MyoKardia, Inc., a clinical stage biopharmaceutical company, discovers, develops, and commercializes targeted therapies for the treatment of serious and neglected rare cardiovascular diseases. Its lead product candidate is mavacamten, an orally administered small molecule, which is in Phase III clinical trial that is designed to reduce left ventricular contractility to alleviate the functional consequences and symptoms of obstructive hypertrophic cardiomyopathy (HCM) and prevent or reverse HCM progression, as well as in Phase II clinical trial for non-obstructive HCM. The company also develops MYK-491, an orally-administered small molecule, which is in Phase IIa clinical trial that is designed to restore normal cardiac muscle contractility in the diseased dilated cardiomyopathy (DCM) heart. Its preclinical programs include MYK-224, a HCM-targeting candidate that is designed to reduce excess cardiac contractility and enhance diastolic function; LUS-1, which is used to counteract a muscle abnormality that results in impaired relaxation of the left ventricle; and ACT-1 targeting genetic DCM due to sarcomeric mutations and impaired calcium regulation. The company has a collaboration with 23andMe, Inc., a consumer genetics and research company; and a strategic collaboration and license agreement with Fulcrum Therapeutics, Inc. to discover, develop and commercialize novel targeted therapies for the treatment of genetic cardiomyopathies. Myokardia, Inc. was founded in 2012 and is headquartered in South San Francisco, California.
Mallinckrodt plc, together with its subsidiaries, develops, manufactures, markets, and distributes specialty pharmaceutical products and therapies in the United States, Europe, the Middle East, Africa, and internationally. It operates in two segments, Specialty Brands and Specialty Generics. The company markets branded pharmaceutical products for autoimmune and rare diseases in the areas of neurology, rheumatology, nephrology, ophthalmology, and pulmonology; and immunotherapy and neonatal respiratory critical care therapies, as well as analgesics and gastrointestinal products. It offers Acthar Gel, an injectable drug for rheumatoid arthritis, multiple sclerosis, infantile spasms, systemic lupus erythematosus, polymyositis, and others; Inomax, a vasodilator to enhance oxygenation and reduce the need for extracorporeal membrane oxygenation; Ofirmev, an intravenous formulation of acetaminophen for pain management; and Therakos photopheresis, an immunotherapy treatment platform, as well as Amitiza, a product for constipation. The company is also developing Terlipressin for the treatment of hepatorenal syndrome; StrataGraft, which is in Phase III and II clinical development for the treatment of burns; MNK-6105 and MNK-6106, an ammonia scavenger for the treatment of hepatic encephalopathy, a neuropsychiatric syndrome associated with hyperammonemia; and SLN500, a ribonucleic acid technology therapy. It markets its branded products to physicians, pharmacists, pharmacy buyers, hospital procurement departments, ambulatory surgical centers, and specialty pharmacies. Mallinckrodt plc has collaboration with Silence Therapeutics plc. The company was founded in 1867 and is based in Dublin, Ireland.
Tetraphase Pharmaceuticals, Inc. engages in the development and production of novel tetracyclines for serious and life-threatening bacterial infections. It also engages in the development of product candidate, eravacycline, a synthetic fluorocycline, as an intravenous and oral antibiotic for use as a first-line empiric monotherapy for the treatment of resistant and multi-drug-resistant infections. The company was founded on July 7, 2006 and is headquartered in Watertown, MA.