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Biogen - Anavex: Biogen will test Anavex’s lead drug candidate, ANAVEX 2-73 in an oligodendrocyte precursor cell (OPC) differentiation assay. This sounds like "Regenerative Advanced Therapy'' as designated in the Cures Act to me.
NEW YORK, NY, September 28, 2016 – Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental diseases including Alzheimer’s disease, other central nervous system (CNS) diseases, pain and various types of cancer, today announced that it has signed a material transfer agreement with Biogen (Cambridge, MA) under which Biogen will test Anavex’s lead drug candidate, ANAVEX 2-73 in an oligodendrocyte precursor cell (OPC) differentiation assay. A satisfactory result from the OPC assay study may lead to an in vivo remyelination study using a chemical demyelination model.
“Battling demyelinating diseases such as multiple sclerosis requires an understanding of the processes that cause remyelination to fail. Remyelination of demyelinated axons is typically a function of oligodendrocyte precursor cells. These studies will examine the therapeutic role ANAVEX 2-73 may play in permitting remyelination in the brain,” said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex.
BioGen testing Anavex's ANAVEX 2-73 in an oligodendrocyte precursor cell differentiation assay should fall within this definition:
This sounds like cell therapy to me as mentioned in the Cures Act:
''The drug is a regenerative medicine therapy, which is defined as a cell therapy, therapeutic tissue engineering product, human cell and tissue product, or any combination product using such therapies or products, except for those regulated solely under Section 361 of the Public Health Service Act and part 1271 of Title 21, Code of Federal Regulations; ''
Cures Act. The Regulatory Explainer for the Act is located here:
http://www.raps.org/Regulatory-Focus/News/2016/11/28/26242/Regulatory-Explainer-21st-Century-Cures-Redux-and-What-it-Will-Mean-for-FDA/
It has many interesting sections that may benefit Anavex. One example is:
Sec. 3011 Qualification of Drug Development Tools
This section also follows on from FDA’s prior work from 2012 in which the agency created a new program known as the Drug Development Tools Qualification Program to qualify — i.e. validate as being useful and appropriate—new biomarkers, clinical outcome assessments, patient reported outcomes and animal models.
This section actually establishes a review pathway at FDA for such biomarkers and other development tools that can be used to help shorten drug development times, aiming to help reduce the high failure rate in drug development.
Under this section, FDA would be required to make publicly available on at least a biannual basis on its website, the following:
All drug development tools qualified, including all surrogate endpoints which were the basis of approval or licensure (as applicable) of a drug or biological product;
information on each qualification submission under the qualification process;
whether external scientific experts were utilized in the development of a qualification plan or the review of a full qualification package;
formal written determinations in response to such qualification submissions;
summary reviews that document conclusions and recommendations for determinations to qualify drug development tools
- See more at: http://www.raps.org/Regulatory-Focus/News/2016/11/28/26242/Regulatory-Explainer-21st-Century-Cures-Redux-and-What-it-Will-Mean-for-FDA/#sthash.SHYTK2oM.dpuf
Cures Act. See this website for a summary that I think is pertinent to AVXL 2-73:
https://multiplesclerosisnewstoday.com/2016/12/22/21st-century-cures-act-may-speed-approval-stem-cell-therapies-for-multiple-sclerosis/
This article mentions a definition for regenerative medicine and advanced therapies :
Under the Cures Act, “regenerative medicine and advanced therapies” are defined so as to include gene therapy, gene-modified cell therapy, therapeutic tissue engineering products, human cell and tissue products, and combination products that use these therapies or products, according to an “explainer” on the new law released by the Regulatory Affairs Professionals Society or RAPS.
What is that combination therapy Biogen is studying in combination with AVXL 2-73?
I think it meets all three based on Jimmy's explanation.
Regenerative Advanced Therapy -- Cures Act. It has to meet all three. Here is a blog that discusses this.
