology, and rare diseases.

In 2015 alone, the company announced $500 million in private financing, the largest private round ever for a biotech; a license and collaboration agreement with Merck to develop mRNA-based antiviral vaccines and passive immunity therapies; and the formation of two venture companies – Valera in infectious diseases and Elpidera, its newly established venture company focused on developing treatments for rare diseases.

“Our mRNA technology is fundamentally disruptive, enabling us to do things not possible – or not done well enough – with current approaches,” said Stéphane Bancel, chief executive officer. “Through our business model, which includes collaborations with industry leaders AstraZeneca, Alexion and Merck, as well as our three venture companies, we are driving more than 50 preclinical programs. We are thrilled to be recognized by CNBC for our far-reaching technology platform, disruptive business model and the potential to bring highly differentiated, completely novel drugs to patients in need.”

Moderna was selected from more than 350 nominated companies which were ranked based on CNBC’s proprietary blend of quantitative and qualitative inputs.

CNBC.com’s special report, disruptor50.cnbc.com , features an in-depth profile of Moderna, an explanation of how CNBC chose and ranked the companies on the list and stories exploring the technologies today’s business decision makers need to know, and how investors can profit from disruptive trends.

For more information on Moderna Therapeutics, please visit www.modernatx.com or follow on Twitter @moderna_tx .

About Messenger RNA Therapeutics ^TM

About Moderna Therapeutics

Moderna is pioneering messenger messenger RNA Therapeutics™ , an entirely new in vivo drug technology that produces human proteins, antibodies and entirely novel protein constructs inside patient cells, which are in turn secreted or active intracellularly. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of diseases and conditions. Moderna is developing and plans to commercialize its innovative mRNA drugs through its own ventures and its strategic relationships with established pharmaceutical and biotech companies. The company currently plans to develop and commercialize its innovative mRNA drugs through a combination of strategic relationships as well as newly formed ventures, including Onkaido LLC , focused on oncology drug development, Valera LLC , focused on infectious diseases, and Elpidera LLC , focused on rare diseases. Founded by Flagship VentureLabs™ , Cambridge-based Moderna is privately held and currently has strategic agreements with AstraZeneca , Alexion Pharmaceuticals , Merck and Vertex Pharmaceuticals . To learn more, visit www.modernatx.com .

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Moderna Appoints Tal Zaks, Former Head of Sanofi Global Oncology, as Chief Medical Officer

Mon, 16 Mar 2015 20:31:14 GMT

CAMBRIDGE, Mass., March 16, 2015 — Moderna Therapeutics, a pioneer in the development of messenger RNA (mRNA) Therapeutics™ across a range of therapeutic applications, announced today that Tal Zaks, M.D., Ph.D., will join Moderna’s executive leadership team as chief medical officer, effective March 16.

Prior to joining Moderna, Dr. Zaks was senior vice president and head of Global Oncology at Sanofi, where he was responsible for all aspects of oncology drug discovery, development and commercialization.

“Moderna holds an unprecedented financial position among pre-clinical biotech companies, with $900 million on its balance sheet and strategic agreements with three industry leaders: AstraZeneca, Alexion and Merck,” said Stéphane Bancel, chief executive officer of Moderna. “As we prepare to initiate several clinical trials in the coming quarters, we require an exceptional chief medical officer, someone with a track record of success in clinical development combined with strong business acumen. Tal is that leader. He has a unique ability to think outside the box and execute for business results. We are delighted that he chose to join Moderna.”

“I am thrilled to be joining Moderna and be at the forefront of translating innovative science into real medicines for patients,” said Dr. Zaks. “I’ve been impressed with the potential of messenger RNA Therapeutics to expand and accelerate drug development for a wide range of unmet medical needs and I look forward to working with the leadership team, our partners and our ventures to fulfill this vision.”

Dr. Zaks began his industry career at GlaxoSmithKline in the genetics research group, where he built the oncology translational medicine team and led translational research on lapatinib as well as the in-licensing and clinical development of foretinib. In addition to his industry work, Dr. Zaks is associate professor of medicine at the University of Pennsylvania, and has served as a volunteer physician at the Philadelphia Veterans Administration Medical Center, treating patients with genitourinary cancers.

Dr. Zaks received his M.D. and Ph.D. from the Ben Gurion University in Israel and conducted post-doctoral research at the U.S. National Institutes of Health. He completed his clinical training in internal medicine at Temple University Hospital followed by a fellowship in medical oncology at the University of Pennsylvania.

For more information on Moderna Therapeutics and potential partnership opportunities, please visit www.modernatx.com .

About Moderna Therapeutics

Moderna is pioneering messenger RNA Therapeutics™ , an entirely new in vivo drug technology that produces human proteins, antibodies and entirely novel protein constructs inside patient cells, which are in turn secreted or active intracellularly. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of disease conditions. The company currently plans to develop and commercialize its innovative mRNA drugs through a combination of strategic relationships as well as newly formed ventures, including Onkaido LLC, focused on oncology drug development, and Valera LLC , focused on infectious diseases. Founded by Flagship VentureLabs™ , Cambridge-based Moderna is privately held and currently has strategic agreements with AstraZeneca , Alexion Pharmaceuticals , and Merck . To learn more, visit www.modernatx.com .

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Moderna and Institut Pasteur Sign Strategic Research Collaboration

Tue, 03 Feb 2015 20:29:35 GMT

Research partnership will advance the development of mRNA vaccines and passive immunity therapies for infectious diseases

CAMBRIDGE, Mass. and PARIS, Feb. 3, 2015 — Moderna Therapeutics, a pioneer in the development of messenger RNA (mRNA) Therapeutics™ and the Institut Pasteur , a recognized public benefit organization with three core missions — research, public health and teaching — at the forefront of biomedical research, today announced a long-term strategic research collaboration for the discovery and development of drugs and vaccines using Moderna’s mRNA Therapeutics™ platform. Valera , a Moderna venture company, will manage the research collaboration for Moderna. Valera is focused exclusively on the advancement of vaccines and therapeutics for the prevention and treatment of viral, bacterial and parasitic infectious diseases.

Said Pr Christian Bréchot, president of the Institut Pasteur, “We look forward to collaborating with Valera and to using Moderna’s mRNA platform to discover and develop new vaccines and treatments to address infectious diseases in an entirely new way. This partnership will be a trump card in our fight against viral and bacterial disease.”

Under the agreement, Valera will sponsor programs of preclinical and clinical research at the Institut Pasteur aimed at identifying and developing new approaches to combatting known and emerging viral and bacterial diseases.

“We are very honored to be working with the Institute that Monsieur Louis Pasteur started more than 125 years ago in Paris. He is the father of the modern fight against infectious disease. Through our partnership with the Institut Pasteur, we will work with world-class researchers and clinicians who will be critical allies as we strive to develop a transformative approach to fighting infectious diseases for patients throughout the world,” said Stephane Bancel, president and CEO of Moderna. “This agreement builds on our strategy of partnering with leaders in critical fields of medicine, from AstraZeneca, Alexion, Merck and DARPA to our recently announced collaboration with the Karolinska Institutet, to advance the development of mRNA-based drugs and vaccines across multiple therapeutic areas.”

About Institut Pasteur

Louis Pasteur created the Institut Pasteur in 1887 as a private non-profit foundation that rapidly became world-renowned for its biomedical research. The main aim of the Institut Pasteur is understanding and preventing diseases throughout the world through excellent scientific and public health research, teaching and other activities. Together with its major contribution to a deeper understanding of fundamental aspects of life, the Institut Pasteur continues to devote a large part of its efforts to infectious diseases, inherited disorders, neurodegenerative diseases and certain cancers. Close to 2,400 people work on its main campus in Paris, which is at the heart of an international network of 32 research institutes on 5 continents. Over the years, 10 Institut Pasteur researchers have received the Nobel Prize. www.pasteur.fr

Contact Information

Marion Doucet, Press Office
+ 33 1 45 68 89 28,
marion.doucet@pasteur.fr

About Valera, a Moderna Venture

Valera, the second venture company formed by Moderna, is focused exclusively on the advancement of vaccines and therapeutics for the prevention and treatment of infectious diseases. Valera is leveraging Moderna’s messenger RNA Therapeutics™ platform, an entirely new in vivo drug technology that produces human proteins, antibodies and entirely novel protein constructs inside patient cells, which are in turn secreted or active intracellularly. For more information please visit www.modernatx.com/ventures .

About Moderna Therapeutics

Moderna is pioneering messenger RNA Therapeutics™ , an entirely new in vivo drug technology that produces human proteins, antibodies and entirely novel protein constructs inside patient cells, which are in turn secreted or active intracellularly. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of disease conditions. The company currently plans to develop and commercialize its innovative mRNA drugs through a combination of strategic relationships as well as newly formed ventures, including Onkaido LLC , focused on oncology drug development, and Valera LLC , focused on infectious diseases. Founded by Flagship VentureLabs™ , Cambridge-based Moderna is privately held and currently has strategic agreements with Merck , AstraZeneca and Alexion Pharmaceuticals . To learn more, visit www.modernatx.com .

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Moderna Announces License and Collaboration Agreement with Merck to Develop Messenger RNA-based Antiviral Vaccines and Passive Immunity Therapies

Tue, 13 Jan 2015 20:27:35 GMT

CAMBRIDGE, Mass., January 13, 2015 — Moderna Therapeutics today announced a license and collaboration agreement with Merck, known as MSD outside the United States and Canada, through a subsidiary, for the discovery and development of vaccines and passive immunity treatments against viral diseases using modified messenger RNA (mRNA). Moderna is a pioneer in the development of mRNA Therapeutics™ across a range of therapeutic applications. Moderna’s work in the collaboration will be led by Valera, its venture focused on the development of mRNA vaccines and therapeutics to fight infectious disease.

The vaccines work of Valera builds on a body of preclinical research at Moderna showing the ability of modified mRNA to express viral antigens in vivo and to induce robust immune responses. Valera’s therapeutic passive immunity programs will expand on Moderna’s research using mRNA to express antibodies that bind to viral and other targets. The robust data in these programs across a range of preclinical infectious disease models, together with the inherent, rapid turn-around time in creating novel mRNA constructs, provide Valera with a potentially powerful and versatile new platform for the creation of a broad array of vaccines and passive immunity therapies.

“Given the tremendous potential for messenger RNA Therapeutics across a wide range of therapeutic applications, establishing long-term strategic relationships with world leaders in their fields will accelerate our ability to bring mRNA products to patients in need,” said Stéphane Bancel, president and CEO of Moderna. “Merck’s worldwide leadership in vaccines and anti-infective treatments make them an ideal collaborator for us, particularly given their strong commitment to innovation and new approaches to prevent and treat serious viral diseases. We are excited to work in collaboration to move these promising programs forward for patients.”

"By combining Merck’s strength in vaccine and antiviral therapeutic development with Moderna’s mRNA Therapeutics technology we are well positioned to develop differentiated candidates with the potential to provide meaningful benefit to patients,” said Dr. Roger M. Perlmutter, president of Merck Research Laboratories. “We look forward to working with the scientific and technical teams at Moderna.”

The three-year research collaboration (with the possibility of a one-year extension) is focused on the development of new mRNA-based treatments and vaccines against four undisclosed viruses. Under the terms of the agreement, Merck will make an upfront cash payment to Moderna of $50 million to give Merck the ability to utilize the granted licenses to commercialize five product candidates, and will make a $50 million equity investment in Moderna. This is in addition to the $450 million financing from other investors previously announced on January 5, 2015. Moderna will be eligible for undisclosed per-product development and commercial milestones under the license as well as tiered royalties on commercial sales. Merck will lead the discovery and development of candidates and commercialization of any products resulting from this license and collaboration agreement, while Moderna will design and synthesize the messenger RNA product candidates directed against selected targets.

Moderna’s mRNA Therapeutics™ platform builds on the discovery that modified mRNA can direct the body’s cellular machinery to produce nearly any protein of interest, from native proteins to antibodies and other entirely novel protein constructs with therapeutic activity inside and outside of cells. In addition to the license and collaboration announced today with Merck, Moderna has ongoing strategic agreements with Alexion Pharmaceuticals in the area of rare diseases, AstraZeneca in cardiovascular disease and some areas of oncology, and DARPA (the Defense Advanced Research Projects Agency) in biodefense.

About Valera LLC, a Moderna Venture

Valera LLC, the second venture company formed by Moderna, is focused exclusively on the advancement of vaccines and therapeutics for the prevention and treatment of infectious diseases. Valera is leveraging Moderna’s messenger RNA Therapeutics™ platform, an entirely new in vivo drug technology that produces human proteins, antibodies and entirely novel protein constructs inside patient cells, which are in turn secreted or active intracellularly. For more information please visit www.modernatx.com/ventures .

About Moderna Therapeutics

Moderna is pioneering messenger RNA Therapeutics™ , an entirely new in vivo drug technology that produces human proteins, antibodies and entirely novel protein constructs inside patient cells, which are in turn secreted or active intracellularly. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of disease conditions. The company plans to develop and commercialize its innovative mRNA drugs through a combination of strategic relationships as well as new formed ventures, including Onkaido LLC , focused oncology drug development, and Valera LLC , focused on infectious disease. Founded by Flagship VentureLabs™ , Cambridge-based Moderna is privately held and currently has strategic agreements with AstraZeneca and Alexion Pharmaceuticals . To learn more, visit www.modernatx.com .

