Cogent Biosciences Announces Submission of New Drug Application for Bezuclastinib in NonAdvanced Systemic Mastocytosis
Rhea-AI Summary
Cogent Biosciences (Nasdaq: COGT) submitted a New Drug Application (NDA) to the FDA on Dec. 30, 2025 for bezuclastinib in NonAdvanced Systemic Mastocytosis based on positive SUMMIT pivotal-trial data.
The filing follows an FDA Breakthrough Therapy designation (Oct 2025) and 48-week SUMMIT results showing statistical significance across all primary and key secondary endpoints, symptom improvements across 11 reported domains, correlated reductions in serum tryptase, and a favorable safety/tolerability profile. Cogent plans two additional NDA submissions for bezuclastinib in GIST and Advanced SM in the first half of 2026 based on PEAK and APEX trial data.
Positive
- NDA submitted to FDA on Dec 30, 2025
- SUMMIT achieved statistical significance on all primary and key secondary endpoints
- Symptom improvement across 11 patient-reported domains through 48 weeks
- Correlated reductions in serum tryptase with symptom improvement
- FDA Breakthrough Therapy designation granted in Oct 2025
Negative
- NDA submission is not an approval; regulatory review timeline and outcome are unspecified
- Additional NDAs for GIST and AdvSM are planned but pending data review in H1 2026
News Market Reaction
On the day this news was published, COGT declined 2.11%, reflecting a moderate negative market reaction. Argus tracked a trough of -3.4% from its starting point during tracking. Our momentum scanner triggered 15 alerts that day, indicating notable trading interest and price volatility. This price movement removed approximately $123M from the company's valuation, bringing the market cap to $5.70B at that time.
Data tracked by StockTitan Argus on the day of publication.
Key Figures
Market Reality Check
Peers on Argus
While COGT was down 2.47% pre‑announcement, key biotech peers like CLDX, AUPH, DYN and IMCR showed smaller declines between 0.21% and 1.91%, suggesting more stock‑specific weakness than a broad sector move.
Historical Context
| Date | Event | Sentiment | Move | Catalyst |
|---|---|---|---|---|
| Dec 08 | AdvSM trial data | Positive | +4.0% | Positive APEX Part 2 AdvSM data with high response rates and good safety. |
| Nov 14 | Conference participation | Neutral | -0.9% | Announcement of presentation slot at Jefferies Global Healthcare Conference. |
| Nov 11 | Financing priced | Negative | +3.0% | Pricing of upsized equity and convertible notes offerings for funding needs. |
| Nov 10 | Financing proposed | Negative | +119.0% | Proposed concurrent common stock and convertible notes offerings announcement. |
| Nov 10 | GIST Phase 3 data | Positive | +119.0% | Positive PEAK Phase 3 GIST results with improved PFS and ORR vs sunitinib. |
Stock has generally reacted positively to strong bezuclastinib data, while financing announcements showed mixed, sometimes divergent, reactions.
Over the past few months, Cogent advanced bezuclastinib across multiple indications. On Nov 10, 2025, positive Phase 3 PEAK GIST data drove a 119.03% move. This was followed by proposed and then priced equity and convertible note offerings in mid‑November, alongside a Q3 10‑Q detailing higher R&D spend and strengthened liquidity. On Dec 8, 2025, positive APEX AdvSM data lifted shares 3.95%. Today’s NDA submission in NonAdvSM builds on this sequence of pivotal readouts and financing for anticipated commercialization.
Market Pulse Summary
This announcement details submission of an NDA for bezuclastinib in NonAdvSM, grounded in the SUMMIT pivotal trial, which met all primary and key secondary endpoints and showed improvement across 11 symptom measures over 48 weeks. It follows FDA Breakthrough Therapy Designation in October 2025 and prior positive Phase 3 results in GIST and AdvSM. Investors may watch for FDA review milestones, additional NDA filings targeted for 1H 2026, and how these programs integrate with the company’s recently expanded balance sheet.
Key Terms
new drug application regulatory
nda regulatory
u.s. food and drug administration regulatory
fda regulatory
breakthrough therapy designation regulatory
systemic mastocytosis medical
gastrointestinal stromal tumors medical
serum tryptase medical
AI-generated analysis. Not financial advice.
WALTHAM, Mass. and BOULDER, Colo., Dec. 30, 2025 (GLOBE NEWSWIRE) -- Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, today announced it has submitted its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for bezuclastinib in NonAdvanced Systemic Mastocytosis (NonAdvSM). The submission is based on positive clinical data from the SUMMIT pivotal trial and follows the Breakthrough Therapy Designation for bezuclastinib in patients with SSM and patients with NonAdvSM who have received prior avapritinib.
