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A profound observation, indeed! It’s an extraordinary time for innovation.
And Christopher Missling is correct about understanding the ability of the body to heal itself.
‘’The Genetics Revolution: ‘The Intersection of Information and Biology’
By understanding the information processes underlying life, we are starting to learn to reprogram our biology to achieve the virtual elimination of disease, dramatic expansion of human potential, and radical life extension.”
– Ray Kurzweil, The Singularity Is Near
Relax!
I meant to say I know of no reason the trials will not occur. Sorry for the typo.
Nidan:
Thank you.
I am not saying that it is a sure thing. I am saying that the odds of some sort of FDA approval are greater here than in most clinical trials because of the indications for CNS diseases that have no effective treatment.
However, I suppose I am saying that Anavex is not on the verge of destruction. I see no evidence of that. There may be a lack of positive feed back due to the delay in a response from the FDA to proceed with the trials that management says will eventually take place. Missling said at the annual meeting yesterday that Anavex has a good relationship with the FDA. No positive response has occurred, but no negative response has occurred. From my personal experience, I am saying that I do not find the FDA delay by itself to be negative or positive. I find it to be normal.
I know of no reason to believe that the planned clinical trials will occur. If the trials occur in the second half of 2018 as announced yesterday, we should know shortly thereafter. The Rett trial, for example, is about 8 weeks.
At yesterday's annual meeting, some statements made that do indicate postive feedback. Some are as follows: Organizations like Angelman, Fragile X, etc. have acknowledged that they are exicited about the drug's positive signal for thier untreatable diseases, there is renewed confidence with the Illuminia data, more funding from third parties is expected, and the move to Europe of the Parkinson's trial that will be less expensive and has other advantages.
I am sure that you have good reasons for your concern, and I would like to know more about the specifics. It is obvious that this drug is new approach to finding an effective treatment for CNS diseases, and I am sure that major pharmacuetical companies and many others are skeptical; however, if Anavex can obtain an approval for any indication anywhere, in the U.S. or Europe, the skepticism may be overcome. After all, nothing else works for many of the CNS diseases for which this drug may be indicated.
I, and others on this board, have commented about the many indications AVXL 2-73 seems to have. If this continues to prove to be the case, it increases the chances of FDA approval for some of those indications, which means it will be used off label for other indications. I have also commented in the past about how most everything the FDA does takes longer than most expect or hope. Of course, FDA processes have been accelerated over the years, but It is still a government bureaucracy. Furthermore, the FDA is dealing with new FDA procedures here in the way the Anavex clinical trials are designed and implemented.
And yes, Missling did make mistakes about how long it would be for these trails to be approved and commenced, but that does not change anything about the science, indications and potential for the drug to be a success. Shareholders that sold yesterday at the price of $1.85 are the ones that made the big mistake in my opinion. However, we are all human. We all make mistakes. Being overly optimistic is sometimes a human mistake as is being overly pessimestic, or for capitulating and dumping your investment at precisely the wrong time.
History does repeat itself. It repeats itself because we are all human with the same characteristic, traits, and emotions no matter of what generation. Missling has made mistakes, but those mistakes are not as grave as some may make them out to be. Humans have overacted to the mistakes he made, but in the end those mistakes will be forgotten and forgiven because that is what humanity does.
Galectin-3 inhibitor improves hepatic ballooning in NASH
This is a link to an article about the presentation Apri 14 at the Liver Congress in Paris:
https://www.healio.com/hepatology/steatohepatitis-metabolic-liver-disease/news/online/%7Bc68938d1-b7cf-47b2-9154-f6e3e5b38e6c%7D/galectin-3-inhibitor-improves-hepatic-ballooning-in-nash
Here is something else I found about about hepatic ballooning in NASH:
Hepatocellular ballooning is a key feature required for the diagnosis of NASH and a component of currently used histological grading and staging systems of NAFLD. However, it represents an ill-defined form of liver cell injury associated with cell swelling and rounding of the cytoplasm, the detection of which is prone to intra- as well as inter-observer variation. Some of the factors that may contribute to ballooning are the rearrangement of the intermediate filament cytoskeleton, accumulation of small-droplet fat in the cytoplasm and dilation of the endoplasmic reticulum. The rearrangement of the intermediate filament cytoskeleton can be demonstrated by the loss of keratin 8/18 immunostaining of the cytoplasm, and may thus be evaluated in the future as a marker for the more objective detection of hepatocellular ballooning in NASH.https://www.ncbi.nlm.nih.gov/pubmed/21476917
The following is a paper dated in 2017 about lack of therapies for treatment of NASH and unmet accellerated need.
