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news.Alexion has completed treatment of the final patient in a pivotal phase III efficacy trial of its investigational compound eculizumab in patients with the chronic orphan blood disorder paroxysmal nocturnal hemoglobinuria.
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Paroxysmal nocturnal hemoglobinuria (PNS) is a rare form of hemolytic anemia characterized by destruction of red blood cells by the immune system.
In accordance with the trial (TRIUMPH)’s design, 85 eligible patients were randomized to receive either eculizumab or placebo in a six-month treatment phase.
Eculizumab is a monoclonal antibody drug that selectively blocks terminal complement, a part of the immune system that is implicated in PNH red blood cell destruction. There currently is no drug specifically available for treatment of PNH, in which patients’ blood cells are deficient in natural inhibitors of terminal complement.
“We are encouraged that every eligible TRIUMPH patient to date has opted to enroll in an open-label extension trial to receive eculizumab,” said Dr Leonard Bell, CEO of Alexion.
The company anticipates that top line clinical results will be available later in the first quarter of 2006.