Akebia Therapeutics
NASDAQ:AKBAAkebia Therapeutics, Inc., a biopharmaceutical company, focuses on the development and commercialization of therapeutics for patients with kidney diseases. The company's lead product investigational product candidate is Vafseo (vadadustat), an oral hypoxia-inducible factor prolyl hydroxylase, which is in Phase III development for the treatment of anemia due to chronic kidney disease (CKD) in dialysis-dependent and non-dialysis dependent patients. It offers Auryxia, a ferric citrate that is used to control the serum phosphorus levels in adult patients with DD-CKD on dialysis; and the treatment of iron deficiency anemia in adult patients with CKD not on dialysis. The company's product pipeline includes AKB-9090, a drug targeting critical-care indications; and AKB-10108, a drug targeting conditions related to premature birth. It has collaboration agreements with Mitsubishi Tanabe Pharma Corporation for the development and commercialization of vadadustat in Japan and other Asian countries, as well as research and license agreement with Janssen Pharmaceutica NV for the development and commercialization of hypoxia-inducible factor prolyl hydroxylase targeted compounds worldwide. Akebia Therapeutics, Inc. was incorporated in 2007 and is headquartered in Cambridge, Massachusetts.
Evelo Biosciences
NASDAQ:EVLOEvelo Biosciences, Inc., a clinical-stage biotechnology company, focuses on discovering and developing oral medicines that act on immune cells in the small intestine with systemic effects. It is developing EDP1815, a whole-microbe product candidate, which has completed a Phase 2 trial for the treatment of psoriais; and is in Phase 2 clinical trial for the treatment of atopic dermatitis. The company is also developing EDP1867, a non-live pharmaceutical preparation for single strain of Veillonella parvula, which is in Phase 1b clinical trial; EDP2939, an investigational oral biologic for the potential treatment of inflammatory diseases; and EDP1908, a product candidate for oncology. Evelo Biosciences, Inc. was incorporated in 2014 and is headquartered in Cambridge, Massachusetts.
Inventiva
NASDAQ:IVAInventiva S.A., a clinical-stage biopharmaceutical company, focuses on the development of oral small molecule therapies for the treatment of non-alcoholic steatohepatitis (NASH) and other diseases. Its lead product candidate is Lanifibranor, which is in Phase III clinical trial to treat NASH. The company also develops Odiparcil for the treatment of patients with mucopolysaccharidoses type VI. In addition, it has a pipeline of earlier stage programs in oncology and other diseases. The company was founded in 2011 and is headquartered in Daix, France.
Mirum Pharmaceuticals
NASDAQ:MIRMMirum Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development and commercialization of novel therapies for debilitating rare and orphan diseases. Its lead product candidate is LIVMARLI (maralixibat), an orally administered and minimally absorbed ileal bile acid transporter (IBAT) inhibitor that is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the United States and internationally. The company is also involved in the commercialization of Cholbam, a cholic acid capsule, which is approved as treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects and for adjunctive treatment of patients with peroxisomal disorders, including peroxisome biogenesis disorder-Zellweger spectrum disorder and Smith-Lemli-Opitz syndrome; and Chenodal, a tablet, which is approved for the treatment of radiolucent stones in the gallbladder, and under Phase 3 development for the treatment cerebrotendinous xanthomatosis. In addition, it develops Volixibat, an oral and minimally absorbed agent designed to inhibit IBAT, currently under Phase 2b clinical trial for the treatment of adult patients with cholestatic liver diseases. The company was incorporated in 2018 and is headquartered in Foster City, California.
Marinus Pharmaceuticals
NASDAQ:MRNSMarinus Pharmaceuticals, Inc., a pharmaceutical company, focuses on development and commercialization of therapeutic products for patients suffering from rare genetic epilepsies and other seizure disorders. It offers ZTALMY (ganaxolone), an oral suspension for the treatment of seizures associated with cyclin-dependent kinase-like 5 deficiency disorder for adult and pediatric patient populations in acute and chronic care, and in-patient and self-administered settings. The company's ZTALMY product candidate acts at synaptic and extrasynaptic GABAA receptors, a target for its anti-seizure, antidepressant, and anxiolytic potential. It is developing ganaxolone for treating genetic epilepsy disorders, such as PCDH19-related epilepsy, and tuberous sclerosis complex. Marinus Pharmaceuticals, Inc. has license agreement with Purdue Neuroscience Company and CyDex Pharmaceuticals, Inc.; and collaboration agreement with Orion Corporation and Tenacia Biotechnology (Shanghai) Co., Ltd. Marinus Pharmaceuticals, Inc. was incorporated in 2003 and is headquartered in Radnor, Pennsylvania.