Neuren - where to from here ?, page-75

I wholeheartedly agree with this and am on record for stating it previously. Hashan uses 30% chance of NNZ-2591 passing a phase 3 trial in PMS I would argue this is a gross underestimate.

Consider the following all of which has been posted/discussed previously:

1. Excellent mouse models generated by single gene knockout.
2. Efficacy in mice demonstrated across all domains with crystal clear dose- response relationship.
3. CNS anatomical changes demonstrated in knockout mice after treatment with normalisation of the overactive dendrite/synapse pattern of the PMS mice.
4. Outstanding phase 2 result in only trial in humans with the condition, with 16 of 18 subjects demonstrating improvement half of these significant improvement after only 13 weeks of treatment.
5. Results surprised the company on the upside described by the usually understated JP as a "Wow result".
6. Low side effect profile.
7. Clear path to commercial success demonstrated very recently by trofinetide.

When comparing to trofinetide in Rett, I would argue that NNZ-2591 has surpassed it on every single metric so far.

When the phase 2 results were announced I ran the available numbers past a few acquaintances in the biostatistical world with brains much larger than mine asking their opinions on likely phase 3 success. With a couple of sensible assumptions and caveats their numbers were MUCH higher than 30% POS.

(This is obviously all my own opinion and I may be completely wrong).