Avutometinib in Combination with Defactinib - (RAMP 201)

October 2, 2026: Verastem Oncology (NASDAQ: VSTM) reported new Phase 2 RAMP 201 analyses for avutometinib plus defactinib in recurrent low-grade serous ovarian cancer (LGSOC) at the IGCS 2026 Annual Global Meeting. In KRAS wild-type disease, molecular profiling showed activity in triple wild-type tumors (KRAS/NRAS/BRAF wild-type) with confirmed ORR 18% (7/39) and median PFS 13 months; in KRAS wild-type tumors with NRAS or BRAF mutations, confirmed ORR was 22% (2/9).

An external control arm analysis comparing RAMP 201 with matched conventional care from GOG 281 showed higher weighted ORR and longer PFS with the combination. In KRAS-mutated LGSOC, weighted ORR was 38.7% vs 0% and median weighted PFS was 22.1 vs 6.5 months. In KRAS wild-type LGSOC, weighted ORR was 22.7% vs 7.9% and median weighted PFS was 11.3 vs 7.7 months.

Verastem also highlighted an encore Phase 2 RAMP 201J presentation in Japanese patients: as of May 29, 2026, 16 efficacy-evaluable patients had ORR 44% and disease control rate 94%; ORR was 71% in KRAS-mutated tumors and 22% in KRAS wild-type tumors. The company said the combination was FDA-approved on May 8, 2025 for KRAS-mutated recurrent LGSOC after prior systemic therapy, with Phase 3 confirmatory trial RAMP 301 ongoing.