The FINANCIAL — The Phase 3 ASCEND study investigating natalizumab in the treatment of secondary progressive multiple sclerosis (SPMS) did not achieve its primary and secondary endpoints, Biogen reported on October 21. During the study, natalizumab was generally well tolerated and adverse events were consistent with its known safety profile.
ASCEND evaluated the efficacy and safety of natalizumab to slow the accumulation of disability progression unrelated to relapse in SPMS patients, an unmet medical need. The majority of study participants had EDSS scores of 6.0 to 6.5 (walking aid required) and were non-relapsing for two years prior to enrollment in the study. The study’s composite primary endpoint evaluated the percentage of patients whose disability had progressed on one or more of three disability measurements comprising the composite endpoint, according to Biogen.
Natalizumab demonstrated a statistically significant effect on upper limb function (one of the three components of the primary composite endpoint) unrelated to relapses. Consistent with the established effects of natalizumab in relapsing multiple sclerosis, analyses of exploratory endpoints suggest that some patients received a benefit from treatment, including reduction of relapses and new MRI lesions.
“While we’re disappointed with these results, we believe this research will provide the MS community important insights into this more advanced patient population, and the benefits that natalizumab may provide in areas such as upper limb function,” said Alfred Sandrock, M.D., Ph.D., group senior vice president and chief medical officer at Biogen. “Given the challenges of treating this advanced stage of MS, these results underscore the importance of treatment early in the course of disease with effective disease-modifying therapies before a patient advances to SPMS.”
SPMS is characterized by ongoing nerve damage or loss and patients experience disability progression with increasingly less frequent relapses. Despite extensive clinical research, treatment options for patients with SPMS are extremely limited and none have demonstrated efficacy in slowing the progression of disability unrelated to relapse.
Natalizumab is a high-efficacy treatment for patients with relapsing forms of MS, including relapsing-remitting MS. The safety and efficacy of natalizumab has been established across a robust clinical program and real-world use with more than a decade of clinical experience demonstrating its benefits on disease progression and sustained efficacy in relapsing MS with a well-characterized safety profile.