Aeglea BioTherapeutics
NASDAQ:AGLEAeglea BioTherapeutics, Inc., a clinical-stage biotechnology company, designs and develops human enzyme therapeutics for the treatment of rare metabolic diseases. The company's therapeutic candidates include pegtarviliase, a polyethylene glycol modified, which is in Phase I/II clinical trial for the treatment of patient with homocystinuria; and pegzilarginase, a recombinant human Arginase 1 that is in Phase III PEACE trial to evaluate the safety and efficacy for the treatment of Arginase 1 deficiency. In addition, its preclinical pipeline includes AGLE-325 for the treatment of cystinuria, as well as other research programs. The company was formerly known as Aeglea BioTherapeutics Holdings, LLC and changed its name to Aeglea BioTherapeutics, Inc. in March 2015. Aeglea BioTherapeutics, Inc. was founded in 2013 and is headquartered in Austin, Texas.
AMAG Pharmaceuticals
NASDAQ:AMAGAMAG Pharmaceuticals, Inc., a pharmaceutical company, develops, manufactures, and commercializes therapeutics for maternal and women's health, and anemia management in the United States. The company markets Feraheme (ferumoxytol), an intravenous iron replacement therapeutic agent for the treatment of iron deficiency anemia in adult patients who have intolerance to oral iron or have had unsatisfactory response to oral iron, as well as patients who have chronic kidney disease; Makena, a hydroxyprogesterone caproate injection to reduce the risk of preterm birth in women pregnant with a single baby who have a history of singleton spontaneous preterm birth; Intrarosa(prasterone) vaginal insert steroid for the treatment of dyspareunia due to menopause; and Vyleesi (bremelanotide), an auto-injector device for the treatment of hypoactive sexual desire disorder in pre-menopausal women. Its product candidates also include AMAG-423 (ovine), an antibody fragment, which is in Phase 2b/3a trial for the treatment of severe preeclampsia in pregnant women; and Ciraparantag, an anticoagulant reversal agent that is in planned Phase 2b trial for the treatment of novel oral anticoagulants or low molecular weight heparin. The company sells Feraheme to authorized wholesalers and specialty distributors. The company has license agreements with Endoceutics, Inc., Palatin Technologies, Inc., Velo Bio, LLC, Prasco, LLC, Perosphere Pharmaceuticals Inc., and Antares Pharma, Inc. AMAG Pharmaceuticals, Inc. was founded in 1981 and is headquartered in Waltham, Massachusetts.
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.
Magenta Therapeutics
NASDAQ:MGTAMagenta Therapeutics, Inc., a biotechnology company focus on improving stem cell transplantation. Its product candidates are designed to bring the curative power of stem cell transplant to patients with blood cancers, genetic diseases, and autoimmune diseases. The company's product portfolio includes MGTA-117, an anti-CD117 antibody conjugated to an amanitin payload that targets hematopoietic stem cells (HSCs) and leukemia cells; and MGTA-45, an anti-human CD45 antibody conjugated to a DNA-interacting payload for HSCs, leukemia cells, and immune cells. In addition, it focus on development of ADC-based conditioning program that targets a receptor of T cells; and novel conditioning and translation stem cell sciences, as well as develops cell therapy program, E478, which is a small molecule aryl hydrocarbon receptor antagonist for stem cell-based gene therapy and genome editing. In addition, the company has a research and clinical collaboration agreement with AVROBIO, Inc. for the treatment of lysosomal storage disorders; and Beam Therapeutics, Inc. to evaluate the potential utility of MGTA-117 for conditioning of patients with sickle cell disease and beta-thalassemia. Magenta Therapeutics, Inc. was formerly known as HSCTCo Therapeutics, Inc. and changed its name to Magenta Therapeutics, Inc. in February 2016. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.
Ovid Therapeutics
NASDAQ:OVIDOvid Therapeutics Inc., a biopharmaceutical company, engages in the development of impactful medicines for patients and families with epilepsies and seizure-related neurological disorders in the United States. The company is developing soticlestat, a novel cholesterol 24 hydroxylase inhibitor, which is in Phase 3 clinical trials for the potential treatment of patients with resistant epilepsies; OV329, a GABA aminotransferase inhibitor which is in Phase 1 clinical trials for the treatment of seizures associated with tuberous sclerosis complex and infantile spasms; and OV350, a small molecule direct activator of the KCC2 transporter, which is in Phase 1 clinical trials for treating epilepsies. It also develops OV815, that focuses on the mutations associated with KIF1A-associated neurological disorder (KAND); OV888 (GV101), a highly selective rock2 inhibitor which is in Phase 1 double-blind multiple-ascending dose trial; OV825, has advanced to potential candidate lead identification for the rare neurodevelopmental condition HNRNPH2 (Bain Syndrome); and OV882, a short hairpin RNA gene therapy for the treatment of Angelman syndrome. The company has license and collaboration agreements with Healx, AstraZeneca AB, H. Lundbeck A/S, Northwestern University, and Graviton, as well as Marinus Pharmaceuticals, Inc. The company was incorporated in 2014 and is headquartered in New York, New York.