License or Buyout?, page-27

Thanks for the great commentary everyone.

I think Neuren’s best chance at Breakthrough Therapy Designation will be for TBI:
-the phase 2 trial has clear clinically significant endpoints, being reduced incidence of adverse events and serious adverse events; and
-the trial size (260 subjects) lends itself to the statistical power the FDA generally likes to see.

If the phase ii trial in TBI is successful, I think we will have a reasonable chance of obtaining BTD. However I can’t see us getting BTD for Fragile X at this stage, given the phase 2 trial design was very similar to Rett…although I would love to be proved wrong.