Biogen Reports Positive Data from Phase I / II Study on Tofersen

Biogen has announced positive interim results from a tofersen phase I / II trial in the study to treat amyotrophic lateral sclerosis (ALS) in adults with confirmed mutation. superoxide dismutase 1 (SOD1).

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

Biogen noted that intermediate data showed a statistically significant reduction in SOD1 protein levels and a numerical trend towards slowing clinical decline in SOD1-SLA patients treated with tofersen.

"We are committed to providing a potential breakthrough treatment to ALS patients."

Michael Ehlers, Senior Vice President of Research and Development at Biogen, said: "Provisional results from this study, which has proven the biology and proof of concept, support the launch of a clinical trial phase III to confirm the efficacy and safety of tofersen in patients with SOD1-SLA and further demonstrate the potential of OAS to target the genetic factor of the disease.

"We are committed to providing ALS patients with potential breakthrough treatment and we are accelerating our efforts to address this urgent and unmet need."

The Phase I / II single-dose, multi-dose, randomized, placebo-controlled, multiple-dose study evaluates the safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy criteria of tofersen in 70 patients with ALS.

Biogen randomized 50 participants with SOD1 mutations to the multiple-escalating part of the study. They received tofersen (20 mg, 40 mg, 60 mg or 100 mg) or placebo for 12 weeks.

In March, Biogen administered the first patient in the VALOR Tofersen Phase III study in adults with ALS and a confirmed SOD1 mutation.