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.
Open FULC dashboard ↗Zevra Therapeutics, Inc. Common Stock
Zevra Therapeutics, Inc., a commercial-stage company, focuses on addressing unmet needs for the treatment of rare diseases in the United States. The company develops its products through Ligand Activated Technology platform. Its lead product candidate is KP1077, consisting of KP1077IH, which is under Phase 2 clinical trial for the treatment of idiopathic hypersomnia, and KP1077N, which is under Phase 1/2 clinical trial to treat narcolepsy. The company is also developing Celiprolol, a prodrug product candidate that is under Phase 3 clinical trial for the treatment of vascular Ehlers Danlos syndrome. In addition, it offers AZSTARYS, a once-daily treatment for attention deficit and hyperactivity disorder in patients aged six years and older; OLPRUVA to treat urea cycle disorders; and MIPLYFFA for the treatment of niemann pick disease type C, an ultra-rare neurodegenerative lysosomal storage disorder. The company has collaboration and license agreement with Commave Therapeutics SA to develop, manufacture and commercialize the company's product candidates containing SDX and d-methylphenidate; and license agreement with Acer and Relief Therapeutics, Inc. to develop and commercialize rights for OLPRUVA. The company was formerly known as KemPharm, Inc. and changed its name to Zevra Therapeutics, Inc. in February 2023. Zevra Therapeutics, Inc. was incorporated in 2006 and is headquartered in Celebration, Florida.
Open ZVRA dashboard ↗