New 12-month tofersen data show clinically meaningful benefit in people with SOD1-ALS

Biogen reported last week that their clinical trial (called VALOR) of tofersen has shown benefit that is clinically meaningful to people with a genetic form of MND. SOD1-MND, arising from a mutation in the SOD1 gene, accounts for approximately 2% of all MND cases.

When the trial started, participants were randomly assigned to receive either the active treatment, tofersen (two thirds of participants), or placebo (one third of participants). After 6 months on the trial, all participants were offered the chance to enter the open label extension (OLE) phase, in which everyone received the active treatment regardless of which group to which they were initially assigned.

Biogen have confirmed early indications that participants who received the active treatment from the start of the trial did better than those who received placebo for the first 6 months. Analysis at 12 months revealed that results favoured the earlier start of tofersen on measures of clinical function, respiratory function, muscle strength and quality of life. Early data also suggests a lower risk of death and permanent ventilation for those who started tofersen earlier.

More information can be found here.