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 inhibitor of the menin-KMT2A interaction, in patients with relapsed or r

SUBMITTER: Issa GC 

PROVIDER: S-EPMC10060155 | biostudies-literature | 2023 Mar

REPOSITORIES: biostudies-literature