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08:13
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Originally posted by ANPto2Dollars:
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An interesting point from this Parent Project DMD video with Capricor from last year. Capricor's CEO said that the FDA viewed their Phase 2 data as interesting, but that they really needed to show in a Phase 3 what is the clinical meaninfulness of a PUL2.0 score, what does 1 or to points on a PUL score actually mean in terms of clinical meaningfulness. So PER might be able to "free-ride" on the back of CAP's data, since CAP will help show to the FDA why PUL scores are clinically meaningful! "We are open to continuing our dialogue with FDA in whatever form that takes. FDA told us on the review of the Hope-2 data that they thought that the data was very exciting, specifically the cardiac implications as well as the skeletal muscle. There's very few if any studies that have shown similar efficacy in DMD, but they really wanted to see a phase 3 trial, and their focus, just so people understand is, that we need to accept the fact that the PUL scores are very relevant, but now we have to tie them to clinical meaningfulness — what does it really mean, what does one PUL point mean? Two PUL points? And fortunately, you know, we think we're going to be able to demonstrate that very cleanly once they are comfortable with the fact that there's clinical meaningfulness, we feel that there's a very clear path to a BLA here." So what do we learn from this statement? That the FDA was "excited" by Capricor's Phase 2 results (our 6 month data is even better than CAP's!) and that CAP's Phase 3 study will help demonstrate to the FDA the clinical meaningfulness of PUL2.0 scores — which is of course our ATL1102's primary endpoint too. So CAP is helping to do our work for us on demonstrating the clinical meaningfulness of PUL scores, and if the FDA views CAP's results as "exciting," if we replicate our Phase 2A results and CAP replicates their phase 2 results in their phase 3 (i.e. ours are better than CAP's) — then there is a chance that the FDA is even more excited by our results than theirs. (Of course, there is the caveat that CAP's data is over a longer period than ours).VIDEO
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And we should remember Caps ph2 trial had just 8 boys on Cap, and 12 on placebo. 20 total.
(20min delay)