First interventional study for asymptomatic FALS carriers ( SOD1)

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Nikki J

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They will monitor neurofilament monthly. When it rises ( which in SOD1 is a sign of imminent symptom onset) they will be randomized into tofersen ( the sod1 aso currently in final trial phase) and placebo. Anyone who then develops ALS will go into an open label extension. Of course the hope is that onset will be prevented or at least delayed.

 

ItsTough

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The BIIB067 Phase 3 results will be finishing up in August, correct?

The preliminary results from Phase 2 seemed incredible. Does the fact that they're starting this study before the Phase 3 results are out give any indication that it is looking good so far in Phase 3?
 

Nikki J

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End of August yes.

they think it is promising yes but until they get all the data and analyze it who knows. They know it is safe though and ultimately the ATLAS model is how to treat FALS
 

GXTrex

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What is meant by the ATLAS model? Is that using ASO's?
 

Nikki J

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Treating presymptomatically by biomarker indication. I just called it a model because if the stars align and we get c9 biomarkers ( need people in studies now) they will do the same IF our aso works but it will be called something else
 

GXTrex

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Oh ok. I dont understand why NFL can be used for SOD1 but not for C9? Isn't NFL released when neurons die? If so it should be able to be used for all FALS.
 

Nikki J

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They think it doesn’t always show. There was a presentation at the mnda and it showed one person who was a very slow progressing c9 with a fairly normal value. I was told it will likely be a panel. They are looking at another test that I think is always elevated in c9 ALS but I think they aren’t sure when it happens
 
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