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Yes, Patrick Cox deserves credit. He too has been unjustifiably scorned at times because of the Semmelweis effect as explained in posts by Dado and others. New scientific findings that contradict and disrupt current beliefs or practices are I suppose naturally opposed by the status quo that are firmly entrenched in what the conventional thinking is of their time. The status quo economically and socially benefit from this. This is sometimes referred to as the Semmelweis effect that Dado pointed out in a post yesterday. Semmelweis was a doctor that discovered in the 1800s that if a doctor performing birth deliveries washed hands with an antiseptic solution before doing doing so it would prevent serious infections that were resulting in deaths. Doctors at his time resented his discovery. His discovery was not recognized until after his death. He died in an asylum from being cruelly beaten by guards. Ironically, it is suspected he was committed to an asylum because he suffered from what is now recognized as Alzheimer's.
Yes, change is afoot. The Cures Act passed this week. It may be that someone like Jim O'Neill may be appointed to head the FDA. As Xena mentioned recently: In a 2014 speech, Jim O'Neill said he supported reforming FDA approval rules so that drugs could hit the market after they’ve been proven safe, but without any proof that they worked, something he called “progressive approval.” The FDA is now caught in a vortex of reform. Of course, Big Pharma (BP) and consumer groups (probably funded by BP) will oppose changes at the FDA that will foster competition for BP. It may be we who are for innovation and drugs that really work, as opposed to simply protecting markets for billion dollar drugs that may not even work (BP's plaque removal drugs for example), may have an advantage because of these changes we are seeing.
Yes, if you were a scientific researcher that recommended to a large pharmaceutical company that the focus should be on removing amyloid plaque and that company spent billions of dollars pursuing clinical trials to develop drugs to remove amyloid plaque, wouldn't it be a little difficult to admit that you may have been wrong?
Could you get fired? Should you be fired? No, because the world is flat and amyloid plaque is the sole cause of Alzheimer's, and silly little Anavex could not possibly have a drug that is in any way effective to treat Alzheimer's. What's more, Anavex has no funds to speak of -- only a few million dollars. Impossible! It takes many billions of dollars to do these trials correctly. Please send more money. We need just a few more billion, and we will get a new block buster Alzheimer's drug on the market.
Yes, thank you. The Times They Are A-Changin'. However, most haven't gotten it yet. You did. Why wouldn't the FDA not favor the adaptive design if done correctly and more accurate it is more efficient and it makes the FDA's job easier? Additionally, more pressure is being exerted on the FDA to speed up its drug approval process -- witness the Cures Act. How can the FDA be more efficient in drug approvals if it clings to the old design?
These are not actual, but projected. Likely, overly optimistic for the upcoming report.
I'm not sure about that. I know of drugs with a large percentage of their sales off label. One example from the past: the epilepsy drug Neurontin.
Yes, I agree. The Senate votes this week on the Cures Act. It should pass. If so, faster FDA approval of drugs to treat these rare diseases for young patient populations is expected. If AVXL 2-73 obtains any approval for any disease, the FDA will be able to rely on data in the clinical trial obtaining approval to approve use of the same drug for other diseases as well (Cures Act specifies that). In any event, a physician may write a prescription for off label use (Alzheimer's for example) of any drug that the FDA has approved for any use.
Great observation. That also explains why it's taking so long and another reason to be patient. We want Anavex to give it the best shot.
Dose optimization: I remember that Missling said that the AVXL 2-73 was yet to be optimized. This factor could result in improved results in the future trial assuming safety is maintained with increased dosage. I am interested in knowing more about this and perhaps other things that have been learned to improve benefits -- besides mono-therapy?
