Cogent Biosciences Submits NDA for Bezuclastinib in AdvSM
Cogent Biosciences, Inc. announced the submission of its New Drug Application (NDA) to the U.S. FDA for bezuclastinib in Advanced Systemic Mastocytosis (AdvSM). This submission is supported by positive data from the APEX pivotal trial, which demonstrated a 65% objective response rate per mIWG criteria and an 81% ORR per PPR criteria, alongside a well-tolerated safety profile. The company anticipates approval in Q4 2026 for bezuclastinib, with two other submissions (PEAK and SUMMIT) also under review. Additionally, Cogent granted inducement equity awards to two new employees.
Key Highlights
- Submitted New Drug Application (NDA) to FDA for bezuclastinib in Advanced Systemic Mastocytosis (AdvSM).
- APEX pivotal trial showed 65% objective response rate (ORR) per mIWG criteria.
- APEX trial demonstrated 81% ORR per pure pathological response (PPR) criteria.
- Bezuclastinib achieved 89% reduction in serum tryptase and bone marrow mast cells.
- 91% reduction in KIT D816V variant allele frequency observed.
- 12-month progression-free survival (PFS) rate of 79% and overall survival (OS) rate of 87%.
- Bezuclastinib was well-tolerated with infrequent dose reductions or discontinuations.
- Granted 7,600 stock options and 7,000 restricted stock units to two new employees.
Price Impact
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