Masitinib, an orally administered tyrosine kinase inhibitor targeting the innate immune system, showed positive signs in treating progressive forms of multiple sclerosis (MS) in the phase III AB07002 trial.
The drug, which specifically downregulates mast cells and microglia, appeared to slow disability progression in people with primary progressive multiple sclerosis (PPMS) and non-active secondary progressive MS (SPMS) over 2 years, reported Patrick Vermersch, PhD, of University of Lille in France, at MS Virtual 2020, the joint ACTRIMS-ECTRIMS meeting.
Point estimates consistently supported efficacy, but not all were significant.
This is the first time that a drug targeting innate immune cells — as opposed to targeting adaptive immune cells like B cells and T cells — has shown positive results in PPMS and non-active SPMS, Vermersch noted. “Despite some atypical endpoints evaluation, the data showed significant difference versus placebo for disability progression using EDSS,” he said.
“Over time, we have accumulated data telling us that to control the progressive forms, we need to control innate immunity,” Vermersch told MedPage Today. “The data obtained with other products in progressive forms — siponimod [Mayzent] in SPMS and ocrelizumab [Ocrevus] in PPMS — showed significant but modest results, and the positive results were driven by patients with baseline characteristics of clinical and MRI activity,” he said.
“Masitinib may provide a new option for physicians with progressive patients,” he added. “We have no therapeutic option for patients without superimposed clinical or MRI activity and it is an urgent need.”
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