Submission based on results of APEX pivotal trial which demonstrated bezuclastinib objective response rate (CR+CRh+PR+CI) of 65% per mIWG criteria and 81% ORR per PPR criteria with a well-tolerated safety profile for AdvSM patients
APEX NDA is third submission for bezuclastinib in six months; PEAK and SUMMIT reviews ongoing and on track for anticipated Q4 2026 approval
WALTHAM, Mass. and BOULDER, Colo., June 30, 2026 (GLOBE NEWSWIRE) -- Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, today announced it has submitted its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for bezuclastinib in Advanced Systemic Mastocytosis (AdvSM). The submission is based on positive clinical data from the APEX pivotal trial.
"We are pleased to announce the submission of our NDA for patients with Advanced Systemic Mastocytosis based on the impressive results from the APEX trial which demonstrated that a highly selective KIT mutant inhibitor like bezuclastinib can deliver clear clinical benefit without the severe tolerability challenges associated with currently available treatments," said Andrew Robbins, President and Chief Executive Officer. "We are working closely with the FDA on all three of our submissions and remain confident that bezuclastinib will achieve its first approvals later this year. I'd also like to congratulate our small, but exceptionally talented, Cogent development team, whose amazing work analyzing, presenting and submitting data from three pivotal trials in less than twelve months has rapidly positioned the company to achieve our vision of creating best-in-class therapies for patients fighting rare, mutational driven diseases."
The NDA submission is supported by data from the pivotal APEX trial, which were most recently presented at the 2026 European Hematology Association (EHA) Congress. As of the March 31, 2026 data cutoff, 81 AdvSM patients were treated with 150 mg of bezuclastinib, including 57 patients with SM-AHN, 11 patients with ASM and 13 patients with MCL. The primary endpoint of response per mIWG-MRT-ECNM was assessed on 68 evaluable patients and showed 65% ORR (CR+CRh+PR+CI), including 57% of patients who achieved CR, CRh or PR as best response.
Additional highlights included:
Key secondary endpoint of response per pure pathological response (PPR) criteria was assessed on 81 patients which showed an 81% ORR (CR+CRh+PR).
Bezuclastinib demonstrated reversal of bone marrow pathobiology including rapid and deep reductions in aberrant CD25 and CD30 expression, normalization of mast cell morphology, normalization of bone marrow cellularity, and improvement in myelofibrosis.
Bezuclastinib demonstrated durable clinical activity and prolonged PFS with a 12-month PFS rate of 79% and a 12-month OS rate of 87%. Median duration of PFS and OS were immature at the time of the data cutoff.
Bezuclastinib achieved clear and clinically significant reductions in objective disease markers for these AdvSM patients: