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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

the mechanisms at work in these disorders lead us to believe that there are strong scientific rationales for undertaking controlled clinical trials to investigate the role of complement inhibition in these two conditions," said Leonard Bell, M.D., Chief Executive Officer of Alexion. "As previously announced, we are working closely and urgently with clinical experts from Europe and North America to design clinical trials of eculizumab for the treatment of aHUS and CAD."

Initial Experience with Eculizumab in aHUS

A poster titled "Successful Treatment of Atypical Hemolytic Uremic Syndrome with the Complement Inhibitor Eculizumab" was presented today at the ASH annual meeting by Dr. Jens Nuernberger of the Department of Nephrology at University Duisburg-Essen in Essen, Germany.

Dr. Nuernberger and his colleagues investigated the potential effect of eculizumab in two patients with aHUS, a rare disease usually caused by a genetic mutation in the complement system. In patients with aHUS, this defect results in hemolysis, formation of tiny blood clots (microvascular thrombosis) and inflammation of blood vessels, often causing acute kidney injury and progressing to end-stage kidney disease. Those who survive aHUS often live with irreversible kidney disease and are dependent upon dialysis to stay alive.

"Our first clinical experience with eculizumab in patients with aHUS is very promising, especially given the devastating nature of this disease and the lack of effective treatments," said Dr. Nuernberger. "Initial data suggest that eculizumab reduced hemolysis, microvascular thrombosis, and blood vessel inflammation, thereby appearing to improve the course of aHUS. If clinical trials confirm these results, eculizumab may then improve our ability to care for these patients."

Initial Experience with Eculizumab in CAD

A poster titled "Long-term Efficacy of the Terminal Comple
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SOURCE Alexion Pharmaceuticals, Inc.
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