Moderna Provides Business Updates and Reports Second Quarter 2019 Financial Results
Phase 2 Personalized Cancer Vaccine (mRNA-4157) study initiated, with first patient consented
Phase 1 CMV vaccine (mRNA-1647) study fully enrolled
Four new Phase 1 studies have begun, two in immuno-oncology and two in infectious diseases
Ended quarter with
New updates announced today include:
Infectious Diseases
- Enrollment complete for Phase 1 CMV vaccine (mRNA-1647) study
- Second planned interim analysis of Phase 1 hMPV and PIV3 vaccine (mRNA-1653) study showed antibody titers remained above baseline at all dose levels at seven months
- First subject dosed in Phase 1 RSV vaccine (mRNA-1172 or Merck V172) study
- First subject dosed in Phase 1 Zika vaccine (mRNA-1893) study
Immuno-Oncology
- First patient consented in Phase 2 PCV (mRNA-4157) study in patients with resected melanoma
- First patient dosed in Phase 1 KRAS cancer vaccine (mRNA-5671 or Merck V941) study
- First patient dosed in Phase 1 study of mRNA encoding IL12 (MEDI1191) injected intratumorally
Rare Diseases
- Six of eight subjects dosed in the third cohort (0.6 mg/kg) of the Phase 1 antibody against chikungunya virus (mRNA-1944) study; the lipid nanoparticle (LNP) formulation used for mRNA-1944 is also utilized in Moderna’s MMA program
- Three clinical sites actively recruiting for Phase 1/2 MMA (mRNA-3704) study
Research
Vertex Pharmaceuticals extended the companies’ research collaboration in cystic fibrosis (CF)
“Since our last quarterly update, our personalized cancer vaccine program with
Summary of Recent Highlights by Modality
Prophylactic vaccines:
- Respiratory syncytial virus (RSV) vaccine (mRNA-1172 or V172): The first subject has been dosed in the Phase 1 study of mRNA-1172 led by
Merck . mRNA-1172 has shown enhanced potency in preclinical studies compared with the companies’ first RSV candidate (mRNA-1777). - Cytomegalovirus (CMV) vaccine (mRNA-1647): The Phase 1 study of mRNA-1647 has been fully enrolled, at a top dose of 300µg. This randomized, observer-blind and placebo-controlled study is designed to evaluate the safety and immunogenicity of mRNA-1647, a vaccine encoding the gB and pentamer complexes of CMV. CMV is a common human pathogen and a leading cause of birth defects.
- Human metapneumovirus (hMPV) and parainfluenza type 3 (PIV3) vaccine (mRNA-1653): Topline data from a second planned interim analysis of an ongoing Phase 1 randomized, observer-blind, placebo-controlled and dose-ranging study in healthy adults showed that hMPV and PIV3 serum neutralizing antibody titers remained above baseline through seven months and the vaccine was generally well-tolerated. Topline data from the study’s first interim analysis, announced in
February 2019 , showed that mRNA-1653 boosted antibody titers one month after vaccination at all dose levels and was generally well-tolerated. Data from the study will be presented at IDWeek onOctober 2 - 6, 2019 inWashington, D.C. In a recent type C meeting with theU.S. Food and Drug Administration (FDA ), the Company discussed a potential path forward to evaluate protection against both hMPV and PIV3 in a single Phase 3 study. Consistent with this development path,Moderna is planning to initiate a Phase 1b study of mRNA-1653 in seropositive toddler subjects as the next step. - Zika virus vaccine (mRNA-1893): The first subject has been dosed in the Phase 1 study of mRNA-1893. 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 . - Publications of note: In May,
Moderna published its clinical data in Vaccine from two Phase 1 studies showing that mRNA vaccines against H10N8 and H7N9 influenza viruses were well-tolerated and elicited robust immune responses. In July,Moderna published preclinical data in theJournal of Infectious Diseases demonstrating the ability of mRNA vaccines to protect against congenital Zika virus infection in mice.
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 vaccines (PCVs) (mRNA-4157, NCI-4650): The first patient has been consented for the randomized Phase 2 study investigating Merck’s pembrolizumab (KEYTRUDA®) in combination with a 1 mg dose of mRNA-4157, compared to pembrolizumab alone, for the adjuvant treatment of high-risk resectable melanoma. Additionally, the protocol for the ongoing Phase 1 trial has been amended to include a cohort of 17 patients who are refractory to PD-1 inhibitors.
