Aurinia Pharmaceuticals
NASDAQ:AUPHAurinia Pharmaceuticals Inc., a commercial-stage biopharmaceutical company, focuses on developing and commercializing therapies to treat various diseases with unmet medical need in the United States. It offers LUPKYNIS for the treatment of adult patients with active lupus nephritis. It has a collaboration and license agreement with Otsuka Pharmaceutical Co., Ltd. The company was incorporated in 1993 and is headquartered in Edmonton, Canada.
Catalyst Pharmaceuticals
NASDAQ:CPRXCatalyst Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on developing and commercializing therapies for people with rare debilitating, chronic neuromuscular, and neurological diseases in the United States. It offers Firdapse, an amifampridine phosphate tablets for the treatment of patients with lambert-eaton myasthenic syndrome (LEMS); and Ruzurgi for the treatment of pediatric LEMS patients. The company develops Firdapse for the treatment of MuSK antibody positive myasthenia gravis and spinal muscular atrophy type. It has license agreements with BioMarin Pharmaceutical Inc.; and collaboration and license agreement with Endo Ventures Limited for the development and commercialization of generic Sabril tablets. The company was founded in 2002 and is based in Coral Gables, Florida.
Karyopharm Therapeutics
NASDAQ:KPTIKaryopharm Therapeutics Inc., a commercial-stage pharmaceutical company, discovers, develops, and commercializes drugs directed against nuclear export for the treatment of cancer and other diseases in the United States. The company discovers, develops, and commercializes novel and small molecule Selective Inhibitor of Nuclear Export (SINE) compounds that inhibit the nuclear export protein exportin 1 (XPO1). Its lead compound, include XPOVIO in combination with bortezomib and dexamethasone for the treatment of adult patients with multiple myeloma; in combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma; and for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL). It also developing Selinexor for treating hematological and solid tumor malignancies, including multiple myeloma, endometrial cancer, myelofibrosis, and DLBCL; and ELTANEXOR for treating Myelodysplastic Neoplasms, as well as verdinexor, KPT-9274, and IL-12 compounds. The company has license agreement with Menarini Group to develop and commercialize selinexor for human oncology indications; license agreement with Antengene Therapeutics Limited to develop and commercialize selinexor, eltanexor, and KPT-9274 for the treatment and/or prevention of human oncology indications, as well as verdinexor for the diagnosis, treatment, and/or prevention of human non-oncology indications; and distribution agreement for the commercialization of XPOVIO with FORUS Therapeutics Inc. Further, it has a collaboration with Bristol Myers Squibb company to evaluate novel cereblon E3 ligase modulator agent mezigdomide in combination with Selinexor in patients with relapsed/refractory multiple myeloma. Karyopharm Therapeutics Inc. was incorporated in 2008 and is headquartered in Newton, Massachusetts.
Rocket Pharmaceuticals
NASDAQ:RCKTRocket Pharmaceuticals, Inc., together with its subsidiaries, operates as a late-stage biotechnology company that focuses on developing gene therapies for rare and devastating diseases. It has three clinical-stage ex vivo lentiviral vector programs for fanconi anemia, a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells; leukocyte adhesion deficiency-I, a genetic disorder that causes the immune system to malfunction; and pyruvate kinase deficiency, a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia. The company also has a clinical stage in vivo adeno-associated virus program for Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure; Plakophilin-2 Arrhythmogenic Cardiomyopathy, an inheritable cardiac disorder; and BAG3 Dilated Cardiomyopathy. It has license agreements with Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Centro de Investigacion Biomedica En Red, and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz; UCL Business PLC; The Regents of the University of California; and REGENXBIO, Inc. Rocket Pharmaceuticals, Inc. was founded in 1999 and is headquartered in Cranbury, New Jersey.