Phelan-McDermid Syndrome, page-95

Mateo, if you have a science background, you’re actually a step ahead of me!

I understand your concern about getting the dosing level right for strong efficacy results.
There are two things that might allay your concern.

Firstly, as Guzz said, the disappointment of trofinetide in TBI, in which a far too low dose was used, isn’t something that Neuren will quickly forget. Following this failure, I would expect them to be acutely aware of getting subsequent trial dosing right.

Secondly, if you haven’t looked at it already, I suggest you check out the NEU announcement of 6 March 2020. A dose-ranging study in a PMS mouse model was carried out over 6 weeks, testing 4 doses. Graphs are provided to show the results. The same optimum dose was identified in similar dose-ranging mouse model studies in Angelman and Pitt Hopkins.

Bottom line?

This clearly demonstrates that the 4x mg/kg dose was the optimum dose level in the mouse model. Comparison with pk data from Neuren’s planned Phase 1 clinical trial will inform the equivalent human dose for the planned Phase 2 trials in patients.