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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.
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.
Shares Menin inhibitors for infant KMT2A-rearranged ALL, Differentiation syndrome, NPM1 mutation, AUGMENT-101: revumenib, the first menin inhibitor, in relapsed leukaemias driven by KMT2A rearrangement or NPM1 mutation.
Shares NPM1 mutation, AUGMENT-101: revumenib, the first menin inhibitor, in relapsed leukaemias driven by KMT2A rearrangement or NPM1 mutation, KMT2A (MLL) rearrangement, Revumenib.
Shares NPM1 mutation, KMT2A (MLL) rearrangement, Revumenib, NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia.
Shares NPM1 mutation, KMT2A (MLL) rearrangement, Revumenib, NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia.
Shares Revumenib, NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia, Menin, Infant acute lymphoblastic leukaemia (KMT2A-rearranged, under one year).
Shares NPM1 mutation, NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia, Menin, Epigenetic therapy roadmap: loosening silenced genes → mutation-specific enzymes → editing the epigenome.
Shares Revumenib, NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia, Menin, Infant acute lymphoblastic leukaemia (KMT2A-rearranged, under one year).
Shares Syndax Pharmaceuticals, Revumenib, Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET), Acute myeloid leukaemia.