Maralixibat Chloride CAS# 228113-66-4 氯马昔巴特

Maralixibat chloride (LUM001) is a potent, selective, orally bioavailable inhibitor of the ileal bile acid transporter (IBAT/ASBT). In preclinical studies, it blocked bile acid reuptake in the terminal ileum with nanomolar potency (IC50 ~80 nM), leading to increased fecal bile acid excretion and reduced serum bile acid levels. In rodent models of cholestasis, maralixibat lowered plasma bile acids, improved liver histology, and reduced markers of pruritus and cholestatic injury. Pharmacokinetic studies showed minimal systemic absorption, indicating a primarily local intestinal mechanism of action. These results supported its development for cholestatic liver diseases, including Alagille syndrome and progressive familial intrahepatic cholestasis.