Moderna Reports 2019 Fourth Quarter and Full Year Financial Results and Highlights Advancements in Core Modalities
Two of three dose cohorts in Phase 2 CMV vaccine (mRNA-1647) dose-confirmation study completed enrollment; third and final cohort rapidly enrolling
Up to
New updates and recent progress include:
Infectious Diseases
- Two of three dose cohorts in Phase 2 CMV vaccine (mRNA-1647) dose-confirmation study completed enrollment, and third and final cohort is more than 85% enrolled; first Phase 2 interim analysis expected in the third quarter of 2020;
Moderna actively preparing for pivotal Phase 3 study, expected to begin in 2021 - First clinical-grade batch of novel coronavirus vaccine (mRNA-1273) shipped and received by
NIH for use in Phase 1 study in theU.S. ; delivered from Company’s cGMP facility in 42 days from sequence selection
Rare Diseases
- Dosing complete for 0.6 mg/kg dose cohort with steroid premedication in the Phase 1 study of antibody against chikungunya virus (mRNA-1944); first participant dosed in additional cohort with two doses of 0.3 mg/kg (without steroid premedication) given one week apart
- First patient enrolled in Phase 1/2 MMA (mRNA-3704) study; actively recruiting patients at
U.S. sites following a protocol amendment expanding the first cohort eligibility criteria to patients 8 years and older
Corporate Updates
- On
February 25, 2020 , the Company received notice that the underwriters had exercised their option to purchase an additional$75 million in shares of common stock in connection with the recently completed public offering. The closing of this additional sale is expected to occur on or aboutFebruary 26, 2020 , subject to the satisfaction of customary closing conditions.
“The Moderna team continues to execute our strategy, including our CMV Phase 2 study enrolling ahead of plan, shipping the coronavirus Phase 1 clinical materials to
Summary of Program Highlights by Modality
Core Modalities
Prophylactic Vaccines:
Infections transmitted from mother to baby
- Cytomegalovirus (CMV) vaccine (mRNA-1647): The first (50 µg) and second (100 µg) cohorts of the Phase 2 dose-confirmation study of mRNA-1647 have completed enrollment, and the third and final cohort (150 µg) is more than 85% enrolled. In February,
Moderna announced that the first interim analysis of this Phase 2 study is expected in the third quarter of 2020. In January,Moderna announced positive seven-month interim data after the third and final vaccination from the Phase 1 study of mRNA-1647. Manufacturing and planning are underway for the pivotal Phase 3 study, which is designed to evaluate the efficacy of mRNA-1647 against primary CMV infection in a population that includes women of childbearing age and is expected to start in 2021.Moderna owns worldwide commercial rights for mRNA-1647. - Zika virus (mRNA-1893): The 10 µg, 30 µg and 100 µg cohorts in the Phase 1 study of mRNA-1893 have completed enrollment. This development candidate is being developed in collaboration with the
U.S. Biomedical Advanced Research and Development Authority (BARDA) within theOffice of the Assistant Secretary for Preparedness and Response at theU.S. Department of Health and Human Services .Moderna owns worldwide commercial rights to mRNA-1893.
Vaccines against respiratory infections
- Human metapneumovirus (hMPV) and parainfluenza type 3 (PIV3) vaccine (mRNA-1653): The Phase 1b age de-escalation study of mRNA-1653 is ongoing.
Moderna previously announced positive data from the second pre-planned interim analysis of the Phase 1 study of mRNA-1653.Moderna owns worldwide commercial rights to mRNA-1653. - Pediatric respiratory syncytial virus (RSV) vaccine (mRNA-1345): mRNA-1345 is a vaccine against respiratory syncytial virus (RSV) in young children encoding for a prefusion F glycoprotein, which elicits a superior neutralizing antibody response compared to the postfusion state. The Company intends to combine mRNA-1345 with mRNA-1653, its vaccine against hMPV and PIV3, to create a combination vaccine against RSV, hMPV and PIV3. There is no approved vaccine for RSV.
Moderna owns worldwide commercial rights to the combined mRNA-1345/mRNA-1653 vaccine. - Respiratory syncytial virus (RSV) vaccine (mRNA-1172 or V172): The Phase 1 study of mRNA-1172 led by Merck is ongoing.
Moderna has licensed worldwide commercial rights to mRNA-1172 to Merck. - Novel coronavirus (SARS CoV-2) vaccine (mRNA-1273): The first clinical-grade batch of mRNA-1273 has been shipped from
Moderna facility to theNIH for use in a Phase 1 study in theU.S. mRNA-1273 was delivered from the Company’s cGMP facility in 42 days from sequence selection.NIH plans to conduct the Phase 1 clinical trial in theU.S. There is no approved vaccine for novel coronavirus. - Influenza H7N9 vaccine (mRNA-1851): Discussions regarding funding the Company’s influenza H7N9 vaccine program through approval are ongoing.
Vaccines against highly prevalent viral infections
- Epstein-Barr virus (EBV) vaccine (mRNA-1189): mRNA-1189 is a vaccine against Epstein-Barr virus (EBV) containing five mRNAs that encode viral proteins (gp350, gB, gp42, gH and gL) in EBV. Similar to Moderna’s CMV vaccine (mRNA-1647), the viral proteins in mRNA-1189 are expressed in their native membrane-bound form for recognition by the immune system. There is no approved vaccine for EBV.
Moderna owns worldwide commercial rights to mRNA-1189.
Systemic Secreted & Cell Surface Therapeutics: In this modality, mRNA is delivered systemically to create proteins that are either secreted or expressed on the cell surface.
- Antibody against the chikungunya virus (mRNA-1944): Dosing is complete for the 0.6 mg/kg cohort with steroid premedication in the Phase 1 study evaluating escalating doses of mRNA-1944 administered via intravenous infusion in healthy adults. The first participant has been dosed in the additional cohort with two doses of 0.3 mg/kg (without steroid premedication) given one week apart. In
September 2019 ,Moderna announced positive interim data from the first analysis of safety and activity in the Phase 1 study.Moderna owns worldwide commercial rights to mRNA-1944. - IL-2 (mRNA-6231): mRNA-6231 is an mRNA encoding for a long-acting tolerizing IL-2. This new autoimmune development candidate is designed to preferentially activate and expand the regulatory T cell population. The Company plans to conduct a Phase 1 study of mRNA-6231 in healthy adult volunteers. mRNA-6231 uses the same LNP formulation as mRNA-1944. The Phase 1 study of mRNA-6231 will be the first clinical demonstration of subcutaneous administration of this delivery technology.
Moderna owns worldwide commercial rights to mRNA-6231. - PD-L1 (mRNA-6981): mRNA-6981 is an mRNA encoding for PD-L1. This new autoimmune development candidate is designed to augment cell surface expression of PD-L1 on myeloid cells to provide co-inhibitory signals to self-reactive lymphocytes. As an initial step to addressing a range of autoimmune indications, the Company intends to pursue proof-of-concept in a Phase 1 study of mRNA-6981 in type 1 autoimmune hepatitis (AIH), a condition that involves liver inflammation and can lead to cirrhosis and liver failure. mRNA-6981 uses the same LNP formulation as mRNA-1944.
Moderna owns worldwide commercial rights to mRNA-6981. - Relaxin (AZD7970): Partnered with AstraZeneca, AZD7970 is in preclinical development for the treatment of heart failure. Under the terms of the collaboration, AstraZeneca would sponsor the Phase 1 trial to assess safety, tolerability and duration of systemic exposure to the Relaxin protein.
Moderna shares worldwide commercial rights to AZD7970 with AstraZeneca. - 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. mRNA-3630 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. mRNA-3630 is in preclinical development.
Moderna owns worldwide commercial rights to mRNA-3630.
Exploratory Modalities
Cancer Vaccines: These programs focus on stimulating a patient’s immune system with antigens derived from tumor-specific mutations to enable the immune system to elicit a more effective anti-tumor response.
- Personalized cancer vaccine (PCV) (mRNA-4157): The randomized Phase 2 study investigating a 1 mg dose of mRNA-4157 in combination with Merck’s pembrolizumab (KEYTRUDA®), compared to pembrolizumab alone, for the adjuvant treatment of high-risk resected melanoma is ongoing. The Phase 1 study is ongoing.
Moderna shares worldwide commercial rights to mRNA-4157 with Merck. - KRAS vaccine (mRNA-5671 or V941): The Phase 1 open-label, multi-center study to evaluate the safety and tolerability of mRNA-5671 both as a monotherapy and in combination with pembrolizumab, led by Merck, is ongoing.
