Can a New Oral Drug Slow Early Alzheimer's Disease? A…

Can a New Oral Drug Slow Early Alzheimer's Disease? A Major Trial Suggests Not Yet Researchers have evaluated whether ceperognastat, an investigational oral drug targeting tau pathology, can slow disease progression in people with early symptomatic Alzheimer's disease. While the drug showed evidence of affecting biological markers linked to the disease, it did not improve cognitive or functional outcomes, according to a randomized phase 2 clinical trial published in JAMA. Alzheimer's disease is characterized by the accumulation of amyloid plaques and tau protein tangles in the brain. Although newer therapies targeting amyloid have shown modest benefits, effective treatments aimed at tau pathology remain an important unmet need. Ceperognastat is an oral O-linked N-acetylglucosaminidase (OGA) inhibitor, designed to reduce abnormal tau accumulation and potentially slow neurodegeneration. The multicenter, randomized, double-blind, placebo-controlled phase 2 trial enrolled 327 adults aged 60 to 85 years with early symptomatic Alzheimer's disease across 72 sites in five countries. Participants were randomly assigned to receive: * Ceperognastat 0.75 mg once daily * Ceperognastat 3 mg once daily * Placebo Treatment continued for up to 124 weeks, with