Cogent Biosciences宣布Bezuclastini治疗非晚期系统性肥大细胞增多症的SUMMIT试验第2部分的总体结果，证明主要终点和所有关键次要终点均具有临床意义且具有高度统计学意义的改善

2025-07-07 19:09

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