Moderna shares worldwide commercial rights to mRNA-5671 with Merck.
Intratumoral Immuno-Oncology: These programs aim to drive anti-cancer T cell responses by injecting mRNA therapies directly into tumors.
- OX40L (mRNA-2416): The first patient has been dosed in the Phase 1 dose escalation cohort of mRNA-2416 in combination with durvalumab (IMFINZI®). The Company has removed the top dose of 8 mg in this cohort based on limitations due to the size of ovarian lesions.
Moderna owns worldwide commercial rights to mRNA-2416. - OX40L/IL-23/IL-36γ (Triplet) (mRNA-2752): The Phase 1 trial evaluating mRNA-2752 as a single agent and in combination with durvalumab in patients with advanced solid tumor malignancies and lymphoma is ongoing. mRNA-2752 is an investigational mRNA immuno-oncology therapy that encodes a novel combination of three immunomodulators.
Moderna owns worldwide commercial rights to mRNA-2752. - IL-12 (MEDI1191): The Phase 1 open-label, multi-center study of intratumoral injections of MEDI1191 alone and in combination with durvalumab in patients with advanced solid tumors, led by AstraZeneca, is ongoing. MEDI1191 is an mRNA encoding for IL-12, a potent immunomodulatory cytokine.
Moderna shares worldwide commercial rights to MEDI1191 with AstraZeneca.
Localized Regenerative Therapeutics: Localized production of proteins has the potential to be used as a regenerative medicine for damaged tissues.
- VEGF-A (AZD8601): The Phase 2a study of AZD8601 for VEGF-A for ischemic heart disease in patients undergoing coronary artery bypass grafting (CABG) surgery with moderately impaired systolic function, led by AstraZeneca, is ongoing.
Moderna has licensed worldwide commercial rights to AZD8601 to AstraZeneca.
Systemic Intracellular Therapeutics: These programs aim to deliver mRNA into cells within target organs as a therapeutic approach for diseases caused by a missing or defective protein.
- Methylmalonic acidemia (MMA) (mRNA-3704): The first patient has been enrolled in the Phase 1/2 open-label, dose escalation study evaluating the safety and tolerability of escalating doses of mRNA-3704 administered via intravenous infusion in patients with isolated methylmalonic acidemia (MMA) due to MUT deficiency. The patient has entered an observational period prior to treatment, which evaluates the patient’s baseline disease prior to starting the treatment period. The Company is planning to initiate several sites outside the
U.S. and has thus far received Medicines and Healthcare products Regulatory Agency (MHRA) approval in theU.K. The objectives of this study are to evaluate safety and tolerability, assess the pharmacodynamic response and characterize the pharmacokinetic profile of mRNA-3704. This is Moderna’s first rare disease program to begin clinical trial enrollment. mRNA-3704 uses the same LNP formulation as mRNA-1944.Moderna owns worldwide commercial rights to mRNA-3704. - Propionic acidemia (PA) (mRNA-3927): Study start-up in the
U.S. is ongoing for the open-label, multi-center Phase 1/2 study of multiple ascending doses of mRNA-3927 in primarily pediatric patients with PA. The objectives of this study are to evaluate the safety and tolerability of mRNA-3927 administered via IV infusion, assess the pharmacodynamic response as assessed by changes in plasma biomarkers and characterize the pharmacokinetic profile of mRNA-3927. mRNA-3927 uses the same LNP formulation as mRNA-1944.Moderna owns worldwide commercial rights to mRNA-3927. - MMA and PA Natural History Study (MaP): This is a global, multi-center, non-interventional study for patients with confirmed diagnosis of MMA due to MUT deficiency or PA and is designed to identify and correlate clinical and biomarker endpoints for these disorders. Enrollment in the study has been completed.
- Phenylketonuria (PKU) (mRNA-3283): Individuals with PKU have a deficiency in phenylalanine hydroxylase (PAH) resulting in a reduced or complete inability to metabolize the essential amino acid phenylalanine into tyrosine. mRNA-3283 encodes human PAH to restore the deficient or defective intracellular enzyme activity in patients with PKU. mRNA-3283 is in preclinical development.