Current and future treatment options in non-alcoholic steatohepatitis (NASH)
Nikos Lazaridis & Emmanuel Tsochatzis
Pages 357-369 | Received 08 Nov 2016, Accepted 07 Feb 2017, Published online: 16 Feb 2017
Download citation https://doi.org/10.1080/17474124.2017.1293523
Introduction: Non-alcoholic steatohepatitis (NASH) is a chronic liver disease that can progress to cirrhosis and hepatocellular carcinoma. Diagnosis of NASH requires a liver biopsy and is defined as presence of hepatic steatosis, ballooning and lobular inflammation with or without fibrosis. Although NASH is the most common cause of liver disease in the west world and among the top three indications for liver transplantation, there are no universally accepted pharmacological therapies and therapeutic advances have been slow.
Areas covered: Current evidence about lifestyle interventions, bariatric surgery and pharmacotherapy is reviewed. Dietary recommendations and lifestyle interventions have shown promising results but are difficult to maintain. At the moment, there is no universally approved medical treatment for NASH. Pioglitazone and vitamin E are recommended by guidelines in selected patients. An increasing number of phase II and III trials in non-cirrhotic NASH are currently recruiting and their preliminary results discussed.
Expert commentary: As NASH is classified as a medical condition of an unmet therapeutic need, it has gained an accelerated access pathway for drug approval based on surrogate endpoints. It is therefore expected that within the next five years, there will be at least one approved agent for the pharmacological treatment of pre-cirrhotic NASH.
So far, today’s low of $2.06 is the exact low of three days ago. If that is the low for today, it matches the yearly low for AVXL shares of $2.06 reached three days ago. Hopefully, it has bottomed, and that will remain the yearly low.
As I remember, Richard Uihlein’s $10 million loan or line of credit projected funds for the company into 2019.
In the meantime, I am interested in the late breaker oral presentation that will be made April 14, at the International Liver Congress in Europe.
Yes, “Making predictions is hard, especially about the future...”
Yogi Berra.
Bob Farrell's 10 rules of investing and other things to think about at this juncture re AVXL:
1. Markets tend to return to the mean over time.
2. Excesses in one direction will lead to an opposite excess in the other direction.
3. There are no new eras—excesses are never permanent.
4. Exponential rapidly rising or falling markets usually go further than you think, but they do not correct by going sideways.
5. The public buys most at the top and the least at the bottom. The downfall of all investors is ultimate ‘greed’ and ‘’fear’’. They don’t sell when markets are near peaks, nor do they buy market bottoms. However, this does not just apply to individuals but many advisors as well.
6. Fear and greed are stronger than long-term resolve.
7. Markets are strongest when they are broad and weakest when they narrow to a handful of blue-chip names.
8. Bear markets have three stages: sharp down, reflexive rebound, and a drawn-out fundamental downtrend.
9. When all the experts and forecasts agree, something else is going to happen.
10. Bull markets are more fun than bear markets.
From the oracle of Omaha, Warren Buffett:
We simply attempt to be fearful when others are greedy and to be greedy only when others are fearful.
Two things I have to add are these:
1. You cannot wish the market to do what you want it to do.
2. We could be close to the ”finish” line. By this I mean we could be close to a bottom for AVXL share price. I remember running marathons where people gave up, and all they had to do was take a few more steps to cross the finish line. However, a good many do not do it for whatever reason, but that may be the difference in success or failure.
