# AUGMENT-101

Source: https://onco.cc/trials/augment-101/  
OnCo record `augment-101` (Trial). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

The trial that turned menin inhibition from an idea into the first approved drug for KMT2A-rearranged leukaemia.

## Summary

KMT2Ar cohort (n=57 efficacy population): CR+CRh 22.8%, ORR 63.2%, most CR/CRh MRD-negative; median OS ~8 months. JCO 2024. Led to November 2024 approval in KMT2Ar acute leukaemia (adults and children ≥1 year) and, with the NPM1 cohort, to the October 2025 NPM1 approval. Differentiation syndrome and QT prolongation were the key toxicities; MEN1 resistance mutations emerged in about a third of relapsing patients.

## Fields

- Kind: Trial
- Status: positive
- Last checked: 2026-09-07
- Registry id: NCT04065399
- Phase: 1/2
- Setting: Relapsed/refractory KMT2A-rearranged or NPM1-mutated acute leukaemia: revumenib monotherapy
- Sponsor: Syndax
- Enrolled: 94
- Result: CR+CRh 22.8%, ORR 63.2% in KMT2Ar cohort.
- Outcomes: CR + CRh (KMT2Ar cohort): Revumenib 22.8%; Overall response rate: Revumenib 63.2%
- Replication: KOMET-001 (ziftomenib, a different menin inhibitor) produced a similar CR rate in NPM1-mutated AML, confirming the target.

## Sources

- ClinicalTrials.gov NCT04065399: https://clinicaltrials.gov/study/NCT04065399

## Connected records

- cancers: [Acute lymphoblastic leukaemia](https://onco.cc/cancers/all-leukemia/), [Acute myeloid leukaemia](https://onco.cc/cancers/aml/), [Acute myeloid leukaemia in children](https://onco.cc/cancers/aml-paediatric/), [Infant acute lymphoblastic leukaemia (KMT2A-rearranged, under one year)](https://onco.cc/cancers/all-infant/), [NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia](https://onco.cc/cancers/aml-npm1-kmt2a/), [Relapsed and refractory acute lymphoblastic leukaemia in children](https://onco.cc/cancers/all-paediatric-relapsed/)
- technologies: [Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET)](https://onco.cc/technologies/epigenetic-drugs/)
- targets: [KMT2A (MLL) rearrangement](https://onco.cc/targets/kmt2a/), [Menin](https://onco.cc/targets/menin/), [NPM1 mutation](https://onco.cc/targets/npm1/)
- drugs: [Revumenib](https://onco.cc/drugs/revumenib/)
- companies: [Syndax Pharmaceuticals](https://onco.cc/companies/syndax/)
- terms: [Differentiation syndrome](https://onco.cc/terms/differentiation-syndrome/)
- people: [Eytan M. Stein](https://onco.cc/people/eytan-stein/), [Ghayas C. Issa](https://onco.cc/people/ghayas-issa/)
- ideas: [Menin inhibitors for infant KMT2A-rearranged ALL](https://onco.cc/ideas/idea-menin-infant-all/)
- key papers: [AUGMENT-101: revumenib, the first menin inhibitor, in relapsed leukaemias driven by KMT2A rearrangement or NPM1 mutation](https://onco.cc/key-papers/paper-augment-101-revumenib-menin-nature-2023/)
- roadmaps: [Epigenetic therapy roadmap: loosening silenced genes → mutation-specific enzymes → editing the epigenome](https://onco.cc/roadmaps/epigenetics-roadmap/)

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