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Investor2014

01/03/24 12:34 PM

#445657 RE: bas2020 #445653

For one reason because both the subject RSBQ and to a lesser degree CGI-I scores are notoriously unreliable.

It is what it is and could likely have been dealt with by a larger and longer trial.

As I have mentioned positive biomarker correlations to the dose arm could be helpful also from the OLE and compassionate patients having been on drug for longer.

We will hopefully get some information on that at some point.

boi568

01/03/24 12:52 PM

#445662 RE: bas2020 #445653

Daybue reported -5.1 treated and -1.7 placebo. That's a 3.4 delta versus our 4.6 delta. They got to stat sig with a much lower dispersion around the mean.

I think their placebo rate was minimized because their trial was effectively unblinded; if your kid wasn't getting diarrhea, you knew she was on the placebo. Conversely, in our trial you knew your chances of your daughter being on the drug was 2 out of 3 due to the trial design (even when she wasn't).

So their placebo effect was low and ours was high. Our placebo effect built up both arms of the trial, as always, inflating to the -12.9, and certainly relative to Daybue.