- Novartis announced positive results from the Phase 3 EXPAND study of once-daily, oral BAF312, also known as siponimod, in patients with secondary-progressive multiple sclerosis. The study included 1,651 patients from 31 countries.
- The topline data showed BAF312 met both its primary and secondary endpoints. The primary endpoint was the reduction in the risk of disability progression compared with placebo.
- Secondary endpoints in the trial included delay in the time to six-month confirmed disability progression, the time to confirmed worsening of at least 20% from baseline in the timed 25-foot walk test, T2 lesion volume and annualized relapse rate.
Novartis expects to present the rest of the trial results as a late breaking oral abstract at the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) on September 17. The big pharma expects to discuss the data with the U.S. Food and Drug Administration to determine next steps for the therapy.
While little was revealed about the results of the EXPAND study, positive results in the hard-to-treat area could be a major turning point for both the disease and MS developers.
Secondary-progressive multiple sclerosis (SPMS) is a form of the disease where patients consistently get worse. Most of the treatments currently on the market treat an earlier form of the disease known as relapsing-remitting multiple sclerosis (RRMS) that is marked by periods of flare up and health. Nearly 80% of these patients are later classified as SPMS when they stop having the periods of health.
Many companies have tried—and failed—to develop treatments for SPMS. Biogen, for one, has made multiple unsuccessful attempts at the market. The big biotech announced in 2015 its MS therapy Tysabri (natalizumab) failed to meet its primary endpoint in a Phase 3 trial in patients with SPMS. More recently, Biogen said it would terminate its late-stage study of its blockbuster Tecfidera for the treatment of SPMS.
BAF312 is meant to be Novartis' follow-up to its oral MS therapy Gilenya (fingolimod), which will likely face generic competition in the next three years.