Business
Cogent Biosciences Announces FDA Breakthrough Therapy Designation for Bezuclastinib
Detailed results from bezuclastinib’s positive SUMMIT trial evaluating bezuclastinib in patients with NonAdvanced Systemic Mastocytosis planned for
About this update from Cogent Biosciences, Inc.
[{"type":"text","content":"Detailed results from bezuclastinib’s positive SUMMIT trial evaluating bezuclastinib in patients with NonAdvanced Systemic Mastocytosis planned for presentation at upcoming scientific conference this yearNDA submission for bezuclastinib planned by YE 2025 WALTHAM, Mass. and BOULDER, Colo., Oct. 20, 2025 (GLOBE NEWSWIRE) -- Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for bezuclastinib in NonAdvanced Systemic Mastocytosis (NonAdvSM) patients previously treated with avapritinib as well as in patients with Smoldering Systemic Mastocytosis; populations with no currently approved standard of care. “We are excited to announce Breakthrough Therapy Designation for bezuclastinib, which highlights the FDA’s recognition of the unmet need for patients with NonAdvanced Systemtic Mastocytosis and the potential for bezuclastinib to redefine the treatment paradigm for this disease,” said Andrew Robbins, Cogent’s President and Chief Executive Officer. “In addition, we recently completed a very productive pre-NDA meeting discussing the results from the SUMMIT pivotal trial and look forward to our continued collaboration with the FDA as we prepare to submit our NDA for NonAdvSM by the end of 2025. We are excited that this designation supports eligibility for Priority Review as we prepare for our planned commercial launch.” The Breakthrough Therapy Designation is based on positive results from the registration-directed SUMMIT trial in which bezuclastinib achieved statistical significance across all primary and key secondary endpoints in patients with NonAdvSM, including the consistent benefit observed in populations with high unmet need. Top-line data were announced in July 2025, and additional data are expected to be presented at an upcoming scientific conference. Breakthrough Therapy Designation is intended to expedite the review of medicines that treat a serious or life-threatening condition and have shown preliminary clinical evidence indicating the potential for substantial improvement over available therapies. The benefits of Breakthrough Therapy Designation include the eligibility for Priority Review, rolling submission of portions of the ...