Verastem (VSTM) 12th Annual Cantor Fitzgerald Global Healthcare Conference summary

Pipeline and clinical development

• Advancing a G12D inhibitor with over 200 patients treated, targeting pancreatic, lung, and colorectal cancers, with significant data updates expected in October.

• Phase II trials are ongoing in all three indications, with plans to initiate phase III studies in the first half of next year, focusing on frontline settings.

• The G12D inhibitor is a dual on/off state molecule, highly selective, with long residence time and strong tolerability, aiming for best-in-class status.

• Combination strategies are prioritized, including with anti-EGFR agents and potential collaborations with pan-RAS or PRMT5 inhibitors.

• Regulatory strategy includes discussions for Accelerated Approval and Breakthrough Therapy designation, with confirmatory phase III studies planned.

Competitive landscape and efficacy benchmarks

• G12D inhibitor shows higher potency and selectivity compared to competitors, with fewer side effects than pan-RAS inhibitors.

• Efficacy benchmarks for Accelerated Approval are set at 30%+ response rates and at least six months durability, with higher targets in competitive tumor types.

• In pancreatic cancer, the drug aims to exceed the 30% response rate seen with other agents, with confidence in efficacy across multiple lines of therapy.

• In lung cancer, a 50% response rate is the competitive benchmark, with the drug expected to perform regardless of prior treatments.

• For colorectal cancer, combination with anti-EGFR agents is key, targeting a 30% response rate for approval.

Commercial progress and market strategy

• Commercial launch of avutometinib and defactinib in low-grade serous ovarian cancer is progressing well, with increased patient uptake and longer treatment durations.

• Market estimates for low-grade serous ovarian cancer range from $200M to $800M, with label expansion efforts underway.

• Uptick in PDAC usage observed, driven by strong clinical data, though reimbursement remains a challenge.

• Strategic partnerships are being explored to fund development, with a focus on maintaining upside and leveraging large partner expertise.

• Collaborations with other RAS pathway players and novel mechanism partners are prioritized, with potential deals expected this year.