Biogen and Ionis Announce Topline Phase 1/2 Study Results of Investigational Drug in Amyotrophic Lateral Sclerosis
CAMBRIDGE, Mass. and CARLSBAD, Calif., May 16, 2024 (GLOBE NEWSWIRE) — Biogen Inc. (Nasdaq:BIIB) and Ionis Pharmaceuticals, Inc. (Nasdaq:IONS) announced the decision to terminate development of BIIB105 (ION541) an investigational antisense oligonucleotide (ASO) for amyotrophic lateral sclerosis (ALS) based on topline results from the Phase 1/2 ALSpire study. BIIB105 was designed to reduce expression of ataxin-2 (ATXN2) protein and demonstrated statistically significant cerebrospinal fluid (CSF) ATXN2 protein reductions in the study. However, over the 6-month placebo-controlled period, treatment with BIIB105 did not result in a reduction in levels of plasma neurofilament light chain (NfL), a marker of neurodegeneration and neuronal damage. Additionally, BIIB105 did not demonstrate an impact on clinical outcome measures of function, breathing, and strength.