Biogen has reported positive interim results of a Phase I/II trial of tofersen being studied to treat amyotrophic lateral sclerosis (ALS) in adults with a confirmed superoxide dismutase 1 (SOD1) mutation.

The antisense oligonucleotide (ASO) Tofersen selectively targets the genetic driver of ALS, a fatal neurodegenerative disease characterised by motor neuron loss in the brain and spinal cord.

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Biogen noted that the interim data highlights a statistically significant reduction in SOD1 protein levels and a numerical trend towards slowing of clinical decline in SOD1-ALS patients treated with tofersen.

“We are committed to bringing a potential breakthrough therapy to patients with ALS.”

Biogen research and development executive vice-president Michael Ehlers said: “The interim results of this study, which achieved proof-of-biology and proof-of-concept, support the initiation of Phase III clinical trial to confirm the efficacy and safety of tofersen in SOD1-ALS patients and further demonstrate the potential of ASOs to target the genetic driver of disease.

“We are committed to bringing a potential breakthrough therapy to patients with ALS and we are expediting our efforts with the aim of addressing this urgent unmet need.”

The randomised, placebo-controlled, single and multiple-ascending dose Phase I/II study is evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics and exploratory efficacy endpoints of tofersen in 70 patients with ALS.

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Biogen randomised 50 participants with SOD1 mutations in the multiple ascending dose portion of the study. They received tofersen (20mg, 40mg, 60mg, or 100mg) or placebo for a period of 12 weeks.

This March, Biogen dosed the first patient in the Phase III VALOR study of tofersen in adults with ALS with a confirmed SOD1 mutation.