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Anavex has enough cash (on hand and acces to additional (ATM) if required) to advance the trials. My belief is that they will enter into a marketing, sales and distribution agreement, highest bidder, when ready to commercialize.
Investment for a life-time and generations after. We may have lost some of the initial battles but we will win the war and build an upire! Can't refute the science.
GLTALongs
Spot on! The science is right. In the end that is what matters most.
The good Dr. is executing a masterful plan. We will se announcements soon for trial initiation for Rett's, PK and Ph-3 AD. This will blow up rapidly and there will be not looking back.
Longs, don't fall into their game and give up your shares too early. Abviously, this is only my opinion and you most do what you need to do. Just don't let them fool you.
GLTALongs
Good idea. This will be better than an insurance policy for elderly care.
GLTALongs
I do believe as well that Dr. Missling is pasionate about providing relief to those who suffer, he had family members afflicted with AD, and that is his top priority.
That said, he is also an astute business man and has always been very consisten and clear when asked abouf partners, funding, etc.: "we will do what is best for the shareholders".
We are in good hands.
I share your interpretation.
The Dr. has been very clear about funding not being an issue (no dilution). I believe him.
My key take aways from the cc:
- There is no doubt that A-273 is at this stage better than anything treating AD.
- Reafirmation that trials for Rett, PK and AD (Ph-2/3) will be the focus for Q1. Hopefully we will have at least 2 of them started in the next couple of months.
- $17M is enough to fund coporate overhead and some of the trials off the ground, but not enough in my opinion to complete the efforts. However, th Dr. sounds very confident that the money is sufficient for the next 2 years so he is definetly counting on additional cash coming from grants or partnerships.
- We will see volatility for the next few days but my belief is that flippers will be moving on and we will start to see more long-term / institutional investors. The potential is too great to not be taken seriously by the market.
GLTALongs
Great add to the discussion. Thank you!! Sad.
From the Australian article:
Because the drug targets homeostasis regulation to help a person’s immune system fight neurological conditions, Anavex Life Sciences chief executive officer Christopher Missling is hopeful it may also prove beneficial for other neurological disorders, including Parkinson’s disease and multiple sclerosis.
There was also encouraging news for biotech company Biogen in its development of dementia drug Aducanumab this month, with early stage results indicating it had reduced the build-up of amyloid plaque in Alzheimer’s patients’ brains.
I read combo, partnership!!
You are correct. They are obligated by SEC rules. While to us the whole world should know what is going on, only those of us with direct interest know about the CTAD poster.
Expect great action tomorrow!
From medical writer attending CTAD:
https://twitter.com/Alz_Gal
I would not be surprised to see highest PPS ever before year end- >15.
Nothing would make me happiey, as investor and human, than to be hear the sweet word with no caveats. This rose in its present state smells better than any other rose in the garden-:)
No wonder Dr. Missling has been more confident in latest interviews: "we will have a PH-3 trial for A-273". "Funding not an issue". He knows what he has and is now in a much stronger position to negotiate partnership.
The detractors will sure be out again with their bogus claims. While they may still scare away the usual weak hands, long-term investors have just been given many reasons to remain believers. Can't wait for Monday's market reaction which I expect to be extremelly positive.
GLTA
That would be a mistake. Let the results speak for themselves without making a statement that yet has some way to go to be true. We are definetely on the right path thou.
This speaks louder than any statistics. Fantastic!
