This is the AVI presentation from this morning:

 

http://www.wsw.com/webcast/maxim3/avii/

 

Not much news. The Phase 2 placebo controlled double blind clinical trial testing 50 and 100 mg/kg dose levels is ready to start (subject to FDA approval of the protocol and IRB approval) at Nationwide Childrens Hospital in Columbus OH. Principal investigators: Drs. Jerry Mendell and Kevin Flanigan.

 

I'll attach a slide I created with the relevant stuff presented about AVI-4658. I love that picture on slide 14! He also quoted Dr. Muntoni who said that the biopsies from the UK trial started to look as non-DMD biopsies.

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Thanks Ofelia. This statement from Dr Muntoni is quite encouraging. Hope and. PRAY that flood gate gets open from here.

Raktim
I forgot to add that he mentioned twice NO immune response observed and reduction in CD3, CD4, CD8 inflammatory markers (meaning disease modification) observed in the UK trial.
Wow! This is very grand news. I didn't hear anything previously about things looking as non-DMD biopsies. Hopefully the no immune response will stick throughout the entire use of exon skipping. But, even if the immune response causes problems later, our guys have something to buy some time. I'll take what ever I can get.
Has there been any indications as to why they are getting no immune response? I dont work on the immunology side of science, but I understand the basica concepts, yet I find it confusing as to what is and isnt casuing immune responses in different studies.


Ofelia Marin said:
I forgot to add that he mentioned twice NO immune response observed and reduction in CD3, CD4, CD8 inflammatory markers (meaning disease modification) observed in the UK trial.
Yes, that is very confusing.

They did not give any explanations, both GSK and AVI report no immune response to date in their exon skipping trials. I did hear explanations from other people working in the exon skipping field (i think Pat posted Annemieke's explanation a while ago in the blog section here) but all are just assumptions.

I also heard that during the Action Duchenne conference, a presenter working on micro-dystrophin (George Dickson) explained why they think that Dr. Mendell's findings regarding the minidystrophin trial might not be correct, that the immune response was triggered by something else (unfortunately I do not have the details, I'm still waiting for them to post the presentations). It looks like there is a lot of debate about possible immune reponse against dystrophin.



Jonathan said:
Has there been any indications as to why they are getting no immune response? I dont work on the immunology side of science, but I understand the basica concepts, yet I find it confusing as to what is and isnt casuing immune responses in different studies.


Ofelia Marin said:
I forgot to add that he mentioned twice NO immune response observed and reduction in CD3, CD4, CD8 inflammatory markers (meaning disease modification) observed in the UK trial.

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