1. Cogent submitted NDA for bezuclastinib for NonAdvSM treatment to the FDA. 2. The drug shows significant clinical benefits based on the SUMMIT trial data. 3. Breakthrough Therapy Designation was awarded for addressing unmet medical needs. 4. Two additional NDA submissions for bezuclastinib are planned for 2026. 5. Bezuclastinib exhibits favorable safety, supporting its chronic use potential.
FAQ
Why Very Bullish?
The positive NDA submission and trial results create strong investor confidence, reminiscent of other biotech gains after successful drug applications, such as Amgen's Enbrel approval boosting its stock significantly.
How important is it?
The article presents key developments that directly pertain to COGT's stock valuation and future revenue potential, significantly influencing investor sentiment.
Why Long Term?
Approval could lead to a sustainable revenue stream from bezuclastinib and additional submissions, similar to how Gilead experienced lasting market impact after launching successful therapies.
Cogent Biosciences Submits New Drug Application for Bezuclastinib
WALTHAM, Mass. and BOULDER, Colo., December 30, 2025 (GLOBE NEWSWIRE) -- Cogent Biosciences, Inc. (Nasdaq: COGT), a leading biotechnology company specializing in precision therapies for genetically defined diseases, has announced the submission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for bezuclastinib in the treatment of NonAdvanced Systemic Mastocytosis (NonAdvSM).
Details of the NDA Submission
The NDA submission is grounded in the successful clinical data from the SUMMIT pivotal trial, which supported the therapy's efficacy. This filing also follows the FDA's Breakthrough Therapy Designation received earlier for patients with systemic mastocytosis and NonAdvSM who have previously received avapritinib.
Andrew Robbins, Cogent’s President and Chief Executive Officer, commented, “This NDA is the first of three planned submissions for bezuclastinib based on positive clinical data from three pivotal trials completed in 2025 for patients with systemic mastocytosis and gastrointestinal stromal tumors (GIST). Building on the exceptional results from the SUMMIT trial, this filing moves us closer to delivering an important disease-modifying therapy to patients with NonAdvSM.”
Significant Clinical Data from the SUMMIT Trial
The SUMMIT trial demonstrated statistical significance across all primary and key secondary endpoints for bezuclastinib, showcasing its clinical benefits across multiple symptom domains. Notably, significant improvements were observed in 11 individual patient-reported symptoms and a substantial reduction in the most severe symptom at baseline.
- Clear correlation of reductions in disease biomarkers such as serum tryptase with symptom improvements
- Data through 48 weeks indicated sustained symptomatic improvement, suggesting potential for long-term efficacy
- Favorable safety and tolerability profile indicating suitability for chronic use in patients with NonAdvSM
Regulatory Milestones and Future Plans
This submission is part of Cogent's strategy to advance bezuclastinib, which was granted Breakthrough Therapy Designation by the FDA in October 2025. The company plans to submit additional NDAs for bezuclastinib targeting GIST and Advanced Systemic Mastocytosis (AdvSM) in the first half of 2026.
Robbins emphasized, “We extend our deep appreciation to the patients, families, clinicians, collaborators, and our Cogent team, all of whom helped make this possible.”
About Cogent Biosciences
Cogent Biosciences specializes in creating innovative therapies tailored for genetically defined diseases. Its lead product, bezuclastinib, serves as a selective tyrosine kinase inhibitor targeting the KIT D816V mutation, which is pivotal in systemic mastocytosis. The company is also advancing other targeted therapies for conditions driven by various genetic mutations, including those in ErbB2, PI3Ka, KRAS, and JAK2.
Cogent Biosciences operates from Waltham, MA, and Boulder, CO. More information is available on their official website at www.cogentbio.com.
Forward-Looking Statements
This article contains forward-looking statements under the Private Securities Litigation Reform Act of 1995 regarding Cogent Biosciences' operational and strategic initiatives. These statements are subject to various risks and uncertainties.
Investors should refer to Cogent’s most recent Quarterly Report on Form 10-Q filed with the SEC for a comprehensive discussion of potential risks affecting future performance.
Contact Information
For more details, please contact:
Christi Waarich
Senior Director, Investor Relations
Email: christi.waarich@cogentbio.com
Phone: 617-830-1653