Moderna owns worldwide commercial rights to mRNA-3283. - Glycogen storage disease type 1a (GSD1a) (mRNA-3745): Individuals with GSD1a have a deficiency in glucose-6-phosphatase resulting in pathological blood glucose imbalance. mRNA-3745 is an IV-administered mRNA encoding human G6Pase enzyme, designed to restore deficient or defective intracellular enzyme activity in patients with GSD1a. mRNA-3745 is in preclinical development.
Moderna owns worldwide commercial rights to mRNA-3745.
Information about each development candidate in Moderna’s pipeline, including those discussed in this press release, can be found on the investor relations page of its website: investors.modernatx.com.
Corporate Updates
- Public offering, strategic alliances and available grant funding: After the closing of the underwriters’ option to purchase additional shares, the Company will have raised
$550 million in net proceeds from the recent financing announced in earlyFebruary 2020 .
The Company has established a wide range of strategic alliances with leading biopharmaceutical companies, as well as grants from government-sponsored and private organizations focused on global health initiatives. As of
With the offering proceeds, access to additional grant funding, and cash and investments of
- Shareholder letter:
Moderna CEOStéphane Bancel published a letter to shareholders onJanuary 6, 2020 .
Key 2020 Investor and Analyst Event Dates
- Manufacturing & Digital Day –
March 4 at Moderna’sNorwood, MA facility - Vaccines Day –
April 14 inNew York City - Science Day –
June 2 inNew York City - R&D Day –
September 17 inNew York City
Fourth Quarter and Full Year 2019 Financial Results (Unaudited)
- Cash Position: Cash, cash equivalents and investments as of
December 31, 2019 and 2018 were$1.26 billion and$1.69 billion , respectively. Net Cash Used in Operating Activities: Net cash used in operating activities was$459.0 million for the year endedDecember 31, 2019 compared to$330.9 million for the year endedDecember 31, 2018 .- Cash Used for Purchases of Property and Equipment: Cash used for purchases of property and equipment decreased
$74.2 million , or 70.2%, to$31.6 million for the year endedDecember 31, 2019 from$105.8 million for the year endedDecember 31, 2018 . Of these amounts, cash disbursements specifically related toModerna Technology Center (MTC) manufacturing facility inNorwood, MA were$14.6 million and$94.5 million for the years endedDecember 31, 2019 and 2018, respectively. Our MTC manufacturing facility opened inJuly 2018 . - Revenue: Total revenue was
$14.1 million for the fourth quarter of 2019 compared to$35.4 million for the fourth quarter of 2018. Total revenue was$60.2 million for the year endedDecember 31, 2019 compared to$135.1 million for the year endedDecember 31, 2018 . The decreases in both periods were mainly due to lower collaboration revenue across all our strategic alliances, particularly AstraZeneca and Merck, driven by our adoption of ASC 606 and the completion of the initial four-year research period under the 2016 Merck Agreement. - Research and Development Expenses: Research and development expenses were
$118.8 million for the fourth quarter of 2019 compared to$150.4 million for the fourth quarter of 2018. Research and development expenses were$496.3 million for the year endedDecember 31, 2019 compared to$454.1 million for the year endedDecember 31, 2018 . The decrease in the fourth quarter was primarily due to a decrease in our in-licensing payments toCellscript, LLC and its affiliate, and a reduction of our lab supplies and materials costs. The increase for the year endedDecember 31, 2019 was primarily due to an increase in personnel related cost, including stock-based compensation, and an increase in clinical trial and manufacturing costs, mainly driven by an increase in the number of employees and costs supporting research and development programs. - General and Administrative Expenses: General and administrative expenses were
$25.9 million for the fourth quarter of 2019 compared to$38.0 million for the fourth quarter of 2018. General and administrative expenses were$109.6 million for the year endedDecember 31, 2019 compared to$94.3 million for the year endedDecember 31, 2018 . The decrease in the fourth quarter was primarily due to a decrease in stock-based compensation, mainly attributable to certain performance-based equity awards with vesting or commencement contingent on our initial public offering in 2018. The increase for the year endedDecember 31, 2019 was primarily due to an increase in insurance, consulting and outside services, and facility related costs, primarily driven by an increase in the number of employees and costs in support of being a public company. - Net Loss: Net loss was
$124.2 million for the fourth quarter of 2019 compared to$141.4 million for the fourth quarter of 2018. Net loss was$514.0 million for the year endedDecember 31, 2019 compared to$384.7 million for the year endedDecember 31, 2018 .
