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Blocking menin, a scaffold protein the leukaemia depends on, produced remissions in heavily pretreated patients with KMT2A-rearranged or NPM1-mutated acute leukaemia.
The phase 1 portion of AUGMENT-101 treated 68 adults and children with relapsed or refractory acute leukaemia carrying a KMT2A rearrangement or NPM1 mutation with oral revumenib (SNDX-5613), a small molecule that disrupts the menin-KMT2A interaction. Among 60 efficacy-evaluable patients the overall response rate was 53% and the rate of complete remission or remission with partial haematological recovery 30%, with most responders MRD-negative. QT prolongation was dose limiting and differentiation syndrome occurred in 16%. Resistance through MEN1 mutations was subsequently described. The phase 2 KMT2A-rearranged cohort met its primary endpoint and revumenib was approved in 2024 for KMT2A-rearranged acute leukaemia and in 2025 for NPM1-mutated AML, the first drugs in a new class.
Revumenib proved that a transcriptional dependency, rather than a kinase, can be drugged in leukaemia, opening treatment for two genetic subgroups that together cover roughly a third of AML plus most infant ALL. It is now approved and is being combined with venetoclax-azacitidine and intensive chemotherapy in front-line trials. Single-agent remissions are often short without transplant.
Shares NPM1 mutation, KMT2A (MLL) rearrangement, Ziftomenib, Revumenib.
Shares Kura Oncology, NPM1 mutation, Ziftomenib, NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia.
Shares NPM1 mutation, KMT2A (MLL) rearrangement, Ziftomenib, Revumenib.
Shares Ziftomenib, Revumenib, NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia, Menin.
Shares AUGMENT-101, KMT2A (MLL) rearrangement, Ziftomenib, Revumenib.
Shares Ziftomenib, Revumenib, NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia, Menin.
Shares NPM1 mutation, Ziftomenib, Revumenib, NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia.
Shares AUGMENT-101, Ziftomenib, Revumenib, NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia.