Akcea Therapeutics
NASDAQ:AKCAAkcea Therapeutics, Inc., a biopharmaceutical company, focuses on developing and commercializing drugs to treat patients with serious and rare diseases in the United States and internationally. The company offers TEGSEDI, which is designed to reduce the production of transthyretin protein. It develops WAYLIVRA, which has completed Phase III clinical study for the treatment of familial chylomicronemia syndrome; and that is in Phase III clinical study for the treatment of familial partial lipodystrophy. The company also develops AKCEA-APO(a)-LRx, which has completed Phase IIb clinical study for treating patients suffering with hyperlipoproteinemia; AKCEA-ANGPTL3-LRx that has completed Phase IIb clinical study for the treatment of multiple lipid disorders; and AKCEA-APOCIII-LRx, which is in Phase II study for the treatment of cardiovascular disease due to elevated triglyceride levels, as well as AKCEA-TTR-LRx to treat the broad population of patients with hereditary and wild-type forms of transthyretin amyloidosis. It has strategic collaboration with Novartis Pharma AG and PTC Therapeutics International Limited. The company was incorporated in 2014 and is headquartered in Boston, Massachusetts. Akcea Therapeutics, Inc. operates as a subsidiary of Ionis Pharmaceuticals, Inc.
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.
Biohaven
NYSE:BHVNBiohaven Ltd., together with its subsidiaries, focuses on discovering, developing, and commercializing therapies for immunology, neuroscience, and oncology worldwide. The company's pipeline products include Troriluzole, which is in Phase 3 clinical trial for the treatment of neurological and neuropsychiatric illnesses; BHV-5500 that blocks glutamate signaling mediated by post-synaptic NMDA receptors; Taldefgrobep Alfa, which is in Phase 3 clinical trial for the treatment of spinal muscular atrophy and obesity; BHV-7000, a candidate in Phase 2/3 clinical trials for the treatment of focal and generalized epilepsy, bipolar disorder, and major depressive disorder; BHV-2100 that is in Phase 1 clinical trials for the treatment of migraines and neuropathic pain; and BHV-8000, a product candidate in Phase 1 clinical trials for the treatment of early Alzheimer's and Parkinson's disease, sclerosis, and amyloid-related imaging abnormalities. It also offers BHV-1300, a product candidate in Phase 1 clinical trials to treat rheumatoid arthritis; BHV-1310 for the treatment of generalized myasthenia gravis and acute exacerbations or flares; BHV-1400 to treat IgA Nephropathy; and BHV-1600 for the treatment of dilated cardiomyopathy. In addition, the company develops BHV-1100, a product candidate in Phase 1a/1b clinical trials for multiple myeloma patients; BHV-1510, a preclinical product that targets carcinomas; and BHV-1500 for Hodgkin's lymphoma. It has license, development, and commercialization agreements with Yale University, AstraZeneca, University of Connecticut, Artizan Biosciences Inc., Reliant Glycosciences LLC, Katholieke Universiteit Leuven, BMS, and Highlightll Pharmaceutical Co. Ltd. The company was formerly known as Biohaven Research Ltd and changed its name to Biohaven Ltd. in September 2022. Biohaven Ltd. was incorporated in 2022 and is headquartered in New Haven, Connecticut.
Ligand Pharmaceuticals
NASDAQ:LGNDLigand Pharmaceuticals Incorporated, a biopharmaceutical company, engages in the development and licensing of biopharmaceutical assets worldwide. Its commercial programs include Kyprolis and Evomela, which are used to treat multiple myeloma; Rylaze, a recombinant erwinia asparaginase for the treatment of acute lymphoblastic leukemia or lymphoblastic lymphoma in adult and pediatric patients; Filspari, a dual endothelin and angiotensin II receptor antagonist in development for rare kidney diseases and non-immunosuppressive treatment indicated for immunoglobulin A nephropathy; Teriparatide injection product for osteoporosis; Vaxneuvance for the prevention of invasive disease caused by streptococcus pneumoniae serotypes; and Pneumosil, a pneumococcal conjugate vaccine to help fight against pneumococcal pneumonia among children. The company also offers TZIELD, a CD3-directed antibody indicated to delay the onset of Stage 3 type 1 diabetes (T1D) in adults and children aged 8 years and older with Stage 2 T1D; Nexterone, a captisol-enabled formulation of amiodarone; Zulresso, a captisol-enabled formulation of brexanolone for the treatment of postpartum depression; and Veklury, an antiviral treatment for moderate or severe COVID-19. In addition, it provides Noxafil-IV, a captisol-enabled formulation of posaconazole for IV use; Duavee for the treatment of post-menopausal symptoms in women; Exemptia for autoimmune diseases; Vivitra for breast cancer; and Bryxta and Zybev for various indications. The company has alliances, licenses, and other business relationships with Amgen, Merck, Pfizer, Jazz, Takeda, Gilead Sciences, and Baxter International. Further, it sells Captisol materials. Ligand Pharmaceuticals Incorporated was incorporated in 1987 and is based in Jupiter, Florida.
Portola Pharmaceuticals
NASDAQ:PTLAPortola Pharmaceuticals, Inc., a biopharmaceutical company, develops and commercializes novel therapeutics in the areas of thrombosis and other hematologic disorders and inflammation in the United States. The company offers Andexxa, an antidote for patients treated with rivaroxaban and apixaban when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding; and Bevyxxa (betrixaban), an oral, once-daily Factor Xa inhibitor for the prevention of venous thromboembolism in adult patients hospitalized for an acute medical illness. It is also advancing cerdulatinib, a dual spleen tyrosine kinase and janus kinases inhibitor in development for the treatment of hematologic cancers. In addition, the company is developing PRT2761, a Syk inhibitor that has completed Phase II clinical trial for the treatment for allergic conjunctivitis. Portola Pharmaceuticals, Inc. has collaboration agreements with Bristol-Myers Squibb; Pfizer Inc.; Bayer Pharma, AG; Janssen Pharmaceuticals, Inc.; Daiichi Sankyo, Inc.; Millennium Pharmaceuticals, Inc.; SRX Cardio; LLC; Ora; and Astellas Pharma Inc. The company was founded in 2003 and is headquartered in South San Francisco, California.