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Profile: Alexion Pharmaceuticals Inc (ALXN.OQ)

ALXN.OQ on NASDAQ Stock Exchange Global Select Market

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Alexion Pharmaceuticals, Inc., incorporated on January 28, 1992, is a biopharmaceutical company. The Company is focused on the development and commercialization of therapeutic products. The Company's products include Soliris (eculizumab), Strensiq (asfotase alfa) and Kanuma (sebelipase alfa). The Company's clinical development programs include Soliris (eculizumab), cPMP (ALXN1101), SBC-103, ALXN1210 (IV) and ALXN1210 (Subcutaneous).

The Company's product, Soliris is the therapeutic approved for patients with either paroxysmal nocturnal hemoglobinuria (PNH) or atypical hemolytic uremic syndrome (aHUS). PNH and aHUS result from chronic uncontrolled activation of the complement component of the immune system. Soliris is designed to inhibit a specific aspect of the component of the immune system and treat inflammation associated with chronic disorders in various therapeutic areas, including hematology, nephrology, neurology and transplant rejection. It is a humanized monoclonal antibody that blocks terminal complement activity. It is approved for the treatment of PNH in the United States, Europe, Japan and in various other territories. It is approved for the treatment of pediatric and adult patients with aHUS in the United States, Europe and Japan.

The Company's product, Soliris (eculizumab) is in the Phase III REGAIN trial of eculizumab for the treatment of Refractory Generalized Myasthenia Gravis (gMG). Eculizumab is also approved for treatment for patients with relapsing neuromyelitis optica spectrum disorder (NMOSD). The Company's product, Strensiq is for the treatment of patients with Hypophosphatasia (HPP). Strensiq is a targeted enzyme replacement therapy and an approved therapy for patients with HPP, and is designed to address the underlying causes of HPP by aiming to restore the genetically defective metabolic process, thereby preventing or reversing the complications in patients with HPP. Its product, Kanuma is for the treatment of patients with Lysosomal Acid Lipase Deficiency (LAL-D). It is a recombinant form of the human LAL enzyme, which is a replacement therapy that is approved for the treatment for patients with LAL-D.

The Company's SBC-103, is a recombinant form of natural human alpha-N-acetyl-glucosaminidase (NAGLU) enzyme, which leads to a buildup of abnormal amounts of heparan sulfate (HS) in the brain and throughout the body. SBC-103 is designed to replace the missing (or deficient) NAGLU enzyme. Its ALXN1101 is in the Phase II/III trial for treatment-naive neonates with Molybdenum Cofactor Deficiency (MoCD) Disease Type A (MoCD Type A). Its ALXN1210 is an anti-C5 antibody that inhibits terminal complement. The ALXN1210 is in the Phase II trial for the treatment of patients with PNH that is designed to measure change in lactate dehydrogenase (LDH) levels and safety in several dosing cohorts and intervals. It is in the Phase III trial for the treatment of aHUS. ALXN1210 is in the Phase I trial for the treatment of Subcutaneous (SC) Delivery.

Company Address

Alexion Pharmaceuticals Inc

121 Seaport Blvd
BOSTON   MA   02210-2050
P: +1475.2302596
F: +1203.2718198

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