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Bourbon and K9: Yes, Anavex is convinced and this former Senior VP of Regulatory Affairs is convinced. We may only speculate, but they know more than we know, and they believe they can obtain a FDA approval for an Anavex drug. Of course, someone may say that this does not mean they will be successful in their efforts, but those that say that certainly do not know what Anavex and this former FDA official knows. I agree no one can say it is a certainty that the FDA will approve anything. You can be sure that this FDA person and Anavex discussed all of the know data -- data this board has speculated about both negatively and positively. At the very least, this is one expert's indirect confirmation that the data is positive. Folks can discount this all they want, but one senior person with 24 years FDA experience thinks Anavex has something worthwhile.
Correct and that the product meets manufacturing regulatory requirements. A product may be approved, but it has to illustrate that it can manufacture the drug in commercial quantities in accord with good standards. The manufacturing facility and process is important to assure continued safety standards, that the drug manufactured contains correct doses as represented, etc.
The U.S. Food and Drug Administration’s Office of Regulatory Affairs (ORA) is the lead office for all agency field activities. ORA inspects regulated products and manufacturers, conducts sample analyses of regulated products and reviews imported products offered for entry into the United States. In pursuit of its mission, ORA also works with its state, local, tribal, territorial and foreign counterparts.
Someone thinks something will get approved. Senior VP of REGULATORY Affairs.
Whiskey:
You are having a difficult time. I understand that. We all have those times at some point in our lives. It seems it may be a good idea to take some time off and not post for a while. However, you have been with this stock for a long time. I remember your many good comments. I have been long for several years. I have had good times and bad times. I have had my ups and downs. However, I think we need to carefully consider that we are now closer to the finish line that may lead to a victory of sorts. We only do not know what that victory may be or when it may happen. Hang in there! Give yourself a break, but also consider giving AVXL some more time. Take care.
You raise some interesting points -- robotics, AI, and the like. This is the future, which is occurring at a faster pace. Kopin is well suited for this. I too doubt China and the USA will pull back from each other benefiting from the transformational advances we will see in these areas even though there may be some posturing from time to time.
Kopin has invested much of its capital in securing various patent protection, and I think that is one reason why it has not reported net earnings that may in the short term boost its share price. It has been building value that is not reflected in the share price. Kopin's intellectual property is well suited for robotics, AI, self driving vehicles, self flying planes, drones, etc.
Robots will perform many more jobs and functions whether for medical and hospital purposes, security, military, manufacturing, construction, transportation, home, etc.
With advances in biotechnology world populations are living longer. As populations continue to age Kopin's technology will be used in our homes and elsewhere. Wearable technology will help the elderly and their families in every day life. Robots will assist and provide company for the elderly and disabled. A son, daughter or caregiver can place sensors and devices around a home and on the person to keep track of elderly parents. These devices will enable instant communication as needed.
Kopin Completes Strategic Agreement with Goertek
Business Wire Business WireApril 24, 2017
WESTBOROUGH, Mass.--(BUSINESS WIRE)--
Kopin Corporation (KOPN) (“Kopin”) today announced that it has closed its previously announced transaction with Goertek Inc. (“Goertek”), receiving approximately $24.6 Million dollars in gross proceeds from Goertek in exchange for issuing approximately 7.6 million shares of Kopin stock. Goertek is a leading innovative global technology company headquartered in Weifang China that designs and manufactures a range of consumer electronics products for brand customers including wearables, virtual and augmented reality (VR and AR) headsets, and audio products.
Wrong!
It is from a subscription service l use, but I paraphrased/summarized the report.
Monday April 17 voting deadline. Kingsdale Advisors contacted me today. I voted all of my shares for all of management's recommendations on directors and proposals. They seemed to be anxious to receive my votes. It took several phone calls. I have more than one account, and Kingsdale had to conference me in with my online brokerage service to obtain a control number for me to vote shares held in that online account. They seemed to be very appreciative that I voted for all proposals. I definitely got the feeling that they need votes for management's proposals to pass. Does anyone know where the vote stands?
