Verastem (VSTM) H.C. Wainwright 4th Annual BioConnect Investor Conference summary

Strategic focus and product overview

• Focused on targeting the RAS/MAPK pathway, with two main programs: a commercial product for low-grade serous ovarian cancer (LGSOC) and a KRAS G12D inhibitor in development.

• Achieved accelerated approval for the LGSOC therapy, demonstrating a 44% response rate in KRAS mutant patients, significantly higher than standard therapies.

• Commercial launch faced Q1 seasonality challenges due to Medicare changes but recovered in subsequent months, with expectations for stronger performance in Q2 and beyond.

• The KRAS G12D inhibitor has shown promising preclinical and early clinical results, with high response rates in pancreatic and lung cancers.

• Phase II studies for the KRAS G12D inhibitor in PDAC, lung, and CRC are set to begin in June, with phase III trials planned for next year.

Commercialization and market insights

• Early launch of the LGSOC therapy saw sicker patients first; ongoing efforts focus on identifying and retaining appropriate patients earlier in their treatment journey.

• Emphasis on education and collaboration with practices to identify eligible patients, especially as diagnostic criteria for LGSOC become more widely recognized.

• Adjusted sales and support strategies to balance patient identification with retention, aiming to replicate clinical trial outcomes in real-world settings.

• Expansion of prescriber base through academic and community outreach, leveraging peer-to-peer education and partnerships with large practices and GPOs.

• Confirmatory studies include both KRAS mutant and wild type patients, with plans to expand the label and promotional efforts pending positive study results.

Clinical development and competitive positioning

• The KRAS G12D inhibitor is differentiated by its isoform specificity, on/off inhibition, and long residence time, potentially offering a better safety profile than pan-RAS inhibitors.

• Achieved target drug concentrations at 900 mg dose, supporting advancement to phase II trials.

• Prioritizing PDAC for proof of concept, with additional focus on CRC and lung cancer; combinations with chemotherapy and targeted agents are being explored.

• Regulatory strategy aims for accelerated approval based on high response rates, with phase III trials serving as confirmatory or primary approval studies.

• Ongoing dose escalation to 1,200 mg to fully characterize safety and support regulatory requirements.