Verubecestat is an investigational drug developed to target the underlying pathology of Alzheimer’s disease by inhibiting the beta-site amyloid precursor protein cleaving enzyme 1 (BACE1). By blocking this enzyme, Verubecestat reduces the production of beta-amyloid peptides, particularly amyloid-beta 42, which are believed to accumulate in the brain and form plaques associated with neuronal damage and cognitive decline in Alzheimer’s patients. The primary application of Verubecestat has been in clinical trials for the treatment of mild to moderate Alzheimer’s disease, as well as in studies involving individuals with prodromal Alzheimer’s. It was designed to slow disease progression by modifying amyloid pathology, potentially preserving cognitive function over time. Due to its oral bioavailability and ability to cross the blood-brain barrier, it was considered a promising candidate for long-term therapy in neurodegenerative disorders linked to amyloid accumulation. However, clinical development was discontinued after Phase III trials failed to show cognitive benefits and raised safety concerns, including liver toxicity and worsening of some cognitive measures. Despite this, Verubecestat contributed valuable data to the understanding of BACE inhibition and the role of amyloid in Alzheimer’s disease, influencing future research directions in neurodegenerative therapeutics.