The delay is in agreement on the Phase 3 trial primary endpoint.
It is acknowledged by both industry and the FDA that endpoint design selection for novel rare diseases is challenged by factors such as lack of regulatory precedence, small trial populations and an often-limited understanding of the disease’s natural history.
The FDA’s view on rare disease endpoints is that it wants to see “sensitive endpoints with as little variability as possible…clinically meaningful and measurable endpoints.” *
Neuren’s job will be to convince the FDA that the novel primary endpoint it is proposing for the pivotal PMS trial meets those criteria. How long that will take, I have no idea.
As for the new indications, Jon has said in the past that they wouldn’t be announced until just before Phase 2 trials are ready to commence. I suspect that no new Phase 2 trials will be starting until the Phase 3 PMS trial is sorted.
*
https://www.biospace.com/fda-launches-pilot-program-to-develop-better-rare-disease-endpoints
(20min delay)