Cogent Biosciences Announces Breakthrough Therapy Designation for Bezuclastinib in Combination with Sunitinib for Patients with Gastrointestinal Stromal Tumors (GIST)

Rhea-AI Summary

Cogent Biosciences (Nasdaq: COGT) announced that the FDA granted Breakthrough Therapy Designation for bezuclastinib combined with sunitinib for patients with GIST after prior imatinib. The designation is based on PEAK trial results showing a 50% reduction in risk of progression or death (HR 0.50, 95% CI 0.39–0.65) and mPFS of 16.5 months vs. 9.2 months for sunitinib alone. The combination showed no new safety risks versus known sunitinib profile. Cogent is on track to complete its PEAK NDA submission under RTOR in April 2026, plans a PEAK full-data presentation in H1 2026, and expects a mid-2026 Phase 2 trial for first-line exon 9 GIST patients.

Positive

• FDA Breakthrough Therapy Designation for the bezuclastinib+sunitinib combination
• PEAK trial: HR 0.50 (50% risk reduction) for progression or death
• Median PFS 16.5 months vs 9.2 months versus sunitinib monotherapy
• No new safety risks observed compared with known sunitinib profile
• NDA accepted for RTOR review; on track for April 2026 submission

Negative

• Regulatory approval still pending; NDA submission completion planned for April 2026

Key Figures

Risk reduction: 50% reduction in risk of progression or death Hazard ratio: HR 0.50 (95% CI: 0.39–0.65) Median PFS combo: 16.5 months +5 more

8 metrics

Risk reduction 50% reduction in risk of progression or death PEAK trial primary endpoint (PFS) vs sunitinib monotherapy

Hazard ratio HR 0.50 (95% CI: 0.39–0.65) Bezuclastinib+sunitinib vs sunitinib in imatinib-treated GIST

Median PFS combo 16.5 months mPFS for bezuclastinib+sunitinib arm in PEAK trial

Median PFS control 9.2 months mPFS for sunitinib monotherapy arm in PEAK trial

BTD granted Breakthrough Therapy Designation FDA designation for imatinib‑treated GIST with bezuclastinib+sunitinib

NDA completion target April 2026 Planned completion of PEAK NDA submission under RTOR

Full PEAK data timing 1H 2026 Planned presentation of full PEAK trial results

Phase 2 start Mid-2026 Planned Phase 2 trial in first-line exon 9 GIST

Market Reality Check

Price: $36.68 Vol: Volume 3,023,959 is 1.24x...

normal vol

$36.68 Last Close

Volume Volume 3,023,959 is 1.24x the 20-day average of 2,430,105, indicating elevated trading interest ahead of this FDA designation news. normal

Technical Shares at $36.68 are trading above the 200-day moving average of $17.14, while still 16.12% below the 52-week high of $43.73.

Peers on Argus

COGT fell 7.21% while key biotech peers showed only modest mixed moves (e.g., CL...

1 Up

COGT fell 7.21% while key biotech peers showed only modest mixed moves (e.g., CLDX -2.41%, AUPH -1.76%, IMCR +0.52%). Momentum scanners flagged only SRPT at +5.87%, reinforcing that COGT’s move appears stock-specific rather than sector-driven.

Historical Context

5 past events · Latest: Jan 20 (Positive)

Pattern 5 events

Date	Event	Sentiment	Move	Catalyst
Jan 20	Regulatory NDA update	Positive	+2.5%	FDA agreement to accept NDA under RTOR for bezuclastinib+sunitinib in GIST.
Jan 12	Pipeline milestones	Positive	-1.7%	Outlined 2026 NDA timelines, trial data presentations, and commercial launch goals.
Jan 06	Conference presentation	Neutral	+1.5%	Announced CEO presentation at the J.P. Morgan Healthcare Conference with webcast access.
Dec 30	NDA submission	Positive	-2.1%	Submitted NDA for bezuclastinib in NonAdvanced SM based on positive SUMMIT data.
Dec 08	Clinical trial results	Positive	+4.0%	Reported positive top-line APEX AdvSM data with strong response rates and tolerability.

Pattern Detected

Recent positive clinical and regulatory milestones often saw modest price reactions, with occasional divergences where favorable updates coincided with short-term pullbacks.

