FDA and Neu have had their meeting and confirmed primary endpoints for a phase 3 trial

2A1591174_NEU.pdf

Nice to get some positive news. There was some concern that the FDA cuts would cause a delay. Clearly it hasn't.

No surprises re the trial protocol. All seems reasonable to me. It will be interesting to see how many patients they need to enrol. For Daybue it was 187

It is kind of interesting for me, at least, to compare with Opthea, which I got badly burnt by. Opthea had a phase 2b trial involving 366 patients, randomised and blinded, showing strongly positive results. The two phase 3 trials completely crashed and burned, showing the drug basically doesn't work.

NNZ-2591 did a phase 2 trial for Phelan-McDermid syndrome. It had a total of 18 patients, open label. No control group, no randomization, no blinding. There were similar small trials for Pitt-Hopkins and Angelman syndromes. Results were based on questionaires completed by caregivers and clinicians. Huge opportunity for bias and placebo effect. Everyone is desperate for anything to help these children, who currently have no effective treatments at all. Obviously people are primed to look for positive improvements. Best to take these results with a MASSIVE degree of scepticism IMO

So I'm treating NNZ-2591 as nothing more than a lottery ticket at this stage. Fortunately, as others have said, the company is is probably around, or even below fair value based on Daybue alone.

The phase 3 trial is expected to start around "mid-year 2025"