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Re: Investor2014 post# 430049

Tuesday, 09/12/2023 11:24:19 AM

Tuesday, September 12, 2023 11:24:19 AM

Post# of 473297
Indeed. That's why the FDA always prefers co-primary endpoints unless there is a bio-marker. The single RSBQ endpoint anchored on CGI-I is not going to be accepted by the FDA as p3.

I have been looking into the correlation between n, effective size, and p.
Trofinetide n=187, ES~=0.4, p=0.0175
RS-001 n=25, ES=0.5, p=0.01 (dose=5ml)
RS-002 n=33, ES=1.9, p>0.5 when AUC and CGI-I anchor are not used (dose=30ml)
So dose-dependent response shows up in ES but p-value changed to insignificant even ES has increased 4x.
To increase the study power, the company has increased the RS-003 n from 60 to 92, 3x RS-002 sample size with the same dosing. The question is what's the correlation between n and p? It's not simply linear.
RS-003 n=92, ES=1.9 (assume the same), p??
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