Sarepta FDA Hearing, page-43

I have just been rereading some previous announcements looking from a viewpoint that the FDA was looking at Sarepta.

Their main concern was finding evidence that the Sarepta drug worked, and some proof of how it worked.

Look the ANP announcements, and at where pretrial ground work has been done. We have biomarkers that we now have patented evidence that ATL1102 acts upon. We have the Extra research from the combination trial investigating the mechanisms of action. We have results from the plasma study showing increased bone density, with the proteind responsible identified. We have PUL2 results from non ambulant boys that have never been seen before. We have MRI results showing a reduction of fatty tissue in muscle mass.

So not only will ANP be able to show regulatory bodies trial results. But they will also be able to prove that ATL1102 was responsible and how it works.

It does look like all the ducks are in a very neat row.

Reread the last quarterly. Have a look at the bit about the combo study again. Particularly the bit about how much dystophin was found in the mouse muscle.

Go way back and gave a look at the MS trial results. Once the tox study is done that can progress.

Lots of news due between now and the end of year.