AUGMENT-101
The trial that turned menin inhibition from an idea into the first approved drug for KMT2A-rearranged leukaemia.
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.
Setting
Relapsed/refractory KMT2A-rearranged or NPM1-mutated acute leukaemia: revumenib monotherapy
Phase
Phase 1/2
Sponsor
Syndax
Registry
Headline result
CR+CRh 22.8%, ORR 63.2% in KMT2Ar cohort.
Reported
2024
Enrolled
94
Replication
KOMET-001 (ziftomenib, a different menin inhibitor) produced a similar CR rate in NPM1-mutated AML, confirming the target.
In plain words
What these results mean for people, not percentages
CR + CRh (KMT2Ar cohort)primarysurrogate endpoint
- 22.8 out of 100 people reached this endpoint with Revumenib.
- This is a surrogate endpoint: it measures the cancer being controlled or absent on scans and tests, which often, but not always, translates into living longer.
Overall response rateresponse endpoint
- 63.2 out of 100 people had their tumour shrink with Revumenib.
- A response rate counts how many people had their tumours shrink by a set amount; shrinking is encouraging but does not by itself show that people live longer or feel better.
Be careful
- There was no comparison group, so this number cannot tell you how much of the effect is due to the treatment.
- These results apply to the people the trial enrolled: Relapsed/refractory KMT2A-rearranged or NPM1-mutated acute leukaemia: revumenib monotherapy. People in a different situation may not see the same effect.
- The trial selected people by a biomarker (NPM1); the result should not be assumed for people whose cancer does not have it.
- Only 94 people took part, so the numbers are less certain than in a large trial.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
94 participants enrolled.
Overall response rate
Revumenib63.2 of 100
| Endpoint | Arm | n | Value | HR (95% CI) | p | Source |
|---|---|---|---|---|---|---|
| CR + CRh (KMT2Ar cohort)primary | Revumenib | 57 | 22.8% | — | — | link |
| 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.