Study summary

A menin-blocking pill brought relapsed NPM1-mutated AML into remission

AUGMENT-101 trial (NPM1) · Phase 2, single-arm · Published August 2025, Blood; approved 2025 (Revuforj)

46.9%of tumors responded

What was tested

Revumenib, a pill that blocks menin, a protein NPM1-mutated and KMT2A-rearranged leukemias depend on, for relapsed or refractory NPM1-mutated AML.

What they found

46.9% responded, and some went on to stem cell transplant.
Differentiation syndrome, a treatable but serious side effect, is a known risk.

What it means

Menin inhibitors (revumenib, ziftomenib) are now approved after relapse and are in phase 3 trials as first treatment.

Blood, via PubMed ↗ ← Back to the outlook

Last reviewed 2026-11-14 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.