The U.S. FDA named the first nine recipients of the recently unveiled commissioner’s national priority voucher (CNPV) program aimed at addressing unmet public health needs by shortening regulatory review times to as little as one to two months. For one of those firms, Disc Medicine Inc., which submitted an NDA for bitopertin for rare genetic disorder erythropoietic protoporphyria in September, that could mean a potential approval before the end of 2025.
Positive results for Revolution Medicines Inc.’s phase I studies of its lead candidate, the RAS-blocker daraxonrasib for treating pancreatic ductal adenocarcinoma, prompted the company to say it’s time for a phase III study in the aggressive cancer. Revolution said it plans to begin a global, randomized and registrational trial in first-line metastatic disease sometime in the fourth quarter of 2025.
Mutations in the GTPase KRAS drive proliferation of many types of tumors, and daraxonrasib (RMC-6236), which inhibits various KRAS mutants, can slow growth of pancreatic and lung cancers. Researchers at City University of New York and Weill Cornell Medical College have demonstrated that it also showed effects in osteosarcoma models expressing mutated KRAS.
Taking an unconventional path to market for its targeted therapies for RAS-addicted cancers, Revolution Medicines Inc. secured access to $2 billion in capital to build its own global commercial infrastructure, instead of partnering outside the U.S. as it had originally intended. “We’ve concluded that the best way for us to achieve our goals with our rich pipeline is to direct our own global development and commercial strategies and to operationalize these both inside and outside the U.S. through our own organization,” Mark Goldsmith, president and CEO of Revolution Medicines (Revmed), told investors June 24.
Revolution Medicines Inc. followed the results offered last month with zoldonrasib (RMC-9805), a RAS G12D (ON) inhibitor, with plenty more about pipeline progress – “an embarrassment of riches,” CEO Mark Goldsmith said – during the firm’s first-quarter update. Shares of the firm (NASDAQ:RVMD) closed May 8 at $41.91, up $4.25, or 11%.
