Axatilimab-csfr

New Drug Application (NDA) for NonAdvSM submitted in December 2025, NDA submission for AdvSM on track for 1H 2026 NDA submission for GIST on track for April 2026; bezuclastinib has the potential to be the first new therapy for second-line GIST in over 20 years Clinical data presentations from all three pivotal trials, PEAK, SUMMIT and APEX, expected at major medical meetings in 1H 2026 Investigational New Drug (IND) applications expected in 2026 for potentially best-in-class pan-KRAS(ON) and selective JAK2 V617F inhibitors Strong financial position with ~$900 million cash to begin 2026, sufficient to fund commercial launches and operations well into 2028 Company to present at the 44th Annual J.P. Morgan Healthcare Conference on Tuesday, January 13, 2026 at 8:15 am PT/11:15 am ET WALTHAM, Mass. and BOULDER, Colo., Jan. 12, 2026 (GLOBE NEWSWIRE) -- Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, today highlighted the company’s key 2026 milestones ahead of its presentation at J.P. Morgan’s 44th annual healthcare conference. “2025 was marked by exceptional progress at Cogent Biosciences,” said Andrew Robbins, President and Chief Executive Officer. “We reported positive results from all three pivotal trials of bezuclastinib in patients with GIST and Systemic Mastocytosis, submitted our first NDA for bezuclastinib in patients with NonAdvSM based on the strength of the SUMMIT data, and put ourselves in a very strong financial position entering the new year. During 2026, we will transform Cogent into a fully integrated commercial stage company with plans to launch bezuclastinib in the second half of the year. In addition, we are investing for the future and plan to submit INDs in 2026 for both our pan-KRAS inhibitor and recently announced JAK2 V617F inhibitor. Our plans are supported by a highly experienced team with proven launch experience backed by a strong balance sheet. We are poised for success and extremely excited about the meaningful impact we can have for patients.” In 2026, Cogent plans to achieve the following milestones: Bezuclastinib Acceptance of the New Drug Application (NDA) for bezuclastinib in NonAdvanced Systemic Mastocytosis (NonAdvSM) by the end of February 2026 Submit an NDA in April 2026 for bezuclastinib in patients with advanced Gastrointestinal Stromal Tumors (GIST) previously treated with imatinib based on the strength of the PEAK pivotal trial Submit an NDA in 1H 2026 for bezuclastinib in patients with Advanced Systemic Mastocytosis (AdvSM) based on the strength of the APEX pivotal trial Present detailed, updated clinical data from each of the three pivotal trials (SUMMIT, PEAK, APEX) at major medical meetings during 1H 2026 Commercialize bezuclastinib following potential FDA approval in 2H 2026 Pipeline Submit Investigational New Drug (IND) applications for CGT1815, Cogent’s novel, selective pan-KRAS(ON) inhibitor and CGT1145, Cogent’s novel, selective JAK2 V617F inhibitor Present clinical data on CGT4859, Cogent’s selective and potent FGFR 2/3 inhibitor, from its Phase 1/2 study in patients with alterations in FGFR2 or FGFR3 Complete dose escalation for both CGT4255, Cogent’s CNS-penetrant, selective mutant ErbB2 inhibitor, and CGT6297, Cogent’s selective PI3Kα inhibitor Bezuclastinib Expanded Access Programs Working with the FDA, Cogent has established active Expanded Access Programs for U.S. patients with GIST or SM who meet disease-specific criteria and could benefit from treatment with bezuclastinib or the combination of bezuclastinib and sunitinib. A growing number of investigational sites now offer access to the bezuclastinib EAPs. For more information please visit: https://www.cogentbio.com/bezuclastinib-program-development/#our-expanded-access-policy New Leadership Appointment Cogent also announced today that Abb Hayden has joined Cogent as Senior Vice President, Sales. Mr. Hayden joins Cogent with over 25 years of industry experience. Previously he served as Vice President of Commercial at Syndax Pharmaceuticals. While at Syndax, he played an integral part in building the commercial infrastructure leading the Field Sales, Marketing, and Clinical Education team to launch Revuforj and Niktimvo. Prior to Syndax, he held roles of increasing responsibility at Adaptive Biotechnologies, Onyx Pharmaceuticals, and Eli Lilly. Mr. Hayden is a graduate of the University of Central Arkansas. J.P. Morgan Presentation Details Cogent will participate in a presentation and Q&A session at the 44th Annual J.P. Morgan Healthcare Conference on Tuesday, January 13, 2026, beginning at 8:15 a.m. PT (11:15 a.m. ET). A live webcast will be accessible in the “Investors & Media” section of the company’s website, www.cogentbio.com, and will be archived for 30 days following the event. Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)Cogent also announced today that, on January 9, 2026, the Compensation Committee of Cogent’s Board of Directors, made up entirely of independent directors, approved the grant of “inducement” equity awards to five new employees under the company’s 2020 Inducement Plan with a grant date of January 9, 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 66,700 shares of Cogent common stock and (ii) 9,700 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. 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, 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 StatementsThis 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 expectation that the FDA will accept our NDA for bezuclastinib in NonAdvSM by the end of February 2026 and approve it for commercial use in the second half of 2026; plans to submit an NDA in April 2026 for bezuclastinib in GIST; plans to submit an NDA in the first half of 2026 for bezuclastinib in AdvSM; plans to present updated clinical data from each of the SUMMIT, PEAK and APEX trials at major medical meetings in the first half of 2026; plans to launch bezuclastinib commercially in the second half of 2026 following FDA approval; plans to submit INDs in 2026 for both our pan-KRAS and JAK2 V617F inhibitors; the potential for bezuclastinib to be the first new therapy for second-line GIST in over 20 years; the expectation that our cash runway is sufficient to fund commercial launches and operations well into 2028; the expectation that Cogent will transform into a fully integrated commercial stage company in 2026; plans to present clinical data in 2026 on CGT4859 and plans to complete dose escalation in 2026 for both CGT4255 and CGT6297. 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 or 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 WaarichSenior Director, Investor Relationschristi.waarich@cogentbio.com 617-830-1653

– Approximately $44 million and $125 million in preliminary (unaudited) Revuforj® (revumenib) 4Q25 and full year 2025 net revenue, respectively – – Continued acceleration in demand following approval in R/R NPM1m AML, with an approximate 38% increase in Revuforj net revenue in 4Q25 vs. 3Q25 - – $56 million and $152 million in preliminary (unaudited) Niktimvo™ (axatilimab-csfr) 4Q25 and full year 2025 net revenue, respectively; Syndax will report its share of the Niktimvo net profit when it reports full year 2025 results – – Ended 2025 with approximately $394 million in cash, cash equivalents and marketable securities (unaudited); fully funded through profitability – – Syndax to present at the 44th Annual J.P. Morgan Healthcare Conference on Monday, January 12, 2026 , at 3:00 p.m. PT / 6:00 p.m. ET . – NEW YORK , Jan. 12, 2026 (GLOBE NEWSWIRE) -- Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company advancing innovative cancer therapies, today announced preliminary, unaudited fourth quarter and full year 2025 financial results and provided additional business updates ahead of its presentation at the 44th Annual J.P. Morgan Healthcare conference. “2025 was a transformational year for Syndax as we secured our third FDA approval and successfully executed the launches of Revuforj and Niktimvo, proving our ability to deliver innovative therapies for patients and advancing the company towards profitability,” said Michael A. Metzger , Chief Executive Officer of Syndax. “With strong momentum and further acceleration of growth going into 2026, we look forward to continuing to build Revuforj and Niktimvo into industry leading franchises and advancing our lifecycle and frontline programs designed to expand our patient impact and unlock the multi-billion-dollar potential of both medicines.” Preliminary 2025 Financial Highlights & Recent Business Updates Revuforj® (revumenib) Approximately $44 million and $125 million in preliminary (unaudited) fourth quarter and full-year 2025 Revuforj net revenue, respectively. Revuforj net revenue increased by approximately 38% in the fourth quarter of 2025 compared to the third quarter of 2025. The Company observed continued acceleration in demand following the FDA’s approval of Revuforj in R/R NPM1m AML on October 24, 2025 , with an approximately 35% increase in total Revuforj prescriptions in the fourth quarter of 2025 compared to the third quarter 2025. Received the ‘Best New