Biogen c9 aso stopped

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Unfortunately I do not have access to message to Dr. Harms anymore about his thoughts. He did say awhile ago that the other ASO's (Wave and Dr. Brown out of UMASS) are different enough so hopefully their results and other C9 treatments could be positive.

Will Biogen try another C9 treatment? Do pharmaceutical companies typically try again after a failed attempt?
 
I am anxious to see the full results. The question is is the target wrong as the seem to say in the release or did it just not work in which case there is more hope for the other studies. If it is a wrong target ( which seems a bit of a leap to be sure) then the other asos are probably going to fail too

tofersen was actually biogen’s second try for a sod1 aso. The first showed some positive biomarker results but there was a problem ( not safety) and it needed to be reformulated. This sounds different but it is early. We will hear more details.
 
The report says it did not support the hypothesis that reducing RNA and DPRs would stop disease progression. It does not say if these were reduced in patients and by how much. Hopefully they weren't reduced enough? There arent many details.

How long does it take to get the full results usually?
 
A few months usually. they will be published and also presented at conferences. Apparently they had proof of target engagement.

if it were a matter of dosing they would have added another cohort. It s concerning too that the treatment group did worse than the placebo.

c9 is so very complicated- much more so than sod1. there are going to be lots of questions and reassessments going forward
 
Biogen's ASO targets the V1 and V3 variants on the mutated and normal chromosome. Wave only targets those on the mutated chromosome. Hopefully that is the issue.
 
Dr Brown said last night he had his c9 aso protocol ready to go but is now waiting to see the biogen data
 
Any word on if Wave's aso clinical trial is still going? Have they commented at all?
 
I haven’t heard anything except Dr Brown said something to the effect that WAVE’s aso had a special design which we knew. Those weren’t his words I can’t remember what he said. WAVE is in a different place having a trial in progress. I think they are still doing single dosing for safety which would be easy to pause if they felt the need. The AAN meeting starts Saturday. I don’t know if biogen will be ready to share anything then. I am sure WAVE is as anxious as Dr Brown to see specific data so they can figure out its applicability to their work
 
What do you mean Waves had a special design? Do you mean that Waves looks to only attack the bad copy of the gene vs both in Biogens? What is Dr. Browns approach?
 
I don’t remember his words. It wasn’t special. Just something positive about their aso. it is somewhat different than biogen which at this point is obviously good

I only know that Dr Brown ‘s spares V2. I don’t know how it is different than biogen. It has to be otherwise it would be a patent issue.
 
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