Cogent Biosciences Files Bezuclastinib NDA After 65% APEX ORR
COGT•Cogent Biosciences has submitted its third New Drug Application for bezuclastinib in Advanced Systemic Mastocytosis, following APEX trial results showing a 65% objective response rate per mIWG criteria and 81% per PPR. The submission complements ongoing PEAK and SUMMIT reviews targeting Q4 2026 approvals.
1. NDA Submission Based on APEX Trial
On June 30, Cogent Biosciences submitted its New Drug Application to the FDA for bezuclastinib in Advanced Systemic Mastocytosis. This marks the company’s third NDA in six months, following the PEAK and SUMMIT submissions still under review.
2. Efficacy Data from APEX Trial
The APEX pivotal trial treated 81 AdvSM patients with 150 mg of bezuclastinib, showing a 65% objective response rate (CR+CRh+PR+CI) among 68 evaluable patients per mIWG criteria and an 81% ORR per PPR criteria. The 12-month progression-free survival rate reached 79% and overall survival rate was 87%, although median values are immature.
3. Safety Profile and Expanded Access
Bezuclastinib demonstrated a favorable tolerability profile with infrequent high-grade treatment-related adverse events; most common events included hair color change (31%), neutropenia (31%) and altered taste (28%). An Expanded Access Program is active for eligible systemic mastocytosis and GIST patients.
4. Regulatory Outlook and Next Steps
Cogent is on track for potential de novo approvals of bezuclastinib in late 2026, pending FDA decisions on all three NDAs. A successful outcome would establish bezuclastinib as a precision therapy for genetically driven mast cell diseases.