Take this for whatever it is worth, and it may not be worth much.
It is reported that GALT is due out with its topline NASH (Nonalcoholic Steatohepatitis) CX trial results in December this year. The company has hinted at the possibility of a Breakthrough Therapy designation if they meet their regulatory endpoints on Hepatic Venous Pressure Gradient (HVPG) and Fibrosis. There seems to be a feeling that there may be a pathway toward provisional approval as they outlined their plans to break the patient groups up into subsets for a follow on phase 3 trial. My understanding is that a few points of improvement in HVPG may result in provisional approval.
Dr Traber said “the study had a rigorous FDA agreed design that measures many parameters in patients with NASH cirrhosis who have not had serious complication and are not yet candidates for a liver transplant.” This FDA agreement may be highlighted to show a lot went into the design of this trial to get an agreement on acceptable endpoints. If you read into this statement a little deeper you can see the FDA may essentially be saying get us the HVPG data and if it meets these endpoints we are going to give you provisional marketing approval with follow-up data.
The trial was designed for 156 and in a way was oversubscribed. Having 152 patients left in the trial at this late stage is encouraging since if you assume the current dropout rate continues then only 5 more patients would be expected to drop out over remaining 5 months of the trial ending in August. The reason this point is important goes to the power of the study which should be approximately 95%. It is difficult for patients to undergo biopsies but for a good reason -- like the maybe the patients feel it is worth the effort. It was previously assumed that there would may be a 25% drop out rate.
There is a hint -- a mere hint--that maybe the end points of the Galt study are being met for possible provisional approval.
Further, the Methacetin breath test employed by Galt in its trial is being evaluated as a diagnostic that measures the level of liver damage. Positive data on this less invasive breath test could be a blessing for all drug companies in the NASH space if it also reduces the need for a liver biopsy. For GALT, if provisional approval is given in December it could potentially mean having to do away with HVPG which is a costly and arduous test in its Phase III trials and allow for the quick recruitment of patients in phase III.
Please do your own due diligence on all of this, and do not rely on what I have said here. My comments are pure speculation based on my understanding of what I have read, which may not be accurate. Furthermore ,I/we can not predict the future of success of the GALT clinical trial no matter what. GALT is a dangerously speculative investment. If the results of this clinical trial fail, those invested in GALT may lose their entire investment.
AXON - strange I think. The stock is trading up pre-market at about $19.46, but the morning news for the AXON is: BASEL, Switzerland, April 11, 2017 /PRNewswire/ -- Axovant Sciences Ltd. (NYSE: AXON) ("Axovant"), a leading clinical-stage biopharmaceutical company focused on the treatment of dementia, today announced the pricing of its underwritten public offering of 6,742,179 of its common shares at a price to the public of $18.54 per share. Gross proceeds to Axovant from the offering are expected to be approximately $125.0 million, before deducting underwriting discounts and commissions and estimated offering expenses. All of the common shares are being offered by Axovant. In connection with this offering, Axovant has granted the underwriters a 30-day option to purchase up to an additional 1,011,326 of its common shares on the same terms and conditions. The offering is expected to close on April 17, 2017, subject to customary closing conditions.
The 180 day lockup restriction pursuant to rule 144 for IPOd PLSE shares will be lifted the first week of May. PLSE shares will likely decline substantially at that time due to a liquidation or sale of a large number of shares formerly restricted.
Yes. I own it. It is not exactly a good comparison with AVXL 2-73 because PLSE is a medical device company. It is easier to gain approval for a medical device versus a drug. Initially, dermatologist will demand PLSE's device for melanoma treatment. However, in the process of testing this device, it was discovered that cancer cells elsewhere in the body were attacked and destroyed by the body's immune system. My simple lay way of explaining this is that normally our immune systems cannot recognize cancer cells. Something is needed to initiate the immune response. Missling sometimes makes references or comparisons to immunotherapy by its drug candidates initiating a response in the body that allows the body to heal itself -- acting on sigma receptors, etc. -- is the way I understand what Missling is saying. It could be that I am simply ignorant and am making the right investment decisions (hopefully) for the wrong reasons, but this is how I am looking at it. Anavex, in my opinion, is a good speculation, but it is longer term than PLSE.
