Verastem Oncology is set to present multiple data analyses from its innovative cancer treatment programs at the upcoming Society of Gynecologic Oncology (SGO) 2025 Annual Meeting, scheduled for March 14-17 in Seattle, Washington. The presentations will spotlight new findings from the company's groundbreaking research in low-grade serous ovarian cancer (LGSOC) treatment.
Key Clinical Data Presentations
Dr. Rachel Grisham will deliver an oral presentation featuring additional analyses from the Phase 2 RAMP 201 trial, examining the combination of avutometinib and defactinib in recurrent LGSOC patients. This presentation, scheduled for March 17, will provide detailed insights into dose intensity and subgroup analyses based on KRAS mutational status.
The data being presented served as the foundation for Verastem's New Drug Application (NDA), which is currently under Priority Review by the FDA. The regulatory agency has set a Prescription Drug User Fee Act (PDUFA) date of June 30, 2025, for the novel combination therapy.
Novel Therapeutic Mechanism
The investigational treatment combines two innovative approaches to targeting cancer cells. Avutometinib functions as a RAF/MEK clamp, providing more complete inhibition of the RAS/MAPK pathway compared to traditional MEK-only inhibitors. This unique mechanism blocks both MEK kinase activity and RAF-mediated MEK phosphorylation, potentially offering more durable anti-tumor responses.
Defactinib, the combination partner, selectively inhibits focal adhesion kinase (FAK) and proline-rich tyrosine kinase-2 (Pyk2). This targeted approach helps address potential resistance mechanisms to RAF and MEK inhibitors, creating a more robust therapeutic strategy.
Regulatory Progress and Clinical Development
The FDA has granted several key designations to the combination therapy, including:
- Breakthrough Therapy Designation for recurrent LGSOC in May 2021
- Orphan Drug Designation for LGSOC treatment
- Priority Review status for the current NDA
Dan Paterson, President and CEO of Verastem Oncology, emphasized the significance of these presentations: "We look forward to sharing these learnings with many of the world's leading gynecologic oncologists at SGO as part of our continued commitment to people living with recurrent low-grade serous ovarian cancer."
Expanding Research Program
Beyond the primary LGSOC program, Verastem is actively pursuing additional applications of their RAF/MEK pathway targeting approach. The company has initiated RAMP 301, an international Phase 3 confirmatory trial, comparing the avutometinib-defactinib combination against standard chemotherapy or hormonal therapy in recurrent LGSOC.
The company has also established strategic collaborations, including a partnership with Amgen to evaluate the combination with LUMAKRAS (sotorasib) in KRAS G12C mutant non-small cell lung cancer, demonstrating the broader potential of their approach in RAS/MAPK pathway-driven cancers.
