Because someone contacted me to ask this question ...he (Missling) is talking about the Neuren trial which is already been P3 approved by FDA and partnered up on. It's a 180 patient trial which the FDA apparently has told them if it met with the same stat significance as their P2, they will be able to file for their NDA out of their P3 trial which starts later this year with their partner. I believe they are using at least one of the metrics that Missling is referring to. Neuren is already well past the point of worrying about what age patients they can treat with the P2s they have run. Maybe Kauffman helped them to design their P3 trial as well and is now helping Anavex do the same? Either way just posting a link where you can find my take on that info. There may be more recent. I don't know. Just passing along. I don't have a dog in the fight either way or either company but seems they are well ahead of anavex in their clinical trials and partnerships. Doesn't mean they have the answer but they do have stat sig in P2 trials.
I think Missling may be saying that the current Rett trial is designed with a P3 in mind with regards to what the regulator will accept as endpoints for approval.
Missling may be saying that since Kaufmann is the recognised specialist in Rett and he is now the CMO of Anavex, the 'authoritative' measure to date is the RSBQ. Hence RSBQ also figures as the first of the secondary efficacy endpoints of the initial P2 safety trial, which moves up to be the primary endpoint in the P3.
And yes the Neuren P3 trial mesage from Acadia says: "The study will use the Rett Syndrome Behaviour Questionnaire (RSBQ)".
More speculation is that Anavex is skirting the juveniles both in the current P2 and in the following P3 to get A2-73 approved and take it from there.
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