Avutometinib

Verastem Inc. (VSTM) is developing Avutometinib for low-grade serous ovarian cancer (LGSOC), with a phase II trial (RAMP 201) showing 31% overall response rate. The company started an NDA submission in May 2024 and expects FDA decision in H1 2025. A phase III study (RAMP 301) is ongoing, and Avutometinib is also being tested in NSCLC and pancreatic cancer trials.

Avutometinib and defactinib combo shows 31% overall response rate in recurrent low-grade serous ovarian cancer patients, with 44% in KRAS mutant and 17% in KRAS wild-type. Median progression-free survival is 12.9 months, 22 months in KRAS mutant. Verastem Oncology plans NDA submission in October 2024.

Dr. Hayslip appointed as Verastem Oncology's chief medical officer to lead avutometinib development programs, including the international Phase 3 RAMP 301 trial, and advance pipeline assets.

The KRAS Inhibitors market is poised for significant growth driven by increasing cancer incidence, treatment access, and a robust pipeline. The market size in the 7MM was $500 million in 2023, expected to rise by 2034. KRAZATI (adagrasib) is projected to outperform LUMAKRAS (sotorasib) in revenue. The US leads in KRAS mutation cases in NSCLC, with 46% of 7MM cases. Competition in G12C NSCLC is intensifying, with Chinese biotechs entering the KRAS space. Despite G12D being the most prevalent KRAS variant, G12C is the primary target in colorectal cancer. KRAZATI is the first KRAS inhibitor approved for KRAS G12C-mutant colorectal cancer. Pancreatic cancer, with 60-90% KRAS mutations, presents a significant market opportunity. Pan-KRAS drugs, targeting multiple mutations, offer broad market potential. Key companies include Novartis, Roche, Genentech, and Mirati Therapeutics. Emerging therapies like JDQ443, Divarasib, and Avutometinib are expected to reshape the market. The KRAS inhibitor market is evolving, driven by high unmet needs, competitive drug development, and regulatory approvals.

Dr. Hayslip appointed as Verastem Oncology's chief medical officer to lead clinical and medical strategy for avutometinib development, including Phase 3 RAMP 301 trial.

Verastem, Inc. (VSTM) develops cancer treatments targeting the RAS/MAPK pathway, focusing on pancreatic, lung, colorectal, and low-grade serous ovarian cancers. Its leading therapy combines Avutometinib and Defactinib, with a rolling NDA submission expected to finalize by 2H2024 for recurrent LGSOC. VSTM also explores additional indications for metastatic pancreatic cancer and mKRAS G12C NSCLC. The company's tight cash runway makes it a high-risk, high-reward investment, but its current valuation and pathway to approval suggest a bullish outlook.