Uses
VTP50469 mesylate is a potent, and selective Menin-MLL1 inhibitor that effectively targets MLL-rearranged and NPM1c+ leukemia. VTP50469 mesylate selectively kills cell lines with MLL rearrangements and NPM1c+ mutations. VTP50469 mesylate displaces Menin from protein complexes and inhibits MLL's chromatin occupancy at specific genes, leading to significant changes in gene expression, differentiation, and apoptosis. VTP50469 demonstrates dramatic reductions in leukemia burden in patient-derived xenograft models of MLL-r acute myeloid leukemia and MLL-r acute lymphoblastic leukemia, with some mice remaining disease-free for over a year post-treatment.