“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 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,” said Andrew Robbins, Cogent’s President and Chief Executive Officer. “We extend our deep appreciation to the patients, families, clinicians, collaborators, and our Cogent team, who all helped make this possible.”
As reported first in July 2025, and more recently at the ASH annual meeting, the SUMMIT trial of bezuclastinib in patients with NonAdvSM achieved statistical significance across all primary and key secondary endpoints.
Bezuclastinib demonstrated clear clinical benefit across all symptom domains, including significant improvements across 11 individual patient reported symptoms as well as the most severe symptom at baseline. Reductions in objective measures of disease, including serum tryptase, correlated with improvements in symptom severity, representing the first time this relationship has been demonstrated in patients with NonAdvSM.
Updated data from the SUMMIT trial through 48 weeks showcased a clear and continued deepening of symptomatic improvement over time, supporting the potential for sustained clinical benefit with longer duration of therapy. Across the SUMMIT trial, bezuclastinib demonstrated a favorable safety and tolerability profile, supporting its potential for chronic use in patients with NonAdvSM.
Bezuclastinib was granted Breakthrough Therapy Designation by the FDA in October 2025, reflecting the agency’s recognition of its potential to address a significant unmet medical need.
NDA submissions for bezuclastinib in Gastrointestinal Stromal Tumors (GIST) and Advanced Systemic Mastocytosis (AdvSM) are on-track for the first half of 2026 based on the strength of clinical results from the PEAK and APEX clinical trials as reported in Q4 2025.
About Cogent Biosciences, Inc.
Cogent Biosciences is a biotechnology company focused on developing precision therapies for genetically defined diseases. The most advanced clinical program, bezuclastinib, is a selective tyrosine kinase inhibitor that is designed to potently inhibit the KIT D816V mutation as well as other mutations in KIT exon 17. KIT D816V is responsible for driving systemic mastocytosis, a serious disease caused by unchecked proliferation of mast cells. Exon 17 mutations are also found in patients with advanced gastrointestinal stromal tumors (GIST), a type of cancer with strong dependence on oncogenic KIT signaling. The company also has an ongoing Phase 1 study of its novel internally discovered FGFR2/3 inhibitor. In addition, the Cogent Research Team is developing a portfolio of novel targeted therapies to help patients fighting serious, genetically driven diseases targeting mutations in ErbB2, PI3Ka, KRAS and JAK2. Cogent Biosciences is based in Waltham, MA and Boulder, CO. Visit our website for more information at www.cogentbio.com. Follow Cogent Biosciences on social media: X (formerly known as Twitter) and LinkedIn. Information that may be important to investors will be routinely posted on our website and X.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding: the company’s plan to deliver an important disease-modifying therapy to patients with NonAdvSM; the potential for bezuclastinib to deliver sustained clinical benefit with longer duration of therapy for patients with NonAdvSM; the potential for bezuclastinib’s chronic use in patients with NonAdvSM; the potential for bezuclastinib to address a significant unmet medical need and the company’s plans to submit two more NDAs for bezuclastinib in GIST and AdvSM in the first half of 2026. The use of words such as, but not limited to, "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "might," "plan," "potential," "predict," "project," "should," "target," "will," or "would" and similar words expressions are intended to identify forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on our current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, our clinical results, the rate of enrollment in our clinical trials and other future conditions. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. We may not actually achieve the forecasts or milestones disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. Such forward-looking statements are subject to a number of material risks and uncertainties including but not limited to those set forth under the caption "Risk Factors" in Cogent's most recent Quarterly Report on Form 10-Q filed with the SEC. Any forward-looking statement speaks only as of the date on which it was made. Neither we, nor our affiliates, advisors or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as result of new information, future events or otherwise, except as required by law. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date hereof.
Contact:
Christi Waarich
Senior Director, Investor Relations
christi.waarich@cogentbio.com
617-830-1653
FAQ
What did Cogent (COGT) announce on Dec 30, 2025 about bezuclastinib?
What clinical evidence supports Cogent's NDA for bezuclastinib (COGT)?
Has the FDA given any special designation to bezuclastinib (COGT)?
When will Cogent (COGT) file additional NDAs for bezuclastinib in GIST and AdvSM?
What safety data did the SUMMIT trial report for bezuclastinib (COGT)?
Does the NDA submission mean bezuclastinib is approved for NonAdvSM?
