The VTP-50469 inhibitor showed strong in vitro and in vivo anti-leukemic activity on NPM1-mutated AML cells, causing a dose-dependent reduction in cell proliferation, a significant downregulation of HOXA/B clusters and MEIS1 gene expression, a marked differentiation of leukemic cells, a reduction of AML engraftment and a prolonged survival in mice PDX models [92, 95, 96]. Here, NPM1 is linked to acute myeloid leukemia.