Tolebrutinib treatment associated with 31 percent risk reduction in six-month confirmed disability progression versus placebo
By Elana Gotkine HealthDay Reporter
WEDNESDAY, April 9, 2025 (HealthDay News) — Tolebrutinib, a brain-penetrant and bioactive Bruton tyrosine kinase inhibitor that modulates persistent immune activation within the central nervous system, slows disability progression in adults with nonrelapsing secondary progressive multiple sclerosis (MS), according to a study presented at the annual meeting of the American Academy of Neurology, held from April 5 to 9 in San Diego.
Robert J. Fox, M.D., from the Mellen Center for Multiple Sclerosis at the Cleveland Clinic, and colleagues conducted a phase 3, double-blind, event-driven trial involving 1,131 adults (age range, 18 to 60 years) with secondary progressive MS, Expanded Disability Status Scale scores of 3.0 to 6.5, documented evidence of disability progression during the previous 12 months, and no clinical relapses during the 24 months before screening. Participants were randomly assigned to receive tolebrutinib or placebo (754 and 377 individuals, respectively).
The researchers found that compared with placebo, tolebrutinib treatment was associated with a 31 percent risk reduction in six-month confirmed disability progression. Six-month confirmed disability improvement was experienced by more participants with tolebrutinib than placebo (a secondary end point). A slight increase in some adverse events was seen with tolebrutinib versus placebo, including respiratory infections. Rare events of high liver enzyme increases were seen with tolebrutinib, which occurred within 90 days of starting treatment.
“The trial found that treatment with tolebrutinib slows progression of disability in nonrelapsing secondary progressive MS, which is really exciting because this is the very first time we’ve identified a treatment that is effective for this form of MS,” Fox told HealthDay News. “Now there are safety concerns, particularly potential liver injury, and that will require careful counseling of patients who may consider this therapy and close monitoring. But finally, I think a bright day has come for … secondary progressive MS because we have finally found a treatment that may be helpful.”
Several authors disclosed ties to Sanofi, which manufactures tolebrutinib and funded the study.
Copyright © 2025 HealthDay. All rights reserved.
