Decitabine

– Total revenue of $68.7 million in 4Q25 and $172.4 million in FY2025 – – Revuforj® (revumenib) net revenue of $44.2 million in 4Q25, a 38% increase vs 3Q25, and $124.8 million in FY2025 – – Niktimvo™ (axatilimab-csfr) net revenue of $56.0 million in 4Q25, a 22% increase vs 3Q25, and $151.6 million in FY2025, resulting in Syndax collaboration revenue of $42.4 million in FY2025 – – Completed enrollment in Phase 2 IPF trial of axatilimab; topline data expected in 4Q26 – – Company to host a conference call today at 4:30 p.m. ET – NEW YORK , Feb. 26, 2026 (GLOBE NEWSWIRE) -- Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company advancing innovative cancer therapies, today reported its financial results for the fourth quarter and full year ended December 31, 2025 , and provided a business update. “We solidified our leadership position and proved the strength of Syndax’s R&D and commercial capabilities in 2025, achieving our third FDA approval and successfully launching two first- and best-in-class medicines. We reached thousands of patients with Revuforj and Niktimvo and generated over $275 million in 2025 sales, rapidly advancing the company towards profitability,” said Michael A. Metzger , Chief Executive Officer. “With strong momentum and multiple growth drivers for both products, including increasing uptake of Revuforj in R/R NPM1m AML and the post-transplant setting, Syndax is well positioned for continued growth in 2026 and beyond.” Mr. Metzger continued, “We’ve also made excellent progress advancing our development programs designed to further unlock multi-billion-dollar opportunities for both our medicines. We are positioned to be first to frontline AML with a menin inhibitor, and to expand our impact on chronic GVHD and other fibrotic diseases through CSF-1R inhibition. Earlier this year, we completed enrollment in our Phase 2 IPF trial and remain on track for topline data later this year which could further unlock Niktimvo’s potential as a novel antifibrotic.” Recent Business Highlights and Anticipated Milestones Revuforj® (revumenib) Achieved $44.2 million in Revuforj net revenue in the fourth quarter of 2025, a 38% increase over the third quarter of 2025. Revuforj net revenue for the full year 2025 totaled $124.8 million . Observed continued acceleration in demand following the FDA’s approval on October 24, 2025 , of Revuforj for the treatment of relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible nucleophosmin 1 mutation (NPM1m) in adult and pediatric patients one year and older who have no satisfactory alternative treatment options. Total Revuforj prescriptions in the fourth quarter of 2025 were approximately 1,150, an approximate 35% increase over the third quarter of 2025. 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. Received the ‘Best New Drug’ award for Revuforj at the Scrip Awards in December 2025 . The award recognizes excellence in pharmaceutical development and the drug that represents the best therapeutic advance in its area. Highlighted the company’s leadership in menin inhibition with the presentation of 12 revumenib abstracts, including three oral presentations, at the 67th American Society of Hematology (ASH) Annual Meeting in December 2025 . The presentations reported compelling results with revumenib in multiple acute leukemia subtypes across the R/R, frontline, and post-stem cell transplant maintenance setting. The data presented included the first real-world evidence for a menin inhibitor. The results showed an overall response rate (ORR) of 77% (10/13), a measurable residual disease (MRD) negativity rate of 75% (9/12), and favorable tolerability among primarily R/R NPM1m, KMT2Ar, and NUP98r acute leukemia patients who received revumenib in combination with standard of care therapies or as a monotherapy outside of a clinical trial. Multiple clinical trials evaluating revumenib across the acute leukemia treatment continuum are ongoing, such as: EVOLVE-2: A pivotal, Phase 3, randomized, double-blind, placebo-controlled trial of revumenib in combination with venetoclax and azacitidine in newly diagnosed NPM1m (primary efficacy analysis population) and KMT2Ar AML patients who are unfit for intensive chemotherapy. The trial is being conducted in collaboration with the HOVON network, a leading cooperative clinical trial group with extensive experience studying novel therapies for hematologic malignancies. REVEAL-ND: A pivotal, Phase 3, randomized, double-blind, placebo-controlled trial of revumenib in combination with intensive chemotherapy in newly diagnosed NPM1m AML patients. SAVE: A Phase 1/2 trial evaluating an all-oral combination of revumenib with venetoclax and decitabine/cedazuridine