Positive results from first human masitinib phase 2 study in Alzheimer's disease published in peer-reviewed journal

Apr 20 2011

AB Science SA (Paris:AB)(NYSE Euronext - FR0010557264 - AB), a pharmaceutical company specializing in the research, development and commercialization of protein kinase inhibitors (PKIs), announces the publication of results from the first human phase 2 study of masitinib carried-out in the treatment of Alzheimer's disease. Entitled, 'Masitinib as an adjunct therapy for mild-to-moderate Alzheimer's disease: a randomised, placebo-controlled phase 2 trial', this article is freely accessible online from BioMed Central's peer-reviewed journal Alzheimer's Research and Therapy (http://alzres.com/content/3/2/16).

• Phase 2 study establishes proof-of-concept that oral masitinib has potential therapeutic benefits in patients with mild-to-moderate Alzheimer's disease

• Overall, results add new scientific data to the important question of the potential role of anti inflammatory agents in the management of Alzheimer's disease

• AB Science is actively preparing to launch a phase 3 study, pivotal in the process of registration of masitinib in this indication

This randomized placebo-controlled phase 2 trial, conducted by Professor François Piette (Hôpital Charles-Foix in Ivry-sur-Seine) and colleagues from 12 study centers across France, investigated the hypothesis that masitinib's targeted inhibitory action on mast cells may reduce the symptoms of Alzheimer's disease. A total of 35 patients were included in this study. Neuroinflammation is thought to be important in Alzheimer's disease pathogenesis. Mast cells are a key component of the inflammatory network and participate in the regulation of the blood-brain barrier's permeability. Masitinib, a selective oral tyrosine kinase inhibitor, effectively inhibits the survival, migration and activity of mast cells.

Professor Marc Verny (Head of the Geriatric Department, Pitié-Salpêtrière hospital in Paris and study co-investigator) commented « Masitinib administered as an add-on therapy to standard care during 24 weeks showed promising signs of retarding the rate of cognitive decline of Alzheimer's disease compared to placebo, with an acceptable tolerance profile. Although the size of this study was too small to make any definitive conclusions about treatment efficacy, the evidence is sufficiently compelling to warrant further phase 3 investigation ».

In summary, patients with mild-to-moderate Alzheimer's disease received masitinib as an adjunct to cholinesterase inhibitor and/or memantine treatment for 24 weeks. Improvement in cognitive function and functional capacity was seen in the masitinib treatment group when compared to a placebo group, as evident through the sustained and statistically significant response in ADAS-Cog, as well as the mean change in ADAS-Cog, MMSE, and ADCS-ADL values relative to baseline. These findings were additionally supported by favorable response in the CIBIC-plus and CDR analyses. Such broad benefits are desirable in Alzheimer's disease as this effectively translates into an improved quality-of-life.

Professor Olivier Hermine, President of the scientific committee of AB Science and co-corresponding author on this paper declared: « Given masitinib's selective targeting of mast cells and specific kinases, the results from this study help establish the role of mast cells in the pathogenesis of neurodegenerative diseases such as Alzheimer's disease. Masitinib may therefore represent an innovative approach in the treatment of Alzheimer's disease. As such, AB Science has launched a clinical development program in the treatment of Alzheimer's disease and is actively preparing to launch a phase 3 study. For this we have already received a positive scientific opinion from the European Medicines Agency (EMA) on the phase 3 study design, which is an important step for the development of masitinib in Alzheimer's disease ».

Source AB Science