- KRAS vaccine (mRNA-5671 or V941): The first patient was dosed in 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. The study, led by
Merck , will enroll patients with KRAS mutant advanced or metastatic non-small cell lung cancer, colorectal cancer or pancreatic adenocarcinoma, and centrally confirmed Human Leukocyte Antigen (HLA) HLA-A*1101 and/or HLA-C*0802 allele expression. mRNA-5671 is designed to generate and present the four most prevalent KRAS mutations as neoantigens to the immune system. These four mutations comprise an estimated 80-90 percent of KRAS mutations in the study indications. - Presentations of note:
Moderna presented interim data from its ongoing Phase 1 study of PCV candidate mRNA-4157, demonstrating safety, tolerability and immunogenicity of mRNA-4157 alone and in combination with pembrolizumab, at the 2019American Society of Clinical Oncology (ASCO ) Annual Meeting. Additionally, theNational Cancer Institute (NCI) also presented data atASCO from its Phase 1 study of PCV mRNA-4650 as a monotherapy for patients with advanced metastatic cancers. The NCI program uses Moderna’s mRNA technology but uses a different neoantigen selection process and study design than Moderna’s Phase 1 mRNA-4157 study.
Intratumoral Immuno-Oncology: These programs aim to drive anti-cancer T cell responses by injecting mRNA therapies directly into tumors.
- OX40L (mRNA-2416): Dosing is ongoing at the highest levels (8 mg) in the Phase 1/2 open-label, multi-center, dose escalation and efficacy study of intratumoral injections of mRNA-2416 in patients with advanced malignancies. A Phase 2 expansion cohort in patients with advanced ovarian carcinoma is preparing to start enrollment; this will include the combination of intratumoral mRNA-2416 with durvalumab (IMFINZI®).
- OX40L + IL23 + IL36γ (Triplet) (mRNA-2752): The first patient was dosed in the combination arm of the Phase 1 trial of mRNA-2752. This study is evaluating mRNA-2752 as a single agent and in combination with durvalumab in patients with accessible solid tumors and lymphomas. mRNA-2752 is an investigational mRNA immuno-oncology therapy that encodes a novel combination of three immunomodulators.
- IL12 (MEDI1191): The first patient was dosed with MEDI1191 monotherapy in the Phase 1 open-label, multi-center study of intratumoral injections of MEDI1191 alone and in combination with a checkpoint inhibitor in patients with advanced solid tumors, being led by
AstraZeneca . MEDI1191 is an mRNA encoding for IL12, a potent immunomodulatory cytokine.
Systemic Secreted Therapeutics: In this modality, mRNA is delivered systemically to create proteins that are secreted outside the cell with the aim of producing pharmaceutically active proteins with therapeutic effects across the human body.
- Antibody against the chikungunya virus (mRNA-1944): Six of eight subjects have been dosed in the third cohort (0.6 mg/kg) of the Phase 1 antibody against chikungunya virus (mRNA-1944) study. This study is evaluating the safety and tolerability of escalating doses of mRNA-1944 via intravenous infusion in healthy adults. mRNA-1944 is the first monoclonal antibody encoded by mRNA to be dosed in a human and the first development candidate from Moderna’s systemic secreted therapeutics modality to start clinical testing. The lipid nanoparticle (LNP) formulation used for mRNA-1944 is also utilized in Moderna’s MMA program.
- Publication of note: In May,
Moderna published preclinical data in Science Immunology showing mRNA encoding an antibody against the chikungunya virus (mRNA-1944) was well-tolerated, resulted in linear dose-dependent protein expression and provided complete protection in preclinical species.
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): Three clinical trial sites are open and actively recruiting patients for the Phase 1/2 open-label, dose escalation study evaluating mRNA-3704 for the treatment of MMA. The objectives of the study are to evaluate safety and tolerability, assess the pharmacodynamic response and characterize the pharmacokinetic profile of mRNA-3704.
