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Any clue where these rumors are coming from other than posters on this board?
Analogizing to SSRIs is a bit tricky as they inhibit re-uptake of a specific neurotransmitter (thus the name), in this case serotonin. The beauty of CRISPR lies in its ability to target multiple sites, excise specific targets, and even insert therapeutic genetic material to catalyze changes in specific exons.
You are correct in suggesting that any manipulation (e.g., suppression) will have a corresponding offset; however, if the initial target has been properly affected, the thought is that such ancillary side affects (if any; it isn’t necessarily a given) would be manageable.
Yes, it’s a long haul and I do not mean to suggest otherwise, but—like any such endeavor—incremental progress continues. Look for news on this front as it is definitely in the hunt.
Your understanding of CRISPR is deficient.
You can use clustered regularly inter spaced short palindromic repeats to point at any place in the genome and do anything you want at that spot.
CRISPR/Cas9 enzyme is a homing device that guides molecular scissors to disable or repair genes. This technique can be used to turn genes on or off.
With respect to AD specifically, CRISPR can be used to “cut and silence” pathological proteins in AD to block further progression of symptoms—regardless of one’s age.
Working with bio-engineers to design cocoon-shaped nanoparticles, doctors can use this application to cross the blood-brain barrier to transport the CRISPR /Cas9 protein, delivering them within the brain.
This process may also be applicable to other CNS disorders such as PK, etcetera.
Yes, of course, development issues are ongoing but there is nothing in theory to prevent this approach from working successfully.
I use Bayesian analysis. Skipping the math (which is important BTW), here’s how it works:
Knowing nothing about success/failure, we start at, reasonably enough, a 50/50 chance of success (Yes, it would be higher as I knew something before investing but I am simply using an unprejudiced case). Then, events happen that either bolster or diminish prospects of success. Each event is factored and prospects for success are recalibrated backward to origin accordingly. Skipping how the sausage is made, I arrive at a new percentage of success. Before “news blackout”, I was at around 80% success for AVXL. Almost impossible to say definitively why updates have not been forthcoming to date (Not saying anything negative about this, just that a void like this is not easy to incorporate into analysis—speculation reigns). Next catalyst—as long as it is a substantive disclosure—will allow an appropriate update.
I can say without reservation that, of all my investments in bio (which began in 2009), I have not seen better prospects for future payoff (for us and patients) than I see for Anavex.
For a certainty. I strongly believe 2-73 will be a boon in our fight against CNS diseases and have a relatively broad application early on and have a degree of staying power. As time progresses, however, other, better, approaches (CRISPR, insulin protocols, and even diet will play an increasing role in treating AD especially).
Just as a “singularity” will be reached in artificial intelligence, so, too, will we see a corresponding breakthroughs in bio medicine. Chemicals will have a diminishing effect in the future but will continue to be the mainstay until more effective approaches are developed. To be sure, I am heavily invested in Anavex (and have even deferred forays into such stocks as $EDIT, for example) and look forward to substantive developments in the foreseeable future for our drug. Just putting things into perspective.
CRISPR is indeed the next up-and-coming approach utilizing gene therapy. IMO, targeting insulin will pay benefits as well. It should be understood that Anavex will temporarily replace SOC in AD (and perhaps other diseases as well if approved) just as Donepezil is currently enjoying this coveted spot. Eventually, other approaches, including CRISPR and maybe even a new Anavex medicine, will replace 2-73 to some extent (it may hang on for some time as new applications are worked out and brought to the market). It’s all in the timing my friends: You gotta “know when to fold’em, know when to . . .”
Trials will start this year. Mark this post. As usual, company will present at CTAD in coming weeks.
You are quite right. The Rett trial will easily fall within the AD timeframe for completion of phase 2/3. A failure, any failure (irrespective of 2-73 eventually crossing the finish line successfully), in the Rett trial would greatly damage investors expectations with respect to 2-73. This could indeed be quite costly for the company. Therefore, modus ponens, the good doc must be extremely confident of success. After all, he has close to $5 mil in shares riding on the outcome of this entire enterprise. Hadn’t considered this angle. Impeccable logic.
Got it. Either way you slice it, each catalyst builds on those from before. As mentioned previously, LBAs require “a high threshold of scientific merit.” Bayesian analysis portends great news to follow.
If there were thousands of drugs targeting AD that failed the outlook for Anavex would be the same. Targeting amyloid plaques has failed and, logically, all other approaches that have failed have also not followed 2-73’s regimen. Failures do not affect Anavex’s chances of success, they only skew stats (percentages) and highlight the ineptitude of ineffective strategies.
LBAs must have "high threshold of scientific merit" per guidelines. Soooo
From truebluetribune oct. 2nd:
Anavex Life Sciences Corp. presently has a consensus price target of $13.33, indicating a potential upside of 222.06%. As a group, “Biotechnology” companies have a potential upside of 55.04%. Given Anavex Life Sciences Corp.’s stronger consensus rating and higher possible upside, research analysts plainly believe Anavex Life Sciences Corp. is more favorable than its competitors.
