July 13, 2026 · JAMA · DOI: 10.1001/jama.2026.12768

Ceperognastat in Early Symptomatic Alzheimer Disease: A Randomized Clinical Trial

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The study aimed to evaluate the efficacy and safety of the oral O-linked N-acetylglucosaminidase inhibitor ceperognastat in slowing disease progression in early symptomatic Alzheimer's disease. Results showed no clinically meaningful benefit in slowing progression compared to placebo, with the ceperognastat 3 mg group experiencing more serious adverse events. Overall, ceperognastat did not demonstrate effectiveness in altering the course of early symptomatic Alzheimer's disease.

Adam S Fleisher, Leanne Munsie, Michele Mancini, Yun-Ju Cheng, Jiaying Guo, Hugh Nuthall, Diana Svaldi Otero, Vikas Kotari, William Kielbasa, Michael Hutton, Miroslaw Brys, Dustin J Mergott, Mark Mintun, Oskar Hansson

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