Syndax Announces Publication of Revumenib Data from the BEAT AML Trial in the Journal of Clinical Oncology and Simultaneous Presentation at EHA 2025

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]