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.
Global Blood Therapeutics
NASDAQ:GBTGlobal Blood Therapeutics, Inc., a biopharmaceutical company, engages in the discovery, development, and delivery of treatments for underserved patient communities with sickle cell disease (SCD). The company offers Oxbryta tablets, an oral, once-daily therapy for SCD. It also evaluates the safety and pharmacokinetics of single and multiple doses of Oxbryta in a Phase II a clinical trial of adolescent and pediatric patients with SCD. In addition, the company is developing its lead product candidate inclacumab, novel human monoclonal antibody, which is in Phase III a clinical trial to reduce the incidence of painful vaso-occlusive crises, and resulting hospital admissions; and GBT601, a hemoglobin polymerization inhibitor that is in Phase I development. It has a license and collaboration agreement with Syros Pharmaceuticals, Inc. to discover, develop, and commercialize therapies for SCD and beta thalassemia; and Sanofi S.A. to two early-stage research programs in SCD. Global Blood Therapeutics, Inc. was incorporated in 2011 and is headquartered in South San Francisco, California.
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.
Ligand Pharmaceuticals
NASDAQ:LGNDLigand Pharmaceuticals Incorporated, a biopharmaceutical company, engages in the development and licensing of biopharmaceutical assets worldwide. Its commercial programs include Kyprolis and Evomela, which are used to treat multiple myeloma; Rylaze, a recombinant erwinia asparaginase for the treatment of acute lymphoblastic leukemia or lymphoblastic lymphoma in adult and pediatric patients; Filspari, a dual endothelin and angiotensin II receptor antagonist in development for rare kidney diseases and non-immunosuppressive treatment indicated for immunoglobulin A nephropathy; Teriparatide injection product for osteoporosis; Vaxneuvance for the prevention of invasive disease caused by streptococcus pneumoniae serotypes; and Pneumosil, a pneumococcal conjugate vaccine to help fight against pneumococcal pneumonia among children. The company also offers TZIELD, a CD3-directed antibody indicated to delay the onset of Stage 3 type 1 diabetes (T1D) in adults and children aged 8 years and older with Stage 2 T1D; Nexterone, a captisol-enabled formulation of amiodarone; Zulresso, a captisol-enabled formulation of brexanolone for the treatment of postpartum depression; and Veklury, an antiviral treatment for moderate or severe COVID-19. In addition, it provides Noxafil-IV, a captisol-enabled formulation of posaconazole for IV use; Duavee for the treatment of post-menopausal symptoms in women; Exemptia for autoimmune diseases; Vivitra for breast cancer; and Bryxta and Zybev for various indications. The company has alliances, licenses, and other business relationships with Amgen, Merck, Pfizer, Jazz, Takeda, Gilead Sciences, and Baxter International. Further, it sells Captisol materials. Ligand Pharmaceuticals Incorporated was incorporated in 1987 and is based in Jupiter, Florida.
Xencor
NASDAQ:XNCRXencor, Inc., a clinical stage biopharmaceutical company, focuses on the discovery and development of engineered monoclonal antibody and cytokine therapeutics to treat patients with cancer and autoimmune diseases. The company provides Sotrovimab that targets the SARS-CoV-2 virus; Ultomiris for the treatment of patients with paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome; and Monjuvi for the treatment of patients with relapsed or refractory diffuse large B-cell lymphoma. It develops Plamotamab, a bispecific antibody, which is in Phase I clinical trial to treat non-Hodgkin lymphoma; Vudalimab, a bispecific antibody, which is in Phase II clinical trial to treat metastatic castration-resistant prostate cancer and other solid tumor types. The company is also developing XmAb306, which is in Phase I clinical trial to treat solid tumors; XmAb104, which is in Phase II clinical trial to treat patients with selected solid tumors; XmAb564 that is in Phase Ia clinical trial to treat autoimmune diseases; AMG 509, which is in Phase I clinical trial to treat prostate cancer; XmAb819 for patients with renal cell carcinoma; XmAb541 for the treatment of ovarian cancer; and XmAb662 which is in Phase I clinical trial to treat patients with solid tumors. In addition, the company develops VIR-3434, which is in Phase II clinical trial for patients with hepatitis B virus infection; and VIR-2482 that is in Phase 2 clinical trial to trat hepatitis B virus. The company develops AIMab7195 to reduce blood serum levels of IgE that mediates allergic responses and allergic disease; Obexelimab to treat autoimmune disease; and Xpro1595 to treat patients with Alzheimer's disease, and depression. It has a license agreement with Caris Life Sciences. Xencor, Inc. was incorporated in 1997 and is headquartered in Pasadena, California.