Cogent Biosciences reported that the full Part 2 data from its SUMMIT trial demonstrate that bezuclastinib delivers clinically meaningful symptom relief and robust reductions in disease biomarkers for patients with non‑advanced systemic mastocytosis. The 118‑patient study showed that 87.4 % of treated patients achieved a ≥50 % drop in serum tryptase, 75.6 % saw a ≥50 % reduction in bone‑marrow mast cells, and 85.7 % experienced a ≥50 % decline in KIT D816V variant allele frequency—outcomes that were statistically superior to placebo.
The trial’s primary endpoint, the Total Symptom Score, improved by an average of 24.3 points for bezuclastinib versus 15.4 points for placebo, confirming the drug’s impact on patient‑reported symptoms. Placebo rates for the secondary endpoints were 12.5 % for tryptase, 18.3 % for bone‑marrow mast cells, and 15.2 % for KIT D816V VAF, underscoring the magnitude of bezuclastinib’s effect relative to standard care.
Safety data reinforce the drug’s suitability for long‑term use. Ninety‑eight point three percent of all treatment‑emergent adverse events were low grade, with the most common being hair‑color change, altered taste, nausea, and mild ALT/AST elevations. Only 5.9 % of patients discontinued due to ALT/AST elevations, and all resolved without lasting impact, indicating a favorable tolerability profile for chronic therapy.
Cogent’s regulatory trajectory is clear: the company received Breakthrough Therapy Designation in October 2025 and plans to file a New Drug Application in December 2025. An investor webcast scheduled for December 8 will discuss the data and the upcoming filing, positioning bezuclastinib as a potential best‑in‑class treatment for a patient population that currently has no approved targeted options.
Management highlighted the trial’s significance, noting that the robust biomarker reductions and symptom improvements support the drug’s clinical value and the company’s confidence in meeting the FDA’s expectations. Cogent’s cash balance of over $390 million as of September 30, 2025 provides a strong runway to support the NDA submission and early commercialization activities.
The positive trial results and accelerated regulatory pathway are expected to strengthen Cogent’s competitive position in the rare‑disease market, potentially expanding its addressable market and setting the stage for future growth in related indications such as advanced systemic mastocytosis and gastrointestinal stromal tumors.
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