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PRAX vs PTCT

Praxis Precision Medicines, Inc. Common Stock and PTC Therapeutics, Inc.

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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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PTC Therapeutics, Inc.

PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines to children and adults living with rare disorders in the United States and internationally. The company offers Translarna and Emflaza for the treatment of Duchenne muscular dystrophy; Upstaza to treat aromatic l-amino acid decarboxylas (AADC) deficiency, a central nervous system disorder; Tegsedi and Waylivra for the treatment of rare diseases; and Evrysdi to treat spinal muscular atrophy (SMA) in adults and children. Its development pipeline products include Sepiapterin for the treatment of phenylketonuria; PTC518 splicing platform, which is being developed for the treatment of Huntington's disease; and inflammation and ferroptosis platforms, including vatiquinone to treat Friedreich's ataxia. The company distributes its products through third-party distributors. It has collaborations with F. Hoffman-La Roche Ltd., Hoffman-La Roche Inc., the SMA Foundation, National Taiwan University, Akcea Therapeutics, Inc., and Shiratori Pharmaceutical Co., Ltd. The company has license and collaboration agreement with Novartis Pharmaceuticals Corporation to develop PTC518 Huntington's disease program. It markets Upstaza, a gene therapy with the brand name Kebilidi in the United States. PTC Therapeutics, Inc. was incorporated in 1998 and is headquartered in Warren, New Jersey.

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