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Need to Know Names of Clinical Trials
Posters often mention the "EXCELLENCE" trial. A nice suggestive name. But what is it? What's it testing, in whom?
Can someone post a listing of the names of the Anavex trials? Just as important as the enigmatic acronyms.
CVR?
Joseph, thanks.
I've added those to the list, including the non-technical acronyms. Readers need to know what all of the acronyms mean. Otherwise, confusion.
Anyone else, post your list. I'll be able to post the list sooner.
I'm going to have to spend some time writing out clear, concise definitions of the acronyms; beyond just what the letters stand for. For example: RSBQ --- Rett Syndrome Behavior Questionnaire.
Good. But what IS the Rett Syndrome Behavior Questionnaire? What do readers need to know about it to make sense of its appearance in postings on this message board? Talk (or an acronym) is cheap. Useful understanding requires effort on the part of both the writer and reader.
Sage, thanks!
What you just posted, a list of acronyms that appear on this message board, is important. Thank you for compiling and posting those. I'll copy your list, get it on my word processor, and update it when I see other acronyms in messages. I'll also try to provide helpful, detailed definitions and information on the acronyms.
All stuff I did when I taught advanced placement biology to talented biology students headed for science and medical student scholarships. My students got big scholarships, and I got awards for my teaching.
This Message Board Needs an Acronym List
In good, acceptable technical writing, acronyms, sequences of otherwise meaningless letters, need to be clearly defined, to be understood by readers. Of course, there is very little acceptable technical writing on this message board. Certainly, a good number of new readers click on to see what's being presented. But postings are perfused with WTHITs --- What The Heck Is This acronyms and new readers click away and go off. Understandably.
So, in my idle moments in the next few days or weeks I'm going to try to assemble a list of the multitude of acronyms lazy message writers so inconveniently use in their postings. Inquiring minds will want to know what all those capital letters stand for. If anyone wants to assist, I'd be delighted. When convenient, post some of the acronyms that confuse readers. Collaboratively, we can bring a bit of clarity on this issue.
Comparison of Three FDA-approved Drugs for Rett Syndrome
This is Anavex 3-71's First Step
As Expected, An Anavex 3-71 Human Clinical Trial
I’m pleased to learn of Anavex’s new placebo-controlled Phase 2 trial of ANAVEX®3-71 for the treatment of schizophrenia. Very much as I expected.
Too early for me to lay out or describe what I believe will be the significance of this new trial. Anything I’d post on the matter would be summarily bashed, by all the usuals, with the usual arguments we’ve seen through the years here. Right now, I’ll keep to myself my understandings of the future of Anavex 3-71 for the company, its shareholders, and patients.
But this won’t be a lengthy trial. Anavex 3-71's abilities to safely and effectively treat schizophrenia will be promptly determined and illustrated.
If those Phase 2 results are positive, the naysaying Anavex experts will, of course, tell us why the required Phase 3 trial will then be a failure and Anavex Life Sciences Corp will fold. There will be some statistical errors or insufficiencies, of course. The game will be over. We’ve seen it all before. I’ll sit back and watch.
We Should Be Honored and Thankful
Ok, But Nature Has Embargo Policies
Now, Let's See the Paper Itself
The Article Will Get Posted Right Here
Deeper Understanding from an Expert
In Weaks or Months
Anavex Has Submitted Actual Data to the EMA
Page 18, in the "Next Steps" portion of the slide:
Peer-reviewed Journal Article Will Appear
On today's presentation slide deck, from the “Next Steps” portion, p18:
Do peer reviews always have good results?
Good question. The concept or process of “peer review,” in regard to the appearance of an article or research report in a recognized scientific journal, is not always understood.
First, what’s a “peer?” A peer is an accomplished science expert having knowledge and experience in a particular field or subject, equal to or better than the author(s) of a paper.
What, then, is “peer review?”
Taking Longer....
Great. We've Got the Answer.
Ah, Some Shorts.
Today's share price gain (now, 7.9%):
Why today’s Up?
The Anavex naysayers who post here make clear that any and all AVXL share price declines following any sort of corporate announcement indicate that “the market didn’t like the latest Anavex news.”
Is the converse ever true? Naysayers, tell us why the AVXL share price is up 7.15% right now. Some could claim that the amorphous “market” “liked” Anavex’s new patent. Right, or wrong? Lots of “You’re in danger” explanations of exactly why AVXL share prices take steep dives. But a 7% gain an hour before the market closes is a nice gain today. What caused this to happen? Fill us in, please.
Next, and Better, Will Be Anavex 3-71
https://pubmed.ncbi.nlm.nih.gov/38073274/
Well, this is an interesting new paper (for which I had available only the abstract). In this study Anavex 3-71 was administered to 28 people, at ascending doses ranging from 5 to 200mg. The study collected resultant pharmacokinetic data.
