Alexion reports positive phase 3 results for eculizumab in patients with rare blood disorder

By E. Janene Geiss

Philadelphia, Jan. 26 - Alexion Pharmaceuticals, Inc. reported Thursday positive results from its pivotal phase 3 placebo-controlled randomized efficacy trial using eculizumab in Paroxysmal Nocturnal Hemoglobinuria patients.

All prespecified primary and secondary endpoints in the international trial were achieved with statistical significance, according to a company news release.

The prespecified, co-primary endpoints were median transfusion rate and hemoglobin stabilization over six months. The median transfusion rate was reduced from 10 units per patient with placebo to no transfusion needed with eculizumab, officials said.

Hemoglobin stabilization was achieved by 49% of eculizumab patients as compared to no patients for placebo, officials said.

The study enrolled 87 patients at 45 sites in the United States, Canada, Europe and Australia.

Paroxysmal Nocturnal Hemoglobinuria, a rare form of hemolytic anemia, is an acquired genetic blood disorder characterized by destruction of red blood cells by the body's complement system, which is a component of the immune system.

Patients with Paroxysmal Nocturnal Hemoglobinuria lack naturally occurring complement inhibitors that prevent red blood cell destruction.

Eculizumab, a long-acting C5 terminal complement inhibitor, is a monoclonal antibody drug that selectively blocks terminal complement activation, officials said.

There currently is no therapy specifically available for treatment of Paroxysmal Nocturnal Hemoglobinuria.

"The ... study represents an important milestone in the development of eculizumab for Paroxysmal Nocturnal Hemoglobinuria and, hopefully, for the patients afflicted with this chronic, severely debilitating and life-threatening disease," said Peter Hillmen, lead investigator and consultant hematologist of the General Infirmary at Leeds in the United Kingdom, in the release.

"Treatment options are extremely limited for Paroxysmal Nocturnal Hemoglobinuria and at present there is no approved therapy. Eculizumab is currently the only potential therapy that addresses hemolysis and the associated morbidities in Paroxysmal Nocturnal Hemoglobinuria," Hillmen added in the release.

All of the prespecified secondary endpoints in the study, including reduction in lactate dehydrogenase, quality of life and transfusion avoidance also were achieved with statistical significance, officials said.

Additionally, eculizumab appeared to be well-tolerated with an adverse event profile comparable to placebo. The most frequent adverse events with eculizumab were headache, nasopharyngitis and back pain, officials said.

Alexion said it previously reached an agreement with the Food and Drug Administration on the design of the study and a companion phase 3 trial under the FDA's Special Protocol Assessment process.

The companion study is an open-label, multi-center clinical trial primarily aimed at generating safety data with eculizumab in a broader population of hemolytic Paroxysmal Nocturnal Hemoglobinuria patients.

Eculizumab has been granted orphan drug status from both the U.S. and European regulatory agencies to treat Paroxysmal Nocturnal Hemoglobinuria.

Alexion, based in Cheshire, Conn., develops therapeutic products aimed at treating patients with severe diseases, including hematologic and cardiovascular disorders, autoimmune diseases and cancer.

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