Hello. This has already been adjudicated. MS is still in Anavex's bailiwick, just not listed in the streamlined and concentrated presentation of their pipeline. See post# 122515
Not sure what a "golden gopher" is, but appreciate your enthusiasm.
I see. So the IR department is LYING. I spoke to them yesterday and they told me point blank that MS, as well as other protocols on the pipeline were ongoing. Too much to get into, you need to read my posts, but MS has NOT been discontinued.
That's pretty much my take as well. Reading anything more into it at this point is (Dare I use the "S" word again?).
Excellent post--"Thereby hangs a tale".
Read his post and it is consistent with recalibration but not indicative of abandonment of previously listed protocols. For example, I specifically asked about epilepsy. "No change". Nothing has been "dropped". There may be some nuances as his post suggest; however, I believe they relate, if at all, to a simple shift in emphasis. I asked if a statement should be expected and the response was matter of factly, "No" as if it was demonstrably unnecessary. So there you have it. If there is more to it than we have been led to believe . . .?
Please see my response to baltimorebullet. Everything copacetic.
No email. Called direct. I was told (and cut me some slack here as I was gathering response "on the fly") NOTHING has changed with respect to their pipeline. I know, sounds strange but response was very calm and measured and not evasive in any manner. In vitro continuing, redid animal models, epilepsy continuing, last conference presentation reflects reworking to some extent. Bottom line: Everything in pipeline continuing unabated.
Wow! The plot thickens. Thanks for the feedback.
Fenfluramine is part of a diet regiment. Now I'm really confused.
https://pubchem.ncbi.nlm.nih.gov/compound/fenfluramine#section=Top
The deletion of epilepsy from pipeline is surprising. I thought excellent progress was being made on this front. I understand streamlining and concentration arguments but still, something is not quite right here. Am I off base?
Thanks for the link. Don't know immediately how Dravet Syndrome seizures generalize to wider applications but sounds promising.
Analogizing to 2-73 for AD into third quarter positive results, a quadrupling of stock price would be relatively tame.
Looks like Zogenix is already in phase three.
How far along in trials are they?
Last sentence: how these things work.
Lane Simonian article:
This article could have been written a year ago, or more. Response:
1. Of course protein kinase C is not inhibited "enough to alter the course of the disease." No one, including Anavex, has suggested 2-73 will indefinitely inhibit protein kinase C.
2. Let's get real and drop the hyperbole: 2-73 most likely will not cure or indefinitely alleviate the eventual progression of AD. None of us honestly believes this or has gone out of their way to strongly suggest such a result for the drug.
3. We know virtually every AD drug fails eventually and our hope is very well grounded in the expectation that 2-73 will significantly improve SOC enough to get FDA backing.
4. We also know enough as investors to shore up profits at the opportune time; some before FDA window, of course. Who holds on to what portion of their investment through the finish line is up to each individual.
On balance, nothing has changed: the weak bail and the committed hold strong. Much too early to get jittery. There will be plenty of time for that as we progress through the trials. I'm well situated with a long position and will remain so. Have a few options I may cut loose when timing appropriate; otherwise, coasting like everybody else who understands how these in segments work.
All the best.
Will do. Good idea.
That's funny. I see a double-reverse head-and-shoulders with an inverted Fibonacci declination trending toward infinity. Of course, I could be wrong.
Have spoken and written to IR only a few times in last couple of years and have always received a relatively prompt, courteous reply. As to how forthcoming the response may be? We'll see.
Did so ystdy and expecting reply.
You're welcome.
Missling now owns close to $5 million in stock. I'm sure he is interested in protecting his wealth as well as helping those in need.
There is much more than MS by the way. Everything is/ should be subject to rule.
Interim results are material (don't. Have to be "final").
So Anavex is NOT under any obligation to disclose. Interesting.
Should be "Carve-outs".
Listing Rule 3.1 is exhaustive; however, pertinent to disclosure of "market sensitive" information:
". . . an entity must disclose all information concerning it that it becomes aware of from any source and of any character, if a reasonable person would expect the information to have a material effect on the price or value of its securities."
When is information market sensitive?
When the information "would, or would be likely to, influence persons who commonly invest in securities in deciding whether to acquire or dispose of" those securities.
If the left side of my brain is working properly (debatable), such information is surely in Anavex's hopper currently. Question then becomes: when must an entity disclose such information?
". . . Immediately upon the entity becoming aware of the information, unless it falls within the carve-pits from disclosure. . ."
"The word 'immediately' does not mean 'instantaneously', but rather 'promptly and without delay.' "
Soooo, as previously posited, are we not in this situation presently?