PATIENT EVENTS: THERAPEUTIC RESPONSE UNEXPECTED
101001 MORE ALERT REGARDING SURROUNDING
101002 FEELS MUCH HAPPIER MAKING JOKES
101003 MUCH HAPPIER WHEN ATTENDING CLINIC APPTS AND ENJOYS MAKING JOKES
AND ENGAGES WELL IN CONVERSATION
101004 BETTER HAND COORDINATION. CALMER AND MORE COMMUNICATIVE
101006 IMPROVING MOODS. READING MORE BOOKS
101007
ABILITY TO PLAY THE PIANO AND READ MUSIC NOTES AT ABOUT 9 MONTHS
INTO TRIAL. SHE USED TO PLAY THE PIANO AT AGE 5 AND LOST HER ABILITY PREALZHEIMER
TRIAL
101010 ABLE TO FOLLOW PLOT WHEN WATCHING MOVIES WHEREAS PREVIOUSLY
COULD NOT
101010 MORE COMPASSION FOR CHILDREN
101011 WIFE THINKS PATIENT IS A BIT MORE CHEERFUL
101013 ABLE TO DO MUCH MORE HOUSEWORK THAN BEFORE
101013 MORE DRIVEN AND UPBEAT LESS ANXIOUS ACCORDING TO CARER
101014
AN INTERNATIONAL ARTIST WHO RESUMED HER PAINTING ABILITIES AND NOW
HAVING AN EXHIBITION IN NOV 2016. WRITTEN A 3 PAGE LETTER TO LONG LOST
BROTHER
101015 PLAYING MORE GOLF NOW BY HIMSELF. MORE CONFIDENT AT GOING OUT BY
HIMSELF
101017 ENJOYED HER TRIP TO BELGIUM - TALKS ABOUT SOME BITS OF HER TRIP
102001 IMPROVED ENGAGEMENT WITH FAMILY/FRIENDS/OUTSIDE WORLD
102008 IMPROVEMENT IN MOOD
102010 FEELING GREAT - IMPROVEMENT IN COGNITION AND MOOD, BALANCE AND
GAIT HAS IMPROVED
103001 PATIENT REMEMBERING SOMETHING HE WOULDN'T HAVE PREVIOUSLY
Great presentation / data.
Curious about the disclosure about paid speaker and consultant fees (Eli Llily?)
This interview is from July 2015. The author was portrayed by the Cabal as being a paid promoter (part of the photo printing scam) and for faking his credentials as a Dr. They ignored the science and everything else as we have come to know. The reason that they are no longer out in force as previously, is that their arguments no longer hold water. Dr. Missling is executing exactly as he has been telling everyone willing to listen, which all of us long-term holders are. I did not edit the patent stuff since it is good to reaffirm everyone that there are no patent issues. Good look back and how the plan continues to evolve.
Kanak Kanti De - Let me see if I understand the science correctly. Is the primary function of Anavex 2-73 to correctly fold back misfolded protein? By stimulating the sigma-1r chaperone? Is there anything else that it does?
Christopher Missling - Correct, however, additionally targeting Sigma-1 receptor and muscarinic receptors, which ANAVEX 2-73 does, is believed to increase cellular plasticity and reduce oxidative stress, inflammation, abeta generation and tau hyperphosphorylation (Note: Please see the publications section on Anavex's website and the following publication for protein misfolding.)
KKD - And adding Aricept to it - that is to reduce the plaque that's already been built up by existing misfolded protein, correct?
CM - Reducing the abeta plaque is also a function of sigma-1 R. Adding Aricept (donepezil) is believed to increase acetylcholine, a messenger for memory and hence work synergistically with ANAVEX 2-73.
KKD - So, A2-73 may work in early stages of the disease, when plaque buildup isn't considerable, but needs a plaque reducing agent in more advanced stages?
CM- Possibly, however, preclinically ANAVEX 2-73 might be sufficient to also reduce abeta by itself. Hence, could be applicable both in MCI and mild-to-moderate Alzheimer's disease. The current Phase 2a is in mild-to-moderate Alzheimer's disease.
KKD - However, since there's no biomarker to properly identify onset of Alzheimer's disease, A2-73 as a monotherapy doesn't work right now, and you need donepezil?
CM - That answer will come from this current Phase 2a. However, preclinical evidence shows ANAVEX 2-73 works very well alone and might have a further boost in combination with donepezil.
KKD - Is this a new formulation, or cannot you simply combine dosages of A2-73 and donepezil?
CM - The outcome of the Phase 2a will determine what the best "ratio" might be if data confirms preclinical findings of synergy with donepezil. Anavex could develop a proprietary new formulation or a standard combination depending on the findings of Phase 2a.
KKD - You are running only the A2-73 trial now according to clinicaltrials.gov, although your press release mentioned both A2-73 and PLUS. Which is correct?
CM - ANAVEX 2-73 is the investigational drug in the trial. ANAVEX PLUS is the potential "combination" drug in case the trial confirms the strong synergy with donepezil and Anavex could develop a proprietary new formulation or a standard combination with donepezil, called ANAVEX PLUS.
