Ionis announces Biogen’s CELIA study did not meet primary endpoint

Ionis Pharmaceuticals (IONS) announced that its partner, Biogen (BIIB), shared compelling topline results from the Phase 2 CELIA study evaluating diranersen, an investigational antisense oligonucleotide, ASO, therapy targeting tau, in individuals with early Alzheimer’s disease, AD. The Celia results provide the first evidence from a randomized Phase 2 study of a tau-directed therapy demonstrating both robust biomarker impact and cognitive benefit in early AD. Key highlights: Based on the strength of the biomarker and efficacy data, Biogen plans to advance diranersen to registrational development; CELIA did not meet its primary endpoint assessing dose response; reductions in tau pathology were observed across all studied doses, with results generally consistent with those observed in the Phase 1b study; Pre-specified analyses of cognitive endpoints demonstrated slowing of clinical decline across all studied doses, particularly at the lowest dose; The safety and tolerability profile of diranersen was generally consistent with the Phase 1b study