Positive Phase III results for Soliris to treat neuromyelitis optica spectrum disorder. Alexion Pharma

Alexion Pharmaceuticals, Inc.announced positive topline results from the Phase III PREVENT study of Soliris (eculizumab) in patients with anti-aquaporin-4 (AQP4) auto antibody-positive neuromyelitis optica spectrum disorder (NMOSD).

NMOSD is a rare, devastating, complement-mediated disorder of the central nervous system characterized by relapses. Each relapse results in stepwise accumulation of disability, including blindness and paralysis, and sometimes premature death. Patients who have anti-AQP4 auto-antibodies represent approximately three quarters of all patients with NMOSD. There are currently no approved therapies for this disease.

The study met its primary endpoint of time to first adjudicated on-trial relapse, demonstrating that treatment with Soliris reduced the risk of NMOSD relapse by 94.2 percent compared to placebo (p < 0.0001). At 48 weeks, 97.9 percent of patients receiving Soliris were free of relapse compared to 63.2 percent of patients receiving placebo. Soliris was generally well tolerated with a safety profile consistent with that seen in previous clinical studies and real-world use in its three approved indications. No cases of meningococcal infection were observed.