Follow-up analyses from the Phase 3 Valor study and the open-label extension study of tofersen, an investigational antisense drug, showed that the drug was effective in slowing down progression of ALS in people with SOD1 mutations. 

The study compared the impact of tofersen when taken earlier (people who were in the drug-treated group), compared to people originally assigned to placebo who began taking tofersen six months later as part of the 12-month open-label extension study. Earlier intervention was shown to slow declines in clinical function, respiratory function, muscle strength and quality of life. 

We are encouraged by Biogen's release of new data suggesting that early administration of tofersen can benefit people living with ALS who have the SOD1 mutation. The data are still preliminary and need to be further validated. We urge Biogen to do so quickly. We were an early funder of the antisense technology underlying tofersen and we are hopeful that the technology will soon benefit everyone living with genetic forms of ALS.

Read more about the open-label extension results.