Mirum Pharmaceuticals Inc. is a clinical-stage therapeutics company developing a novel approach for treating cholestatic liver diseases, with an immediate focus on rare pediatric conditions. The company's lead product candidate, maralixibat, is a Phase 3-ready investigational oral drug with an established safety profile and efficacy data in several indications, including Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC).  The company's lead product candidate, maralixibat, is a Phase 3-ready investigational oral drug with an established safety profile and efficacy data in several indications, including Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC).