Sanofi has shared positive results from a late-stage study of its investigational BTK inhibitor, tolebrutinib, in patients with non-relapsing secondary progressive multiple sclerosis (nrSPMS).
The phase 3 HERCULES trial evaluated an oral dose of the candidate in MS patients who had stopped experiencing confirmed relapses but continued to experience accumulation of disability.
Results presented at this year’s European Committee for Treatment and Research in MS (ECTRIMS) conference showed that tolebrutinib delayed the time to onset of six-month confirmed disability progression by 31% compared to placebo.
Sanofi also reported that the number of patients who experienced confirmed disability improvement increased in the tolebrutinib arm, at 10% versus 5% in the placebo cohort.
The company outlined that the results will “form the basis” for future discussions with global regulatory authorities, with submissions starting in the second half of 2024.
Affecting approximately 2.9 million people worldwide, multiple sclerosis (MS) occurs when the immune system attacks the protective myelin sheath that covers the nerves and disrupts communication between the brain and the rest of the body.
Relapsing MS, characterised by attacks of worsening neurologic function followed by partial or complete recovery periods, accounts for about 85% of initial diagnoses, while nrSPMS is less common.
Tolebrutinib is being assessed in various forms of the disease, including primary progressive MS.
Data from the phase 3 GEMINI 1 and 2 trials of the drug in relapsing MS was also shared at ECTRIMS.
The studies did not meet their shared primary endpoint of reducing annualised relapse rate compared to Sanofi’s current oral MS therapy Aubagio (teriflunomide), but a pooled analysis showed that tolebrutinib delayed the time to onset of six-month confirmed disability worsening by 29%.
Houman Ashrafian, head of research and development at Sanofi, said: “With no treatment options currently available for the broad population of patients with secondary progressive MS, tolebrutinib has demonstrated its ability to delay disability by targeting underlying drivers of the disease.
“We look forward to discussing these results with healthcare authorities and are eager to see the results of tolebrutinib in primary progressive MS when they become available next year.”