AI Summary
This article discusses the safety, tolerability, and pharmacokinetics of the antisense oligonucleotide BIIB078 in adults with C9orf72-associated amyotrophic lateral sclerosis (ALS), based on a phase 1 study. The study did not show a reduction in neurofilament levels or benefit on clinical outcomes compared to the placebo group, leading to the discontinuation of BIIB078 clinical development. Despite this, the results will contribute to understanding the complex pathobiology of C9orf72-associated ALS.
On the basis of these phase 1 study results, including secondary and exploratory findings showing no reduction in neurofilament levels and no benefit on clinical outcomes relative to the placebo cohort, BIIB078 clinical development has been discontinued. However, these results will be informative in furthering our understanding of the complex pathobiology of C9orf72-associated ALS.