Alexion Pharmaceuticals
NASDAQ:ALXNAlexion Pharmaceuticals, Inc. develops and commercializes various therapeutic products. The company offers ULTOMIRIS for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS); and SOLIRIS for the treatment of PNH, aHUS, generalized myasthenia gravis (gMG), and neuromyelitis optica spectrum disorder (NMOSD). It also provides Strensiq for patients with hypophosphatasia; Kanuma (sebelipase alfa) for the treatment of patients with lysosomal acid lipase deficiency; and Andexxa, a reversal agent for patients treated with rivaroxaban or apixaban. In addition, the company is developing ALXN1210 (Intravenous) that is in Phase III clinical trials for the treatment of gMG, NMOSD, ALS, COVID-19, and HSCT-TMA; ALXN1210 (Subcutaneous), which is in Phase III clinical trials for PNH and aHUS; ALXN1820 that is in Phase I clinical trial, a therapeutic antagonist of properdin; and ALXN1720, which is in Phase I clinical trial for the treatment of disease states involving dysregulated terminal complement activity. Further, it is developing ALXN1840 that is in Phase III clinical trials for the treatment of Wilson disease; ALXN1830, which are in Phase I clinical trials for neonatal Fc receptor; ALXN2040 and ALXN2050 to treat diseases associated with dysregulation of the complement alternative pathway; ALXN1850, an enzyme replacement therapy; ALXN2060 for treating transthyretin amyloidosis; and ALXN2075 for treatment of relapsed/refractory chronic lymphocytic leukemia. The company serves distributors, pharmacies, hospital, hospital buying groups, and other healthcare providers in the United States and internationally. Alexion Pharmaceuticals, Inc. has collaboration and license agreement with Halozyme Therapeutics, Inc.; and agreements with Dicerna Pharmaceuticals, Inc., Zealand Pharma A/S, Caelum Biosciences, Inc., Stealth BioTherapeutics Corp., and Affibody AB. The company was founded in 1992 and is headquartered in Boston, Massachusetts.
Horizon Therapeutics Public
NASDAQ:HZNPHorizon Therapeutics Public Limited Company, a biotechnology company, focuses on the discovery, development, and commercialization of medicines that address critical needs for people impacted by rare, autoimmune, and severe inflammatory diseases. Its portfolio comprises 12 medicines in the areas of rare diseases, gout, ophthalmology, and inflammation. It markets TEPEZZA (teprotumumab-trbw) for intravenous infusion; KRYSTEXXA (pegloticase injection) for intravenous infusion; RAVICTI (glycerol phenylbutyrate) oral liquid; PROCYSBI (cysteamine bitartrate) delayed-release capsules and granules for oral use; ACTIMMUNE (interferon gamma-1b) injection for subcutaneous use; BUPHENYL (sodium phenylbutyrate) tablets and powder for oral use; QUINSAIR (levofloxacin) solution for inhalation; and UPLIZNA (inebilizumab-cdon) injection for intravenous use. The company also markets PENNSAID (diclofenac sodium topical solution) for topical use; DUEXIS (ibuprofen/famotidine) tablets for oral use; RAYOS (prednisone) delayed-release tablets for oral use; and VIMOVO (naproxen/esomeprazole magnesium) delayed-release tablets for oral use. It has collaboration agreements with HemoShear Therapeutics, LLC; Alpine Immune Sciences, Inc.; Halozyme Therapeutics, Inc.; Arrowhead Pharmaceuticals, Inc.; Q32 Bio Inc.; and Xeris Biopharma Holdings, Inc. The company was formerly known as Horizon Pharma Public Limited Company and changed its name to Horizon Therapeutics Public Limited Company in May 2019. Horizon Therapeutics Public Limited Company was founded in 2005 and is headquartered in Dublin, Ireland.
MyoKardia
NASDAQ:MYOKMyoKardia, 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 allosteric modulator of cardiac myosin, which is in Phase III clinical trial for the treatment of hypertrophic cardiomyopathy (HCM). The company also develops Danicamtiv, an orally administered small molecule, which completed Phase I and Phase IIa clinical trials on patients with dilated cardiomyopathy. Its preclinical programs include MYK-224, a small molecule program for the treatment of HCM targeting hypercontractility and impaired relaxation; ACT-1 for the treatment of diastolic dysfunction; and LUS-1 for the treatment of diastolic dysfunction. Myokardia, Inc. was founded in 2012 and is headquartered in Brisbane, California.
Royalty Pharma
NASDAQ:RPRXRoyalty Pharma plc operates as a buyer of biopharmaceutical royalties and a funder of innovations in the biopharmaceutical industry in the United States. It is also involved in the identification, evaluation, and acquisition of royalties on various biopharmaceutical therapies. In addition, the company collaborates with innovators from academic institutions, research hospitals and not-for-profits, small and mid-cap biotechnology companies, and pharmaceutical companies. Its portfolio consists of royalties on approximately 35 commercial products and 14 development-stage product candidates that address various therapeutic areas, such as rare disease, cancer, neuroscience, immunology, respiratory, infectious disease, hematology, and diabetes. The company was founded in 1996 and is based in New York, New York.