RCZY-711

RCZY-711 is a novel, highly potent, and orally bioavailable small molecule inhibitor of the menin-MLL (KMT2A) interaction. Developed by Rongchang Pharmaceuticals, it is designed to treat acute leukemias characterized by KMT2A rearrangements or NPM1 mutations. By disrupting the menin-MLL complex, RCZY-711 inhibits the aberrant expression of HOX genes and MEIS1, which drive leukemogenesis. Preclinical studies have demonstrated subnanomolar binding affinity, potent growth inhibition in specific cancer cell lines, and significant anti-tumor efficacy in acute myeloid leukemia (AML) xenograft models, including those with MLL-AF4 fusions. It exhibits favorable drug-like ADME properties and a clean safety profile in vitro.