NEW YORK, Aug. 26, 2025 (GLOBE NEWSWIRE) -- Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company advancing innovative cancer therapies, today announced that Michael A. Metzger, Chief Executive Officer, as well as members of the Syndax management team, will participate in the following upcoming investor conferences: Citi’s 2025 BioPharma Back to School Conference with a fireside chat on Tuesday, September 2, 2025, at 4:45 p.m. ET.H.C. Wainwright 27th Annual Global Investment Co ...

Core Insights - Syndax Pharmaceuticals reported strong financial results for Q2 2025, with significant growth in net revenues from its products Revuforj and Niktimvo, indicating a positive trajectory towards profitability [2][3][4]. Financial Performance - Revuforj (revumenib) generated $28.6 million in net revenue for Q2 2025, reflecting a 43% increase compared to Q1 2025 [4][8]. - Niktimvo (axatilimab-csfr) achieved $36.2 million in net revenue in its first full quarter post-launch, with Syndax recording $9.4 million in collaboration revenue from this product [3][11]. - Total revenue for Q2 2025 was reported at $38.0 million, combining Revuforj and Niktimvo revenues [8]. - The company had cash, cash equivalents, and investments totaling $517.9 million as of June 30, 2025, which is expected to support operations until profitability [7][14]. Product Updates - Revuforj is under Priority Review by the FDA for the treatment of relapsed or refractory mutant NPM1 acute myeloid leukemia (mNPM1 AML), with a PDUFA action date set for October 25, 2025 [4][17]. - Niktimvo is positioned as a treatment for chronic graft-versus-host disease (GVHD) and has shown rapid sales growth since its launch [11][21]. Clinical Trials and Research - Multiple ongoing clinical trials for revumenib are evaluating its efficacy in various acute leukemia settings, including pivotal Phase 3 trials [5][18]. - Recent data from the AUGMENT-101 trial indicated an overall response rate of 48% for patients with R/R mNPM1 AML, with a complete remission rate of 26% [4][5]. Expense Management - Research and development expenses increased to $62.2 million in Q2 2025, up from $48.7 million in the same period last year, primarily due to costs associated with revumenib trials [9]. - Selling, general, and administrative expenses rose to $43.8 million from $29.1 million year-over-year, reflecting increased marketing efforts for Revuforj [10]. Future Outlook - The company anticipates stable operating expenses over the next few years, which, combined with product revenues, is expected to lead to profitability [14]. - For Q3 2025, total expenses are projected to be between $95 million and $100 million, excluding non-cash stock compensation [13].

Company Overview - Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company focused on advancing innovative cancer therapies [3] - The company'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] Upcoming Financial Results - Syndax Pharmaceuticals will report its second quarter 2025 financial results and provide a business update on August 4, 2025 [1] - A conference call and live audio webcast will be hosted by the management at 4:30 p.m. ET on the same day [1] Accessing the Conference Call - The live audio webcast and accompanying slides can be accessed through the Events & Presentations page on the company's website [2] - Domestic and international dial-in numbers are provided for the conference call, with a specific Conference ID: Syndax2Q25 [2] - A replay of the conference call will be available approximately 24 hours after the event and will remain accessible for 90 days [2]

Core Insights - The FDA has granted Priority Review for Syndax Pharmaceuticals' supplemental New Drug Application (sNDA) for Revuforj® (revumenib) targeting relapsed or refractory (R/R) mutant NPM1 (mNPM1) acute myeloid leukemia (AML) with a PDUFA action date set for October 25, 2025 [1][5] - Revuforj is positioned to be the first and only menin inhibitor approved for both R/R mNPM1 AML and R/R KMT2Ar acute leukemia, expanding its indication from its initial approval in 2024 [2][5] - The sNDA is supported by positive pivotal data from the AUGMENT-101 trial, with results published in the journal Blood and presented at the European Hematology Association (EHA) Annual Congress Meeting in June 2025 [2][5] Company Overview - Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company focused on innovative cancer therapies, with Revuforj being a first-in-class oral menin inhibitor [4][22] - The company aims to lead in the therapeutic class of menin inhibitors, supported by compelling data and established relationships with clinicians and payers [2][22] Industry Context - Mutant NPM1 (mNPM1) AML is characterized by mutations in the NPM1 gene, occurring in approximately 30% of adult AML cases, and is associated with high relapse rates and poor prognosis [3] - There are currently no approved therapies that selectively target the underlying mechanisms of mNPM1 AML, highlighting a critical unmet need in the treatment landscape [3]