It's a free country. Take your choice. You may choose to fail or you may choose to succeed.
Think about it!
I second all of that!
Yes, a pragmatic assessment.
Yes, value is being added to the drug, and at some point that will be reflected in the share price. Actually, I think this will be evident as early as next week - more likely the end of this next week.
Yes, Anavex should move forward. Share price should increase.
Yes. Look at the long-term chart. How long did this stock stay at a low level? This is part of something I posted before about how one may make real money by investing in biotech:
”You have to be patient and you need to make sure that you do not lose your position because a stock like Anavex can have very sudden, abrupt moves. If you sell too soon, you may miss the big move. Anavex is attempting to do what no drug company has ever done before by finding effective treatments for CNS diseases that have no effective treatment. If it succeeds in obtaining drug approval or even looks like it will obtain drug approval, there should be a surge in share price...”
The way you may lose your position is to become impatient, sell too soon, and miss the sudden, abrupt surge the moment success/approval becomes apparent. Of course, the reverse may happen just as abruptly once a failure is evident. However, the AVXL share price is depressed. A short has little to gain compared to what may be lost on the upside. A long holding even a few shares may have little to lose for a shot at a big move.
I believe that if you want to make real money investing, you will have to do it by picking stocks in disruptive technology and biotechnology with emphasis on the latter. Of course, it is fraught with risks, but I think Anavex is increasing the odds that it will obtain approval of AVXL 2-73. You have to be patient and you need to make sure that you do not lose your position because a stock like Anavex can have very sudden, abrupt moves. If you sell too soon, you may miss the big move. Anavex is attempting to do what no drug company has ever done before by finding effective treatments for CNS diseases that have no effective treatment. If it succeeds in obtaining drug approval or even looks like it will obtain drug approval, there should be a surge in share price, but I certainly cannot predict what that will be. I will wait and see.
It may be that during the drug approval process Anavex will attempt to obtain approval for a label that may broadly cover seizures to result in AVXl 2-73 being covered by insurance for CNS diseases involving seizures.
No, off label use is not covered by private insurance. It is also my understanding that Medicare and Medicaid will not cover off label use. However, I have seen situations were some drug companies have promoted drugs off label to increase sales. As you know a doctor may write a prescription off label, but it is illegal for a drug company to promote its drugs off label. Pfizer paid fines in the hundreds of millions for promoting its drug Neurontin off label. I must rephrase this to say that off label promotion by a company of its drugs off label is illegal in the United States. Such promotion may be legal outside the United States.
Yes, we need to be patient, and you appear to be more patient than most. We want Anavex to succeed, and I believe the delay in the Rett trial is because Anavex has been collaborating with the Rett Foundation and the FDA to put together the most effective clinical trial it can under new FDA guidlines. Although the start of the trial has been delayed, it may be that all of this preparation involving the patient group and the FDA increases chances that AVXL 2-73 may be approved sooner. If the drug is approved for use for treating Rett patients, the drug will also be prescribed off label for a variety of CNS diseases that have no effective treatment.
Talon thinks the Rett trial will commence before that date. I agree.
World Orphan Drug Congress on April 25-27, 2018
April 25 - April 28
http://anavex.com/event/world-orphan-drug-congress-april-25-27-2018/
Sorry about my grammar. I have had a couple of glasses of wine tonight.
