Syndax reports publication of revumenib data from BEAT AML trial

Syndax (SNDX) Pharmaceuticals announced that data from the BEAT AML trial of revumenib in combination with venetoclax and azacitidine in newly diagnosed mutant NPM1 and KMT2A-rearranged acute myeloid leukemia patients were published in the Journal of Clinical Oncology and simultaneously presented in an oral session at the 30th European Hematology Association Annual Congress Meeting being held June 12-15, 2025, in Milan, Italy and virtually. The publication and EHA presentation report updated results from the Phase 1b BEAT AML trial evaluating the safety and clinical activity of revumenib in combination with venetoclax/azacitidine in newly diagnosed older adults with mNPM1 or KMT2Ar AML. The trial is being conducted as part of The Leukemia & Lymphoma Society’s Beat AML Master Clinical Trial. As of September 2024, 43 patients were enrolled and treated in BEAT AML across two dose levels of revumenib in combination with venetoclax and azacitidine. Overall, 79% of patients had mNPM1 AML and 21% had KMT2Ar AML. Revumenib was generally well tolerated at both dose levels in combination with venetoclax and azacitidine without a maximal tolerated dose identified. The most common overall non-hematologic treatment-emergent adverse events (TEAEs) of any grade were nausea (60%), constipation (53%), QTc prolongation (44%), hypokalemia (44%), and vomiting (42%). Overall Grade greater than or equal to3 non-hematologic AEs were rare and similar between both dose levels. In the intent-to-treat population, the observed rate of complete remission was 67%, composite complete remission was 81%, and the overall response rate was 88%. Among 37 patients with measurable residual disease response assessment, 100% were MRD negative by centralized flow cytometry testing. The median duration of CRc was 12.0 months. 23% of patients had proceeded to hematopoietic stem cell transplantation as of the February 2025 data cut off. In an early analysis of survival from this single-arm trial, the median overall survival observed was 15.5 months. Subset analysis showed a CRc rate of 77% and an observed median OS of 15.5 months in mNPM1 patients with intermediate risk by ELN 2024, and a CRc rate of 89% and observed median OS of 18.0 months in KMT2Ar patients. In contrast, historical data from newly diagnosed mNPM1 patients with intermediate risk treated with venetoclax and azacitidine show a CRc of 57% and median OS of 9.9 months.1 In newly diagnosed KMT2Ar AML patients treated with venetoclax and hypomethylating agent therapy, a CRc rate of 43% and median OS of 2.5 months was observed in a retrospective analysis.