Ann: Trofinetide successful in Phase 2 trial in Fragile X, page-27

Stockstoker if your asking whether anyone has the pre-specified statistical analysis plan I think you going to draw a big zero.

So until NEU publishes these results (if they do) I suspect the announcement today is as good as you are going to get.

And as with all P2 exploratory results from micro-caps the results are very much in the eye of the beholder.

On the face of it the ABC results are stronger than I expected and the ABC is as close to a gold standard as you will get.

But because the scoring is modified (using the new Sansone method) it is very difficult to translate these results back to a common metric for the ABC.

In theory the new scoring should result in better sensitivity to change but quite what this means in terms of what would constitute a clinically significant level of change is anyones guess.

You would assume that any type of correction for multiple testing would wipe out the statistical significance of any of the individual tests. And this is the reason that Ns and p values are not provided for the individual tests. In its place NEU continue with this idea of a pattern of results permutation testing idea.

The clinical global improvement scale is probably the key here. And as in Retts this looks pretty intransigent. So you do start to wonder about the strength of the blinding leading to these differences in measures by reporter.

But as I say … eye of the beholder type of thing. The clincher I think will whether the results are intended to be sent off to peer review and published.