On Monday, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation (BTD) to Cogent Biosciences, Inc.’s (NASDAQ:COGT) bezuclastinib in combination with sunitinib for patients with Gastrointestinal Stromal Tumors (GIST).
The trial included patients who received prior treatment with Novartis AG’s (NYSE:NVS) Gleevac (imatinib).
Pfizer Inc. (NYSE:PFE) sells sunitinib under the brand Sutent.
Bezuclastinib’s Efficacy Outshines Current Standards
This Breakthrough Therapy Designation is based on results from the PEAK trial, which 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%.
Median PFS was 16.5 months for the bezuclastinib combination vs. 9.2 months for sunitinib monotherapy.
The combination was well tolerated, and no new safety risks were observed when compared to the known safety profile of sunitinib.
Earlier this month, the FDA agreed to accept Cogent’s bezuclastinib plus sunitinib combo regimen New Drug Application (NDA) under the FDA’s Real-Time Oncology Review (RTOR) program.
The 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.
What Next?
Cogent is expected to initiate the RTOR process immediately, with completion of the NDA submission expected in April 2026.
Cogent plans to present full results from the PEAK trial at a major medical meeting during the first half of 2026.
Additionally, in mid-2026, Cogent expects to initiate a Phase 2 trial of the bezuclastinib plus sunitinib combination for first-line GIST patients with exon 9 mutations who are naive to, or recently initiated treatment with, imatinib.
In December 2025, Cogent Biosciences submitted its NDA to the FDA for bezuclastinib in Non-Advanced Systemic Mastocytosis.
The submission is based on clinical data from the SUMMIT pivotal trial and follows the Breakthrough Therapy Designation for bezuclastinib in patients with SSM and patients with NonAdvSM who have received prior Sanofi SA’s (NASDAQ:SNY) Ayvakit (avapritinib).
Price Action: COGT stock is up 1.87% at $37.35 at the last check on Monday, according to Benzinga Pro data.
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