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Moderna Launches New Venture Valera LLC for Infectious Diseases

Thu, 08 Jan 2015 20:25:22 GMT

Moderna venture to advance mRNA vaccines and mRNA-based passive immunity therapies

CAMBRIDGE, Mass., Jan. 8, 2015 — Moderna Therapeutics, a pioneer in the development of messenger RNA (mRNA) Therapeutics™, today announced the launch of Valera LLC , a new Moderna venture focused exclusively on the advancement of vaccines and therapeutics for the prevention and treatment of viral, bacterial and parasitic infectious diseases.

The vaccines work of Valera builds on a body of preclinical research at Moderna showing the ability of modified mRNA to express viral antigens in vivo and to induce robust immune responses. Valera’s therapeutic passive immunity programs will expand on Moderna’s research using mRNA to express antibodies that bind to viral and other targets. The robust data from these programs across a range of preclinical infectious disease models, together with the inherent, rapid turn-around time in creating novel mRNA constructs, provide Valera with a potentially powerful and versatile new platform for the creation of a broad array of vaccines and passive immunity therapies.

“We are thrilled to launch Valera to bring sharp focus to developing mRNA Therapeutics for a wide range of infectious diseases, which remain a hugely significant global health threat,” said Stéphane Bancel, president and CEO of Moderna. “We believe that mRNA offers unique advantages when it comes to the rapid design and manufacture of new vaccines and therapies, and we have seen promising signs of efficacy in our preclinical work.”

Mr. Bancel will continue to serve as interim president of Valera. Moderna has engaged the global executive search firm Korn Ferry (NYSE:KFY) to recruit a president for Valera.

Giuseppe Ciaramella, Ph.D., former vice president and head of immunology and biotherapeutics at Moderna, will serve as chief scientific officer of Valera. Dr. Ciaramella has nearly two decades of global industry experience at AstraZeneca, Boeringher Ingelheim, Pfizer and Merck focused on the discovery and development of small molecule and biological clinical candidates, with a particular focus on antivirals and biotherapeutics. The plan in 2015 is for Dr. Ciaramella to hire and lead a team of 15 scientists to drive early-stage discovery and development at Valera.

Michele Keough, formerly with Moderna’s R&D strategy and operations group, will serve as head of programs and alliance management at Valera. Prior to Moderna, Ms. Keough served as senior director, program and alliance management at Genzyme, a Sanofi company, where she led several of Genzyme’s strategic partnerships with biotechnology companies.

Moderna is actively recruiting for the position of chief medical officer of Valera.

Valera will expand its team significantly in 2015 and will work out of Moderna’s Venture Labs at 320 Bent Street, Cambridge, Mass.

About Valera LLC, a Moderna Venture

About Moderna Therapeutics

Moderna is pioneering messenger RNA Therapeutics™ , an entirely new in vivo drug technology that produces human proteins, antibodies and entirely novel protein constructs inside patient cells, which are in turn secreted or active intracellularly. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of disease conditions. The company currently plans to develop and commercialize its innovative mRNA drugs through a combination of strategic relationships as well as newly formed ventures, including Onkaido LLC , focused on oncology drug development, and Valera LLC , focused on infectious diseases. Founded by Flagship VentureLabs™ , Cambridge-based Moderna is privately held and currently has strategic agreements with AstraZeneca and Alexion Pharmaceuticals . To learn more, visit www.modernatx.com .

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Onkaido Announces Appointment of Michael J. Morin, Ph.D., as Chief Scientific Officer

Wed, 07 Jan 2015 20:23:22 GMT

Former Pfizer research executive to lead scientific efforts in oncology drug development leveraging Moderna’s messenger RNA Therapeutics™ platform

CAMBRIDGE, Mass., Jan. 7, 2015 — Onkaido Therapeutics , a venture company formed, funded and wholly-owned by Moderna Therapeutics, a pioneer in the development of messenger RNA (mRNA) Therapeutics™ , announced today that Michael J. Morin, Ph.D., joins Onkaido as chief scientific officer. Launched in January 2014 , Onkaido is Moderna’s first venture company, focused exclusively on the use of Moderna’s mRNA platform for the development and commercialization of oncology treatments.

“At Onkaido, we are dedicated to growing our mRNA Therapeutics platform in oncology to transform the lives of patients with cancer,” said Stephen Kelsey, M.D., president of Onkaido. “With Mike’s exceptional track record in the discovery and development of cancer treatments, he will bring critical expertise and leadership to Onkaido’s team of top scientists.”

Dr. Morin’s oncology career spans academia and industry, including 17 years at Pfizer serving in a variety of positions, most recently as vice president of global research and development in Groton, CT, overseeing antibacterials, immunology and cancer drug discovery. During Dr. Morin’s time at Pfizer, some 40 clinical candidates were discovered under his leadership, most notably the discovery through Phase 2 proof-of-concept for erlotinib (Tarceva™), a small molecule inhibitor of the epidermal growth factor receptor tyrosine kinase (a blockbuster drug marketed by Roche and Astellas). Following his time at Pfizer, Dr. Morin worked as an advisor to venture-backed biotechnology and publicly-held pharmaceutical companies and has continued his active support of a variety of cancer research organizations at the local and national level.

“I am pleased to join Onkaido as chief scientific officer to help advance new mRNA-based oncology treatments from discovery to commercialization,” said Dr. Morin. “I am truly excited by the opportunity to lead research with Moderna’s mRNA Therapeutics platform – a technology with real potential to transform the way cancer is treated.”

For more information on Onkaido, please visit onkaido.com . For more information on Moderna Therapeutics please visit modernatx.com .

About Onkaido, a Moderna Venture

Onkaido, a venture company formed, funded and wholly-owned by Moderna, is focused exclusively on the advancement of oncology products for previously undruggable targets and as a superior alternative to existing drug modalities. Leveraging Moderna’s messenger RNA Therapeutics™ platform, an entirely new in vivo drug modality that produces human proteins, and antibodies and entirely novel protein constructs inside patient cells, Onkaido currently plans to rapidly turn scientific innovation into cancer therapies that can make a real difference for patients.

About Moderna Therapeutics

Moderna is pioneering messenger RNA Therapeutics™ , an entirely new in vivo drug technology that produces human proteins, antibodies and entirely novel protein constructs inside patient cells, which are in turn secreted or active intracellularly. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of disease conditions. The company plans to develop and commercialize its innovative mRNA drugs through a combination of strategic relationships as well as new formed ventures, including Onkaido LLC , focused oncology drug development, and Valera LLC , focused on infectious disease. Founded by Flagship VentureLabs™ , Cambridge-based Moderna is privately held and currently has strategic agreements with AstraZeneca and Alexion Pharmaceuticals . To learn more, visit www.modernatx.com .

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Moderna to Present at 33rd Annual J.P. Morgan Healthcare Conference

Tue, 06 Jan 2015 20:21:11 GMT

CAMBRIDGE, Mass., Jan. 6, 2015 — Moderna Therapeutics, a pioneer in the development of messenger RNA (mRNA) Therapeutics™, a revolutionary new treatment modality to enable the in vivo production of therapeutic proteins, announced today that Stéphane Bancel, president and chief executive officer of Moderna, will present a company overview at the 33rd Annual J.P. Morgan Healthcare Conference.

Bancel will focus on Moderna’s efforts to accelerate drug discovery and development across a series of drug modalities and therapeutic areas, each powered by Moderna’s proprietary approach for utilizing modified mRNA to instruct native cellular machinery to make therapeutic proteins in vivo.

The presentation will take place on Tuesday, January 13, 2015 at 8:30 a.m. PT in Elizabethan D at the Westin St. Francis Hotel in San Francisco.

For more information on Moderna Therapeutics, including how messenger RNA works, partnership opportunities, and open job opportunities, visit www.modernatx.com .

About Moderna Therapeutics

Moderna is pioneering messenger RNA Therapeutics™ , an entirely new in vivo drug technology that produces human proteins, antibodies and entirely novel protein constructs inside patient cells, which are in turn secreted or active intracellularly. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of disease conditions. The company currently plans to develop and commercialize its innovative mRNA drugs through a combination of strategic relationships as well as newly formed ventures, including Onkaido LLC , focused oncology drug development. Founded by Flagship VentureLabs™ , Cambridge-based Moderna is privately held and currently has strategic agreements with AstraZeneca and Alexion Pharmaceuticals . To learn more, visit www.modernatx.com .

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Moderna Closes $450 Million Financing to Support Growth of Messenger RNA Therapeutics™ Platform across Diverse Therapeutic Areas

Mon, 05 Jan 2015 20:17:33 GMT

CAMBRIDGE, Mass., January 5, 2015 —Moderna Therapeutics, a pioneer in the development of messenger RNA (mRNA) Therapeutics™, announced today that it has raised $450 million in new funding to support the further expansion of its mRNA Therapeutics™ platform across multiple modalities and therapeutic areas. Since the company’s founding in 2011 by Flagship VentureLabs™, Moderna has secured more than $950 million in funding through financing activities and commercial partnerships.

This funding round, Moderna’s largest to date, will be used to further accelerate drug discovery and development across a series of drug modalities and therapeutic areas, each powered by Moderna’s proprietary approach for utilizing modified mRNA to instruct native cellular machinery to make therapeutic proteins in vivo. To continue to drive these programs forward, Moderna plans immediate growth and expansion, including the addition of more than 100 industry leaders, drug development experts and scientists to its current team of 145 employees during the coming months.

Participating in the financing round were new investors Viking Global Investors LP, Invus, RA Capital Management, and Wellington Management Company, LLP, as well as existing investors AstraZeneca and Alexion Pharmaceuticals. Additional existing investors participated in the financing.

“The strong support from our investors is a testament to the incredible work done by our team over the past three years in unlocking the potential of mRNA Therapeutics to drive transformational advances for patients,” said Stéphane Bancel, president and CEO of Moderna. “With $800 million in cash after this financing, we are moving rapidly to support the exponential growth of our mRNA Therapeutics platform with new investments, partnerships and ventures, and are committed to recruiting the best industry talent to support this growth at all levels, including bench scientists, seasoned drug hunters and leaders for our new ventures.”

Together with partners AstraZeneca, Alexion and DARPA (The Defense Advanced Research Projects Agency), Moderna is in active development of 45 preclinical programs in oncology, cardiovascular disease, rare diseases, and infectious diseases. These include programs underway at Onkaido, a Moderna venture focused exclusively on oncology, and at other therapeutically-focused ventures in development. In addition, Moderna is investing in early-stage drug discovery and development through its long-term strategic collaboration with Karolinska Institutet (KI) and Karolinska University Hospital (KUH), announced in October 2014.

For more information on Moderna Therapeutics, including how messenger RNA works, partnership opportunities, and open job opportunities, visit www.modernatx.com .

About Moderna Therapeutics

Moderna is pioneering messenger RNA Therapeutics™ , an entirely new in vivo drug technology that produces human proteins, antibodies and entirely novel protein constructs inside patient cells, which are in turn secreted or active intracellularly. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of disease conditions. The company currently plans to develop and commercialize its innovative mRNA drugs through a combination of strategic relationships as well as newly formed ventures, including Onkaido LLC , focused oncology drug development. Founded by Flagship VentureLabs™ , Cambridge-based Moderna is privately held and currently has strategic agreements with AstraZeneca and Alexion Pharmaceuticals . To learn more, visit www.modernatx.com .

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Moderna to Collaborate with Karolinska Institutet and Karolinska University Hospital on Discovery of New Messenger RNA Therapeutics™

Thu, 16 Oct 2014 20:59:09 GMT

Strategic research and clinical partnership will advance state-of-the-art discoveries on the use of messenger RNA (mRNA) Therapeutics™ to treat serious diseases

CAMBRIDGE, Mass. and STOCKHOLM, Sweden, October 16, 2014 —Moderna Therapeutics today announced a strategic, long-term collaboration with Karolinska Institutet (KI) and Karolinska University Hospital (KUH) for the discovery and development of innovative drugs using Moderna’s messenger RNA (mRNA) Therapeutics™ technology. mRNA Therapeutics™ enable the in vivo production of both intracellular proteins and secreted proteins. As a result, Moderna’s platform has the potential to speed the development and manufacture of treatments for many diseases that are currently untreatable with existing pharmaceutical approaches.

"This project is an important step in advancing medical science," said Professor Hans-Gustaf Ljunggren, Dean of Research at Karolinska Institutet. "It will help achieve our common goal of rapidly advancing new drug candidates into the clinic."

Under the terms of the partnership, Moderna will sponsor research grants for scientists at both institutions to conduct preclinical research with novel mRNA Therapeutics™. As this pre-clinical work is successfully completed, Moderna will conduct clinical trials of new drug candidates at Karolinska University Hospital.

“As a leading medical center, we continually strive to improve the treatment of serious diseases,” said Professor Mats Eriksson, Karolinska University Hospital. “Our clinical researchers are excited to work with Moderna’s groundbreaking mRNA Therapeutics platform and speed the advancement of new treatments to patients.”

To solidify the scientific and clinical collaboration between the organizations, and to optimize the output of this important partnership, Moderna is creating a new laboratory in Stockholm, Sweden, located in the Novum building next to the Karolinksa University Hospital Huddinge campus.

“Moderna is investing heavily to bring mRNA Therapeutics to patients, and our science is accelerating rapidly,” said Stéphane Bancel, President and founding CEO of Moderna. “This partnership puts our mRNA Therapeutics platform in the hands of Karolinska’s world-class scientists and clinical researchers to develop new drugs and therapeutic approaches that cannot be done with small molecules or biologics – bringing new hope to patients with serious diseases.”

“Strategically, we view this, our first academic partnership, as highly complementary to our existing drug discovery and development efforts, both with our pharmaceutical partners AstraZeneca and Alexion and with Moderna ventures such as Onkaido,” added Bancel. “Given the broad potential of this revolutionary drug technology, it was critical to us to work closely with a leading academic medical institution. We are honored to be partnering with one of the best academic medical research institutions in the world.”

