Company description

We are a biopharmaceutical company committed to developing and commercializing new medicines to improve the lives of patients diagnosed with challenging RAS/MAPK pathway-driven cancers. We market AVMAPKI FAKZYNJA CO-PACK (avutometinib capsules; defactinib tablets) in the United States (“U.S.”), 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. We are also conducting RAMP 301, a Phase 3 trial designed to evaluate avutometinib plus defactinib versus Investigator’s Choice of Treatment (“ICT”) in patients with recurrent LGSOC with and without a KRAS mutation. This trial will serve as a confirmatory study for the initial U.S. indication and has the potential to expand the indication regardless of KRAS mutation status. Results of the RAMP 301 trial may also support future regulatory filings outside of the U.S. Our pipeline includes clinical-stage programs, preclinical research programs and externally partnered early-stage programs. Our focus is on novel small molecule drugs developed both as monotherapy and in combination, which inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including targeting RAS directly with KRAS G12D inhibition, targeting the pathway downstream with RAF/MEK inhibition, and targeting the parallel pathway that drives resistance with FAK inhibition.

Read the full filing description · filed 4 Mar 2026

At a glance

Sector

Healthcare

Industry

Pharmaceutical Preparations