Anavex Life Sciences Corp (AVXL)

[sokol]

Yes Xena, and please note this from the article: “In these programs drugs for serious illnesses are rapidly approved on the basis of limited clinical trial data or data reliant on surrogate outcome measures ...... rather than clinical.”

And, consider the upcoming presentation by Anavex at CTAD on Friday, December 6 at 5:30 p.m. entitled: “LB19 - Novel analytics framework for augmenting single-arm Phase 2a open label trials with Real-World external control data: Application to the Blarcamesine (ANAVEX®2-73) study in Alzheimer’s disease matched with propensity corrected patients from Alzheimer’s Disease Neuroimaging Initiative (ADNI) exploring treatment effect on cognition at Interim two-year (104-Week) time-point.” To be presented by Mohammad AFSHAR (1), Coralie WILLIAMS (1), Nanthara SRITHARAN (1), Frederic PARMENTIER (1), Federico GOODSAID (2), Christopher MISSLING (3)
(1) Ariana Pharma, France, (2) Regulatory Pathfinders, United States, (3) Anavex, United States.

The terms used in describing this presentation such as “Real-World external control data”, “propensity corrected patients” may be revealing. For example, see this article:

An Introduction to Propensity Score Methods for Reducing the Effects of Confounding in Observational Studies
- Peter C. Austin

“ The propensity score is the probability of treatment assignment conditional on observed baseline characteristics. The propensity score allows one to design and analyze an observational (nonrandomized) study so that it mimics some of the particular characteristics of a randomized controlled trial. In particular, the propensity score is a balancing score: conditional on the propensity score, the distribution of observed baseline covariates will be similar between treated and untreated subjects. I describe 4 different propensity score methods: matching on the propensity score, stratification on the propensity score, inverse probability of treatment weighting using the propensity score, and covariate adjustment using the propensity score. I describe balance diagnostics for examining whether the propensity score model has been adequately specified.”

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3144483/

I believe it may be that Anavex and its team has compared the Blarcamesine treated patients in their 104 week study to propensity corrected patients from the ADNI that were not treated with Blarcamesine to mimic a randomized study. In other words, they used information from the ADNI database to find nontreated patients (AD patients that were not teated with Blarcamesine) that were otherwise comparable to patients that were administered Blarcamesine for 104 weeks. And, I assume that the presentation will show how comparable non-treated propensity corrected patients from the ADNI database compare to treated patients in the 104 week Blarcamesine study - a surrogate outcome measure so to speak that mimics a randomized trial.

Of course, Anavex is also conducting randomized clinical trials of AVXL 2-73 as we all know, and it is important that those clinical trials are demonstrating positive results. Hopefully, the ongoing clinical trial results compares favorably with what Anavex will reveal at the CTAD presentation this coming Friday in San Diego at 5:30 p.m. - 8:30 p.m. E.T. - 7:30 p.m. CST - 6:30 p.m M.T. However, I doubt that we will have much in the way of information about the ongoing clinical trials at the time of this CTAD presentation. In any event, it will be interesting to see what it is that Anavex presents at CTAD 2019.