KKD - Without donepezil in the mix, can you run a meaningful trial with A2-73 alone? Or do you plan to combine donepezil at some point when the dispute is settled and the patent is granted?
CM - Phase 2a will answer the impact of ANAVEX 2-73 alone. There is no patent dispute.
KKD - That brings me to the patent question - what is its status?
CM - All patents are irrevocably owned by Anavex.
KKD - What were the terms of your original agreement with Dr Alexandre Vamvakides?
CM - 2007 Original Agreement, between Assignor (Vamvakides) and Assignee (Anavex) regarding ANAVEX 2-73 was amended in late 2012 in order to clarify language regarding IP ownership for Anavex of all IP. Also to be noted is that potential royalty to A Vamvakides is 6% is on net income - not net sales.
KKD - What is the importance of Dr Steffen Thomas in this patent dispute?
CM - There is no patent dispute. Dr Steffen Thomas joining the company likely confirms that there are no IP concerns. Since Dr Steffen reviewed all IP files before joining.
KKD - Does it relate to a claimed foreign priority by Dr Vamvakides, and do you think that claim has merit?
CM - Don't understand what this claim is in reference to.
KKD - So, until you get the patent assigned to you, you cannot begin the Anavex Plus trial, correct?
CM - No. All patents are irrevocably assigned or owned by Anavex.
KKD - There is speculation that the dispute can be settled against added compensation to Dr Vamvakides. Is that correct?
CM - There is no patent dispute. Dr Vamvakides is required according to amended 2012 license agreement if requested by Anavex to comply with signing IP related paperwork. If Dr Vamvakides decides not to comply with the request, it does not change the course of the filing.
KKD - So, based on all that, how much of a hurdle would you say the patent dispute is to your progression towards market?
CM - There is no patent dispute. There is no hurdle.
KKD - Anavex began life as Thrifty Printing, a digital to photo print business targeting corner stores that didn't work out. In 2007, when Dr Vamvakides joined Anavex, he was the only one with real medical expertise. How far would you say the company has come since then? What sort of medical and related business expertise do you have today?
CM - Anavex has now a team of big pharma and biotech experts, doubled the number of Scientific Advisors which is mostly Medical Doctors, raised a company's record amount of $10M in one funding transaction, advanced after that quickly into Phase 2a, licensed additional promising compounds into the company. Anavex is significantly more advanced that in 2007.
KKD - Are you looking at licensing deals and/or outright acquisition by anybody? What are the prospects of that?
CM - Anavex will do what is best for shareholder value.
KKD - By how much do you expect the cash burn rate to increase once Anavex Plus trial begins and you reach advanced stages?
CM - Phase 2a already started and will be finished by year end 2015. All within budget of monthly burn rate of $300k-$500k per month.
KKD - Are you looking for an uplisting to Nasdaq in the near future?
CM - Yes. That is very likely.
KKD - Give our readers 5 reasons investors would be interested in AVXL.
Alzheimer's disease is a significant unmet need since no cure has been found yet.
ANAVEX 2-73 and pipeline targets novel mechanism of action further "upstream" of the Alzheimer's disease pathology by targeting Sigma-1 receptor and muscarinic receptors, which is believed to increase cellular plasticity and reduce oxidative stress, inflammation, abeta generation and tau hyperphosphorylation. All these effects are believed to be relevant to reduce Alzheimer's disease pathology.
Safety profile of ANAVEX 2-73 is clean based on Phase 1 and interim Phase 2a data.
Anavex management team and Scientific Advisory Board, consisting of Alzheimer's disease experts like Jeff Cummings, Paul Aisen, Norm Relkin.
Valuation still very attractive compare to AXON.
===
Follow up questions:
KKD - Question about patents
I heard from some sources that Dr Vamvakides has not signed an assignment document for the '352 patent. So I checked that at USPTO. The assignment document is only signed by Tangui Maurice. It has not been signed by Dr Vamvakides.
Secondly, this is ongoing for a year, because your last two 10-Ks mention it.
Third, Dr Vamvakides filed a patent very similar to the '352 patent.
