AB Science has received approval from several European countries to initiate the confirmatory phase 3 study of masitinib in ALS

Core Viewpoint - AB Science has received approval from several European countries to initiate a confirmatory Phase 3 study of masitinib in amyotrophic lateral sclerosis (ALS), following validation by the EMA and FDA [1][2]. Group 1: Study Design and Population - The confirmatory Phase 3 study, AB23005, is a multicenter, randomized, double-blind, placebo-controlled trial designed to confirm the efficacy and safety of masitinib in ALS patients [3]. - The study will enroll 408 patients with ALS, focusing on those with normal disease progression and no total loss of function [4]. - The optimal population for the study is defined as patients before complete loss of function, representing approximately 75% of the total ALS patient population [5][6]. Group 2: Clinical Data and Mechanism of Action - Previous studies, including the Phase 2B/3 study AB10015, indicated a median survival increase of +12 months for the targeted patient population [5]. - Masitinib's mechanism of action involves targeting mast cells and microglia, which has been supported by preclinical studies demonstrating its immunomodulatory properties [11][22]. Group 3: Statistical Considerations - The AB23005 study aims to enroll approximately 200 patients per treatment group, more than double the number in the previous study, to enhance statistical power [10]. - The optimal population included approximately 90 patients per treatment group in the AB10015 study, where masitinib showed statistically significant effects on key endpoints [9]. Group 4: Intellectual Property and Regulatory Status - AB Science has secured patent protection for masitinib in ALS until 2037, with potential extensions in certain jurisdictions [14][15]. - Masitinib has received orphan drug designation from both the EMA and FDA, granting it marketing exclusivity for 10 years in Europe and 7 years in the United States [17].