New development candidate for combination respiratory COVID-19 booster + seasonal flu booster vaccine

New pediatric combination development candidate for RSV + hMPV vaccine

Interim Phase 1 data from RSV vaccine candidate in older adults significantly boosted neutralizing antibody titers above baseline

New infectious disease therapeutic vaccine candidate, to complement Epstein-Barr virus prophylactic vaccine

Phase 2 randomized, placebo-controlled study of personalized cancer vaccine fully enrolled; primary endpoint is recurrence free survival at 12 months

Company continues to scale with 37 programs in development, including 22 in ongoing clinical studies

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Moderna, Inc. (Nasdaq: MRNA), a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, today announced significant advances across its portfolio of mRNA pipeline programs being presented at the Company’s fifth annual R&D Day today.

“I am proud of the progress that the Moderna team has made in advancing our best-in-class mRNA pipeline while addressing the global COVID-19 pandemic. We believe our mRNA platform can solve the world’s greatest health challenges, from diseases impacting millions, to ultra-rare diseases impacting dozens, to medicines personalized down to the individual level,” said Stéphane Bancel, Chief Executive Officer of Moderna. “Today we are announcing the first step in our novel respiratory vaccine program with the development of a single dose vaccine that combines a booster against COVID-19 and a booster against flu. We are making progress on enrolling patients in our rare disease programs, and we are fully enrolled in our personalized cancer vaccine trial. We believe this is just the beginning of a new age of information-based medicines.”

Updates and recent progress include:

COVID-19 Vaccine Development

• Moderna completed the rolling submission process for a Biologics License Application (BLA) for its COVID-19 vaccine (mRNA-1273) in the U.S.
• Robust antibody responses have been observed in a Phase 2 study of a third dose/booster of mRNA-1273

New Development Candidates

• Combination vaccine candidate (mRNA-1073) that combines Moderna’s COVID-19 vaccine and flu vaccine candidate
• Pediatric combination vaccine candidate (mRNA-1365) that combines Moderna’s RSV vaccine candidate with its hMPV vaccine candidate
• EBV therapeutic vaccine candidate (mRNA-1195)
• Ultra-rare disease, Crigler-Najjar Syndrome Type 1 (mRNA-3351); Moderna to provide investigational mRNA CN-1 therapy to the nonprofit Institute for Life Changing Medicines (ILCM) free of charge

Infectious Diseases

• Phase 1 study of quadrivalent seasonal flu vaccine candidate (mRNA-1010) fully enrolled
• Positive interim data from Phase 1 study of RSV vaccine candidate (mRNA-1345) from older adult cohort (ages 65-79 years)
• Preparing for a global Phase 2/3 study with approximately 34,000 participants of RSV vaccine candidate (mRNA-1345); expected to begin by the end of 2021

Oncology

• Phase 2 randomized, placebo-controlled study of personalized cancer vaccine (PCV) (mRNA-4157)in combination with Merck’s pembrolizumab (KEYTRUDA®), compared to pembrolizumab alone, for the adjuvant treatment of high-risk resected melanoma is fully enrolled

Cardiovascular

• Phase 2a study of AZD8601 VEGF-A, being developed for patients with ischemic heart disease undergoing coronary artery bypass grafting surgery with moderately impaired systolic function, led by AstraZeneca, has completed recruitment after enrollment of the low dose cohort

Rare Diseases

• Enrollment of the first cohort in Propionic Acidemia (mRNA-3927) Phase 1/2 Paramount study is complete
• Investigational New Drug application (IND) open and Orphan Drug Designation granted by U.S. FDA for GSD1a program (mRNA-3745)

Corporate Update

• Moderna Genomics (MGX) will leverage Moderna’s current mRNA and lipid nanoparticle (LNP) platform and will pursue novel technology within nucleic acids

Moderna continues to scale, now with 37 programs in development across 34 development candidates1, including 22 in ongoing clinical studies. The Company’s updated pipeline can be found at www.modernatx.com/pipeline. Moderna and collaborators have published more than 80 peer-reviewed manuscripts.

Select Program Highlights

Core Modalities

Prophylactic Vaccines: Moderna is developing vaccines against viral diseases where there is unmet medical need – including vaccines against acute respiratory infections, vaccines against persistent infections, as well as vaccines against threats to global public health.

