Sanofi’s Tolebrutinib Shows Mixed Results in Multiple Sclerosis Trials

Mixed Trial Results:
Sanofi's investigational BTK inhibitor, tolebrutinib, missed primary efficacy endpoints in two Phase III trials (GEMINI 1 and GEMINI 2) for relapsing multiple sclerosis (RMS) but succeeded in a third trial (HERCULES) for non-relapsing secondary progressive multiple sclerosis (nrSPMS).

HERCULES Trial Success:
The HERCULES study showed that tolebrutinib significantly delayed disability progression in patients with nrSPMS, a population with significant unmet medical needs.

Liver Safety Concerns:
Despite previous liver safety concerns that led to a partial clinical hold in 2022, preliminary analysis indicated that tolebrutinib's liver safety profile was consistent with previous studies.

Future Plans:
Sanofi plans to file for approval for tolebrutinib in nrSPMS "as soon as possible" and will present detailed findings from the trials at the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) later in September 2024.

Industry Context:
Other pharmaceutical companies like Merck KGaA and Roche have also faced challenges with their BTK inhibitors due to liver safety issues and efficacy concerns.