AMX0035: Phase II Clinical Trial to Evaluate Safety, Tolerability, and Biomarker Engagement of AMX0035 in Patients with ALS.

Protocol title: A multicenter, randomized, double-blind, placebo-controlled 24-week study evaluating the safety, tolerability, efficacy, pharmacokinetics and biological activity of AMX0035 The objective of this study is to determine the safety and efficacy of AMX0035 in subjects with Amyotrophic Lateral Sclerosis (ALS). ALS is a progressive neurodegenerative disease for which there is no cure. There is just one medicine approved specifically for treating ALS, Rilutek (riluzole), and it only provides a modest benefit for subjects. ALS also exacts a significant economic burden. AMX0035 has demonstrated efficacy in models of neurodegeneration, classical activation of neuroinflammation, and bioenergetics deficits. The individual components of AMX0035, PB and TUDCA have demonstrated efficacy in in vivo models of ALS, Parkinson’s, Alzheimer’s, ischemia, and many others. Each individual component has also been tested in small clinical trials of ALS subjects and was found to be safe and well-tolerated, and hit primary endpoints of efficacy. The first trial under this IND will be a randomized double-blind placebo-controlled Phase II trial to evaluate the safety and efficacy of AMX0035 for treatment of ALS. The program is designed to demonstrate that treatment can slow the decline in function, muscle strength, and vital capacity, and to assess the impact of AMX0035 therapy on biomarkers of ALS including blood levels of phosphorylated axonal neurofilament H subunit and 18 kDa translocator protein PET tracer uptake. This Phase II trial would also serve as the basis for the design of a pivotal trial in this subject population.