On January 20, 2026 Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, reported that the U.S. Food and Drug Administration (FDA) has agreed to accept its New Drug Application (NDA) for bezuclastinib in combination with sunitinib for patients with Gastrointestinal Stromal Tumors (GIST) who have received prior treatment with imatinib under the Real-Time Oncology Review (RTOR) program.
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"This milestone reflects the FDA’s recognition of the significant unmet need facing patients with imatinib resistant GIST," said Andrew Robbins, Cogent’s President and Chief Executive Officer. "Based on positive results from the PEAK trial, the bezuclastinib combination has the potential to be the first new approval in this patient population in over 20 years. We look forward to the continued, close collaboration with the FDA as we advance bezuclastinib toward commercialization."
As announced in November 2025, the bezuclastinib combination in the PEAK trial demonstrated a substantial and highly statistically significant clinical benefit on the primary endpoint of progression free survival (PFS), reducing risk of disease progression or death compared to the current standard of care by 50% (hazard ratio of 0.50, 95% CI: 0.39 – 0.65). mPFS, as assessed by blinded independent central review, was 16.5 months for the bezuclastinib combination vs. 9.2 months for sunitinib monotherapy. Additionally, the bezuclastinib combination demonstrated an unprecedented overall response rate (ORR) in imatinib-resistant patients, with 46% of patients treated with the bezuclastinib combination achieving an objective response compared to 26% of patients treated with sunitinib. At the time of this analysis, data for overall survival remains immature.
The bezuclastinib combination was generally well tolerated, and no unique risks were observed with the novel combination when compared to the known safety profile of sunitinib.
The FDA’s RTOR program allows an applicant to pre-submit components of its NDA to allow the FDA to review clinical trial data before the complete filing is submitted and aims to provide a more efficient review process to ensure safe and effective treatments are available to patients as early as possible. Cogent is expected to initiate the RTOR process immediately with completion of the NDA submission expected in April 2026.
Full results from the PEAK trial will be presented at a major medical meeting during the first half of 2026. Additionally, Cogent expects to initiate in mid-2026 a Phase 2 trial investigating the benefit of the bezuclastinib combination for first-line GIST patients with exon 9 mutations who are naive to, or recently initiated treatment with, imatinib.
(Press release, Cogent Biosciences, JAN 20, 2026, View Source [SID1234662090])