Update on tofersen clinical program to treat ALS -Biogen

Statement to ALS Community ( summarised) :Tofersen is an investigational molecule for superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). A mutation in the SOD1 gene is believed to be a genetic driver of disease in approximately two percent of all ALS patients. The safety and efficacy of tofersen is still being investigated in a Phase III placebo-controlled clinical study. We designed our Phase III study to provide the scientific answers necessary to file applications for regulatory approval as quickly as possible and to expose as few patients as possible to placebo.For these reasons, the study is short in duration and includes only a small number of patients.Since our Phase III tofersen study completed its enrollment at the end of 2020, we have heard and listened to the voices of many patients with SOD1-ALS and their families seeking to gain access to tofersen before the study is completed and before tofersen is confirmed to be safe and effective. Biogen has engaged with regulators, clinical investigators, ethicists, and patient advocates to explore all potential ethical avenues to provide access to tofersen, outside of the ongoing Phase III study.We do not believe it is fair to ask participants in this study to continue to receive placebo while other SOD1-ALS patients are offered access to tofersen, but we do believe that access could be provided as soon as the placebo-controlled study has ended. However, until the safety and efficacy have been established, we will prioritize early access for a subset of the most rapidly progressing patients with this rare, severe disease. Beginning in mid-July 2021, after patients in this study currently on placebo transition to active therapy, and before the safety and efficacy of tofersen are established, compassionate use access will be allowed for a subset of the SOD1-ALS population with the most rapidly progressive disease. In the fall of 2021, if results from the Phase III study indicate that tofersen is safe and effective, and if no further studies are required, we will initiate an early access program (EAP) for the broad SOD1-ALS population.