Cogent Biosciences Announces Positive Top-line Results Achieving Statistical Significance Across All Primary and Key Secondary Endpoints from the SUMMIT Trial of Bezuclastinib in Patients with Non-Advanced Systemic Mastocytosis

-- Patients treated with bezuclastinib showed a superior mean change in total symptom score at 24 weeks (-24.3 points vs. -15.4 points, -8.91 point placebo-adjusted difference; p=0.0002), compared to patients treated with placebo, establishing new benchmarks for placebo-adjusted and absolute symptomatic improvement for this patient population --

-- Bezuclastinib demonstrated a powerful effect on mast cell burden, with 87.4% of patients treated with bezuclastinib achieving at least 50% reduction in serum tryptase compared to 0% of patients treated with placebo --

-- Bezuclastinib demonstrated a favorable safety and tolerability profile supporting chronic use in this patient population --

-- Bezuclastinib NDA submission to FDA expected by end of 2025; strong financial position with $237 million current cash balance and access up to an additional $350 million via recently announced debt facility with SLR Capital Partners --

-- On track to share pivotal trial results from PEAK in GIST and APEX in AdvSM in 2H 2025 --

-- Cogent to host investor conference call and webcast today at 8:00 a.m. ET --

WALTHAM, Mass. and BOULDER, Colo., July 07, 2025 (GLOBE NEWSWIRE) -- Cogent Biosciences, Inc. (NASDAQ: COGT) today announced positive top-line results from the registration-directed Part 2 of the SUMMIT clinical trial of bezuclastinib in patients with non-advanced systemic mastocytosis (NonAdvSM) demonstrating clinically meaningful and highly statistically significant improvements across the primary and all key secondary endpoints, including patient-reported symptoms and objective measures of mast cell burden. Based on these data, Cogent is on track to submit its first new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for bezuclastinib in NonAdvSM by the end of 2025. In addition, Cogent plans to present detailed results from the SUMMIT trial at an upcoming medical meeting later this year.

“We have been eagerly awaiting this day and are thrilled to announce bezuclastinib’s performance in the SUMMIT trial, demonstrating clinically meaningful and statistically significant results across all trial endpoints,” said Andrew Robbins, Cogent’s President and CEO. “Our team is already at work on our first New Drug Application for bezuclastinib that we expect to file with the FDA later this year. We are committed to providing bezuclastinib access to the thousands of patients with Non-Advanced Systemic Mastocytosis as quickly as possible, including through our recently announced Bezuclastinib Expanded Access Program.”

The SUMMIT trial, which was designed to assess the clinical benefit of bezuclastinib versus placebo, achieved its primary endpoint with a highly statistically significant difference in the mean change in total symptom score (TSS) at 24 weeks (p=0.0002). TSS was assessed by the Mastocytosis Symptom Severity Daily Diary (MS2D2). The bezuclastinib arm had a mean reduction of 24.3 points in TSS at 24 weeks, versus the placebo arm which had a mean reduction of 15.4 points in TSS, resulting in a placebo-adjusted TSS improvement of 8.91 points. In addition, the SUMMIT trial demonstrated highly statistically significant benefit across all key secondary endpoints, including reduction of serum tryptase on which 87.4% of bezuclastinib-treated patients had ≥50% reduction, compared to no patients in the control arm (87.4% vs. 0%; p