Anavex Life Sciences Corp (AVXL)

[sokol]

Nidan:


What is Real-World data?:

Real world data (RWD) in medicine is data derived from a number of sources that are associated with outcomes in a heterogeneous patient population in real-world settings, such as patient surveys, clinical trials, and observational cohort studies. Real-world data refer to observational data as opposed to data gathered in an experimental setting such as a randomized controlled trial (RCT). They are derived from electronic health records (EHRs), claims and billing activities, product and disease registries. Real world data includes evidence, real world evidence, gathered by Anavex over many months - 104 + months.

If I correctly understand the title of Anavex’s presentation, what Anavex and its team has done is to compare 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.

So, you may also want to know what propensity corrected patients means. 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/

Of course we are familiar with the ADNI:

“The Alzheimer’s Disease Neuroimaging Initiative (ADNI) unites researchers with study data as they work to define the progression of Alzheimer’s disease (AD). ADNI researchers collect, validate and utilize data, including MRI and PET images, genetics, cognitive tests, CSF and blood biomarkers as predictors of the disease. Study resources and data from the North American ADNI study are available through this website, including Alzheimer’s disease patients, mild cognitive impairment subjects, and elderly controls.”

http://adni.loni.usc.edu/

The Anavex presentation is 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)
timepoint
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

In conclusion, I surmise that this presentation late Friday of this week is about a study/analysis Anavex did with Ariana, et al. using data from its clinical trials over 104 months and comparing that with data from ADNI to mimic a randomized trial - a virtual randomized trial. Of course, as you know, Anavex is also in the process of conducting actual randomized clinical trials with AVXL 2-73 as we speak, and I think it is important to wait and see what those trials reveal.