- Program will expand access in certain regions where Revuforj is not available commercially, as permitted by local regulations - - Program will be launched in parts of Eurasia, Central and Southeast Europe, Israel, the Middle East and Turkey, Latin America, and Africa - NEW YORK, Jan. 07, 2026 (GLOBE NEWSWIRE) -- Syndax Pharmaceuticals (Nasdaq: SNDX) and the World Orphan Drug Alliance (WODA) today announced a collaboration to expand access to the Company’s first-in-class menin inhibitor, Revuforj® (revume ...

NEW YORK, Jan. 05, 2026 (GLOBE NEWSWIRE) -- Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company advancing innovative cancer therapies, today announced that Michael A. Metzger, Chief Executive Officer, will 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. A live webcast of the presentation will be available in the Investor section of the Company's website at www.syndax.com, where a replay will also be avai ...

Core Insights - Syndax Pharmaceuticals' Revuforj® (revumenib) was awarded Best New Drug at the Scrip Awards 2025, highlighting its significance in pharmaceutical development [1][3] - Revuforj is a first-in-class menin inhibitor, recently approved by the FDA for treating relapsed or refractory acute leukemia with specific genetic mutations [2][4] Company Overview - Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company focused on innovative cancer therapies, including Revuforj and Niktimvo™ [22] - The company is committed to advancing cancer care through its pipeline and ongoing clinical trials [22] Product Details - Revuforj is FDA approved for treating relapsed or refractory acute leukemia with KMT2A translocation and acute myeloid leukemia with NPM1 mutation in patients aged one year and older [2][4] - The drug has received Orphan Drug Designation and Fast Track designation from the FDA, indicating its potential in treating serious conditions [6] Clinical Trials and Future Prospects - Multiple clinical trials for Revuforj are ongoing or planned, including studies in combination with standard therapies for newly diagnosed patients [5] - The company aims to unlock the full potential of its pipeline through continued research and development efforts [22]

Core Insights - Syndax Pharmaceuticals presented new data on Revuforj (revumenib) at the 67th ASH Annual Meeting, highlighting its efficacy and safety in treating acute leukemia, particularly in patients with NPM1m, KMT2Ar, and NUP98r mutations [1][2][3] Efficacy and Safety Data - The overall response rate (ORR) for revumenib was reported at 77% (10/13) in a retrospective review, with a complete remission (CR) rate of 31% (4/13) and 75% (9/12) achieving measurable residual disease (MRD) negativity [1][4][5] - In a Phase 2 SAVE trial, the ORR was 86% (18/21) and the CR rate was 76% (16/21), with 100% MRD negativity among responders [6][11] - Revumenib demonstrated a favorable safety profile, with only 24% (4/17) experiencing Grade 3 or 4 non-hematological adverse events, and low rates of dose reductions and discontinuations at 6% each [5][13] Clinical Trials and Future Directions - Syndax has initiated pivotal frontline trials for revumenib in patients unfit for intensive chemotherapy and in fit patients, aiming to expand clinical data supporting its use [2][23] - Multiple ongoing and planned trials are evaluating revumenib in combination with standard therapies for newly diagnosed patients with specific genetic mutations [22][23] Pediatric Applications - A retrospective review of revumenib as post-HSCT maintenance in children showed that all ten patients were alive at a median follow-up of 19 months, with a 90% relapse-free survival rate [7][8] Company Overview - Syndax Pharmaceuticals is focused on advancing innovative cancer therapies, with Revuforj being a first-in-class menin inhibitor approved for treating relapsed or refractory acute leukemia [22][40]

Core Insights - Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company focused on innovative cancer therapies [3] - The CEO, Michael A. Metzger, will participate in a fireside chat at the 8th Annual Evercore Healthcare Conference on December 4, 2025 [1] - A live webcast of the event will be available on the company's website, along with a replay for a limited time [2] Company Overview - Syndax's pipeline includes FDA-approved therapies such as Revuforj (revumenib), a menin inhibitor, and Niktimvo™ (axatilimab-csfr), a monoclonal antibody targeting the CSF-1 receptor [3] - The company is committed to advancing cancer care and is conducting several clinical trials across various treatment stages [3]

Core Insights - Syndax Pharmaceuticals reported total revenue of $45.9 million for Q3 2025, marking a 21% increase compared to Q2 2025 [1] - The company achieved FDA approval for Revuforj on October 24, 2025, for the treatment of relapsed or refractory NPM1m acute myeloid leukemia (AML) [1][5] - Syndax has $456.1 million in cash and investments, which is expected to fund the company to profitability [1][8] Financial Performance - Revuforj generated $32.0 million in net revenue for Q3 2025, a 12% increase from Q2 2025, with total prescriptions rising by 25% [4][5] - Niktimvo reported $45.8 million in net revenue, with Syndax recording $13.9 million in collaboration revenue from this product [9][11] - The net loss attributable to common stockholders for Q3 2025 was $60.7 million, or $0.70 per share, an improvement from a net loss of $84.1 million, or $0.98 per share, in the same period last year [13][29] Business Highlights - Strong demand for Revuforj and Niktimvo resulted in over $75 million in combined net sales for the quarter [2] - Revuforj was included in the NCCN Guidelines for R/R NPM1m AML as a category 2A recommended treatment option [5] - The company is advancing multiple clinical trials for Revuforj and Niktimvo, targeting various indications and patient populations [10][18] Research and Development - Research and development expenses decreased to $56.3 million in Q3 2025 from $71.0 million in the prior year, primarily due to the absence of a milestone payment related to Niktimvo's approval [11] - Selling, general, and administrative expenses increased to $44.9 million from $31.1 million, driven by higher commercial costs associated with the launches of Revuforj and Niktimvo [12] Future Outlook - The company expects total operating expenses for 2025 to be between $380 million and $385 million, excluding an estimated $45 million in non-cash stock compensation [14] - Syndax anticipates that its current cash reserves, combined with expected product revenue, will enable it to reach profitability [15]

