Verastem (VSTM) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Company overview and oncology platform

• Focused on small molecules targeting the MAPK/RAS pathway, with avutometinib and defactinib as lead assets.

• Lead program targets low-grade serous ovarian cancer (LGSOC), a rare, chemotherapy-resistant disease affecting younger women.

• LGSOC has a long disease course, with mean survival around 10 years and poor response to standard therapies.

• Combination therapy aims to improve tolerability and efficacy for long-term treatment.

Clinical development and data updates

• Interim pivotal study data for LGSOC reported earlier in the year; mature data to be presented in mid-October.

• Rolling NDA submission for accelerated approval began in May, with final clinical module to be submitted by year-end.

• RAMP 201 study completed, showing higher response rates for the combination versus single agent, especially in KRAS-mutant patients.

• Confirmatory Phase III (RAMP 301) is underway, randomizing against standard of care with progression-free survival as the primary endpoint.

Pipeline expansion and partnerships

• Pancreatic cancer is the next major indication, with early data showing an 83% response rate in combination with standard of care.

• Partnerships with Amgen and BMS for G12C inhibitor combinations; early preclinical and clinical data are promising.

• G12D inhibitor licensed from GenFleet entered the clinic in July, with plans to accelerate US development.

• Additional early-stage programs in gynecologic, colorectal, and breast cancers are ongoing.