Phelan-McDermid Syndrome, page-72

Agent- 86

Your guess on this is as good as mine because I really don’t know.

All I can offer is a couple of observations.

Firstly, as I think I’ve said before, if NNZ-2591 is successful in Phase 2 PMD, I think it unlikely that Neuren will be running a Phase 3 trial in PMD or any other indication. My feeling is that, whether we like it or not, it will be Acadia running the show.

That said, I believe that Neuren will continue to work towards making NNZ-2591 a Phase 3-ready asset by completing all the necessary manufacturing requirements (very important step) and meeting with the FDA (and possibly EMA) in a post-Phase 2 meeting to ascertain what the FDA’s pivotal trial requirements are. With respect to trial design, I think that any potential acquirer would prefer to control that.

The clinical trials website suggests that there has been just one Phase 3 trial commenced in PMD, which is still in progress – and that’s a randomized, double-blind placebo-controlled 12 week treatment trial in 7-18 yr olds with PMD and ASD, using a pre-existing drug (lithium). There appears to be just one recruitment site in Paris, and they are aiming to enrol just 22 patients and expect that to take 2 years.

Other current Phase 2 trials in PMD are small (in a couple of cases, just 6 participants) and recruiting at just one or two sites.

However, I’d expect any Phase 3 trial of NNZ-2591 in PMD to be considerably larger than the current Phase 2, where all 20 participants are receiving the drug.