Alexion Pharma: Soliris Inhibits TMA, Improves Renal Function In AHUS Patients

(RTTNews) - Alexion Pharmaceuticals, Inc. (ALXN) reported that researchers presented data from four trials, all demonstrating Soliris' clinical benefits for the treatment of atypical hemolytic uremic syndrome or aHUS, a genetic, chronic, ultra-rare disease associated with vital organ failure and premature death. Soliris is the first and only approved safe and effective treatment for pediatric and adult patients with aHUS. In two large, prospective, multinational studies, Soliris inhibited systemic complement-mediated thrombotic microangiopathy or TMA and improved renal function in both pediatric and adult patients with aHUS. The data were presented at Kidney Week 2013, the annual meeting of the American Society of Nephrology in Atlanta.

Also, the ASN meeting featured the presentation of three-year update data from two pivotal Phase 2 extension studies that highlighted the long-term benefits of Soliris therapy in patients with aHUS.

aHUS is an ultra-rare, life-threatening, chronic genetic disease that can progressively damage vital organs, leading to stroke, heart attack, kidney failure, and death.

The study met its primary endpoint, with 30 of 41 patients (73%) achieving a complete TMA response at 26 weeks. Forty of 41 patients achieved platelet count normalization by week 26, and the mean increase in platelet count from baseline was 135x109/L, demonstrating inhibition of TMA.2

Soliris significantly improved renal function with a mean increase in eGFR from baseline of 29 mL/min/1.73m2. Importantly, out of the 24 patients who were on dialysis at baseline, 20 patients discontinued dialysis by week 26.2

"This is the largest study in aHUS and the results confirm those from previous prospective trials, in which ongoing Soliris treatment led to sustained inhibition of complement-mediated TMA, rapid and sustained improvements in hematological parameters, and continued, on-going improvement in renal function in adult patients with aHUS,"said Fadi Fakhouri, M.D., Ph.D., Centre Hospitalier Universitaire de Nantes, Nantes, France.