Core Insights - Revumenib, in combination with venetoclax and azacitidine, shows promising clinical activity in older patients with newly diagnosed mNPM1 and KMT2Ar AML, achieving a 67% complete remission (CR) rate and an 88% overall response rate (ORR) [1][2][6] - The ongoing Phase 3 EVOLVE-2 trial is evaluating the efficacy of this combination in newly diagnosed mNPM1 AML patients who are unfit for intensive chemotherapy [1][2] Group 1: Clinical Trial Results - The BEAT AML trial reported a 67% CR rate (29 out of 43 patients) and an 88% ORR (38 out of 43 patients) among newly diagnosed older adults with mNPM1 or KMT2Ar AML [1][6] - Among 37 patients assessed for measurable residual disease (MRD), 100% were found to be MRD negative [6] - The median duration of composite complete remission (CRc) was 12.0 months [6] Group 2: Patient Demographics and Safety - As of September 2024, 43 patients were enrolled, with 79% having mNPM1 AML and 21% KMT2Ar AML; the median age was 70 years [4][5] - Revumenib was generally well tolerated, with common non-hematologic treatment-emergent adverse events including nausea (60%), constipation (53%), and QTc prolongation (44%) [5][6] Group 3: Comparative Outcomes - The observed median overall survival (OS) was 15.5 months, with a CRc rate of 77% in mNPM1 patients and 89% in KMT2Ar patients [7][8] - Historical data indicated lower CRc rates and median OS for patients treated with venetoclax and azacitidine alone, highlighting the potential of revumenib in improving outcomes [8] Group 4: Future Developments - Revumenib is being developed for the treatment of relapsed or refractory AML with mNPM1 mutations, with a supplemental NDA submitted in April 2025 [10] - The company is conducting multiple trials of revumenib in combination with standard-of-care agents across various treatment settings [10][25]

Core Insights - Syndax Pharmaceuticals announced new data from the pivotal AUGMENT-101 trial of Revuforj (revumenib), a first-in-class menin inhibitor, for patients with relapsed or refractory (R/R) mutant NPM1 (mNPM1) and NUP98-rearranged (NUP98r) acute myeloid leukemia (AML) [1][2][16] Efficacy Results - In the efficacy-evaluable pivotal R/R mNPM1 AML population, the complete remission plus complete remission with partial hematologic recovery (CR+CRh) rate was 26% (20/77) and the overall response rate (ORR) was 48% (37/77) [1][5][4] - Robust responses were observed across various subgroups, regardless of co-mutations, number of prior lines of therapy, or prior venetoclax exposure [7][2] - Among patients with R/R NUP98r AML, 60% (3/5) achieved morphological remission [10] Survival Data - The median overall survival (OS) for all efficacy-evaluable Phase 2 R/R mNPM1 AML patients was 4.8 months, while responders had a median OS of 23.3 months [6][6] Safety Profile - Revumenib was generally well-tolerated, with only 4.8% (4/84) of patients discontinuing treatment due to treatment-related adverse events [8] - The safety profile was consistent with previously reported data, with differentiation syndrome occurring in 29% of patients [19][24] Future Developments - The company submitted a supplemental NDA for Revuforj for R/R mNPM1 AML, building on the FDA approval for R/R acute leukemia with a KMT2A translocation [2][16] - Ongoing and planned trials include combinations of revumenib with standard-of-care agents in various AML populations [11][16]

Group 1 - Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company focused on innovative cancer therapies [2] - The company'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 [2] - Syndax is committed to advancing cancer care and is conducting several clinical trials across various treatment stages [2] Group 2 - Michael A. Metzger, CEO of Syndax, and the management team will participate in investor conferences, including the Jefferies Global Healthcare Conference on June 5, 2025, and the Goldman Sachs 46th Annual Global Healthcare Conference on June 11, 2025 [1][3] - A live webcast of the fireside chats will be available on the company's website, with replays accessible for a limited time [1]