Well, Drs. Kaminsky and Missling would not be appearing together to announce that the preparations for the clinical trial and the trial itself is a dud. Moreover, this is telling in a very positive way:
“World Orphan Drug Congress USA 2018 - Conference Day One @ 11:25
Empowered educated patients influencing pre-clinical development and working with industry to optimize drug discovery
Patient groups role guiding research, disease understanding, supporting pre-clinical development and follow up experiments
Driving translational research and the influence on formulation, routes to delivery and overall clinical development
Effective partnerships between advocacy and industry to expand collaborations that can support enrollment and physician engagement
Christopher Missling, President and CEO, Anavex Life Sciences
Steven Kaminsky, Chief Science Officer, International Rett Syndrome Foundation”
This is indicating that the Rett Foundation and Anavex have been working together pre-clinically to optimize drug discovery, that the Rett Foundation (and possibly other patient groups) have been guiding research, disease understanding and supporting pre-clinical development. I do not mean to say just the Anavex pre-clinical development, but certainly the Rett Foundation is supporting Anavex. It mentions effective partnerships in doing so. And, as you point out, Steven Kaminsky, Rett’s Chief Science Officer is appearing for this presentation that you mention. Although we have had much negative comment about why the Rett clinical trial has not commenced, the Rett Foundation and its Chief Science Officer most definitely is not viewing the situation as negative!
The negative opinions and speculation on this board are not evidence, but this is evidence that is favorable for this clinical trial.
March 7 - 14 looks interesting for Anavex. Plus, April 25 - 27 may have something to offer regarding Orphan drug developments.
http://anavex.com/event/asent-20th-annual-meeting-march-7-10-2018/
30th Annual Roth Conference on March 11-13, 2018
March 11 - March 13
http://anavex.com/event/30th-annual-roth-conference-march-11-13th-2018/
Cowen 38th Annual Health Care Conference on March 12 – 14, 2018
March 12 - March 14
http://anavex.com/event/cowen-38th-annual-health-care-conference-march-12-14-2018/
World Orphan Drug Congress on April 25-27, 2018
April 25 - April 28
http://anavex.com/event/world-orphan-drug-congress-april-25-27-2018/
Either the low of tomorrow, March 1, or the low of Friday, March 2, may be lower than the low of today, $2.25. However, if that occurs, selling is likely exhausted, and the short term share price at least is likely to move up. In other words, I would not consider it to be that bad of a thing if we made new lows tomorrow or Friday. If this occurs, and there is no guarantee, the share price is likely to move higher beginning next week. I would then like to see other things develop next week to see if the move is considerably up from this very depressed level.
This is the lowest we have seen the share price since 2015, I believe, although the company is in a much better position than it was in 2015.
Personally, I would like to see the above (first paragraph) occur, but I know others on this board may go ballistic judging solely by lower low in the next two days. Of course, I am in this stock for the long term. I have kept abreast of the science, design of the clinical trials and more. I have a different perspective than those that view this stock short term. If I were short term, I would be very upset, but I am not.
Gottlieb Outlines the FDA’s Future Approach to Precision Medicine in Davos
February 5, 2018
The FDA plans to base future approvals of precision medicines largely on clinical trials that examine long-term durability and safety issues, Commissioner Scott Gottlieb said during a panel discussion at the World Economic Forum in Davos, Switzerland. Gottlieb said the key regulatory issues will be less about determining efficacy, especially with products demonstrating a strong proof-of-principle and early observations of effectiveness in small trials — and instead more focused on product issues related to potential off-target effects and their implications. The agency will also seek accelerated approvals for precision medicines designed to optimize benefits for particular groups of patients, especially through genomic or molecular profiling. “I think we’re really at an inflection point right now, where we’re … defining the modern rules for how these technologies are going to be regulated,” he said. “We’re going to be looking at accelerated approval endpoints for earlier approval on questions of efficacy, with more vigorous long-term follow up in some of these constructs where we have authority to do that under accelerated approval.”
Well, despite Christopher Columbus’s adventurous voyage and discovery of America in 1492, I suppose there are still some people somewhere that still think the world is flat and that there is no such thing as new discoveries. There may be nothing you can do to change their minds, but thank you Fireman for trying.
Yes, Anavex alluded to Illumina and its sequencing technology on Feburary 7. Saying this in part:
”The company has completed both RNA and whole exome DNA genome sequencing from ANAVEX®2-73-treated patients utilizing Illumina HiSeq 2500 Next Generation Sequencing (NGS) technology. The analysis of this data has characterized several genomic alterations in well-characterized targets that have the potential to be used as biomarkers, which the Company plans to use to identify optimal patients in forthcoming clinical trials. This novel, precision-medicine approach will enable enriched clinical trial populations, more robust regulatory submissions and better characterized clinical trial designs.”
