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Apellis Pharmaceuticals Inc is a commercial-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel therapeutic compounds to treat diseases with high unmet needs through the inhibition of the complement system, which is an integral component of the immune system, at the level of C3, the central protein in the complement cascade.
Mirum Pharmaceuticals Inc is a biopharmaceutical company focused on the identification, acquisition, development, and commercialization of novel therapies for debilitating rare and orphan diseases. Its product, Livmarli, a novel, orally administered, minimally-absorbed ileal bile acid transporter IBAT) inhibitor (IBATi), is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS). It is involved in the development of Maralixibat for the treatment of pediatric patients with progressive familial intrahepatic cholestasis, or PFIC, and Alagille syndrome, or ALGS. The company is also involved in the development of volixibat for the treatment of adult patients with cholestatic liver diseases.