Anavex Life Sciences Corp (AVXL)

[falconer66a]

I'm in because blarcamesine works.

What drives your confidence in absolute success of Avxl?

A crucial, seminal question for actual or potential AVXL shareholders. Anavex Life Sciences Corp is a start-up biotechnology firm, with no sales revenues, attempting to advance and bring to market several new, drugs, which have heretofore never been seen, used, or understood. Moreover, the Anavex drugs are targeted at recalcitrant central nervous system diseases.

With all of that, the chances of success for the Anavex drugs would appear quite dismal. For example, Anavex, with their drug blarcamesine (Anavex 2-73), is attempting to prove, in clinical trials, that it can effectively treat, of all things, Alzheimer's disease. But, existing major drug companies in the last two decades have spent billions of dollars trying to do this, to treat Alzheimer's and each has failed. How, then, might this tiny drug start-up company be able to beat Alzheimer's? Doesn't seem possible at all.

Well, not until the unique, propitious biochemistry of blarcamesine is understood. Indeed, nothing else is like it. Simply, the evidence, both in murines (lab rodents) and early data in real humans, show that blarcamesine really does fix pathological anomalies in diseased or malfunctioning neurons, including those as the venue for Alzheimer's.

Detailing how this happens would require several days of itemized explanations of neuron physiology, biochemistry, and structure, telling how blarcamesine uniquely binds to sigma-1 receptor proteins, which can then direct facilitated homeostasis (cell maintenance feedback loops), restore autophagy (internal consumption and recycling of chemical wastes), and most significantly, restore proper protein folding. In the simplest perspective, Alzheimer's is caused by agglomerations of waste proteins, which are no longer removed in either neurons or nerves. That's because the enzymes and reaction sequences that normally do this fail because the functioning enzymes in this waste-clearing process are mis-folded; poorly synthesized. Equivalent to trying to open a lock or start a car with a bent, "misshaped" key. Straighten the key and the lock can be opened. Properly fold the waste-clearing enzymes, and tau and beta-amyloid proteins causing Alzheimer's won't accumulate.

That's the briefest summary of blarcamesine's mechanism of action (MOA) in Alzheimer's. It is also being targeted at a genetic disease in young girls, Rett syndrome. Early, preliminary clinical data show profound good results. In summary, just as it was done in transgenic mice (with human Rett syndrome genes and symptoms) the drug in humans with Rett syndrome has markedly reduced glutamate levels, and conversely and propitiously restored toward normal levels concentrations of gamma-amino butyric acid (GABA). In mice with Rett syndrome symptoms, the drug essentially terminated the disease's symptoms. An early safety and tolerability study of the drug in a few girls with Rett syndrome likewise exhibited wonderful outcomes. A large clinical study in humans is underway, with results to appear sometime this year.

Why have I, a small retail stock investor purchased over several years a considerable number of AVXL shares? Well, I'm a biologist; I understand the deep science involved with blarcamesine. I've reviewed the dozens of papers of studies on the drug, both in preclinical murine studies, and in recent years, the early studies in real humans. For me, the key is this. Blarcamesine has two things going for it. First, it has proven to be exceptionally safe; no disqualifying side effects of any sort. Very unusual for drugs acting in the central nervous system. Secondly, in no trial of the drug for any of the several diseases being targeted has it failed to produce favorable therapeutic outcomes. In both mice and men the drug has worked, safely; at low doses, taken orally.

Therefore, I am confident that the clinical outcomes of the on-going blarcamesine human trials will be universally positive; presently for Rett syndrome and Alzheimer's, but followed by Parkinson's disease dementia and the several other diseases eventually being targeted by the company.

Check back in five years. By then, Anavex Life Sciences Corp will be a major global pharmaceutical, marketing a few drugs that will transform 21st-century medical practice. Until then, either take a moderate AVXL position (use only discretionary funds), or just sit back and watch the story continue to develop. In any case, it will be interesting.