Biogen announced final results from a Phase 1/2 study of tofersen (BIIB067) for the potential treatment of superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS) were published in The New England Journal of Medicine. A mutation in the SOD1 gene is believed to be a genetic driver of disease in approximately two percent of all ALS cases. The final data demonstrate proof of concept and efficacy.
The phase 1/2 trial randomized, placebo-controlled, single- and multiple-ascending dose study evaluated the safety, pharmacokinetics, pharmacodynamics and exploratory efficacy endpoints. In the multiple ascending dose (MAD) portion of the study, participants with SOD1-ALS were randomized to receive tofersen (20 mg, 40 mg, 60 mg or 100 mg) or placebo for 12 weeks. The primary objective of the MAD portion of the study was to evaluate the safety, tolerability and pharmacokinetics of tofersen. The most common adverse events in people who received one or more doses of tofersen (n=38) were headache, procedural pain, post-lumbar puncture syndrome and falls.
The secondary outcome was the change from baseline in cerebrospinal fluid (CSF) SOD1 protein concentratio...
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