Cogent Biosciences' bezuclastinib shows 65% ORR in AdvSM; NDA in June 2026
Cogent Biosciences announced updated positive clinical results from its APEX trial for bezuclastinib in advanced systemic mastocytosis (AdvSM) patients, showing a 65% objective response rate (ORR) per mIWG criteria and 81% ORR per PPR criteria. The drug also demonstrated significant reductions in mast cell burden and a favorable safety profile. The company plans to submit the New Drug Application (NDA) for bezuclastinib in June 2026 and aims for a launch later this year for both systemic mastocytosis and GIST, following FDA approval.
Key Highlights
- APEX NDA for bezuclastinib in AdvSM to be submitted in June 2026.
- Bezuclastinib achieved 65% ORR (CR+CRh+PR+CI) per mIWG criteria in AdvSM patients.
- Bezuclastinib achieved 81% ORR per PPR criteria in AdvSM patients.
- 91% of patients achieved ">=50%" reduction in KIT D816V variant allele frequency.
- 89% of patients achieved ">=50%" reduction in bone marrow mast cells or clearance of aggregates.
- 12-month PFS rate was 79% and 12-month OS rate was 87%.
- Most frequent TRAEs included hair color change (31%), neutropenia (31%), altered taste (28%), thrombocytopenia (25%), and ALT/AST elevations (21%).
Price Impact
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