Ann: US FDA Type C Meeting for ATL1102 in DMD, page-70

”Firstly.. @Akki there is no issue with the primate data. The only issue with the primate data and the P2a trial is that is was for 6 months. EMA regulations need 6 months of tox data for a rare disease.. so we are all good with the EMA. FDA requires tox data for 9 months for rare diseases (12 months for non rare diseases)... so a 3 month gap. Will the FDA give ATL1102 the go ahead or make them do a 9 month tox study??”

George 1972: Spot on
FDA rules was 12 months for animal trial but they changed it to 9 months ( 6 months for rodents)
So the FDA
has to weigh up whether Atl1102 can produce the brilliant data for DMD.. and with a higher dose combined with a longer trial surpass those results.

FDA is aware that a drug is required urgently to treat these DMD sufferers so for the next 4 weeks they will be in talks with Anp and the US DMD community.
Or
like the Europeans: say the 12 month Trial is a success the FDA may consider waiving the longer animal studies because
of the Urgency to find a solution..... and accept the result as “Done”....So Anp can commercialise Atl1102 for DMD in Europe and USA...

The other Alternative is to see if the European trial is going well and to start the 9 month rox study on monkeys.

Itsa feels that with the Monkey trial for 6 months and the DMD trial for 6 months ...should be enough to do a trial in the US....
So Atl 1102 is derisked and it’s only time in the US..
No major problems..
Anp will be the next CSL as we take the Global stage $$$$$... it’s only a 12 month trial...