Verastem Oncology's Investigational Drug Delivers Encouraging Responses Across Multiple Cancers
Verastem Oncology (NASDAQ:VSTM) reported encouraging early clinical results for its experimental KRAS G12D inhibitor VS-7375, with the drug demonstrating antitumor activity across pancreatic, colorectal, and lung cancers, alongside a favorable safety profile.
The company also said it aligned with the U.S. Food and Drug Administration (FDA) on the design of a Phase 3 trial evaluating the drug in first-line metastatic pancreatic cancer.
VS-7375 Shows Promising Results In Pancreatic Cancer
In the ongoing TARGET-D 101 Phase 1/2 trial, VS-7375 demonstrated a 41% confirmed objective response rate (ORR) among 22 patients with metastatic pancreatic ductal adenocarcinoma (mPDAC) treated with the 900 mg once-daily monotherapy dose in the second-line and later settings.
The results included one confirmed complete response and another unconfirmed complete response.
The confirmed response rate reached 50% in second-line patients and 36% among those treated in the third- and fourth-line settings. The disease control rate was 86%, while six-month progression-free survival and overall survival rates stood at 67% and 80%, respectively.
The company said the 900 mg dose produced deeper tumor reductions and higher response rates than the 600 mg dose, supporting its selection for further evaluation.
Biomarker findings also showed that all 12 evaluable pancreatic cancer patients with detectable KRAS G12D circulating tumor DNA at baseline achieved at least a 50% reduction in variant allele frequency.
Seven patients achieved complete clearance of detectable KRAS G12D circulating tumor DNA.
Phase 3 Pancreatic Trial Results Extend To Other Cancers
Verastem plans to initiate a Phase 3 study by year-end 2026, comparing two VS-7375 combination regimens against standard-of-care chemotherapy in first-line mPDAC. The experimental arms will evaluate VS-7375 with an anti-EGFR therapy and VS-7375 with gemcitabine and nab-paclitaxel.
In third-line and later metastatic colorectal cancer, VS-7375 at 900 mg combined with an anti-EGFR therapy produced a 33% ORR among six patients, including one confirmed and one unconfirmed response.
In advanced non-small cell lung cancer, the 600 mg monotherapy dose achieved a 43% confirmed ORR among 23 patients treated in the second-line and later settings.
In a separate first-line lung cancer cohort, the drug combined with standard treatment achieved a 75% ORR among eight patients.
Safety Profile Supports Further Development
VS-7375 maintained a manageable safety profile across monotherapy and combination regimens at both 600 mg and 900 mg. The company continues evaluating the higher dose across tumor types and combination settings.
Verastem said it aligned with the FDA on a three-arm Phase 3 study evaluating VS-7375 combinations and standard chemotherapy in first-line pancreatic cancer. The company plans to initiate the trial by year-end 2026.
CEO Transition And Stock Offering
Separately, Verastem appointed Michael Bailey president and CEO, effective January 1, 2027. He will succeed Daniel Paterson, who plans to retire but remain on the board and serve as an adviser.
The company also priced an offering of 30.937 million shares at $8 each, alongside pre-funded warrants for up to 312,503 additional shares at $7.9999 apiece.
Gross proceeds of approximately $250 million will support clinical development, including VS-7375, working capital, and general corporate purposes.
VSTM Price Action: Verastem shares were up 19.33% at $8.89 during premarket trading on Wednesday, according to Benzinga Pro data.
Photo via Shutterstock