I plan on not raising my expectations to such a high level that I may be disappointed in the news we are about to hear. We should keep in mind that if the clinical trial demonstrates that at 41 weeks the participating patients in this trial have at least maintained a cognitive and activities level above what Alzheimers' patients traditionally or historically experience without AVXL 2-73 that is an encouraging and successful result. Heretofore, nothing has stopped or slowed the progression of this disease, and Alzheimer's patients have continued to decline over such a period of time. Having said that, I am hopeful that a larger phase 3 trial exhibits even better AVXL 2-73 effects. It is becoming clearer to me that mono-therapy is the path going forward. Perhaps also some other lessons have been learned that may be of benefit in the larger trial.
Makes sense that this inference is correct. He most likely intended to convey "combination".
Thank you.
Thank you. I agree.
Frrol:
Is this consistent with your interpretation?
https://pbs.twimg.com/media/CoADe8XUMAIiq0O.jpg
Raja: Where did Pitor obtain the chart in this first tweet below or did he create this?
https://mobile.twitter.com/PiotrPietrzkiew/status/805036090088361984/photo/1
Also, it looks like the chart in the below tweet projects that at 41 weeks AVXL 2-73 alone brings patient improvement up to close to healthy?
https://pbs.twimg.com/media/CoADe8XUMAIiq0O.jpg
What is your take on this?
Yes, remember Galileo was imprisoned for holding the belief that the Earth revolves around the Sun. His belief went against all convential thinking during his time.
I suspect that today there is a bias and dependency of researchers's careers in pursuing and defending theories they have espoused. Billions have been spent on the amyloid palque theory. It's difficult to admit an error or failure to any degree. It may be that an amyloid plaque removal drug will eventually prove to be helpful after we address the root cause of Alzheimer's disease. However, up to now the amyloid plaque hypothesis has been a failure.
No drug to date has demonstrated any treatment to even slow the progression of Alzheimer's disease. The latest reports we have received about the AVXL 2-73 clinical trial suggest that patients in this small study have not experienced the expected ongoing cognitive decline normally expected in Alzheimer's patients. Indeed, the reports this summer and fall claim that AVXL 2-73 clinical trial patients show "improvements on tasks within the Cogstate battery''. This suggests that there is hope that AVXL 2-73 may prove to slow the progression of Alzheimer's disease in some patients. If so, that is a first as difficult as it will be for convential thinking to accept.
Yes, intriguing ('Therapeutic Response Unexpected'). Increased independent activity and better coordination are two that seem to me to be most unlikely to be due to a placebo effect.
16 events reported I think needs explaining.
Does anyone know if events like this have ever been reported in any other clinical trial of an AD drug?
'Therapeutic Response Unexpected'. A sample of verbatim terms reported include:
Improved Engagement with Family/Friends
Improvement in Mood
Increased Independent Activity
Feeling Happier
Improved Alertness
Better Coordination
Better Coping
If the Cures Act passes (the House votes on it Wednesday), AVXL 2-73 for Rett may have faster approval. Here are some key provisions:
1. Authorizes innovation prize competitions to advance biomedical science for diseases that are serious and represent a significant burden. (Section 2002)
2. Provides opportunities for new researchers and promotes earlier research independence. (Section 2021)
3. Supports high-risk, high-reward research that has the potential to transform the scientific field. (Section 2036)
4. Expedites the development and review of drugs for subgroups of rare disease patients by authorizing FDA to rely on data submitted in previously approved applications involving similar gene targeting technology. (Section 3012)
5.Reauthorizes the pediatric priority review voucher program incentivizing the development of drugs for rare pediatric diseases. (Section 3013)
If the FDA approves the drug for Rett, physicians may then write prescriptions off label.
Cures Act
I received a long email from the Energy and Commerce Committee regarding the Cures: 21st Century Cures Bill Released... the U.S. House of Representatives will vote this Wednesday. Here's some parts of the email I received:
House Will Vote Wednesday on $6.3 Billion Game-Changing Medical Innovation Package, Including New Research Funding, Mental Health Reforms, and State Grants to Fight Opioid Abuse....
