Making it pivotal is a big deal and changing the primary outcomes is also a big deal.
The primary end point is Drug exposure-dependent response of the Rett Syndrome Behaviour Questionnaire (RSBQ) Total score. That is a behavioral description change.
The second one is Incidence of Adverse Events.
The secondary endpoint contain what may be considered the usual P2 PK measures and two more behavioral related scales.
There is nothing that could be considered biomarkers.
Appreciate your comments, too! Sometimes I skim over your posts and think that they are just "promos", but you definitely have a clear understanding of the science, etc.