Biogen and Ionis halt BIIB105 development for ALS due to lack of efficacy in Phase 1/2 study.

Biogen and Ionis Pharmaceuticals halt BIIB105 (ION541) development for ALS due to lack of efficacy in reducing neurodegeneration and improving functional measures in Phase 1/2 ALSpire study. The drug demonstrated a reduction in ATXN2 protein levels but failed to impact clinical outcome measures. Companies continue to explore other ALS treatments, including Ionis' Phase 3 ulefnersen program for genetic FUS-ALS.

May 16, 2024
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