Cogent Biosciences Announces FDA Acceptance of New Drug Application (NDA) for Bezuclastinib in Patients with Advanced Systemic Mastocytosis (AdvSM)

FDA acceptance of the AdvSM NDA and strong APEX data add to Cogent’s three active bezuclastinib reviews across KIT-driven diseases.

Rhea-AI Summary

Cogent Biosciences (COGT) reported FDA acceptance of its New Drug Application for bezuclastinib in patients with Advanced Systemic Mastocytosis (AdvSM), with a PDUFA target action date of June 29, 2027.

The FDA indicated there is currently no plan to hold an advisory committee meeting and has not identified potential review issues. The NDA is supported by the pivotal APEX trial, in which bezuclastinib showed a 65% overall response rate by mIWG-MRT-ECNM criteria, including 57% of patients achieving CR, CRh or PR, and an 81% response rate by pure pathological response criteria as of March 31, 2026. Twelve‑month progression‑free survival was 79% and twelve‑month overall survival was 87%, with median PFS and OS not yet mature.

Additional NDAs for bezuclastinib in NonAdvSM and GIST remain under FDA review with PDUFA dates of December 30, 2026 and November 30, 2026, respectively. Cogent also detailed active U.S. expanded access programs for SM and GIST and disclosed option grants for 39,300 shares and 33,600 RSUs to nine new employees under its 2020 Inducement Plan.

Positive

• NDA accepted for AdvSM with FDA PDUFA target action date of June 29, 2027
• APEX trial ORR 65% by mIWG-MRT-ECNM, with 57% achieving CR, CRh or PR
• APEX PPR response rate 81% in AdvSM as of March 31, 2026 data cutoff
• 12‑month PFS 79% and OS 87% in APEX, with median PFS/OS not yet mature
• Two additional NDAs under review in NonAdvSM (PDUFA Dec 30, 2026) and GIST (PDUFA Nov 30, 2026)
• Expanded Access Programs active for eligible U.S. SM and GIST patients needing bezuclastinib-based treatment

Negative

• Equity inducement grants of options for 39,300 shares and 33,600 RSUs create incremental dilution risk

News Explained

The employee awards can add equity over time, but vesting conditions defer delivery across four years and options also require exercise.

On September 10 and September 14, 2026, Cogent Biosciences approved equity awards for nine new employees, creating conditional future equity issuance rather than making the full package vested immediately.

The package includes 39,300 nonqualified options to purchase common stock, each with a 10-year term and an exercise price equal to the applicable grant-date closing price.

The options vest 25% after one year and monthly thereafter for 36 months, while 33,600 RSUs vest in equal annual installments over four years, in each case subject to continued employment.

Market Context

Before publication, COGT closed at $33.41 with a 0% 24-hour change; the prior June 30 AdvSM NDA subm...

Analysis

Before publication, COGT closed at $33.41 with a 0% 24-hour change; the prior June 30 AdvSM NDA submission was followed by a -0.1% reaction, providing directly comparable regulatory context.

Key Figures

PDUFA target action date: June 29, 2027 Overall response rate: 65% CR, CRh or PR best response: 57% +5 more

PDUFA target action date

June 29, 2027

AdvSM NDA

Overall response rate

65%

APEX pivotal trial; mIWG-MRT-ECNM criteria

CR, CRh or PR best response

57%

APEX pivotal trial

Pure pathological response

81%

APEX pivotal trial

12-month progression-free survival

79%

APEX pivotal trial

12-month overall survival

87%

APEX pivotal trial

Inducement options

39,300 shares

Awards to nine new employees

Inducement RSUs

33,600 RSUs

Awards to nine new employees

Previous Clinical trial Reports

2 past events · Latest: Jun 30

Same Type 2 events

1. Jun 30

   NDA submission

   24h Move

   -0.1%

   AdvSM NDA submitted with APEX trial data supporting the application

2. Jun 12

   APEX trial data

   24h Move

   +1.5%

   Detailed APEX AdvSM data reported clinically meaningful response and durability

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Key Terms

nda, pdufa, expanded access program, tyrosine kinase inhibitor, +2 more

6 terms

nda regulatory

"accepted its New Drug Application (NDA) for bezuclastinib"

