Profile: Alexion Pharmaceuticals Inc (ALXN.O)
7 Mar 2014
Alexion Pharmaceuticals, Inc. (Alexion), incorporated on January 28, 1992, is a biopharmaceutical company focused on serving patients with severe and ultra-rare disorders through the development and commercialization of life-transforming therapeutic products. Its marketed product Soliris (eculizumab) is the first and only 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 also evaluating other indications for Soliris in other diseases in which chronic uncontrolled complement activation is the underlying mechanism, and it is progressing with other biotechnology product candidates in ultra-rare and severe disorders, which are in various stages of development. On February 7, 2012, it acquired Enobia Pharma Corp. (Enobia). On February 8, 2011, it acquired patents and assets from Orphatec Pharmaceuticals GmbH (Orphatec). On January 28, 2011, it acquired Taligen Therapeutics, Inc. (Taligen). It acquired Asfotase alfa in February 2012.
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 the therapeutic areas of hematology, nephrology, including transplant rejection and neurology. Soliris is a humanized antibody which, administered at the doses prescribed, generally blocks complement activity for one to two weeks after a single dose. Soliris was approved for the treatment of aHUS by the Food and Drug Administration (FDA) and the EC, during the year ended December 31, 2011. Soliris was granted orphan drug designation for the treatment of Atypical Hemolytic Uremic Syndrome (aHUS) in the United States and Europe. Soliris is a therapy approved for the treatment of patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). aHUS is a chronic and life-threatening disease, in which uncontrolled complement activation causes blood clots in small blood vessels throughout the body (thrombotic microangiopathy) leading to kidney failure, stroke, heart attack and death. Shiga-toxin producing E. Coli Hemolytic Uremic Syndrome (STEC-HUS) is a disorder that results from exposure to Enterohemorrhagic E.Coli, (EHEC). Its STEC-HUS development program was initiated in connection with EHEC in Germany in May and June 2011. Dense Deposit Disease (DDD) also called Type II membrano-proliferative glomerulonephritis, is a form of glomerulonephritis, associated with genetic mutations in complement inhibitor genes leading to sustained complement activation and inflammation.
Myasthenia Gravis (MG) is an autoimmune syndrome characterized by complement activation leading to failure of neuromuscular transmission. NMO is a autoimmune disease of the central nervous system (CNS) that affects the optic nerves and spinal cord. Age-related macular degeneration is a medical condition usually affecting older adults, in which uncontrolled complement activation results in a loss of vision in the center of the visual field (the macular) and complement-mediated damage to the retina. AMD is a cause of visual impairment in older adults.
Hypophosphatasia (HPP) is a genetic, and life-threatening metabolic disease characterized by defective bone mineralization and impaired phosphate and calcium regulation, leading to progressive damage to multiple vital organs, including destruction and deformity of bones, profound muscle weakness, seizures, impaired renal function, and respiratory failure. 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 directly to the deficient tissue.
Molybdenum Cofactor Deficiency (MoCD) Disease Type A (MoCD Type A) is a metabolic disorder characterized by severe and rapidly progressive neurologic damage and death in newborns, MoCD Type A results from a genetic deficiency in cyclic Pyranopterin Monophosphate (cPMP), a molecule that enables production of certain enzymes, the absence of which allows neurotoxic sulfite to accumulate on the brain. As of December 31, 2011, there is no approved therapy available for MoCD Type A. The Company acquired assets related to a cPMP replacement therapy from Orphatec Pharmaceuticals GmbH in February 2011. There has been some early clinical experience with the cPMP replacement therapy in a small number of children with MoCD type A.
TT30 is an alternative complement inhibitor with a mechanism of action from Soliris. The Company acquired a portfolio of preclinical product candidates, including TT30, in January 2011. TT30 is being investigated in a Phase I single dose, dose escalating safety and pharmacology study.
Samalizumab is a humanized monoclonal antibody directed against the cell surface protein CD200. Samalizumab is designed to modulate the immune system and destroy tumors expressing the CD200 protein. The FDA authorized its IND to evaluate the activity of samalizumab, an antibody to the immune regulator CD200, in patients with chronic lymphocytic leukemia (CLL). CLL is a type of cancer of the blood and bone marrow. Enrollment and dosing has been completed in its Phase I dose-escalation clinical study of samalizumab in patients with treatment refractory CLL or multiple myeloma.
Alexion Pharmaceuticals Inc
352 Knotter Drive
CHESHIRE CT 06410
Company Web Links
- British watchdog wants U.S. biotech Alexion to justify cost of drug
- Alexion Pharma sees $2 billion sales, lower tax rate in 2014
- UPDATE 1-Alexion Pharma sees $2 bln sales, lower tax rate in 2014
- Alexion Pharma sees Soliris sales topping $2 bln in 2014
- UPDATE 2-Roche CEO does not rule out move into rare diseases