Verastem Posts 83% Response Rate in Pancreatic Cancer Trial

Company plans Phase 3 trial in 2026

Verastem Oncology (Nasdaq: VSTM) announced encouraging results from its RAMP 205 trial in advanced pancreatic cancer. The study is evaluating a combination of targeted therapies with standard chemotherapy in newly diagnosed patients.

At the selected Phase 2 dose level, 83% of patients (10 of 12) experienced tumor shrinkage, with 8 confirmed and 2 unconfirmed partial responses. This dosing level has now advanced into an expansion cohort of up to 29 patients. Verastem plans to launch a Phase 3 registrational trial in 2026.

The RAMP 205 treatment includes:

• Avutometinib, which blocks cancer cell growth by targeting the MEK and RAF proteins in the MAPK pathway

• Defactinib, which targets tumor resistance by inhibiting FAK, a protein that helps cancer cells survive and spread

Additional Findings from the Trial

Across all dose groups, 92% of patients (48 of 52) saw tumor reduction. Importantly, safety data remained consistent with earlier findings. No new side effects have emerged. Verastem has already begun enrolling patients in the expanded Phase 2 portion of the trial.

Lung and Pancreatic Cancer Programs Advancing

Beyond pancreatic cancer, Verastem is also advancing its KRAS G12D inhibitor, VS-7375, which targets a common cancer-driving mutation. The company is co-developing the drug with GenFleet Therapeutics.

Notably, early data from a study in China showed a 43% response rate in pancreatic cancer and 33% in non-small cell lung cancer (NSCLC). Most side effects were mild and related to digestion. Verastem plans to launch a U.S. trial for VS-7375 later this year.

FDA Approval for Ovarian Cancer Treatment

In May, the FDA granted accelerated approval for Verastem’s combination of avutometinib and defactinib. The therapy, now marketed as AVMAPKI™ FAKZYNJA™ CO-PACK, is approved for KRAS-mutated low-grade serous ovarian cancer. It is the first treatment ever approved for this specific cancer.

Verastem is also evaluating the same combination in other hard-to-treat cancers, including pancreatic, lung, and colorectal tumors.