The U.S. Food and Drug Administration (FDA) has awarded Breakthrough Therapy Designation to Cogent Biosciences, Inc. for its innovative treatment combining bezuclastinib with sunitinib for patients with Gastrointestinal Stromal Tumors (GIST). This designation follows promising results from the PEAK trial, where the combination therapy significantly reduced the risk of disease progression or death by 50% compared to the current standard treatment.
The PEAK trial demonstrated a marked improvement in progression-free survival (PFS), with a median PFS of 16.5 months for the bezuclastinib and sunitinib regimen, compared to just 9.2 months for patients receiving sunitinib alone. Notably, the combination therapy was well tolerated, showing no new safety risks compared to the known profile of sunitinib, which is marketed by Pfizer Inc. under the brand name Sutent.
Regulatory Path and Future Trials
Earlier this month, the FDA accepted Cogent’s New Drug Application (NDA) for the bezuclastinib and sunitinib combination under the Real-Time Oncology Review (RTOR) program. This program allows for the pre-submission of components of the NDA, enabling the FDA to review clinical trial data before the complete filing is submitted. Cogent plans to initiate the RTOR process immediately, with the NDA submission expected to be finalized by April 2026.
Additionally, Cogent intends to share full results from the PEAK trial at a significant medical meeting in the first half of 2026. The company is also preparing to launch a Phase 2 trial of the combination for first-line GIST patients with exon 9 mutations who are either new to treatment or have recently started on imatinib.
In December 2025, Cogent submitted an NDA for bezuclastinib in the treatment of Non-Advanced Systemic Mastocytosis, building on clinical data from the SUMMIT pivotal trial. This submission follows the previous Breakthrough Therapy Designation for bezuclastinib in patients with Systemic Mastocytosis and Non-Advanced Systemic Mastocytosis who have received prior treatment with Sanofi SA’s Ayvakit (avapritinib).
Market Response
Following the announcement of the Breakthrough Therapy Designation, shares of Cogent Biosciences saw an increase of 1.87%, bringing the stock price to $37.35, according to Benzinga Pro data. The positive market response reflects investor confidence in Cogent’s potential to significantly impact the treatment landscape for GIST patients.
As the company moves forward with its plans, the medical community and patients alike are hopeful that this new treatment will deliver improved outcomes and offer a new avenue of hope for those battling this challenging form of cancer.
