In this context, the inhibition of the KMT2A/menin complex by chemical agents, such as VTP-50469 and MI-3454, demonstrated significant anti-leukemic activity, reducing cell proliferation, downregulating HOXA/B clusters and MEIS1 gene expression, promoting a marked differentiation of leukemic cells, and reducing the AML engraftment and survival in mouse patient-derived xenograft (PDX) models [113,114]. Here, MEN1 is linked to acute myeloid leukemia.