Hi Sankiado
I doubt very much that ANP would be applying for this grant funding, and i think partly you answered youir question yourself,
For the funding that ANP were to receive i doubt they would want to share ROI with the stage we are now at,
I know they say would take interest from clinical trials but i think this type of funding from what can be read it looks more to be directed at early research than clinical trials
In research early early stage for another indication then maybe,
Not only that it could be detrimental to any talks with potential pharmas if they were to involve a third party
I couldn't see especially for the amount involved where would it take us.
Diamond actually covers this in the last interview by saying their are two parts to our market one being the scientific and one being capital talks about bringing long term investment onboard not sure when thats gonna happen though
I think any association with any of the DMD organisations would be primarily to get the message out there that we have had a successful trial and to create a following from within the DMD community to support early access shopuld our PH2b be succesful
This they have proven as having a very strong voice in their support to bring sareptas drug to market after initially being knocked back by the FDA
And as we have already heard Pat pledged her support from the PPMD community to help in anyway they could, but i doubt this will be cash orientated
They have already supported us in a major way by suggesting in the Ph2b that we monitor cardiac response in these children
They could also support us through connections both in the US and their tie up with the UK based community in getting the word out to parents that a trial is coming up that shows great promise in none ambulant children and therefore help us recruit our 75 children for the 2b far sooner than we could by going it alone, Pat will already have a data base of the sick children one would think.
This is how i believe they will be a massive support behind us
and as i have said before they should have been approached pre ph2 just to have knocked on Pats door and introduced ourselves would not have done us any harm infact it just goes to show through their reluctance to approach the DMD they actually let us as shareholders down in a big way once more, if they had spoken to Pat pre Ph2 then we could well have monitored the cardiac muscle, and even maybe, had even greater results than we now have
But this is Diamond and a very reserved board that we have i could list questions that Diamond has dodged or not answered through these online interviews and cannot for the life of me understand why he just does not come clean with the answers.
Would it be detrimental to inform the interviewer when asked what would be the cost for a Ph2b trial his question was totally ignored
Would it be detrimental to inform Pat when she asked the question of dosing what were they thinking for the Ph2b
When asked will the 2b trial involve ambulant children in the EU Diamond answered the question we are just looking at none ambulant children for the EU trial
But when Pat threw in the curved ball will that be the same approach for the FDA ie none ambulant only
Diamond fell apart and threw Gil under the bus and left Gil to save the day, yet they say
Why didnt he just say Pat our target is cd49D and this biomarker is more prevalent in none ambulant boys than ambulant boys and as we saw the drug had effect on CD49d by reducing in numbers whilst these boys were taking ATL1102
Perhaps down the track in early onset of the disease when ambulant boys start to show signs of excess cd49d we could trial the drug on the back of the 2b success hopefully
personally on researching this it would appear its not a case of ambulant or none ambulant its more a case of speed progression of the disease from onset to wheelchair
And for all those that are hailing this as the be all and end all in the inflammation market then i think you need to come back down to earth
Atl1102c for whatever reason appears to have effect on excess cd49d in the system so our ideal target with reference to further indications, then i am presuming we will be targeting diseases where cd49d is most prevalent in the illness and of course this is not just my rambling on this is backed up by clinical representation from pinto himself
Results
We studied 75 DMD patients at different stages of their disease and observed that increased percentages of circulating CD4+CD49dhi and CD8+CD49dhi T lymphocytes were correlated with both severity and a more rapid progression of the disease. Moreover, T+CD49d+ cells were also found in muscular inflammatory infiltrates. Functionally, T cells from severely affected patients exhibited higher transendothelial and fibronectin-driven migratory responses and increased adhesion to myotubes, when compared to control individuals. These responses could be blocked with an anti-CD49d monoclonal antibody. Conclusion
CD49d can be used as a novel biomarker to stratify DMD patients by predicting disease progression for clinical trials. Moreover, anti-CD49d peptides or antibodies can be used as a therapeutic approach to decrease inflammation-mediated tissue damage in DMD.
We demonstrate that circulating CD49dhi T cells can be successfully used as a biomarker for disease progression in DMD patients. Moreover, this seems to be specific for DMD since it is not found in IBM patients, who also suffer from an inflammatory muscle disease.
its all here for those that have not gone through it before
