Revumenib

Arellano M. L, et al. Blood, 2025.

In the AUGMENT-101 study, Revumenib was investigated for its therapeutic potential in relapsed or refractory (R/R) NPM1-mutated (NPM1m) acute myeloid leukemia (AML). The phase 2 portion of this study enrolled 84 patients, with a median age of 63 years, who had received at least one dose of Revumenib administered every 12 hours in 28-day cycles. Experimental methods included stratifying patients with or without strong CYP3A4 inhibitors and carefully monitoring for complete remission (CR) or CR with partial hematologic recovery (CRh). Key endpoints comprised CR+CRh rates, overall response rate (ORR), and safety profiles. The primary analysis was conducted on 64 adult patients, revealing a CR+CRh rate of 23.4% (P=.0014, 1-sided) and an ORR of 46.9%. Median CR+CRh duration was 4.7 months. Notably, five responders proceeded to hematopoietic stem cell transplantation, indicating clinically significant remissions. Revumenib's tolerability and efficacy, particularly in a heavily pretreated and older patient cohort, underscore its therapeutic potential in targeting NPM1m AML through menin inhibition. These findings establish Revumenib as a promising candidate for further clinical exploration in AML therapy.