Advocates and opposition of the 21st Century Cures Act alike waited with bated breath throughout December to learn if President Obama would approve the act into a law before the New Year, and as of December 15 he did. This massive piece of legislation will have far reaching impacts on numerous components of medical research, product development, and health care delivery. It is an especially important law for regenerative medicine experts and patients alike, given its goal of accelerating the time it takes new regenerative medicine techniques and devices to enter the market. Many have lauded the 21st Century Cures Act as an important victory for patients and doctors, though it certainly isn’t without its opposition.
A New Category is Born: Regenerative Advanced Therapy
One of the most important aspects of the 21st Century Cures Act for regenerative medicine advocates is the new option for regenerative medicine products be categorized as “regenerative advanced therapy,” If designated as such, the products become eligible for priority review by the FDA. Regenerative advanced therapy is considered a product aimed at treating or curing a serious disease, especially with the potential to provide currently unmet medical needs. In addition, the product must fall under the Act’s definition of “cell therapy, therapeutic tissue engineering products, human cell and tissue products, and combination products using any such therapies or products.”
Since it seems that the majority of regenerative medicine products in development meet these standards, the 21st Century Cures Act offers an exciting opportunity for regenerative medicine to skip over time consuming roadblocks on the path to meet patient needs. Anything currently in stages of development to help resolve serious conditions like paralysis, MS, dementia, and so much more could be soon available to patients in desperate need, and this is a huge step of progress in the medical world.
Wide Ranging Benefits
The implications of more accessible regenerative medicine treatments are impressive. First, the number, length, and complexity of clinical trials would be reduced because the FDA would allow sponsors to conduct confirmatory clinical trials after the product or technology becomes available on the market. This means that patients can receive life-changing treatments sooner, and the companies are still held liable for their promises because they can have their accelerated approval withdrawn if their products do not achieve expected responses.
Furthermore, as observed by FDA Commissioner Robert Califf, the Act will create “a research network and a public-private partnership to assist developers in generating definitive evidence about whether their proposed therapies indeed provide clinical benefits that are hoped for.”
Overall, assuming that the FDA leadership requires compelling and high-quality scientific evidence before providing accelerated approval, patients with debilitating conditions will have a realistic chance of obtaining revolutionary regenerative medicine treatments to change their lives forever.
http://www.regenerativemedicinenow.com/21st-century-cures-act/
You get it, and you got it first!
Life is a but a series of moments as Emily Dickson related to in her poem “Forever is composed of moments.” It is important to make the best of our moments during our lives, and it is important that we remember our moments. This is why we do things like taking photographs or keeping diaries to capture moments that we desire to remember and share. Alzheimer’s is a disease that robs the afflicted of the series of moments composing their lives -- moments that they may recall and share in their old age. That’s cruel. Hopefully, this cruelty will be eliminated soon. That's my wish for 2017.
Why is there usually a relatively large block purchase at the very end of the trading day like today at 29,555 shares?
I have been a longterm Anavex stockholder. Anavex was claimed by many, who have come and gone, to be a sham. Such was alleged in a lawsuit that has been dismissed with prejudice never to see the light of day again. All the while these negative allegations have been made and pending, I continued to accumulate shares.
I sold some shares on pops only to buy more after a price drop. I did my due diligence before purchasing shares, and I continue to study the fundamentals and technicals day by day. I make every attempt to manage my expectations and exercise necessary patience. I have no idea how long it may take for Anavex to succeed or fail, but I have learned to persist and plan to prevail. I am here for the duration never to be shaken by a Plato or a Satan, but I will increase my position as they too are forsaken.
I agree. Good point.
Right. I believe it is inappropriate at this point for Anavex speak about a patient's decision to "switch" to mono-therapy. Anavex cannot / should not make any statements about a patient's choice. Such statements may also unduly stress other patients and and interfere with their physicians' decisions about treatment decisions. It may be comforting to you as an investor, but Anavex should not do that. It cannot say anything directly or indirectly that suggests AVXL 2-73, an unapproved drug, is superior to SOC.
I am convinced that mono-therapy is so far is proving to be superior. Any "ambiguity" as you say may be explained away by what I said in my previous post. # 86519.
