Positive Phase III results for ALXN 1210 in patients with paroxysmal nocturnal hemoglobinuria (PNH) to be presented at EHA meeting.- Alexion Pharma.

Alexion Pharmaceuticals, Inc. has announced that positive results from one of the two large Phase III studies of ALXN 1210, the Company�s investigational long-acting C5 complement inhibitor, in patients with paroxysmal nocturnal hemoglobinuria (PNH) were selected for presentation during the Late-Breaking Oral Session (at 11:45 a.m. CET ) on Sunday, June 17, 2018 at the Annual Conference of the European Hematology Association (EHA) in Stockholm, Sweden .

As previously announced, weight-optimized treatment every eight weeks with ALXN 1210 demonstrated non-inferiority to treatment every two weeks with Soliris (eculizumab) in complement inhibitor treatment-na�ve patients with PNH on the two co-primary endpoints and all four key secondary endpoints. The numeric results for all these endpoints, including breakthrough hemolysis (one of the key secondary endpoints), favored ALXN 1210 and are consistent with the immediate and complete C5 inhibition observed by the end of the first infusion of ALXN 1210 and sustained throughout the entire 26-week treatment period. The safety profile of ALXN 1210, based on almost 180 patient years of experience, was similar to that of Soliris.

Comment: In Phase III clinical studies in complement inhibitor-na�ve patients with PNH, and patients with PNH who had been stable on Soliris, intravenous treatment with ALXN 1210 every eight weeks demonstrated non-inferiority to intravenous treatment with Soliris every two weeks, with numeric results for all primary and key secondary endpoints favoring ALXN 1210. ALXN 1210 is also currently being evaluated in a Phase III clinical study in complement inhibitor-na�ve patients with aHUS, administered intravenously every eight weeks. In addition, Alexion plans to initiate a Phase III clinical study of ALXN 1210 delivered subcutaneously once per week as a potential treatment for patients with PNH and aHUS. Alexion also plans to initiate the development of ALXN 1210 as a potential treatment for patients with generalized MG (gMG) and patients with immunoglobulin A nephropathy .