November 1, 2021 – The Phase 3 VALOR results for tofersen did not show a statistically significant change in disease progression as assessed by the ALSFRS-R scale, however a number of other outcome measures suggested the treatment was able to have an effect on people with SOD1-ALS. As more open label extension data is shared, and further discussion across the field occurs, there will be a stronger consensus on what the data reveals about tofersen.
In other promising news related to this therapy, Biogen recently initiated the ATLAS study, which will start treatment of tofersen in people living with an SOD1 mutation before symptoms of ALS begin. The study will look at whether early treatment with tofersen can significantly delay the onset of clinical symptoms. Participants randomized to placebo would be provided active drug immediately upon diagnosis. Never clinically tested before, early intervention has long been considered a key area for study in ALS. The ATLAS trial is an important next step not only in the evolution of tofersen for SOD1-ALS but in paving the way for future ALS trials.
Read more in the International Alliance of ALS/MND Associations Scientific Advisory Council (SAC) briefing note on tofersen.
October 19, 2021 – The ALS community has been eagerly anticipating the results of the Phase 3 VALOR trial for tofersen, an experimental drug being evaluated as a therapy for a genetic form of ALS caused by mutations in the gene SOD1. The results were presented earlier this week at the American Neurological Association (ANA) 2021 virtual meeting.
Although the primary endpoint of ALSFRS-R was not met in the same manner many anticipated, the data does provide some hopeful signals. Other data from the study showed positive indications that the therapy may affect disease progression but further discussion and analysis across the field will help to better understand the complexity of the results and what this means for next steps. The many people and families around the world affected by SOD1-ALS who participated in the study deserve incredible thanks for their contributions. More information to come. You can read the Biogen press release here.