Verastem (VSTM) Guggenheim SMID Cap Biotech Conference summary

Pipeline and regulatory progress

• NDA for avutometinib and defactinib in low-grade serous ovarian cancer (LGSOC) accepted with priority review; FDA decision expected summer 2025.

• Ongoing daily interactions with the FDA; launch preparations underway for potential U.S. approval.

• Additional data readouts expected in frontline metastatic pancreatic cancer and triplet combination studies mid-year.

• Early data in pancreatic cancer showed over 80% response rate; further updates and phase II dose selection expected by mid-2025.

• G12D inhibitor program advanced after meeting PK, efficacy, and safety criteria; U.S. IND submission planned this quarter.

Clinical data and commercial opportunity

• LGSOC response rates above 30% overall and 40% in KRAS mutant, compared to 6%-13% for standard of care.

• 82% of patients experienced tumor shrinkage; clinical benefit rate of 60% overall, 70% in KRAS mutant.

• Market opportunity estimated at $4 billion, with both incident and prevalent patient segments; long therapy duration supports annuity model.

• Initial uptake expected to be modest, building over time as guidelines and awareness increase.

• U.S. launch preparations include patient engagement, HCP education, and partnership with IQVIA for infrastructure and analytics.

Regulatory and guideline inclusion

• Expectation for initial approval in KRAS mutant LGSOC; high likelihood of NCCN guideline inclusion within a month of approval.

• Confirmatory phase III RAMP 301 study enrolling both wild type and mutant; strong confidence in positive outcomes for all-comers.

• Ex-U.S. progress most advanced in Japan with bridging study; groundwork ongoing in Europe and China.