Biogen Inc. announced new 12-month data for tofersen, an investigational antisense drug for people with superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). The data show that earlier initiation of tofersen compared to delayed initiation (six months later in the open-label extension [OLE] study) slowed declines in clinical function, respiratory function, muscle strength, and quality of life. At the time of the analysis, because the majority of participants survived without permanent ventilation (PV), the median time to death or PV could not be estimated. However, early survival data suggest a lower risk of death or PV with earlier initiation of tofersen. These results are based on new integrated data from the pivotal Phase 3 VALOR study and its OLE study. At the time of the analysis, because the majority of participants survived without PV, the median time tdeath or PV anand median time to death, could not be estimated. However, early survival data suggest a lower risk of death or PV (Hazard ratio [HR] 0.36; 95% CI: 0.137, 0.941) and death (HR 0.27; 95% CI: 0.084, 0.890) with earlier initiation of
tofersen. Biomarker Results The latest 12-month results show that reductions in total SOD1 protein (a marker of target engagement) and neurofilament (a marker of axonal injury and neurodegeneration) were sustained over time.