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news.Alexion Pharmaceuticals, a biopharmaceutical company, has reported positive results from Aegis, an open-label registration study examining Soliris treatment of Japanese patients with paroxysmal nocturnal hemoglobinuria.
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The pre-specified primary efficacy endpoint of change in hemolysis was achieved with an 86% reduction. Key secondary endpoints including fatigue and transfusions/anemia were also achieved.
In the Aegis open-label clinical study, investigators examined the safety and efficacy of Soliris in Japanese patients with paroxysmal nocturnal hemoglobinuria (PNH) relative to the Shepherd and Triumph studies. The primary endpoints of the study were reduction of hemolysis and safety following 12 weeks of Soliris treatment.
The study results showed that hemolysis, the primary trial endpoint, was rapidly and significantly reduced with Soliris treatment. A standard measure of hemolysis, lactate dehydrogenase, decreased 86% from a median of 1,814 U/L at baseline to a median of 244 U/L following 12 weeks of treatment (P<0.001; normal range 103-223 U/L).
Control of hemolysis resulted in an improvement in anemia as indicated by a reduction in packed red blood cell (PRBC) transfusion requirements. Transfusions were reduced 71% from a mean of 5.2 (+/-1.04) PRBC units/patient during the 12-week pre-treatment period to 1.5 (+/-0.67) units/patient following 12 weeks of Soliris treatment (P<0.001 for the pre-specified median change). Transfusion independence was achieved in 67% (14/21) of patients who were transfusion dependent prior to treatment (P<0.001), the company said.
The improvement in anemia with Soliris treatment was also indicated by increased hemoglobin levels. Hemoglobin levels increased from a median of 7.6 g/dL (7.9 +/- 0.3) at baseline to a median of 9.0 g/dL (8.9 +/- 0.4) following 12 weeks of Soliris treatment (P<0.001).
According to the company, patient fatigue levels, as measured by the FACIT-Fatigue instrument, significantly improved within one week of Soliris treatment, with a median increase of five points at 12 weeks. A change of three or more points is considered clinically meaningful. The drug appeared to be safe and well tolerated in study patients.
Leonard Bell, CEO of Alexion, said: The Aegis results show that Soliris treats the hallmarks of PNH in Japanese patients with levels of safety and efficacy consistent with those demonstrated in the US and the European study populations.