Relative strength breakout as of yesterday! Bullish!
A primary system I use is giving a relative strength buy signal.
Conceivably, AVXL 2-73 may obtain additional exclusivity because of this: House Bill 1223
Bill to Amend FD&C Act to authorization extension of exclusivity periods for certain drugs that are approved for a new indication for a rare disease or condition. Introduced by Rep. Bilirakis (R-FL); Rep. Butterfield (D-NC); Rep. McCaul (R-TX)
This bill was introduced Feb. 27. The full text is not available yet.
Two House Bills Introduced 2/27 to Amend FD&C Act re: pediatric cancer and rare disease approval process and exclusivity periods.
Following up on the President’s speech last night about speeding up FDA approval, I looked up two bills that have been introduced to amend the Federal FD&C Act that may have an impact on FDA approval process and exclusivity periods for pediatric cancer and rare disease drugs. Both bills were introduced on February 27, 2017 and are slated to go before the House Energy & Commerce Committee.
The full text of each bill is not yet available.
House Bill 1223
Bill to Amend FD&C Act to authorization extension of exclusivity periods for certain drugs that are approved for a new indication for a rare disease or condition. Introduced by Rep. Bilirakis (R-FL); Rep. Butterfield (D-NC); Rep. McCaul (R-TX)
House Bill 1231 (Senate Bill 456 is companion bill)
Bill to Amend the FD&C Act to establish program to increase the development of new drugs to treat pediatric cancer
Introduced by Rep. McCaul (R-TX); Rep. Butterfield (D-NC); Rep. Clarke (D-WY); and Rep. Duffy (R-WI)
Can Anything Be Learned From Alzheimer's Drug Failures?
http://www.seekingalpha.com/article/4047215
Efficacy. A very good reason why AVXL needs to demonstrate efficacy:
"...some of the names Trump is reportedly considering for the critical FDA commissioner job. One potential candidate, Peter Thiel associate Jim O'Neill, has advocated a system where drugs can be approved the minute they're shown to be safe, rather than the dual safety and effectiveness standards which the agency must now consider.
But that radical approach has already shown poor results in the marketplace. Sarepta Therapeutics (SRPT, +0.22%), which won a controversial but pioneering approval to treat the rare disease Duchenne muscular dystrophy, has been shunned by a number of insurance companies because (as the FDA admitted in its own approval) the treatment doesn't have proven efficacy."
http://fortune.com/2017/02/15/trump-fda-deregulation-biopharma/
It's true that bad news travels like wildfire. If it were bad news, we would know it. Look, we have three trials that haven't even started. There's no news to be had with that. And, any calamity would be reported. I think remain cool, calm and collective. Don't try roller skating in a buffalo herd that gets spooked ever now and then. This is a highly volatile stock, but I like it.
Correct. All three AVXL clinical trials will be funded. It is time to change the subject to something more constructive.
About Noble for those, like me, that do not know much. Noble Life Science Partners focus is:
''NOBLE LIFE SCIENCE PARTNERS COMBINES SCIENTIFIC KNOWLEDGE AND INDUSTRY EXPERIENCE WITH CAPITAL MARKETS EXPERTISE TO ACCELERATE THE GROWTH AND PROFITABILITY OF UNDERVALUED EMERGING GROWTH COMPANIES IN BIOTECHNOLOGY, SPECIALTY PHARMACEUTICALS AND MEDICAL TECHNOLOGY''
https://noblecapitalmarkets.com/life-science#lsp-focus
It is a division of Noble Financial Capital Markets. It is a private company and is a full service investment banking firm dedicated to the small cap market. It provides services to emerging growth companies as well as mergers and acquisitions advisory, securities placement, networking services, etc.