Drug’ award for Revuforj at the Scrip Awards 2025. The award recognizes excellence in pharmaceutical development and the drug that represents the best therapeutic advance in its area. Initiated REVEAL-ND, a Phase 3, randomized, double-blind, placebo-controlled trial of revumenib in combination with intensive chemotherapy in newly diagnosed patients with NPM1m AML in November 2025 . The trial has dual primary endpoints of measurable residual disease (MRD) negative complete remission (CR) and event free survival (EFS) to support the potential for accelerated approval and full approval, respectively. Initiated a multi-regional Managed Access Program which will expand access to Revuforj outside the U.S. This program enables physicians to prescribe Revuforj to appropriate patients outside the U.S. where the drug is not approved but access to novel medicines is permitted by local regulations and where funding can be secured. Niktimvo™ (axatilimab-csfr) $56 million and $152 million in preliminary (unaudited) fourth quarter and full-year 2025 Niktimvo net revenue, respectively. Syndax will report its 50% share of the Niktimvo net commercial profit, defined as net product revenue minus the cost of sales and commercial expenses, when the Company reports its full year 2025 results. The Company expects its share of the product contribution to amount to approximately 25-30% of Niktimvo net revenue. Cash Position & 2026 Financial Guidance Preliminary (unaudited) year-end 2025 cash, cash equivalents, and marketable securities of approximately $394 million . For the full year of 2026, the Company expects total research and development plus selling, general and administrative expenses to be approximately $400 million , excluding the impact of $50 million in estimated non-cash stock compensation expense. Syndax expects that its cash position, combined with its anticipated product revenue, interest income, and stable operating expense base, will enable the company to reach profitability. *The preliminary fourth quarter and full year 2025 financial results are preliminary, unaudited, subject to adjustment, and provided as an approximation in advance of the Company’s announcement of complete financial results in early 2026. Key 2026 Priorities & Anticipated Milestones Drive Revuforj and Niktimvo net revenue growth and invest thoughtfully in pipeline development and data generation to fuel further growth. Complete enrollment in MAXPIRe Phase 2 trial of axatilimab in idiopathic pulmonary fibrosis (IPF) in January 2026 , with topline data on track for the second half of 2026. Advance global enrollment in EVOLVE-2 (unfit NPM1m and KMT2Ar AML) and REVEAL-ND (fit NPM1m), two Phase 3 frontline trials of revumenib in combination with low-intensity and high-intensity regimens, respectively. Initiate RAVEN, a Phase 2 collaborative trial of revumenib in combination with venetoclax and azacitidine in newly diagnosed KMT2Ar patients who would be considered eligible, or fit, for intensive chemotherapy in 2026. Further leadership in menin and CSF-1R inhibition through the publication and presentation of clinical trial data and real-world evidence, including revumenib data from the frontline and post-HSCT maintenance setting. Initiate a program in 2026 designed to generate proof-of-principle clinical data with revumenib in myelofibrosis (MF). J.P. Morgan Healthcare Conference Presentation and Webcast Michael Metzger , Chief Executive Officer of Syndax, will discuss these updates as part of a webcast presentation at the 44th Annual J.P. Morgan Healthcare Conference on Monday, January 12, 2026 , at 3:00 p.m. PT / 6:00 p.m. ET . A live webcast of the event can be accessed from the Investor section of the Company's website at www.syndax.com, where a replay of the event will also be available for a limited time. About Revuforj® (revumenib) Revuforj (revumenib) is an oral, first-in-class menin inhibitor that is FDA approved for the treatment of relapsed or refractory (R/R) acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation as determined by an FDA-authorized test in adult and pediatric patients one year and older. Revuforj is also indicated for the treatment of R/R acute myeloid leukemia (AML) with a susceptible nucleophosmin 1 (NPM1) mutation in adult and pediatric patients one year and older who have no satisfactory alternative treatment options. Multiple trials of revumenib are ongoing or planned across the treatment landscape, including in combination with standard of care therapies in newly diagnosed patients with NPM1m or KMT2Ar AML. Revumenib