Tofersen demonstrates positive results in Phase I/II trial for amyotrophic lateral sclerosis

Biogen (MA, USA) has announced positive results from a Phase I/II study of tofersen – an antisense oligonucleotide – for the potential treatment of SOD1 amyotrophic lateral sclerosis (ALS). These results have been published in The New England Journal of Medicine. The Phase I/II study of tofersen was a randomized, placebo-controlled, single- and multiple-ascending dose study that evaluated the safety, pharmacokinetics, pharmacodynamics and exploratory efficacy endpoints in individuals living with SOD1-ALS. In the multiple-ascending dose part of the study, participants with SOD1-ALS were randomized to receive either tofersen (20 mg, 40 mg, 60 mg or 100 mg) or placebo for...