Amicus Therapeutics
NASDAQ:FOLDAmicus Therapeutics, Inc., a biotechnology company, focuses on discovering, developing, and delivering medicines for rare diseases. Its commercial product and product candidates include Galafold, an oral precision medicine for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene variant; and Pombiliti + Opfolda, for the treatment of late-onset. It has collaboration and license agreements with the University of Pennsylvania to research and develop parvovirus gene therapy products; and GlaxoSmithKline. The company was incorporated in 2002 and is headquartered in Princeton, New Jersey.
Galapagos
NASDAQ:GLPGGalapagos NV, an integrated biopharmaceutical company, engages in the discovery, development, and commercialization of?various medicines for high unmet medical need. Its pipeline products include filgotinib, a JAK1 inhibitor that is in various phases of clinical trials for the treatment of rheumatoid arthritis, Crohn's disease, ulcerative colitis, ankylosing spondylitis, psoriatic arthritis, and uveitis. The company's pipeline products also comprise GLPG3667 that has completed Phase 1b trial; and GLPG5101 and GLPG5201, CD19 CAR-T product candidate manufactures at point-of-care, that is in Phase I/II in relapsed/refractory non-Hodgkin lymphoma and chronic lymphocytic leukemia. The company has collaboration agreements with Gilead Sciences, Inc.; AbbVie S.à r.l.; and Novartis Pharma AG. Galapagos NV was incorporated in 1999 and is headquartered in Mechelen, Belgium.
Insmed
NASDAQ:INSMInsmed Incorporated is a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases. Insmed's first commercial product is ARIKAYCE® (amikacin liposome inhalation suspension), which is approved in the United States for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen for adult patients with limited or no alternative treatment options. MAC lung disease is a rare and often chronic infection that can cause irreversible lung damage and can be fatal. Insmed's earlier-stage clinical pipeline includes INS1007, a novel oral reversible inhibitor of dipeptidyl peptidase 1 with therapeutic potential in non-cystic fibrosis bronchiectasis and other inflammatory diseases, and INS1009, an inhaled formulation of a treprostinil prodrug that may offer a differentiated product profile for rare pulmonary disorders, including pulmonary arterial hypertension.
Intra-Cellular Therapies
NASDAQ:ITCIIntra-Cellular Therapies, Inc., a biopharmaceutical company, focuses on the discovery, clinical development, and commercialization of small molecule drugs that address medical needs primarily in neuropsychiatric and neurological disorders by targeting intracellular signaling mechanisms in the central nervous system (CNS) in the United States. The company offers CAPLYTA for the treatment of schizophrenia and bipolar depression in adults. It is also involved in developing Lumateperone, which is in Phase 3 clinical trial for the treatment of various depressive disorders, as well as additional neuropsychiatric indications. In addition, the company is developing Lenrispodun (ITI-214) for the treatment of Parkinson's disease, CNS, and other disorders; ITI-1284 for the treatment of neuropsychiatric disorders and behavioral disturbances in dementia; and ITI-333 for substance use disorders, pain, and psychiatric comorbidities, including depression, anxiety, and sleep disorders. Intra-Cellular Therapies, Inc. was founded in 2002 and is headquartered in New York, New York.
Arcus Biosciences
NYSE:RCUSArcus Biosciences, Inc., a clinical-stage biopharmaceutical company, develops and commercializes cancer therapies in the United States. The company's pipeline products include Domvanalimab, an anti-TIGIT antibody, which is in Phase 2 and Phase 3 clinical trial; and AB308, an investigational anti-TIGIT monoclonal antibody, which is in Phase 1b clinical trial to study people with advanced solid and hematologic malignancies. It also develops Etrumadenant, a dual A2a/A2b adenosine receptor antagonist, which is in Phase 2 clinical trial; Quemliclustat, a small-molecule CD73 inhibitor, which is Phase 1b and Phase 2 clinical trial; Zimberelimab, an anti-PD-1 antibody, which is in Phase 2 clinical trial for metastatic cell lung cancer and monotherapy; and AB521, an oral and small-molecule inhibitor of HIF-2a, which is in Phase 1 clinical trial for the treatment of Von Hippel-Lindau disease. In addition, the company's preclinical pipeline products include AB598, a CD39 antibody; and AB801, a small molecule Axl inhibitor. It has a clinical collaboration with AstraZeneca to evaluate domvanalimab in combination with durvalumab in a registrational phase 3 clinical trial in patients with unresectable Stage 3 NSCLC; and BVF Partners L.P. to support the discovery and development of compounds for the treatment of inflammatory diseases. Arcus Biosciences, Inc. was incorporated in 2015 and is headquartered in Hayward, California.