Fourth, you added a patent lawyer to your team.
All this gives some credence to the source that there may be a patent issue.
Q: Are you having an issue for Dr Vamvakides to sign the patent assignment document? What are the implications of this, if any?
CM - All patent applications are owned by Anavex, independently of who filed them (Dr Vamvakidis or Anavex). Dr Vamvakides is required according to license agreement to comply with signing IP related paperwork. Sometimes this process can be delayed, but has no implications. If a signatory cannot be provided, e.g. if a signer cannot be reached, the patent application continues - just an additional form is required. Hence, it does not change the course of the filing. Anavex is confident of its intellectual property rights in the cited patent applications.
KKD - Your PR from yesterday mentions Anavex 2-73 as the investigational drug. However, in the same release, you mention that most of the 12 patients have taken donepezil.
Now, were they given donepezil as part of the trial, or were they taking donepezil therapeutically?
Q: Either way, did the trial measure Anavex 2-73 alone, or Anavex 2-73 PLUS donepezil?
Can you clarify that? This follows from question 6 from before.
CM - The trial measures both: ANAVEX 2-73 effect alone and ANAVEX 2-73 on top of donepezil. Patients are also allowed with being on donepezil (before they started the trial). We will break this down when full data of the trial will be disclosed. For the interim 12 patients, this breakdown has not been looked at yet.
For those who may have missed this:
https://www.insiderfinancial.com/anavex-life-sciences-corp-nasdaqavxl-is-changing-the-game-in-alzheimers/118101/
“We are very impressed with the rational and efficient clinical trial path Anavex has embarked upon by adopting population pharmacokinetics (PK) and adaptive trial design in its current ANAVEX 2-73 Phase 2a study in Alzheimer’s disease,” said Mohammad Afshar, CEO of Ariana Pharma. “Ariana has the expertise to fully support Anavex’s precision medicine approach to develop more effective treatments for devastating diseases including Alzheimer’s, Parkinson’s and Rett syndrome.”
Ariana specializes in clinical patient stratification using KEM®, a proprietary decision support technology for rapid and systematic analysis of multi-parametric/multi-objective data. Ariana’s technology uncovers signals and complex relationships overlooked by conventional statistical analysis that minimizes the risk of data over-fitting. This approach increases clinical trial success and reduces drug development risks by systematically identifying patient subgroups most benefiting from a drug and endpoints that best capture its effect. Ariana is one of the first companies to implement the new FDA draft enrichment guidelines for patient stratification and trial enrichment. Ariana’s expertise spans multiple medical indications and therapeutic areas, including immunological and neurological diseases and cancer.
Not unusual for small cap companies, especially those with no revenue, to not conduct quarterly earnings calls. I did write to Anavex suggesting that Dr. Missling should hold quarterly investor calls to do exactly as you suggest. The response was that this is being considered.
I believe that there is enough evidence already that A-273 may be, at minimum, better than the current standard of care for AD. If Biogen, although testing for MS, is pleased with their experiment results, no doubt they would want to pursue a true partnership deal for more than MS. It is all about the results folks. If good, no way to hold us back with BS arguments like: scam, photo co., 4 employees, bogus trials, promotional PRs, Missling's hair, etc. etc.
Enjoy the weekend longs! For those on the other side of the ledger, something for you to ponder on:
------
TWST: Regarding Biogen, can you elaborate a little bit on the agreement terms as to which party does what?
Dr. Missling: It is a material transfer agreement in which Biogen will explore ANAVEX 2-73 in an in-house remyelination experiment of multiple sclerosis at its own costs. We are just providing the compound. After that, the evaluation will take place depending on what the findings are, and then discussions are expected to continue from that point on.
---------------
...and then, a partnership happens and...
I don't think we can dismiss partnership for phase 3. All I am saying is that the more that can be proven, through data, the better any deal will be. Maybe we get a big catalyst after we see the 48-52 week results.
Create the highest possible value for shareholders. In other words, no need to rush into a partnership now. The highest value will come when the outcome is more certain. Missling is holding up for big payback IMHO.
--------------
TWST: Did you want to say anything further on partnerships?
