Apellis Pharmaceuticals
NASDAQ:APLSApellis Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery, development, and commercialization of therapeutic compounds through the inhibition of the complement system for autoimmune and inflammatory diseases. It offers EMPAVELI for the treatment of paroxysmal nocturnal hemoglobinuria, C3 glomerulopathy and immune complex membranoproliferative glomerulonephritis, and hematopoietic stem cell transplantation-associated thrombotic microangiopathy; and SYFOVRE for treating geographic atrophy secondary to age-related macular degeneration and geographic atrophy (GA). The company also develops APL-3007, a small interfering RNA, or siRNA, which is in a Phase 1 clinical trial, as well as an oral complement inhibitor that is in preclinical development. It has a collaboration and license agreement with Swedish Orphan Biovitrum AB (publ) for development and commercialization of pegcetacoplan; and a collaboration with Beam Therapeutics Inc. focused on the use of Beam's base editing technology to discover new treatments for complement-driven diseases. The company was incorporated in 2009 and is based in Waltham, Massachusetts.
Grifols
NASDAQ:GRFSGrifols, S.A. operates as a plasma therapeutic company in Spain, the United States, Canada, and internationally. The company provides immunoglobulin to treat immunodeficiencies; albumin used to restore circulatory volume and protein loss in pathophysiological conditions, such as liver cirrhosis, cardiocirculatory failure, trauma and severe burns; alpha-1 proteinase inhibitor, a plasma protein, used to treat a genetic disease known as alpha-1; factorVIII/von Willerbrand factor and factor IX, clotting factors for the treatment of hemophilia A and von Willebrand's disease, as well as hemophilia B; antithrombin III to treat hereditary antithrombin deficiency; Fostamatinib, a spleen tyrosine kinase inhibitor; combination of fibrinogen and enzyme thrombin that acts as a biological sealant to control surgical bleeding; and plasma exchange with albumin used to treat Alzheimer's disease. It markets diagnostic testing equipment, reagents, and other equipment; biological products; manufactures and sells plasma to third parties; and involves in research activities, as well as markets pharmaceutical products for hospital pharmacies. In addition, the company offers Yimmugo PID, an immunology drug; and Yimmugo ITP, a hematology drug. Further, it develops Xembify Pre-filled syringes, FlexBag, and Prolastin vials; Xembify Biweekly dosing, Prolastin-C, Fostamatinib2, and VISTASEAL which are in Phase IV development stage; Xembify, Albumin 20% and 5%, Fibrinogen, Trimodulin, Cytotec pregnancy, and AMBAR-Next in Phase III development stage; and AKST4290 that is in Phase II clinical development. Additionally, it offers recIG, Alpha-1 AT in non-cystic fibrosis bronchiectasis, ATIII, GIGA 2339, GIGA564, and OSIG. It has collaboration agreements with Canadian Blood Services for the processing of other plasma-derived products and with GIANT; and GigaGen to develop recombinant polyclonal immunoglobulin therapies. The company was founded in 1909 and is headquartered in Barcelona, Spain.
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.
Intra-Cellular Therapies
NASDAQ:ITCIIntra-Cellular Therapies, Inc., a biopharmaceutical company, focuses on the discovery, clinical development, and commercialization of small molecule drugs that address medical needs primarily in neuropsychiatric and neurological disorders by targeting intracellular signaling mechanisms in the central nervous system (CNS) in the United States. The company offers CAPLYTA for the treatment of schizophrenia and bipolar depression in adults. It is also involved in developing Lumateperone, which is in Phase 3 clinical trial for the treatment of various depressive disorders, as well as additional neuropsychiatric indications. In addition, the company is developing Lenrispodun (ITI-214) for the treatment of Parkinson's disease, CNS, and other disorders; ITI-1284 for the treatment of neuropsychiatric disorders and behavioral disturbances in dementia; and ITI-333 for substance use disorders, pain, and psychiatric comorbidities, including depression, anxiety, and sleep disorders. Intra-Cellular Therapies, Inc. was founded in 2002 and is headquartered in New York, New York.