Pharmacokinetics describes what the body does to a drug, referring to its movement into, though, and then out of the body; noting its time periods of absorption, bioavailability, distribution, metabolism, and excretion. All crucial information to understand for any drug, but especially for a new drug candidate — essential information that would appear in a new drug application (NDA).
The title of the paper indicated that the new drug in development, Anavex 3-71, will be targeted “for Treatment of Frontotemporal Dementia, Schizophrenia, and Alzheimer Disease.”
I find several things to be very interesting.
First, of course, is the plainly-stated target diseases of Frontotemporal Dementia, Schizophrenia, and Alzheimer Disease for Anavex, this time using Anavex 3-71. This signals a new, major chapter in the Anavex story. To date, Anavex’s lead molecule in both murine and human trials has been Anavex 2-73, blarcamesine. Now, Anavex 3-71 will be in play — perhaps (I think very likely) to replace blarcamesine. The spotlight on blarcamesine may start to fade, as Anavex 3-71 takes center stage.
Of particular interest was the dosage ranges in the study, from 5mg on up to 200mg. It is noted that in animal trials Anavex 3-71 was dosed in microgram units, not milligram units. A microgram is one-one thousandth of a milligram. Anavex 3-71 is far more active than blarcamesine; hence the much smaller dosages in the animal studies.
This was a study in humans. At the low end, a mere 5mg was administered. At the high end, 200mg. In a human clinical trial for efficacy and safety, dosages will be somewhere within the margins of this study.
Here are the most important, significant pharmacokinetic data for Anavex 3-71:
The We Really Know Crowd Will Tell....
Rett is a Very Rare Disease; Not Alzheimer's
How many have Alzheimer's Disease?
I Was Wrong — By a Factor of 10
Yesterday, in my conjecture that eventually blarcamesine might gain widespread use as a general-health prophylactic, where most people in the developed world would take a small dose to prevent the onset of many common diseases, I noted that the daily cost might be in the range of a single dollar, $1.00. With that price, the cost burden would not be great. Anavex would have annual global sales revenues in the tens to hundreds of billion dollars.
But I was wrong, by a factor of at least 10.
Actually, soon enough, blarcamesine will be replaced with a similar Anavex sigma-1 receptor agonist (activator), Anavex 3-71. It has been used and proven in tests in transgenic murines, lab rodents who have the human genes for CNS diseases inserted in their genomes. And, yes, Anavex 3-71 was therapeutically effective.
But Anavex 3-71 is dosed, administered in microgram amounts, not the milligram amounts used when administering and testing blarcamesine. A microgram, 0.000,001 gram, is one-millionth of a gram; or, 0.001 milligram, a thousandth of a milligram.
Simply, very tiny amounts of Anavex 3-71 are required, compared to blarcamesine; probably not in one-thousandth fractions; more likely in one-hundredth amounts. But, at whatever doses prove effective, they will be far smaller than the milligram dosages of blarcamesine.
The per-kilogram manufacturing costs of both drugs will be essentially equivalent. But ten to a hundred times more doses can be made with the Anavex 3-71. Simply, it works more powerfully, at far lower dosages than blarcamesine. Therefore, dose costs to customers will be lower than for blarcamesine; but with a larger profit margin for Anavex.
With Anavex 3-71, everyone wins; including AVXL shareholders.
Hundreds of Millions of Blarcamesine Users?
The plausibility of my blarcamesine prophylaxis conjectures ever materializing, understandably, appear to be improbable, at best. At least for the first years of blarcamesine sales, Anavex will be highly profitable by providing effective treatment for the CNS diseases they presently target. For AVXL shareholders, that will be greatly rewording.
But, for several factors I’ll lay out, eventual sales of blarcamesine to hundreds of millions of people in the developed world for the purpose of general health prophylaxis, disease prevention, is not out of question. It could really happen, for these reasons.
First, of course, blarcamesine will have to prove itself to be utterly safe, without obviating adverse events (side effects) when taken chronically, for long, continuing periods. This will be revealed when the drug is used to treat or prevent Alzheimer’s disease, for lengthy, continuing dosing periods.
Finally, of course, a preponderance of evidence must show that the drug is, actually, a safe and effective general-health prophylactic; that when taken in low doses for continuing periods of time various age-related or developmental diseases are prevented or minimized. Such evidence, too, could be derived from people taking the drug for a CNS disease, where it not only stopped or reversed that disease’s progression, but concurrently improved the general health of patients on the drug.
What, then, if such prophylactic dosings cost, say, $10 a day; $3,650 a year? For a family of four, that would be an annual blarcamesine cost of $14,600. Pretty expensive. Only a minority of families would have or would use such funds for a drug that doesn’t treat any disease they presently have; merely prevents diseases often far in the future.