in pediatric and adult patients with newly diagnosed and R/R AML or mixed-lineage acute leukemia (MPAL) harboring either NPM1m, KMT2Ar, or NUP98r alterations. The trial is being conducted by investigators from MD Anderson Cancer Center. New data presented at the 2025 ASH Annual Meeting from the first cohort of newly diagnosed patients showed a complete remission (CR) rate of 76% (16/21), an ORR of 86% (18/21), and a MRD negativity rate among responders of 100% (18/18). Intensive chemotherapy: Two ongoing Phase 1 trials evaluating the combination of revumenib with intensive chemotherapy (7+3) in newly diagnosed NPM1m or KMT2Ar acute leukemia patients. Preliminary data presented from both trials at the 2025 ASH Annual Meeting showed that the safety profile of the combination was consistent with the profile for intensive chemotherapy alone, along with high rates of response and MRD negativity. BEAT AML: A Phase 1 trial evaluating the combination of revumenib with venetoclax and azacitidine in newly diagnosed older adults (≥60 years) with NPM1m or KMT2Ar AML. The trial is being conducted as part of the Leukemia & Lymphoma Society's Beat AML® Master Clinical Trial. Data presented at the 2025 European Hematology Association (EHA) Congress and simultaneously published in the Journal of Clinical Oncology showed a CR rate of 67% (29/43), an ORR of 88% (38/43), and a MRD negativity rate of 100% (37/37) among responders. Post-transplant maintenance: A Phase 1 trial evaluating the safety and preliminary efficacy of revumenib as post-transplant maintenance after hematopoietic stem cell transplant (HSCT) in patients with KMT2Ar or NPM1m acute leukemia. The trial is being conducted by investigators from the City of Hope Medical Center . Break Through Cancer: A Phase 2 trial studying whether the combination of revumenib and venetoclax can eliminate MRD in patients with AML and extend progression-free survival. The trial is being conducted by Break Through Cancer, a collaboration between leading U.S. cancer research centers. INTERCEPT: A Phase 1 trial evaluating the use of novel therapies, including revumenib, to target MRD and early relapse in AML. The trial is being conducted by the Australasian Leukaemia and Lymphoma Group as part of the INTERCEPT AML master clinical trial. The Company expects to present additional revumenib data at major medical meetings throughout 2026, including additional real-world evidence and data from the frontline and post-HSCT maintenance setting. The Company expects the RAVEN trial to initiate in the second half of 2026. RAVEN is 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. Niktimvo™ (axatilimab-csfr) Achieved $56.0 million in Niktimvo net revenue in the fourth quarter of 2025, a 22% increase over the third quarter of 2025. Niktimvo net revenue for the full year 2025 totaled $151.6 million . Syndax and Incyte are co-commercializing Niktimvo. Syndax records 50% of the Niktimvo net commercial profit, defined as net product revenue minus the cost of sales and commercial expenses. Syndax’s share of the Niktimvo product contribution, reported as collaboration revenue, was $19.4 million and $42.4 million in the fourth quarter and full year 2025, respectively. Presented data from 11 axatilimab abstracts, including three oral presentations, at the 2025 ASH Annual Meeting. The abstracts highlighted the potential for axatilimab to provide long-term benefit in recurrent or refractory chronic graft-versus-host disease (GVHD) and the tolerability of axatilimab with ruxolitinib in newly diagnosed chronic GVHD. Presented data from nine axatilimab abstracts, including one oral presentation, at the Tandem Meetings (Transplantation & Cellular Therapy Meetings of ASTCT® and CIBMTR®) in February 2026 . The data presented included a comprehensive analysis of axatilimab in patients with chronic GVHD-related bronchiolitis obliterans syndrome (BOS) in two clinical studies. The results show clinical and symptom responses across a spectrum of lung involvement. Two trials evaluating axatilimab in combination with standard of care therapies in newly diagnosed chronic GVHD patients are ongoing, including: A Phase 2, open-label, randomized, multicenter trial of axatilimab in combination with ruxolitinib in patients ≥ 12 years of age with newly diagnosed chronic GVHD. A pivotal Phase 3, randomized, double-blind, placebo-controlled, multi-center trial of axatilimab in combination with corticosteroids in patients ≥ 12 years of age with newly diagnosed chronic GVHD. Completed enrollment in MAXPIRe, a Phase 2, 26-week randomized, double-blinded, placebo-controlled trial of axatilimab on top of standard of care in patients with idiopathic