Core Insights - Syndax Pharmaceuticals will host an in-person investor event on December 8, 2025, at 7:00 a.m. ET during the ASH Annual Meeting to discuss data updates from its Revuforj and Niktimvo programs [1] Company Overview - Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company focused on innovative cancer therapies, with key products including Revuforj (revumenib), an FDA-approved menin inhibitor, and Niktimvo™ (axatilimab-csfr), an FDA-approved monoclonal antibody targeting the CSF-1 receptor [3] - The company is committed to advancing its pipeline and is conducting several clinical trials across various treatment stages [3]

Core Insights - Syndax Pharmaceuticals announced the acceptance of 23 abstracts, including six oral presentations, for the 67th American Society of Hematology (ASH) Annual Meeting, highlighting the company's advancements in menin inhibition and CSF-1R inhibition [1][2] Revumenib - Revumenib abstracts demonstrate promising results across various acute leukemia subtypes, including relapsed/refractory (R/R) settings and post-HSCT scenarios [1][2] - New frontline datasets will showcase the tolerability of Revumenib in combination with standard therapies, achieving high rates of complete remission and minimal residual disease (MRD) negativity [2][5] - The first real-world evidence for a menin inhibitor will be presented, along with a retrospective review of its use in post-transplant settings [2][5] - Key presentations include a Phase 2 study of Revumenib combined with venetoclax and decitabine/cedazuridine in newly diagnosed acute myeloid leukemia (AML) [7] - Additional presentations will cover efficacy and safety by leukemia type in patients with R/R KMT2Ar acute leukemia [7] Axatilimab - Axatilimab abstracts will focus on its long-term benefits in R/R chronic graft-versus-host disease (GVHD) and its tolerability when combined with ruxolitinib in newly diagnosed chronic GVHD [1][2] - An oral presentation will detail the safety and feasibility of transitioning dosing from 0.3 mg/kg every 2 weeks to 0.6 mg/kg every 4 weeks in the pivotal Phase 2 AGAVE-201 trial [4][10] - Presentations will also include interim safety analyses from a Phase 2 trial of axatilimab combined with ruxolitinib in newly diagnosed chronic GVHD [8][10] Company Overview - Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company focused on innovative cancer therapies, with a pipeline that includes Revuforj (revumenib) and Niktimvo (axatilimab) [44] - The company is committed to advancing its clinical trials across various treatment landscapes, aiming to unlock the full potential of its product candidates [44]

Core Insights - Syndax Pharmaceuticals will report its third quarter 2025 financial results and provide a business update on November 3, 2025 [1] - A conference call and live audio webcast will be hosted by Syndax's management at 4:30 p.m. ET on the same day [1] Company Overview - Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company focused on innovative cancer therapies [3] - The company's pipeline includes Revuforj (revumenib), an FDA-approved menin inhibitor, and Niktimvo™ (axatilimab-csfr), an FDA-approved monoclonal antibody targeting the CSF-1 receptor [3] - Syndax is committed to advancing cancer care and is conducting several clinical trials across various treatment stages [3]

FDA Approval and Indication - Revuforj获得FDA批准，用于治疗复发或难治性（R/R）急性髓系白血病（AML），该白血病在1岁及以上的成人和儿童患者中具有易感的NPM1突变，且没有令人满意的替代治疗方案[8] - Revuforj还被批准用于治疗具有KMT2A易位的R/R急性白血病，适用于1岁及以上的成人和儿童患者[11] - Revuforj是首个也是唯一获批用于多种急性白血病亚型的menin抑制剂，适用于成人和儿童[8, 12] Clinical Data and Efficacy - AUGMENT-101关键试验数据显示，Revuforj在R/R NPM1m AML患者中，完全缓解（CR）加上伴有部分血液学恢复的CR（CRh）率为23%[22] - AUGMENT-101试验的中位CR/CRh时间为2.8个月，CR/CRh的中位持续时间为4.5个月[22] - AUGMENT-101试验中，47%的患者达到总缓解率[22] - AUGMENT-101试验的CR/CRh患者中，63%达到MRD阴性[22] - 在缓解者亚组分析中观察到23个月的中位总生存期[22] Market Opportunity and Commercial Strategy - Revuforj在美国R/R急性白血病市场拥有超过20亿美元的市场机会[28] - Revuforj在R/R和一线治疗环境中拥有超过50亿美元的美国市场机会[9, 29] - Revuforj有望成为最大的靶向AML疗法，在可寻址的AML人群中占据40-45%的份额[30, 31] - 通过2025年第二季度，Revuforj已实现65%的一级/二级账户渗透率[34] - Revuforj已列入NCCN指南，用于R/R NPM1m AML和KMT2Ar急性白血病[12, 26]