Core Insights - Syndax Pharmaceuticals has appointed Dr. Nicholas Botwood as the new Head of Research and Development and Chief Medical Officer, bringing over 25 years of experience in oncology drug development [1][2] - Dr. Botwood previously held senior roles at Bristol Myers Squibb, where he led medical oncology and was responsible for the medical strategy in the U.S. [2][3] - The company aims to leverage Dr. Botwood's expertise to accelerate the growth of its oncology franchises and enhance its R&D strategy [2][3] Company Overview - Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company focused on innovative cancer therapies [4] - The company's pipeline includes FDA-approved products such as Revuforj® (revumenib) and Niktimvo™ (axatilimab-csfr) [4] - Syndax is committed to advancing cancer care through ongoing clinical trials and aims to unlock the full potential of its product pipeline [4]

Core Insights - Syndax Pharmaceuticals announced positive results from the pivotal Phase 2 portion of the AUGMENT-101 trial for revumenib in relapsed or refractory mutant NPM1 acute myeloid leukemia (mNPM1 AML) patients, achieving nearly 50% overall response rate [1][2] - The company submitted a supplemental New Drug Application (sNDA) for revumenib in April 2025 under the FDA's Real-Time Oncology Review (RTOR) program [1][2] Group 1: Trial Results - The primary efficacy endpoint was met with a complete remission (CR) plus CR with partial hematological recovery (CRh) rate of 23% among the first 64 adult patients [5] - The overall response rate (ORR) was reported at 47%, with 17% of responders proceeding to hematopoietic stem cell transplant (HSCT) while in remission [6] - The median overall survival (OS) for all patients was 4.0 months, while responders had a median OS of 23.3 months [7] Group 2: Patient Demographics and Treatment Background - The efficacy-evaluable population had a median age of 65, with 36% having received three or more prior lines of therapy [4] - 75% of patients were previously treated with venetoclax, indicating a heavily pretreated population [4] Group 3: Safety Profile - The safety population included 84 patients, with treatment-emergent serious adverse events occurring in ≥5% of patients, including febrile neutropenia (21%) and differentiation syndrome (13%) [8] - The safety profile of revumenib was consistent with previously reported data, with 12% of patients experiencing dose reductions due to adverse events [8] Group 4: Background on mNPM1 AML - Mutations in the NPM1 gene are the most common genetic alteration in adult AML, observed in approximately 30% of cases [9] - There are currently no approved targeted therapies specifically for mNPM1 AML, highlighting a significant unmet medical need [9] Group 5: Revumenib Overview - Revumenib is an oral, first-in-class selective menin inhibitor, previously approved for R/R acute leukemia with KMT2A translocation [10][11] - The drug is in development for R/R mNPM1 AML, with ongoing trials planned for combination therapies [11]

Financial Performance - Syndax Pharmaceuticals reported $20.0 million in net revenue from Revuforj® (revumenib) in the first full quarter of its launch [1][4] - Niktimvo™ (axatilimab-csfr) generated $13.6 million in net revenue in its first partial quarter of launch, as reported by Incyte [1][8] - The company had cash, cash equivalents, and investments totaling $602.1 million as of March 31, 2025, which is expected to fund operations until profitability [1][7] Business Developments - The company submitted a supplemental New Drug Application (sNDA) for revumenib for the treatment of relapsed or refractory (R/R) mutant NPM1 acute myeloid leukemia (AML) [1][4] - A pivotal frontline trial of revumenib in combination with venetoclax and azacitidine for mNPM1 and KMT2Ar AML has been initiated [1][4] - Multiple ongoing trials are evaluating revumenib in various combinations and settings, including a Phase 1 trial showing a 100% overall response rate in newly diagnosed mNPM1 or KMT2Ar AML patients [4][5] Research and Development - Research and development expenses for the first quarter of 2025 increased to $61.6 million from $56.5 million in the prior year, driven by costs related to axatilimab and ongoing clinical trials [10] - Selling, general, and administrative expenses rose to $41.0 million from $23.0 million, primarily due to increased employee-related expenses and marketing efforts for Revuforj [11] - The company plans to initiate multiple trials of revumenib in combination with standard care regimens for newly diagnosed acute leukemia patients starting in the second half of 2025 [5][10] Future Outlook - For the second quarter of 2025, the company expects research and development expenses to be between $70 million and $75 million, with total operating expenses projected to be between $110 million and $115 million [13] - The company anticipates that its current cash position, along with expected product revenue, will enable it to reach profitability [14]