This section highlights FDA-related milestones and regulatory updates for drugs developed by Cogent Biosciences (COGT).
Over the past two years, Cogent Biosciences has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
bezuclastinib and Bezuclastinib. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
bezuclastinib - FDA Regulatory Timeline and Events
bezuclastinib is a drug developed by Cogent Biosciences for the following indication: In patients with non-advanced systemic mastocytosis.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- bezuclastinib
- Announced Date:
- July 7, 2025
- Indication:
- In patients with non-advanced systemic mastocytosis
Announcement
Cogent Biosciences, Inc. announced positive top-line results from the registration-directed Part 2 of the SUMMIT clinical trial of bezuclastinib in patients with non-advanced systemic mastocytosis (NonAdvSM) demonstrating clinically meaningful and highly statistically significant improvements across the primary and all key secondary endpoints, including patient-reported symptoms and objective measures of mast cell burden.
AI Summary
Cogent Biosciences announced positive top-line results from Part 2 of its SUMMIT clinical trial for bezuclastinib in patients with non-advanced systemic mastocytosis (NonAdvSM). The trial showed that bezuclastinib led to a clinically meaningful improvement in patient-reported symptoms and a significant reduction in mast cell burden. Specifically, patients on bezuclastinib experienced a 24.3-point decrease in total symptom score compared to a 15.4-point decrease in the placebo group, with a statistically significant p-value of 0.0002.
Moreover, 87.4% of patients in the treatment group achieved at least a 50% reduction in serum tryptase levels—a key objective measure—while no patients in the placebo group reached this milestone. These strong and statistically significant outcomes, along with a favorable safety profile, support bezuclastinib as a promising new treatment option as Cogent moves toward its FDA New Drug Application submission by the end of 2025.
Read Announcement
Bezuclastinib - FDA Regulatory Timeline and Events
Bezuclastinib is a drug developed by Cogent Biosciences for the following indication: Advanced Systemic Mastocytosis (AdvSM).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Bezuclastinib
- Announced Date:
- February 10, 2025
- Indication:
- Advanced Systemic Mastocytosis (AdvSM)
Announcement
Cogent Biosciences, Inc announced an upcoming poster for bezuclastinib at the 2025 American Academy of Allergy Asthma & Immunology Annual Meeting (AAAAI) being held in San Diego, CA, February 28-March 3, 2025.
AI Summary
Cogent Biosciences, Inc. announced that it will present a research poster on its drug bezuclastinib at the 2025 American Academy of Allergy Asthma & Immunology Annual Meeting. The poster will focus on patients with non-advanced systemic mastocytosis who received 100 mg of bezuclastinib for at least 48 weeks, highlighting the long-term benefits of the treatment. The presentation is part of a Phase 2 clinical trial known as the Summit Trial.
The session, titled "Novel Mechanisms of Mast Cells, Basophils and IgE," is scheduled for March 1, 2025, from 9:45 am to 10:45 am PT at the San Diego Convention Center. The poster, labeled as number 520, will provide important insights into the efficacy and safety of the treatment.
Read Announcement- Drug:
- Bezuclastinib
- Announced Date:
- December 9, 2024
- Indication:
- Advanced Systemic Mastocytosis (AdvSM)
Announcement
Cogent Biosciences, Inc. announced updated clinical results from the Open Label Extension (OLE) portion of SUMMIT, a clinical trial evaluating bezuclastinib in patients with nonadvanced systemic mastocytosis (NonAdvSM).
AI Summary
Cogent Biosciences recently shared updated clinical results from the Open Label Extension (OLE) of the SUMMIT trial, which is testing bezuclastinib in patients with nonadvanced systemic mastocytosis (NonAdvSM). The trial data revealed a 56% average improvement in the Total Symptom Score (TSS) at 24 weeks, with 76% of patients experiencing at least a 50% reduction in their symptoms. Moreover, 89% of patients saw more than a 50% decrease in serum tryptase levels by four weeks, and 95% of those with elevated baseline tryptase reached levels below 20 ng/ml by week 24.
The strong symptomatic and pharmacodynamic improvements indicate that bezuclastinib could offer rapid and sustained benefits for NonAdvSM patients. In addition to these promising results, enrollment in the SUMMIT Part 2 trial was completed ahead of schedule with 179 patients, and top-line results from this study are expected in July 2025.
Read Announcement- Drug:
- Bezuclastinib
- Announced Date:
- December 8, 2024
- Indication:
- Advanced Systemic Mastocytosis (AdvSM)
Announcement
Cogent Biosciences, Inc reported positive updated data from Part 1 of the Company's ongoing Phase 2 APEX clinical trial evaluating bezuclastinib in patients with advanced systemic mastocytosis (AdvSM) at the 66th American Society of Hematology (ASH 2024) Annual Meeting & Exposition taking place December 7-10, 2024 in San Diego, CA.
AI Summary
Cogent Biosciences, Inc. presented encouraging updated data from Part 1 of its ongoing Phase 2 APEX trial at the 66th American Society of Hematology (ASH) 2024 Annual Meeting in San Diego. The study is evaluating the safety, efficacy, and pharmacological profile of bezuclastinib in patients with advanced systemic mastocytosis (AdvSM). Results showed high overall response rates, especially in the 100 mg BID dosing group, where patients achieved an 83% response rate per mIWG criteria and an 88% response rate per pure pathological response criteria. The median time to response was 2.2 months, and many patients experienced significant improvements in key disease biomarkers. These early findings support bezuclastinib's potential to provide rapid and deep clinical responses with a manageable safety profile, offering a promising new treatment approach for AdvSM patients. Part 2 of the trial is on track with top-line data expected in mid-2025.
