Alexion Commences Dosing in AEGIS Registration Study of Soliris(R) (eculizumab) in Patients with PN...( CHESHIRE Conn. Jan. 7 /- AlexionP...),Health

CHESHIRE, Conn., Jan. 7 /PRNewswire-FirstCall/ -- Alexion Pharmaceuticals, Inc. (the "Company," Nasdaq: ALXN) today announced that it has commenced dosing in the AEGIS study, a single registration study to evaluate the safety, efficacy, and pharmacology of Soliris(R) (eculizumab) as a treatment for Japanese patients with paroxysmal nocturnal hemoglobinuria (PNH). When fully enrolled, the open-label study, authorized by Japan's Pharmaceutical and Medical Device Administration (PMDA), is expected to include approximately 25 patients with PNH at clinical sites throughout Japan. Each PNH patient in the AEGIS study will be dosed with Soliris for 12 weeks after enrollment.

PNH is a rare acquired genetic blood disorder defined by hemolysis, in which patients' red blood cells are destroyed by complement, a component of the body's immune system. The primary efficacy endpoint of the study is reduction of hemolysis from baseline. The study will also measure the effect of Soliris on other clinical manifestations of PNH, including blood transfusion requirements, thromboses (blood clots), and kidney function. Patients' overall quality of life, including fatigue, will also be assessed.

The inclusion and exclusion criteria for the AEGIS study are similar to those used in one of Alexion's previous Phase 3 studies of Soliris as a treatment for PNH, the SHEPHERD study. SHEPHERD examined the safety and efficacy of eculizumab in a broad and diverse population of patients with PNH, including enrollment of patients with minimal transfusion requirements and/or evidence of thrombocytopenia. (1) Based on the SHEPHERD study and the TRIUMPH Phase 3 study, (2) Soliris was approved as the first treatment for patients with PNH by the U.S. Food and Drug Administration in March 2007 and by the European Commission in June 2007.

"Commencing patient dosing with eculizumab in the Japanese registration study represents an important step toward providing patients with PNH in J
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