Profile: Alexion Pharmaceuticals Inc (ALXN.O)
25 Aug 2016
Alexion Pharmaceuticals, Inc., incorporated on January 28, 1992, is a biopharmaceutical company. The Company is focused on the development and commercialization of life-transforming therapeutic products. The Company operates through innovation, development and commercialization of life-transforming therapeutic products segment. The Company's marketed products include Soliris (eculizumab), Strensiq (asfotase alfa) and Kanuma (sebelipase alfa). The Company's clinical programs include Soliris (eculizumab), ALXN 1101, ALXN 1007, SBC-103, ALXN 1210 and ALXN 5500.
In the Company's complement franchise, it offers Soliris for patients with either paroxysmal nocturnal hemoglobinuria (PNH), a life-threatening and ultra-rare genetic blood disorder, or atypical hemolytic uremic syndrome (aHUS), a life-threatening and ultra-rare genetic disease. PNH and aHUS are severe and ultra-rare disorders resulting from chronic uncontrolled activation of the complement component of the immune system. Soliris is designed to inhibit a specific aspect of the complement component of the immune system and thereby treat inflammation associated with chronic disorders in several therapeutic areas, including hematology, nephrology, transplant rejection and neurology. Soliris is a humanized monoclonal antibody that blocks terminal complement activity.
The Company has completed enrollment of patients in a Phase III multinational, placebo-controlled registration trial of eculizumab in patients with refractory generalized Myasthenia Gravis (MG). The Company is enrolling patients in a global, randomized, double-blind, placebo-controlled to evaluate eculizumab as a treatment for patients with relapsing neuromyelitis optica spectrum disorder (NMOSD). The Company has completed enrollment in a single, multinational, placebo-controlled delayed kidney transplant graft function (DGF) registration trial. The Company has completed a randomized, open-label, multicenter Phase II clinical trial of eculizumab presensitized kidney transplant patients at an elevated risk of antibody mediated rejection (AMR) receiving kidneys from living donors.
Strensiq (asfotase alfa)
In the Company's metabolic franchise, it markets Strensiq for the treatment of patients with Hypophosphatasia (HPP). HPP is a genetic ultra-rare disease characterized by defective bone mineralization that can lead to deformity of bones and other skeletal abnormalities. Strensiq is a targeted enzyme replacement therapy. It is designed to address underlying causes of HPP by aiming to restore the genetically defective metabolic process, thereby preventing or reversing the severe and potentially life-threatening complications in patients with HPP.
Kanuma (sebelipase alfa)
The Company offers Kanuma for the treatment of patients with Lysosomal Acid Lipase Deficiency (LAL-D). Kanuma is a recombinant form of the human LAL enzyme. It is an enzyme-replacement therapy, which is approved for the treatment for patients with LAL-D.
cPMP (ALXN 1101)
The Company has completed evaluation of its synthetic form of cyclic Pyranopterin Monophosphate (cPMP) replacement therapy in a Phase I healthy volunteer study. The Company has completed enrollment in a multi-center, multinational open-label clinical trial of synthetic cPMP in patients with Molybdenum Cofactor Deficiency (MoCD) Type A switched from treatment with recombinant cPMP. The Company has commenced the Phase II/III pivotal open-label, single-arm trial of ALXN1101 for treatment-naive neonates with MoCD Type A.
The Company's product candidate, ALXN 1007, is a humanized antibody designed to target rare and severe inflammatory disorders and is a product of its antibody discovery technologies. The Company has completed enrollment in both a Phase I single-dose, dose escalating safety and pharmacology study in healthy volunteers, as well as in a multi-dose, dose escalating safety and pharmacology study in healthy volunteers. The Company is conducting a proof-of-concept study in patients with an ultra-rare disorder, gastrointestinal graft versus host disease (GI-GVHD). The Company is conducting Phase II proof-of-concept study in patients with non-criteria manifestations of anti-phospholipid syndrome (APS).
The Company's product candidate, 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.
The Company's product candidate, ALXN 1210, is a next-generation complement inhibitor in development for PNH and other indications. The Company is conducting a multiple-ascending dose study of ALXN 1210 to evaluate the safety and efficacy of ALXN 1210. In addition, it has over two clinical studies of ALXN 1210 in patients with PNH. The Company also has initiated an open-label, multi-dose Phase II study of ALXN 1210 in patients with PNH that is designed to measure change in lactate dehydrogenase (LDH) levels and safety in several dosing cohorts and intervals.
Alexion Pharmaceuticals Inc
100 College St
NEW HAVEN CT 06510-3210
Company Web Links
- BRIEF-Alexion Pharmaceuticals-FDA recently completed routine current good manufacturing practice inspection of Co's Smithfield, Rhode Island Manufacturing Facility
- BRIEF-Alexion reports Q2 non-gaap EPS $1.13
- U.S. RESEARCH ROUNDUP-D.R. Horton, Humana, Chicago Bridge & Iron
- BRIEF-Alexion presents new SBC-103 data in patients with MPS iiiB
- BRIEF-Alexion Pharmaceuticals announces data from phase 1/2 study of ALXN1210