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.
Mirati Therapeutics
NASDAQ:MRTXMirati Therapeutics, Inc., a commercial-stage oncology company, develops novel therapeutics to address the genetic and immunological promoters of cancer in the United States. The company provides KRAZATI, an oral targeted treatment option for adult patients with KRAS G12C-mutated locally advanced or metastatic non-small cell lung (NSCLC), as well as in clinical development as a monotherapy and in combination with other agents. It also develops Sitravatinib, an investigational spectrum-selective kinase inhibitor in Phase 3 clinical development that inhibits receptor tyrosine kinases (RTKs), including TAM family receptors, split family receptors, and RET, which overcomes resistance to checkpoint inhibitor therapy through targeted reversal of an immunosuppressive tumor microenvironment that enhances antigen-specific T cell response and expanding dendritic cell-dependent antigen presentation. In addition, the company pipeline comprises the MRTX1719, an investigational synthetic lethal PRMT5 inhibitor designed to target the PRMT5/methylthioadensoine (MTA) complex and is in clinical development; MRTX0902, a selective SOS1 inhibitor that improves anti-tumor efficacy in combination with targeted mitogen-activated protein kinase (MAPK)-pathway inhibitors, and is in clinical development; and MRTX1133, an investigational lead KRAS G12D compound. It has a collaboration and license agreement with BeiGene, Ltd. to develop, manufacture, and commercialize sitravatinib; and Zai Lab Ltd. to research, develop, manufacture, and commercialize adagrasib in various indications. Mirati Therapeutics, Inc. was founded in 1995 and is headquartered in San Diego, California.