Revumenib may be safe and effective in the treatment of nucleophosmin 1-mutated acute myeloid leukemia

1. In pretreated patients with nucleophosmin 1-mutated (NPM1m) acute myeloid leukemia (AML), nearly half of patients treated with revumenib showed a clinically meaningful response with an overall response rate (ORR) of 46.9%. 

Evidence Rating Level: 1 (Excellent)

NPM1 is the most common mutation found in adult AML, yet no targeted therapies for patients with NPM1m AML currently exist. In patients with refractory or relapsed (R/R) NPM1m AML, there is no standard of care and the prognosis of patients in this setting remains poor. Revumenib is a novel agent that was recently approved for the treatment of R/R lysine methyltransferase 2A (KMT2A)-translocated acute leukemia based on data from the phase ½ AUGMENT-101 trial. This study therefore provides primary efficacy data for revumenib in R/R NPM1m AML in the AUGMENT-101 trial. Between October 2021 and September 2024, the first 64 adult patients (median[range] age, 19-84 years; 59.4% female) enrolled in the study were included in the efficacy-evaluable group while 84 patients (median[range] age, 63[11-84] years; 59.5% female) who received at least 1 dose of revumenib and not included in the efficacy-evaluable group comprised the safety population. Patients in the study were heavily pre-treated with 34.5% having received at least 3 previous lines of therapy. The primary efficacy end point was met with the proportion of patients achieving composite complete remission (CRc) being 29.7% (95% CI, 18.9-42.4) and the ORR being 46.9% (95% CI, 34.3-59.8). Four patients (4.8%) required treatment discontinuation due to a treatment-related adverse event. Overall, this study found that among patients with R/R NPM1m AML, revumenib demonstrated a promising efficacy and safety profile. 

Click to read the study in Blood

Image: PD

©2025 2 Minute Medicine, Inc. All rights reserved. No works may be reproduced without expressed written consent from 2 Minute Medicine, Inc. Inquire about licensing here. No article should be construed as medical advice and is not intended as such by the authors or by 2 Minute Medicine, Inc.