WASHINGTON, DC – House and Senate health committee leaders have released the final "21st Century Cures” bill and announced that the House of Representatives will vote Wednesday on a $6.3 Billion landmark medical innovation package that will accelerate the discovery, development, and delivery of new cures and treatments and provide new funding for the National Institutes of Health and Food and Drug Administration.
Among other things, the act is to provide and $1.6 billion for the BRAIN initiative to improve our understanding of diseases like Alzheimer's and speed diagnosis and treatment.
If I am not mistaken, I think federal laws and regulations prohibit a company from selling a drug that the FDA has not approved. They have to provide it for free. Many small companies cannot afford to provide experimental drugs to the general public for free. This is why the federal laws and the FDA need to be dealt with. However, there will be serious lobbying by the major pharmaceutical companies to block reformation. The structure of the FDA and the federal drug laws serve big pharma well. The federal laws and the FDA serve as a barrier to entry of innovative drugs by small companies. BP does not like competition for the blockbuster drugs in their pipelines that are protected by some form of exclusivity -- patents, etc. That is why BP file phony patents, pay generics to stay off the market (Google pay for delay), file phony patents in the FDA Orange Book, etc. to block competition.
I sent this to the WSJ:
Dear Daniela:
I have been a long time WSJ subscriber. I encourage you and others to become more comfortable in researching and writing about innovative drugs that have possibilities rather than to continue to dwell on mainstream big pharma drugs that do not work. The Lilly "throw in the towel" PR this week about its amyloid plaque drug, solanezunab, is a capitulation that the amyloid theory approach to combatting Alzheimer's has not worked to arrest the Alzheimer's epidemic.
''While there is no cure, prevention or treatment to slow the progression of Alzheimer's disease, there are five prescription medications approved by the U.S. Food and Drug Administration (FDA) to treat its symptoms.''...... http://www.alz.org/i-have-alz/treatments-and-research.asp. None of the five FDA Alzheimer's approved drugs work to alter the progression of the disease.
On Nov. 22, 2016 of this very same week Anavex Life Sciences Corp. (AVXL) announced a positive 41-week update from its Phase 2a study in mild-to-moderate Alzheimer’s disease (AD) patients for ANAVEX 2-73, which targets cellular homeostasis.
Anavex Life Sciences Announces Data on 41-Week Treatment of ANAVEX 2-73 for Patients with Alzheimer’s Disease - Investigational Treatment suggests to curb Cognitive and Functional Decline.
Read more: http://www.nasdaq.com/press-release/anavex-life-sciences-announces-data-on-41week-treatment-of-anavex-273-for-patients-with-alzheimers-20161122-00252#ixzz4QwSDtLT5
''At 41 weeks, Alzheimer’s patients taking a daily oral dose of ANAVEX 2-73 in the exploratory, not yet dose optimized Phase 2a clinical trial, showed a stabilization of cognitive and functional measures. This data of stabilization is promising since Alzheimer’s disease is a progressive disease where current therapeutics are only able to temporarily slow the worsening of dementia symptoms and not stop the disease from progressing.''
It is interesting too that Anavex and Lily are scheduled to be present at the 9TH CLINICAL TRIALS ON ALZHEIMER'S DISEASE (CTAD) DECEMBER 8-10, 2016. This seems to me to be an opportunity for a report about an alternative to the plaque amyloid theory.
Why not focus more on what may work than what does not work?
Thank you.
Charles Schwab and Institutional investors investing in Anavex recently.
http://www.mideasttime.com/schwab-charles-investment-management-inc-invests-313000-in-anavex-life-sciences-corp-avxl/1398706.html
Charles Investment Management Inc. purchased a new stake in Anavex Life Sciences Corp. (NASDAQ:AVXL) during the second quarter, Holdings Channel reports. The institutional investor purchased 51,200 shares of the company’s stock, valued at approximately $313,000. Schwab Charles Investment Management Inc. owned 0.14% of Anavex Life Sciences Corp. as of its most recent filing with the SEC.
