Sanofi’s multiple sclerosis drug meets Phase II goal

Results from a Phase IIb clinical trial of Sanofi’s investigational small molecule, SAR442168, have demonstrated an encouraging profile in multiple sclerosis patients.

SAR442168 is an oral, brain-penetrant, selective Bruton’s tyrosine kinase (BTK) inhibitor. It is thought to act by regulating adaptive and innate immune cells associated with neuroinflammation in the brain and spinal cord.

Sanofi gained the rights to develop and commercialise the drug candidate worldwide as part of a licence agreement with Principia Biopharma.

The randomised, placebo-controlled, cross-over, double-blind 12-week dose-ranging Phase IIb trial assessed SAR442168 in people with recurring multiple sclerosis.

A group of participants in the study were treated with one of four SAR442168 doses during the initial 12 weeks and later crossed over to placebo for four weeks.

The remaining arms were given placebo for four weeks and then crossed over to Sanofi’s drug candidate.

Data showed that the trial reached its primary endpoint, where SAR442168 significantly decreased multiple sclerosis disease activity, which was determined using magnetic resonance imaging.

SAR442168 was observed to be well-tolerated without any new safety findings. The drug candidate’s safety profile was consistent with prior Phase I data.

Sanofi will use the drug candidate’s dose-response curve for a decrease in brain MRI lesion activity to select the Phase III dose.

Participants who complete the week 16 visit will be able to enrol in a long-term safety follow-up study.

Sanofi Research and Development global head John Reed said: “This molecule may be the first B-cell-targeted MS therapy that not only inhibits the peripheral immune system but also crosses the blood-brain barrier to suppress immune cells that have migrated into the brain, while also modulating the brain-resident microglia cells that have been implicated in MS progression.

“Building on Sanofi’s heritage in multiple sclerosis, we are encouraged by these clinical results and look forward to rapidly advancing our brain-penetrant BTK inhibitor into pivotal clinical trials.”

The company will launch four Phase III studies in relapsing and progressive multiple sclerosis patients to assess SAR442168’s impact on relapse rates, disability progression and central nervous system damage.

Last month, Sanofi reported positive results from the ASCEND and ASCEND-Peds trials of olipudase alfa.