An NDA, or nondisclosure agreement, is a legal contract that keeps certain information private between parties. It’s like a promise not to share sensitive details, helping protect business ideas, strategies, or data from being leaked or used without permission. For investors, NDAs help ensure that confidential information remains secure, enabling trust and open communication during business discussions.

pdufa regulatory

"assigned a Prescription Drug User Fee Act (PDUFA) target action date"

PDUFA is the Prescription Drug User Fee Act, the U.S. law under which drug companies pay fees that fund the FDA's review of new medicines. In company news the term usually appears as the PDUFA date, the target deadline by which the FDA aims to decide on a drug application; that date tells investors when to expect the approval or rejection decision for the product.

expanded access program regulatory

"established active Expanded Access Programs (EAPs) for U.S. patients"

A program that allows patients with serious or life‑threatening conditions to receive an experimental drug or therapy before it is fully approved by regulators, when they cannot join clinical trials. Investors care because expanded access can change a treatment’s market perception, create early real‑world safety or demand signals, and affect regulatory timelines and potential revenue — like a pre‑order system that also reveals how the product performs outside controlled testing.

tyrosine kinase inhibitor medical

"Bezuclastinib is a selective tyrosine kinase inhibitor"

A tyrosine kinase inhibitor is a type of drug that blocks specific proteins in cells that act like on/off switches for growth and survival signals, often used to stop cancer cells from multiplying. For investors, these drugs matter because their clinical trial results, regulatory approvals, safety profiles, and patent status drive sales potential and company valuation—think of them as precision tools whose effectiveness and market exclusivity determine commercial success.

restricted stock units financial

"33,600 restricted stock units (RSUs)"

Restricted stock units are a type of company reward where employees are promised shares of stock, but they only fully own these shares after meeting certain conditions, like staying with the company for a set time. They matter because they can become valuable assets and are often used to motivate employees to help the company succeed.

nonqualified options financial

"nonqualified options to purchase 39,300 shares"

Nonqualified options are employee stock options that give a holder the right to buy company shares at a set price for a limited period, like a coupon letting you buy a product later at today’s price. They matter to investors because when exercised the difference between market price and the set price is taxed as regular income (affecting the holder’s tax bill) and can dilute existing shares and create a tax deduction for the company.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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NDA supported by positive results from the APEX pivotal trial demonstrating clinically meaningful responses and evidence of disease modification in patients with AdvSM

PDUFA target action date set for June 29, 2027

WALTHAM, Mass. and BOULDER, Colo., Sept. 15, 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 accepted its New Drug Application (NDA) for bezuclastinib in patients with Advanced Systemic Mastocytosis (AdvSM) and assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 29, 2027. In addition, the FDA communicated that at this time, there is no plan to hold an advisory committee, nor have they identified any potential review issues.

“With the FDA’s acceptance of our NDA in Advanced Systemic Mastocytosis, we have reached another important regulatory milestone for bezuclastinib and are one step closer to potentially bringing this therapy to patients with AdvSM,” said Andrew Robbins, Cogent’s President and Chief Executive Officer. “Together with the NDAs currently under review for NonAdvSM and GIST patients, this milestone further reinforces the potential of bezuclastinib across multiple patient populations with KIT-driven diseases. With an excellent commercial and medical team in place, we are excited and ready for the potential launches later this year.”

The NDA acceptance is supported by data from the APEX pivotal trial, which was most recently presented at the 2026 European Hematology Association (EHA) Congress, in which bezuclastinib demonstrated clinically meaningful responses and durable clinical activity in patients with AdvSM. As of the March 31, 2026 data cutoff, the primary endpoint of response per mIWG-MRT-ECNM criteria demonstrated a 65% overall response rate, including 57% of patients achieving CR, CRh or PR as best response. Response by pure pathological response (PPR) criteria was 81%. Bezuclastinib also demonstrated durable clinical activity, with a 12-month progression-free survival rate of 79% and 12-month overall survival rate of 87%, with median PFS and OS not yet mature.

