SHERIDAN, WYOMING – July 8, 2025 – Cogent Biosciences is accelerating its strategic trajectory with plans to submit its first New Drug Application (NDA) to the FDA by year-end, following strong Phase II data for its investigational tyrosine kinase inhibitor, bezuclastinib. The move positions Cogent as a serious contender in the systemic mastocytosis treatment landscape, directly challenging Blueprint Medicines' market presence.
Strategic Differentiation in a Competitive Field
The Phase II SUMMIT trial showed that bezuclastinib achieved its primary endpoint, significantly improving total symptom scores compared to placebo. The treatment arm recorded a mean reduction of 24.3 points, versus 15.4 points in the placebo arm, based on a proprietary scoring system developed by Cogent. Secondary endpoints were also met, with 87.4% of patients experiencing at least a 50% reduction in serum tryptase levels—a key biomarker for mastocytosis—while no such reduction was observed in the placebo group.
Analysts at Leerink Partners emphasized the drug’s competitive positioning, stating, “We continue to believe that [bezuclastinib] has a meaningful [systemic mastocytosis] opportunity by first addressing the ~20–30% of higher symptom burden patients not adequately treated by Ayvakit.”
Market Implications and Clinical Relevance
Cogent’s announcement significantly boosted investor confidence, driving a 26% surge in its share price on Monday. The SUMMIT trial’s outcome is poised to reshape how unmet needs in systemic mastocytosis are addressed, particularly for patients unresponsive or intolerant to Blueprint’s Ayvakit, which received FDA approval in May 2023 for a subset of patients.
In a high-profile comparison, Cogent CEO Andy Robbins underscored the safety concerns surrounding Ayvakit at the recent Jefferies Global Healthcare Conference, stating it “was so toxic that it was killing patients,” as reported by STAT.
Favorable Safety Profile Enhances Marketability
Bezuclastinib’s safety profile could provide a crucial edge in market adoption. Reported side effects in the SUMMIT trial included changes in hair color (69.5%), altered taste (23.7%), and elevated liver enzymes (22.0%), but lacked the more severe complications associated with Ayvakit, such as edema, cognitive effects, and hemorrhagic events.
Leerink analysts attributed this favorable profile to the drug’s “higher selectivity and lack of brain penetrance,” suggesting improved tolerability may translate into broader clinical adoption.
Commercial Outlook and Forward Path
The company plans to present a more comprehensive dataset later this year at a leading medical conference, further supporting its NDA submission strategy. Leerink’s financial forecast places peak revenue expectations for bezuclastinib at approximately $2.5 billion, underscoring the drug’s commercial potential.
“In total, we model ~$2.5bn for peak [bezuclastinib’s] revenues,” concluded the analysts, signaling high expectations for the product’s market performance if regulatory approval is secured.
Learn more at www.cogentbio.com