Topline Results from Phase 2 CELIA Study of Diranersen (BIIB080): First Study to Show Reduction in Tau Pathology and Cognitive Benefit in Patients with Early Alzheimer’s Disease
Biogen • • general
Key Points:
- Biogen announced topline results from the Phase 2 CELIA study of diranersen, a tau-targeting antisense oligonucleotide (ASO) therapy for early Alzheimer’s disease, showing significant biomarker impact and cognitive benefit despite not meeting the primary dose-response endpoint.
- The study demonstrated robust reductions in cerebrospinal fluid tau and tau pathology via PET imaging across all doses, with cognitive decline slowing particularly at the lowest dose of 60 mg every 24 weeks.
- Diranersen’s safety and tolerability profile was consistent with previous Phase 1b data, though a higher incidence of serious adverse events was observed at the highest dose.
- CELIA enrolled 416 participants with early Alzheimer’s disease and evaluated three dosing regimens over 76 weeks; an ongoing extension study is assessing long-term safety and efficacy.
- Based on these results, Biogen plans to advance diranersen to registrational development and will present data at the Alzheimer’s Association International Conference (AAIC) 2026 and other scientific meetings.