Verastem (VSTM) 3rd Annual Guggenheim Healthcare Innovation Conference summary

Portfolio and leadership

• Avutometinib/defactinib CO-PACK, a RAF/MEK and FAK inhibitor, was approved in May 2025; sales now cover the commercial force, making the asset break-even.

• The recent capital raise is intended to fund accelerated VS-7375 development across pancreatic, colorectal and lung cancers.

• Dan Paterson is transitioning out after serving as CEO since 2011; Michael Bailey is expected to become CEO on January 1.

VS-7375 data and dosing

• October 14 data were described as potentially best-in-class; development targets PDAC, CRC and NSCLC, with PDAC and CRC studies expected to complete enrollment by year-end and NSCLC enrollment to follow early next year.

• The 900 mg dose showed dose-proportional exposure, was characterized more fully than GenFleet’s 600 mg dose, and had tolerability described as identical to 600 mg.

• Main adverse events were predominantly low-grade GI disorders, mostly grade 1, with ~10%-15% grade 2; the overall safety profile was described as clean.

• In 900 mg previously treated PDAC, the 41% response rate included all dosed patients in the intent-to-treat population and confirmed responses; results were described as consistent across second-line and later settings.

• The 22-patient PDAC dataset included two complete responses; six-month landmark PFS was 67%, which management characterized as competitive.

Pancreatic cancer development

• TARGET-D 201’s main cohort was nearly fully enrolled, with completion expected before year-end; management is targeting a potential Q2 submission, subject to mature duration-of-response data and further FDA alignment.

• Management said approval could potentially come as early as the end of next year and described second-line PDAC as a potential first-to-market opportunity for a G12D inhibitor.

• TARGET-D 201 is randomizing second-line patients between monotherapy and cetuximab combination to establish contribution of components; planned enrollment is 80 patients per arm, with a potential mid-year readout.

• Early cetuximab-combination observations included responses at subtherapeutic doses and deeper, faster responses at higher doses; phase I data may be presented in Q1 next year, with randomized data expected mid-year as they mature.

• The frontline PDAC phase III plan includes cetuximab or GemNab combinations, not FOLFIRINOX; frontline GemNab and cetuximab data are planned for a medical meeting in Q2 next year.