Cogent Biosciences Secures FDA Breakthrough Therapy Status For Bezuclastinib + Sunitinib Combo To Treat Gastrointestinal Stromal Tumors (GIST)

Cogent Biosciences, Inc., a biotechnology company focused on creating precision therapies for genetically defined diseases, announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for bezuclastinib when used together with sunitinib. This designation applies to patients with Gastrointestinal Stromal Tumors (GIST) who have previously been treated with imatinib and now require more effective treatment options.

Andrew Robbins, President and Chief Executive Officer of Cogent Biosciences, stated that the company is pleased to receive this designation, as it reflects the strong potential of the bezuclastinib and sunitinib combination therapy. According to him, this recognition underscores the possibility that the combination could offer significantly better outcomes for patients whose GIST has become resistant to imatinib. He added that the company looks forward to continued collaboration with the FDA as they work toward bringing a new treatment option to this patient group for the first time in over two decades.

The FDA’s decision is supported by data from the PEAK trial, which showed a substantial clinical advantage for the combination therapy. The study demonstrated a strong and statistically significant improvement in progression-free survival, reducing the risk of disease progression or death by 50 percent compared to the current standard treatment. The median progression-free survival, based on an independent central review, was 16.5 months for patients receiving the bezuclastinib combination, compared with 9.2 months for those receiving sunitinib alone. The combination was well tolerated in the trial, and no new safety concerns emerged beyond the known side effects associated with sunitinib. Breakthrough Therapy Designation is granted to medicines that show early clinical evidence of offering a meaningful improvement over available therapies for serious or life-threatening conditions.

Earlier this month, the FDA also agreed to review Cogent’s New Drug Application under the Real-Time Oncology Review program. This program enables companies to submit parts of their application early so that the FDA can begin reviewing clinical data before the full application is submitted. This approach is designed to streamline the review process and make effective treatments available to patients sooner.

Cogent plans to present the full results from the PEAK trial at a major medical meeting in the first half of 2026. The company also expects to launch a Phase 2 trial in mid-2026 to evaluate the benefits of the bezuclastinib and sunitinib combination as a first-line treatment for GIST patients with exon 9 mutations, including those who have not yet been treated with imatinib or who have only recently begun imatinib therapy.