Research into innovative small molecule therapeutics with disease modifying potential in Alzheimer’s disease (AD) has been conducted at Alzheon since 2013.
This research and development has yielded a novel clinical candidate, ALZ-801, a phase 3-ready, first-in-class, small molecule oral inhibitor of amyloid aggregation and neurotoxicity – hallmarks of AD. ALZ-801 emerged as a drug candidate through the re-evaluation of tramiprosate, the active molecule of a prodrug ALZ-801, which had been previously studied for the treatment of AD in two large phase 3 studies in North America and Europe, and demonstrated a favourable safety profile but failed to show conclusive clinical efficacy in the overall Alzheimer’s population. ALZ-801 is a novel optimised prodrug of tramiprosate based on insights into tramiprosate’s mechanism of action and clinical results in genetically-defined subset of AD patients.