Cogent Biosciences submits NDA for bezuclastinib in NonAdvSM

Cogent Biosciences (COGT) submitted its New Drug Application to the U.S. Food and Drug Administration for bezuclastinib in NonAdvanced Systemic Mastocytosis. The submission is based on positive clinical data from the SUMMIT pivotal trial and follows the Breakthrough Therapy Designation for bezuclastinib in patients with SSM and patients with NonAdvSM who have received prior avapritinib. As reported first in July, and more recently at the ASH annual meeting, the SUMMIT trial of bezuclastinib in patients with NonAdvSM achieved statistical significance across all primary and key secondary endpoints. Bezuclastinib demonstrated clear clinical benefit across all symptom domains, including significant improvements across 11 individual patient reported symptoms as well as the most severe symptom at baseline. Reductions in objective measures of disease, including serum tryptase, correlated with improvements in symptom severity, representing the first time this relationship has been demonstrated in patients with NonAdvSM. Updated data from the SUMMIT trial through 48 weeks showcased a clear and continued deepening of symptomatic improvement over time, supporting the potential for sustained clinical benefit with longer duration of therapy. Across the SUMMIT trial, bezuclastinib demonstrated a favorable safety and tolerability profile, supporting its potential for chronic use in patients with NonAdvSM.