Thanks. That's a very sensible observation.
What is the placebo effect?
Even though they don’t act on the disease, placebos affect how some people feel. This happens in up to 1 of 3 people. A change in a person’s symptoms as a result of getting a placebo is called the placebo effect. Usually the term “placebo effect” speaks to the helpful effects a placebo has in relieving symptoms. This effect usually lasts only a short time. It’s thought to have something to do with the body’s natural chemical ability to briefly relieve pain and certain other symptoms.
https://www.cancer.org/treatment/treatments-and-side-effects/clinical-trials/placebo-effect.html
The placebo effect may in some people (1/3 it says above) relieve symptoms. However, can the placebo effect cure a patient, either temporarily or permanently? If one had a clinical spinal cord drug trial in progress, and patients begin to walk when prior to that they could not walk, can that be due to the placebo effect?
And, "The World Will be a Better Place."
No, Noble says the presentation will be available approximately 24 hours following the the live presentation, meaning if you are present at the conference site you will hear his presentation otherwise you must wait for about 24 hours. That's Noble saying that. Not Anavex. It's Noble's conference.
Anything is possible, and I'm not bothered by the combination patent pending with the United States patent office for approval. This patent when issued may not help, but I certainly do not see how it hurts anything. And, it may very well help. It is common practice for drug companies to obtain as many patents as possible. My best guess is that Anavex's strategy is to obtain approval for AVXL 2-73, if possible, for Rett Syndrome, which will give 7 years Orphan Drug Protection. In the meantime, I expect many more patent applications will be filed and other exclusivity protection for 2-73 will be pursued.
Anavex has very competent patent and IP counsel. These people should keep everything confidential. That is their duty, and Anavex should not waive any confidentiality whether it is the attorney client privilege or anything else. Once waived, that privilege no longer exists.
I also believe that Anavex is justified in not disclosing it's full and complete patent strategy. Why do that? That may temporarily make some investors happy, but it only aids potential competitors in the long run. I believe it is in our best interest that Anavex maintain confidentiality about all of this. Does anyone expect Anavex to waive confidentiality? Does anyone expect Anavex to disclose its trade secrets and all other matters that if it otherwise keeps confidential is best for the company and its investors?
Personally, I would be shocked if Anavex spilled the beans about any of this that should remain confidential.
Let's see if Missling says mote about this Monday at the Noble conference. Perhaps someone should suggest to the PR people that he do so.
Yes, the more care and skill spent in identifying responders the more likely the success in obtaining approval, but this requires a great deal of work and study. Possibly a large number of candidates must be considered and screened to discover those that are more likely to respond. Anavex does not have to establish that AVXL 2-73 is effective for every possible, probable or proposed patient. It just has has to establish that it is effective for a sufficient number of patients as compared to the placebo. This takes time. It is enormously expensive to conduct a clinical trial as we all know. It is best to take time beforehand and take the best shot. Any company, especially a tiny company, should take the time to determine the clearest path to gain success for such an expensive endeavor.
This may explain why it is taking so long to start phase 3 for AVXL 2-73. This is from the article you referenced:
''Problems can arise with randomization, drug supply, and the recruitment of patients when there are adaptive changes due to dose selection, sample-size increases, or population enrichment. It is critical to ensure that the sample size at the interim analysis is adequate for making the adaptive decision. If patients are enrolled too rapidly relative to the time needed to observe the primary end point, the planned enrollment might be completed before adequate information is available for an adaptive decision to be taken. To date, regulatory agencies have opined favorably about adaptive designs.
FUTURE OF ADAPTIVE TRIALS
More widespread use of adaptive trial designs could accelerate the discovery process, especially if coupled with other evolving trial concepts, such as large, simple trials. Advances in adaptive trial design will require further dissemination and acceptance of the sometimes complex statistical methods. There is an intuitive appeal of adaptive trial design and its attempt to identify the patients who are most likely to derive benefit from a therapy, and this feature will resonate well with most doctors and patients."
