Earlier this year, the global biotechnology company Biogen announced its decision to discontinue development and commercialisation of aducanumab for early Alzheimer's disease (AD), continuing to advance lecanemab and accelerating BIIB080, an antisense oligonucleotide (ASO) targeting tau. BIIB080 is the first tau targeting ASO to enter human AD clinical trials. Findings from a Phase Ib trial in people with AD showed that BIIB080 was generally well tolerated with favorable trends on multiple exploratory endpoints of cognition and activities of daily living in AD, building upon prior results which showed a reduction of tau protein in the cerebral spinal fluid (CSF t-tau) and tau positron emission tomography (PET) across brain regions.
These findings support further investigation of BIIB080 in the ongoing CELIA phase II trial evaluating its efficacy, safety and tolerability in people with mild cognitive impairment (MCI) due to AD or mild AD dementia. The company plans to enrol 735 participants aged 50-80 years who will receive either a low dose or high dose of BIIB080, or a placebo, as an injection into the fluid around the spinal cord once every 12 weeks or 24 weeks. The primary outcome is the dose response in change of CDR-SB from baseline after 76 weeks. Secondary measures include ADCS-ADL-MCI, ADAS-Cog 13, MMSE, iADRS, ADCOMS and adverse events. After 76 weeks of treatment, eligible participants will move in the Long-Term Extension Period, where all participants will receive the drug for an additional 96 weeks. The trial is being carried out in around 140 sites across North America, Europe and Asia.
The University College London Hospitals NHS Foundation Trust (UCLH) has recruited the first participant in the UK into the CELIA trial. Dr Catherine Mummery is leading the trial which is taking place at the NIHR UCLH Clinical Research Facility. The recruitment for this study is also opened in Oxford. The CELIA study in Oxford is a collaboration between Oxford Health Clinical Research Facility (OH CRF), Oxford University Hospitals Clinical Research Facility (OUH CRF) and Oxford University Hospitals Pharmacy and Neurology departments.