Ceperognastat in Early Symptomatic Alzheimer Disease

Does the -linked -acetylglucosaminidase (OGA) inhibitor ceperognastat affect progression of early symptomatic Alzheimer disease vs placebo?In this randomized phase 2 trial of 326 participants, prespecified success criteria for the primary outcome (slower change in the Integrated Alzheimer’s Disease Rating Scale score at week 100 in participants with baseline low to medium tau levels on positron emission tomography) were not met. Posterior mean change in score from baseline at week 100 was −8.39 with ceperognastat 0.75 mg, −13.27 with ceperognastat 3 mg, and −10.07 with placebo (16% less and 32% more clinical progression, respectively, with ceperognastat vs placebo).-linked -acetylglucosaminidase inhibition with ceperognastat did not demonstrate a beneficial effect on Alzheimer disease progression.

Corresponding Author: Adam S. Fleisher, MD, Eli Lilly and Company, 893 S Delaware St, Indianapolis, IN 46225 (afleisher@lilly.com).

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