HAO472

HAO472 is an orally bioavailable small molecule inhibitor of the Menin-KMT2A (Mixed Lineage Leukemia 1 or MLL1) protein-protein interaction, developed by Jiangsu Hengrui Medicine. It is specifically designed to treat patients with relapsed or refractory acute myeloid leukemia (AML), particularly those with KMT2A rearrangements or NPM1 mutations. By binding to Menin, HAO472 prevents its association with KMT2A fusion proteins, thereby disrupting the transcriptional complex responsible for maintaining the leukemic phenotype and promoting the differentiation of myeloid blasts. The drug is currently being evaluated in early-phase clinical trials to determine its safety, tolerability, and preliminary efficacy in genetically defined subsets of leukemia.