In this study, revumenib exhibited a promising overall response rate of 46.9% in patients with relapsed or refractory NPM1-mutated acute myeloid leukemia. The median overall survival was 4.0 months and the complete remission plus complete remission with hematologic recovery rate was 23.4%. The treatment had a manageable safety profile with notable adverse events including QT prolongation and differentiation syndrome.
Study
|
Phase 1/2 open-label study [AUGMENT-101] |
| Relapsed or refractory NPM1-mutated acute myeloid leukemia |
| Revumenib every 12 hours in 28-day cycles (n=84)
|
Efficacy
|
CR + CRh: 23.4% |
| ORR: 46.9% |
| mOS: 4.0 mos [2.5-7.2] |
| mEFS: 3.0 mos [2.0-3.8] |
| mOS in pts with CR+CRh: 23.3 mos
|
Safety
|
QTcF prolongation (42.9%); differentiation syndrome (19.0%); anemia (27.4%); thrombocytopenia (16.7%) |
| Discontinuation due to AE: 4.8%
|
Blood. Published online 2024 Sep 18.
Arellano ML, Thirman MJ, DiPersio JF New Indication: Revumenib for Rel/Ref NPM1-Mutated AML
http://doi.org/10.1182/blood.2025028357
Reviewed by Ulas D. Bayraktar, MD on Oct 26, 2025
