A phase 3 study evaluating the efficacy and safety of oral, once-daily siponimod (BAF312, Novartis) in patients with secondary progressive multiple sclerosis (SPMS) has met its primary endpoint of a reduction in the risk of disability progression compared with placebo. The study represents the largest randomized, controlled trial in SPMS to date.
Topline results, including primary and key secondary endpoints, will be presented in September at the 32nd Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) in London.
The EXPAND trial was a randomized, double-blind, placebo-controlled study comparing siponimod 2 mg with placebo in 1,651 subjects with SPMS. The study’s endpoint was an improvement in the time to three-month confirmed disability progression, as measured by the Expanded Disability Status Scale (EDSS), compared with placebo. Secondary endpoints included the delay in the time to six-month confirmed disability progression compared with placebo; the time to confirmed worsening of at least 20% from baseline in the timed 25-foot walk test (T25FW); the T2 lesion volume; the annualized relapse rate; and the safety and tolerability of siponimod in subjects with SPMS.
Siponimod is a selective modulator of specific types of the sphingosine-1-phosphate (S1P) receptor. This receptor is commonly found on the surface of specific cells in the central nervous system (CNS) that are responsible for causing CNS damage that drives the loss of function in SPMS. Siponimod enters the brain and, by binding to these specific receptors, may prevent the activation of these harmful cells, helping to reduce the loss of physical and cognitive function associated with SPMS.
Source: Novartis; August 25, 2016.