WASHINGTON (dpa-AFX) - Alexion Pharmaceuticals Inc. (ALXN) Saturday announced the presentation of final data from two Phase-2 studies of Soliris as a treatment for patients with atypical hemolytic uremic syndrome, or aHUS. Data showed that the studies met their primary endpoints with high levels of statistical and clinical significance.
Separately, researchers presented for the first time findings from a retrospective clinical study of eculizumab in pediatric patients with aHUS.
Findings from these studies were presented at the 16th Congress of the European Hematology Association in London.
aHUS is a chronic, ultra-rare disease characterized by thrombotic microangiopathy, the formation of blood clots in small blood vessels throughout the body, causing a reduction in platelet count and life-threatening damage to the kidney, brain, and other vital organs.
Alexion's Soliris (eculizumab) is a first-in-class terminal complement inhibitor, and is approved in the U.S., European Union, Japan and other territories as the first treatment for patients with PNH - a debilitating, ultra-rare blood disorder.
Alexion said it has filed marketing applications with the U.S. Food and Drug Administration and the European Medicines Agency for eculizumab as a treatment for patients with aHUS.
Elaborating on the phase-2 data, the company said researchers today presented the findings of eculizumab in 17 adult and adolescent patients who were resistant or intolerant to plasma exchange/infusion and received eculizumab. Fifteen patients received eculizumab therapy for 26 weeks.
Data showed that the study met its primary endpoint and key secondary endpoints with high levels of statistical and clinical significance. For the primary endpoint, platelet count increased from baseline through week 26 by a point estimate 73×109/L.
Researchers also presented final data from a Phase 2 study of Soliris in aHUS patients who were receiving chronic plasma exchange/infusion. In the 26-week study, 20 patients continued to receive plasma exchange/infusion during an 8-week observation period, and then discontinued plasma exchange/infusion and commenced eculizumab treatment. In the study, 80% of patients achieved TMA event-free status, the primary endpoint, defined as at least 12 consecutive weeks of stable platelet count, absence of plasma exchange/infusion, and no new dialysis. The most frequently reported adverse events were diarrhea, headache, hypertension and nausea (generally mild to moderate in severity.
Separately, researchers presented for the first time, findings from a retrospective clinical study of eculizumab in pediatric patients with aHUS. The analysis showed that eculizumab treatment significantly reduced TMA, as platelet counts were normalized in 93% and 73% of patients achieved TMA-event free status. Importantly, 53% of patients had improved renal function with eculizumab treatment.
Further, four of six patients with dialysis in the 30 days prior to eculizumab did not require dialysis during eculizumab treatment. The most frequently reported adverse events were fever, upper respiratory tract infection, diarrhea and cough.
Additionally, Alexion said it is currently enrolling patients in a Phase 2, open-label, single-arm, multi-center study of Soliris in pediatric patients with aHUS in the United States, European Union and Canada.
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