IMP5471

IMP5471 is an orally administered small molecule inhibitor of the Hedgehog signaling pathway, specifically targeting the SMO (Smoothened) protein. It is being developed by IMPACT Therapeutics for the treatment of cancers associated with aberrant Hedgehog pathway activation, including basal cell carcinoma and medulloblastoma. Preclinical studies have demonstrated high in vitro and in vivo pharmacodynamic activity, high selectivity, good oral bioavailability in animal models, and a wide safety window. The drug has advanced to Phase I clinical trials for cancer indications in China. There are no currently approved drugs with this mechanism for malignant hematological diseases in China[1][5][6].