Ann: Trading Halt, page-100

As we prepare for Phase 2 results on Monday, a reminder that these are the mouse model results for Pitt Hopkins:



The left-hand column is the wild type (i.e. typical) mice. The second column is the knockout (i.e. Pitt Hopkins) mice with no treatment. The third column is the wild type mice with NNZ-2591. Most importantly, the final column is the knockout mice with NNZ-2591. One can observe that the knockout mice with treatment tend towards the wild type mice in each of the above characteristics.

Those results are similar to the Phelan-McDermid mouse model:


We now have an affirmation of the Phelan-McDermid results in the form of positive safety and efficacy data from a Phase 2 human study.

Finally, the Angelman and Prader-Willi mouse model results for NNZ-2591 are similar again:





Exciting stuff.