Syndax Reports 88% Response Rate Revumenib Combo AML Trial

Syndax Pharmaceuticals has reported promising clinical trial results for revumenib, a menin inhibitor, in combination with venetoclax and azacitidine for newly diagnosed mutant NPM1 (mNPM1) and KMT2A-rearranged (KMT2Ar) acute myeloid leukemia (AML) patients. The Phase 1b BEAT AML trial demonstrated a 67% complete remission rate and 88% overall response rate, with all patients assessed showing measurable residual disease negativity, and median overall survival of 15.5 months. The treatment was generally well-tolerated, with common side effects including nausea, constipation, and QTc prolongation, and no maximum tolerated dose identified. Further data from the pivotal AUGMENT-101 trial in relapsed or refractory mNPM1 AML patients showed a 26% complete remission rate and an overall response rate of 48%, with median overall survival reaching 23.3 months for responders. Syndax is continuing to enroll patients in ongoing trials, including the Phase 3 EVOLVE-2 study evaluating revumenib with venetoclax and azacitidine for patients unfit for intensive chemotherapy, highlighting the drug's potential as a cornerstone therapy for menin-dependent acute leukemias. The company maintains a strong financial position with expectations of stock value upside based on analyst forecasts.