Menin Inhibitors in Acute Myeloid Leukemia: From Molecular Rationale to Clinical Practice

Background

Acute Myeloid Leukemia (AML) harboring KMT2A rearrangements (KMT2Ar) or NPM1 mutations (mNPM1) constitutes a substantial and molecularly coherent subset of AML characterized by aberrant activation of homeobox (HOX) gene programs driven by the menin–MLL transcriptional complex. Disruption of this interaction through small-molecule menin inhibitors represents a compelling therapeutic strategy.