pulmonary fibrosis (IPF) in the first quarter of 2026. The Company expects to report topline data in the fourth quarter of 2026. Fourth Quarter and Full Year 2025 Financial Results As of December 31, 2025 , Syndax had cash, cash equivalents, and short-term investments of $394.1 million and 87.7 million common shares and prefunded warrants outstanding. Total revenue for the fourth quarter of 2025 was $68.7 million , which consisted of $44.2 million in Revuforj net revenue, $19.4 million in Niktimvo collaboration revenue, and $5.1 million in milestone, license and royalty revenue. Total revenue for the full year 2025 was $172.4 million , which consisted of $124.8 million in Revuforj net revenue, $42.4 million in Niktimvo collaboration revenue, and $5.1 million in milestone, license and royalty revenue. The Niktimvo collaboration revenue is derived from the $151.6 million in Niktimvo net revenue that was previously reported by the Company's partner Incyte for the full year 2025. Syndax records 50% of the Niktimvo net commercial profit, defined as net revenue (recorded by Incyte) minus the cost of sales and commercial expenses. Fourth quarter 2025 research and development expenses increased to $78.6 million from $65.5 million for the comparable prior year period, and for the full year 2025 increased to $258.8 million compared to $241.6 million for 2024. The year-over-year increase was primarily due to increased clinical, medical, and employee-related expenses. Fourth quarter 2025 selling, general and administrative expenses increased to $49.9 million from $37.7 million for the comparable prior year period, and for the full year 2025 increased to $179.7 million compared to $120.9 million for 2024. The year-over-year increase was primarily due to increased employee-related expenses and increased sales and marketing expenses related to the U.S. commercial launches of Revuforj and Niktimvo. For the three months ended December 31, 2025 , Syndax reported a net loss attributable to common stockholders of $68.0 million , or $0.78 per share, compared to a net loss attributable to common stockholders of $94.2 million , or $1.10 per share, for the comparable prior year period. For the year ended December 31, 2025, Syndax reported a net loss attributable to common stockholders of $285.4 million or $3.29 per share, compared to a net loss attributable to common stockholders of $318.8 million or $3.72 per share for the comparable prior year period. Financial Guidance 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 operating expense base will remain stable over the next couple of years. As a result, Syndax expects that its cash, cash equivalents and short-term investments, combined with its anticipated product revenue, collaboration revenue and interest income, will enable the Company to reach profitability. Conference Call and Webcast In connection with the earnings release, Syndax's management team will host a conference call and live audio webcast at 4:30 p.m. ET today, Thursday, February 26, 2026 . The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company's website. Alternatively, the conference call may be accessed through the following: Conference ID: Syndax4Q25 Domestic Dial -in Number: (800) 590-8290International Dial-in Number: (240) 690-8800Live webcast: https://sndx-4q25.open-exchange.net For those unable to participate in the conference call or webcast, a replay will be available on the Investors section of the Company's website at www.syndax.com approximately 24 hours after the conference call and will be available for 90 days following the call. 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, including a Phase 2 combination trial with ruxolitinib (NCT06388564) and a Phase 3 combination trial with steroids (NCT06585774). 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. 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, the potential use of its product candidates to treat various cancer indications and fibrotic diseases, and Syndax's expected full year total operating expenses, including its estimated non-cash stock compensation expense. 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. References 1. Overall response rate (ORR) includes CR, CRh, CRp, CRi, MLFS, and PR; Composite complete remission (CRc) includes CR, CRh, CRp, and CRi.CR = Complete remissionCRh = Complete remission with partial hematologic recoveryCRp = Complete remission with incomplete platelet recoveryCRi = Complete remission with incomplete count recoveryMLFS = Morphologic leukemia-free statePR = Partial response Syndax Contact Sharon Klahre Syndax Pharmaceuticals, Inc. sklahre@syndax.com Tel 781.684.9827 SNDX-G Source: Syndax Pharmaceuticals, Inc.

