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 bis 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.