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Verastem Oncology Plans for FDA Submission of Avutometinib-Defactinib Combo in Ovarian Cancer

NEW YORK – Verastem Oncology on Wednesday said it will seek accelerated approval for the combined use of its RAF/MEK inhibitor avutometinib and FAK inhibitor defactinib in patients with recurrent low-grade serous ovarian cancer after a Phase II trial yielded positive interim results.

Based on patients' response rates seen so far with avutometinib alone and combined with defactinib in the registration-directed RAMP 201 trial, the firm selected the combination regimen to move forward in the trial. Patients with KRAS-mutant and KRAS-wild type recurrent low-grade serous ovarian cancer are eligible to partake in the study.

Verastem said it recently met with the US Food and Drug Administration to discuss results from the ongoing Phase II RAMP 201 trial and the regulatory path forward for avutometinib-defactinib. Based on those discussions, the firm could potentially pursue accelerated approval based on mature data from RAMP 201 as well as the Phase II FRAME study, which also explored the activity of avutometinib-defactinib in ovarian cancer. Verastem is also in talks with the FDA about conducting a confirmatory study for the combination treatment.

The data from RAMP 201 appears to support Verastem's bid to seek approval for avutometinib-defactinib in an all-comer population, though the trial did stratify patients based on KRAS mutation status. Of 29 evaluable patients in the RAMP 201 trial, avutometinib-defactinib demonstrated an objective response rate of 28 percent in all patients, with 27 percent of KRAS-mutant patients responding and 29 percent of KRAS wild-type patients responding. The disease control rate across all patients was 93 percent. The avutometinib monotherapy arm, comparatively, showed a response rate of 7 percent and a disease control rate of 90 percent.

Verastem plans to present additional data from RAMP 201 at a medical conference later this year.