白血病的最新治疗进展有哪些？ 白血病的最新治疗进展正以前所未有的速度重塑临床实践，其核心已从传统的“地毯式轰炸”化疗，全面转向以精准分层、靶向治疗、免疫疗法和细胞治疗为核心的“精准歼灭”时代。其进展可归纳为以下几个关键方向。 一、精准靶向治疗：针对特定基因突变的“精确制导” 这是当前进展最迅速、成果最丰硕的领域，尤其体现在急性髓系白血病（AML）的治疗中。新药研发和联合策略正不断填补治疗空白。 1. 针对特定突变的新药获批与应用：2025年，美国FDA批准了两种Menin抑制剂（Revumenib和Ziftomenib），用于治疗携带NPM1突变的复发/难治性AML患者，为这类预后不佳的患者提供了全新的靶向选择。同时，针对IDH1/2突变的抑制剂（如Ivosidenib、Enasidenib）和针对FLT3-ITD突变的抑制剂（如吉瑞替尼、奎扎替尼）已成为标准治疗的一部分，显著改善了相应突变患者的预后。最新的临床试验显示，IDH1抑制剂Olutasidenib联合阿扎胞苷，在复发难治性IDH1突变AML患者中实现了51%的整体反应率。 2. BCL-2抑制剂的联合治疗深化：以维奈克拉（Venetoclax）为代表的BCL-2抑制剂，与去甲基化药物（如阿扎胞苷）或低剂量化疗联合，已成为老年或不适合强化疗AML患者的一线标准方案。研究进一步通过VEN-PRS风险模型对患者进行精准分层，以预测联合治疗疗效，指导个体化决策。 3. 克服耐药的新机制与联合策略：针对FLT3-ITD抑制剂耐药这一临床瓶颈，2025年的研究揭示了其通过募集STAT1信号通路导致T细胞耗竭的新机制，这为联合使用FLT3抑制剂与CD276抑制剂等免疫治疗提供了理论依据，预示着双靶向联合治疗将成为克服耐药的新方向。 二、免疫与细胞治疗：重塑人体自身的“抗癌军团” 这类疗法通过激活或改造免疫系统，为复发难治患者带来了治愈希望。 1. CAR-T细胞疗法的靶点拓展与突破：CAR-T疗法已超越经典的CD19靶点，向更广谱和更具挑战性的白血病类型进军。针对AML，CLL1靶点CAR-T在中国成功治疗了化疗后转化为AML的儿童患者，实现了癌细胞清零。针对T细胞急性淋巴细胞白血病（T-ALL），CD7靶点CAR-T也成功治愈了复发患者，解决了T细胞肿瘤中“敌我难分”的难题。双靶点CAR-T（如CD19/CD22）等迭代技术正在探索中，以应对靶点逃逸。 2. 双特异性抗体的崛起：这类药物像“智能桥梁”一样将T细胞与肿瘤细胞连接起来。例如，贝林妥欧单抗（CD3×CD19双抗）已成为B细胞急性淋巴细胞白血病（B-ALL）的重要治疗选择。在AML领域，针对CD33等靶点的双特异性抗体也处于积极研发阶段。 三、治疗策略的范式革新：从“延长生存”到“追求治愈” 1. 基于微小残留病（MRD）的精准分层与干预：MRD监测已成为指导治疗决策的核心。在造血干细胞移植（HSCT）领域，移植前追求MRD阴性、移植后对MRD转阳进行“抢先干预”已成为标准策略。研究表明，经维奈克拉治疗后达到MRD阴性的患者，其无进展生存期是未转阴者的2.9倍。 2. 功能性治愈与有限疗程治疗：对于某些特定亚型的患者，如IDH1/NPM1共突变的AML患者，通过靶向药实现深度缓解后长期停药已成为可能，开启了“功能性治愈”的新时代。在慢性淋巴细胞白血病（CLL）中，固定疗程的联合方案（如维奈克拉+奥妥珠单抗）也显示出不劣于持续治疗的疗效，且心脏毒性更低，让患者摆脱终身服药的负担。 3. 移植技术的优化与新药应用：移植预处理方案也在更新，例如FDA于2025年批准了苏消安联合氟达拉滨作为新的预处理方案。同时，将靶向药物（如FLT3抑制剂、IDH1抑制剂）纳入移植前后的桥接或维持治疗，以提升疗效和降低复发风险，已成为临床研究的热点。 四、前沿探索与未来方向 研究正在向更基础的机制和更整合的策略深入。 1. 代谢靶向与肿瘤微环境：研究发现白血病干细胞依赖骨髓微环境提供的牛磺酸等代谢物来增殖，靶向其转运蛋白（TAUT）成为潜在新方向。针对甲硫氨酸、谷氨酰胺等代谢通路的药物也在探索中，通常与现有疗法联合以克服耐药。 2. 新型药物形式与联合疗法：抗体药物偶联物（ADC）、表观遗传学药物（如地西他滨）等与传统治疗或靶向治疗的联合方案不断被验证。针对TP53突变等高危患者，靶向放疗（如抗CD33核素抗体）联合化疗也显示出前景。 总结，白血病治疗已进入一个精准化、个体化、免疫化的新纪元。其进展不仅体现在众多靶向新药和免疫疗法的获批上，更深刻地体现在以MRD指导临床决策、追求功能性治愈、多机制联合克服耐药等治疗范式的根本性转变中。这些进展共同为患者，尤其是难治复发患者，带来了前所未有的生存希望和生活质量提升。