VTP50469

VTP50469 is a potent, selective, and orally bioavailable small molecule inhibitor of the Menin-KMT2A (formerly MLL1) protein-protein interaction. It binds to Menin with high affinity, specifically targeting the pocket where KMT2A binds, thereby displacing oncogenic KMT2A fusion proteins or wild-type KMT2A from the Menin complex. This disruption leads to the suppression of a leukemogenic gene expression program, particularly the HOXA/MEIS1 signaling axis, which is critical for the survival of specific leukemia subtypes. VTP50469 has demonstrated significant anti-leukemic activity in preclinical models of KMT2A-rearranged and NPM1-mutated acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL), inducing cell cycle arrest, apoptosis, and myeloid differentiation. It was originally developed by Vitae Pharmaceuticals, which was subsequently acquired by Allergan and later AbbVie.

Other names

VTP50469VTP-50469VTP 50469