Cogent continues to advance bezuclastinib toward potential approvals and commercialization across its three indications. The Company’s NDA for NonAdvanced Systemic Mastocytosis is under FDA review, with a PDUFA target action date of December 30, 2026, and the NDA for GIST is also under FDA review, with a PDUFA target action date of November 30, 2026. Together, these regulatory milestones represent significant progress toward bringing bezuclastinib to patients across a broad spectrum of KIT-driven diseases.

Bezuclastinib - Expanded Access Program
Working with the FDA, Cogent has established active Expanded Access Programs (EAPs) for U.S. patients with SM or GIST who meet disease-specific criteria and could benefit from treatment with bezuclastinib or the combination of bezuclastinib and sunitinib. For more information please visit: https://www.cogentbio.com/bezuclastinib-program-development/#our-expanded-access-policy.

Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
Cogent also announced today that, on September 10, 2026, the Compensation Committee of Cogent’s Board of Directors, made up entirely of independent directors, approved the grants of “inducement” equity awards to nine new employees under the company’s 2020 Inducement Plan with grant dates of September 10, 2026 and September 14, 2026. The awards were approved in accordance with Listing Rule 5635(c)(4) of the corporate governance rules of the Nasdaq Stock Market. The employees received, in the aggregate, (i) nonqualified options to purchase 39,300 shares of Cogent common stock and (ii) 33,600 restricted stock units (RSUs). Each option has a 10-year term, an exercise price equal to the closing price of Cogent’s common stock on the grant date, and a 4-year vesting schedule with 25% vesting on the 1-year anniversary of the grant date and the remainder vesting in equal monthly installments over the subsequent 36 months, provided such employee remains employed through each such vesting date. The RSUs vest annually in equal installments over 4 years from the grant date, provided such employee remains employed through each such vesting date.

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, the Cogent Research Team is developing a portfolio of novel targeted therapies to help patients fighting serious, genetically driven diseases targeting mutations in 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  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 potential for regulatory approval and commercialization of bezuclastinib across three indications: patients with GIST, NonAdvSM and AdvSM; bezuclastinib’s potential across multiple KIT-driven diseases and the potential commercial launches of bezuclastinib later this year. 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

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

What clinical data supported the NDA for bezuclastinib in Advanced Systemic Mastocytosis?

The NDA is supported by the pivotal APEX trial in AdvSM. As of the March 31, 2026 data cutoff, the primary endpoint of response per mIWG-MRT-ECNM criteria showed a 65% overall response rate, including 57% of patients achieving complete response (CR), CR with partial hematologic recovery (CRh) or partial response (PR) as best response. By pure pathological response (PPR) criteria, the response rate was 81%. Bezuclastinib also showed durable activity, with a 12‑month progression‑free survival rate of 79% and a 12‑month overall survival rate of 87%, and median PFS and OS were not yet mature.

What are the PDUFA timelines for Cogent’s other bezuclastinib NDAs?

Cogent has two additional bezuclastinib NDAs under FDA review. The NDA for NonAdvanced Systemic Mastocytosis has a PDUFA target action date of December 30, 2026. The NDA for gastrointestinal stromal tumors (GIST) has a PDUFA target action date of November 30, 2026.

What Expanded Access Programs are available for bezuclastinib?

Working with the FDA, Cogent has established active Expanded Access Programs (EAPs) in the United States for patients with systemic mastocytosis (SM) or GIST who meet disease‑specific criteria and could benefit from treatment with bezuclastinib or the combination of bezuclastinib and sunitinib. Additional information is provided on the company’s website at its bezuclastinib program development and expanded access policy page.

What are the key terms of the inducement equity awards granted to new Cogent employees?

On September 10, 2026, the Compensation Committee, composed solely of independent directors, approved inducement awards under Cogent’s 2020 Inducement Plan for nine new employees, with grant dates of September 10 and 14, 2026. In aggregate, the employees received (i) nonqualified options to purchase 39,300 shares of common stock and (ii) 33,600 restricted stock units (RSUs). Each option has a 10‑year term, an exercise price equal to the closing price on the grant date, and vests over 4 years, with 25% on the first anniversary and the remainder in equal monthly installments over the next 36 months, subject to continued employment. The RSUs vest in equal annual installments over 4 years, also contingent on continued employment.