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.
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.
Zogenix
NASDAQ:ZGNXZogenix, Inc. is a pharmaceutical company. The firm engages in the development and commercialization of central nervous system (CNS) therapies and products for the treatment orphan diseases and other CNS disorders. Its products include Fintepla and MT1621. The company was founded by Stephen James Farr, Cam L. Garner, Roger L. Hawley, Bret E. Megargel, Jonathan M. N. Rigby, Scott L. Glenn, and John J. Turanin on May 11, 2006 and is headquartered in Emeryville, CA.
Zai Lab
NASDAQ:ZLABZai Lab Limited develops and commercializes therapies to treat oncology, autoimmune disorders, infectious diseases, and neuroscience. Its commercial products include Zejula, an orally administered poly polymerase 1/2 inhibitor; Optune, a cancer therapy that uses electric fields tuned to specific frequencies to kill tumor cells; NUZYRA for acute bacterial skin and skin structure infections, and community acquired bacterial pneumonia; Qinlock to treat gastrointestinal stromal tumors, and VYVGART, a human IgG1 antibody fragment for myesthenia gravis. The company also develops Tumor Treating Fields, a portable device for delivery of electric fields; Repotrectinib, a tyrosine kinase inhibitor (TKI) to target ROS1 and TRK A/B/C in TKI-naïve- or -pretreated cancer patients; Tisotumab vedotin, an antibody drug conjugate; Adagrasib for treating KRAS-G12C-mutated NSCLC, colorectal cancer, and pancreatic cancer; and Bemarituzumab to treat gastric and gastroesophageal junction cancer patients. In addition, it develops Sulbactam/durlobactam, a combination of a beta-lactam antibiotic and a beta-lactamase inhibitor for the treatment of serious infections caused by Acinetobacter; KarXT for the treatment of psychiatric and neurological conditions. It has license and collaboration agreement with Tesaro, Inc. to develop, manufacture, and commercialize niraparib; NovoCure to develop and commercialize Tumor Treating Fields; Deciphera to develop and commercialize ripretinib; Paratek Bermuda Ltd. to develop, manufacture, and commercialize omadacycline; argenx, to develop and commercialize efgartigimod; BMS to develop and commercialize tisotumab vedotin and repotrectinib; Mirati to research, develop, manufacture, and commercialize adagrasib; Amgen to develop and commercialize bemarituzumab; and Innoviva to develop and commercialize Sulbactam-Durlobactam; Karuna to develop and commercialize KarXT. The company was incorporated in 2013 and is headquartered in Shanghai, China.