For more information on Karolinska Institutet and Karolinska University Hospital, please visit ki.se and karolinska.se .

For more information on Moderna Therapeutics please visit modernatx.com .

About Karolinska Institutet

Onkaido Therapeutics, a venture company formed, funded and wholly-owned by Moderna, is focused exclusively on the advancement of oncology products for previously undruggable targets and as a superior alternative to existing drug modalities. Leveraging Moderna’s messenger RNA Therapeutics™ platform, an entirely new in vivo drug modality that produces human proteins or antibodies inside patient cells, Onkaido plans to rapidly turn scientific innovation into cancer therapies that can make a real difference for patients. onkaido.com

About Karolinska University Hospital

Karolinska University Hospital is one of Europe's largest university hospitals and together with Karolinska Institutet has a leading role within the field of medical breakthroughs. The hospital aims to always put the patient first by providing the best possible medical expertise, treatment and care. Through innovation and active collaboration with industry and academia, it is committed to being internationally prominent in medicine, research and education.

About Moderna Therapeutics

Moderna is pioneering messenger RNA Therapeutics™ , an entirely new in vivo drug modality that produces human proteins or antibodies inside patient cells, which are in turn secreted or active intracellularly. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of disease conditions. Moderna has developed a broad intellectual property estate, including more than 320 patent applications covering novel nucleotide chemistries and drug compositions. The company plans to develop and commercialize its innovative mRNA drugs through a combination of strategic relationships as well as new formed ventures, like Onkaido LLC , its oncology Drug Development Company. Founded by Flagship VentureLabs™ , Cambridge-based Moderna is privately held and currently has strategic agreements with AstraZeneca and Alexion Pharmaceuticals . To learn more, visit www.modernatx.com .

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Moderna Appoints Oncology Leader Dr. Stephen Kelsey as President of Onkaido

Tue, 01 Jul 2014 21:02:13 GMT

Former Genentech executive to lead Moderna’s first venture company, focused exclusively in novel biology for oncology drug development

CAMBRIDGE, Mass., July 1, 2014 — Moderna Therapeutics, the pioneer in developing messenger RNA (mRNA) Therapeutics™ , a revolutionary treatment modality to enable the in vivo production of therapeutic proteins, announced today that Stephen Kelsey, M.D., will become president of Onkaido Therapeutics, Moderna’s oncology drug development company, effective July 21. Launched in January of this year , Onkaido is Moderna’s first venture company, focused exclusively on developing and commercializing mRNA-based oncology treatments.

“As we continue to grow Moderna and perfect our mRNA Therapeutics platform, we are also focused on building a transformational oncology company that will benefit patients and society. This requires hiring the best oncology talent to lead Onkaido,” said Stéphane Bancel, president and founding chief executive officer, Moderna. “Steve brings a wealth of experience in oncology drug development to his new role. His knowledge and leadership, combined with the team of Onkaido scientists and Moderna’s innovative mRNA technology, will help speed a new class of cancer drugs to patients around the world.”

Dr. Kelsey has extensive pharmaceutical industry experience in oncology. After 16 years as an academic clinician, he started his industry career at Sugen, and later was vice president of hematology/oncology at Genentech. While at Genentech, Dr. Kelsey played a significant role in the development of key products Perjeta®, Kadcyla® and Erivedge®, as well as other molecules in the company’s oncology portfolio. He left Genentech in 2009 to run Geron’s oncology division, where he served for four years as executive vice president, research and development, and chief medical officer helping to develop therapeutics and vaccines to fight cancer.

Most recently Dr. Kelsey was senior vice president, new projects at the cancer therapies company Medivation. Dr. Kelsey graduated with a Doctorate of Medicine (M.D.) from the University of Birmingham, UK and is a fellow of both the Royal Colleges of Physicians and Pathologists, UK.

For his new role as President of Onkaido, Dr. Kelsey will relocate to Cambridge from San Francisco.

“It is a huge privilege to be invited to lead Onkaido,” said Dr. Kelsey. “Moderna’s mRNA platform holds great potential for many therapeutic areas but especially for oncology, a field with many targets still undiscovered. With mRNA Therapeutics, we have the opportunity to rapidly develop new cancer therapies that address unmet medical needs currently untreatable by existing approaches.”

For more information on Onkaido Therapeutics, please visit onkaido.com . For more information on Moderna Therapeutics please visit modernatx.com .

About Onkaido Therapeutics

About Moderna Therapeutics

Moderna is pioneering messenger RNA Therapeutics™ , an entirely new in vivo drug modality that produces human proteins or antibodies inside patient cells, which are in turn secreted or active intracellularly. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of disease conditions. Moderna has developed a broad intellectual property estate, including more than 320 patent applications covering novel nucleotide chemistries and drug compositions. The company plans to develop and commercialize its innovative mRNA drugs through a combination of strategic relationships as well as new formed ventures, like Onkaido LLC, its oncology Drug Development Company. Founded by Flagship VentureLabs , Cambridge-based Moderna is privately held and currently has strategic agreements with AstraZeneca and Alexion Pharmaceuticals . To learn more, visit www.modernatx.com .

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Moderna Named to CNBC Disruptor 50 List

Tue, 17 Jun 2014 21:04:28 GMT

Messenger RNA Therapeutics™ leader joins short list of major innovators for potential to transform the treatment of many diseases

CAMBRIDGE, Mass., June 17, 2014 — Moderna Therapeutics , the pioneer in developing messenger RNA (mRNA) Therapeutics™, today announced it was named to the 2014 CNBC Disruptor 50 list . CNBC recognizes Moderna as one of the top 50 companies whose innovations are having a dramatic impact across their industries, and are poised for hyper-growth. Moderna is considered a Disruptor 50 for its revolutionary mRNA Therapeutics technology platform, which has the potential to speed the development and manufacture of treatments for many diseases that are currently untreatable with existing technologies.

Moderna is creating first-in-class in vivo medicines called mRNA Therapeutics. mRNA Therapeutics are designed to directly utilize the body’s natural processes to enable the in vivo production of both intracellular proteins, which remain within the cells, and secreted proteins, which are released into the bloodstream and act to restore function elsewhere in the body. This is a quantum change in the way protein therapeutics are traditionally produced and used, and has the potential to transform the treatment of a broad range of diseases.

“We are proud to be recognized by CNBC as a Disruptor 50 for our technology platform and the work we are doing to speed the development and delivery of much-needed medicines to patients,” said Stéphane Bancel, president and founding chief executive officer. “Moderna is just three years old, yet we are moving quickly to deliver on the potential of our mRNA Therapeutics platform through our alliances with key industry leaders, substantial capital and rapid hiring of top scientists. We now have more than 170 talented team members to bring mRNA Therapeutics to patients.”

mRNA Therapeutics have unique advantages over other drug modalities, as they can be made and dosed with unprecedented ease and speed, reducing time and costs involved in the traditional drug development and manufacturing process:

Proteins are produced in vivo, enabling the ability to go after undruggable targets using current small or large molecules.
mRNA Therapeutics can be produced and tested in just weeks, expediting the process from concept to first-in-man studies in under a year.
mRNA Therapeutics are made using a cell-free production process, enabling rapid, cost-effective GMP manufacturing.

Moderna – having signed strategic option agreements with AstraZeneca and Alexion Pharmaceuticals – already has several preclinical programs underway in cardiovascular, cancer, infectious diseases, and rare genetic diseases.

Moderna recently announced its expansion in Cambridge, Mass. to include a second office location at 320 Bent Street, bringing its total office space to about 100,000 square feet. The new office will house Moderna Venture laboratories, non-GMP production facilities and the relocated offices of Moderna’s headquarters. The company’s former headquarters at 200 Technology Square in Cambridge is now dedicated to research and development and counts an added floor for GMP manufacturing.

The CNBC Disruptor 50 finalists were selected with help from the National Venture Capital Association, which called for nominations from the leading venture capital firms and individual leaders in the venture capital world. More than 50 firms and individuals collectively submitted more than 500 companies for consideration, double the nominees received for the 2013 Disruptor 50 list.

Nominated companies were then put through a months-long process of researching and scoring, using a proprietary blend of quantitative criteria such as amount of venture capital raised and the estimated size of the company’s addressable market, along with qualitative criteria, including originality and creating a new market or ecosystem.

For more information on Moderna Therapeutics, please visit modernatx.com .

About Moderna Therapeutics

Moderna is pioneering messenger RNA Therapeutics™ , an entirely new in vivo drug modality that produces human proteins or antibodies inside patient cells, which are in turn secreted or active intracellularly. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of disease conditions. Moderna has developed a broad intellectual property estate, including more than 320 patent applications covering novel nucleotide chemistries and drug compositions. The company plans to develop and commercialize its innovative mRNA drugs through a combination of strategic relationships as well as new formed ventures, like Onkaido LLC, its oncology Drug Development Company. Founded by Flagship VentureLabs , Cambridge-based Moderna is privately held and currently has strategic agreements with AstraZeneca and Alexion Pharmaceuticals . To learn more, visit www.modernatx.com .

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Moderna Announces Expansion to New Facility in Kendall Square

Thu, 12 Jun 2014 21:06:12 GMT

Company Doubles Footprint in Cambridge Due to Rapid Growth

CAMBRIDGE, Mass., June 12, 2014 — Moderna Therapeutics , the pioneer in developing messenger RNA (mRNA) Therapeutics™, is announcing the company’s expansion to 320 Bent Street in the Kendall Square area of Cambridge, a move that adds nearly 50,000 square feet of office, laboratory and manufacturing facilities to Moderna’s operations in Massachusetts.

“Moderna’s rapid growth has led to our expansion in Cambridge – a city with the right resources and talent to fuel our continued success,” said Stéphane Bancel, founding president and CEO of Moderna. “Less than three years after launching the company, our team has grown to about 170 people in Cambridge, we have expanded our facilities to more than 100,000 square feet, and we are continuing to hire.”

The new site at 320 Bent Street will house Moderna Venture Incubator laboratories, non-GMP production facilities, and the relocated offices of Moderna’s headquarters. The company’s former headquarters at 200 Technology Square in Cambridge will remain occupied by Moderna and be dedicated to research and development and GMP manufacturing.

“In Massachusetts we invest in the life sciences because we are choosing to shape our own future,” said Governor Deval Patrick. “I congratulate Moderna Therapeutics on their expansion and look forward to their continued contributions and job creation here in the Commonwealth.”

Since 2011, Moderna has received more than $1.4 million in tax incentives from the Massachusetts Life Sciences Center (PDF File) (MLSC) based upon commitments to create more than 70 new jobs in the Commonwealth.

The pace of Moderna’s development is driven by progress with its research on the mRNA Therapeutics™ technology platform, its proprietary research programs, and the research programs of the organizations with which Moderna has strategic agreements, AstraZeneca, Alexion Pharmaceuticals and DARPA (the Defense Advanced Research Projects Agency).

“On behalf of the team at the Massachusetts Life Sciences Center, congratulations to Moderna Therapeutics on the company’s expansion into this new space in Kendall Square,” said Susan Windham-Bannister, Ph.D., president and CEO of the MLSC. “We are pleased to have awarded Moderna tax incentives over the past three years totaling more than $1.4 million, tied to their new job creation in Massachusetts, and we look forward to the great advances in messenger RNA Therapeutics that will occur in this new facility.”

Moderna is accelerating the number of research programs using mRNA Therapeutics™, with 16 research programs currently ongoing. In addition, AstraZeneca will soon add another 15 programs to its eight programs already underway, and Alexion has initiated two programs since signing an agreement with Moderna in January, and intends to add more programs soon.

“Moderna is firing on all cylinders, making important discoveries on the core science behind mRNA drugs, advancing research programs and filing key patents to drive future growth,” said Bancel. “We are grateful to the Commonwealth of Massachusetts and the City of Cambridge for their support. Moderna is committed to investing in Cambridge – an important global hub for biotechnology research, development and commercialization.”

“Moderna is a great example of a Massachusetts success story—a company that is growing here because of its great science, great talent, support from our partners in government, and collaboration with key strategic partners in industry,” said Robert K. Coughlin, president and CEO of MassBio, the life sciences trade organization. “We congratulate Stéphane and his team for reaching the milestone of this expansion and wish them the best of luck in finding treatments and cures for patients in need around the world.”

For more information on Moderna Therapeutics and potential partnership opportunities, please visit modernatx.com .

About Moderna Therapeutics

Moderna is pioneering messenger RNA Therapeutics™ , an entirely new in vivo drug modality that produces human proteins or antibodies inside patient cells, which are in turn secreted or active intracellularly. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of disease conditions. Moderna has developed a broad intellectual property estate, including more than 320 patent applications covering novel nucleotide chemistries and drug compositions. The company plans to develop and commercialize its innovative mRNA drugs through a combination of strategic relationships as well as new formed ventures, like Onkaido LLC, its oncology Drug Development Company. Founded by Flagship VentureLabs , Cambridge-based Moderna is privately held and currently has strategic agreements with AstraZeneca and Alexion Pharmaceuticals . To learn more, visit www.modernatx.com .

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Moderna Forms Technology Advisory Board to Provide Expertise for the Company’s GMP Manufacturing Strategy

Wed, 21 May 2014 21:08:15 GMT

CAMBRIDGE, Mass., May 21, 2014 — Moderna Therapeutics , the pioneer in developing messenger RNA (mRNA) Therapeutics™, a revolutionary treatment modality to enable the in vivo production of therapeutic proteins, announced today the formation of a Technology Advisory Board.

