A sign marks a Biogen facility in Cambridge, Massachusetts, U.S. January 26, 2017. REUTERS/Brian Snyderexperimental treatment for an inherited form of amyotrophic lateral sclerosis failed to reach its main goal, but secondary measures and biomarkers showed favorable trends, the company said on Sunday.
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 slower-progressing disease, the difference was 1.4 points.
"Despite the fact that there was no statistically significant difference in the primary endpoint, there is a clinical signal here," said Dr. Timothy Miller, the study's lead investigator and ALS Center Director at Washington University School of Medicine, St. Louis. He presented the tofersen data at the annual meeting of the American Neurological Association.