An experimental drug originally developed to treat lymphomas is the first-ever to show an effect against a form of multiple sclerosis for which no approved treatments are available, researchers reported at the American Academy of Neurology Annual Meeting in San Diego.
Sanofi's tolebrutinib, an investigational oral Bruton's tyrosine kinase inhibitor, demonstrated a 31% delay in the onset of six-month confirmed disability progression in patients with non-active, non-relapsing secondary progressive multiple sclerosis, in a clinical trial.
"This is the first clinical trial showing a positive effect in delaying disability progression in non-relapsing SPMS, a later form of the disease where neurological function gradually worsens over time and disability increases relentlessly," study leader Dr. Robert Fox of the Cleveland Clinic said in a statement.
With 1,131 patients enrolled in the trial, the rate of confirmed disability progression at six months was 22.6% in the tolebrutinib group versus 30.7% in the placebo group, according to a report of the study published in The New England Journal of Medicine.
More patients receiving tolebrutinib achieved improvement in disability, with a six-month confirmed disability improvement rate of 8.6% versus 4.5% with placebo, the researchers also reported.
Markers of disease activity, including inflammation and tissue damage, also were reduced with tolebrutinib compared with placebo.
Serious adverse events, particularly liver complications, were more frequent with tolebrutinib, which is currently under review for potential U.S. approval.
"It appears that about one in 200 patients will have severe elevation of liver enzymes during the first three months of use, so careful monitoring is important, and the drug should be stopped immediately in those with liver enzyme elevations," Fox said.
Separately, in two studies of patients with relapsing multiple sclerosis, tolebrutinib was not superior to Sanofi's Aubagio (teriflunomide) in decreasing annualized relapse rates, according to a second report in same journal.
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