Dr. Missling: For larger markets like Alzheimer’s disease, partnerships for commercialization are very common. This is something that no small company can market by itself and, for that reason, at some point in time, there will be certainly discussions in that direction.
TWST: When might that be happening? Perhaps pre-Phase 3 or afterwards?
Dr. Missling: Our goal is always to create the highest possible value for shareholders. When you enter into such a collaboration or partnership for a larger indication, we will always try to aim for improving the outcome for the shareholders.
-------------------
Good. I expect that we will get information between now and year-end to help us all make the call on how to proceed with our investment.
Good luck!
Indeed! While the Dr. needs to remain cautious, he is being so but at the same time quite direct in what will be done. You can only do that if you have enough confidence in what you know (and he knows a lot more about the data and business developments than what he can now say), you have your ducks aligned to execute, and confidence in your ability to make it happen.
Shorts will claim this interview as another hyped promo. From my point of view, this is a sober but very confident indication of where our CEO is taking the company going forward.
BTW - my interpretation about financing: ~12M in cash, LP in place if needed, partnerships, grants (track record in place). Not concrete but backed up by some facts.
I respect your interpretation. I have mine-neither one of us is right or wrong. By your language you might be betting against the company; I am betting for. Time will tell but my money (lots of it) is on the long side.
Cheers!
1 more:
-----
Dr. Missling: We will conduct the Phase 2/3 for Alzheimer’s.
------
Translation: we are confident in the results/data so far. The FDA is onboard. We can handle the financing. We have resources (internal and external) to conduct the trials.
CEO's are very cautions to make such statements which can land them in legal trouble. Missling has so far delivered on all milestones he has announced-perhaps not to the satisfaction of all stakeholders, but delivered he has indeed. I believe we have the right man, with scientific and business experience, at the helm.
Long for the long...
Agree. Lots of gems in this interview. Cautious but quite firm about the answers. Many to pick from but here is one:
--------------------------------------
TWST: Do you have the financing you need to get through the development of ANAVEX 2-73 at this current stage?
Dr. Missling: Yes, we believe that sufficient financing in place and also because we are working with several foundations on specific diseases like the Michael J. Fox Foundation that has supported us very strongly for exploring ANAVEX 2-73 in a pre-clinical study of Parkinson’s disease, for which positive data was recently reported on September 22nd. And we also received support from the Rettsyndrome.org foundation, which also supported exploring ANAVEX 2-73 in a pre-clinical study in Rett syndrome, a rare disease for which Anavex received FDA orphan designation this year. We might continue these collaborations if the data continues to be promising.
TWST: Do you have any other agreements in place that you wanted to mention to the investor community? Also, are you seeking any agreements in order to move the company forward? And, if so, can you elaborate on what you might be seeking at this time?
Dr. Missling: We announced on September 28th that Biogen has signed a material transfer agreement with Anavex to explore ANAVEX 2-73 for a completely different indication, multiple sclerosis, which is the main focus of Biogen’s portfolio. We obviously are excited to work with Biogen to explore the potential of ANAVEX 2-73 in multiple sclerosis. On October 5th, we announced a collaboration with Ariana Pharma under which we’ll use Ariana’s proprietary KEM® (Knowledge, Extraction, Management) patient stratification technology to potentially accelerate ANAVEX 2-73’s Phase 2/3 Alzheimer’s clinical development timelines. KEM® is a comprehensive and FDA-tested clinical data analysis system that enables full exploitation of complex datasets of smaller numbers of patients.
---------------------------------
Full interview from 8k. Reassuring indeed!!
Christopher U. Missling, MS, PhD, MBA, President and CEO of Anavex Life Sciences Corp.
Dr. Missling, President and CEO of Anavex, has over 20 years of healthcare industry experience within large pharmaceutical companies, the biotech industry and investment banking. Prior to joining Anavex, he served as the Chief Financial Officer of Curis and ImmunoGen. In addition, at Aventis (now Sanofi), Dr. Missling served as head of financial planning on all aspects of financial strategy and M&A. His career experience also includes working as an investment banker in the healthcare practice at Deutsche Bank, serving pharmaceutical, biotech, and diagnostic companies, as well as serving as the head of healthcare investment banking at Brimberg & Co. in New York. Dr. Missling has an MS and PhD from the University of Munich in Chemistry and an MBA from Northwestern University Kellogg School of Management.
