Sanofi's experimental BTK inhibitor SAR442168 hits main goal of mid-stage MS study

Sanofi announced Thursday that a Phase IIb study of the experimental BTK inhibitor SAR442168 in patients with recurring multiple sclerosis (MS) met its primary endpoint. Results of the trial showed that the oral, brain-penetrant, selective small molecule significantly reduced disease activity associated with MS as measured by magnetic resonance imaging (MRI).

"We believe our BTK inhibitor has the potential to transform how MS is treated," remarked John Reed, head of global R&D, adding "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."

In the randomised trial, 60 patients with recurring MS received one of four doses of SAR442168 for the first 12 weeks, then crossed over to placebo for four weeks. Meanwhile, another 60 patients with recurring MS received four weeks of placebo before crossing over to SAR442168.

According to Sanofi, SAR442168 demonstrated a dose-response relationship in the reduction of new active gadolinium (Gd)-enhancing T1-hyperintense brain lesions after 12 weeks of treatment. The company noted that safety findings were consistent with the previously reported Phase I study, while detailed results from the Phase IIb trial, including advanced imaging endpoints, will be presented at an upcoming medical meeting.

Sanofi indicated that four Phase III studies of SAR442168, in both relapsing and progressive forms of MS, are planned to be initiated in the middle of this year. Sanofi obtained global rights to develop and commercialise the drug, previously known as PRN2246, under an agreement with Principia Biopharma potentially worth more than $800 million.


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