Vaccines against acute respiratory infections

COVID-19 vaccine development

• Moderna COVID-19 Vaccine (mRNA-1273): The World Health Organization (WHO) and health agencies in more than 50 countries have granted emergency use authorization or emergency use listingfor the use of the Moderna COVID-19 vaccine in adults. In addition, Moderna has received authorization for use of its COVID-19 vaccine in adolescents ages 12 and up in the European Union and other jurisdictions.On August 25, 2021, Modernacompleted the rolling submission process for a Biologics License Application (BLA) for the vaccine in the U.S.Moderna is working with additional health agencies on the authorization and/or approval of its vaccine in additional jurisdictions2. Moderna retains worldwide rights to develop and commercialize the Moderna COVID-19 Vaccine.
  • Final Analysis of Phase 3 COVE Study: In the final analysis of the Phase 3 COVE study, the Moderna COVID-19 Vaccine showed 93% efficacy (95% CI), with the efficacy remaining durable through six months after administration of the second dose. In this analysis, the Moderna COVID-19 vaccine showed 98.2% efficacy against severe COVID-19 disease and 100% efficacy against death caused by COVID-19. Sub-group analyses were consistent across different populations and the safety profile based on extended safety follow-up was consistent with the Phase 3 COVE study primary results.
  • Addressing Variants of Concern:Moderna has four development candidates against SARS-CoV-2 variants of concern, including three which have been administered in a Phase 2/3 clinical trial. Initiation of a clinical cohort with the fourth candidate is planned in the coming weeks.The Company’s strategy is to develop booster vaccines against current variants of concern and against potential future variants of concern.
    • mRNA-1273.351: Variant-specific candidate against the Beta variant
    • mRNA-1273.617: Variant-specific candidate against the Delta variant
    • mRNA-1273.211: Multivalent candidate combining the Beta-specific variant and mRNA-1273
    • mRNA-1273.213: Multivalent candidate combining the Beta-specific and Delta-specific candidates
  • Booster (Third) Dose:Moderna has submitted for a booster (third) dose of mRNA-1273 at the 50 µg dose level for the following: Emergency Use Authorization (EUA) with the U.S. FDA, Conditional Marketing Approval (CMA) with the European Medicines Agency (EMA) and to additional regulatory agencies. In the amended Phase 2 study, a booster dose of mRNA-1273 at the 50 µg dose level boosted neutralizing titers significantly above the Phase 3 benchmark. After a third dose, a similar level of neutralizing titers was achieved across age groups, notably in older adults (ages 65 and above). The safety profile following dose 3 was similar to that observed previously for dose 2 of mRNA-1273. An additional analysis showed that a booster dose of mRNA-1273 at the 50 µg dose level induced robust antibody responses and significantly increased geometric mean titers (GMT) for all variants of concern including importantly, Delta (B.1.617.2) by 42.3-fold.
• Additional Clinical Studies of mRNA-1273
  • Phase 2/3 “TeenCOVE” study of mRNA-1273 in adolescents: The Phase 2/3 study of mRNA-1273 in adolescents ages 12-17 years showed that no cases of COVID-19 were observed after two doses of vaccine using the primary case definition, consistent with a vaccine efficacy of 100%. Generally well tolerated, the majority of adverse events were mild or moderate in severity. The most common solicited local adverse event was injection site pain. The most common solicited systemic adverse events after the second dose of mRNA-1273 were headache, fatigue, myalgia and chills. The Conditional Marketing Authorization (CMA) for Spikevax3 in the European Union (EU) has been expanded to include adolescents 12 years of age and older. In addition, the Japanese Ministry of Health, Labor and Welfare, Health Canada, as well as other regulatory agencies around the world have also authorized Moderna's COVID-19 vaccine for ages 12 to 17. Moderna has filed for an EUA for adolescents with the U.S. FDA.
  • Phase 2 “KidCOVE” study of mRNA-1273 in young children: The Phase 2 study of mRNA-1273 in pediatric population ages 6 months to 11 years is ongoing. The Company selected the 50 µg dose for expanded enrollment in the 6 to