This new Technology Advisory Board will bring together a world-class panel of experts to advise Moderna on building state-of-the-art GMP (Good Manufacturing Practice) manufacturing capabilities to deliver high-quality mRNA Active Pharmaceutical Ingredient (API) for its own clinical programs, and for organizations with which Moderna has strategic agreements. These include AstraZeneca, Alexion Pharmaceuticals and DARPA (the Defense Advanced Research Projects Agency).

The Board will focus initially on scaling the production of mRNA from micrograms to kilograms to full commercial scale, and on ensuring the use of best-in-class purification techniques and analytical tools.

“Moderna’s strategy is to own its R&D technology and its API GMP manufacturing,” said Stéphane Bancel, President and founding CEO of Moderna. “We are on a path to grow our manufacturing lot size from the micrograms of our early days, to grams today and kilograms tomorrow.”

“We will also develop proprietary manufacturing processes to ensure our mRNA is of the highest purity, resulting in a very short manufacturing cycle so we can produce a lot in just days, not weeks,” added Bancel. “With the right tools and technology, we can move with unprecedented speed from a drug concept to a first-in-human clinical trial, so we can serve patients faster. This group of world-class experts from academia and industry will complement our talented internal team, providing the right expertise to accomplish this important goal.”

The members of the Technology Advisory Board are:

Dr. Noubar Afeyan, Ph.D. , co-founder and chairman of Moderna, managing partner and CEO of Flagship Ventures, founder and CEO of PerSeptive Biosystems, Ph.D. in Biochemical Engineering from M.I.T.
Dr. John Aunins, Ph.D. , executive vice president, CMC, of Seres Health, Inc., former VP of process development at Merck Inc., Ph.D. in Chemical Engineering from M.I.T.
Dr. Scott Canute , former president of manufacturing at Genzyme, former president of manufacturing at Eli Lilly, MBA from Harvard Business School
Professor Fred Regnier, Ph.D. , J.H. Law distinguished professor of chemistry at Purdue University
Professor James Swartz, Ph.D. , professor of chemical engineering at Stanford University, former engineer at Genentech and Eli Lilly
Professor Jack Szostak, Ph.D. , Nobel Prize in Medicine 2009, Howard Hughes medical institute investigator, professor of genetics at Harvard Medical School, and Alex Rich distinguished investigator, Dept. of Molecular Biology and the Center for Computational and Integrative Biology at Massachusetts General Hospital
Professor Richard Willson, Ph.D. , Huffington-Woestemeyer professor in chemical and biomolecular engineering at the University of Houston

For more information on Moderna Therapeutics and potential partnership opportunities, please visit www.modernatx.com .

About Moderna Therapeutics

Moderna is pioneering messenger RNA Therapeutics™ , an entirely new in vivo drug modality that produces human proteins or antibodies inside patient cells, which are in turn secreted or active intracellularly. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of disease conditions. Moderna has developed a broad intellectual property estate, including more than 320 patent applications covering novel nucleotide chemistries and drug compositions. The company plans to develop and commercialize its innovative mRNA drugs through a combination of strategic relationships as well as new formed ventures, like Onkaido LLC, its oncology Drug Development Company. Founded by Flagship VentureLabs , Cambridge-based Moderna is privately held and currently has strategic agreements with AstraZeneca and Alexion Pharmaceuticals . To learn more, visit www.modernatx.com .

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Moderna Appoints Biotechnology Investment Banking Veteran Lorence Kim as Chief Financial Officer

Thu, 06 Mar 2014 21:10:30 GMT

CAMBRIDGE, Mass., March 6, 2014 — Moderna Therapeutics , the pioneer in developing messenger RNA (mRNA) Therapeutics™, a revolutionary treatment modality to enable the in vivo production of therapeutic proteins, announced today that Lorence Kim, M.D. will join Moderna’s executive leadership team as chief financial officer effective April 21.

Prior to joining Moderna, Dr. Kim was managing director and co-head of the U.S. biotechnology investment banking effort at Goldman Sachs. While at Goldman Sachs, Dr. Kim was a member of the Healthcare Investment Banking Group for nearly 14 years and was named a managing director in 2008.

“Moderna is now in a strong financial position, with significant funding from investors and strategic agreements with pharmaceutical leaders such as AstraZeneca and Alexion Pharmaceuticals. As we build the company, we have many options ahead of us, and our strategic choices will have a huge impact on our long-term success. This is why we looked for an atypical CFO,” said Stéphane Bancel, founding president and CEO of Moderna.

“Lorence is very accomplished in the healthcare investment community and a great match for Moderna. His addition to the Moderna leadership team increases our ability to reach our full potential and deliver many transformative mRNA drugs to patients.”

Dr. Kim has deep expertise in corporate finance and mergers and acquisitions (M&A) for the pharmaceutical and biotechnology industries, including raising several billion dollars in equity and equity-linked financings and advising on more than $55 billion in M&A transactions.

Specifically, Dr. Kim’s role as chief financial officer will focus on forging strategic agreements and partnerships with proven industry leaders, as well as structuring and financing Moderna’s internal capabilities, in order to maximize the benefit of the company’s transformative technology for patients.

“I am thrilled to be joining such an innovative and forward-looking company,” said Dr. Kim. “I’ve been consistently impressed with the vast potential of Moderna’s novel technology, and it will be exciting to expand and accelerate its reach while in this new role. Moderna compelled me with its unique combination of disruptive science and technology, $450 million of capital to invest and a very experienced team with a broad strategic vision. I look forward to helping the company grow in this extremely exciting time.”

Dr. Kim earned his AB, magna cum laude, from Harvard University in Biochemical Sciences. He earned his MBA in Healthcare Management as a Palmer Scholar from the Wharton School of the University of Pennsylvania and his MD from the University of Pennsylvania School of Medicine.

For more information on Moderna Therapeutics and potential partnership opportunities, please visit www.modernatx.com .

About Moderna Therapeutics

Moderna is pioneering messenger RNA Therapeutics™ , an entirely new in vivo drug modality that produces human proteins or antibodies inside patient cells, which are in turn secreted or active intracellularly. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of disease conditions. Moderna has developed a broad intellectual property estate, including more than 250 patent applications covering novel nucleotide chemistries and drug compositions. The company plans to develop and commercialize its innovative mRNA drugs through a combination of strategic relationships as well as newly formed ventures. Founded in late 2010 by Flagship VentureLabs , Cambridgebased Moderna is privately held and currently has strategic agreements with AstraZeneca and Alexion Pharmaceuticals . To learn more, visit www.modernatx.com .

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Moderna Launches Onkaido Therapeutics to Focus on the Development of mRNA Therapeutics™ in Oncology with $20 Million Capital Commitment

Tue, 14 Jan 2014 21:12:03 GMT

CAMBRIDGE, Mass., Jan. 14, 2014 — Moderna Therapeutics , the pioneer in developing messenger RNA (mRNA) Therapeutics™, a revolutionary treatment modality to enable the in vivo production of therapeutic proteins, announced today the creation of Onkaido Therapeutics, a new venture focused exclusively on developing mRNA-based oncology treatments. Onkaido Therapeutics will launch with $20 million in committed capital from Moderna. The new company will focus initially on 15 pre-clinical drug candidates in the areas of apoptosis, central regulatory nodes and immunotherapy. Several of these candidates are already under evaluation in in vivo disease models. By establishing Onkaido as a standalone unit, Moderna intends to accelerate the development of mRNA Therapeutics™ for oncology and deliver the potential of these new cancer treatments to patients.

Stephen Hoge, M.D., senior vice president, corporate development and new drug concepts at Moderna will take on the added role of founding chief executive officer for Onkaido, and Peter F. Smith, Ph. D., former head of non-clinical discovery and development at Millennium Pharmaceuticals, will serve as founding chief scientific officer.

“The vast potential for messenger RNA Therapeutics will only be tapped by simultaneously industrializing our platform and developing our key programs in the clinic as quickly and safely as possible,” said Stéphane Bancel, president and founding CEO, Moderna Therapeutics. “Moderna’s novel technology holds great therapeutic promise in oncology. We will benefit by enabling Stephen, Peter and the scientific team to focus intensely on the best, most rapid path toward turning our discoveries into cancer therapies that make a difference for patients.”

mRNA Therapeutics™ are designed to elude the body’s innate immune response and work with its natural processes, enabling the in vivo production of both intracellular proteins, which remain within the cells, and secreted proteins, which are released into the bloodstream and act to restore function elsewhere in the body. As a result, the mRNA Therapeutics™ platform will enable Onkaido to reach known anti-cancer targets that are not addressable through current approaches.

“Basic research in the past decade has uncovered innumerable targets in cancer, but our ability to reach them therapeutically remains limited,” said Dr. Stephen Hoge, founding CEO, Onkaido Therapeutics. “We are thrilled and humbled by the opportunity to use this new modality to do things that have never been done before in the fight against cancer.”

The new oncology company will be led by proven industry leaders with combined decades of experience in biotech strategy and clinical development, as well as expertise in oncology:

Stephen Hoge, M.D. is Moderna’s senior vice president, corporate development and new drug concepts, and founded Moderna’s internal efforts in oncology upon joining Moderna. Prior to Moderna, Dr. Hoge was a partner at McKinsey & Company and a leader in the firm’s global Healthcare and Corporate Finance practices advising senior management teams of leading biotech and pharmaceutical companies on a wide range of strategic, financial and operational issues. Before that, he was a physician in New York City.

Peter F. Smith, Ph.D. has 30 years of pharmaceutical leadership experience with previous roles held at Millennium Pharmaceuticals, Searle/Monsanto, Merck Research Laboratories and SmithKline. At Millennium, Dr. Smith served as senior vice president responsible for all non-clinical discovery and development efforts and helped lead all research and development. Over the course of his career, Dr. Smith has also driven the non-clinical development of several major, marketed drugs including Celebrex, Inspra and Velcade. Dr. Smith holds a doctorate in pharmacology and toxicology from the University of Arizona and is a sought after industry expert with more than 50 academic publications to-date.

About Onkaido Therapeutics

About Moderna Therapeutics

Moderna is pioneering messenger RNA Therapeutics™ , an entirely new in vivo drug modality that produces human proteins or antibodies inside patient cells, which are in turn secreted or active intracellularly. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of disease conditions. Moderna has developed a broad intellectual property estate, including more than 250 patent applications covering novel nucleotide chemistries and drug compositions. The company plans to develop and commercialize its innovative mRNA drugs through a combination of strategic relationships as well as newly formed ventures. Founded in late 2010 by Flagship VentureLabs , Cambridgebased Moderna is privately held and currently has strategic agreements with AstraZeneca and Alexion Pharmaceuticals . To learn more, visit www.modernatx.com .

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Alexion Pharmaceuticals and Moderna Therapeutics Announce Exclusive Strategic Agreement to Develop Messenger RNA Therapeutics™ for Rare Diseases

Mon, 13 Jan 2014 21:14:33 GMT

CHESHIRE, Conn. and CAMBRIDGE, Mass., Jan. 13, 2014 — Alexion Pharmaceuticals, Inc. (Nasdaq:ALXN) and Moderna Therapeutics today announced an exclusive strategic agreement for the discovery and development of messenger RNA Therapeutics™ to treat rare diseases. Alexion is a global leader in the development and commercialization of breakthrough therapies for patients with severe and life-threatening rare diseases. Moderna is a pioneer in developing messenger RNA (mRNA) Therapeutics™, a highly innovative treatment modality to enable the in vivoproduction of therapeutic proteins.

Messenger RNA Therapeutics™ are designed to directly utilize the body’s natural processes to enable the in vivo production of both intracellular proteins, which remain within the cells, and secreted proteins, which are released into the bloodstream and act to restore function elsewhere in the body. As a result, the mRNA Therapeutics™ platform has the potential to speed the development and manufacture of treatments for many rare diseases that are currently untreatable with existing technologies.

Under the agreement, Alexion will make an upfront payment to Moderna of $100 million to purchase 10 product options to develop and commercialize treatments for rare diseases with Moderna’s mRNA Therapeutics™ platform. Alexion will lead the discovery, development and commercialization of the treatments produced through this broad, long-term strategic agreement, while Moderna will retain responsibility for the design and manufacture of the messenger RNA against selected targets. Following option exercise by Alexion, Moderna will be entitled to drug development and commercial milestone payments, as well as high single to double digit royalties on commercial sales. In addition, Alexion has made a $25 million preferred equity investment into Moderna today.

“This broad and long-term strategic agreement represents a significant expansion of Alexion’s capabilities for drug discovery, and provides us with the strong potential to further our mission of developing novel and breakthrough therapies for patients with severe and life-threatening rare diseases,” said Leonard Bell, M.D., Chief Executive Officer of Alexion Pharmaceuticals. “We consider Moderna’s mRNA Therapeutics to be an especially exciting drug discovery platform, and we look forward to combining Moderna’s deep expertise in mRNA with Alexion’s significant global resources and expertise in drug discovery, clinical studies, regulatory affairs and commercialization for the benefit of patients worldwide.”

“Moderna’s strategy is to aggressively seek to commercialize its mRNA platform for patients in need,” said Stéphane Bancel, President and founding CEO of Moderna. “Given the broad potential of this new drug modality, we wanted to forge a few long-term strategic relationships with best-in-class companies. As we were looking for a global leader with a proven track record in the discovery, development and global commercialization of treatments for patients with rare diseases, entering into a strategic agreement with Alexion became an obvious choice for us. We were very impressed with the Alexion team’s passion to treat patients with severe and life-threatening diseases, its biology expertise, as well as its clinical and commercial capabilities. We are very pleased and honored to work with such a dedicated company to bring new medicines to patients.”

