Profile: Alexion Pharmaceuticals, Inc. (ALXN.O)

Alexion Pharmaceuticals, Inc. (Alexion), incorporated in January 1992, is a biopharmaceutical company engaged in the discovery, development and delivery of biologic therapeutic products for the treatment disease, including hematologic and neurologic diseases, cancer and autoimmune disorders. The Company has one marketed product, Soliris (eculizumab), which is a therapy approved for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH). In June 2007, the European Commission (E.C.) approved the use of Soliris for patients with PNH in the European Union, which also serves as the basis for approval in Iceland and Norway. Alexion has focused its product development programs on anti-inflammatory therapeutics for diseases.

Soliris is designed to inhibit a specific aspect of the complement component of the immune system, and thereby treat inflammation related to chronic hematologic, neurologic and autoimmune disorders. Alexion submitted an application for marketing authorization in Australia for Soliris for the treatment of patients with PNH and the application was accepted for priority review. Soliris has received Orphan Drug Designation in Australia. In September 2008, the Company was granted limited marketing authorization for Soliris for the treatment of patients with PNH in Switzerland. In January 2009, Health Canada approved the use of Soliris for patients with PNH in Canada. In October 2008, it completed the 12-week AEGIS study of Japanese patients. PNH is a debilitating acquired genetic deficiency blood disorder defined by the destruction of red blood cells. Patients with PNH have an acquired genetic deficiency in certain protective proteins on the surface of their blood cells, allowing their own complement system to attack and destroy these blood cells.

Myasthenia gravis (MG) is an autoimmune syndrome characterized by the failure of neuromuscular transmission. Patients with MG produce antibodies that damage communication between an individual's nerve ending and muscle. During the year ended December 31, 2007, the Company filed an investigational new drug (IND) with the United States food and drug administration (FDA) to initiate clinical development and received authorization from the FDA in July 2008. Asthma is a chronic respiratory disease that results in bronchial inflammation and airway constriction that prompts asthma’s symptoms, including shortness of breath, chest tightness and wheezing. During the year ended December 31, 2008, the Company completed a phase I/II proof of concept study of IV eculizumab in allergic asthmatic patients.

Multifocal motor neuropathy (MMN) is a rare autoimmune disorder in which autoantibodies attack the nerve-muscle junctions. Patients with MMN demonstrate a slow progressive asymmetrical weakness of limbs without sensory loss. Antibodies and complement activation products have been identified at the nerve-muscle junctions in diseases similar to MMN. As of December 31, 2008, the Company is developing an antibody for the treatment of B-Cell chronic lymphocytic leukemia (B-CLL), an incurable chronic cancer that results from expansion of B-lymphocytes and other cancers including blood tumors, such as multiple myeloma (MM). Its antibody binds to CD200, a protein that is upregulated on the surface of B-CLL and MM tumor cells. Alexion’s antibody targets CLL and MM cells and blocks the interaction of CD200 with the CD200 receptor, with the objective of enhancing the body’s immune response to these tumors.