PC-0120

PC-0120 is an orally bioavailable small molecule inhibitor developed by Changtai Pharmaceutical (also known as PharmaMax Corp) for the treatment of acute myeloid leukemia (AML). It specifically targets the protein-protein interaction between Menin and KMT2A (formerly known as MLL). This interaction is a critical driver in specific genetic subtypes of leukemia, including those with KMT2A rearrangements or NPM1 mutations. By disrupting the binding of KMT2A to Menin, PC-0120 inhibits the transcription of leukemogenic genes such as HOXA9 and MEIS1, which are responsible for maintaining the undifferentiated, proliferative state of leukemic blasts. The drug is currently being evaluated in Phase 1 clinical trials to determine its safety, tolerability, and preliminary anti-tumor activity in patients with relapsed or refractory AML.