Anavex Life Sciences Corp (AVXL)

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Things to Know

A Promising New Alzheimer’s Treatment Drug

The Problem. Alzheimer’s disease debilitates and kills ever-increasing numbers of aging Americans. The Alzheimer’s Foundation of America estimates that as many as 5.1 million Americans may have the disease, and the number is increasing with our large, aging population. The disease is devastating to both its victims and their care givers. Treatments and care-giving costs are massive, stretching collectively into the many billions of dollars.

Treatments. Effectively, there are none presently. A few, very expensive drugs can temporarily, for just a few weeks or months, merely slow the aggravating progression of symptoms. No treatment drug or protocol either stops or cures the inevitable progression to profound disability and ultimate death from the disease. A diagnosis of Alzheimer’s disease is a curse for which modern medicine offers no hope.

Treatment Research. The cause of Alzheimer’s symptoms is almost universally perceived to be the accumulation in or near nerve cells (neurons) of two waste proteins, agglomerations of beta-amyloid plaque, and neurofibrillary tangles, insoluble twisted fibers composed largely of the protein tau, composing the tau tangles.

Current research has strived to remove these neuron wastes that complicate normal brain nerve functions. Plainly, and tragically, all of these efforts have failed. The research drugs targeted at waste proteins have either failed to reduce their presence, or have concomitantly caused serious, even lethal side effects.

The other approach has been to prompt the immune system to chemically destroy and remove the waste proteins. These efforts, too, have failed in every case.

Until now, there have been no drugs that have demonstrated any safe, effective potential to successfully treat Alzheimer’s in any useful way. The disease continues to be an un-treatable curse.

Until now.

A Successful New Drug in Development. A different treatment approach is required. Instead to attempting to pharmaceutically remove beta-amyloid plaques and tau tangles, one company’s researchers have intelligently targeted — successfully — the root cause of those waste protein agglomerations. Instead of attempting to remove them after their formation, after they persist and continue to cause nerve dysfunction, the new approach is to keep them from ever forming, or facilitate their removal by natural, innate cellular processes (as occurs in normal, non-diseased neurons). This approach will revolutionize Alzheimer’s treatment (along with a number of other neurodegenerative diseases).

Children and young adults virtually never get Alzheimer’s. It appears in middle to older aged individuals; after earlier years of mental normalcy. With age, neurons in many people fail to work normally. The new drug restores youthful “homeostasis,” consistent, normal cell status or function, in a powerfully unique and successful way.

The proper function of two structures, organelles, in the neuron are essential. Mitochondria are tiny cellular structures that extract energy from digested food molecules and store that energy in adenosine triphosphate, ATP. ATP is then used in virtually all cellular reactions and processes to power them. ATP is the cell’s energy resource. Without adequate ATP, cellular processes are diminished or inhibited, and disease ensues.

The other essential organelle is the endoplasmic reticulum. Endoplasmic reticula (among other things) take ATP from adjacent, connected mitochondria and use its energy to properly fold and lock together complex proteins, primarily essential enzymes. Virtually all cellular chemistry is mediated by controlling enzymes, much as a key unlocks a lock. If a key, in this case an enzyme in a neuron, is bent or misfolded, the lock, or the life-supporting chemical reaction cannot occur.

In most neurodegenerative conditions, especially Alzheimer’s, endoplamic reticula become separated from the mitochondria. They no longer can receive sufficient ATP to properly fold proteins into normal reaction-controlling enzymes. The cell fails to function properly, or even dies (as in the case of advanced Alzheimer’s neurons).

The solution? Find a molecule that re-connects and maintains the mitochondrion-endoplasmic reticulum connection. Keep the two organelles connected, so proper, healthful enzymes can be normally synthesized — as was the case before Alzheimer’s struck.

The Revolutionizing Molecule. Wonderfully, such a molecule has been found, and has been tested in early trials on humans, with extremely positive results. The molecule is Anavex 2-73, a patented proprietary chemical of Anavex Life Sciences Corp. (AVXL:NASDAQ).

Early clinical trials of Anavex 2-73 have established the following facts.

1. Few or No Side Effects. After a year of administration to people with mid to moderate levels of Alzheimer’s, no serious, debilitating, or disqualifying side effects appeared. For drugs treating nerve or brain conditions, this is exceptionally rare.

2. Stops or Improves Alzheimer’s Symptoms. Remarkably, Anavex 2-73 in dosing ranges between 10 to 50 mg (very small amounts) in a one-year clinical trial period, either a) stopped the progression of symptoms, keeping them at the initial dosing level; or b) remarkably reversed the severity of symptoms, trending at the end of the 52-week trial toward mental and functional normalcy.

Larger, substantiating clinical trials of Anavex 2-73 for Alzheimer’s should be forthcoming. But preliminary lab data in animals, followed by the profound findings of the recently-reported clinical trial in humans are revolutionary. Now, there is profound hope for a) successful treatment of Alzheimer’s, and b) termination of the progression of the disease at its earliest, mildest degrees.

The Future. Anavex 2-73 holds the promise of solving the expanding Alzheimer’s problem, first in America, and then in the rest of the world. The sooner Food and Drug Administration (FDA) approval is attained for this safe and effective drug, the sooner millions will no longer be medically and financially ruined by this frightful disease.

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Study Data. Scrutinize the clinical results of an early 52-week trial of Anavex 2-73 here:
http://www.anavex.com/wp-content/uploads/CTAD-Anavex-December-2016-2.pdf

Notice the minimal and insignificant adverse events (side effects) on page 18.

The data plot on page 25 (the yellow doted line) shows cognition results.

Page 33 enumerates the positive results of Anavex 2-73