The menin inhibitor revumenib in KMT2A-rearranged or NPM1-mutant leukaemia

Abstract Targeting critical epigenetic regulators reverses aberrant transcription in cancer, thereby restoring normal tissue function 1–3 . The interaction of menin with lysine methyltransferase 2A (KMT2A), an epigenetic regulator, is a dependence in acute leukaemia caused by either rearrangement of KMT2A or mutation of the nucleophosmin 1 gene ( NPM1 ) 4–6 . KMT2A rearrangements occur in up to 10% of acute leukaemias and have an adverse prognosis, whereas NPM1 mutations occur in up to 30%, forming the most common genetic alteration in acute myeloid leukaemia 7,8 . Here, we describe the results of the first-in-human phase 1 clinical trial investigating revumenib (SNDX-5613), a potent and selective oral…

• MS
  Memorial Sloan-Kettering Cancer Center

  Award: CA008748
• SP
  Syndax Pharmaceuticals
• NI
  National Institutes of Health

  Awards: P30 CA008748, CA088084, K12 CA088084
• NC
  National Cancer Institute

  Awards: CA088084, CA008748, P30 CA008748, K12 CA088084