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Biogen and Ionis’s SOD1-antisense oligonucleotide tofersen failed a first phase III trial, raising questions about the next steps for this drug and for future ALS trials.
Biogen and Ionis’s tofersen missed the primary endpoint in a first phase III trial of an antisense candidate for amyotrophic lateral sclerosis (ALS). The findings, reported at the American Neurological Association (ANA) meeting in October, were another blow for patients with the rare, fatal neurodegenerative disease. But there were hints of a possible effect — raising hopes that earlier use of the drug might show efficacy.
