AB Science announces the identification of a plasma biomarker that indicates the activity of masitinib in treating ALS

Core Insights - AB Science has identified a plasma biomarker that indicates the activity of masitinib in treating Amyotrophic Lateral Sclerosis (ALS) and can identify patients with pro-inflammatory microglia, which are targeted by masitinib [1][2] - This biomarker is also applicable to progressive forms of Multiple Sclerosis (MS) and Alzheimer's disease, potentially enhancing treatment response identification and registration chances in neurodegenerative diseases [1][2] Biomarker Characteristics - The biomarker is blood-based, easy to collect, and can be accurately evaluated using ELISA [4] - It is produced by pro-inflammatory microglia, activates microglia and astrocytes, and contributes to a neuroinflammation feedback loop [4] - It is predictive of survival in ALS, potentially explaining why masitinib could extend survival in specific patients [4] Clinical Development - The biomarker will be introduced in the phase 3 program of masitinib for ALS, progressive MS, and Alzheimer's disease to validate its mechanism of action and clinical relevance [3] - Once validated, it could facilitate patient registration by determining the best responders to treatment and serve as a surrogate endpoint of efficacy [3] Regulatory Context - The FDA encourages the incorporation of exploratory biomarkers in all phases of ALS drug development, which may lead to discussions on surrogate endpoints for accelerated approval [3]