Valiltramiprosate, a potent inhibitor of amyloid-β oligomer formation, was tested in a 78-week phase III trial in APOEε4/ε4 homozygotes with early Alzheimer’s disease (AD).
The overall study population showed significant slowing of hippocampal atrophy but no significant clinical improvement on the primary cognitive endpoint.
Prespecified analyses in the mild cognitive impairment (MCI) subgroup demonstrated nominally significant slowing in cognitive decline and functional measures alongside imaging benefits.
The treatment was generally well tolerated, with mainly gastrointestinal adverse events and no increased risk of amyloid-related imaging abnormalities (ARIA).
