Anavex Life Sciences Corp (AVXL)

[falconer66a]

Two other CNS diseases blarcamesine will treat.

...the normalization of GABA/glutamate [by blarcamesine therapy] could then be used as a surrogate endpoint across other diseases which hallmark imbalanced GABA/glutamate levels.

As a biologist, able to sufficiently understand the cellular biology and chemistry of the Anavex molecules, I’ve been following the progression of the company’s drug development for longer than six years. In that period, using only discretionary funds in my family budget, I’ve accumulated several thousand shares of AVXL. In 2023 I expect them to be worth quite a bit.

Of course, the AVXL share price ascent will rest on the clinical successes in the three ongoing drug trials, first (soon) with Rett syndrome, then with Parkinson’s disease dementia, and finally the big, game-changing Alzheimer’s study. The incessant quibbling here of poor management, unmet data release “deadlines,” etc. will finally evaporate.

And, yes, in 2023 there will be another new message board topic: What other diseases can blarcamesine now be used to treat? By then, the drug’s mechanism of action (MOA) will be no longer in question. Blarcamesine, as a unique sigma-1 receptor ligand activates that protein, causing it to propitiously facilitate a diversity of favorable cell processes. Those processes suppress or minimize the symptoms of Rett syndrome, Parkinson’s disease, and Alzheimer’s. Three otherwise recalcitrant central nervous system diseases without useful treatments. Others, not just biologists, will then be asking, “If this drug works so well and so safely against these three CNS diseases, why won’t it work on others?”

Whereupon, for me, enters the GABA/glutamate optimization in girls with Rett syndrome. The Phase I safety and tolerability trials on a few girls with Rett showed that the drug was safe, and it also favorably increased GABA concentrations and reduced glutamate levels. There is no reason to believe that those results won’t continue into the ongoing trial; validating the Phase I results.

For me, it’s the GABA question. I have a rare CNS disease, hereditary spastic paraplegia (HSP). In the long motor neurons controlling the adductor and other muscles in my legs, GABA, gamma-aminobutyric acid, is deficient. I can walk, but only with a walker; can no longer ascend staircases. HSP has caused a neurogenic bladder condition; now have a suprapubic catheter.

GABA is a helpful inhibitor of extraneous nerve impulses. It effectively blocks “nerve static” signals, calming and smoothing nerve impulses. With normalized levels of GABA, muscles are properly controlled and signaled by nerves. When GABA is deficient (as in the case of Rett syndrome) all sorts of nerve impulse problems occur: “Rett syndrome, a serious neurodevelopmental disorder, has been associated with an altered expression of different synaptic-related proteins and aberrant glutamatergic and ?-aminobutyric acid (GABA)ergic neurotransmission.”
https://pubmed.ncbi.nlm.nih.gov/31947619/

SO, when blarcamesine is authorized for Rett syndrome, my neurologist, if his institution will allow it, will prescribe blarcamesine for my HSP, off label. It will be very expensive. But I’ll liquidate a portion of my AVXL position to underwrite my personal experiment with blarcamesine. Of course, the rest of the HSP community will spot the GABA factor, too; will petition to gain FDA approval for HSP outright, or seek a prompt clinical trial.

HSP will be a new CNS disease to be treated with blarcamesine. Another similar disease, primary lateral sclerosis, PLS, will be another one.