Akcea Therapeutics
NASDAQ:AKCAAkcea Therapeutics, Inc., a biopharmaceutical company, focuses on developing and commercializing drugs to treat patients with serious and rare diseases in the United States and internationally. The company offers TEGSEDI, which is designed to reduce the production of transthyretin protein. It develops WAYLIVRA, which has completed Phase III clinical study for the treatment of familial chylomicronemia syndrome; and that is in Phase III clinical study for the treatment of familial partial lipodystrophy. The company also develops AKCEA-APO(a)-LRx, which has completed Phase IIb clinical study for treating patients suffering with hyperlipoproteinemia; AKCEA-ANGPTL3-LRx that has completed Phase IIb clinical study for the treatment of multiple lipid disorders; and AKCEA-APOCIII-LRx, which is in Phase II study for the treatment of cardiovascular disease due to elevated triglyceride levels, as well as AKCEA-TTR-LRx to treat the broad population of patients with hereditary and wild-type forms of transthyretin amyloidosis. It has strategic collaboration with Novartis Pharma AG and PTC Therapeutics International Limited. The company was incorporated in 2014 and is headquartered in Boston, Massachusetts. Akcea Therapeutics, Inc. operates as a subsidiary of Ionis Pharmaceuticals, Inc.
Intercept Pharmaceuticals
NASDAQ:ICPTIntercept Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development and commercialization of therapeutics to treat progressive non-viral liver diseases in the United States, Europe, and Canada. The company markets Ocaliva, a farnesoid X receptor agonist used for the treatment of primary biliary cholangitis in combination with ursodeoxycholic acid in adults. It is also developing Ocaliva for various indications, including nonalcoholic steatohepatitis and NASH; and other product candidates in various stages of clinical and preclinical development. The company has a license agreement with Aralez Pharmaceuticals Canada Inc. to develop and commercialize bezafibrate in the United States. It markets its products through an internal commercial organization and third-party distributors. The company was incorporated in 2002 and is headquartered in Morristown, New Jersey. As of November 8, 2023, Intercept Pharmaceuticals, Inc. operates as a subsidiary of Alfasigma S.p.A..
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.
MyoKardia
NASDAQ:MYOKMyoKardia, Inc., a clinical stage biopharmaceutical company, discovers, develops, and commercializes targeted therapies for the treatment of serious and neglected rare cardiovascular diseases. Its lead product candidate is Mavacamten, an orally administered allosteric modulator of cardiac myosin, which is in Phase III clinical trial for the treatment of hypertrophic cardiomyopathy (HCM). The company also develops Danicamtiv, an orally administered small molecule, which completed Phase I and Phase IIa clinical trials on patients with dilated cardiomyopathy. Its preclinical programs include MYK-224, a small molecule program for the treatment of HCM targeting hypercontractility and impaired relaxation; ACT-1 for the treatment of diastolic dysfunction; and LUS-1 for the treatment of diastolic dysfunction. Myokardia, Inc. was founded in 2012 and is headquartered in Brisbane, California.
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.