I spoke to Jon Pilcher regarding RSBQ and this is his...

I spoke to Jon Pilcher regarding RSBQ and this is his response:

Everyone seems to be fixating on the subscales, none of which were an endpoint in the clinical trials. The co-primary endpoint was the Total score. If you read the whole manuscript, the concluding paragraph of the Discussion states "In summary the analyses presented here support the use of the RSBQ in children and adults with RTT". It also states that the Total scale has "good psychometric properties". So the manuscript is actually supportive, not critical. It recommends some reorganisation of the subscales, but that doesn't impact the Total scale. Neither the manuscript nor the E&P report says anything about Acadia submitting or having to submit any new analysis - someone has just made that up.

As a reminder, at Neuren's End of Phase 2 Meeting with FDA the use of RSBQ Total and CGI-I as co-primary endpoints for the Phase 3 trial was agreed. The elevation of CGI-I to co-primary (a higher bar) was a specific request from FDA in order that a positive CGI-I result would provide corroboration of the clinical meaningfulness of a positive RSBQ result, recognising that RSBQ had not been used in a registration trial before and was not specifically designed for interventional trials.

Regards, Jon