247AD201 CELIA

A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Assess the Efficacy, Safety, and Tolerability of BIIB080 in Subjects with Mild Cognitive Impairment Due to Alzheimer’s Disease or Mild Alzheimer’s Disease Dementia

01/07/2023 to 31/12/2027

Intraneuronal NFTs composed of abnormal hyperphosphorylated tau protein are a neuropathological hallmark of AD, along with extracellular plaques containing Aβ. Tau pathology has been shown to correlate with neuronal loss and cognitive decline [Morris 2011; Spires-Jones 2009]. Previous nonclinical proof-of-concept work with ASOs in a transgenic mouse model with established tau pathology has demonstrated that an ASO targeted to the human MAPT mRNA reverses tau deposition and prevents neurodegeneration [DeVos 2017].
BIIB080 is an 18-nucleotide chimeric 2ʹ-MOE/DNA modified ASO that selectively reduces MAPT mRNA. BIIB080 is designed to bind to MAPT pre-mRNA and prevent translation of tau protein by promoting the degradation of the MAPT-encoded RNA. BIIB080 is in development for the treatment of AD.