New Medical Therapies™

Renal Failure

November 1, 2010

Alexion issued positive interim results from two phase II trials of eculizumab for the treatment of atypical Hemolytic Uremic Syndrome. The first open label trial enrolled 17 adolescent and adult subjects who were resistant to plasma therapy. The subjects received eculizumab for up to 26 weeks. The primary endpoint of the study is the change in platelet count, a measure of thrombotic microangiopathy (TMA). Data showed a significant increase in platelet count with eculizumab treatment compared to baseline (p<0.0001). Key secondary clinical endpoints were also positive. The second open label trial enrolled 20 adolescent and adult subjects who were receiving plasma therapy chronically prior to entering the study. The primary endpoint was TMA Event-Free Status, as defined by stable platelet counts, absence of plasma therapy and no new dialysis. In this interim analysis of 15 subjects treated with eculizumab for at least 12 weeks, a significant 87% achieved the primary endpoint. Key secondary clinical endpoints were also positive. Eculizumab was well-tolerated, with the most common adverse events including anemia, diarrhea, headache, nausea and hypertension.