AU5-5

AU5-5 is an orally bioavailable, highly selective small molecule inhibitor of Fms-like tyrosine kinase 3 (FLT3) currently under development by Aucentra Therapeutics. It demonstrates picomolar potency against wild-type FLT3 and a broad spectrum of FLT3 mutations, including FLT3-ITD and FLT3-D835Y, which are frequently associated with resistance to existing therapies. Preclinical data indicate that AU5-5 induces high levels of cytotoxicity in FLT3-positive leukemic cells and can achieve complete remission in animal models of acute myeloid leukemia (AML). Furthermore, the compound has shown significant synergy when combined with the BCL-2 inhibitor venetoclax, suggesting potential utility in overcoming venetoclax resistance in hematologic malignancies.