It takes time to "attempt to identify the patients who are most likely to derive benefit from a therapy''. However, identifying patients that will benefit is key to regulatory approval of a drug for those patients, and I can see how this resonates well with doctors and patients.
People have negatively speculated about the delay in the announcement of the start of phase three -- about it taking so long, but this article gives a possibly rational explanation why it takes time and planning to do an adapative design. However, the time and planning increases the odds that the drug will be approved. Once approved, the drug will be used for all Alzheimer's patients becuase presently there is no effective treatment for Alzheimer's. All of this is consistent with what Missling has said about adapative designs.
See this: http://www.nejm.org/doi/full/10.1056/NEJMra1510061#t=article
The FDA only acts in a ministerial capacity. It does not decide whether a patent is valid and enforceable. Courts do that. You surmise correctly about the possibility of blocking of generics. Big pharma has used Orange Book listing of "patents" to block generic competition. Big pharma has been sued for violating antitrust laws for wrongfully listing patents in the Orange Book.
See this as one example: Wrongful "Orange Book" Listing Raises Red Flag with FTC; Leads to Consent Order with Biovail Corp. Concerning its Drug Tiazac
https://www.ftc.gov/news-events/press-releases/2002/04/wrongful-orange-book-listing-raises-red-flag-ftc-leads-consent
There are other cases where companies were sued for wrongful Orange Book listings to block generics. Another examplw, BMS was sued regarding blocking generic competition for Buspar.
I like your post.
''The only thing constant is change.'' Heraclitus
And...
“Uncertainty is the only certainty there is, and knowing how to live with insecurity is the only security.”
? John Allen Paulos
My best judgment is that it is premature for anyone to say anything definitively.
Most great investments begin in discomfort. The things most people feel good about – investments where the underlying premise is widely accepted are unlikely to be available at bargain prices. Rather, bargains are usually found among things that are controversial, that people are pessimistic about, and that have maybe have performed badly for a long while (look at the long term chart for AVXL - AVXL's stock performed badly for a long time). Buying AVXL stock has at times been uncomfortable. Sometimes it is uncomfortable owing AVXL stock. It is NEVER easy to do things that entail discomfort, but being uncomfortable comes with the territory when investing in new ideas.
Innovative ideas like this, AVXL's new forms of drugs, have to be lonely. By definition, non-consensus ideas that are popular and widely held or obvious are an oxymoron. Thus, such new ideas are oxymoron and uncomfortable. Non-conformists don’t enjoy the warmth that comes with being at the center of the herd. Unconventional ideas often appear imprudent because what AVXL is doing is something that is NOT "what everyone does.” You will never make any serious money doing what everyone else does. Most of the other drug companies, big pharma, are doing something else -- something else that's not working.
This is really the bottom-line: whether you dare to be different. Do you dare to look wrong. Any new, innovative, revolutionary idea has to go through the stage of looking wrong because you are doing something that no one else is doing.
As an investor, don't worry about the herd thinks or is doing. Do diversify your investments (don't invest money here that you cannot afford to lose). However, do not believe that you should never buy so much of something that if it doesn’t work or that you will look bad. If you worry about temporarily looking bad you must understand that it will keep you from buying enough of something that works for it to make much of a difference for the better.
Continue to do due diligence. I do that. So far, I think Anavex may be disruptive. Remember Kodax? A Kodax employee first developed a digital camera, but Kodax rejected this new technology. Kodax could not fathom change. Kodax went bankrupt. I will maintain my long position waiting to see what results emerge. However, it will take time. In the meantime, I struggle to have patience. I watch and listen, but I ignore all of the noise. I have been long for several years. Although I have bought and sold small amounts here and there, I have continually increased my position. I think I am right. If you think you are right, sit tight!
There is also a New Clinical Investigation Exclusivity – 3 years. A clinical investigation that provides a "new" basis for approval of an application can qualify for this exclusivity.
