Earlier this week, Biogen announced the results of their VALOR study, which evaluated the use of an antisense drug called tofersen to treat MND caused by the genetic mutation SOD1.
While we were disappointed to learn that the trial has failed to meet its primary endpoint, promising secondary endpoint data support the potential for tofersen to provide some benefit to patients. It may well be that the drug needs to be administered earlier in disease or may require a longer timeframe to show true disease modification.
Additionally, Biogen also announced their intent to continue with expanded eligibility for tofersen for its ongoing early access program to all people with SOD1-ALS. Biogen has also initiated the ATLAS study to determine if pre-symptomatic treatment of SOD1 mutation carriers may represent more optimal timing of intervention.