Biogen, Ionis terminate development of BIIB105 based on data from ALSpire study

Biogen (BIIB) and Ionis Pharmaceuticals (IONS) announced the decision to terminate development of BIIB105 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 protein and demonstrated statistically significant cerebrospinal fluid 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, a marker of neurodegeneration and neuronal damage. Additionally, BIIB105 did not demonstrate an impact on clinical outcome measures of function, breathing, and strength. During the 6-month placebo-controlled portion of the study, the most common adverse events in BIIB105 treated participants were procedural pain, headache and fall. AEs leading to study discontinuation were higher in the BIIB105 group compared with the placebo group.