Replies to post #321379 on Anavex Life Sciences Corp (AVXL)

[Bourbon_on_my_cornflakes]

SPOT ON: How could you account for one tiny company coming up with two such pharmaceuticals? The odds against just one are unreal.

This can be “accounted for” quite easily. None of the other pharmaceuticals, large or small, simply haven’t discovered or developed the drugs Anavex has. That’s because the others deal in what they know; what their scientists are specialists in. They don’t go off into the molecular hinterlands playing around with strange new molecules. Intelligently (and profitably) they stick with what they know or see.

Look at electric car companies. Two decades ago there were none. Then along came Tesla. Now it seems they IPO a new electric car company every month.

The good thing is we have patent protection so that there aren't 20 sigma-1 startups in the works.

But it is a warning that we need to keep moving fast, as competition will come.

[HMB2010]

Good stuff falconer66a, thanks for post.

Godspeed Anavex !!

[wildcard235]

None of the other pharmaceuticals had Dr. Alexandre Vamvakides.

Sigma receptors were investigated in the 1960's for a particular medical issue (I've forgotten which one), and were determined to be unrelated to that issue. Further investigation into sigma receptors was abandoned.

Dr. Vamvakides, a relatively young researcher at the time, realized that the large number of sigma receptors throughout the human body(1) had to have some function(s), and he began working to discover what those functions might be.

Anavex began in 2004 with a portfolio of over 30 sigma-receptor compounds developed by Dr. Vamvakides, lead investor Athanasios (Tom) Skarpelos, and Harvey Lalach as President. Dr. Panos Kontzalis came on as the first CEO in Jan2007. At some point, Dr. Kontzalis resigned, and Dr. Hervé de Kergrohen became the CEO 16Jun2009. Together, they all got A2-73 through it's Phase 1 clinical trial for safety, but Kergrohen and Lalach failed to secure adequate funding to continue.

After a period of dormancy, Skarpeolos took over and secured $10M in funding from Lincoln Park Capital Fund, and just as important, he recruited Dr. Christoper U. Missling as the new CEO. Dr. Missling held an MBA in addition to his PhD, and had significant experience as a CFO at other pharmaceutical companies.

And soon, Anavex will have its first of many FDA approvals. It took a lot of people a lot of work and a lot of years to get us where we are today. And all we investors have to do is be patient.

(1): "Sigma receptors are unique drug-binding proteins, present in the central nervous system as well as in various peripheral tissues. Sigma receptors are found in endocrine, immune, and reproductive tissues and in high density in liver and kidney. Thus, it is likely that sigma receptors have important functions outside the nervous system and sub-serve a more general role than a neurotransmitter receptor" (WD Wayne, Pharmaceutica Acta Helvetiae 2000; 74, 211-218).

[boi568]

What is the MOA for 1066? I have not seen it described here or elsewhere, so I did not jump to the conclusion it also involved the Sigma 1 receptor. (Hence my question to you last night.)

[abew4me]

Excellent post falconer66a. Well done sir!

I'm reposting your response to boi...and the response to your post by wildcard as well.

**************************************************************************************************

Pure brilliance, actually.

Quote:
"...let's assume for purposes of discussion that both 2-73 and 1066 will be megablockbuster drugs. How could you account for one tiny company coming up with two such pharmaceuticals? The odds against just one are unreal."

This can be “accounted for” quite easily. None of the other pharmaceuticals, large or small, have discovered or developed the drugs Anavex has. That’s because the others deal in what they know; what their scientists are specialists in. They don’t go off into the molecular hinterlands playing around with strange new molecules. Intelligently (and profitably) they stick with what they know or see.

And they haven’t known or seen anything useful at the sigma-1 receptor protein. First, its innate functions and mechanisms of action were until recently unknown, unrecognized. Just a nice little protein lodged between the mitochondrion and the attached endoplasmic reticulum. Should anyone want to look around (inside any cell, but particularly in neurons), there are hundreds of distinct proteins. No one at any of the other pharmaceuticals had any reason to scrutinize the sigma-1 receptor protein.