I believe the decision to discontinue any other therapy of any patient participant in this or any trial must be a decision made by each patient and the patient's physician. Furthermore, it may be that this would need to be reported to the FDA. It is questionable whether anyone that is not the patient's physician would recommend that the patient discontinue an FDA approved drug prescribed by the patient's physician and take instead a mono-therapy that has not been approved by the FDA. I see serious implications in pursuing the path of "switching" a patient to an unapproved mono-therapy -- "efficacy" has not been established! Anavex would, in my opinion, be foolish to "switch" patients to the mono-therapy and announce it to the world as you say. Frankly, I do not think Anavex can legally do that. Moreover, I do not think a physician would risk "switching" a patient from an approved drug (SOC) to an unapproved drug!
Perhaps the PR people, including Missling, are not aware of yesterday's order of dismissal especially during this holiday period when many people within the chain of communication are out of pocket.
This 3 year extension is an extension of "FDA exclusivity" irrespective of any patent:
"Other" Exclusivity - 3 years for a "change" if criteria are met:
• Granted to drug when application or supplement contains reports of new clinical investigations (not bioavailability
studies) conducted or sponsored by applicant and essential for approval
• Runs from time of NDA approval
• Bars FDA from approving, for a three year period, any ANDA or 505(b)(2) application that relies on the information supporting the approval of the drug or the change to the drug for which the information was submitted and the exclusivity granted
• Described in 21 CFR 314.108
It is a good time to accumulate shares at this price. The volume is low. It is a holiday week. I am buying.
Excellent! A smart move.
Not yet. Traveling today.
It may refer to the collaboration between Anavex and the Rhett Foundation.
It may refer to the collaboration between Anavex and the Rhett Foundation.
Yes, I suppose we need to ask Anavex investor relations. I did some searches as well and could not find it.
Yes, apparently Anavex holds the trademark for the term SIGMACEPTOR™ , which is good, but patents are better. Take a look at page 6 of the 10k:
"Patents, Trademarks and Intellectual Property
Anavex holds ownership or exclusive rights to three issued U.S. patent and eight U.S. patent applications with various international counterpart applications, all of which relate to drug candidates and methods associated therewith. The most recent U.S. patent application was filed in November, 2016. We regard patents and other intellectual property rights as corporate assets. Accordingly, we attempt to optimize the value of intellectual property in developing our business strategy including the selective development, protection, and exploitation of our intellectual property rights."
The above is from page 6 of the 10k Anavex Annual report. What about this new patent application filed last month? We need to know more about what that covers?
"Patents, Trademarks and Intellectual Property
Anavex holds ownership or exclusive rights to three issued U.S. patent and eight U.S. patent applications with various international counterpart applications, all of which relate to drug candidates and methods associated therewith. The most recent U.S. patent application was filed in November, 2016. We regard patents and other intellectual property rights as corporate assets. Accordingly, we attempt to optimize the value of intellectual property in developing our business strategy including the selective development, protection, and exploitation of our intellectual property rights."
The above is from page 6 of the 10k Anavex Annual report. NOTE THAT THE MOST RECENT PATENT APPLICATION WAS FILED NOVEMBER 16, 2016! What is this patent about?
CBD: You did the right thing buying today.
Great! Thanks. So far, I have sent that article to several members of congress as well as Barron's Editors. I will send more this article that Mike found:
http://www.mdedge.com/ehospitalistnews/article/120066/alzheimers-cognition/sigma-1-agonist-presses-forward-after-positive
I think you have convinced me. Persistence pays off. I think Cramer should at least read this article that mike found:
http://www.mdedge.com/ehospitalistnews/article/120066/alzheimers-cognition/sigma-1-agonist-presses-forward-after-positive
I second that!
I think Missling will be pursuing government grants, especially in this new environment with passage of the Cures Act, etc.
You may be correct, but isn't Cramer's tv audience mainly small investors that call in with questions. Wouldn't Missling rather spend time with potential partners like Biogen, the Rhett Foundation, Fox Foundation, and other similar entities that may fund his various trials? I'm not saying what you suggest is not a good idea, but it seems Missling may be more inclined to do what he has been doing to secure funding.
