Verastem (VSTM) RBC Capital Markets Global Healthcare Conference 2026 summary

Clinical development and differentiation

• VS-7375 was developed with GenFleet, focusing on oral bioavailability and on/off inhibition, with a long residence time on target and promising pharmacokinetics at higher doses, replicating positive preclinical and Chinese clinical results in the U.S.

• Patient populations in the U.S. and China are similar in mutational burden and prior therapies, with U.S. data guiding regulatory submissions and dose escalation beyond what was achieved in China.

• GI side effects observed in China have been mitigated in the U.S. through prophylactic ondansetron and feeding, with no significant liver or hematologic toxicity seen in the U.S., enabling higher dosing.

• Mature data on confirmed responses, PFS, and OS will be key for regulatory review, with safety and PK updates expected in June and efficacy data in the second half of the year across PDAC, CRC, and lung cohorts.

Regulatory and strategic outlook

• FDA feedback prompted the separation of registration-directed phase II protocols by indication, with plans for randomized phase III studies to support both accelerated and full approvals.

• Accelerated approval is not guaranteed, but strong data could enable rapid advancement, especially in CRC and lung where there is perceived white space and potential for combination regimens.

• G12D-specific inhibitors may offer better tolerability and combinability compared to pan-RAS agents, with ongoing studies in monotherapy and combinations (e.g., with gemcitabine, cetuximab, PD-1 inhibitors).

• The competitive landscape remains open due to high unmet need, with room for multiple agents and differentiation by efficacy and side effect profiles.

Commercial performance and launch strategy

• The commercial launch of Avmapki Fakzynja showed initial growth, with a strategic refocus and new leadership aiming to improve patient starts, therapy persistence, and earlier line use.

• January was soft due to typical insurance and out-of-pocket reset issues in the oral oncology space, but momentum improved in February and March, supported by new campaigns and data analytics investments.

• Efforts include educating physicians, leveraging EMR algorithms and chart audits to identify patients, and expanding the sales force for broader and deeper market penetration.

• Patient compliance is addressed through education on side effect management and dose re-initiation, with support from medical affairs and nurse educators to reinforce best practices.