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DEC 30 2026
UPCOMING · 210 DAYS TO PDUFA

Cogent Biosciences bezuclastinib $COGT

for non-advanced systemic mastocytosis (NonAdvSM)

NDA · Standard Review

Accepted MAR 16 2026.

About

Systemic mastocytosis is a rare disorder where defective mast cells accumulate in tissues, causing unpredictable allergic-like reactions and organ damage. Bezuclastinib is a selective inhibitor that blocks the KIT D816V mutation—a faulty growth signal driving mast cell overproduction—by precisely targeting the malfunctioning protein. By shutting down this abnormal signal at its source, bezuclastinib may reduce mast cell burden and stabilize symptoms in patients whose current treatments fall short.

Pivotal trial

NCT05186753 Phase 2 active not recruiting NCT05186753

This is a multi-part, randomized, double-blind, placebo-controlled Phase 2 clinical study comparing the safety and efficacy of bezuclastinib (CGT9486) plus best supportive care (BSC) with placebo plus BSC in patients with nonadvanced systemic mastocytosis (NonAdvSM), including indolent systemic mastocytosis and smoldering systemic mastocytosis, whose symptoms are not adequately controlled by BSC. This study will be conducted in three parts

Source: ClinicalTrials.gov

Also in development