Recent Company History

Over the past few months, Cogent reported multiple positive milestones for bezuclastinib. An 8-K on Nov 10, 2025 highlighted strong Phase 3 PEAK GIST data, followed by equity and convertible note offerings in mid‑November 2025. Subsequent filings detailed an NDA for NonAdvanced SM on Dec 30, 2025 and 2026 milestone guidance on Jan 12, 2026. On Jan 20, 2026, Cogent announced RTOR NDA plans for GIST. Today’s Breakthrough Therapy Designation builds directly on those PEAK trial results and ongoing NDA activities.

Market Pulse Summary

This announcement underscores a major regulatory milestone: FDA Breakthrough Therapy Designation for...

Analysis

This announcement underscores a major regulatory milestone: FDA Breakthrough Therapy Designation for bezuclastinib plus sunitinib in imatinib‑treated GIST, supported by PEAK data showing a 50% risk reduction and mPFS of 16.5 vs 9.2 months. It builds on prior disclosures about RTOR‑based NDA plans and future Phase 2 work in first‑line exon 9 GIST. Investors may watch upcoming full PEAK data in 1H 2026, NDA progress toward the targeted April 2026 completion, and how the broader bezuclastinib franchise advances across indications.

Key Terms

breakthrough therapy designation, new drug application (nda), real-time oncology review (rtor), progression free survival (pfs), +4 more

8 terms

breakthrough therapy designation regulatory

"has granted Breakthrough Therapy Designation (BTD) for bezuclastinib in"

A breakthrough therapy designation is a regulatory fast-track given to a drug or treatment that shows early signs of providing a major improvement over existing options for a serious condition. Think of it as a VIP lane that can speed up development and more intensive guidance from regulators, which matters to investors because it can shorten time to market, reduce development risk and potentially increase a company’s value — though it does not guarantee approval.

new drug application (nda) regulatory

"Cogent will submit the PEAK New Drug Application (NDA) under previously"

A new drug application (NDA) is a formal request submitted to regulatory authorities to gain approval for a new medication to be sold and used by the public. It is a comprehensive review process that examines the drug’s safety, effectiveness, and manufacturing quality. For investors, an NDA approval can signal a potential breakthrough product and influence a company's stock value.

real-time oncology review (rtor) regulatory

"under the FDA’s Real-Time Oncology Review (RTOR) program which allows"

Real-Time Oncology Review (RTOR) is a regulatory program that lets drug regulators review key clinical data as it becomes available instead of waiting for a finished application, so the assessment can proceed in parallel with the company's final submission. For investors, RTOR matters because it can shorten the time between final data and a decision—like letting an inspector check a building while it's being finished—reducing approval uncertainty and potentially accelerating a therapy’s market entry and revenue timing.

progression free survival (pfs) medical

"clinical benefit on the primary endpoint of progression free survival (PFS),"

Progression free survival (PFS) is the amount of time after a treatment starts during which a patient’s disease does not get worse. Investors watch PFS because it’s a commonly reported measure in clinical trials that can indicate a drug’s effectiveness earlier than overall survival, much like measuring how long a dam holds before leaks reappear; stronger PFS results can speed regulatory decisions and affect a drug’s commercial prospects.

hazard ratio medical

"reducing risk of disease progression or death compared to the current standard of care by 50% (hazard ratio of 0.50,"

A hazard ratio is a way scientists compare the chance of something happening over time between two groups, like patients taking different medicines. If the ratio is high, it means one group is more likely to experience the event sooner or more often, which helps determine how effective a treatment is or how risky a situation might be.

blinded independent central review medical

"mPFS, as assessed by blinded independent central review, was 16.5"

Blinded independent central review is a quality-control step in clinical trials where outside medical experts, who do not know which patients received the experimental therapy, re-examine key measurements (like scans or lab results) to prevent bias. Think of it as neutral referees watching game footage without knowing the teams, which gives investors greater confidence that the trial results are fair, more reliable for regulators, and less likely to be overturned or disputed.

phase 2 trial medical

"expects to initiate a Phase 2 trial investigating the benefit of the"

A phase 2 trial is an intermediate-stage clinical study that tests whether a new treatment works and is reasonably safe in a group of patients who have the condition it targets. Think of it as a field test of a prototype product: it checks real-world effectiveness and side effects on a modest number of users to decide whether the treatment should move to larger, definitive testing. Investors watch phase 2 results because positive outcomes can sharply increase the likelihood of regulatory approval and future sales, while failures often halt development.

first-line medical

"Phase 2 trial investigating the benefit of the bezuclastinib plus sunitinib combination for first-line GIST patients"

First-line describes the initial or primary therapy, drug, or treatment recommended for a medical condition — the option doctors try first because it is proven, safest, or simplest. For investors, first-line status matters because treatments used first tend to reach more patients, set pricing benchmarks and become the standard of care; think of it as the default app on a phone that most users adopt before trying alternatives.