Several other institutional investors have also bought and sold shares of the company. American International Group Inc. acquired a new position in Anavex Life Sciences Corp. during the second quarter valued at approximately $115,000. Nationwide Fund Advisors acquired a new position in Anavex Life Sciences Corp. during the second quarter valued at approximately $130,000. Rhumbline Advisers acquired a new position in Anavex Life Sciences Corp. during the second quarter valued at approximately $192,000. Teachers Advisors Inc. acquired a new position in Anavex Life Sciences Corp. during the second quarter valued at approximately $314,000. Finally, California State Teachers Retirement System acquired a new position in Anavex Life Sciences Corp. during the second quarter valued at approximately $418,000. Institutional investors and hedge funds own 11.06% of the company’s stock.
Thank you. This is all very good and encouraging.
Is AVXL 2-73 better or worse than the Alzheimer's drugs the FDA has approved up to now?
Previous to AVXL 2-73 clinical trials THIS WAS SAID:
''While there is no cure, prevention or treatment to slow the progression of Alzheimer's disease, there are five prescription medications approved by the U.S. Food and Drug Administration (FDA) to treat its symptoms.''...... (amyloid and such)
...Alzheimer's or dementia may change your sleep patterns. You may have difficulty sleeping, take daytime naps, and/or experience other shifts in your sleep pattern. Researchers are not sure why these sleep changes occur.
http://www.alz.org/i-have-alz/treatments-and-research.asp
In July of this year the AVXL 2-73 clinical trial results were updated SAYING THIS:
''In a disease state where progression is invariable over time, a sustained or stable MMSE and ADCS-ADL score is considered a positive outcome.''
“The demonstration of an extended period of both cognitive and functional stability out to 31 weeks in a patient population that would normally be expected to experience ongoing cognitive decline is an encouraging milestone in the development of ANAVEX 2-73. The 31-week data also validates the earlier observation of improvements on tasks within the Cogstate battery. The specificity and consistency of these benefits suggest that ANAVEX 2-73 can sustain activation of attentional and working memory functions with repeated dosing in Alzheimer’s disease,” said Associate Professor Stephen Macfarlane, FRANZCP, Head of Clinical Governance, Dementia Centre HammondCare, who is conducting the study. “Of noticeable interest was also HAM-D data showing beneficial effects of ANAVEX 2-73 on insomnia, agitation and anxiety at 31 weeks, which might suggest an additional important role of ANAVEX 2-73 for the amelioration of behavioral and psychological symptoms of dementia (BPSD).”''
http://www.anavex.com/?news=anavex-presents-31-week-efficacy-data-from-phase-2a-study-of-anavex-2-73-in-alzheimers-patients-at-aaic-2016
''...no statistically significant decline from baseline was reported, which is impressive.”
http://www.anavex.com/?news=anavex-confirms-data-from-phase-2a-study-of-anavex-2-73-in-alzheimers-patients-presented-at-aaic-2016
In a very recent interview Missling said this about AVXL 2-73 (from Xena's summary)
4) 2-73 is believed to address underlying cause and prevent progression
5) 2-73 works by restoring homeostasis of cells
6) Sigma 1 not utilized on a daily basis - is a safeguard.
7) 2-73 restores balance through s-1 receptor - Missling specifically states that there is a possibility to regain cognition
So, what may be expected from here? Do we expect AVXL 2-73 to be better than the 5 Alzheimer's drugs approved by the FDA up to now? Do we expect the December reported results to produce indications that AVXL 2-73 may be better than the 5 AZ drugs approved so far? I think so.
I think the big barrier to entry or drug approval in the United States is the FDA. The FDA needs reform and restructuring to speed up the approval process for drugs that are (1) safe, (2) potentially beneficial for dreaded diseases, and/or (3) potentially beneficial for diseases or conditions that presently have no effective treatment.
