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Friday, 06/30/2023 11:40:05 AM

Friday, June 30, 2023 11:40:05 AM

Post# of 464769
Several posters are asking why isn't Wall Street responding more to the recent OLE RETT PR claiming disease modifying effects. If the PR had swayed institutional or retail investors that the chance of Rett approval was increased based on the OLE, then the price should have gone up. I think CM was saving this PR in the wings (the 12 then 36 OLE data was complete a year ago, surely RSBQ results from 24 patients was available months ago) to release right before he would exercise his options in the hope there would be a little bump allowing him to keep a few thousand more shares in a tax neutral manner. --- still this quirk wouldn't prevent WS from buying if the data was convincing.

As usual, Anavex released an incomplete picture. What are the actual RSBQ numbers? What exact endpoints, what statistical measures, one sided or two-sided? How was missing data imputed? Why just p values? It is not hard a table of treated vs placebo at 0 and 2 weeks and OLE at 24 and 48 (maybe 36). 10 minutes with Excel. Also, RSBQ can't measure disease progression. Claims of disease modification are exaggerated. Indeed, pediatric patients average a higher RSBQ that adult patients - let that sink in (links below) . Why would one expect any population mean change over 12 weeks or over 4 to 48 weeks. The cohorts are small and likely distinct. Weirdly, in the 2a, the same placebo patients gained points. Usually for a scale not changing over time like the RSBQ, placebo patients move in the same direction as treated patients, yet this cohort lost ground in 12 weeks on placebo.

These incomplete data releases and overhyped claims do not inspire confidence. Dr. Kun seemed to have no influence on the quality of this PR

I'll wait for Excellence and keep my unchanged small position anticipating a 20-25% likelihood that data is good enough for an NDA.



https://pubmed.ncbi.nlm.nih.gov/37104862/
https://www.aaidd.org/docs/default-source/prepressarticles/rett-syndrome-behaviour-questionnaire-psychometric-characterization-and-revised-factor-structuredcc25eae9e8c6329b425ff0000b6faa6.pdf?sfvrsn=bd4e3d21_0
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