AI-generated analysis. Not financial advice.

-   Cogent will submit the PEAK New Drug Application (NDA) under previously announced RTOR designation; on track to complete NDA submission in April 2026

WALTHAM, Mass. and BOULDER, Colo., Jan. 26, 2026 (GLOBE NEWSWIRE) -- Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for bezuclastinib in combination with sunitinib for patients with Gastrointestinal Stromal Tumors (GIST) who have received prior treatment with imatinib. 

“We are excited to announce this Breakthrough Therapy Designation which recognizes the potential for the bezuclastinib combination to substantially improve upon the currently available treatment options for patients with imatinib-resistant GIST,” said Andrew Robbins, Cogent’s President and Chief Executive Officer. “We look forward to the continued collaboration with the FDA as we work to bring the first new treatment option in over twenty years to this patient population.”

This Breakthrough Therapy Designation is based on results from the PEAK trial which demonstrated a substantial and highly statistically significant clinical benefit on the primary endpoint of progression free survival (PFS), reducing risk of disease progression or death compared to the current standard of care by 50% (hazard ratio of 0.50, 95% CI: 0.39 – 0.65). mPFS, as assessed by blinded independent central review, was 16.5 months for the bezuclastinib combination vs. 9.2 months for sunitinib monotherapy. The combination was well tolerated, and no new safety risks were observed when compared to the known safety profile of sunitinib. Breakthrough Therapy Designation is intended to expedite the review of medicines that treat a serious or life-threatening condition and have shown clinical evidence indicating the potential for substantial improvement over available therapies.

Earlier this month, the FDA agreed to accept Cogent’s NDA under the FDA’s Real-Time Oncology Review (RTOR) program which allows an applicant to pre-submit components of its NDA to allow the FDA to review clinical trial data before the complete filing is submitted and aims to provide a more efficient review process to ensure safe and effective treatments are available to patients as early as possible.

Cogent plans to present full results from the PEAK trial at a major medical meeting during the first half of 2026. Additionally, in mid-2026 Cogent expects to initiate a Phase 2 trial investigating the benefit of the bezuclastinib plus sunitinib combination for first-line GIST patients with exon 9 mutations who are naive to, or recently initiated treatment with, imatinib.

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. In addition to bezuclastinib, the Cogent Research Team is developing a portfolio of novel targeted therapies to help patients fighting serious, genetically driven diseases initially targeting mutations in FGFR2/3, ErbB2, PI3Kα, 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 plans to submit its PEAK NDA under RTOR and to complete the NDA submission in April 2026; the potential for the bezuclastinib combination to substantially improve upon the currently available treatment options for patients with imatinib-resistant GIST; the anticipated benefits of FDA’s RTOR and Breakthrough Therapy Designation; plans to present the full results from the PEAK trial at a major medical meeting during the first half of 2026 and the expectation to initiate in mid-2026 a Phase 2 trial investigating the benefit of the bezuclastinib plus sunitinib combination for first-line GIST patients with exon 9 mutations who are naive to, or recently initiated treatment with, imatinib. 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 Annual Report on Form 10-K and/or 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 about bezuclastinib for GIST on January 26, 2026?

The company said the FDA granted Breakthrough Therapy Designation for bezuclastinib plus sunitinib for imatinib‑resistant GIST based on PEAK trial benefits.

What were the PEAK trial results that supported the FDA Breakthrough Therapy Designation for COGT?

PEAK showed a 50% reduction in risk of progression or death (HR 0.50, 95% CI 0.39–0.65) and mPFS of 16.5 vs 9.2 months versus sunitinib.

When does Cogent (COGT) plan to submit the PEAK NDA under RTOR?

Cogent is on track to complete the PEAK NDA submission in April 2026 under the FDA Real‑Time Oncology Review program.

Will the bezuclastinib plus sunitinib combination present new safety concerns according to Cogent (COGT)?

The company reported the combination was well tolerated and observed no new safety risks compared to the known sunitinib profile.

When will Cogent (COGT) present full PEAK trial results and what are next trials?

Cogent plans a full PEAK presentation in H1 2026 and expects to start a Phase 2 trial in mid‑2026 for first‑line exon 9 GIST patients.