Aptinyx
NASDAQ:APTXAptinyx Inc., a clinical-stage biopharmaceutical company, focuses on the discovery, development, and commercialization of novel, proprietary, and synthetic small molecules for the treatment of brain and nervous system disorders. Its lead product candidate NYX-783, an N-methyl-D-aspartate receptors (NMDAr) modulator that is in Phase I clinical trial to treat opioid use disorder, or OUD; and in preclinical trial for the treatment of alcohol use disorder, or AUD. The company has a research collaboration agreement with Allergan plc. Aptinyx Inc. was incorporated in 2015 and is headquartered in Evanston, Illinois.
Eton Pharmaceuticals
NASDAQ:ETONEton Pharmaceuticals, Inc., a specialty pharmaceutical company, focuses on developing, acquiring, and commercializing pharmaceutical products for rare diseases. The company offers ALKINDI SPRINKLE, a replacement therapy for adrenocortical insufficiency in children under 17 years of age; Carglumic Acid for the treatment of acute and chronic hyperammonemia due to N-acetylglutamate Synthase deficiency; Betaine Anhydrous for the treatment of homocystinuria; and Nitisinone for the treatment of tyrosinemia type 1. It also provides Zeneo hydrocortisone autoinjector for the treatment of adrenal crisis. Eton Pharmaceuticals, Inc. was incorporated in 2017 and is based in Deer Park, Illinois.
Fulcrum Therapeutics
NASDAQ:FULCFulcrum Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing products for improving the lives of patients with genetically defined diseases in the areas of high unmet medical need in the United States. Its product candidates are losmapimod, a small molecule for the treatment of facioscapulohumeral muscular dystrophy is under phase III clinical trial; and pociredir, a fetal hemoglobin inducer for the treatment of sickle cell disease and beta-thalassemia is under phase I clinical trial. The company is also discovering drug targets for the treatments of rare neuromuscular, muscular, central nervous system, and hematologic disorders, as well as cardiomyopathies and pulmonary diseases. Fulcrum Therapeutics, Inc. has collaboration and license agreement with Acceleron Pharma Inc. to identify biological targets to modulate specific pathways associated with a targeted indication within the pulmonary disease space; and MyoKardia, Inc. to discover and develop therapies for the treatment of genetic cardiomyopathies. Fulcrum Therapeutics, Inc. was Incorporated in 2015 and is headquartered in Cambridge, Massachusetts.
Strongbridge Biopharma
NASDAQ:SBBPStrongbridge Biopharma Plc operates as a commercial-stage biopharmaceutical company, which focuses on the development and commercialization of therapies for rare diseases. Its pipeline is comprised of Keveyis, Macrilen, Recorlev, and Veldoreotide. The company was founded in 1996 and is headquartered in Trevose, PA.
scPharmaceuticals
NASDAQ:SCPHscPharmaceuticals Inc., a pharmaceutical company, engages in the development and commercialization of various pharmaceutical products. Its lead product candidate is FUROSCIX that consists of formulation of furosemide, which is delivered through an on-body infusor for treatment of congestion due to fluid overload in adults with chronic heart failure and kidney disease, as well as consists of subcutaneous loop diuretic that delivers IV equivalent diuresis at home; and FUROSCIX On-Body Infusor, a drug-device combination product consisting of FUROSCIX. The company's product pipeline also includes SCP-111 (furosemide injection), an investigational pH neutral aqueous furosemide formulation that is being developed for subcutaneous administration outside of the hospital setting, including patient self-administration in the home; and SCP-111 Autoinjector, an investigational single-entity, drug-device combination product candidate consisting of a prefilled syringe containing SCP-111, preloaded into a commercially available, fixed single dose, disposable, two step mechanical autoinjector. It has a collaboration agreement with West Pharmaceutical Services, Inc. The company was incorporated in 2013 and is headquartered in Burlington, Massachusetts.