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New Phase 3 data for Soliris
BOSTON & PHILADELPHIA—Alexion Pharmaceuticals, Inc. has announced that the New England Journal of Medicine (NEJM) published positive data from the Phase 3 PREVENT study of Soliris (eculizumab), a first-in-class complement inhibitor, in adult patients with anti-aquaporin-4 (AQP4) auto antibody-positive neuromyelitis optica spectrum disorder (NMOSD). Today’s NEJM online publication coincides with the American Academy of Neurology (AAN) Annual Meeting, May 4-10, 2019 in Philadelphia, where the data will be presented in the Emerging Science session on May 7.
NMOSD is a rare autoimmune, inflammatory disorder of the central nervous system characterized by sudden and unpredictable relapses, also known as attacks. Each relapse results in stepwise accumulation of disability — including blindness, paralysis and sometimes premature death. Uncontrolled complement activation triggered by anti-AQP4 auto antibodies is a major underlying mechanism of the disease. There is currently no approved therapy for patients with NMOSD.
“Patients with NMOSD live in constant fear of an attack or relapse. In this devastating disease, where each relapse results in further disability, preventing relapses is the primary goal of treatment,” said Sean Pittock, M.D., principal investigator of the PREVENT study, lead author of the NEJM article and director of Mayo Clinic’s Center for Multiple Sclerosis and Autoimmune Neurology and Mayo’s Neuroimmunology Laboratory in Rochester, Minnesota. “The results from the PREVENT study, the first placebo-controlled Phase 3 study in NMOSD, are groundbreaking and demonstrate that the vast majority of patients receiving eculizumab did not experience a relapse.”
As previously announced Soliris data stated, 97.9 percent of patients receiving Soliris were relapse-free at 48 weeks, compared with 63.2 percent of patients receiving placebo. The new data published in NEJM to be presented at the AAN meeting confirm that the significant relapse reduction observed in the PREVENT study was sustained through three years of treatment. All patients receiving Soliris monotherapy, and the vast majority of patients receiving Soliris in addition to immunosuppressive therapy (IST) were relapse-free. The safety profile of Soliris was consistent with that seen in other clinical studies.
“The substantial reduction in relapse risk sustained through three years of Soliris treatment was consistent across all patients, regardless of their baseline immunosuppressive therapy use. These results provide hope for a promising new way of treating patients with NMOSD, who currently have no approved treatment options,” added John Orloff, M.D., executive vice president and head of research & development at Alexion.
The most common adverse events observed in the PREVENT study were upper respiratory tract infection (29% of patients in the Soliris group vs. 13% in the placebo group), headache (23 vs. 23%), nasopharyngitis (21 vs. 19%) and nausea (17 vs. 26%). The serious adverse events that were reported for more than one patient in either group were pneumonia (three patients in the Soliris group vs. one patient in the placebo group), cellulitis, sepsis and urinary tract infection (two patients for each event in the Soliris group vs. zero patients in the placebo group). One patient receiving Soliris and concomitant supportive IST died from infectious pleural effusion. The patient had an extensive history of pulmonary disease and was an active smoker. No cases of meningococcal infection were observed in the study.
The U.S. Food and Drug Administration (FDA), the European Medicines Agency and the Japanese Pharmaceuticals and Medical Devices Agency are reviewing Alexion’s applications for approval of Soliris as a treatment for patients with NMOSD who are anti-AQP4 antibody-positive. The FDA has granted priority review and set a Prescription Drug User Fee Act action date of June 28, 2019.