Hi
@Uboy nothing wrong with your suggestion
However I tend to think a little different from you on this occasion
I believe the data should be acceptable in a comparison across both trials and therefore crosslinked and accepted by both the FDA and EMA
But I think combination therapy will be saved for down the track
IMHO we will take on a partner for the FDA route, because I believe that partner, as has been said would want input into the trials
And as such that partner would want to extract full value from that trial moving forwards into the future
So I could see the FDA route taking a more comprehensive approach
They have discussed and it has been touted that 1102 could well be used in all boys including ambulant boys
Go back to the initial talks with Pat from the PPMD society and Diamonds stuttering when asked would 1102 be taking the trials forwards in none ambulant boys only, he replied that the EU trial would take that approach but never commented on the FDA approach, she also asked would their hearts be monitored and would an extention to the trial be available on completion of the trial
Well we can see the latter, was taken onboard via the EMA route as the open label extention has been added to the trial
So I am thinking
The FDA route trial would look something like this
A placebo arm
A none ambulant arm
A ambulant arm
A 25mg dosing arm
A higher dose arm
Monitoring of the hearts of these children
Extended open label period post trial
Questionable
The Ambulant boys taking part would have to be screened intially for elevated measures of CD49d to participate
FDA would have to approve larger dosing than their restricted 25mg
This would I believe satisfy the major advocacy groups supporting us and also any pharma engaging in our future
As you can imagine this would incur far greater an expense than our EU trial, hence I believe the partnership will be required to take us down this track.
These are just my ramblings on how I see things going
Whichever way you look at it things look very rosy for this company moving forwards
We just have to sit tight and hope the deal been brokered comes up to expectations
Those expectations have not been set by myself, we all know here what deals have been brokered in this space, management have set the seed of expectation in constant presentations
All they have to do now is deliver
See how we go
