Verastem Oncology

Verastem Oncology plans to submit an NDA in Oct 2024 for avutometinib and defactinib combo to treat recurrent low-grade serous ovarian cancer with KRAS mutations. Data from the RAMP 201 trial showed a 31% ORR in the overall population and a 44% ORR in KRAS-mutated patients, with a median DOR of 31.1 months. The combo was well-tolerated, with no new safety signals.

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.

S.B., A.D., T.J.F., I.M.G., L.G., R.K.J., G.K., J.M.L., G.V.L., K.P.-J., H.I.S., J.T., R.R.W., M.W., Y.L.W. have disclosed various financial interests, including funding, advisory roles, patents, and equity. C.M.B., E.B., M.W.B., and K.P. declare no competing interests.

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.

FDA granted orphan drug designation to avutometinib and defactinib for pancreatic cancer treatment. In the RAMP 205 study, the combination with SOC showed an 83% ORR. The study aims to determine MTD and RP2D, with preliminary efficacy and safety data reported. Avutometinib and defactinib are also being studied for ovarian cancer.

Verastem Oncology announced interim results from the RAMP 205 trial for metastatic pancreatic cancer, showing an 83% ORR in one cohort. They're also submitting an NDA for a KRAS mutant ovarian cancer treatment and collaborating on NSCLC trials. VSTM stock dropped 59% premarket Friday.

Verastem Oncology's NDA for avutometinib and defactinib combo, targeting recurrent KRAS mutant LGSOC, received FDA Priority Review with a PDUFA date of June 30, 2025. This potential first-ever FDA-approved treatment for the condition is based on Phase 2 RAMP 201 trial results, showing durable responses and tolerability.