Anavex Life Sciences Corp (AVXL)

[JB3729]

For those that haven't read this and could possibly be swayed by BS posted on this message board -

From IR -

In terms of the number of participants in the Phase 2a trial, this is because of the adaptive design of the trial (versus a trial that includes patients receiving a placebo). Here is a summary of comments made by Dr. Tasos Zografidis, the Vice President Clinical Operations of Anavex, and Dr. Christopher Missling, President and CEO, in a conference call earlier this year, which provides additional insight into the reasons Anavex is using an adaptive trial design.

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TZ: For background information, there are two approaches in a clinical trial. One is the placebo controlled study. Here you have to estimate the number of patients needed if you are testing a hypothesis since you need a certain power; you need a certain number of patients. However, the methodology that we are using is very different, we just measure whatever effect is there. If you measure the effect and are able to reproduce it mathematically (mathematical modeling), then you can calculate the number of patients needed so as to see the effect. By using the adaptive trial methodology with population PK modeling, we are implementing mathematical modeling along with probability density function statistical methodology. That means that the results are looked at in confidence intervals. In the first approach you first estimate the number of patients and then you "see" the effect. In the second approach you first measure the effect and then you calculate the patients needed so as to truly "see" the effect. So, these are two opposite approaches with same outcome.


CM: We want to get a signal and the adaptive clinical trial allows us to increase the dose or decrease the dose until we see a signal. The mathematical model that Tasos speaks of sufficiently allows us to measure the data from these 32 patients because we are measuring many data points in this trial, from blood samples and the various cognitive tests, which allows for sufficiently precise data to learn how the drug works in order to select the number of patients needed for a Phase 3 pivotal trial. The number could range anywhere from 100 to 300 and the goal is to figure that number out.

The key is to understand the difference between a statistical standard trial, which is based on the placebo versus active dose in a number of patients, and the adaptive trial design statistical method used. The FDA has written a very eloquent guideline which you can find on the fda.gov website (http://www.fda.gov/downloads/Drugs/Guidances/ucm201790.pdf). The FDA states that with the adaptive trial design, which we are using, you need fewer patients, you are able to do this in a shorter period of time, you are more likely to demonstrate the effect of the drug, and you are getting more information on the treatment effect as well as a better understanding of dose response information in subgroups. Lastly, you are able to identify and optimize the parameters needed for Phase 3 effects. So, this Phase 2a trial is a very important stepping stone and building block for a successful Phase 3 trial. We want to avoid the same failure that the other Alzheimer drug trials experienced, and by implementing a different, innovative trial design for ANAVEX-73 in Alzheimer’s treatment, we are leading a more efficient study than a conventional study.