Menin inhibitor MI-503 exhibits potent anti-cancer activity in osteosarcoma.

Small molecule Menin inhibitor recently has emerged as a new therapeutic by targeting the interaction of histone methyltransferase MLL1 (KMT2A) with Menin. MLL1 is associated with aggressive osteosarcoma (OS) in young adults. The purpose of the study is to explore whether Menin inhibitors have therapeutic effects in OS.