MS Drug Fails to Meet Primary Endpoint in Phase 3 Study

Some patients with MS received a benefit from natalizumab involving the reduction of relapses and new MRI lesions.

Some patients with MS received a benefit from natalizumab involving the reduction of relapses and new MRI lesions.

A phase 3 investigational study that evaluated natalizumab in the treatment of secondary progressive multiple sclerosis (SPMS) yielded negative results for the medication. The drug did not meet the primary or secondary endpoints of the study, according to Biogen.

During the study, natalizumab was well-tolerated and adverse events were consistent with its known safety profile.

The study, ASCEND, analyzed safety and efficacy in natalizumab to slow the accumulation of disability progression unrelated to relapse in SPMS patients. The majority of participants required a walking aid and were non-relapsing for 2 years prior to enrollment in the study.

The primary endpoint of the study was to evaluate the percentage of patients whose disability progressed on 1 or more of 3 disability measurements comprising the composite endpoint. The treatment demonstrated a significant effect on upper limb functionality unrelated to relapses.

Analyses of exploratory endpoints suggested that some patients received a benefit from treatment involving the reduction of relapses and new MRI lesions, which was found to be consistent with the established effects of natalizumab in the condition.

“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, MD, PhD, 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 significant research into the area, treatment options for patients with SPMS remain limited and none have demonstrated efficacy in slowing the progression of the disease.

Natalizumab is a highly effective treatment option for patients with relapsing forms of MS, including relapsing-remitting MS.

The safety and efficacy of natalizumab has been established across studies and real-world use with more than 10 years of clinical experience, demonstrating benefits on disease progression and sustained efficacy in relapsing MS with a well-characterized safety profile.