ACE-2223-1

ACE-2223-1 is a small molecule drug candidate developed by Acelot, Inc. for the treatment of amyotrophic lateral sclerosis (ALS) and TDP-related frontotemporal dementia (FTD-TDP). It is the lead compound of the ACE-2223 program, designed using advanced AI and machine learning tools, including generative AI and molecular dynamics simulations. The drug targets the TDP-43 protein, aiming to restore its healthy function by disaggregating toxic oligomers and reducing the mislocalization and aggregation of TDP-43, which are pathological hallmarks of sporadic ALS. ACE-2223-1 specifically refers to a solid oral formulation that has been evaluated in pharmacokinetic studies in healthy adults to assess the impact of food on its absorption.