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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Tonix Pharmaceuticals Holding Corp., a biopharmaceutical company, focuses on developing and commercializing therapeutics to treat and prevent human disease. It markets Zembrace SymTouch and Tosymra for the treatment of acute migraine with or without aura in adults. Its portfolio focuses on central nervous system disorders, as well as immunology, infectious disease, and rare disease product candidates. The company's products pipeline include TNX-102 SL (cyclobenzaprine HCl sublingual tablet), for the management of fibromyalgia; and is being developed to prevent and treat acute stress reaction, posttraumatic stress disorder, Alzheimer's disease, alcohol use disorder, and multi-site pain associated with Long COVID. It also develops TNX-1300, a double-mutant cocaine esterase, which is in Phase 2 clinical trials for the treatment of cocaine intoxication; and TNX-1900, an intranasal potentiated oxytocin for the treatment of binge eating disorder, adolescent obesity, and bone health in pediatric autism, as well as for social anxiety disorder. In addition, the company develops TNX-1500, an Fc-modified humanized monoclonal antibody indicated to prevent organ transplant rejection, as well as to treat autoimmune conditions; TNX-1700, a recombinant Trefoil Factor Family 2 fusion protein for the treatment of gastric and colorectal cancers; TNX-801 that is in the pre-IND development stage for protection against smallpox and mpox; and TNX-1800, a live virus vaccine for SARS-CoV-2. Its rare disease portfolio consists of TNX-2900, an intranasal potentiated oxytocin to treat for Prader-Willi syndrome. The company has collaboration agreement with Bilthoven Biologicals to advance TNX-801 and Makana Therapeutics, Inc. to study TNX-1500 for human-compatible organs and cells for the treatment of organ failure. Tonix Pharmaceuticals Holding Corp. is headquartered in Chatham, New Jersey.
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