Safety and tolerability studies of antisense oligonucleotide BIIB078 in adults with amyotrophic lateral sclerosis

The Phase 1 trial evaluated the safety and pharmacokinetics of BIIB078, an antisense oligonucleotide targeting C9orf72 in amyotrophic lateral sclerosis (ALS). Conducted across six countries with 106 participants, the study found that while adverse events were mostly mild or moderate, BIIB078 did not show significant clinical benefit or neurofilament reduction compared to placebo.