Axsome Therapeutics
NASDAQ:AXSMAxsome Therapeutics, Inc., a biopharmaceutical company, engages in the development of novel therapies for central nervous system (CNS) disorders in the United States. The company's commercial product portfolio includes Auvelity (dextromethorphan-bupropion), a N-methyl-D-aspartate receptor antagonist with multimodal activity indicated for the treatment of major depressive disorder; and Sunosi (solriamfetol), a medication indicated to the treatment of excessive daytime sleepiness in patients with narcolepsy or obstructive sleep apnea. It is also developing AXS-05, which is in Phase III clinical trial to treat Alzheimer's disease agitation, as well as that has completed phase II clinical trial for the treatment of smoking cessation; AXS-07, an investigational medicine that has completed Phase III trials for the acute treatment of migraine; AXS-12, an investigational medicine, which is in Phase III trial to treat narcolepsy; AXS-14, a selective and potent norepinephrine reuptake inhibitor that has completed Phase III trial for the treatment of fibromyalgia and other conditions; and solriamfetol, a dual-acting dopamine and norepinephrine reuptake inhibitor, which has completed a Phase 2 trial for treating attention-deficit/hyperactivity disorder, and is in phase 2 major depressive, binge eating, and shift work disorder. Axsome Therapeutics, Inc. has a research collaboration agreement with Duke University for evaluating AXS-05 in smoking cessation. The company was incorporated in 2012 and is based in New York, New York.
MacroGenics
NASDAQ:MGNXMacroGenics, Inc., a biopharmaceutical company, develops, manufactures, and commercializes antibody-based therapeutics to treat cancer in the United States. Its approved product is MARGENZA (margetuximab-cmkb), a human epidermal growth factor receptor 2 (HER2) receptor antagonist indicated, in combination with chemotherapy, for the treatment of adult patients with metastatic HER2-positive breast cancer who have received two or more prior anti-HER2 regimens. The company's pipeline of immuno-oncology product candidates includes MGC018, an antibody drug conjugate (ADC), which targets solid tumors expressing B7-H3; Enoblituzumab, a monoclonal antibody that targets B7-H3; and MGD024, an investigational bispecific CD123 × CD3 DART molecule to minimize cytokine-release syndrome for patients with hematologic malignancies. In addition, it develops Lorigerlimab, a monoclonal antibody that targets the immune checkpoints PD-1 and cytotoxic T-lymphocyte-associated protein 4; Tebotelimab, an investigational tetravalent DART molecule for PD-1 and lymphocyte-activation gene 3; Retifanlimab, a humanized monoclonal antibody targeting programmed death receptor-1; and IMGC936, an ADC that targets ADAM9, a cell surface protein over-expressed in various solid tumor types. Further, the company develops MGD014 and MGD020, a DART molecule to target the envelope protein of human immunodeficiency virus infected cells and CD3 on T cells; Teplizumab for the treatment of type 1 diabetes; and PRV-3279, a CD32B × CD79B DART molecule for the treatment of autoimmune indications. It has collaborations with Incyte Corporation; Zai Lab Limited; I-Mab Biopharma; and Janssen Biotech, Inc. The company was incorporated in 2000 and is headquartered in Rockville, Maryland.
Omeros
NASDAQ:OMEROmeros Corporation, a clinical-stage biopharmaceutical company, discovers, develops, and commercializes small-molecule and protein therapeutics, and orphan indications targeting inflammation, complement-mediated diseases, cancers, and addictive and compulsive disorders. The company's clinical programs include Narsoplimab (OMS721/MASP-2) that has completed pivotal studies for hematopoietic stem-cell transplant-associated thrombotic microangiopathy (HSCT-TMA); that is in Phase III clinical trial for immunoglobulin A nephropathy (IgAN); and Phase II clinical trial to treat COVID-19. It also develops OMS1029 that is in phase I clinical trials for long-acting second-generation antibody targeting lectin pathway disorders; OMS906 that is in phase Ib clinical trials for Paroxysmal nocturnal hemoglobinuria, complement 3 glomerulopathy, and other alternative pathway disorders; OMS527 that is in phase I clinical trials for addictions and compulsive disorders, and movement disorders; and OMS405 that is in phase II clinical trials for opioid and nicotine addiction. In addition, the company develops MASP-2-small-molecule inhibitors for the treatment of aHUS, HSCT-TMA, and age-related macular degeneration; GPR174 inhibitors and chimeric antigen receptor T-Cell and adoptive T-Cell therapies for various cancers; and G protein-coupled receptors for treating immunologic, immuno-oncologic, metabolic, CNS, cardiovascular, musculoskeletal, and other disorders. Omeros Corporation was incorporated in 1994 and is headquartered in Seattle, Washington.
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.
Travere Therapeutics
NASDAQ:RTRXTravere Therapeutics, Inc. is a biopharmaceutical company. It engages in the identification, development, commercialization, and distribution of therapies to people living with rare diseases. Its products include Chenodal, Cholbam, and Thiola. The company was founded by Martin Shkreli on February 8, 2008 and is headquartered in San Diego, CA.