Galapagos
NASDAQ:GLPGGalapagos NV, an integrated biopharmaceutical company, engages in the discovery, development, and commercialization of?various medicines for high unmet medical need. Its pipeline products include filgotinib, a JAK1 inhibitor that is in various phases of clinical trials for the treatment of rheumatoid arthritis, Crohn's disease, ulcerative colitis, ankylosing spondylitis, psoriatic arthritis, and uveitis. The company's pipeline products also comprise GLPG3667 that has completed Phase 1b trial; and GLPG5101 and GLPG5201, CD19 CAR-T product candidate manufactures at point-of-care, that is in Phase I/II in relapsed/refractory non-Hodgkin lymphoma and chronic lymphocytic leukemia. The company has collaboration agreements with Gilead Sciences, Inc.; AbbVie S.à r.l.; and Novartis Pharma AG. Galapagos NV was incorporated in 1999 and is headquartered in Mechelen, Belgium.
Ionis Pharmaceuticals
NASDAQ:IONSIonis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics in the United States. The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI, an antisense injection for the treatment of polyneuropathy caused by hereditary transthyretin amyloidosis in adults; and WAYLIVRA, an antisense medicine for treatment for familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy. It also develops medicines for various indications that are in phase 3 study, including Eplontersen as a monthly self-administered subcutaneous injection to treat all types of ATTR; Olezarsen for patients with FCS and severe hypertriglyceridemia (SHTG); Donidalorsen for patients with hereditary angioedema; ION363 for patients with amyotrophic lateral sclerosis; Tofersen to inhibit the production of superoxide dismutase 1; Pelacarsen for patients with established cardiovascular disease and elevated lipoprotein(a); and Bepirovirsen to inhibit the production of viral proteins associated with hepatitis B virus. In addition, the company develops IONIS-FB-LRx to inhibit the production of complement factor B and the alternative complement pathway; and ION224 to reduce the production of diacylglycerol acyltransferase 2. It has a strategic collaboration with Biogen for the treatment of neurological disorders; and collaboration and license agreement with Metagenomi, Inc, AstraZeneca, Bayer AG, GlaxoSmithKline plc, Novartis, Roche, Swedish Orphan Biovitrum AB, and PTC Therapeutics. The company was incorporated in 1989 and is based in Carlsbad, California.
Jazz Pharmaceuticals
NASDAQ:JAZZJazz Pharmaceuticals plc identifies, develops, and commercializes pharmaceutical products for unmet medical needs in the United States, Europe, and internationally. The company offers Xywav for cataplexy or excessive daytime sleepiness (EDS) with narcolepsy and idiopathic hypersomnia; Xyrem to treat cataplexy or EDS with narcolepsy; Epidiolex for seizures associated with Lennox-Gastaut and Dravet syndromes, or tuberous sclerosis complex; Zepzelca to treat metastatic small cell lung cancer, or with disease progression on or after platinum-based chemotherapy; Rylaze for acute lymphoblastic leukemia or lymphoblastic lymphoma; Enrylaze to treat acute lymphoblastic leukemia and lymphoblastic lymphoma; Defitelio to treat severe hepatic veno-occlusive disease; and Vyxeos for newly-diagnosed therapy-related acute myeloid leukemia. It also develops Zanidatamab to treat HER2-expressing gastroesophageal adenocarcinoma (GEA), and patients with HER2-expressing metastatic GEA; Zepzelca for the treatment of patients with select relapsed/refractory solid tumors based on limited response in three solid tumor cohorts; JZP815, a pan-RAF kinase inhibitor that targets components of the mitogen-activated protein kinase; JZP898, a conditionally-activated interferon alpha molecule; Epidiolex to treat LGS, DS, and TSC; Suvecaltamide to treat parkinson's disease tremor; JZP150, a fatty acid amide hydrolase inhibitor program to treat post-traumatic stress disorder; and JZP441 to treat narcolepsy, IH, and other sleep disorders. The company has licensing and collaboration agreements with XL-protein GmbH to extend the plasma half-life of selected asparaginase product candidates; Redx Pharma plc for preclinical activities Ras/Raf/MAP kinase pathway program; and Autifony Therapeutics Limited on discovering and developing drug candidates targeting two different ion channel targets associated with neurological disorders. The company was incorporated in 2003 and is headquartered in Dublin, Ireland.
Mylan
NASDAQ:MYLMylan N.V., together with its subsidiaries, develops, licenses, manufactures, markets, and distributes generic, branded-generic, brand-name, and over-the-counter (OTC) pharmaceutical products in North America, Europe, and internationally. It offers active pharmaceutical ingredients and finished dosage forms; and antiretroviral medicines to treat HIV/AIDS. The company also provides prescription products, such as EpiPen Auto-Injector; Perforomist Inhalation Solution; Dymista; Creon; and Influvac, as well as YUPELRI, an inhalation solution for the maintenance treatment of patients with chronic obstructive pulmonary diseases. In addition, it markets OTC products, including Cold-EEZE, MidNite, Vivarin, Brufen, CB12, and EndWarts. The company offers its products to therapeutic areas, such as cardiovascular, CNS and anesthesia, dermatology, diabetes and metabolism, gastroenterology, immunology, infectious disease, oncology, respiratory and allergy, and women's health. Its customers include retail pharmacies, wholesalers and distributors, payers, and insurers and governments, as well as institutions, such as hospitals. Mylan N.V. has collaboration and license agreements with Pfizer Inc.; Momenta Pharmaceuticals, Inc.; Theravance Biopharma, Inc.; Biocon Ltd.; Fujifilm Kyowa Kirin Biologics Co. Ltd; and Revance Therapeutics, Inc. The company was founded in 1961 and is based in Hatfield, the United Kingdom.
Ultragenyx Pharmaceutical
NASDAQ:RAREUltragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Japan, Europe, and internationally. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for treating long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidatures include DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; UX701, for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; Bayer Healthcare LLC; GeneTx; Mereo; University of Pennsylvania; Arcturus Therapeutics Holdings Inc.; Solid Biosciences Inc.; Regeneron; Abeona; and Daiichi Sankyo Co., Ltd. The company was incorporated in 2010 and is headquartered in Novato, California.