Replies to post #469449 on 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

[abew4me]

Hoskuld...You asked me: What would lead you to believe that Dr. Kun Jin designed the P2 schizo trial using 3-71?

Very simple. He is probably the most experienced person on their staff...and he has successfully designed other trials. (See below)

"During his [25 years] tenure at the agency, Dr. Jin has contributed extensively to statistical review issues and trial designs surrounding the regulatory approval of drugs for the treatment of neurological diseases including Alzheimer’s disease, Parkinson’s disease, migraine, epilepsy, and multiple sclerosis, as well as rare diseases, such as ALS and DMD."

So, let me ask you a question. Why would you conclude that "he totally was not "the one that designed the trial"?

Additionally, you were incorrect to insinuate that Dr. Kun Jin is somehow responsible for designing the failed P3 Rett trial for pediatrics. Why would you insinuate that?