About Alexion

Alexion Pharmaceuticals, Inc. is a biopharmaceutical company focused on serving patients with severe and rare disorders through the innovation, development and commercialization of life-transforming therapeutic products. Alexion is the global leader in complement inhibition and has developed and markets Soliris ^® (eculizumab) as a treatment for patients with PNH and aHUS, two debilitating, ultra-rare and life-threatening disorders caused by chronic uncontrolled complement activation. Soliris is currently approved in nearly 50 countries for the treatment of PNH, and in the United States, European Union, Japan and other countries for the treatment of aHUS. Alexion is evaluating other potential indications for Soliris in additional severe and ultra-rare disorders beyond PNH and aHUS, and is developing other highly innovative biotechnology product candidates across multiple therapeutic areas. This press release and further information about Alexion Pharmaceuticals, Inc. can be found at: www.alexionpharma.com .

Contact:
Alexion Pharmaceuticals, Inc.
Irving Adler
203-271-8210
Executive Director, Corporate Communications

Media:
Kim Diamond
203-439-9600
Senior Director, Corporate Communications

Investors:
Rx Communications
Rhonda Chiger
917-322-2569

About Moderna Therapeutics

Moderna is pioneering messenger RNA Therapeutics™ , an entirely new in vivo drug modality that produces human proteins or antibodies inside patient cells, which are in turn secreted or active intracellularly. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of disease conditions. Moderna has developed a broad intellectual property estate, including more than 250 patent applications covering novel nucleotide chemistries and drug compositions. The company plans to develop and commercialize its innovative mRNA drugs through a combination of strategic relationships as well as newly formed ventures. Founded in late 2010 by Flagship VentureLabs , Cambridgebased Moderna is privately held and currently has strategic agreements with AstraZeneca and Alexion Pharmaceuticals . To learn more, visit www.modernatx.com .

Safe Harbor Statement

This news release contains forward-looking statements, including statements related to anticipated target selection, drug discovery, clinical development, regulatory and commercial milestones resulting from the agreement between Alexion and Moderna. Forward-looking statements are subject to factors that may cause Alexion's results and plans to differ from those expected, including for example, outcomes of pre-clinical and clinical studies, success in manufacturing target products, decisions of regulatory authorities regarding marketing approvals, and a variety of other risks set forth from time to time in Alexion's filings with the Securities and Exchange Commission, including but not limited to the risks discussed in Alexion's Quarterly Report on Form 10-Q for the period ended September 30, 2013, and in Alexion's other filings with the Securities and Exchange Commission. Alexion does not intend to update any of these forward-looking statements to reflect events or circumstances after the date hereof, except when a duty arises under law.

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Moderna Therapeutics to Present at 32nd Annual J.P. Morgan Healthcare Conference

Mon, 06 Jan 2014 21:15:36 GMT

CAMBRIDGE, Mass., Jan. 6, 2014 — Moderna Therapeutics , the pioneer in developing messenger RNA (mRNA) Therapeutics™, a revolutionary new treatment modality to enable the in vivo production of therapeutic proteins, announced today that President and founding Chief Executive Officer Stéphane Bancel will present a company overview at the 32nd Annual J.P. Morgan Healthcare Conference.

Bancel will focus on the potential of Moderna’s mRNA therapeutics to transform drug development across a wide range of diseases and improve patients’ lives.

The presentation will take place on Tuesday, January 14 at 8:30 a.m. PT in Elizabethan C/D at the Westin St. Francis Hotel in San Francisco.

For more information on Moderna Therapeutics please visit www.modernatx.com .

About Moderna Therapeutics

Moderna is pioneering messenger RNA Therapeutics™ , an entirely new in vivo drug modality that produces human proteins or antibodies inside patient cells, which are in turn active intracellularly or secreted. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of disease conditions. Moderna has developed a broad intellectual property estate, including more than 250 patent applications with more than 12,000 claims ranging from novel nucleotide chemistries to specific drug compositions. The company plans to develop and commercialize its innovative mRNA drugs—initially for rare diseases and oncology—while partnering drug candidates in other therapeutic areas in order to rapidly deliver this innovation to patients. Moderna is a privately held company based in Cambridge, Massachusetts. Visit www.modernatx.com to learn more.

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Moderna Therapeutics Appoints Theo Melas-Kyriazi to Board of Directors

Tue, 17 Dec 2013 22:21:35 GMT

CAMBRIDGE, Mass., December 17, 2013 —Moderna Therapeutics, the pioneer in developing messenger RNA (mRNA) Therapeutics™, a revolutionary new treatment modality to enable the in vivo production of therapeutic proteins, announced today that Theo Melas-Kyriazi, CFO of Levitronix Technologies LLC and a director of Valeant Pharmaceuticals International (NYSE: VRX and TSX: VRX) has joined Moderna’s board of directors.

Melas-Kyriazi brings extensive financial and executive leadership experience to Moderna, having previously served as CFO and vice president of corporate strategy for Thermo Electron Corporation (now Thermo Fisher Scientific). He also has experience as CEO of Thermo Spectra Corporation, a former subsidiary of Thermo Electron, and as a board member and investor in several early stage health sciences companies. Melas-Kyriazi joins Moderna’s board as the company embarks on the next phase of its growth strategy focused on clinical development and building the world’s leading mRNA platform.

“I am thrilled that Theo is joining the Moderna board of directors. He brings important expertise and skill to the company as we scale up to build the leader in mRNA therapeutics,” said Stéphane Bancel, president and founding CEO of Moderna. “With his experience as CFO of Thermo Electron, a large, public life sciences company, and as a director of Valeant for the last ten years, during a period of extraordinary growth, he will be a tremendous asset to help guide Moderna’s future growth.”

“I am truly excited to be joining the board of Moderna, a company with tremendous potential to transform drug development,” said Melas-Kyriazi. “Moderna’s novel platform and ambitious vision stand out among early biotechnology leaders, and I look forward to helping the company deliver on its mission to improve the lives of patients across a broad range of diseases.”

About Moderna Therapeutics

Moderna is pioneering messenger RNA Therapeutics™ , an entirely new in vivo drug modality that produces human proteins or antibodies inside patient cells, which are in turn active intracellularly or secreted. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of disease conditions. Moderna has developed a broad intellectual property estate, including more than 200 patent applications with more than 10,000 claims ranging from novel nucleotide chemistries to specific drug compositions. The company plans to develop and commercialize its innovative mRNA drugs—initially for rare diseases and oncology—while partnering drug candidates in other therapeutic areas in order to rapidly deliver this innovation to patients. Moderna is a privately held company based in Cambridge, Massachusetts. Visit www.modernatx.com to learn more.

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Moderna Closes New Financing Round Raising $110M to Develop Messenger RNA Therapeutics™

Wed, 20 Nov 2013 22:15:20 GMT

Latest round boosts capital reserves to advance numerous drug candidates into clinical testing directly and through strategic partners

CAMBRIDGE, Mass., November 20, 2013 — Moderna Therapeutics , the pioneer in developing messenger RNA (mRNA) Therapeutics™, a revolutionary new treatment modality to enable the in vivo production of therapeutic proteins, announced today the completion of a new financing round, raising $110 million. In aggregate, Moderna has secured more than $415 million in financing and partnerships to date from the pharmaceutical and government sectors, as well as from private investors led by founding investor Flagship Ventures.

“We are pleased with the confidence and enthusiasm that our investors continue to demonstrate by providing the resources to advance Moderna’s clinical development platform,” said Stéphane Bancel, president and founding CEO of Moderna. “With their support, and $340 million in the bank and available now, we are working hard to deliver on the promise of messenger RNA Therapeutics™ to provide transformative medicines for patients.”

This round of financing underscores a successful and significant year for Moderna. Emerging from stealth mode in December 2012, the company has garnered the attention of the scientific community, and attracted growing interest in its mRNA platform in 2013 as demonstrated by:

The completion of one of the biggest pre-clinical partnerships in biotech history with AstraZeneca , which made an upfront payment of $240M for the right to 40 drug options in cardiovascular, metabolic diseases and oncology
The publication in Nature Biotechnology of the first peer-reviewed, scientific article describing the ability of messenger RNA Therapeutics™ to stimulate in vivo production of human proteins to achieve a long-term therapeutic effect
Receipt of a grant of up to $25M from the Defense Advanced Research Projects Agency (DARPA) for the development of therapies to defend against infectious diseases and engineered biological threats
Recruitment of a dozen biotech industry veterans

“Given the potential of Moderna’s technology to transform the treatment of a wide range of diseases, we have made the strategic decision to remain private at this time, even in the face of a favorable public market environment for biotech IPOs,” added Bancel.

“The significant support we have received from our investor base and partners will enable us to strike the right balance between urgency and care to build a durable scientific platform and propel the field forward, without the short-term distractions that can side-track a young public company from its mission.”

About Moderna Therapeutics

Moderna is pioneering messenger RNA Therapeutics™ , an entirely new in vivo drug modality that produces human proteins or antibodies inside patient cells, which are in turn active intracellularly or secreted. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of disease conditions. Moderna has developed a broad intellectual property estate, including more than 200 patent applications with more than 10,000 claims ranging from novel nucleotide chemistries to specific drug compositions. The company plans to develop and commercialize its innovative mRNA drugs—initially for rare diseases and oncology—while partnering drug candidates in other therapeutic areas in order to rapidly deliver this innovation to patients. Moderna is a privately held company based in Cambridge, Massachusetts. Visit www.modernatx.com to learn more.

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Moderna Therapeutics Named to the World Economic Forum’s Community of “Global Growth Companies”

Thu, 24 Oct 2013 22:12:26 GMT

Innovator in mRNA therapeutics™ recognized for potential to develop drugs for untreatable diseases

CAMBRIDGE, Mass., October 24, 2013 — Moderna Therapeutics , the pioneering company developing messenger RNA (mRNA) therapeutics™, a revolutionary new treatment modality to enable the in vivo production of therapeutic proteins, has been named to the World Economic Forum’s community of the fastest-growing “Global Growth Companies” (GGC).

Moderna is one of three North American global health companies to receive this year’s honor and is recognized by the Forum for being an industry leader in innovative mRNA therapeutics. The company’s initial focus is on developing and commercializing its therapeutics in oncology and rare diseases, and it will partner future drug candidates in areas such as cardiovascular and metabolic disorders. In addition, Moderna was recently awarded a grant by the United States Defense Advanced Research Projects Agency (DARPA) to develop mRNA drugs to protect against a wide range of known and unknown emerging infectious diseases and engineered biological threats.

“When choosing entrants to our Community of Global Growth Companies, we assess companies on their business model, annual revenues and growth rates, executive leadership and market position,” said David Aikman, Managing Director, Head of New Champions, World Economic Forum. “Moderna is a pioneering group with clear potential to shape the future in its relevant business sectors and so is a perfect fit to our GGC community.”

“We take our commitment to delivering on the promise of messenger RNA therapeutics very seriously, as it has the potential to transform the lives of patients with a wide range of diseases, including many that are untreatable today,” said Stéphane Bancel, president and founding CEO of Moderna. “We are honored to be recognized for our efforts to advance our platform and ensure its potential is realized on a global scale, and we look forward to being a member of the World Economic Forum community.”

The World Economic Forum’s Community of Global Growth Companies (GGC) was formed in 2007 to engage dynamic high-growth companies with the potential to be tomorrow’s industry leaders and to become a driving force of economic and social change. Members of the Community of Global Growth Companies convene every year at the Chinese-hosted Annual Meeting of the New Champions and at the World Economic Forum’s regional meetings, and collaborate through the private area of the World Economic Forum's website, an exclusive online networking platform for business and government leaders.

About the World Economic Forum

The World Economic Forum is an independent international organization committed to improving the state of the world by engaging leaders in partnerships to shape global, regional and industry agendas. Incorporated as a foundation in 1971, and headquartered in Geneva, Switzerland, the World Economic Forum is impartial and not-for-profit; it is tied to no political, partisan or national interests ( www.weforum.org ).

About Moderna Therapeutics

Moderna is pioneering messenger RNA Therapeutics™ , an entirely new in vivo drug modality that produces human proteins or antibodies inside patient cells, which are in turn active intracellularly or secreted. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of disease conditions. Moderna has developed a broad intellectual property estate, including more than 200 patent applications with more than 10,000 claims ranging from novel nucleotide chemistries to specific drug compositions. The company plans to develop and commercialize its innovative mRNA drugs—initially for rare diseases and oncology—while partnering drug candidates in other therapeutic areas in order to rapidly deliver this innovation to patients. Moderna is a privately held company based in Cambridge, Massachusetts. Visit www.modernatx.com to learn more.

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Moderna Therapeutics Announces Expansion to New Headquarters and Laboratories at 200 Tech Square

Thu, 17 Oct 2013 22:10:06 GMT

CAMBRIDGE, Mass., October 17, 2013 — Moderna Therapeutics, the pioneering company developing messenger RNA therapeutics™ , a revolutionary new treatment modality to enable the in vivo production of therapeutic proteins, announces today the expansion of their headquarters to Technology Square in Cambridge, MA. With the addition of the new space, Moderna will have up to 100 full-time employees at two locations by the end of 2013.

“We are thrilled to expand our presence in Massachusetts, the country’s most important hub of biotech innovation, which has been a great source of talent and ideas to Moderna from its earliest days,” said Stéphane Bancel, president and founding CEO of Moderna. “With this facility, we intend to deliver on the promise of messenger RNA therapeutics™ by developing transformative new medicines for a wide range of diseases, including many that are untreatable today.”

The new site will be the center of Moderna’s R&D and provide a significant increase in laboratory space for formulation, screening, and both in-vivo and in-vitro studies. The facility houses administrative and corporate needs as well as IT specialists and is equipped with state of the art technology to enable virtual collaboration and cloud-based computing. Moderna’s previous Cambridge headquarters will continue to serve as an additional site for manufacturing and quality.

