Verastem, Inc.

Verastem, Inc. is a biopharmaceutical company that develops and commercializes new medicines for patients with challenging RAS/MAPK pathway-driven cancers. The company markets AVMAPKI FAKZYNJA CO-PACK (avutometinib capsules; defactinib tablets) in the United States. This product is the first treatment specifically FDA-approved for adults with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy. AVMAPKI FAKZYNJA CO-PACK received accelerated approval in the U.S. on May 8, 2025. Verastem is also conducting RAMP 301, a Phase 3 trial evaluating avutometinib plus defactinib versus Investigator's Choice of Treatment (ICT) in patients with recurrent LGSOC, both with and without a KRAS mutation. This trial serves as a confirmatory study for the initial U.S. indication and has the potential to expand the indication regardless of KRAS mutation status. Results from RAMP 301 may also support future regulatory filings outside of the U.S. The company's pipeline includes clinical-stage programs, preclinical research programs, and externally partnered early-stage programs. Their focus is on novel small molecule drugs developed as monotherapy and in combination, which inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth. This includes directly targeting RAS with KRAS G12D inhibition, targeting the pathway downstream with RAF/MEK inhibition, and targeting the parallel pathway that drives resistance with FAK inhibition. For clinical-stage pipeline programs, Verastem is evaluating VS-7375, an oral KRAS G12D (ON/OFF) inhibitor, for the treatment of patients with KRAS G12D mutated cancers, including pancreatic ductal adenocarcinoma (PDAC), non-small cell lung cancer (NSCLC), colorectal cancer (CRC), and other KRAS G12D mutated cancers. The company also has two undisclosed assets at the discovery phase targeting RAS/MAPK pathway-driven cancers as part of the GenFleet Therapeutics collaboration.