And, even if they did, they had no molecules that could in any usefully way affect it.

So, as with most of everything else useful in medical science, with Anavex there has been “new science,” things that at the start appear pretty nebulous and without probable application. Every field of science experiences this, but particularly medicine.

Classically, take the “germ theory,” the strange and weird notion (back then) that some sort of invisible “microbes” are the actual cause of many diseases. These “germs” (disease-producers) can move into organisms and produce all sorts of diseases. Of course, medical science utterly rejected this, for decades. Pasteur, Lister, and a few others finally won the argument, but only after “the real professionals” for decades rejected the scientific evidence.

Check the grief and push-back Semmelwiess got for decades trying to keep microbes from mothers during delivery:
https://en.wikipedia.org/wiki/Ignaz_Semmelweis

Scientists don’t like to have their bodies of knowledge discarded, or even questioned. The germ theory did that. In physics, Einstein disrupted things profoundly.

(Currently, I’m working with a university research team in my particular area of ecological science, where we are, similar to Anavex, developing very new ecological techniques and protocols that will solve a major ecological problem. Implementation will take a few more years; in the mean time we’ve had to convince intelligent sceptics that our stuff really works. “Hard to believe!” New ecological science; being rejected for now. Later, broad applications and understandings.)

So, why and how Anavex science? Because a few people had the vision to see that their unique, proprietary molecules could produce wonderful therapeutic (even prophylactic) outcomes. All of that went against what was known about, say, Parkinson’s and Alzheimer’s diseases. Before, no one knew how the sigma-1 receptor protein actually worked, or what it caused to happen in cells. Now, Anavex knows, and has in-house molecules that modulate and promote the multiple functions of the sigma-1 receptor.

There will be blow-back (understood), but I’ll make the claim now: In time, a Nobel Prize or two will be awarded to those at Anavex who have been instrumental in bring forth Anavex science. In the last century antibiotics changed medicine profoundly. In the 21st century, Anavex will make probably even greater improvements — all because a few individuals were not restrained by conventional understandings or practice. Like all great scientists, they could look beyond what is known and accepted, seeing new things that needed to be studied and perfected.

Why Anavex? Scientific brilliance, unconstrained by conventional knowledge or perspective.

**********************************************************************************

response by wildcard:


None of the other pharmaceuticals had Dr. Alexandre Vamvakides.

Sigma receptors were investigated in the 1960's for a particular medical issue (I've forgotten which one), and were determined to be unrelated to that issue. Further investigation into sigma receptors was abandoned.

Dr. Vamvakides, a relatively young researcher at the time, realized that the large number of sigma receptors throughout the human body(1) had to have some function(s), and he began working to discover what those functions might be.

Anavex began in 2004 with a portfolio of over 30 sigma-receptor compounds developed by Dr. Vamvakides, lead investor Athanasios (Tom) Skarpelos, and Harvey Lalach as President. Dr. Panos Kontzalis came on as the first CEO in Jan2007. At some point, Dr. Kontzalis resigned, and Dr. Hervé de Kergrohen became the CEO 16Jun2009. Together, they all got A2-73 through it's Phase 1 clinical trial for safety, but Kergrohen and Lalach failed to secure adequate funding to continue.

After a period of dormancy, Skarpeolos took over and secured $10M in funding from Lincoln Park Capital Fund, and just as important, he recruited Dr. Christoper U. Missling as the new CEO. Dr. Missling held an MBA in addition to his PhD, and had significant experience as a CFO at other pharmaceutical companies.

And soon, Anavex will have its first of many FDA approvals. It took a lot of people a lot of work and a lot of years to get us where we are today. And all we investors have to do is be patient.

(1): "Sigma receptors are unique drug-binding proteins, present in the central nervous system as well as in various peripheral tissues. Sigma receptors are found in endocrine, immune, and reproductive tissues and in high density in liver and kidney. Thus, it is likely that sigma receptors have important functions outside the nervous system and sub-serve a more general role than a neurotransmitter receptor" (WD Wayne, Pharmaceutica Acta Helvetiae 2000; 74, 211-218).