Verastem announces two-year median follow-up data on AVMAPKI FAKZYNJA combo

Verastem (VSTM) Oncology announced two-year median follow-up data from the Phase 2 RAMP 201 clinical trial that evaluated AVMAPKI FAKZYNJA combination therapy in patients with recurrent low-grade serous ovarian cancer will be presented today during an oral plenary session at the Society of Gynecologic Oncology 2026 Annual Meeting on Women’s Cancers taking place in San Juan, Puerto Rico, April 10-13. “Patients who remained on treatment with avutometinib plus defactinib for two years were able to maintain the same level of response and duration of therapy as seen in the primary analysis, suggesting that patients can stay on the combination for a long period of time, derive benefit and have manageable toxicity during long-term administration of these medications,” said Rachel Grisham, RAMP 201 Presenting Investigator, Section Head, Ovarian Cancer at Memorial Sloan Kettering Cancer Center and Global Lead Principal Investigator of GOG-3097/ENGOT-ov81/GTG-UK/RAMP 301. “As we approach the one-year FDA approval anniversary of avutometinib in combination with defactinib, this analysis reinforces progress in bringing a durable and clinically meaningful option to patients.” In the updated analysis, with ongoing patients presenting a median follow-up of 24.9 months, efficacy measures, including median duration of response and median progression free survival, and safety, are consistent with the primary analysis, which was conducted more than 13 months prior. Fifty percent of patients with KRAS-mutated and 30 percent with KRAS wild-type LGSOC remained on therapy for more than one year. Adverse events were consistent with the primary analysis with no new safety signals observed, and a 12 percent discontinuation rate due to adverse events. Efficacy analyses included patients with LGSOC from the FRAME and RAMP 201 studies, while safety analyses included a broader population from the FRAME, RAMP 201, and RAMP 202 studies. All efficacy endpoints, including overall response rates, duration of response, and best target lesion response, suggest the best therapeutic effect is achieved with the FDA approved dose of avutometinib 3.2 mg twice weekly plus defactinib 200 mg twice daily. While a lower avutometinib dose may mitigate treatment emergent adverse events, it may also compromise the efficacy. Importantly, these analyses demonstrated that TEAEs can be monitored and managed with dose interruptions, and subsequently resume treatment at the approved dose level, to allow patients to stay on treatment.