Did you see some years ago where Jon Stewart slammed Jim Cramer for Jim's shady days as a hedge fund trader? I think you may still be able to Google for that and watch it. You may learn something about Jim Cramer.
I believe that KOLs are likely heavily endowed by large pharmaceutical companies. Therefore, although they proclaim to be independent, they are dependent. I begin to learn this years ago when I realized the tentacles of a large. Chemical company within colleges/universities throughout the U.S. Large companies fund various departments of colleges/universities with grants. They also constantly sprinkle small "gifts" to individuals in key departments of these institutions whether it be in the form of dinners, entertainment, devices, etc. Many of these so-called scientific experts are biased.
An excellent synopsis of the success and progress of the AVXL 2-73 Alzheimer's drug clinical trial. Thank you.
I will have to reply more about this later. But just for now, the U.S. FDA exclusivity only has application in the U.S. Therefore, the Greek patents have no relation to the FDA exclusivity, which FDA exclusivity starts to run once the FDA approves the drug in the U.S. Also, the assignment of the invention by the inventors may cover more than just any patents the inventors may have filed in Greece or elsewhere. We are not privy to the assignments, but I am just thinking the assignments were very broad. Also, I really do not know that much about Greek patents -- how long they run, etc. Am I making sense though about everything else other than the Greek patents that I may try to discover more about?
Following up, sorry may last reply was not in response about what you asked regarding the 2015 patent. I think the 2015 patent likely does only relate to melanoma.
Yes, I agree. However, I am not sure about the Greek patents. Where these covered in the assignments that I just mentioned in my post to bsparks?
Yes. My understanding is that no other competitor in the U.S. (the most desirable drug market in the world) will be able to copy the chemical entity (AVXL 2-73) during the FDA exclusivity periods whether it be straight New Chemical Entity (NCE) or chemical entity for Orphans Designation. And let's face it: What other company is as far along as Anavex on its FDA trials for this drug.
Moreover, other competitors may be spooked or blocked patent wise because (1) Anavex has an assignment of from both inventors of the invention although one inventor was controversial or possibly contesting his assignment (U.S. patent statutes only require an assignment from one of the two inventors -- remember the SA article about that?) (2) It is my understanding that Anavex has a U.S. Patent Application on file for AVXL 2-73 mono-therapy alone (per Orveko -- where is Orveko?) that may be revived at any time.
And, Anavex I am sure has calculated the FDA exclusivity the periods as you say to arrive at something like 7-9 years exclusivity based on FDA exclusivity alone without U.S. patent protection that you and I know Anavex is bound to be pursuing along many lines -- not only in the U.S. but also elsewhere. Anavex has competent patent counsel. I believe it is reasonable to think AVXL 2-73 will have more exclusivity through patents as well as the U.S. FDA exclusivity I have mentioned.
Exclusivity/Patent protection.
I have posted something like this before, but here it is. Anavex will have some form of exclusivity for AVXL 2-73 for AD alone not to mention the exclusivity it may have for all of the other uses. It certainly has 5 years FDA exclusivity in the U.S. if there is efficacy for the treatment of AD and FDA approval is granted. However, Anavex is approaching approval for Rhett Syndrome first. Anavex has Orphan Drug Designation for AVXL 2-73 for Rhett. Orphan Drug Designation means faster approval of the drug for that use (for Rhett), and the exclusivity period for an Orphan Drug approval is 7 years. What's more , is that any FDA approval of a drug for any use will open the door for physicians to write off label prescriptions for that durg for any use whether it be for Rhett, Alzheimer's, Parkinson's or anything else.
It may also wind up with 20 years exclusivity from the date of filing (maybe more with patent extensions) patent protection because of the application it has on file in the U.S. about AVXL 2-73 as a monotherapy for AD, which is potentially revivable at any time. Remember too that Anavex allegedly has some foreign patent protection for AVXL alone including, but not limited to, that once controversial Greek patent assigned to Anavex.
Anavex has competent patent counsel. I believe more patent protection will be forthcoming.
If efficacy is proven, any way you look at it Anavex has some exclusivity and a valuable drug.