Read Announcement- Drug:
- Bezuclastinib
- Announced Date:
- September 3, 2024
- Estimated Event Date Range:
- July 1, 2025 - December 31, 2025
- Target Action Date:
- 2025-H2
- Indication:
- Advanced Systemic Mastocytosis (AdvSM)
Announcement
Cogent Biosciences, Inc announced that top-line results expected 2H 2025
AI Summary
Cogent Biosciences, Inc. provided key updates on their clinical trial progress for bezuclastinib, a promising KIT mutant inhibitor. The Phase 3 PEAK trial for gastrointestinal stromal tumors (GIST) enrolled 413 patients and recently passed an interim futility analysis, keeping the study on track. Top-line results for the PEAK trial are expected by the end of 2025, supporting plans for a New Drug Application in this patient group.
Additionally, the registration-directed SUMMIT trial for nonadvanced systemic mastocytosis has seen accelerated enrollment, with completion expected in the first quarter of 2025. Top-line results for the SUMMIT trial are projected to be available in the second half of 2025, reflecting strong global interest and commitment to advancing precision therapies for genetically defined diseases.
Read Announcement- Drug:
- Bezuclastinib
- Announced Date:
- September 3, 2024
- Estimated Event Date Range:
- January 1, 2025 - March 31, 2025
- Target Action Date:
- Q1 - 2025
- Indication:
- Advanced Systemic Mastocytosis (AdvSM)
Announcement
Cogent Biosciences, Inc announced that Registration-directed SUMMIT trial in NonAdvanced Systemic Mastocytosis (NonAdvSM) now on track to complete enrollment in Q1 2025
AI Summary
Cogent Biosciences, Inc. announced that its registration-directed SUMMIT trial for NonAdvanced Systemic Mastocytosis (NonAdvSM) is progressing well. The company now expects to complete patient enrollment in this global trial during the first quarter of 2025, which is roughly three months ahead of the original schedule. Top-line results from the study are anticipated in the second half of 2025. This update highlights the strong global interest and rapid enrollment driven by the need for new, precision therapies. The SUMMIT trial is designed to evaluate bezuclastinib’s potential as a best-in-class KIT mutant inhibitor, reinforcing Cogent’s commitment to advancing treatments for genetically defined diseases. Overall, the accelerated timeline for this trial represents a significant milestone in their efforts to provide effective therapies for patients with NonAdvSM.
Read Announcement- Drug:
- Bezuclastinib
- Announced Date:
- September 3, 2024
- Indication:
- Advanced Systemic Mastocytosis (AdvSM)
Announcement
Cogent Biosciences, Inc today provided several updates from the company's ongoing registration-directed clinical trials of its potential best-in-class KIT mutant inhibitor, bezuclastinib.
AI Summary
Cogent Biosciences, Inc. shared important updates on its registration-directed clinical trials for its promising KIT mutant inhibitor, bezuclastinib. The company confirmed that its Phase 3 PEAK trial for patients with gastrointestinal stromal tumors (GIST) has completed enrollment with 413 patients, surpassing its target. An interim futility analysis was conducted based on progression-free survival, with the Independent Data Monitoring Committee recommending that the study continue without changes. Additionally, the SUMMIT trial, which evaluates bezuclastinib in nonadvanced systemic mastocytosis (NonAdvSM), is progressing well, with patient enrollment expected to finish by the first quarter of 2025 and results anticipated in the second half of the year. These advancements underscore strong patient interest and the potential of bezuclastinib as a best-in-class therapy targeting KIT mutations.
Read Announcement - Drug:
- Bezuclastinib
- Announced Date:
- June 27, 2024
- Indication:
- Advanced Systemic Mastocytosis (AdvSM)
Announcement
Cogent Biosciences, Inc. announced it has reached alignment with the U.S. Food and Drug Administration (FDA) on the Company's novel patient reported outcome measure, Mastocytosis Symptom Severity Daily Diary (MS2D2), for use in Part 2 of the registration-directed SUMMIT trial evaluating bezuclastinib in Nonadvanced Systemic Mastocytosis (NonAdvSM) patients.
AI Summary
Cogent Biosciences, Inc. announced that it has reached alignment with the U.S. Food and Drug Administration (FDA) on its new patient-reported outcome measure, the Mastocytosis Symptom Severity Daily Diary (MS2D2). The MS2D2 is designed to track patient symptoms from the beginning of treatment and throughout the trial, helping to capture changes in the severity of key symptoms over time. This FDA agreement allows the diary to be used in Part 2 of the registration-directed SUMMIT trial, which evaluates bezuclastinib in patients with Nonadvanced Systemic Mastocytosis (NonAdvSM). In the trial, data gathered by the MS2D2 will contribute to a Total Symptom Score used as the primary endpoint for measuring treatment effectiveness. Cogent Biosciences plans to complete enrollment in SUMMIT Part 2 by the second quarter of 2025 and to report top-line results by the end of 2025.
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