Reiterating Financial Guidance
Moderna expects net cash used in operating activities and for purchases of property and equipment in 2020 to be similar to 2019, between$490 million and$510 million .
Investor Call and Webcast Information
About
Headquartered in
1
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, but not limited to, statements concerning: the closing of the sale of additional shares to the underwriters in connection with the Company’s recent public offering, and the net proceeds of the offering following such closing; initiating clinical trial sites outside of the
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CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS |
|||||||||||||||
(Unaudited, in thousands) |
|||||||||||||||
|
Three Months Ended |
|
Years Ended |
||||||||||||
|
2019 |
|
2018 |
|
2019 |
|
2018 |
||||||||
Revenue: |
|
|
|
|
|
|
|
||||||||
Collaboration revenue |
$ |
10,553 |
|
|
$ |
32,816 |
|
|
$ |
48,036 |
|
|
$ |
122,512 |
|
Grant revenue |
3,502 |
|
|
2,605 |
|
|
12,173 |
|
|
12,556 |
|
||||
Total revenue |
14,055 |
|
|
35,421 |
|
|
60,209 |
|
|
135,068 |
|
||||
Operating expenses: |
|
|
|
|
|
|
|
||||||||
Research and development |
118,754 |
|
|
150,429 |
|
|
496,309 |
|
|
454,082 |
|
||||
General and administrative |
25,857 |
|
|
38,023 |
|
|
109,620 |
|
|
94,252 |
|
||||
Total operating expenses |
144,611 |
|
|
188,452 |
|
|
605,929 |
|
|
548,334 |
|
||||
Loss from operations |
(130,556 |
) |
|
(153,031 |
) |
|
(545,720 |
) |
|
(413,266 |
) |
||||
Interest income |
7,984 |
|
|
8,894 |
|
|
38,530 |
|
|
27,023 |
|
||||
Other (expense) income, net |
(1,837 |
) |
|
2,879 |
|
|
(7,526 |
) |
|
1,835 |
|
||||
Loss before (benefit from) provision for income taxes |
(124,409 |
) |
|
(141,258 |
) |
|
(514,716 |
) |
|
(384,408 |
) |
||||
(Benefit from) provision for income taxes |
(169 |
) |
|
168 |
|
|
(695 |
) |
|
326 |
|
||||
Net loss |
$ |
(124,240 |
) |
|
$ |
(141,426 |
) |
|
$ |
(514,021 |
) |
|
$ |
(384,734 |
) |
Net loss attributable to common stockholders |
$ |
(124,240 |
) |
|
$ |
(144,099 |
) |
|
$ |
(514,021 |
) |
|
$ |
(401,857 |
) |
Net loss per share attributable to common stockholders, |
$ |
(0.37 |
) |
|
$ |
(1.14 |
) |
|
$ |
(1.55 |
) |
|
$ |
(4.95 |
) |
Weighted average common shares used in net loss per |
334,392,128 |
|
126,298,266 |
|
330,802,136 |
|
81,114,183 |
|
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CONDENSED CONSOLIDATED BALANCE SHEETS AND STATEMENTS OF CASH FLOWS DATA |
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(Unaudited, in thousands) |
|||||||||
|
|
||||||||
|
2019 |
|
2018 |
||||||
Cash, cash equivalents and investments |
$ |
|
1,262,987 |
|
|
$ |
|
1,694,417 |
|
Total assets |
|
1,589,422 |
|
|
|
1,962,149 |
|
||
Total liabilities |
|
414,612 |
|
|
|
431,908 |
|
||
Total stockholders’ equity |
|
1,174,810 |
|
|
|
1,530,241 |
|
||
|
|
|
|
||||||
|
Years Ended |
||||||||
|
2019 |
|
2018 |
||||||
Net cash used in operating activities |
$ |
|
(458,968 |
) |
|
$ |
|
(330,865 |
) |
Cash used for purchases of property and equipment (1) |
|
(31,554 |
) |
|
|
(105,766 |
) |
(1) Includes
View source version on businesswire.com: https://www.businesswire.com/news/home/20200226005280/en/
Media:
Senior Manager, Corporate Communications
203-470-5620
Colleen.Hussey@modernatx.com
Investors:
Head of Investor Relations
617-209-5834
Lavina.Talukdar@modernatx.com
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