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Profile: Alexion Pharmaceuticals Inc (ALXN.O)

ALXN.O on Nasdaq

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28 Apr 2016
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Alexion Pharmaceuticals, Inc. (Alexion), incorporated on January 28, 1992, is a biopharmaceutical company. The Company is focused on the development and commercialization of life-transforming therapeutic products. As of June 23, 2015, the Company had eight product candidates in clinical trials for 11 indications. The Company's product pipeline includes complement inhibitor portfolio, metabolic rare disease portfolio and preclinical candidates. Its complement inhibitor portfolio includes Soliris, ALXN1007, ALXN1210 and ALXN5500. Its metabolic rare disease portfolio includes Strensiq, Kanuma, ALXN1101 and SBC-103. The Company's preclinical candidates include mRNA Therapies, SBC-105 and other complement inhibitors.

Complement inhibitor portfolio

The Company’s Soliris (eculizumab) is a humanized antibody therapeutic approved for patients with two ultra-rare and severe disorders resulting from chronic uncontrolled activation of the complement component of the immune system: paroxysmal nocturnal hemoglobinuria (PNH), an ultra-rare and life-threatening blood disorder, and atypical hemolytic uremic syndrome (aHUS), an ultra-rare and life-threatening genetic disease. The Company is conducting advanced clinical development studies for eculizumab for other indications, including refractory myasthenia gravis (MG), relapsing neuromyelitis optica (NMO) and delayed graft function (DGF). It is conducting early clinical development studies for eculizumab for the treatment of antibody mediated rejection (AMR).

The Company’s product, eculizumab, is in Phase II clinical trial for the treatment of antibody mediated rejection (AMR), Phase III clinical trial for the treatment of NMO, Phase II for the treatment of MG and Phase III for the treatment of Delayed Kidney Transplant Graft Function. ALXN 1007 is a humanized anti-inflammatory antibody for the treatment of rare and inflammatory disorders. The Company commenced a Phase II study of ALXN 1007 in patients with anti-phospholipid syndrome (APS). APS is an ultra-rare autoimmune, hypercoagulable state caused by anti-phospholipid antibodies. ALXN 1007 is in Phase II clinical trial for the treatment of Gastrointestinal Graft-versus-Host Disease (GI-GVHD) and Phase II clinical trial for the treatment of anti-phospholipid syndrome. The Company is also conducting early clinical development studies for complement inhibitor portfolio ALXN1210 and ALXN5500.

Metabolic rare disease portfolio

The Company's Strensiq (asfotase alfa) is a bone-targeted enzyme replacement therapy for the treatment of the underlying cause of Hypophosphatasia (HPP). Asfotase alfa, a targeted enzyme replacement therapy in Phase II clinical trials for patients with HPP, directly addresses the morbidities and mortality of HPP by targeting alkaline phosphatase to the deficient tissue.

The Company's Kanuma (sebelipase alfa) is a recombinant form of the human LAL enzyme being developed as an enzyme replacement therapy for patients with Lysosomal Acid Lipase Deficiency (LAL-D), a genetic, chronic and rare metabolic disease associated with multi-systemic organ damage, including hepatic fibrosis, cirrhosis, liver failure, accelerated atherosclerosis, cardiovascular disease and other devastating consequences. The Company is also conducting preclinical studies on Asfotase alfa for the treatment for patients with Neurofibromatosis Type 1 (NF-1). The Company's ALXN1101 (cPMP Replacement Therapy) is an enzyme co-factor replacement therapy for the treatment of patients with molybdenum cofactor deficiency (MoCD) type A.

The Company's SBC-103 is an enzyme replacement therapy. The Company is investigating SBC-103 for the treatment for patients with MPS IIIB, a rare, devastating and life-threatening disease that presents in children during the first few years of life and causes progressive and severe cognitive decline, behavioral problems, speech loss, increasing loss of mobility and premature death.

Preclinical candidates

The Company's preclinical candidates include mRNA therapies, SBC-105 and other complement inhibitors. Its SBC-105 is being investigated for the treatment of generalized arterial calcification of infancy (GACI).

Company Address

Alexion Pharmaceuticals Inc

100 College St
NEW HAVEN   CT   06510-3210
P: +1203.2722596
F: +1302.6555049

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