Fulcrum Therapeutics, Inc. Common Stock
Fulcrum Therapeutics, Inc., a clinical-stage biopharmaceutical company, develops small molecules to improve the lives of patients with genetically defined diseases in the areas of high unmet medical need in the United States. Its clinical-stage product candidate is pociredir, a fetal hemoglobin inducer, which is in phase 1b clinical trial for the treatment of sickle cell disease and beta-thalassemia. The company also discovers drug targets for potential treatment inherited aplastic anemias, such as of diamond-blackfan anemia, shwachman-diamond syndrome, and fanconi anemia, as well as novel fetal hemoglobin inducers, fibrotic disorders, and cardiomyopathies which is in preclinical trial. It has collaboration and license agreement with MyoKardia, Inc. to identify biological targets that are capable of modulating genes of interest with relevance to cardiomyopathies; and licensce agreement with CAMP4 which includes certain small molecule compounds, composition of matter and method of use patent rights, and know-how to research, develop, manufacture, use, and commercialize; and Sanofi for the development and commercialization of losmapimod, an oral small molecule being investigated for the treatment of facioscapulohumeral muscular dystrophy. Fulcrum Therapeutics, Inc. was Incorporated in 2015 and is headquartered in Cambridge, Massachusetts.
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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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