Will Anavex Treat Multiple CNS Diseases?
Will Anavex Life Sciences Corp be a one-trick pony (well, a one-drug pharmaceutical) able to treat only a few thousand girls with a very rare genetics-based central nervous system disease (Rett syndrome)? Or, will several of the drugs in the corporate pipeline successfully treat a multitude of CNS and other diseases with mitochondrial and related etiologies? Let’s check the evidence.
Personally, of the three CND diseases Anavex 2-73 is being clinically tested in real humans (Rett syndrome, Parkinson’s Disease Dementia, and Alzheimer’s Disease) I had the greatest concern with the prospects of the Rett trial. A mutation in the X chromosome causes severe neurodevelopmental anomalies. Girls have two X chromosomes, so in most cases one X chromosome functions adequately to allow development, albeit with the severe symptoms of the disease. Boys have only one X chromosome. The matching other one is the Y chromosome, which cannot have a Rett mutation. A Rett mutation in the single X chromosome is lethal. Baby boys born with the gene do not survive long. For little girls with the gene, one functioning gene in one X chromosome allows survival; but not a biochemical scenario that appears to be directly correctable by Anavex 2-73.
But that’s not what the evidence shows. First, mice with an induced Rett syndrome status were treated with Anavex 2-73 in their drinking water. Their symptoms were suppressed, more normal neuromuscular activity and controls appeared. Somehow, the Anavex sigma-1 receptor agonist restored more normalized neuromuscular control — without discernable side effects. For Rett syndrome mice, both safe and effective.
Those well-replicated and precisely-defined data allowed the first testing on humans with Rett, the subject of the recent Anavex announcement. The six older girls with Rett, like the lab mice before, also had safe symptomatic improvements.
For Rett syndrome, there is now a substantiated mouse/human prediction of safety and efficacy; for a disease condition for which the proper folding of various proteins wouldn’t appear to be the sole factor of efficacy. Whatever the exact mechanism(s) of action were in the girls, there is now no doubt that Anavex 2-73 successfully minimizes to some degree various Rett symptoms. No other drug has been able to show this (at least without appreciable side effects; absent with Anavex 2-73).
And, those results came from older girls, whose nerves and neuromuscular reflexes had been degraded and disturbed for many years. If administered early on, at the first indications of a Rett status in a baby girl, it is probable that nerve and muscle development could proceed much more normally. Good chance the Anavex drug can best suppress Rett syndromes if administered at the earliest indication.
Ok, the efficacy against Rett symptoms is settled (lots more to follow; treatment protocols, regulatory approval for sales and use, etc.). Do the Rett data portend anything for the other two diseases in clinical trial?
Well, they, too, have had detailed murine studies done. Rats or mice with genetically or chemically induced Parkinson’s and Alzheimer’s have been dosed with Anavex 2-73. The results of those detailed lab trials provided the information to allow the start of human trials, however yet preliminary they are.
Readers here are familiar with Australian Alzheimer’s patients being treated with Anavex 2-73. I won’t detail those results. They continue to be very successful, appearing to either maintain or improve cognition. They substantiate, replicate the results (at least to a degree) of transgenic rats with Alzheimer’s being treated with Anavex 2-73. Two murine (rats and mice)/human successes: Rett syndrome, and Alzheimer’s.
The first data from the Parkinson’s trial could appear within a few months. Same thing. Lab rats or mice with Parkinson’s symptoms showed improved health with Anavex 2-73. What are the probabilities of an Anavex Parkinson’s success? My moderate AVXL position is based upon what I believe will be the outcome.
Great potential, now, for Anavex Life Sciences Corp to be able to successfully treat three CNS diseases: Rett syndroms, Parkinson’s Disease Dementia, and Alzheimer’s Disease. With success of any one of these, Anavex Life Sciences Corp will be a (very) going concern.
But the Anavex pipeline lays out a number of other diseases and conditions being targeted. Those didn’t arise merely in casual discussions over lunch. Doubtless, there are abundant murine data showing other particular disease and condition efficacies.
Simply, the Anavex sigma-1 receptor agonists have a multitude of “downstream” treatment outcomes, against a wide variety of diseases and conditions. Privately, Anavex principals have seen and know the multitude of yet-concealed murine studies. Like the unobserved portions of an iceberg, as it were. (Didn’t someone in Anavex use that phrase once upon a time?)
The data I’d most want to see is the ability of the Anavex molecules to a) prevent the onset of various diseases, or b) to clearly suppress and reverse disease symptoms at their onset. An anti-aging drug, as it were.
Presently, we are in but the first pages of the first chapter of the Anavex story. Big things ahead. Things will get very interesting in the next several years.
AI
