Ann: ATL1102 DMD Paediatric Investigation Plan submitted to EMA, page-21

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