Moderna Eyes Cancer Vaccine Filing as It Expands Beyond COVID

Key Points

• Moderna plans to pursue regulatory filing for intismeran, its personalized mRNA cancer vaccine developed with Merck, after a Phase 3 melanoma trial met its primary and secondary endpoints. The companies are first focusing on a biologics license application.
• Intismeran is being studied across additional cancers, including lung, renal, bladder, pancreatic and gastric cancers, while Moderna works with Merck to address hospital logistics for individualized vaccine manufacturing and delivery.
• Moderna is expanding beyond COVID-19 with programs in off-the-shelf cancer vaccines, autoimmune disease and rare diseases, including a propionic acidemia treatment expected to produce pivotal data by year-end.

Moderna NASDAQ: MRNA executives emphasized the company’s evolution beyond COVID-19 vaccines during a discussion with Bernstein, highlighting its mRNA platform, personalized cancer vaccine program intismeran, rare-disease pipeline and plans to expand therapeutics development.

Chief Executive Officer Stéphane Bancel said Moderna has been an mRNA platform company since its founding and described the COVID-19 vaccine as an unplanned but necessary response to the pandemic rather than the company’s long-term strategy. He said Moderna currently has a vaccine business with five approved products and two product launches planned this year, alongside oncology, rare disease and autoimmune programs.

Bancel said the company believes it is now the only remaining broad mRNA platform company following changes among competitors, including CureVac’s failed late-stage COVID vaccine, Sanofi’s decision to stop mRNA vaccine programs following its acquisition of Translate Bio, and BioNTech’s announced organizational changes.

Intismeran Moves Toward Regulatory Filing

Much of the discussion focused on intismeran, Moderna’s individualized mRNA cancer vaccine being developed with Merck. Chief Development Officer David Berman said the Phase 3 melanoma study met both its primary endpoint and a secondary endpoint of distant metastasis-free survival at the first interim analysis.

Berman characterized the study as a rigorously controlled, blinded trial involving approximately 1,100 patients that compared intismeran plus Keytruda with Keytruda alone. He said the company believes the results provide a strong case for regulatory approval, though Moderna plans to focus first on filing its biologics license application.

The executives said the program could extend into other cancer settings. Berman cited ongoing studies in lung cancer, renal cell carcinoma, bladder cancer, pancreatic cancer and gastric cancer. Moderna has three Phase 3 lung cancer trials, including an adjuvant trial where it expects randomization to be completed later this year, according to Berman.

Berman said Moderna is also studying intismeran in earlier stages of disease, including a Phase 3 trial in stage 1 lung cancer and a randomized Phase 2 trial in non-muscle-invasive bladder cancer. He said the company’s view is that the treatment may be active without a PD-1 inhibitor, potentially creating opportunities in settings where checkpoint inhibitors are not commonly used.

Manufacturing and Commercial Preparation

Bancel said Moderna owns the proprietary work involved in selecting the 34 mutations included in each individualized cancer vaccine. External partners provide DNA sequencing data from healthy and cancer cells, while Moderna conducts the downstream analysis and selection process, he said.

He described intismeran manufacturing as a synthetic, cell-free liquid process that can be scaled through smaller equipment footprints and faster cycle times. Bancel said the principal bottleneck may be hospital operations rather than manufacturing, particularly the ability to rapidly obtain biopsies and related patient information.

Moderna and Merck are establishing teams to support hospitals and standardize those processes, according to Bancel. He said the companies have built a digital platform to track operational steps while maintaining patient privacy.

Under the collaboration, Moderna and Merck share costs equally, including commercial, clinical, manufacturing and capital expenditures, with remaining profit split evenly between the companies, Bancel said. He declined to discuss potential pricing before additional clinical data are presented.

Broader Oncology and Rare-Disease Programs

Berman also outlined Moderna’s efforts to develop off-the-shelf cancer vaccines. The company has a Phase 1/2 program in the United Kingdom for people with Lynch syndrome, an inherited condition associated with increased cancer risk. The long-term goal is cancer prevention, he said.

Other early oncology efforts include shared-antigen cancer vaccines and a Phase 1 multiple myeloma program that uses three T-cell engagers in a single treatment. Berman said the company expects to share data from the multiple myeloma study later this year. Moderna is also advancing an in vivo CAR-T program and other autoimmune disease programs.

In rare disease, Bancel said the company expects pivotal data for propionic acidemia by year-end. He described the program as the first of what Moderna expects could become a broader liver-focused rare-disease franchise, using mRNA to direct the liver to produce missing or deficient proteins.

Bancel said Moderna has published work involving more than 12 rare genetic liver-disease models and intends to advance additional programs using the same lipid delivery approach and manufacturing infrastructure as its propionic acidemia candidate.

Long-Term Platform Strategy

Bancel said Moderna recently raised $2.6 billion through convertible notes and remains focused on long-term platform development rather than near-term earnings. He said the company’s sales declined sharply after the pandemic and that it restructured while continuing to invest in its pipeline.

Looking ahead, Bancel pointed to artificial intelligence as a potential accelerator for mRNA drug development, citing Moderna’s rapid design of its COVID-19 vaccine candidate after the viral sequence became available. He said the company’s platform could allow it to respond rapidly to new biological discoveries across vaccines, oncology, autoimmune disease and rare diseases.

About Moderna (NASDAQ:MRNA)

Moderna, Inc is a biotechnology company that develops messenger RNA (mRNA) medicines and vaccines. Its platform is designed to provide cells with instructions to produce proteins that may help prevent or treat infectious diseases, cancer and other medical conditions.

The company's commercial products include Spikevax, an mRNA vaccine for COVID-19, and mRESVIA, an mRNA vaccine for respiratory syncytial virus (RSV) in older adults. Moderna also maintains a pipeline of investigational programs in areas such as seasonal influenza, cytomegalovirus, oncology and rare diseases.

Founded in 2010 and headquartered in Cambridge, Massachusetts, Moderna serves markets in the United States and internationally through regulatory approvals, commercial partnerships and distribution arrangements.

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