MI-503

MI-503 is a potent, orally bioavailable small molecule inhibitor of the menin-MLL (KMT2A) protein-protein interaction. The menin-MLL complex is crucial for sustaining the transcriptional program essential to the growth and survival of cancers with MLL1 fusions and other tumors reliant on menin-MLL driven epigenetic dysregulation. MI-503 has demonstrated selective, anti-proliferative activity in preclinical models of MLL leukemia, hepatocellular carcinoma, castration-resistant prostate cancer, and osteosarcoma, functioning via inhibition of histone H3K4 methylation and suppression of oncogenic transcription factors such as c-Myc and Mcl-1. It is characterized by strong in vivo and in vitro efficacy, favorable pharmacokinetic profiles, and low observed toxicity in animal models[1][2][3][5][7][8].

Other names

MI-503MI503MI 5031-((1H-Pyrazol-4-yl)methyl)-4-methyl-5-((4-(6-(2,2,2-trifluoroethyl)thieno[2,3-d]pyrimidin-4-ylamino)piperidin-1-yl)methyl)-1H-indole-2-carbonitrile