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Investor2014

02/11/24 2:42 AM

#451634 RE: Steady_T #451629

I agree.

To use your argument, the heightened expectations of the placebo group should be the same for the treatment group.


And I am not forgetting that placebo also applies in the drug arm, a concept many seem to struggle with. Placebo effect linked to subjective score giver expectations and wishes bias is a likely reason that both arms had much better absolute scores than trofinetide.

The variability (up to 300% in RSBQ per Missling and the papers he quotes) coupled with Kaufmann's proposed baseline imbalance in the placebo arm and 2:1 drug/placebo arm size, altogether makes the EXCELLENCE, and the other two even smaller Anevex Rett trials, at best inconclusive at this point.

We agree that biomarkers, although not accepted standard ones in Rett, could shed more light on what is going on.

My chances of approval from such a deep analysis exercise have to remain low. Here again as with EMA and AD, I won't be into assigning percentages before we get some more Bayesian inference update points.