Moderna recently updated the study protocol to widen the age bracket of the first cohort for this trial to allow for the enrollment of pediatric patients (now includes patients 8 years and older, a modification from adolescents aged 12-18.) This is Moderna’s first rare disease program to advance into clinical testing. - MMA and propionic acidemia (PA) Natural History Study (MaP): As of
July 15, 2019 , a total of 71 patients have been enrolled in the study (35 MMA, 36 PA). 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 and is designed to identify and correlate clinical and biomarker endpoints for these disorders. - Publication of note: In July,
Moderna published preclinical data in EBioMedicine, a Lancet publication, showing mRNA therapy demonstrated long-term efficacy and safety, with dose-dependent and reproducible biomarker responses, in mouse models of MMA.
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: https://investors.modernatx.com/.
Research Update
- Vertex CF research collaboration: In
July 2016 ,Moderna and Vertex announced an exclusive research collaboration and licensing agreement aimed at the discovery and development of mRNA therapeutics for the treatment of CF. Based on preclinical work to date, Vertex has extended this collaboration through the first quarter of 2020 with options to extend further based on future progress. Pulmonary mRNA delivery represents a potential new route of administration forModerna .
Annual R&D Day
Moderna will host its annual R&D Day inNew York City onSeptember 12, 2019 .
Investor Call and Webcast Information
Second Quarter 2019 Financial Results
- Cash Position: Cash, cash equivalents and investments as of
June 30, 2019 andDecember 31, 2018 were$1.44 billion and$1.69 billion , respectively. - Net Cash Used in Operating Activities: Net cash used in operating activities was
$256.1 million for the six months endedJune 30, 2019 compared to$159.6 million for the six months endedJune 30, 2018 . Net cash used in operating activities includes$22.0 million and$25.0 million for the six months endedJune 30, 2019 and 2018, respectively, of in-licensing payments toCellscript, LLC and its affiliate, mRNARiboTherapeutics, Inc. , to sublicense certain patent rights. After the first quarter of 2019, we have no further in-licensing payment obligations to Cellscript and its affiliate. - Cash Used for Purchases of Property and Equipment: Cash used for purchases of property and equipment was
$18.2 million for the six months endedJune 30, 2019 compared to$66.0 million for the six months endedJune 30, 2018 . - Revenue: Total revenue was
$13.1 million for the three months endedJune 30, 2019 compared to$28.9 million for the three months endedJune 30, 2018 . Total revenue was$29.1 million for the six months endedJune 30, 2019 compared to$57.9 million for the six months endedJune 30, 2018 . OnJanuary 1, 2019 , we adopted Accounting Standards Codification (ASC) Topic 606, Revenue from Contracts with Customers (ASC 606), using the modified retrospective transition method applied to those contracts which were not completed as ofJanuary 1, 2019 . The total revenue decreases in 2019 were due to decreases in collaboration revenue across all our strategic alliances, particularlyAstraZeneca andMerck , largely driven by our adoption of ASC 606. Total revenue under the previous revenue recognition standard would have been$16.9 million and$55.5 million for the three months and six months endedJune 30, 2019 , respectively. - Research and Development Expenses: Research and development expenses were
$128.5 million for the three months endedJune 30, 2019 compared to$104.5 million for the three months endedJune 30, 2018 . The increase was primarily attributable to an increase in personnel related costs including stock-based compensation, an increase in lab supplies and materials, and an increase in clinical trial and manufacturing costs. Research and development expenses were$259.1 million for the six months endedJune 30, 2019 compared to$194.6 million for the six months endedJune 30, 2018 . The increase was primarily attributable to an increase in personnel related costs including stock-based compensation, an increase in clinical trial and manufacturing costs, an increase in lab supplies and materials, and an increase in consulting and outside services. - General and Administrative Expenses: General and administrative expenses were
$28.5 million for the three months endedJune 30, 2019 compared to$21.4 million for the three months endedJune 30, 2018 . The increase was mainly due to an increase in personnel related costs including stock-based compensation, and an increase in consulting and outside services. General and administrative expenses were$55.8 million for the six months endedJune 30, 2019 compared to$37.7 million for the six months endedJune 30, 2018 . These increases were mainly due to the additional costs of operating as a publicly traded company, including an increase in personnel related costs and stock-based compensation, an increase in consulting and outside services, and an increase in information technology, facility and insurance related costs. - Net Loss: Net loss was
$135.1 million for the three months endedJune 30, 2019 compared to$90.6 million for the three months endedJune 30, 2018 . Net loss was$267.7 million for the six months endedJune 30, 2019 compared to$163.0 million for the six months endedJune 30, 2018 .