What, however, if a 10mg prophylactic dose of blarcamesine can be sold (and purchased) for $1.00? That’s a yearly cost of $360; a fee health insurance companies would gladly cover, as it would significantly reduce their expenditures used to treat any of the big diseases the drug is preventing.
In all of this, here’s the most important information. Anavex has patents protecting their efficient and effective manufacture of blarcamesine. With those protected chemical synthesis processes, it is very probable that 10mg doses of blarcamesine can be made for pennies on the dose. A small snowflake, or a grain of salt or sand are all typically 1mg. 10mg isn’t much. Unlike other drugs, blarcamesine is not a complex, gigantic molecule. Using Anavex’s patent-protected chemical processes, the drug will be inexpensive to manufacture, perhaps in the realm of a penny a milligram or so.
If these numbers prove to be accurate, blarcamesine sales may eventually bring tens or hundreds of billions of dollars in annual revenues to Anavex Life Sciences Corp. Good health for hundreds of millions. Wonderful dividends for owners of AVXL shares. Let’s watch to see how or if this develops.
Your Concern is Understood
Be Assured, Anavex Knows — By Themselves
Pre- and Post-natal Blarcamesine Developmental Defect Prophylaxis
It is now well-demonstrated that blarcamesine, by its activation of the sigma-1 receptor protein, favorably modulates or promotes a number of propitious cell-keeping processes, the ones involved in autophagy, the multitude of processes within cells that remove cellular wastes and maintain internal cell processes. The term autophagy, from auto-, meaning “self,”and -phagy, meaning “to eat,” is where the cell eats itself; but consumes only the cell’s internal wastes, toxins, or non-functioning organelles. Autophagy is the cell’s essential internal cleanup processes.
But, as organisms age, autophagic processes decline. Age-related pathologies thereby increase. Hence, there is the prospect that the administration of blarcamesine in late middle age will maintain age-inhibiting autophagy, prophylactically yielding fewer age-related diseases. As I’ve indicated before, Anavex surely has clinical data on this, derived from typical aging studies done in certain lab organisms, such as the lab roundworm Ceanorhabtidis elegans. There is the eventual prospect that blarcamesine or Anavex 3-71 will be typically and commonly prescribed for and taken by everyone, to safely and effectively maintain autophagic processes; to thereby improve and maintain health throughout life.
But blarcamesine induces other favorable cell processes. One is involved in gene expression, where genes in chromosomes have the chromatin surrounding them chemically unwrapped, allowing the now freed-up gene, a length of DNA, to be transported to a ribosome where the genetic information produces a specific protein. Those proteins are specific enzymes that catalyze subsequent chemical reactions that enliven the cell.
Key is this. Gene expression, the converting of the DNA of specific genes to cellular chemicals that make the cell function, must be an error-free and efficient process. If gene expression goes wholly wrong, the cell dies. But fractional or partial gene expression is merely pathogenic. The cell and the organ system in which it resides continues to function, but in a diseased state. Such is the case with the neurons involved in Alzheimer’s disease.
But here is a new, but related blarcamesine conjecture — one that I’m certain Anavex has confirmatory data on but has the good sense to keep quiet about. At the right time, it will be an Anavex media announcement; but most likely after blarcamesine is already approved as a CNS disease therapy.
The conjecture is this: that blarcamesine may prove to be a safe, effective, and inexpensive birth defect prophylactic. Many birth defects are caused by erroneous gene expression starting with the growth of the embryo, right after egg is fertilized, before the mother even knows she’s pregnant. Therefore, women desiring to have a baby would be prescribed a daily dose of blarcamesine, which would diffuse into the egg, before conception, and be available to implement fully functional autophagy and gene expression, starting right in the zygote, the one-celled fertilized egg. The mother would continue to take blarcamesine through her entire pregnancy, during which the zygote, then the embryo, then the fetus, and then the infant would develop with minimized chances of defects caused by compromised gene expression.
But, of course, gene expression doesn’t terminate at birth. During a child’s physical development, all sorts of genetic or physiological problems can occur — many of which might well be obviated by the presence of daily or weekly doses of blarcamesine.
Blarcamesine prophylaxis — from conception through all of life.
Who Could Trust the EMA? Why?
When Blarcemasine Is Approved, ‘CEOChecker’ Will Warn Everyone
Really?
Neonatal Administration for Rett Syndrome Will Be Successful
No TV Ads for Blarcamesine
Consider, Anavex Has New Hires
Learn About a Genetic Heat Map
Anavex Will Not Sellout Its Drug Manufacture
EMA Knows, Will Be Supportive
The Share Price Ascent Has Begun
Safety and Efficacy Don’t Count?