Biogen reported topline Phase 2 CELIA results for diranersen (BIIB080), an antisense oligonucleotide targeting tau in early Alzheimer’s disease.
The trial missed its primary endpoint, which was a dose-response effect on clinical decline (CDR-SB) at week 76.
Despite the miss, pre-specified analyses showed reductions in tau pathology and signals of slower cognitive decline across doses, with the strongest effect at the lowest dose.
Biogen said the biomarker and efficacy findings were compelling enough to advance diranersen into registrational development / phase 3 planning.
Safety was generally consistent with earlier studies, though serious adverse events were reported more often at the highest dose.