Cogent Biosciences, Inc. (NASDAQ: COGT) has released positive top-line results from the Summit trial of bezuclastinib in patients with non-advanced systemic mastocytosis (nonadvsm). The results show statistically significant improvements across primary and key secondary endpoints.
The primary endpoint, which measured the mean change in total symptom score (TSS) at 24 weeks, demonstrated a highly statistically significant difference (p=0.0002). Patients treated with bezuclastinib showed a superior mean change in TSS compared to those treated with a placebo. The mean reduction of TSS at 24 weeks in the bezuclastinib arm was 24.3 points, compared to 15.4 points in the placebo arm, resulting in a placebo-adjusted TSS improvement of 8.91 points.
Furthermore, the trial showed highly statistically significant benefit across all key secondary endpoints, including a reduction in serum tryptase. Specifically, 87.4% of patients treated with bezuclastinib achieved at least a 50% reduction in serum tryptase compared to 0% of patients treated with a placebo (p<0.0001).
The majority of treatment emergent adverse events (TEAEs) were of low grade, with hair color change, altered taste, nausea, and ALT/AST elevations being the most frequent TEAEs reported on bezuclastinib treatment.
Andrew Robbins, President and CEO of Cogent Biosciences, expressed excitement about the results, stating that the company is committed to providing access to bezuclastinib to patients with non-advanced systemic mastocytosis as quickly as possible, including through an expanded access program.
The company expects 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. Additionally, Cogent plans to present detailed results from the Summit trial at an upcoming medical meeting later this year.
Cogent Biosciences also announced that it remains on track to provide top-line results from both the Peak and Apex trials during the second half of 2025.
Bezuclastinib is a selective tyrosine kinase inhibitor designed to potently inhibit the KIT D816V mutation as well as other mutations in KIT exon 17. It is aimed at addressing serious diseases caused by unchecked proliferation of mast cells, such as systemic mastocytosis and gastrointestinal stromal tumors.
The company is focused on developing precision therapies for genetically defined diseases and has ongoing research into a portfolio of novel targeted therapies to help patients fighting serious, genetically driven diseases.
Cogent Biosciences, Inc. is based in Waltham, MA, and Boulder, CO.
The company's current cash balance is $237 million, with access to up to an additional $350 million via a recently announced debt facility with SLR Capital Partners. The market has reacted to these announcements by moving the company's shares 6.34% to a price of $10.40. For the full picture, make sure to review Cogent Biosciences's 8-K report.
