Anavex Life Sciences Corp (AVXL)

[Doc328]

This paper goes over the thinking used to design the Exellence Rett study based on the good data from the small US Rett trial.

The new big is small: Leveraging knowledge from small trials for rare disease drug development: Blarcamesine for Rett syndrome
https://bpspubs.onlinelibrary.wiley.com/doi/full/10.1111/bcp.15843

They assumed that results from week 7 to week 12 would simply be extending the line from week 0 to week 7 out to week 12. Also that 30 mg would naturally be better than 5 mg (both of these in figure 6) The problem was only partially the initial design but the stupidity not to significantly adjust the N up after the failed Avatar trial (yes a fail using the accepted endpoints) is 100% on Missling. 12 weeks was not better than 7, 30 mg was not better than 5mg. The paper showed a great knowledge of Bayesian statistics to design the initial trial --- only problem is they did not form a new posterior hypothesis by adjusting the prior hypothesis after the Avatar data was available and increasing N to 180 or so. The study was also hampered by the 2:1 randomization. Maybe the new Big is still the old Big.

Ene Ette's resume differs from Dr. Jin but both are expert clinical trial statistician

http://www.anoixiscorp.com/about/ette-ene.htm