Moderna (MRNA) is expanding its mRNA platform beyond infectious disease into oncology and rare genetic disorders, executives told attendees at the Bernstein Insights Healthcare Leaders and Disruptors forum on Wednesday.
Chief Executive Stéphane Bancel opened the presentation by stressing the company’s long-held positioning. “Moderna has never been a COVID-19 vaccine company. Moderna has been, since day one, an mRNA platform company,” he said.
Moderna raised $2.6 billion through convertible notes to support its broadened pipeline and accelerate development timelines, the company said. Revenue for the last twelve months was $2.23 billion, while gross profit margin sat at negative 66%. Earnings per share were -$8.01.
The most significant clinical update centered on intismeran (mRNA-4157 / V940), a personalized neoantigen cancer vaccine developed with Merck. The Phase III adjuvant melanoma study met both its primary endpoint and a key secondary endpoint—distant metastasis-free survival—at its first interim analysis. Moderna noted the trial marks the first adjuvant study to hit both endpoints at an interim readout and the first adjuvant trial to beat PD-1 monotherapy when added to treatment. The trial enrolled 1,100 patients, and the program is now being studied across nine ongoing trials in multiple tumor types.
Under the partnership with Merck, costs and profits are shared equally through a structure described as a virtual joint venture rather than a legal entity, with expenses split across sales, studies, manufacturing, capital spending and operating costs.
David Berman, Moderna’s chief development officer, called the earlier Phase II data “a landmark phase II trial” and described it as the most rigorous immunotherapy cancer trial he has seen. “Intratumoral does not need a PD-1 to be active,” Berman said, calling the finding “a really important point.”
On the rare-disease front, Moderna expects pivotal data for its propionic acidemia therapy by the end of 2024. The liver-targeted treatment uses an intravenous lipid injection rather than an intramuscular approach. Preclinical data has been published across more than 12 rare genetic disease models, including methylmalonic acidemia. The company also highlighted its Lynch syndrome program—a Phase I/II trial in the United Kingdom focused on cancer interception in high-risk patients. Lynch syndrome affects roughly 1 in 400 people and can raise the risk of certain cancers up to tenfold.
Bancel pointed to Moderna’s manufacturing scale-up during the pandemic as proof of platform capability: production grew from fewer than 100,000 doses across all clinical trials in 2019 to 1 billion doses within 18 months. Each patient cassette uses approximately 34 neoantigens, with about one-third expected to be immunogenic.
The company currently has five approved vaccine products. Moderna acquired Translate Bio and has previously partnered with CureVac and BioNTech.
Shares closed Wednesday at $154.04, down 2.55%, and were trading at $156.55 in pre-market session, up 1.63%.
