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First Clinical Experience With Soliris(R) in Treating Patients With Two Rare Complement-Mediated Diseases Presented at ASH Annual Meeting
Date:12/7/2008

CHESHIRE, Conn., Dec. 7 /PRNewswire-FirstCall/-- Soliris(R) (eculizumab), a terminal complement inhibitor developed by Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), was associated with an improvement in hematologic parameters in a limited number of patients with two other rare and severe complement-mediated diseases treated outside of a clinical trial, according to patient cases presented today at the 50th Annual Meeting of the American Society of Hematology (ASH).

Physicians reported on the first clinical experience with Soliris in patients with atypical Hemolytic Uremic Syndrome (aHUS) and Cold Agglutinin Disease (CAD), two rare and serious diseases. Like patients with PNH, patients with aHUS are missing or have defective complement inhibitors that help to regulate the body's immune system. These patients suffer from hemolysis, blood clotting which can be measured in part as reduced number of circulating platelets, and kidney damage. In two patients with aHUS, Soliris administration was associated with improvements in platelet levels and reduction in hemolysis. Patients with CAD suffer from an autoimmune attack on their red blood cells leading to severe complement activation and hemolysis (destruction of those blood cells), anemia, and poor quality of life. In one patient with CAD, Soliris treatment was associated with reduced hemolysis, absence of the need for blood transfusions and improved symptoms of fatigue and anemia. Eculizumab appeared to be well tolerated in these patients, with safety observations that were consistent with those reported from controlled trials in patients with PNH.

"We are encouraged by this initial clinical experience with eculizumab in a very limited number of aHUS and CAD patients. These observations and other knowledge of
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SOURCE Alexion Pharmaceuticals, Inc.
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