TWST: Can you tell us what Avanex is?
Dr. Missling: Anavex is a precision medicine company specializing in the development of small molecule technologies for the treatment of neurodevelopmental and neurodegenerative diseases. Neurodegeneration is the largest unmet medical need and includes Alzheimer’s and Parkinson’s disease. Neurodevelopmental indications, which are caused by genetic dysfunction, include autism, autism spectrum disorder, Fragile X, Rett syndrome and epilepsy.
TWST: Before we go on to other things, can you tell about the SIGMACEPTOR Discovery Platform is? Also, is this something that you are just using internally in the company or is it something that you outlicense or seek to outlicense?
Dr. Missling: SIGMACEPTOR is a high-throughput screening for discovering small molecule drugs with different affinity for targeting the sigma-1 receptor, and yes, it indeed can be outlicensed. SIGMACEPTOR is the core of the company because this is the basis for discovering compounds that target the sigma-1 receptor, among other important targets, which are relevant and involved in restoring cellular functions and homeostasis. This might be critical to improving the lives of patients suffering from both neurodevelopmental and neurodegenerative indications.
TWST: When you call it a discovery platform, can you provide a little more insight into what the platform consists of? Is it a screening system or a particular process?
Dr. Missling: We focus on small molecules, which are orally available, versus biologics, which typically must be injected. If you can give a patient a pill or oral formulation to take, then that is easier to administer compared to an injection for which you will need a physician or a nurse. Also, some people just don't like or tolerate injections. But the focus indeed is on small molecules.
TWST: What drug candidates are the farthest along in the pipeline? Where are they and when could they potentially be commercialized?
Dr. Missling: The most of advanced compound in our pipeline is ANAVEX 2-73, which is now in a Phase 2a clinical trial in 32 mild-to-moderate Alzheimer’s patients. The next most advanced compounds are ANAVEX 3-71 and ANAVEX 1-41, which are both at the pre-IND stage. The next stage of clinical trials is in preparation for ANAVEX 2-73: a larger Phase 2/3 study as well as another Phase 2 in an orphan indication, and it could be Rett syndrome.
TWST: Is this for Alzheimer's?
Dr. Missling: We will conduct the Phase 2/3 for Alzheimer’s.
TWST: Tell us, if you could, a little bit of a science behind the Alzheimer’s candidate, as it seems there is new information on ways to intervene in this disease process. Could you tell us how it works?
Dr. Missling: The underlying pathology of Alzheimer’s is not fully understood. The most studied hypothesis is the over-expression of beta amyloid. However, the disease is likely more complex, there are also other pathological manifestations like tau overexpression, but also inflammation, mitochondrial dysfunction and calcium imbalance in parallel. Irrespective of that, we are focusing on restoring cellular homeostasis, by activating the sigma-1 receptor, a protein that we all have in our body that is not utilized unless cells are not functioning well or out of balance, i.e. out of homeostasis. If and when cells are impaired, the sigma-1 receptor activation might help those cells to regain functionality through restoring homeostasis. We believe Alzheimer’s is a chronic neurodegenerative disease directly correlating with age, and it is similar in that way to Parkinson’s and other chronic dementia diseases. On the other hand when you have a genetic predisposition where the cells are constantly stressed or proteins are misfolded, or out of homeostasis, the body’s own repair system is probably over-challenged, overburdened, but with small molecules, you can increase the expression of the sigma-1 receptor, potentially restoring homeostasis and potentially reducing those dysfunctions. The difference would be the positive features mentioned before; we are seeing a reduction in amyloid beta but also in tau and inflammation. Calcium balance is restored and oxidative stress and mitochondrial dysfunction are improved. So our approach is not one hypothesis-dependent approach. It is utilizing a more comprehensive approach. It is more macro-management of disease rather than a micro-management approach to the disease stage. So, we are basically in sync with any hypothesis because the sigma-1 receptor is reacting independently to whatever form of dysfunction is caused by the disease. Possibly, an analogy is immuno-stimulation in oncology, where you similarly harness the body’s own existing armoire of defense by activating the body’s own immune system to help to fight the disease, and we are essentially utilizing a similar approach by activating the sigma-1 receptor, which restores cellular homeostasis.
