Moderna-Merck gets USFDA Breakthrough Therapy Designation for personalized mRNA cancer vaccine in combination with KEYTRUDA to treat high risk melanoma

"The FDA's Breakthrough Designation for mRNA-4157/V940 in combination with KEYTRUDA reflects the excitement that we have for the potential promise of individualized cancer treatments," said Stephen Hoge, M.D., Moderna's President. "mRNA-4157/V940 in combination with KEYTRUDA provided the first demonstration of efficacy for an investigational mRNA cancer treatment in a randomized clinical trial and potentially represents a new frontier in treating melanoma and other cancers. We look forward to publishing the full data set and sharing the results at an upcoming oncology medical conference, as well as continuing discussions with health authorities. We are grateful to the FDA for this designation."

"This is an important milestone in the development of mRNA-4157/V940 in combination with KEYTRUDA," said Dr. Eric H. Rubin, senior vice president, global clinical development, Merck Research Laboratories. "We look forward to working with the FDA, in collaboration with Moderna, to conduct a rigorous and rapid clinical development program with a focus on addressing the needs of this important patient population."

The FDA's Breakthrough Therapy Designation is granted to expedite the development and review of drugs that are intended to treat a serious condition, and when preliminary clinical evidence indicates that the product may demonstrate substantial improvement over available therapies on at least one clinically significant endpoint.

"The companies will continue to discuss the results with regulatory authorities, initiate a Phase 3 study in adjuvant melanoma in 2023, and rapidly expand to additional tumor types, including non-small cell lung cancer," the company stated.

mRNA-4157/V940 is a novel investigational messenger ribonucleic acid (mRNA)-based personalized cancer vaccine consisting of a single synthetic mRNA coding for up to 34 neoantigens that is designed and produced based on the unique mutational signature of the patient's tumor. Upon administration into the body, the algorithmically derived and mRNA-encoded neoantigen sequences are endogenously translated and undergo natural cellular antigen processing and presentation, a key step in adaptive immunity.

Personalized cancer vaccines are designed to prime the immune system so that a patient can generate a tailored antitumor response specific to their tumor mutation signature. mRNA-4157/V940 is designed to stimulate an immune response by generating specific T cell responses based on the unique mutational signature of a patient's tumor. KEYTRUDA is an immunotherapy that works by increasing the ability of the body's immune system to help detect and fight tumor cells.