New Long-Term Data on Tolebrutinib for Relapsing Multiple Sclerosis Show Favorable Safety Outcomes

Results from research presented Monday, April 24 at the American Academy of Neurology (AAN) 2023 Annual Meeting revealed that tolebrutinib (SAR442168; Sanofi, Paris, France) administered as treatment for relapsing multiple sclerosis (MS) was not associated with significant treatment-emergent adverse events (TEAEs). These results were obtained from a long-term extension (LTE) study (NCT03996291) associated with a phase 2b dose-finding study for tolebrutinib in relapsing MS (NCT03889639).

Individuals who had completed the dose-finding study were eligible for the LTE study. In all, 124 participants completed part A of the LTE which took place from weeks 0 to 24 weeks and involved administration of 1 of 4 doses (5, 15, 30, or 60 mg/d) before transitioning to part B which took place from weeks 24 to 120 during which 60 mg/d was administered. According to primary outcome results, no serious adverse events or dose effects were reported in part A, and no safety signals were seen in part B. The most common TEAEs were COVID-19, headache, nasopharyngitis, upper respiratory infection, bacterial cystitis, arthralgia, back pain, and pharyngitis. Data from secondary outcomes reported from the phase 2b study revealed favorable annualized relapse rate (ARR) and stable mean Expanded Disability Status Scale (EDSS) scores.  

Tolebrutinib is a Bruton tyrosine kinase (BTK) inhibitor being investigated for use as treatment for relapsing multiple sclerosis (MS). BTK inhibitors inhibit the BTK enzyme which plays a critical role in the B-cell receptor signaling pathway.