Verastem announces results from avutometinib, defactinib trial at IGCS 2023

Verastem announced results of the efficacy and safety of avutometinib and defactinib in recurrent low-grade serous ovarian cancer, or LGSOC, following prior systemic therapy. The results of this planned subgroup analysis of Part A of the Phase 2 RAMP 201 trial were presented as a late-breaking abstract in an oral presentation during a plenary session at the annual global meeting of the International Gynecologic Cancer Society, or IGCS 2023, November 5-7 in Seoul, Korea. This planned subgroup analysis was performed to assess efficacy and safety in prior lines of therapy. The analysis also evaluated efficacy in the context of best response to most recent prior treatment in the metastatic/recurrent setting. In the combination arm, the observed ORRs were consistent across patients who received 1-3 and greater than or equal to4 lines of therapy. Prior to enrollment in RAMP 201, only 2/23 patients responded to their last prior treatment in the metastatic/recurrent setting, whereas the combination of avutometinib and defactinib yielded an ORR of 43.5% in this subgroup. The safety profiles of avutometinib and defactinib were similar in the less and more heavily pretreated subgroups and both analyses were consistent with previously reported safety data. The majority of treatment-emergent adverse events were mild to moderate. Initial results of RAMP 201 Part A, presented at the American Society for Clinical Oncology Annual Meeting in May 2023, demonstrated an ORR of 45% and tumor shrinkage in 86% of evaluable patients that were treated with the combination of avutometinib and defactinib. Safety and tolerability were favorable and consistent with previously reported data. As previously announced, Verastem Oncology intends to file for accelerated approval with the FDA for the combination of avutometinib and defactinib based on mature data from the RAMP 201 trial, together with the results of the investigator-initiated FRAME trial. The company plans to initiate its Phase 3 confirmatory trial of avutometinib and defactinib in LGSOC versus standard of care, or SOC, chemotherapy or hormone therapy before the end of the year.