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DEC 30 2026
UPCOMING · 152 DAYS TO PDUFA

Cogent Biosciences bezuclastinib $COGT

for non-advanced systemic mastocytosis (NonAdvSM)

NDA · Standard Review

Accepted MAR 16 2026.

About

Non-advanced systemic mastocytosis is a rare disorder where defective mast cells accumulate in the body, often causing severe and unpredictable allergic-type symptoms. Bezulcastinib is a small molecule drug designed to selectively block the activity of the mutated KIT D816V protein that drives this abnormal mast cell growth. By targeting this specific mutation, the drug could potentially reduce the burden of defective cells and alleviate the debilitating symptoms of the disease.

Pivotal trial

NCT04996875 Phase 2 recruiting NCT04996875

This is an open-label, two-part Phase 2 study investigating CGT9486 for the treatment of patients with Advanced Systemic Mastocytosis (AdvSM), including patients with Aggressive SM (ASM), SM with Associated Hematologic Neoplasm (SM-AHN), and Mast Cell Leukemia (MCL).

Source: ClinicalTrials.gov

Also in development