Biogen’s TYSABRI for Secondary Progressive MS Fails to Meet Phase 3 Trial Endpoints

Biogen Inc. recently reported that the Phase 3 ASCEND clinical trial study testing TYSABRI efficacy in patients with secondary progressive multiple sclerosis (SPMS) did not achieve its primary and secondary goals. According to the company, the comprehensive results of the study will be revealed at a future medical conference.

TYSABRI (natalizumab) is an effective monoclonal antibody therapy indicated for the treatment of patients with relapsing variants of multiple sclerosis (MS), including relapsing-remitting multiple sclerosis (RRMS). It exerts its therapeutic action by disrupting key molecule interactions, leading to a blockage of leukocyte migration to inflamed tissues. Although it is thought to stop the activity of inflammatory cells, TYSABRI’s mode of action has not been completely elucidated.

The drug has been approved in over 65 countries, and in the U.S. is recommended as a mono therapy for relapsing MS. In Europe, this medicine is recommended for patients suffering from high activity or rapidly evolving RRMS, even when subjected to other therapies such as beta interferon. TYSABRI, in previous studies, has been shown to be effective in slowing disease progression and reducing the relapse rates, and in the ASCEND study, it was generally well-tolerated and adverse events were consistent with its known safety profile, Biogen reported.

SPMS relates to severe events in multiple sclerosis, such as progressive nerve damage and loss, and it has very limited and poorly effective treatment options.

“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 Dr. Alfred Sandrock, MD, group senior vice president and chief medical officer at Biogen, in a press release. 

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The placebo-controlled and randomized ASCEND study included 889 SPMS patients in 15 countries, distributed randomly into two treatment groups: placebo or 300 mg of natalizumab. Its primary endpoint was the percentage of patients with confirmed progression of disability in one or more of three disability measurements. Results showed that the drug had a statistically significant improvement effect in upper limb function, one of the three disability points under evaluation. Further analysis of the patient’s response suggested that, in fact, only some SPMS patients benefited from TYSABRI treatment, with less relapses and MRI lesions (secondary endpoints).

“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,” Dr. Sandrock said.