Gburgin, the answer to your question about whether significant and successful off label use for AD (once AVXL 2-73 is approved for Rhetts) would bring in the generic makers is no as long as any exclusivity is pending. No generic may be authorized by the FDA as long as any exclusivity is in force for AVXL 2-73 whether it be FDA exclusivity and/or patent exclusivity. Patents too are listed in the FDA's Orange Book in the FDA's ministerial capacity. As long as any patent for AVXL 2-73 is listed in the FDA's Orange Book, it will not authorize a generic version of AVXL 2-73 for any use.
Anavex may list a patent for AVXL 2-73 in the FDA's Orange Book after the Rhetts approval is granted and before any exclusivity expires. The 7 year FDA exclusivity period for approval of this drug for Rhetts gives Anavex 7 years to develop more patent protrction.
And yes, I do think that there may be additional FDA exclusivity for this drug for the treatment of Alzheimer's or any other disease besides Rhetts; however, I need to confirm this.
Yes, Reyeton, you are correct and that example of an off label prescription for arthritis as well as the severe side effects, which makes it understandable why your wife declined to use that prescription, is good one. Comparing that that drug to AVXL 2-73, we have evidence of the safety of AVXL 2-73 as well as evidence of less side effects. Plus, there are many alternative drug treatments for arthritis, but no effective drug to treat Alzheimer's for example.
Despite what some have said on this board, FDA orphan approval of AVXL 2-73 for Rhetts is a big deal. It will mean 7 years FDA New Chemical Entity exclusivity, and you can bet that doctors will write off label prescriptions for conditions other than for Rhetts. The latter is especially true if the clinical trials for Alzheimer's, Parkinson's, etc., are demonstrating positive results. Plus, Anavex may in the interim file for and obtain longer patent protection. You can bet that families will be requesting prescriptions from doctors for the dreaded diseases that AVXL 2-73 may benefit. Who wouldn't request an off label prescription for AVXL 2-73 for Alzheimer's when no other drug on the market is actually working? And, doctors will write those off label prescriptions as I know they have written off label prescriptions for other drugs in the past.
Yes, doctors may write off label prescriptions, meaning writing prescriptions for diseases that are not intended by the FDA approved label. If AVXL 2-73 gains FDA approval for Rhetts, doctors may write prescriptions all the conditions you mention plus they may write prescriptions for Alzheimer's, Parkinson's, and other diseases. However, Anavex may not promote its drug off label and may not advertise its drug for any condition other than for the condition the drug has been approved. I know of instances where substantial sales of a drug have occurred from doctors writing prescriptions for that drug off label.
Thank you. I realize now that my post was inaccurate. I was going back in my patent notes, and I picked up the wrong patent numbers in my attempt to identify the newest patent filed.
Patent filed the latter part of 2016?
It will be interesting to see what the most recent Anavex patent application is about. This is U.S. patent application 15/194792. It has not been published yet, to my knowledge. Perhaps someone more astute than me may elaborate. Orveko commented about this sometime the latter part of last year:
''1) U.S. patent application 15/194792 -
Title and information not yet known (this patent has not been published yet), however this patent application is a child of both 14/395581 and 14/865862, so is likely related to Anavex 19-144 and/or Anavex 2-73.
2) U.S. patent application 14/395581 - "Optimization and therapeutic valorization of the symptomatic treatment of alzheimer's disease with rivastigmine, galantamine or donepezil, by selected aminotetrahydrofurans acting as mixed sigma-1 / muscarinic ligands"
Priority date: 03/28/2013
Filing date: 04/03/2013
Publication date: 03/12/2015
3) U.S. patent application 14/865862 - "Prototypical brain protective activity of tetrahydro-n-methyl-2,2-diphenyl-3-furanomethanamine (AE37Met)"
Priority date: 03/28/2013
Filing date: 09/25/2015
Publication: date 01/14/2016 ''