Biogen Trial Of ALS Drug Misses Main Goal, But Dev't Continues

A late-stage trial of Biogen Inc.'s experimental treatment for an inherited form of amyotrophic lateral sclerosis (ALS) failed to reach its main goal, but secondary measures and biomarkers showed favorable trends, the company said.

Photo Insert: Despite the setback, Biogen will continue its research.

Biogen will engage with regulators and other stakeholders "to understand the meaningfulness of this data and potential paths forward," Toby Ferguson, head of the neuromuscular development unit at Biogen, told Deena Beasley of Reuters.

The company is treating trial patients in a follow-on study and recently launched a Phase 3 trial of the drug, tofersen, in patients who are not yet experiencing ALS symptoms.

Tofersen, administered directly into the spinal canal each month, is designed to suppress the production of SOD1, a protein that can accumulate to toxic levels in ALS patients with mutations in a specific gene. Around 2% of ALS cases are believed to be caused by the genetic mutation.

After 28 weeks of treatment, the 108-patient trial showed a 1.2-point difference on a scale evaluating functional status for patients with fast-progressing ALS who were given tofersen compared to placebo patients, which was not statistically significant.

In the group of patients with the slower-progressing disease, the difference was 1.4 points. An improvement of at least 2 points would be clinically meaningful, Guggenheim Partners said in a recent research report.

Biogen also detailed results from secondary trial endpoints, including breathing ability and muscular strength, indicating that patients treated with tofersen fared better than placebo patients, and that placebo patients switched to the drug in the extension phase of the study experienced similar gains.

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