“In Massachusetts we invest in the life sciences because we are choosing to shape our own future,” said Governor Deval Patrick. “I congratulate Moderna on their new facility and look forward to the jobs and economic opportunities they will bring.”

“On behalf of the team at the Massachusetts Life Sciences Center, congratulations to Moderna on its expansion here in the Commonwealth,” said MLSC President & CEO Dr. Susan Windham-Bannister. “We are pleased to have awarded Moderna 2011 and 2012 Tax Incentives tied to their new job creation in Massachusetts and look forward to the great R&D efforts in ‘messenger RNA Therapeutics’ that will be taking place in this new space.”

About the Massachusetts Life Sciences Center

The Massachusetts Life Sciences Center (MLSC) is an investment agency that supports life sciences innovation, research, development and commercialization. The MLSC is charged with implementing a 10-year, $1-billion, state-funded investment initiative. These investments create jobs and support advances that improve health and well-being. The MLSC offers the nation’s most comprehensive set of incentives and collaborative programs targeted to the life sciences ecosystem. These programs propel the growth that has made Massachusetts the global leader in life sciences. The MLSC creates new models for collaboration and partners with organizations, both public and private, around the world to promote innovation in the life sciences. For more information, visit www.masslifesciences.com .

About Moderna Therapeutics

Moderna is pioneering messenger RNA Therapeutics™ , an entirely new in vivo drug modality that produces human proteins or antibodies inside patient cells, which are in turn active intracellularly or secreted. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of disease conditions. Moderna has developed a broad intellectual property estate, including more than 200 patent applications with more than 10,000 claims ranging from novel nucleotide chemistries to specific drug compositions. The company plans to develop and commercialize its innovative mRNA drugs—initially for rare diseases and oncology—while partnering drug candidates in other therapeutic areas in order to rapidly deliver this innovation to patients. Moderna is a privately held company based in Cambridge, Massachusetts. Visit www.modernatx.com to learn more.

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DARPA Awards Moderna Therapeutics a Grant for up to $25 Million to Develop Messenger RNA Therapeutics™

Wed, 02 Oct 2013 22:07:54 GMT

Research to focus on antibody production for immune defense

CAMBRIDGE, Mass., October 2, 2013 — Moderna Therapeutics, the company pioneering messenger RNA therapeutics™ , a revolutionary new treatment modality to enable the in vivo production of therapeutic proteins, announced today that the Defense Advanced Research Projects Agency (DARPA) has awarded the company up to $25 million to research and develop its messenger RNA therapeutics™ platform as a rapid and reliable way to make antibody-producing drugs to protect against a wide range of known and unknown emerging infectious diseases and engineered biological threats.

Messenger RNA therapeutics™ can be designed to tap directly into the body’s natural processes to produce antibodies without exposing people to a weakened or inactivated virus or pathogen, as is the case with the vaccine approaches currently being tested. As a result, Moderna’s messenger RNA therapeutics™ platform has the potential to speed the development and manufacture of treatments that can produce a safer, more reliable and more robust immune response than existing technologies.

“We are honored to be chosen by DARPA for this important grant, which will greatly accelerate our efforts to develop antibody messenger RNA therapeutics™ to combat a wide range of infectious diseases,” said Stéphane Bancel, president and founding CEO of Moderna. “We were awarded this major grant after an intense and rigorous scientific review, and it is a testament to our team’s progress and to the profound implications of messenger RNA therapeutics™ that our work was funded. We look forward to further expanding the development of our platform into this critically important new therapeutic area.”

This $24.6 million grant could support research for up to 5 years to advance promising antibody- producing drug candidates into preclinical testing and human clinical trials. The company also received a $0.7 million “seedling” grant from DARPA in March to begin work on the project.

This grant is part of a DARPA program called ADEPT: PROTECT (Autonomous Diagnostics to Enable Prevention and Therapeutics: Prophylactic Options to Environmental and Contagious Threats). The goal is to develop platform technologies that can be deployed safely and rapidly to provide the U.S. population with near-immediate protection against emerging infectious diseases and engineered biological weapons, even in cases when the pathogen or infectious agent is unknown. For more information about DARPA, visit http://www.darpa.mil/about-us/about-darpa .

About Moderna Therapeutics

Moderna is pioneering messenger RNA Therapeutics™ , an entirely new in vivo drug modality that produces human proteins or antibodies inside patient cells, which are in turn active intracellularly or secreted. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of disease conditions. Moderna has developed a broad intellectual property estate, including 144 patent applications with 6,910 claims ranging from novel nucleotide chemistries to specific drug compositions. The company plans to develop and commercialize its innovative mRNA drugs—initially for rare diseases and oncology—while partnering drug candidates in - 2 - other therapeutic areas in order to rapidly deliver this innovation to patients. Moderna is a privately held company based in Cambridge, Massachusetts. Visit www.modernatx.com to learn more.

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Moderna Therapeutics Named a “Fierce 15” Biotech Company for 2013

Tue, 24 Sep 2013 22:05:38 GMT

CAMBRIDGE, Mass., September 24, 2013 —Moderna Therapeutics, the pioneering company developing messenger RNA therapeutics™ , a revolutionary new treatment modality to enable the in vivo production of therapeutic proteins, announced today that FierceBiotech has named Moderna as one of 2013’s Fierce 15 .

The award designates Moderna as one of the most promising private companies in the biotech industry. Companies from around the world are selected based on strength of technology, venture funding, and their potential to positively impact the treatment of human diseases.

“With its messenger RNA therapies, Moderna has the potential to reinvent the way protein therapies are developed and created,” says Executive Editor Ryan McBride. “AstraZeneca validated this potential in a landmark deal that gives Moderna the cash and resources to become a major player in oncology and rare diseases, making this company one to watch closely.”

“We are honored to be selected as a Fierce 15 company and recognized for our vision in the field of messenger RNA therapeutics™,” said Stéphane Bancel, president and founding CEO of Moderna. “This award comes at an exciting time for us, as we recently published our first proof of concept study in Nature Biotechnology, providing important validation of our platform’s potential to treat many diseases that cannot be addressed today. We are thrilled to be moving forward quickly and safely to translate our technology into treatment for patients in need.”

About FierceBiotech

FierceBiotech is the biotech industry’s daily monitor – a free email newsletter and web resource providing the latest biotech news, articles, and resources related to clinical trials, drug discovery, FDA approval and regulation, biotech company deals and more. More than 90,000 top biotech professionals rely on FierceBiotech for an insider briefing on the day’s top stories. Sign up is free at www.fiercebiotech.com .

About Moderna Therapeutics

Moderna is pioneering messenger RNA Therapeutics™ , an entirely new in vivo drug modality that produces human proteins or antibodies inside patient cells, which are in turn active intracellularly or secreted. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of disease conditions. Moderna has developed a broad intellectual property estate, including 144 patent applications with 6,910 claims ranging from novel nucleotide chemistries to specific drug compositions. The company plans to develop and commercialize its innovative messenger RNA drugs—initially for rare diseases and oncology—while partnering drug candidates in other therapeutic areas in order to rapidly deliver this innovation to patients. Moderna is a privately held company based in Cambridge, Mass. Visit www.modernatx.com to learn more.

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Study Published in Nature Biotechnology Demonstrates Ability of Moderna’s Novel Therapeutic Paradigm to Repair and Regenerate Damaged Heart Tissue

Sun, 08 Sep 2013 22:03:39 GMT

Messenger RNA therapeutics™ successfully induced high-level, transient expression of VEGF-A protein in the heart, stimulating the growth of cardiovascular cells and improving heart function and survival in an animal model of myocardial infarction

CAMBRIDGE, Mass., Sept. 8, 2013 — Moderna Therapeutics, the pioneering company developing messenger RNA therapeutics™ , a revolutionary new treatment modality to enable the in vivo production of therapeutic proteins, announced today that two of its academic co-founders published a study in Nature Biotechnology demonstrating that Moderna’s messenger RNA therapeutics™ platform induced in vivo production of proteins to stimulate blood vessel growth, repair damaged heart tissue, and improve outcomes in a mouse model of myocardial infarction (MI).

The research, led by Moderna’s academic co-founder Kenneth Chien, M.D., Ph.D., with the help of academic co-founder Derrick Rossi, Ph.D., provides proof-of-concept that messenger RNA therapeutics™ provide an effective and robust platform for gene transfer that could be applicable across a number of diseases. The Nature Biotechnologypaper is the first peer-reviewed scientific publication describing the ability of messenger RNA therapeutics™ to stimulate in vivo production of human proteins and to achieve a long-term therapeutic effect.

Researchers injected mice with a single dose of VEGF-A-coded messenger RNA in the heart shortly following myocardial infarction to trigger production of the VEGF-A protein. VEGF-A (vascular endothelial growth factor-A) is an important angiogenic factor protein that mediates the growth of blood vessels. Mice injected with messenger RNA went on to generate new cardiovascular cells and show improvements on measures of cardiac function and survival when compared with controls.

“Regeneration of heart tissue through the mobilization of heart progenitor cells holds tremendous potential for the treatment of myocardial infarction and other forms of heart injury but has remained an elusive goal for over a decade,” said Dr. Chien. “Over the past 20 years, many labs have attempted to repair damaged heart muscle in animal models by delivering VEGF-A and other factors to the heart using recombinant proteins, DNA plasmids, and engineered viruses—with little if any clinical success. The results we have seen using messenger RNA therapeutics™ mark the start of a new and exciting phase of drug development with potentially profound implications for patients.”

In the study, the mice injected with VEGF-A-coded messenger RNA produced a “pulse-like” expression of a significant quantity of VEGF-A near the injection site. The production of VEGF-A in turn mobilized heart progenitor cells to produce functional, intact blood vessels in the heart and led to marked improvements in heart function and improved long-term survival of the mice for at least one year post-MI. Researchers attribute these effects to a reduction in the size of infarct region and an increase in capillary density in the affected region. In this way, the high-level, transient production of VEGF-A led to a sustained therapeutic improvement by altering the behavior of heart progenitor cells.

Introduction of modRNA was not associated with an innate immune response, further supporting the safety and effectiveness of this novel approach.

“We are extremely proud that our messenger RNA therapeutics™ platform enabled Ken to realize his vision and clinical intuition of the last 20 years,” said Stéphane Bancel, president and founding CEO of Moderna. “Beyond the considerable implications for the treatment of heart injury, this publication provides an important validation of the potential of messenger RNA therapeutics™ across a wide range of diseases. The ability to stimulate protein production within cells will enable the treatment of many diseases that cannot be addressed today. What is also exciting is the fact that the Moderna team has considerably improved our technology since its performance for this groundbreaking study. The current generation of our technology is capable of producing 50 times more protein per dose, and our R&D team has many new improvements in the making, further increasing the potential of messenger RNA therapeutics™ to transform drug development and improve patients’ lives.”

Moderna in March announced an exclusive strategic option agreement with AstraZeneca to discover, develop, and commercialize messenger RNA therapeutics™ for the treatment of serious cardiovascular, metabolic, and renal diseases as well as selected targets in oncology. Targets covered under the agreement included the use of VEGF in the treatment of cardiovascular disease.

“Cardio-metabolic medicine is a core therapy area for AstraZeneca, and part of our research focuses on identifying novel therapies that will activate progenitor cells within the heart to turn into healthy heart cells. Recent findings by Dr. Chien and his team will play an important role in our work with Karolinska Institutet to advance cardiac regenerative therapy and our collaboration with Moderna Therapeutics to discover and develop messenger RNA therapeutics™,” said Dr. Marcus Schindler, Head of the AstraZeneca Cardiovascular and Metabolic Disease Innovative Medicines Unit.

About Moderna Therapeutics

Moderna is pioneering messenger RNA Therapeutics™ , an entirely new in vivo drug modality that produces human proteins or antibodies inside patient cells, which are in turn active intracellularly or secreted. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of disease conditions. Moderna has developed a broad intellectual property estate, including 144 patent applications with 6,910 claims ranging from novel nucleotide chemistries to specific drug compositions. The company plans to develop and commercialize its innovative messenger RNA drugs—initially for rare diseases and oncology—while partnering drug candidates in other therapeutic areas in order to rapidly deliver this innovation to patients. Moderna is a privately held company based in Cambridge, Mass. Visit www.modernatx.com to learn more.

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Biotech Industry Veteran Joseph Bolen Joins Moderna Therapeutics to be President of Research and Development

Wed, 31 Jul 2013 22:00:42 GMT

CAMBRIDGE, Mass., July 31, 2013 —Moderna Therapeutics, the pioneering company developing messenger RNA therapeutics™ , a revolutionary new treatment modality to enable the in vivo production of therapeutic proteins, announced today that Joseph Bolen, Ph.D., joined Moderna as its president of research and development (R&D).

Dr. Bolen, the chief scientific officer and global head of oncology research at Millennium: The Takeda Oncology Company has served in senior leadership positions at Millennium since 1999. As the Millennium CSO and global head of research, Dr. Bolen has a proven track record of leading a large and multi-faceted international R&D organization while fostering a culture of scientific innovation within a global commercial-stage company. He has more than 30 years of industry and research experience and has been at the forefront of cancer and immunology research since starting his careeras a scientist at the National Institutes of Health (NIH).

“Messenger RNA therapeutics™ promises to vastly expand the number of diseases we can treat and usher in an entirely new era of drug discovery and development,” said Stéphane Bancel, president and founding CEO of Moderna. “To advance our internal programs in genetic rare diseases and oncology and support partners in many additional therapeutic areas, we are building an R&D organization that is second to none. Joe’s deep scientific knowledge and strong vision will help us to create a culture and a structure that can move rapidly to turn scientific innovation into therapies that make a true difference for patients. I am very happy and honored to partner with such an accomplished and thoughtful scientific leader and drug hunter as Joe to build a very special company for patients and our team.”

