Anavex Life Sciences Corp (AVXL)

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My Reasons

What odds are you giving for successful phase 2b/3 AD?

Here’s my bet — and why I think it’s valid.

Chances that the top line data at the end of the long, big, valid Phase 3 Alzheimer’s clinical trial of blarcamesine will be “successful?” 100% How so?

The first thing that disqualifies a proposed new drug, especially one that works like no others, is toxicity. The drug produces “adverse events,” (AEs), side effects that when compared to whatever favorable therapeutic outcomes limit eventual uses. The drug may produce degrees of symptomatic relief; well and good. But, if at the same time it also produces AEs that cause pain, discomfort, or new pathologies, the efficacy/AE ratio is unfavorable. The drug works, but at a great health-diminishing cost. Patients and physicians will be reluctant to use it.

Case in point: Aduhelm. For Alzheimer’s, that new waste protein-targeting drug does, to a degree, reduce the waste proteins that are involved in Alzheimer’s symptoms. But at the same time it causes swelling of the brain, which evokes a new set of severe symptoms. The physicians at my nearby Cleveland Clinic have proclaimed they won’t prescribe, use, or have in the hospital pharmacy any Aduhelm. AEs too severe; not effectively counterbalanced by therapeutic outcomes.

What evidence supports my contention of blarcamesine safety? All of it; from early preclinical tests in murines (lab rodents) on up through the Phase 1 safety and tolerability trials in real humans. Before all of those, there would have been the rather standard toxicity tests in typical lab animals, to check for (among other things) teretogenicity (induction of birth defects), endocrine disruptions (hormone anomalies), neuron function tests, oncogenic (cancer-induction) tests, and many others. Had blarcamesine failed any of those, the drug would have never been allowed to progress to the early human trials (Phase 1 safety and tolerability studies).

Of course, a few AEs were detected. A bit of dizziness or dizziness; none of which was severe or enduring. Not a disqualifier. Blarcamesine is safe.

What, then, indicates blarcamesine will effectively treat Alzheimer’s? All of the evidence, human and murine, from the many studies of it’s unique mechanism of action (MOA). Consult the graphics of this on the Anavex website; too detailed to expound upon here. But, simply, all of the evidence shows that activation of the sigma-1 receptor protein by blarcamesine causes that protein to function normally, which restores a diversity of reaction pathways and neuron cell-keeping functions that are disrupted or inadequate in the neurons of Alzheimer’s patients. Whenever this has been tested, either in murines or humans, blarcamesine has been effective. Not a shred of plausible evidence that this won’t be the case in the big Phase 3 trial that will terminate later this year (in the second half). In the many murine and early human trials blarcamesine’s safety and efficacy are already clearly established.

But those studies were too short or small, and lacked proper, accepted statistical analyses. Insufficient data points to prompt or allow the FDA to approve the drug. The bigger and longer Phase 3 trial results will suffice. Blarcamesine will be proven to either halt or reverse Alzheimer’s symptoms; safely. Thereafter, medical history will be changed.