was previously granted Orphan Drug Designation for the treatment of AML, ALL and acute leukemias of ambiguous lineage (ALAL) by the U.S. FDA and for the treatment of AML by the European Commission . The U.S. FDA also granted Fast Track designation to revumenib for the treatment of adult and pediatric patients with R/R acute leukemias harboring a KMT2A rearrangement or NPM1 mutation and Breakthrough Therapy Designation for the treatment of adult and pediatric patients with R/R acute leukemia harboring a KMT2A rearrangement. About Niktimvo™ (axatilimab-csfr) Niktimvo (axatilimab-csfr) is a first-in-class colony stimulating factor-1 receptor (CSF-1R)-blocking antibody approved for use in the U.S. for the treatment of chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg (88.2 lbs). In 2016, Syndax licensed exclusive worldwide rights to develop and commercialize axatilimab from UCB. In September 2021 , Syndax and Incyte entered into an exclusive worldwide co-development and co-commercialization license agreement for axatilimab in chronic GVHD and any future indications. Axatilimab is being studied in frontline combination trials in chronic GVHD – a Phase 2 combination trial with ruxolitinib (NCT06388564) and a Phase 3 combination trial with steroids (NCT06585774) are underway. Axatilimab is also being studied in an ongoing Phase 2 trial in patients with idiopathic pulmonary fibrosis (NCT06132256). About Syndax Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company advancing innovative cancer therapies. Highlights of the Company's pipeline include Revuforj® (revumenib), an FDA-approved menin inhibitor, and Niktimvo™ (axatilimab-csfr), an FDA-approved monoclonal antibody that blocks the colony stimulating factor 1 (CSF-1) receptor. Fueled by our commitment to reimagining cancer care, Syndax is working to unlock the full potential of its pipeline and is conducting several clinical trials across the continuum of treatment. For more information, please visit www.syndax.com/ or follow the Company on X and LinkedIn. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, relating to our business, operations, and financial conditions, including but not limited to our preliminary financial results for the fourth quarter and full year 2025 for both Revuforj and Niktimvo, preliminary year-end 2025 cash, cash equivalents, restricted cash and marketable securities, current beliefs, as well as expectations and assumptions regarding the future of our business, future plans and strategies, our development and commercialization plans. Words such as "anticipate," "believe," "could," "estimate," "expects," "intend," "may," "plan," "potential," "predict," "project," "should," "will," "would" or the negative or plural of those terms, and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These forward-looking statements are based on Syndax's expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties. Actual results may differ materially from these forward-looking statements. Forward-looking statements contained in this press release include, but are not limited to, statements about the progress, timing, clinical development and scope of clinical trials, the reporting of clinical data for Syndax's product candidates, the acceptance of Syndax and its partners' products in the marketplace, sales, marketing, manufacturing and distribution requirements, and the potential use of its product candidates to treat various cancer indications and fibrotic diseases. Many factors may cause differences between current expectations and actual results, including: unexpected safety or efficacy data observed during preclinical or clinical trials; clinical trial site activation or enrollment rates that are lower than expected; changes to Revuforj's or Niktimvo’s commercial availability; changes in expected or existing competition; changes in the regulatory environment; failure of Syndax's collaborators to support or advance collaborations or product candidates; and unexpected litigation or other disputes. Other factors that may cause Syndax's actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Syndax's filings with the U.S. Securities and Exchange Commission, including the "Risk Factors" sections contained therein. Except as required by law, Syndax assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available. Niktimvo is a trademark of Incyte.All other trademarks are the property of their respective owners. Contact Sharon Klahre Syndax Pharmaceuticals, Inc. sklahre@syndax.com Tel 781.684.9827 SNDX-G Source: Syndax Pharmaceuticals, Inc.