Novel Alzheimer's Drug Targeting Tau Shows Promise in Mid-Stage Trial

The tau-targeting agent diranersen did not meet the primary endpoint of the phase II CELIA trial, but that isn't stopping the drug from moving forward.

The placebo-controlled study is the first to show a cognitive benefit and a reduction in tau pathology in people with early Alzheimer's disease, reported Catherine Mummery, MBBS, PhD, of University College London, in a presentation at the Alzheimer's Association International Conference (AAIC).

All diranersen doses tested in CELIA reduced tau PET signals and cerebrospinal fluid (CSF) tau in people with mild cognitive impairment or mild dementia due to Alzheimer's disease, Mummery said. Every dose slowed cognitive decline.

Because the lowest dosage performed best, the drug missed its primary endpoint of demonstrating a clear dose response.

"However, there was consistency in treatment effect across all dose arms and consistent favorable trends seen across all secondary cognitive and composite measures," Mummery said. Based on that, diranersen will advance to a phase III trial.