BOSTON — September 28, 2026

Verastem Oncology announced the publication in Gynecologic Oncology of two-year follow-up data from the Phase 2 RAMP 201 study evaluating AVMAPKI FAKZYNJA CO-PACK (avutometinib capsules; defactinib tablets) in women with recurrent low-grade serous ovarian cancer (LGSOC). With a median follow-up of 24.9 months, the updated analysis reported a median duration of response (mDoR) of 31.1 months and median progression-free survival (mPFS) of 12.9 months across patients with KRAS-mutated and KRAS wild-type disease. The company reported that efficacy measures remained consistent with the primary analysis, while no new safety signals were identified. The RAMP 201 results supported the U.S. FDA’s May 2025 accelerated approval of AVMAPKI FAKZYNJA CO-PACK for adults with KRAS-mutated recurrent LGSOC following prior systemic therapy.

Long-Term RAMP 201 Analysis Shows Durable Responses

The updated RAMP 201 analysis included ongoing patients with more than two years of follow-up and showed sustained disease-control measures across KRAS subgroups. Among 17 patients included in the long-term analysis, median duration of response was 31.1 months overall and in the KRAS-mutated subgroup, while the KRAS wild-type subgroup had a median duration of response of 12 months. Median progression-free survival was 12.9 months overall, 19.6 months for KRAS-mutated disease and 12.7 months for KRAS wild-type disease. The company also reported that the median duration of response in KRAS wild-type patients increased from 9.2 months in the primary analysis to 12 months in the longer-term analysis. Fifty percent of patients with KRAS-mutated LGSOC and 30% of those with KRAS wild-type disease remained on treatment for more than one year. These findings describe longer-term outcomes from the study and do not represent results from a randomized confirmatory comparison.

Safety Profile Remains Consistent at Two Years

Verastem reported that the safety profile in the longer-term analysis remained consistent with the primary RAMP 201 analysis, with no new safety signals identified. The company reported a 12% discontinuation rate due to adverse events in the updated analysis. AVMAPKI FAKZYNJA CO-PACK combines avutometinib, which inhibits MEK activity and blocks RAF-mediated reactivation of MEK, with defactinib, a focal adhesion kinase (FAK) inhibitor. The combination is designed to inhibit signaling within the RAS/MAPK pathway and mechanisms associated with treatment resistance. The FDA approved the co-pack in May 2025 under the accelerated approval pathway for adults with KRAS-mutated recurrent LGSOC who have received prior systemic therapy. The indication remains subject to confirmatory evidence of clinical benefit.

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RAMP 301 Continues Confirmatory Development

Verastem is continuing the international Phase 3 RAMP 301 trial as the confirmatory study for the approved KRAS-mutated recurrent LGSOC indication. RAMP 301 is evaluating avutometinib plus defactinib against standard chemotherapy or hormonal therapy in recurrent LGSOC patients with and without KRAS mutations. The company is also studying the combination with standard-of-care chemotherapy in first-line advanced pancreatic cancer in the Phase 2 RAMP 205 trial. The longer-term RAMP 201 findings provide additional follow-up from the registration-directed Phase 2 program, while the ongoing Phase 3 study is intended to generate comparative evidence required to verify clinical benefit. AVMAPKI FAKZYNJA CO-PACK is currently approved in the United States only for KRAS-mutated recurrent LGSOC after prior systemic therapy; its use in other indications remains investigational.

Source :Verastem Oncology press release