Mirum Pharmaceuticals, Inc. Common Stock
Mirum Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development and commercialization of novel therapies for debilitating rare and orphan diseases. Its lead product candidate is LIVMARLI (maralixibat), an orally administered and minimally absorbed ileal bile acid transporter (IBAT) inhibitor that is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the United States and internationally. The company is also involved in the commercialization of Cholbam, a cholic acid capsule, which is approved as treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects and for adjunctive treatment of patients with peroxisomal disorders, including peroxisome biogenesis disorder-Zellweger spectrum disorder and Smith-Lemli-Opitz syndrome; and Chenodal, a tablet, which is approved for the treatment of radiolucent stones in the gallbladder, and under Phase 3 development for the treatment cerebrotendinous xanthomatosis. In addition, it develops Volixibat, an oral and minimally absorbed agent designed to inhibit IBAT, currently under Phase 2b clinical trial for the treatment of adult patients with cholestatic liver diseases. The company was incorporated in 2018 and is headquartered in Foster City, California.
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Novartis AG engages in the research, development, manufacture, distribution, marketing, and sale of pharmaceutical medicines in Switzerland and internationally. The company offers Entresto, an angiotensin receptorneprilysin inhibitor for the treatment of symptomatic chronic heart failure with reduced ejection fraction (HFrEF); Cosentyx to treat plaque psoriasis, pso riatic arthritis, ankylosing spondylitis, and nonradiographic axial spondy loarthritis; Kisqali, a selective oral cyclin dependent inhibitor of kinases 4 and 6 (CDK4/6); Promacta/Revolade to treat immune thrombocytopenia (ITP), thrombocytopenia, and patients with severe aplastic anemia (SAA); Tafinlar+Mekinist, an oral combination therapy to treat patients with certain types of cancers; and Jakavi for the treatment of myelofibrosis, polycythemia vera, and acute or chronic graftversushost disease (GvHD). It also provides Tasigna to treat philadelphia chromosomepositive chronic myeloid leukemia in the chronic and/or accelerated phase; Xolair for the treatment of allergic asthma and nasal polyps or severe chronic rhinosi nusitis with nasal polyps; Ilaris to treat fever syndromes, Still's disease, and acute gouty arthritis; Pluvicto to treat prostatespecific membrane anti genpositive metastatic castrationresistant prostate cancer; Sandostatin SC and Sandostatin LAR to treat acromegaly carcinoid tumors and other types of functional gastro intestinal and pancreatic neuroendocrine tumors; Zolgensma for the treatment of genetic root cause of spinal muscular atrophy; Lucentis; Leqvio to reduce LDL cholesterol; Lutathera; Scemblix; and Fabhalta. The company focuses on therapeutic areas, such as cardiovascular, renal and metabolic, immunology, neuroscience, oncology, and hematology. It has a license and collaboration agreement with Alnylam Pharmaceuticals, Inc. to develop, manufacture, and commercialize Leqvio (inclisiran), a therapy to reduce LDL cholesterol. Novartis AG was incorporated in 1996 and is headquartered in Basel, Switzerland.
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