Soliris(R) Therapy provided clinical improvements in PNH Patients regardless of disease severity

Soliris(R) (eculizumab) therapy reduced hemolysis, fatigue, thromboses (blood clots) and transfusion requirements in patients with a rare blood disorder called paroxysmal nocturnal hemoglobinuria (PNH), including those who might have been expected to have less severe disease, according to data from an ongoing open-label clinical study presented at the 49th Annual Meeting of the American Society of Hematology Meeting in Atlanta.

The data were highlighted in an oral presentation titled, "High Incidence of Progression to Significant Disease Burden in Paroxysmal Nocturnal Hemoglobinuria Patients with Lower Levels of Hemolysis, Mild Anemia and Minimal Transfusion: Clinical Improvement with Eculizumab Therapy."

In the study, Soliris was associated with significant long-term clinical improvements in patients with PNH, regardless of baseline degree of hemolysis, anemia or transfusion requirements. The study also demonstrated that patients who might have been expected to have less severe disease, considering their baseline clinical characteristics, suffered from significant disease burden.

"PNH patients once thought to have less severe disease based on their clinical characteristics actually face significant disease burden from anemia, fatigue, impaired quality of life, blood transfusion requirements and blood clot risk," said Monica Bessler, MD, PhD, lead author of the study and Professor of Medicine, Professor of Pharmacology and Molecular Biology, Washington University in St. Louis School of Medicine. "The results presented today show that, in this study group, regardless of disease severity, long- term Soliris treatment provides important clinical improvements in their disease signs, symptoms and complications."

"We continue to observe that the PNH patient population includes a wide range of patients with a broad clinical profile," said Leonard Bell, MD, Chief Executive Officer of Alexion Pharmaceuticals. "The results presented today provide further evidence for the utility of Soliris therapy in patients with diverse manifestations of PNH. We remain committed to our goal that all patients who can benefit from Soliris will have access to it."

Soliris, developed by Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN), is the first therapy approved for the treatment of patients with PNH, a rare, debilitating and life-threatening blood disorder defined by the destruction of red blood cells, or hemolysis. Soliris is a complement inhibitor indicated for the treatment of patients with PNH to reduce hemolysis. In patients with PNH, hemolysis can cause thromboses, kidney disease, liver dysfunction, disabling fatigue, impaired quality of life, recurrent pain, shortness of breath, pulmonary hypertension, intermittent episodes of dark colored urine (hemoglobinuria), and anemia.

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