Dr. Bolen will oversee all aspects of R&D at Moderna, including discovery research, preclinical and clinical development, and clinical operations. His team will drive the discovery and development of compounds to support Moderna’s existing and future research partnerships, as well as its internal efforts in rare diseases and oncology.

“The global scientific community is just beginning to understand the significant promise of modified mRNA technology to advance science and improve human health. I am tremendously excited about expanding our knowledge of this new scientific field and pioneering the clinical application of this modality, which holds such great potential to treat a vast array of human diseases,” Dr. Bolen said. “As someone who has dedicated his career to the discovery and development of innovative new medicines for people with cancer, I believe the therapeutic potential of Moderna’s novel technology in oncology is particularly compelling. I am excited to work with the dedicated scientists and physicians at Moderna to build a respected R&D organization that is both scalable and flexible, with a clear focus on bringing better medicines to people with life-threatening illnesses.”

Prior to joining Millennium in 1999, Dr. Bolen held senior R&D positions at Hoechst Marion Roussel, Schering-Plough, and Bristol-Myers Squibb. He served as a member of the graduate faculty in the Department of Molecular Biology at Princeton University and spent 10 years at NIH where he madesignificant contributions to the discovery of a class of proteins known as tyrosine kinase oncogenes as key regulators of the immune system. He has published more than 200 peer-reviewed scientific articles and book chapters and presented hundreds of lectures and seminars at leading universities and national and international scientific meetings. Dr. Bolen graduated from the University of Nebraska with a B.S. degree in Microbiology & Chemistry and a Ph.D. in Immunology and conducted his postdoctoral training in Molecular Virology at the Kansas State University Cancer Center.

About Moderna Therapeutics

Moderna is pioneering messenger RNA Therapeutics™ , an entirely new in vivo drug modality that produces human proteins or antibodies inside patient cells, which are in turn active intracellularly or secreted. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of disease conditions. Moderna has developed a broad intellectual property estate, including more than 144 patent applications with more than 6,910 claims ranging from novel nucleotide chemistries to specific drug compositions. The company plans to develop and commercialize its innovative mRNA drugs—initially for rare diseases and oncology—while partnering drug candidates in other therapeutic areas in order to rapidly deliver this innovation to patients. Moderna is a privately held company based in Cambridge, Massachusetts. Visit www.modernatx.com to learn more.

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Industry Leader John Reynders Joins Moderna Therapeutics as Chief Information Officer

Wed, 22 May 2013 21:58:43 GMT

CAMBRIDGE, Mass., May 22, 2013 — Moderna Therapeutics, the pioneering company developing messenger RNA therapeutics™ , a revolutionary new treatment modality to enable the in vivo production of therapeutic proteins, announced today that John Reynders will join Moderna in early July as its first Chief Information Officer.

Reynders is a proven leader in the design and implementation of information systems underlying all phases of drug development, from discovery to translation, clinical operations and commercial preparedness. John has also been at the vanguard of the pharmaceutical industry’s efforts in big data analytics, cloud-based computing, and innovation networks. He joins Moderna from AstraZeneca, where he served as Vice President of R&D Information, and has held senior leadership positions over the past decade at Johnson & Johnson, Lilly, and Celera Genomics.

“We are building a company capable of driving innovation on a scale and at a pace that were unimaginable until recently, and our information systems must be able to fuel that vision,” said Stephane Bancel, founding president and CEO of Moderna. “John has long been on the forefront of the information revolution that is reshaping drug development and healthcare, and he has a strong vision for how we can design an information resource capable of propelling Moderna – and the entire field of messenger RNA therapeutics™ – well into the future.”

Reynders will lead a team at Moderna in creating an information infrastructure critical to all aspects of the company's innovation model, including the use of genomic data in drug design; the integration and instant sharing of knowledge from a variety of preclinical and clinical settings; and the seamless interaction of a wide network of collaborators, partners, and potential spinoff companies.

“It is tremendously exciting to be involved in designing the drug company of the future at this critical moment in Moderna’s scale up, when knowledge of the potential application of messenger RNA therapeutics™ is exploding,” Reynders said. “For Moderna and the entire field to fully capitalize on this profound advance for patients will require a full end-to-end informatics integration of the Moderna pipeline, creating the industry’s first fully digital biotech. It will also require all of us to design new ways of working together, not just within the walls of Moderna but with a vast network of collaborators around the world. This is a once in a lifetime opportunity and I am proud to be a part of it.”

About Moderna Therapeutics

Moderna is pioneering messenger RNA Therapeutics™ , an entirely new in vivo drug modality that produces human proteins or antibodies inside patient cells, which are in turn active intracellularly or secreted. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of disease conditions. Moderna has developed a broad intellectual property estate including 120 patent applications with 6,500 claims ranging from novel nucleotide chemistries to specific drug compositions. The company plans to develop and commercialize its innovative mRNA drugs – initially for rare diseases and oncology – while partnering drug candidates in other therapeutic areas in order to rapidly deliver this innovation to patients. Based in Cambridge, Massachusetts, Moderna is privately held and was founded in 2010 by Flagship VentureLabs in association with leading scientists from Boston Children’s Hospital and Massachusetts Institute of Technology. Visit www.modernatx.com to learn more.

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Henri A. Termeer Joins Moderna Therapeutics Board of Directors

Tue, 30 Apr 2013 21:56:47 GMT

CAMBRIDGE, Mass., April 30, 2013 —Moderna Therapeutics, the pioneering company developing messenger RNA therapeutics™ —a revolutionary new treatment modality to enable the in vivo production of therapeutic proteins—announced today that Henri A. Termeer, Genzyme’s retired chairman, president, and CEO, has joined Moderna’s board of directors.

Over nearly 30 years at Genzyme, Termeer built one of the world’s most innovative and commercially successful biotechnology companies. Under his leadership, Genzyme pioneered new models for sustainably bringing first-ever therapies to patients who were previously underserved. Termeer joins Moderna’s board as the company embarks on the next phase of its growth strategy, strengthened by a strategic option agreement with AstraZeneca to develop new medicines for cardiometabolic diseases and by the continued progress of its preclinical programs in rare diseases and oncology.

“Messenger RNA therapeutics™ hold the same revolutionary potential as recombinant proteins did when they were first introduced nearly 30 years ago,” said Noubar Afeyan, Chairman of Moderna’s board and CEO of Flagship Ventures, the company’s lead investor. “Henri’s perspective will be invaluable as we work to deliver this new category of medicines for the treatment of serious diseases.”

“It is an exciting time to be joining the board of Moderna, which I believe has the potential to transform the way in which protein therapeutics are developed, and to address diseases that are not currently reachable by recombinant technologies,” Termeer said. “Moderna shares the same vision, ambition, and potential as many of the early biotechnology leaders. I look forward to helping the company deliver on its vast potential to improve the lives of patients across a wide array of disease areas.”

“Henri has unparalleled experience in building visionary companies and we are proud and excited to welcome him to our board,” said Stéphane Bancel, founding president and CEO of Moderna. “Under his leadership, Genzyme became known for its steadfast commitment to patients, and for constantly pushing new boundaries in order to bring biomedical innovation to those who were not well served by traditional approaches. This perspective will be enormously helpful to Moderna as we work to unlock the vast potential of messenger RNA therapeutics™.”

About Henri A. Termeer

Henri A. Termeer served as chairman, president and chief executive officer of Genzyme Corporation for nearly three decades. Mr. Termeer was appointed president of Genzyme in 1983, two years after the company’s founding. He became its chief executive officer in 1985 and chairman in 1988. He retired from Genzyme in June 2011 following the acquisition of Genzyme by Sanofi in a transaction valued at more than $20 billion.

Widely acknowledged for his contributions to the biotechnology industry and health care field, Mr. Termeer is active in the areas of humanitarian assistance, policy issues, and innovation in providing access to health care.

Mr. Termeer is a Board member of Massachusetts Institute of Technology Corporation and serves on its Executive Committee, a director of Massachusetts General Hospital, Board member of Partners HealthCare, a member of the Board of Fellows of Harvard Medical School and on the Board of the Biotechnology Industry Organization (BIO). He also serves on the Boards of Abiomed, Inc., AVEO Pharmaceuticals, Verastem, Inc., and Medical Simulation Corporation. In 2008, he was appointed to Massachusetts Governor Deval Patrick ’s Council of Economic Advisors and he is a co-chair of the Leadership Council of the Massachusetts Life Sciences Collaborative. Mr. Termeer is also Chairman Emeritus of the New England Healthcare Institute, a nonprofit, applied research health policy organization he was instrumental in founding. He was elected a Fellow of the American Academy of Arts and Sciences in 1999.

Mr. Termeer was Chairman of the Federal Reserve Bank of Boston’s Board of Directors from 2010-2011 and served on the Board of Directors of the Pharmaceutical Research and Manufacturers of America. Mr. Termeer studied economics at the Economische Hogeschool (Erasmus University, The Netherlands) and earned an M.B.A from the Darden School at the University of Virginia. Mr. Termeer has been recognized by several highly regarded organizations for his contributions to the health care and entrepreneurial fields.

About Moderna Therapeutics

Moderna is pioneering messenger RNA Therapeutics™ , an entirely new in vivo drug modality that produces human proteins or antibodies inside patient cells, which are in turn active intracellularly or secreted. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of disease conditions. Moderna has developed a broad intellectual property estate including 120 patent applications with 6,500 claims ranging from novel nucleotide chemistries to specific drug compositions. The company plans to develop and commercialize its innovative mRNA drugs—initially for rare diseases and oncology—while partnering drug candidates in other therapeutic areas in order to rapidly deliver this innovation to patients. Based in Cambridge, Massachusetts, Moderna is privately held and was founded in 2010 by Flagship VentureLabs in association with leading scientists from Boston Children’s Hospital and Massachusetts Institute of Technology. Visit www.modernatx.com to learn more.

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AstraZeneca and Moderna Therapeutics Announce Exclusive Agreement to Develop Pioneering Messenger RNA Therapeutics™ in Cardiometabolic Diseases and Cancer

Thu, 21 Mar 2013 21:54:39 GMT

AstraZeneca today announced an exclusive agreement with Moderna Therapeutics to discover, develop and commercialise pioneering messenger RNA therapeutics™ for the treatment of serious cardiovascular, metabolic and renal diseases as well as cancer. Messenger RNA therapeutics™ are an entirely new treatment approach that enables the body to produce therapeutic protein in vivo, opening up new treatment options for a wide range of diseases that cannot be addressed today using existing technologies.

Under the terms of the agreement, AstraZeneca will make an upfront payment of $240 million. AstraZeneca will have exclusive access to select any target of its choice in cardiometabolic diseases, as well as selected targets in oncology, over a period of up to five years for subsequent development of messenger RNA. In addition, Moderna is entitled to an additional $180 million for the achievement of three technical milestones.

Through this agreement, AstraZeneca has the option to select up to 40 drug products for clinical development and Moderna will be entitled to development and commercial milestone payments as well as royalties on drug sales ranging from high single digits to low double digits for each product. AstraZeneca will lead the preclinical, clinical development and commercialisation of therapeutics resulting from the agreement and Moderna will be responsible for designing and manufacturing the messenger RNA against selected targets.

Moderna’s unique approach uses proprietary messenger RNA containing naturally occurring nucleotide analogues, which are designed to stimulate the body’s natural ability to produce intracellular and secreted therapeutic proteins without triggering an innate immune response. The secreted proteins will be released into the bloodstream to potentially restore function elsewhere in the body. Using messenger RNA also has the potential advantage of dramatically reducing the time and expense associated with creating therapeutic proteins using current recombinant technologies.

Pascal Soriot, Chief Executive Officer of AstraZeneca, said: “Today’s agreement signals an exciting move for AstraZeneca and our focus on innovation. Together with Moderna, we are pushing the boundaries of science in the pioneering field of messenger RNA therapeutics™. Where current drug discovery technologies can target only a fraction of the disease-relevant proteins in the human genome, we have the potential to create completely new medicines to treat patients with serious cardiometabolic diseases and cancer.”

Stephane Bancel, President and founding CEO of Moderna Therapeutics, said: “This multi-year strategic agreement with AstraZeneca is a very exciting and special moment for the Moderna team. The Company’s strategy is to develop, manufacture, and commercialize innovative drugs initially in rare diseases and oncology as well as to partner other therapeutic areas with best-in-class companies. We share a common vision with AstraZeneca for how mRNA therapeutics™ will enable many new innovative drugs for targets which are totally undruggable today. We look forward to this unique opportunity where Moderna can apply its research platform, broad intellectual property portfolio and know-how to potentially contribute significantly to AstraZeneca’s pipeline.”

Effectiveness of the agreement is contingent on expiration or termination of the waiting period under the Hart Scott-Rodino Antitrust Improvements Act.

About AstraZeneca

AstraZeneca is a global, innovation-driven biopharmaceutical business that focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of cardiovascular, metabolic, respiratory, inflammation, autoimmune, oncology, infection and neuroscience diseases. AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. For more information please visit: www.astrazeneca.com

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About Moderna Therapeutics

Moderna is a pioneering an entirely new drug modality using messenger RNA Therapeutics™ to produce, in vivo, human proteins or antibodies inside patient cells that are in turn active intracellularly or secreted into the serum. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to exisiting drug modalities for a wide range of disease conditions. Moderna has developed a broad intellectual property estate including 125 patent filings with 6,500 claims ranging from novel nucelotide chemistries to specific drug compositions. The company plans to develop and commercialize its innovative mRNA drugs initially for rare diseases and oncology while partnering drug candiates in other therapeutics areas in order to rapidly maximize patient impact. Based in Cambridge MA, privately held Moderna was founded in 2010 by Flagship VentureLabs in association with leading scientists from Boston Children's Hospital and Massachusetts Institute of Technology. Visit www.modernatx.com to learn more.

