Axatilimab-csfr

Sept. 19, 2025 -- Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company advancing innovative cancer therapies, today announced that the National Comprehensive Cancer Network® (NCCN®) Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Acute Myeloid Leukemia were updated to include revumenib as a category 2A recommendation for relapsed or refractory (R/R) acute myeloid leukemia (AML) with an NPM1 mutation (mNPM1).1 The update was based on positive pivotal results from the AUGMENT-101 trial of revumenib which were published in the journal Blood in 2025.2 The NCCN Guidelines for AML and acute lymphoblastic leukemia (ALL) continue to also include revumenib as a category 2A recommendation for R/R acute leukemia with a KMT2A rearrangement. “The inclusion of revumenib as a recommended treatment option for R/R NPM1 mutated AML in the NCCN Guidelines underscores the strength of our clinical data in this population and further solidifies revumenib’s leading position,” said Nick Botwood, MBBS, Head of Research & Development and Chief Medical Officer at Syndax. “Given the pivotal role NCCN Guidelines play in guiding the decision-making process for clinicians, payers, patients, and other key stakeholders in the U.S. and beyond, this is a major milestone for Syndax and the entire acute leukemia community.” The Company has submitted a supplemental New Drug Application (sNDA) seeking the approval of revumenib for the treatment of R/R mNPM1 AML. The sNDA has been granted Priority Review by the FDA and assigned a Prescription Drug User Fee Act (PDUFA) target action date of October 25, 2025. The NCCN is a not-for-profit alliance of 33 leading cancer centers devoted to patient care, research, and education. Through the leadership and expertise of clinical professionals at NCCN Member Institutions, NCCN develops resources that present valuable information to the numerous stakeholders in the health care delivery system. Mutations in the NPM1 gene are the most common genetic alteration observed in AML, occurring in approximately 30% of adults with AML. Mutations in this gene play a critical role in the development of mutant NPM1 (mNPM1) AML, an aggressive blood cancer associated with high rates of relapse. Patients with relapsed or refractory mNPM1 AML have a poor prognosis and critical unmet need. Similar to KMT2A-rearranged acute leukemia, mNPM1 AML is highly dependent on the menin-KMT2A interaction and disruption of this interaction has been shown to lead to downregulation of certain leukemogenic genes. mNPM1 AML is routinely diagnosed through currently available screening techniques. There are currently no approved therapies that selectively target the underlying disease mechanisms driving mNPM1 AML. 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 in adult and pediatric patients one year and older. The FDA has granted Priority Review to Syndax’s supplemental New Drug Application (sNDA) seeking the approval of revumenib for the treatment of R/R mNPM1 AML. The sNDA is being reviewed under the FDA's Real-Time Oncology Review (RTOR) program, which allows for a more efficient review and close engagement between the sponsor and FDA. The sNDA is supported by positive pivotal data from the AUGMENT-101 trial of revumenib in patients with R/R mNPM1 AML. Results from this population were published in the journal Blood and presented at the 2025 European Hematology Association (EHA) Annual Congress Meeting. Additionally, 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 mNPM1 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. 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. References NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Acute Myeloid Leukemia (Version 1.2026 – September 18, 2025); NCCN makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way. Martha L. Arellano, et al. Menin inhibition with revumenib for NPM1-mutated relapsed or refractory acute myeloid leukemia: the AUGMENT-101 study. Blood 2025. https://doi-org.libproxy1.nus.edu.sg/10.1182/blood.2025028357 The content above comes from the network. if any infringement, please contact us to modify.