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MIRM vs PRAX

Mirum Pharmaceuticals, Inc. Common Stock and Praxis Precision Medicines, 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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Praxis Precision Medicines, Inc. Common Stock

Praxis Precision Medicines, Inc., a clinical-stage biopharmaceutical company, engages in the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance. The company's platforms include cerebrum, a small molecule platform for CNS small molecule therapies to develop orally available precision therapies; and solidus to discover and develop antisense oligonucleotide. It also develops Ulixacaltamide, a small molecule inhibitor of T-type calcium channels that is in Phase 3 clinical trial for the treatment of essential tremor; Vormatrigine, a small molecule targeting the hyperexcitable state of sodium-channels in the brain to treat focal onset epilepsy; Relutrigine for the treatment of developmental and epileptic encephalopathies; PRAX-020 to treat KCNT1; and PRAX-050. In addition, the company develops Elsunersen, a clinical-stage ASO designed to regulate NaV1.2 expression for the treatment of SCN2A-DEE; PRAX-080 that targets PCDH19-related developmental epilepsy; PRAX-090 for targeting SYNGAP1 loss-of-function; and PRAX-100 that targets SCN2A LoF mutations. It has a license agreement with RogCon Inc.; and research collaboration and license agreement with Ionis Pharmaceuticals, Inc. Praxis Precision Medicines, Inc. was incorporated in 2015 and is based in Boston, Massachusetts.

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