Anavex Life Sciences Corp (AVXL)

[falconer66a]

As expected.

The close alignment of the Anavex science and the actual, not conceptual or theoretical effects of blarcamesine in humans with various central nervous system (CNS) diseases continues. The new clinical results, from a very low dose (5mg) of blarcamesine for older girls with Rett syndrome, were exactly as I expected.

First, and for drugs treating CNS diseases, safety is so important. Blarcamesine once again, in real humans, yielded a very, very favorable safety profile. No report of severe or disqualifying adverse events ("side effects"). Drugs that mess with the endocrine system, disrupt brain chemistries, or affect genes or DNA have poor prognoses for clinical approval. In neither murines (lab rodents) nor humans have any of these appeared. Profound therapeutic safety, as shown in the new Rett data. So rare for drugs working in the CNS.

But most importantly, for older girls with Rett syndrome, blarcamesine works. Simply, in the trial every desired clinical endpoint was achieved. Alone, those data will mandate FDA approval. But approval is further prompted by the fact that no other drug comes close to providing the favorable clinical results of blarcamesine for non-pediatric Rett. Three conditions must be met for the FDA to approve a new drug:

1) It must be safe. Adverse events can't outweigh therapeutic results. With Rett, blarcamesine meets this criterion.

2) It must be effective, provide demonstrated, significant therapeutic outcomes. This criterion is met (as the new clinical results reveal).

3) It must match or exceed both the safety profiles and therapeutic efficacies of existing drugs. For non-pediatric Rett, there are none.

Let the reader determine, then, the regulatory fate of blarcamesine for the treatment of Rett syndrome in girls with this debilitating CNS disease.

Once again, I note that whenever blarcamesine has been administered to human beings with a targeted CNS disease, a) there have been no safety issues, and b) there have been measured, useful symptomatic improvements.

Biochemists focusing on the theoretical aspects of the new results must attempt to explain them, at the base molecular level. Once again, the unique mechanism(s) of action (MOA) of blarcamesine, facilitating optimal sigma-1 receptor protein function, is further substantiated. Becomes ever more difficult to negate this MOA as it would favorably apply to other CNS diseases (such as Parkinson's disease dementia, Alzheimer's disease, etc.) — all of the ones listed in the Anavex pipeline. To see them, scroll down on this: https://www.anavex.com/