Anavex Life Sciences Corp (AVXL)

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I Retract Rett Trial Concerns. Murine Data Positive!

http://www.anavex.com/my_uploads/ANAVEX-2-73-as-a-Potential-Treatment-for-Rett-Syndrome.pdf

Fireman, thank you for bringing this Anavex presentation to my attention. I was negligent in not finding and studying it.

In short, it thoroughly affirms my earlier contention that the Anavex people must have had very positive treatment results with lab rodents for the treatment of Rett-like genetics and symptoms with Anavex 2-73.

In the Rett-like mice, as expected, the drug failed to provide complete relief from the many symptoms of the rodent Rett-like genetics. But relief was sufficient—if it occurs to the same degree in humans in the upcoming trial—that FDA approval appears absolutely certain.

The presentation, as it should, first noted the profound safety, lack of high-level adverse events, in the Australian human trial with Anavex 2-73. And no adverse events in the mice were noted or presented.

The positive outcomes are summarized on slide 30:

Administration of ANAVEX 2-73 results in both significant and dose related improvements in an array of behavioral paradigms in the MECP2 HET Rett syndrome disease model.

These behavioral paradigms measure different aspects of muscular coordination, balance, motor learning and muscular strengths, some of the core deficits observed in Rett syndrome.

The efficacy of ANAVEX 2-73 in additional different disease-relevant models — Infantile Spasms and Fragile X, Autism-related Disorders — in combination with existing clinical safety data supports exploration of ANAVEX 2-73 as a potential therapeutic in these disorders.

Clinical efficacy may also be evident in patients with CDKL5 mutations given that this gene has been implicated in both Rett Syndrome and X-linked Infantile Spasm Syndrome.

Clearly, the Anavex principals knew exactly what they were doing when they arranged for this new, 12-week trial of Anavex 2-73 for treatment of girls with Rett syndrome. The murine data, coupled with the now well-authenticated human safety profile of the drug, assures the highest probability of positive, symptom-suppressing results.

Those who reject model murine diseases as valid pre-tests for human efficacies, of course, will not be much impressed by the data in this presentation. Personally, I can discover no reason the mouse tests or results in this study were in any way flawed.

At the least, I now expect results from the Anavex Rett trial to be supportive of an FDA approval of Anavex 2-73 for Rett. That should happen even if it only moderately suppresses a few of the many Rett symptoms, inasmuch as there few or no other treatments presently available; the standard of care hurdle is minimal.

But, collectively, the murine results imply positive broad-spectrum results. For the girls suffering with this horrendous disease, we all hope for that comprehensive good outcome. Here, there is good evidence it can happen.