Cogent Biosciences has submitted its New Drug Application (NDA) to the US Food and Drug Administration (FDA) for bezuclastinib in NonAdvanced Systemic Mastocytosis (NonAdvSM). The submission is based on positive clinical data from the SUMMIT pivotal trial and follows the Breakthrough Therapy Designation (BTD) for bezuclastinib in patients with SSM and patients with NonAdvSM who have received prior avapritinib.

“This NDA is the first of three planned submissions for bezuclastinib based on positive clinical data from three pivotal trials completed in 2025 for patients with systemic mastocytosis and GIST. Building on the exceptional results from the SUMMIT trial, this filing moves us closer to delivering an important disease-modifying therapy to patients with NonAdvSM. We extend our deep appreciation to the patients, families, clinicians, collaborators, and our Cogent team, who all helped make this possible,” said Andrew Robbins, President and Chief Executive Officer, Cogent.

As reported first in July 2025, 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 favourable safety and tolerability profile, supporting its potential for chronic use in patients with NonAdvSM.

Bezuclastinib was granted Breakthrough Therapy Designation by the FDA in October 2025, reflecting the agency’s recognition of its potential to address a significant unmet medical need.

NDA submissions for bezuclastinib in Gastrointestinal Stromal Tumours (GIST) and Advanced Systemic Mastocytosis (AdvSM) are on-track for the first half of 2026 based on the strength of clinical results from the PEAK and APEX clinical trials as reported in Q4 2025.