Assumptions, page-25

We note Exondys 51 received accelerated approval on the basis of three small studies of a similar size to the RCT for ATL1102 i.e. 10 to 15 patients.

The approval remains controversial and for this reason it is highly doubtful that the FDA would consider an accelerated approval for ATL1102 unless the data was absolutely exceptional i.e. highly statistically significant on primary and secondary endpoints.


The above doesn't make much sense to me. They are saying the FDA would not give us accelerated approval, yet they have already with sketchier outcomes. so.... if our outcome is not sketchy at all??? What will happen? Our data appears to be exceptional and best in class, so to refuse partial approval would be quite contradictory. We need top line results to know further thought direction here. And a confirmatory trial would be a great outcome and IMO the most likely.

I believe Dr Clary was put in place to advocate our results come next year. We have also yet to partner which could have a huge impact on our FDA approach. NB you mention the CAP deal wasn't provided in their report, and instead NEU was. NEU had their partner pay for future trials, CAP did not, and naturally the upfront reflects that.