Syndax(US:SNDX) Globenewswire·2025-06-12 11:00Core 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]