Alexion Pharmaceuticals, Inc. today announced positive topline results from the Phase 3 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.
"These results far exceeded our expectations. The remarkable reduction in relapse risk demonstrates the unique ability of Soliris® to inhibit complement, and suggests a promising new treatment for NMOSD," said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. "Given that patients currently have no approved therapies, we are moving quickly to discuss these results with regulators and file for approval in the U.S., EU, and Japan."
"The primary goal in treating NMOSD is relapse prevention as each relapse further increases disability, which makes this disease so devastating. For decades, we have been hoping for a therapy that can prevent relapse and subsequent accumulation of disability by addressing a critical underlying cause of the disease," said Michael Levy, M.D., Ph.D., Associate Professor at Johns Hopkins University, and Director of the Neuromyelitis Optica Clinic in Baltimore, MD. "The substantial effect of Soliris® seen in this groundbreaking randomized, controlled study in NMOSD could potentially become a turning point for patients and their families who live in constant fear of relapse.
A new era of targeted therapy with antibody–drug conjugates