Avagacestat is primarily investigated for its potential in treating Alzheimer's disease. It functions as a gamma-secretase inhibitor, targeting the amyloid precursor protein processing pathway to reduce the formation of amyloid-beta peptides, which are implicated in the development of Alzheimer's. Clinical trials have explored its efficacy and safety in slowing disease progression, though results have shown mixed outcomes and challenges with side effects. Research continues to assess its therapeutic potential and optimize its use in neurodegenerative conditions.