Verastem presents updated data from RAMP 201 clinical trial

Verastem announced updated data from the Phase 2 RAMP 201 clinical trial evaluating the combination of avutometinib, an oral RAF/MEK clamp, and defactinib, an oral, selective FAK inhibitor, in patients with recurrent low-grade serous ovarian cancer, or LGSOC. The data were published as a late-breaking abstract and additional detailed findings will be presented in an oral plenary session at the International Gynecologic Cancer Society, or IGCS, 2024 Annual Meeting in Dublin, Ireland. The primary analysis of the RAMP 201 trial, with a data cutoff of June 30, showed a confirmed overall response rate, or ORR, by blinded independent central review of 31% in all evaluable patients with measurable disease with approximately 12 months of follow up. Among patients with KRAS mutant LGSOC, the confirmed ORR was 44% and for patients with KRAS wild-type LGSOC the confirmed ORR was 17%. The median duration of response, or DOR, was 31.1 months in all evaluable patients, with 31.1 months in the KRAS mt population and 9.2 months in the KRAS wt population. The median progression-free survival, or PFS, was 12.9 months in all evaluable patients, with 22 months in the KRAS mt population and 12.8 months in the KRAS wt population. The disease control rate, or DCR, at 6 or more months was 61% in the total evaluable population, 70% in KRAS mt population and 50% in KRAS wt population. The updated data continue to demonstrate avutometinib in combination with defactinib is generally well-tolerated, with a 10% discontinuation rate due to adverse events, or AEs, and no new safety signals were identified. The most common treatment-related AEs for the combination were nausea, diarrhea, and increased blood creatine phosphokinase levels. A Type A meeting was recently held with the FDA, during which the company aligned with the FDA on the company’s plans to complete the new drug application, or NDA, submission in October for adult patients with recurrent KRAS mt LGSOC, who received at least one prior systemic therapy, based on the mature data from the RAMP 201 trial. The company plans to seek Accelerated Approval from the FDA and request Priority Review. At this time, the FDA did not recommend pursuing a KRAS wt indication under accelerated approval. This strategic approach allows the company to potentially reach the market more efficiently while mapping out a path forward with the FDA for the KRAS wild-type indication, including leveraging data from the ongoing RAMP 301 Phase 3 trial. RAMP 301, which is currently enrolling patients with recurrent LGSOC regardless of KRAS mutation status, will serve as a confirmatory study for the initial indication and has potential to expand the indication regardless of KRAS mutation status.