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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.
Celcuity Inc is a clinical-stage biotechnology company pursuing development for oncology. The Company's lead therapeutic candidate is gedatolisib, a potent pan-PI3K and mTOR inhibitor. Its mechanism of action and pharmacokinetic properties are differentiated from other currently approved and investigational therapies that target PI3K or mTOR alone or together. A Phase 3 clinical trial, VIKTORIA-1, evaluating gedatolisib in combination with fulvestrant with or without palbociclib in patients with HR+/HER2- advanced breast cancer is currently enrolling patients.