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Long-Term Efficacy, Safety Outcomes Data for PNH Patients Treated with Alexion's Soliris

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Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) today announced the presentation of new data demonstrating the long-term benefits of Soliris® (eculizumab) in patients with paroxysmal nocturnal hemoglobinuria (PNH), a debilitating, ultra-rare and life-threatening blood disorder characterized by complement-mediated hemolysis (destruction of red blood cells). Data including the clinical benefits of Soliris in the treatment of patients with PNH and atypical hemolytic uremic syndrome (aHUS) were presented at the 54th Annual Meeting of the American Society of Hematology (ASH) in Atlanta:

Ten-year data from a large cohort of patients with PNH from the United Kingdom (UK) confirm the long-term safety and efficacy of chronic Soliris therapy and demonstrate the impact of Soliris on quality of life.1 The long-term safety of Soliris was also demonstrated in PNH clinical development trials in which patients were treated continuously with Soliris and followed for up to 5.5 years.2 Data from an international PNH registry showed that Soliris significantly reduced the risk of thromboembolism (TE) in patients with PNH.3 Researchers from South Korea presented data confirming that hemolysis is an independent risk factor for TE in patients with PNH.4 Researchers in Japan observed a rare genetic polymorphism in the terminal complement protein C5 in Japanese patients with PNH who had no or minimal reduction in lactate dehydrogenase (LDH) while on Soliris therapy (9 out of 250 treated, or 3.6%).5 The ASH meeting also featured presentations of two-year data highlighting the long-term benefits of chronic Soliris therapy in patients with aHUS,6,7 an ultra-rare, genetic disease characterized by complement-mediated thrombotic microangiopathy (TMA), the formation of blood clots in small vessels throughout the body. Soliris, a first-in-class terminal complement inhibitor, specifically targets uncontrolled complement activation, and is approved in the United States, European Union, Japan and other countries as the first and only treatment for patients with PNH. Soliris is also approved in the US and EU as the first and only treatment for patients with aHUS.

“Extensive clinical data and real-world clinical experience spanning more than 10 years further establish the benefits that Soliris is providing to patients with PNH,” said Leonard Bell, M.D., Chief Executive Officer of Alexion. “The presentation of the two-year aHUS data also demonstrates the continued benefits of long-term Soliris therapy in patients with aHUS, and underscores the important role of hematologists in diagnosing and treating patients with this severe and ultra-rare complement-mediated disease.”

Ten-Year Experience with Soliris in All Patients with PNH Treated in the UK

In a poster presentation today, researchers presented data from all 153 patients who participated in a nationally commissioned PNH service led by two UK medical centers. The objective of the study was to describe the long-term safety, efficacy, and outcomes in patients with PNH from the UK who received Soliris treatment from May 2002 to April 2012.1

The results demonstrated that the significant clinical benefits and long-term safety of Soliris were sustained over 10 years of chronic treatment. The investigators reported that long-term Soliris treatment led to an improvement in survival when compared with historical controls, and a significant reduction in the incidence of TE.8,9 Researchers also reported data on the effect of discontinuation of anti-coagulant therapy in selected PNH patients. Transfusion independence was observed in the majority of patients, and the number of units transfused was significantly reduced in those patients still receiving transfusions. Results also confirmed the long-term safety and efficacy of continuous Soliris treatment.1

Researchers presented the following data in support of these conclusions:1

Long-term Soliris therapy significantly reduced intravascular hemolysis by 83.4%, as assessed by levels of LDH (p<0.001). The researchers noted that UK patients on long-term Soliris therapy had improved survival compared with previously published historical controls.8,9 Researchers also compared the survival of PNH patients treated with Soliris to a normal population of the same age and gender. Although the survival of PNH patients after 10 years of Soliris therapy was slightly inferior to this normal healthy population group, the causes of death in PNH patients were related to bone marrow failure and not due to hemolysis or TE associated with the underlying PNH. In the 12 months prior to starting Soliris, 36 thrombotic episodes were reported in 22 patients. None of those patients had a further thrombotic episode while on Soliris therapy. In the most recent 12 months on therapy, TEs were significantly reduced with only 3 events reported in a total of 3 patients (p<0.05). Of 117 patients transfused in the 12 months before receiving Soliris, 77 (65.8%) were transfusion-independent in the most recent 12 months on treatment. Among those patients still receiving transfusions (n=40), there was a significant 69% reduction in the number of units transfused, from a median of 26 units, 12 months before therapy, to 8 units in the most recent 12 months on therapy (P<0.05).

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