And, why should there be no credit or recognition what so ever for drugs that are approved for use in humans in other countries that are not approved in the United States. The FDA has in the past refused, without requiring costly prohibitive U.S. clinical trials, to even approve European sunscreens in the United States although these sunscreens have been used for years in Europe and in some cases proven to be more effective in blocking harmful UVA rays than sunscreens approved in the United Stares. I bought a sunscreen to use while in Europe, but I am not allowed, thanks to the FDA, to buy and use the same sunscreen in the U.S.
I suppose being a human in Europe is not the same as being a human in the United Stares.
Yes, I think it helps to some degree. The midweek announcement produced an opportunity to spook the herd because the short entourage had it set up them to cause a stampede. Announcing it on Saturday gives the herd time to digest news and to weigh the information. It, for example, gives this board as well as others a chance to comment on the results in a calmer environment and to come to our senses.
Following up on my last post, this is what I posted on Nov. 1.
"Remember that Missling and Nell Rebowe will be at the Global Mizuho Investor Conference (MIC NY). November 14, 2016. This conference is by invitation only. There will be some one on one sessions and sessions where there will be groups. I tried to register for this conference, fly to NY, and see if I could find out first hand what is going on with Anavex. I was also interested in attending a group session where drug pricing is to be discussed. I doubt there will be any publicity emanating from the conference, but it is a good venue for networking with companies in the biotechnology business."
I do not expect much, if anything, to be divulged about the conference on Monday. It is an invitational only event and is closed to all others that have not received an invitation. It is, however, a good opportunity for Anavex to network.
I really do not know. I can only speculate that results of the clinical trial were coming together to show something in common. It may mean several things. One may be that it may be Donepezil and 2-73 in combination may not be the way to go, but that is purely a guess. Or, it may simply be that it is now more likely that 2-73 alone has (regardless of Donepezil) has more affect on the disease. Of course, there are many other possibilities. Not knowing what you know, we can only guess.
I believe Xena is correct. We may may take a few more steps backwards, but odds are we take more steps, if not leaps, forward.
Remember that Missling and Nell Rebowe will be at the Global Mizuho Investor Conference (MIC NY). November 14, 2016. This conference is by invitation only. There will be some one on one sessions and sessions where there will be groups. I tried to register for this conference, fly to NY, and see if I could find out first hand what is going on with Anavex. I was also interested in attending a group session where drug pricing is to be discussed. I doubt there will be any publicity emanating from the conference, but it is a good venue for networking with companies in the biotechnology business.
What is significant near term is that Biogen's resources are behind this, and any positive announcements along the way about the Biogen/AVXL 2-73 study of AVXL 2-73 for demyelinating diseases should move Anavex share price up near term even though the process as a whole will take quite some time.
Millstone:
Thank you for your insight. I am sure you are correct the more I think about it. Biogen would not be a party to some deal that was simply thrown together. It was carefully done by skillful people at Biogen that have expertise in deal making, licensing, and the science involved. It is a big deal.
Yes, and many times when you strike up a new romance, you may not kiss on the first date. Relationships as a rule take time.
“As we looked at how the landscape was evolving ...... and the corresponding fit with our strategic priorities about where we could best allocate resources, based on our own expertise and competency, ...... it wasn't as good of a fit as other things which we could allocate our resources towards,” said Biogen EVP of R&D Michael Ehlers on the Oct. 26 call about the decision.
The above quote illustrates what I was trying to say in my last post last night when I said this about the Anavex - Biogen collaboration: "Many people within Biogen that have expertise have mulled this over very carefully. This is not to say that succes by Biogen in its efforts here for treating this complicated disease is in any way a given, but it definitely gives a great deal of credibility to the Anavex science. Biogen's decision was not lighty made.''
