Sanofi shares promising late-stage results for BTK inhibitor in progressive MS

Sanofi has announced encouraging results from a late-stage clinical trial of its investigational BTK inhibitor, tolebrutinib, in patients with non-relapsing secondary progressive multiple sclerosis (nrSPMS). The phase 3 HERCULES trial examined an oral dosage of the drug in MS patients who no longer experienced confirmed relapses but continued to face accumulating disability.

Sanofi presented the findings at the European Committee for Treatment and Research in MS (ECTRIMS) conference, revealing that tolebrutinib delayed the onset of six-month confirmed disability progression by 31% compared to a placebo. Additionally, the number of patients showing confirmed disability improvement doubled in the tolebrutinib group, reaching 10% compared to 5% in the placebo cohort.

The company emphasized that these results will serve as a foundation for future discussions with global regulatory authorities, with submission processes expected to begin in the latter half of 2024.

Multiple sclerosis (MS) affects roughly 2.9 million people globally and is characterized by the immune system attacking the protective myelin sheath covering nerve fibers, thereby disrupting communication between the brain and other parts of the body. Relapsing MS, which involves periods of worsening neurologic function followed by partial or complete recovery, accounts for about 85% of initial MS diagnoses. NrSPMS, however, is less common.

Tolebrutinib is also being tested in various other forms of MS, including primary progressive MS. Data from the phase 3 GEMINI 1 and 2 trials investigating the drug in relapsing MS were also presented at ECTRIMS. Although these studies did not meet their primary goal of reducing the annualized relapse rate compared to Sanofi's existing oral MS treatment, Aubagio (teriflunomide), a pooled analysis indicated that tolebrutinib delayed the time to onset of six-month confirmed disability worsening by 29%.

Houman Ashrafian, head of research and development at Sanofi, stated, "With no current treatment options available for the broad population of patients with secondary progressive MS, tolebrutinib has shown its potential to delay disability by targeting the underlying drivers of the disease. We look forward to discussing these findings with healthcare authorities and are eager to see the results of tolebrutinib in primary progressive MS when they become available next year."