Patent protection is governed by the Patent and Trademark Office and generally, runs 20 years from the date of filing (there may be some extensions of the 20 year period and some I mention below). FDA approval involves varying periods of exclusivity as follows:
Orphan Drug (ODE) - 7 years
New Chemical (NCE)- 5 years
"Other" Exclusivity - 3 years for a "change" if criteria are met
Pediatric Exclusivity (PED) - 6 months added to existing Patents/Exclusivity
Patent Challenge – (PC) – 180 days (this exclusivity is for ANDAs only)
Exclusivity revisited. Anavex will have some form of exclusivity for AVXL 2-73 for AD alone not to mention the exclusivity it may have for all of the other uses. It certainly has 5-7 years FDA exclusivity in the U.S. if there is efficacy for the treatment of AD and FDA approval is granted. It may also wind up with 20 years from the date of filing (maybe more with patent extensions) patent protection because of the application it has on file in the U.S. about AVXL 2-73 as a monotherapy for AD, which is potentially revivable at any time. Remember too that Anavex allegedly has some foreign patent protection for AVXL alone including, but not limited to, that once controversial Greek patent assigned to Anavex.
Anavex has competent patent counsel. I believe more patent protection will be forthcoming.
If efficacy is proven, any way you look at it Anavex has some exclusivity and a valuable drug.
Patent protection is governed by the Patent and Trademark Office and generally, runs 20 years from the date of filing (there may be some extensions of the 20 year period and some I mention below). FDA approval involves varying periods of exclusivity as follows:
Orphan Drug (ODE) - 7 years
New Chemical (NCE)- 5 years
"Other" Exclusivity - 3 years for a "change" if criteria are met
Pediatric Exclusivity (PED) - 6 months added to existing Patents/Exclusivity
Patent Challenge – (PC) – 180 days (this exclusivity is for ANDAs only)
Do not be the least bit dissuaded by the usual critics such as AF who are suffering from the Semmelweis effect: We may expect to be hearing the same old opposition arguments: 1. It is a small study and proves nothing (as if there is no such thing as an adaptive and efficient FDA trial). 2. Many other Alzheimer's clinical trials initially showed some promise only later to fail in phase 3 (as if we should not conduct any more clinical trials to treat ALzheimer's -- and ironically at the same time these critics will be writing bits of encouraging reports about amyloid plaque studies by Biogen and others). 3. Anavex is too small and does not have sufficient capital and resources to complete a successful phase three (as if might is always right). 4. The Anavex clinical trial has been conducted in the wilds of Australia of all places (as if this should be a negative). 5. We should be skeptical of this Mc fellow conducting this trial (as if he as never been associated with the likes of Lilly or Big Pharma). 6. That Big Pharma is key to the R&D for genuine innovation and the the role of guardians of our health (as if Big Pharma never does anything to block drug competition such as Pay for Delay -- Google that for more information). 7. That AVXL 2-73 is claimed to treat AZ "symptoms" of all all things (as if this should be a negative -- glossing over the fact that all currently approved FDA drugs for Alzheimer's only treat symptoms -- none of which FDA approved drugs I believe relieve insomnia and anxiety prevalent in the disease).
Yes, there will continue to be opposition. Unfortunately, the Semmelweis syndrome is a disease in itself that is not quickly cured.
Yes, I am familiar with what helped your arthritis. I believe it is certainly safe. It is unfortunate the FDA is keeping it from us.
I subscribe to Patrick Cox's Transformational Technologies news letter. I have also attended John Mauldin's Strategic Investment Conference for the past 6 years. Patrick Cox joined Mauldin Economics a few years ago. I have met Patrick. He is a sincerely genuine hardworking analyst. His work is beneficial to society especially age related diseases. Patrick believes aging is a disease. If we focus on transformational bioscience drugs to treat aging and age related diseases, we will solve the healthcare crises. A drug like AVXL 2-73 is one example.
Despite Patrick's good work, he is attacked by people like this AF and others that oppose new scientific findings that contradict widely held beliefs and practices even when it should be evident to them that those current practices are not working. We can expect more opposition to AVXL 2-73 , but we should not be dissuaded by these attacks.