2019 Expected Cash Position
Moderna reiterated its expectation for cash, cash equivalents and investments atDecember 31, 2019 to be in the range of$1.15 billion to $1.20 billion .
About
Headquartered in
Forward Looking Statement
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: sharing new clinical data in the near term; the advancement and expansion of Moderna’s development pipeline and the creation of potential new modalities; the anticipated commencement of Moderna’s clinical studies, including the Phase 1b study of mRNA-1653 in seropositive toddler subjects and the Phase 2 expansion cohort of mRNA-2416 in patients with advanced ovarian carcinoma; whether mRNA-5671 will generate and present KRAS neoantigens from the four most prevalent KRAS mutations; and the Company’s expected cash, cash equivalents, and investments at
KEYTRUDA is a registered trademark of
MODERNA, INC. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (Unaudited, in thousands) |
||||||||||||||||
|
|
Three Months Ended June 30, |
|
Six Months Ended June 30, |
||||||||||||
|
|
2019 |
|
2018 |
|
2019 |
|
2018 |
||||||||
Revenue: |
|
|
|
|
|
|
|
|
||||||||
Collaboration revenue |
|
$ |
10,030 |
|
|
$ |
25,831 |
|
|
$ |
24,145 |
|
|
$ |
53,291 |
|
Grant revenue |
|
3,053 |
|
|
3,020 |
|
|
4,963 |
|
|
4,599 |
|
||||
Total revenue |
|
13,083 |
|
|
28,851 |
|
|
29,108 |
|
|
57,890 |
|
||||
Operating expenses: |
|
|
|
|
|
|
|
|
||||||||
Research and development |
|
128,496 |
|
|
104,479 |
|
|
259,071 |
|
|
194,603 |
|
||||
General and administrative |
|
28,523 |
|
|
21,387 |
|
|
55,806 |
|
|
37,704 |
|
||||
Total operating expenses |
|
157,019 |
|
|
125,866 |
|
|
314,877 |
|
|
232,307 |
|
||||
Loss from operations |
|
(143,936 |
) |
|
(97,015 |
) |
|
(285,769 |
) |
|
(174,417 |
) |
||||
Interest income |
|
10,322 |
|
|
6,401 |
|
|
21,294 |
|
|
11,610 |
|
||||
Other (expense) income, net |
|
(1,764 |
) |
|
171 |
|
|
(3,584 |
) |
|
(12 |
) |
||||
Loss before income taxes |
|
(135,378 |
) |
|
(90,443 |
) |
|
(268,059 |
) |
|
(162,819 |
) |
||||
(Benefit from) provision for income taxes |
|
(324 |
) |
|
158 |
|
|
(348 |
) |
|
158 |
|
||||
Net loss |
|
$ |
(135,054 |
) |
|
$ |
(90,601 |
) |
|
$ |
(267,711 |
) |
|
$ |
(162,977 |
) |
MODERNA, INC. CONDENSED CONSOLIDATED BALANCE SHEETS AND STATEMENTS OF CASH FLOWS DATA (Unaudited, in thousands) |
|||||||
|
June 30, |
|
December 31, |
||||
|
2019 |
|
2018 |
||||
Cash, cash equivalents and investments |
$ |
1,435,378 |
|
|
$ |
1,694,417 |
|
Total assets |
1,685,279 |
|
|
1,962,149 |
|
||
Total liabilities |
346,725 |
|
|
431,908 |
|
||
Total stockholders’ equity |
1,338,554 |
|
|
1,530,241 |
|
||
|
|||||||
|
Six Months Ended June 30, |
||||||
|
2019 |
|
2018 |
||||
Net cash used in operating activities |
$ |
(256,147 |
) |
|
$ |
(159,634 |
) |
Cash used for purchases of property and equipment |
(18,181 |
) |
|
(65,989 |
) |
View source version on businesswire.com: https://www.businesswire.com/news/home/20190807005154/en/
Source:
Media:
Dan Budwick
1AB
973-271-6085
dan@1abmedia.com
Investors:
Lavina Talukdar
Head of Investor Relations
617-209-5834
lavina.talukdar@modernatx.com