Verastem Oncology announced that it has finalized with the U.S. Food and Drug Administration (FDA) the design of its confirmatory Phase 3 trial to evaluate the combination of avutometinib and defactinib for the treatment of recurrent low-grade serous ovarian cancer (LGSOC). RAMP 301, a randomized global confirmatory trial, will evaluate the efficacy and safety of avutometinib and defactinib versus standard of care (SOC) chemotherapy and hormonal therapy in patients with recurrent LGSOC. RAMP 301 is expected to begin enrollment in the second half of this year.

RAMP 301 is the follow-up confirmatory study for full approval in recurrent LGSOC. The Company intends to file for Accelerated FDA Approval for the combination of avutometinib and defactinib based on mature data from the Company’s Phase 2 registration-directed RAMP 201, together with the results of the investigator-initiated FRAME trial. The Company recently reported results of Part A of RAMP 201 including confirmed objective response rates (ORR) by blinded independent central review of 45% (13/29; 95% CI: 26%,64%) with a tolerable safety profile.?

RAMP 301 is an international collaboration between The GOG Foundation, Inc. (GOG) and the European Network of Gynaecological Oncological Trial groups (ENGOT) sponsored by Verastem Oncology. The trial will enroll approximately 270 patients who will be randomized to either the combination of avutometinib and defactinib or SOC chemotherapy (pegylated liposomal doxorubicin, paclitaxel, topotecan) or hormone therapy (letrozole, anastrozole).  Selection of the SOC regimen will be based on the preference of the investigator. The primary endpoint is progression free survival (PFS) by Blinded Independent Central Review. Secondary endpoints include overall response rates, duration of response, disease control rate, safety and tolerability, patient reported outcomes and overall survival.

The RAMP 301 trial will be led globally and in the U.S. by Rachel Grisham, MD, Section Head, Ovarian Cancer and Director, Gynecologic Medical Oncology at Memorial Sloan Kettering Cancer Center in Westchester, NY. Susana Banerjee, MBBS, MA PhD, FRCP, global and lead investigator of the RAMP 201 study, Consultant Medical Oncologist at The Royal Marsden NHS Foundation Trust and Team Leader in Women’s Cancers at The Institute of Cancer Research, London, will be leading the RAMP 301 trial in Europe.