Although this is certainly encouraging, my understanding is that Biogen will first perform tests in a laboratory setting. If laboratory results are positive, this may lead to human trials. AVXL 2-73 safety in humans will help. The companies would also likely obtained fast track approval for use in treating MS and other similar CNS diseases involving the myelin sheath that protects neurons or nerves in the human body. This will all take time. The FDA regulatory wheels grind slowly.
What is significant is that Biogen's resources are behind this, and any positive announcements along the way about the Biogen/AVXL 2-73 study should move Anavex share price up near term even though the process as a whole will take quite some time.
A rehash of the Biogen collaboration, etc.:
Biogen is investigating whether Avxl 2-73 is useful in restoring the myelin sheath or providing some alternative to protecting neurons in place of the myelin sheath.
http://www.webmd.com/multiple-sclerosis/guide/ms-or-als#1:
''Nerve cells in your body are wrapped in thin coverings called myelin sheaths. They protect these cells, similar to how insulation protects electrical wires.
When you have MS, your body attacks the myelin sheaths in your brain and spinal cord.''
MS is but one of the demyelinating diseases being looked at.
Biogen will have to either acquire patent rights from Anavex or do a partnership with Anavex if it desires to commercially pursue the use of AVXL 2-73.
Biogen is not expending the effort of this investigation just for the heck of it. This investigation is expensive. Many people within Biogen that have expertise have mulled this over very carefully. This is not to say that succes by Biogen in its efforts here for treating demyelinating diseases is in any way a given, but it definitely gives a great deal of credibility to the Anavex science. Biogen's decision was not lighty made.
Another way to look at the deal, is that Biogen is doing at its expense what Anavex would otherwise have to do at its expense to gain approval from the FDA to use AVXL 2-73 for MS and other demyelinating diseases.
What does Anavex have to lose in this Biogen deal? What does Anavex have to gain? Is Anavex a better bet today because of the Biogen deal? Is it a better bet for other reasons -- Michael Fox Foundation (Parkinson's study), the Rett Syndrome Foundation (Rhett Syndrome study). Should investors be willing to take a risk along with Biogen, the Michael Fox Foundation, and the Rett Foundation? All of the entities mentioned have put their money on the line along with Anavex as well as the the Australian Government relative to Alzheimer's.
With all of these developments, would you be surprised to see the Anavex share price trade in its higher ranges? I would not.
Congratulations that you are in a position to do that, and I certainly hope it works out well for you. I have for quite some time had, for me at least, a substantial position. I bought a few more shares today at $3.07 and $3.06. I wish you and all longs well. Most of all, I am hopeful for a treatment for the serious CNS diseases that the Anavex science may address.
Rocalinda: Thank you. Another good part in addition to what you put in bold: "Anavex confirms that there has been no change with regards to its science, data or the fundamentals of the Company."
It is a company whose website says it has developed a medical device that does this:
COGNISION™
While numerous QEEG/ERP studies have been successfully performed in research settings around the world, these studies have generally lacked the diagnostic reliability necessary to be clinically useful for most physicians. This is primarily due to the inherent difficulty in implementing research-oriented equipment on a scale large enough to obtain the high accuracy and repeatability rates needed for clinical use. Furthermore, the complex research-oriented hardware and methods are not practical enough to be used in real-world clinical settings.
The COGNISION™ System was designed from the outset to overcome these limitations. The system is designed to be easy and practical enough to use in a physician’s office, while also being able to support the large-scale data collection requirements of the most demanding research applications. Additionally, the extremely difficult task of analyzing the QEEG/ERP data is automatically accomplished with Neuronetrix’s proprietary pattern recognition software, reCOGNISION™.
The COGNISION™ System is a highly integrated, internet enabled, cognitive assessment system designed to perform validated QEEG and ERP tests in normal office settings. The COGNISION™ Headset is battery-powered and wireless to be patient- and user-friendly. The COGNISION™ Software System has many advanced features to facilitate automated testing and analysis even in large, multicenter clinical trials.
Both Rebowe and Missling will be involved in both conferences on November 13 and November 14.