Verastem Oncology Reports Promising RAMP 201 Data for Avutometinib and Defactinib in Low-Grade Serous Ovarian Cancer
Verastem Oncology has announced encouraging results from its Phase 2 RAMP 201 trial, showcasing the combination of avutometinib and defactinib in patients with recurrent low-grade serous ovarian cancer (LGSOC). The study reported an overall response rate (ORR) of 31%, with a notable 44% in patients with KRAS mutations and 17% in those with KRAS wild-type, despite prior treatments including chemotherapy and MEK inhibitors.
Patients receiving this combination therapy achieved a median progression-free survival (PFS) of 12.9 months overall, with those having KRAS mutations experiencing a longer median PFS of 22 months. The data, which were presented as a late-breaking abstract at the International Gynecologic Cancer Society (IGCS) 2024 Annual Meeting in Dublin, Ireland, reveal the potential of this therapy to enhance treatment options for patients with LGSOC, a rare and difficult-to-treat cancer.
The trial’s results indicated that 61% of patients maintained disease control for six months or longer. Importantly, the combination therapy was well-tolerated, with only a 10% discontinuation rate due to adverse events. The most commonly reported side effects included nausea (67%), diarrhea (58%), and elevated blood creatine phosphokinase levels (60%).
Professor Susana Banerjee, Global Lead Investigator, emphasized the significance of these findings, stating that the robust response rates and manageable side effects could establish this combination as the new standard of care for patients with recurrent LGSOC, a population that previously had limited effective treatments.
In a recent meeting with the U.S. Food and Drug Administration (FDA), Verastem aligned on plans to submit a New Drug Application (NDA) in October 2024, focusing on adult patients with recurrent KRAS mutant LGSOC who have undergone at least one prior systemic therapy. The company aims to secure Accelerated Approval and Priority Review for this indication. However, the FDA advised against pursuing a KRAS wild-type indication under Accelerated Approval at this time.
The mature data from the RAMP 201 trial underscore the potential of the combination therapy to improve treatment outcomes for patients, with a commitment to make it widely available. Dr. John Hayslip, Chief Medical Officer of Verastem, expressed confidence in the therapy’s ability to transform the treatment landscape for recurrent LGSOC.
Dan Paterson, President and CEO of Verastem, announced the anticipated NDA submission and the company’s goal of introducing the first FDA-approved treatment specifically for recurrent KRAS mutant LGSOC in the U.S. by 2025.
Conference Call and Webcast
Verastem will hold an investor conference call and webcast on October 17, 2024, at 4:30 p.m. EDT to discuss the RAMP 201 trial data. Investors can join the call by dialing (844) 763-8274 for local or (412) 717-9224 for international access, or they can listen to the live webcast on the company’s website.
About RAMP 201
The RAMP 201 trial is a multicenter study aimed at evaluating the efficacy and safety of the combination therapy for recurrent LGSOC. The trial consists of multiple parts, with the current focus on the combination of avutometinib and defactinib.
About Verastem Oncology
Verastem Oncology is dedicated to developing innovative therapies for patients with cancer, with a focus on RAS/MAPK-driven tumors. Their pipeline includes drugs targeting critical signaling pathways that promote cancer growth and survival. For more information, visit Verastem’s website and follow them on LinkedIn.