See this from Febuary 7: Anavex Life Sciences Provides Clinical Programs Update and Reports Fiscal First Quarter 2018 Financial Results. http://anavex.com/anavex-life-sciences-provides-clinical-programs-update-reports-fiscal-first-quarter-2018-financial-results/
Of course, the significance did not hit home until Bio hit me/us in the face with the great post today. We all owe Bio a great debt of gratitude.
Superb!
Fireman: Excellent post!
Ok. I will bet. In the spirit of moving forward without looking backward, my bet is a move in share price to $4.68 between now and June, and a move to at least $7.00 before the end of this year. Now, please, don’t anyone on this board take my bet too seriously because I am making this bet like someone betting on a sporting event. In other words, it’s just a bet, right? However, I must warn everyone that I consider myself to be very lucky, and I am very lucky simply to be alive and well today. So, remind me in June whether I met the first target, and do the same after December 31, 2018. And if you are inclined to ridicule me for being wrong, do that too because I can take the heat. With all of that in mind, I wish everyone the best, and most of all I wish for advancements in treating all CNS diseases.
Yes, someone, including most of us as well, thinks AVXL will be a winner. It should be. “And the world will be a better place for you and me. You just wait and see.” Have a good weekend.
I fully agree. If anyone watched the Davos panel that included the new head of the FDA, Anavex did precisely what that panel discussed about more efficient clinical trials and faster approval. Anavex improved its chances or odds for approval of its drug. Here is my key quote that is aligned with what was discussed at Davos: 'We are expecting to leverage these important findings to more precisely target patients based on specific genomic profiles that may be highly responsive to ANAVEX®2-73,” said Christopher U Missling, PhD, President and Chief Executive Officer of Anavex. “This should allow us to improve upon existing trial designs and expedite other aspects of our clinical development program.” '
From the indicators, it looks to me like we have confirmation that AXXL selling is exhausted, and we should see the share price increase from here.
However, overall it looks like the long bull market where we saw the Dow keep making record highs has or is about to end. It has been the practice "to buy the dips", but there is a lot of fear in the market as exhibited by CBOE Volatility Index. The DOW is swinging widely. The "buy the dips" strategy is no longer a good or comforting approach for investors.
Yes, I am 76 and work out with a trainer 3 days a week, cycle 2 days a week, jog and do other things outdoors 2 days a week. Recently, Outside Magazine had a pretty good article about probiotics and athletic performance. Those foods you mentioned are good. I have kefir each morning - good source. I have to cut this short because I cycle this morning.
Talon: Thank you. I agree. Anavex must be striving for a ACTC candidacy spot, which I think it highly likey and will happen meaning additional funding for Anavex with more exposure or attention for its AD trial.
I believe you are correct. It is a good buy although that may not be any comfort to those that are down. I am down on my AVXL investment, but I am staying the course. I will get to the end of this tunnel. I have my shades ready so the bright light will not hurt my eyes.
That is a good one. Some good humor sometimes helps. Thank you.
As Yogi Berra said: "When you come to a fork in the road, take it" & "It ain't over til it's over" That sums it up for me. I have taken the fork in the road, and it is not over until it is over.
We have embarked on a journey to discover the unknown. How to effectively treat AD, PD, Rett Syndrome, and possibly other serious and complex diseases. We feel like we have come to a fork in the road, and we do not know which way to turn. However, if you are to complete the journey you must remain hopeful for treatments that are presently unknown. This is a long and arduous journey -- far from over. No one should have expected it would be easy in the first place. To accomplish something no one has accomplished cannot be easy. If it were obvious and easy to treat these diseases, someone would have already done it. No one has although many have tried. If you know of a better fork or path to take, take it. Or, you may be frustrated, and you just want to quit. I am not one to quit, and I do not know of any better way. I will, win or lose, continue on this trip I commenced several years ago.