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Moderna Appoints Divakar Ramamakishnan, Ph.D., as Senior Vice President of Process Development, Manufacturing and Quality

Wed, 16 Jan 2013 21:51:56 GMT

CAMBRIDGE, Mass., January 16, 2013 —Moderna Therapeutics, which is developing a new platform designed to spur the body to create its own therapeutic proteins to treat a wide range of diseases, announced today that Divakar Ramakrishnan, Ph.D., is joining the company as Senior Vice President of Process Development, Manufacturing and Quality. His appointment is effective immediately.

Moderna is pioneering messenger RNA Therapeutics™, a new biotherapeutic modality to restore protein function by enabling the body to produce its own healing proteins. In his new role, Dr. Ramakrishnan will lead drug substance, drug product and analytical development. He will also be responsible for scaling production of high purity mRNA in a current good manufacturing practices (cGMP) environment to support preclinical and clinical programs and designing a pilot manufacturing facility.

“I am very pleased to welcome Divakar as the newest member of our executive team,” said Stéphane Bancel, President and founding CEO of Moderna. “As we begin our transition into clinical development, we need a leader with unwavering commitment to quality, and the expertise needed to manage complex processes to manufacture mRNA products at the highest purity. Divakar brings unrivaled expertise in bioprocess development, manufacturing and supply chain management, and has personally led a GMP commercial plant and a network of six sites. His addition will enable us to continue our rapid progress to deliver a new, safe treatment modality to clinicians and patients.”

Dr. Ramakrishnan joins Moderna from Eli Lilly and Company. As vice president of manufacturing he supported Lilly’s animal health business with operational and supply chain responsibilities for six global sites and more than 60 contract manufacturers. He joined Lilly in 1998 as a senior scientist in Lilly Research Laboratories, and subsequently held positions including head of bioprocess development, senior director of biotech manufacturing strategy and manufacturing strategic planning and vice president of manufacturing science and technology. Dr. Ramakrishnan was also general manager of the Lilly Kinsale site in Ireland, where he led commercialization and manufacturing operations for biotech and chemical technologies, and has served as a management consultant for Cook Pharmica, LLC, developing business strategy and recruiting and directing their start-up operations and technical teams. He holds a Ph.D. in Chemical Engineering from Pennsylvania State University and an MBA from Harvard Business School.

“I am so pleased to join the Moderna team and help maximize the potential of Moderna’s innovative mRNA technology platform to address unmet medical needs for patients,” said Dr. Ramakrishnan. “I believe that Moderna has the same transformative power as recombinant DNA technology, which marked the advent of modern biotechnology thirty years ago, and expect this advance will have a similar impact for patients as well as industry.”

About Moderna Therapeutics

Moderna is revolutionizing the restoration of protein function with messenger RNA Therapeutics™ , a new treatment modality that enables the body to produce healing proteins in vivo. Moderna has created a novel method for producing a wide range of drugs in the body through the delivery of messenger RNA (mRNA) and is using this approach to develop first-ever treatments for a wide range of diseases that cannot be addressed today using existing technologies, and to drastically reduce the time and expense associated with creating therapeutic proteins using recombinant technologies. Visit www.modernatx.com to learn more.

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Moderna Appoints Stephen Hoge as Senior Vice President of Corporate Development and New Drug Concepts

Wed, 19 Dec 2012 18:23:06 GMT

CAMBRIDGE, Mass., December 19, 2012 —Moderna Therapeutics, which is developing a new platform designed to spur the body to create its own therapeutic proteins to treat a wide range of diseases, announced today that Stephen Hoge, MD, currently a partner at McKinsey & Company, will join the company as Senior Vice President of Corporate Development and New Drug Concepts on January 1st, 2013. In this newly created role, Dr. Hoge will lead corporate development efforts, including corporate strategy and business development, as well as work with scientists around the world to develop novel drug concepts enabled by messenger RNA.

Moderna is pioneering the development of messenger RNA Therapeutics ™, a novel biotherapeutic modality with the unprecedented capability of stimulating the body’s natural ability to produce therapeutic proteins. The successful development of messenger RNA Therapeutics could drastically improve the ease and speed with which novel drugs may be developed a nd dosed, potentially opening up hundreds of new targets for drug development. Given the wide variety of potential applications, a core pillar of Moderna’s business strategy will be to partner with companies, patient organizations, and others to rapidly accelerate the development of new treatments for patients.

“I am delighted and very proud to welcome Stephen Hoge, who brings a unique combination of skills to this critical position – including a deep understanding of biological pathways and a proven mastery of drug portfolio valuation,” said Stéphane Bancel, president and founding CEO of Moderna. “Stephen’s energy, creativity, passion to help patients, and vision to treat diseases in a fundamentally new way will make him a great addition to the Moderna executive team.”

Prior to Moderna, Dr. Hoge was a partner at McKinsey & Company and a leader in the firm’s global Healthcare and Corporate Finance practices. At McKinsey he advised senior management teams of leading biotech and pharmaceutical companies on a wide range of strategic, financial, and operational issues. He also led McKinsey’s thinking on R&D portfolio development and valuation. Prior to joining the firm, Dr. Hoge was a physician in New York. He holds an MD with thesis from the University of California, San Francisco and a BS in Neuroscience from Amherst College.

“I am thrilled to be joining a terrific team and a very special company,” said Dr. Hoge. “The most disruptive ideas are deceptively simple. When you really start to understand Moderna’s technology, you realize that it changes everything; not just biology, but also operations, finance, and even core beliefs about timelines and the structure of R&D. The potential implications are so profound that I knew I had to be a part of this company.”

About Moderna Therapeutics

Moderna is revolutionizing the restoration of protein function with messenger RNA Therapeutics™ , a new treatment modality that enables the body to produce healing proteins in vivo. Moderna has created a 2 novel method for producing a wide range of drugs in the body through the delivery of messenger RNA (mRNA) and is using this approach to develop first-ever treatments for a wide range of diseases that cannot be addressed today using existing technologies, and to drastically reduce the time and expense associated with creating therapeutic proteins using recombinant technologies. Visit www.modernatx.com to learn more.

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Moderna Announces $40 Million In Financing To Advance Development Of New Biotherapeutic Modality: Messenger RNA Therapeutics™

Thu, 06 Dec 2012 18:25:04 GMT

Company announces appointment of Executive Leadership Team and Scientific Advisory Board

CAMBRIDGE, Mass., Dec. 6, 2012 — Moderna Therapeutics announced today that it has closed more than $40 million in financing to date, led by Flagship Ventures and private investors, which will be used to advance multiple programs toward clinical stage development. Moderna is pioneering messenger RNA Therapeutics™, a novel biotherapeutic modality with the unprecedented capability of stimulating the body's natural ability to produce therapeutic proteins. If successful in human clinical trials, this advance will usher in the first entirely new way of making therapeutic proteins since the development of recombinant technology more than 30 years ago, with dramatic implications for both patients and industry.

Moderna was founded within Flagship VentureLabs™, an innovation foundry dedicated to institutional entrepreneuring. Over the past 18 months, Moderna has conducted proof-of-concept studies in preclinical models, including non-human primates, and has demonstrated the ability to induce in vivo production of dozens of intracellular and secreted therapeutic proteins through intramuscular, subcutaneous or intravenous administration across multiple preclinical models. The company anticipates publishing and presenting the results of these studies in 2013.

Moderna has appointed an accomplished executive leadership team and scientific advisory board, and implemented a comprehensive intellectual property strategy surrounding messenger RNA Therapeutics, filing over 80 patent applications reciting more than 4,000 claims covering novel chemical modifications, RNA engineering, formulation, composition of matter, route of administration and dosing. The company has established preclinical programs in four key therapeutic areas: oncology supportive care, inherited genetic disorders, hemophilia and diabetes.

"Our messenger RNA Therapeutics platform has the potential to revolutionize the treatment of a wide variety of illnesses by opening up a completely new biotherapeutic modality, and by offering a technologically and financially superior path for the discovery, creation and endogenous production of therapeutic proteins," said Stephane Bancel, president and founding CEO of Moderna. "In the last 18 months, we have charged ahead in this space, vigorously pursuing a robust IP portfolio. We have hired an experienced employee base and cultivated leadership and advisory teams who are offering deep insights as we advance key programs into clinical development. We are working with great urgency and great care to translate this groundbreaking science into new treatments for patients."

Moderna’s Leadership Team

Before joining Moderna, Bancel served for five years as the CEO of bioMerieux, a world leader in diagnostics, where he led the company through 10 successful acquisitions in the U.S., Europe and Asia/Pacific; nearly doubled the company's sales growth rate to above nine percent per year; and doubled the company's market capitalization despite the 2008 global financial crisis. Prior to his time at bioMerieux, Stephane was Managing Director of Eli Lilly in Belgium and Executive Director of Global Manufacturing Strategy and Supply Chain at Eli Lilly in Indianapolis, Indiana. He started at Lilly in its UK manufacturing plant outside London. Before joining Lilly he also served as Asia-Pacific Sales and Marketing Director for bioMerieux while based in Tokyo. He holds a Master of Engineering from Ecole Central Paris, a Master of Science in Chemical Engineering from the University of Minnesota and an MBA from Harvard Business School, and is named as an inventor on more than 45 patent filings in the field of messenger RNA (mRNA) technology.

Bancel has assembled a seasoned executive team composed of biotech and pharmaceutical professionals who have played key roles in successfully developing and commercializing biotech products. Susan Whoriskey, Ph.D., senior vice president of technology strategy, served on the founding executive teams of Momenta Pharmaceuticals and Cubist Pharmaceuticals. Chief scientific officer Tony de Fougerolles, Ph.D., brings more than 15 years of research and development experience to this role, having served previously as chief scientific officer at Tolerx, and on the management team at Alnylam Pharmaceuticals as vice president of research for immunology, metabolic and viral disease. Louis O'Dea, MB, BCh, BAO, chief medical officer and head of regulatory affairs, previously led clinical and regulatory activities at Radius Health and was worldwide head of clinical development for reproductive health and metabolism at Serono.

Moderna's leadership is supported by an engaged and experienced board of directors, chaired by Noubar Afeyan, Ph.D., co-founder of Moderna, and also co-founder, managing partner and CEO of Flagship Ventures, a leading early-stage venture capital firm. Dr. Afeyan is joined on the board by: Robert Carpenter, President, Boston Medical Investors, Inc.; Peter Barton Hutt, Senior Counsel, Covington & Burling; Robert Langer, Sc.D., David H. Koch Institute Professor at MIT; John Mendlein, Ph.D., Executive Chairman and CEO of aTyr Pharma; Derrick Rossi, Ph.D., assistant professor in the Stem Cell Regenerative Biology Department at Harvard Medical School and Harvard University; and Timothy Springer, Ph.D., Latham Family Professor at Harvard Medical School and Professor of Medicine at Children's Hospital Boston. Board members Robert Langer and Derrick Rossi are academic co-founders of Moderna, along with scientific advisory board member Kenneth Chien from Harvard University.

"Moderna's promise rivals that of the earliest biotechnology companies over 30 years ago -- adding an entirely new drug category to the pharmaceutical arsenal in the fight against important diseases," said Noubar Afeyan, co-founder and chairman of Moderna. "The executive team, board of directors and scientific advisors all combine the expertise and passion needed to create an unparalleled company that fits the extraordinary scope of this opportunity," he added.

Moderna has assembled a scientific advisory board of world-renowned experts led by Jack Szostak, Ph.D.,2009 winner of the Nobel Prize in medicine, Howard Hughes Medical Institute investigator, professor of genetics atHarvard Medical School and Alex Rich Distinguished Investigator, Department of Molecular Biology at the Center for Computational and Integrative Biology at Massachusetts General Hospital. The scientific advisory board also includes Douglas Cole, MD, General Partner, Flagship Ventures; Kenneth Chien, MD, Ph.D., professor in the Department of Cell and Molecular Biology and Department of Medicine at the Karolinska Institute, and visiting professor in the Department of Stem Cell and Regenerative Biology, Harvard University.; David Liu, Ph.D., Professor of Chemistry and Chemical Biology at Harvard University, a Howard Hughes Medical Institute Investigator and a Senior Associate Member of the Broad Institute of Harvard and MIT; Douglas Melton, Ph.D., Thomas Dudley Cabot Professor in the Natural Sciences at Harvard University and an Investigator of the Howard Hughes Medical Institute; Elizabeth Nabel, MD, President of the Brigham and Women's Hospital (BWH) and Professor of Medicine,Harvard Medical School; and Ulrich H. von Andrian, M.D., Mallinckrodt Professor of Immunopathology at Harvard Medical School. Board members Robert Langer, Derrick Rossi and Timothy Springer also sit on the scientific advisory board.

About Moderna Therapeutics

Moderna is pioneering messenger RNA Therapeutics™ , an entirely new treatment modality that enables the body to produce therapeutic proteins in vivo. Moderna is using this approach to develop first-ever treatments for a wide range of diseases that cannot be addressed today using existing technologies, and to drastically reduce the time and expense associated with creating therapeutic proteins using recombinant technologies. The company has demonstrated proof-of-concept using messenger RNA Therapeutics for a variety of indications and is moving multiple programs into the clinic. Visit www.modernatx.com to learn more.

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