Verastem Oncology has put itself into the center of the KRAS race. Initial data from its Phase 1/2 TARGET-D 101 study of VS-7375, an oral inhibitor of the KRAS G12D mutation licensed from China’s GenFleet Therapeutics, showed confirmed responses in half of a small group of previously treated pancreatic cancer patients, and the company paired the readout with a roughly $250 million stock offering to fund late stage development. Shares rose about 16 to 18 percent on October 14.
The numbers need their denominators kept attached. At the 900 milligram daily dose, four of eight second line pancreatic patients responded. Across 22 pancreatic patients treated in the second line or later, the confirmed response rate was 41 percent, including two complete responses, with a disease control rate of 86 percent, six month progression free survival of 67 percent and six month overall survival of 80 percent, with 16 of the 22 still on treatment. In third and fourth line patients the response rate was 36 percent. In lung cancer, 43 percent of 23 patients on the 600 milligram dose responded. The trial has enrolled about 295 patients across pancreatic, colorectal and lung tumors. Side effects were mainly gastrointestinal and the drug was generally well tolerated at the doses reported.
The comparison driving investor interest is Revolution Medicines’ Rasonque, approved in August for metastatic pancreatic cancer. Its overall second line response rate was in the mid 30 percent range, but regulatory review documents showed the G12D subgroup, about 43 percent of its pivotal trial, responded at only 24 percent. G12D accounts for roughly 26 percent of all KRAS mutations, the largest single share. A G12D selective drug that holds up in larger cohorts would attack the incumbent exactly where it is weakest.
Verastem priced about 30.94 million shares at $8 in the accompanying offering, a premium to the prior close, adding to $136.4 million of cash and investments held at the end of June. The company plans a Phase 3 pancreatic trial by the end of 2026 and further late stage studies in lung and colorectal cancers, and announced a chief executive transition, with Michael Bailey taking over on January 1, 2027. Single arm Phase 1/2 response rates in pancreatic cancer have flattered drugs before; the Phase 3 design, where Verastem says it has aligned with the FDA, is where this claim will be tested.