The current phase 3 trial is for people with ALS. It is full and due to complete at the end of August. The early phase results looked good and anecdotally people say they are seeing stabilization , slowing or even improvements. They are going to do a presymptomatic trial and we are expecting it to enroll soon.
the biogen c9 trial is the same technology IF it has similar results they plan a presymptomatic trial for C9 IF they have reliable biomarkers. The neurofilament they are using for sod1 may not be a reliable for c9. This is why carriers need to be in studies
Theoretically yes but they are looking at efficacy things like frs svc and potential biomarkers like neurofilament and the c9 protein. They added another cohort at a higher dose and they must have had a reason. The original sod1 aso by biogen had only a phase 1 and then they reformulated it to what is now tofersen. There was a several year gap and tgey had to start again at phase 1 because it is a different drug.
i have hope for it but we need to be prepared that it could fail.