Phase III AB09004 study of Masican shows efficacy and safety in mild-to-moderate Alzheimer’s disease.

Study AB09004 was the first successful phase III randomized trial in mild-to-moderate Alzheimer’s disease of a drug targeting innate immune cells of the neuroimmune system. Masitinib at 4.5 mg/kg/day showed significant benefit over placebo according to the primary analysis, with an acceptable safety profile.

Primary efficacy analysis (based on multiple endpoints, each tested at a significance level of 2.5%) was the least-squares mean change from baseline to week-24 in either the 11-item Alzheimer’s Disease Assessment Scale - cognitive subscale (ADAS-cog), or the 13-item Alzheimer’s Disease Cooperative Study Activities of Daily Living Inventory scale (ADCS-ADL). Results showed that masitinib can generate a significant treatment effect relative to placebo in the primary endpoint of change from baseline in ADAS-Cog, an instrument that measures the effect on cognition and memory. Specifically, masitinib 4.5 mg/kg/day (n=182) showed significant benefit relative to placebo (n=176), with a respective change in ADAS-cog from baseline of -1.46 (representing an overall improvement in cognition) versus +0.69 (representing increased cognitive deterioration); a corresponding ADAS-cog between-group difference of -2.15 (97.5%CI [-3.48, -0.81]), p=0.0003.

It was also seen that masitinib generated a benefit in Alzheimer’s Disease Cooperative Study Activities of Daily Living (ADCS-ADL) score, an instrument that assesses self-care and activities of daily living. Specifically, masitinib 4.5 mg/kg/day showed a change in ADCS-ADL from baseline of +1.01 (representing an overall functional improvement) versus -0.81 for placebo (representing increased functional deterioration); a corresponding ADCS-ADL between-group difference of +1.82 (97.5%CI [(-0.15, 3.79]), p=0.038. The safety of masitinib as an adjunct to cholinesterase inhibitor and/or memantine was acceptable and consistent with its known tolerability profile.