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MIRM vs STOK

Mirum Pharmaceuticals, Inc. Common Stock and Stoke Therapeutics, Inc. Common Stock

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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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Stoke Therapeutics, Inc. Common Stock

Stoke Therapeutics, Inc., an early-stage biopharmaceutical company, engages in the development of treatments for severe genetic diseases by upregulating protein expression. The company utilizes its proprietary Targeted Augmentation of Nuclear Gene Output (TANGO) approach in developing antisense oligonucleotides (ASOs) to selectively restore protein levels. Its lead product candidates include STK-002, which is in preclinical stage for the treatment of autosomal dominant optic atrophy; and Zorevunersen (STK-001), an investigational new medicine for the treatment of Dravet syndrome is being evaluated in phase I/II clinical trials. The company also develops programs focused on various targets, including haploinsufficiency diseases of the central nervous system and eye. It has a license and collaboration with Biogen Inc. for the development and commercialization of zorevunersen medicine for the treatment of Dravet syndrome; and Acadia Pharmaceuticals Inc. for the discovery, development, and commercialization of novel RNA-based medicines for the treatment of genetic neurodevelopmental diseases. The company was formerly known as ASOthera Pharmaceuticals, Inc. and changed its name to Stoke Therapeutics, Inc. in May 2016. Stoke Therapeutics, Inc. was incorporated in 2014 and is headquartered in Bedford, Massachusetts.

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