On 28 February, researchers have published results of the Phase III clinical trial of masitinib in mild-to-moderate Alzheimer’s disease (AD) in the journal Alzheimer's Research & Therapy. Masitinib is an oral drug classed as a tyrosine kinase inhibitor that targets important cells for immunity. The 24-week trial was a multicenter, double-blind, randomised and placebo-controlled study evaluating the efficacy and safety of masitinib as an add-on therapy to cholinesterase inhibitors and/or memantine in people with mild-to-moderate dementia due to probable AD. A total of 718 participants from 119 hospital clinics and specialised AD centers in 20 countries were randomised to receive either masitinib or placebo. Results demonstrated that masitinib administered at 4.5 mg/kg/day significantly slowed cognitive deterioration relative to placebo in the primary endpoint of ADAS-Cog, an instrument that measures the effect on cognition and memory.
The safety of masitinib was acceptable and consistent with the known tolerability profile for masitinib. The company AB Science has initiated a confirmatory Phase III study (AB21004). This study will enrol 600 people with confirmed clinical diagnosis of mild and moderate AD. The primary objective of the study will be to evaluate the effect of masitinib 4.5 mg/kg/day on absolute change from baseline in ADCS-ADL score and in ADAS-Cog-11. The Phase III trial will recruit participants in several EU countries who will receive either placebo or masitinib as an add-on therapy to standard of care (cholinesterase inhibitors and/or memantine). The article, titled ‘Masitinib for mild-to-moderate Alzheimer’s disease: results from a randomized, placebo-controlled, phase 3, clinical trial’ is freely accessible online from the journal website: https://alzres.biomedcentral.com/articles/10.1186/s13195-023-01169-x