Presymptomatic trials?

GXTrex

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@Nikki J
Is toferson designed for symptomatic, pre symptomatic, or both for SOD1?

My family is c9 but I am obviously hoping for toferson to have good results for those impacted.
 

Nikki J

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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
 

GXTrex

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@Nikki J

So they would start the pre symptomatic treatment when biomarkers indicate ALS is starting?

If they can't find reliable biomarkers for c9 or another genetic defect, would they just wait for the disease to manifest before starting future treatments?
 

Nikki J

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I think the trials will have a placebo but yes

and yes probably. You can’t know if it works by just randomly giving carriers drugs. Especially with c9 onset being so variable.

moved posts to avoid hijacking
 

GXTrex

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@Nikki J
Sorry about hijacking
At least if they found a viable treatment it can be given as soon as possible for all c9 pals. Is there any headway on c9 biomarkers?
 

Nikki J

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Yes I think the DIALS study is looking at a couple of things. They will find something ( or a panel of tests more likely I heard) which is why study participation is critical
 

GXTrex

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Do you know when BIIB078 results should be released?
 

Nikki J

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Phase 1 is finishing at the end of September. After that if they see promise they will do another phase.
 

GXTrex

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Phase one is just is it safe in humans though right? Not if it's effective?
 

Nikki J

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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 they 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.
 
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