I’m happy with the primary outcome endpoints agreed with the FDA...

I’m happy with the primary outcome endpoints agreed with the FDA and I suspect that Neuren will be happy as well.

There will be co-primary endpoints, as was also used in the registrational trial for trofinetide in Rett, and as in that trial, one of the endpoints will be assessed by physicians and the other by caregivers.

The physician-rated PMSA-C endpoint (previously referred to as the CGI-I) was probably the standout result in the Phase 2 PMS trial,  with improvement seen in 16 out of 18 patients and a mean score of 2.4 (Wilcoxon signed rank test p<0.0001).




Details of the VABS-3 Receptive-Raw Score endpoint results in the Phase 2 PMS trial I don’t think have been previously provided. However, the fact that the greatest area of improvement seen by caregivers was in communication is clear from the graph below.





We now know that excellent results were seen on the VABS-3 Receptive-Raw Score in the Phase 2 PMS trial– improvement was seen in 16 out of 18 patients and there was a mean improvement of 7.5 from a mean baseline of 29.0 (Wilcoxon signed rank test p=0.0001).

Improved communication is not only one of the most desired areas for improvement nominated by caregivers for all of these neurodevelopmental disorders, it is also, happily, the area in which both trofinetide and NNZ-2591 have demonstrated the greatest impact.