TWST: Is ANAVEX 2-73 potentially a drug that somebody could take and an early stage when they are pre-symptomatic in order to arrest the disease? I know many of these medications ideally should be taken pre-symptomatically if and when we can get the appropriate diagnostic tools.
Dr. Missling: Yes.
TWST: But can you elaborate on the stage that this could be taken?
Dr. Missling: We presented preclinical data that ANAVEX 2-73 could halt the disease and it could improve cognitive function. It was also published that ANAVEX 2-73 might be able to prevent the symptoms of Alzheimer’s in a pre-clinical animal model. So the answer to the question is, it could be both; it could be possibly utilized for either situations or time points.
TWST: Forgive me if I'm overstepping my bounds here, but it almost sounds like something that, if it had a clean safety profile, you could even take as a prophylactic or a vaccination because you just alluded to the fact that it improved cognitive function in people. Is this at all possible do you think?
Dr. Missling: We would have to prove that in clinical trials before claiming that.
TWST: Right, you're saying it's not out of the question at this stage?
Dr. Missling: It is not out of the question, but we cannot claim that at this point.
TWST: If all goes well, when might be the earliest timeframe ANAVEX 2-73 could be on the market? I'm assuming, given the disease it's targeting, that the FDA would want to expedite approval.
Dr. Missling: It's too early for us to give a time point for that because we are really thinking step by step to establish safety first, and the current study is running for 52 weeks. But today, it's really hard to tell because it depends on the data.
TWST: Do you have the financing you need to get through the development of ANAVEX 2-73 at this current stage?
Dr. Missling: Yes, we believe that sufficient financing in place and also because we are working with several foundations on specific diseases like the Michael J. Fox Foundation that has supported us very strongly for exploring ANAVEX 2-73 in a pre-clinical study of Parkinson’s disease, for which positive data was recently reported on September 22nd. And we also received support from the Rettsyndrome.org foundation, which also supported exploring ANAVEX 2-73 in a pre-clinical study in Rett syndrome, a rare disease for which Anavex received FDA orphan designation this year. We might continue these collaborations if the data continues to be promising.
TWST: Do you have any other agreements in place that you wanted to mention to the investor community? Also, are you seeking any agreements in order to move the company forward? And, if so, can you elaborate on what you might be seeking at this time?
Dr. Missling: We announced on September 28th that Biogen has signed a material transfer agreement with Anavex to explore ANAVEX 2-73 for a completely different indication, multiple sclerosis, which is the main focus of Biogen’s portfolio. We obviously are excited to work with Biogen to explore the potential of ANAVEX 2-73 in multiple sclerosis. On October 5th, we announced a collaboration with Ariana Pharma under which we’ll use Ariana’s proprietary KEM® (Knowledge, Extraction, Management) patient stratification technology to potentially accelerate ANAVEX 2-73’s Phase 2/3 Alzheimer’s clinical development timelines. KEM® is a comprehensive and FDA-tested clinical data analysis system that enables full exploitation of complex datasets of smaller numbers of patients.
TWST: Regarding Biogen, can you elaborate a little bit on the agreement terms as to which party does what?
Dr. Missling: It is a material transfer agreement in which Biogen will explore ANAVEX 2-73 in an in-house remyelination experiment of multiple sclerosis at its own costs. We are just providing the compound. After that, the evaluation will take place depending on what the findings are, and then discussions are expected to continue from that point on.
TWST: Does Anavex own most of its IP, or are you sharing it with others?
Dr. Missling: Yes, the company owns it.
TWST: I know you are knee-deep in some very important work, but if, let us say all goes well, where do you see the company going in the future? Do you want to stay in the neurology area? Do you see increased partnerships in the future? Can you elaborate on any kind of longer-term vision?
Dr. Missling: Yes, we are focusing on the central nervous system. And I mentioned specifically indications like Alzheimer’s and Parkinson’s disease, and then also for the neurodevelopmental orphan designations or rare diseases like Rett syndrome, infantile spasm and Fragile X, which is a special form of autism. Then, we also work on improving pain management. So we have a balanced pipeline, but the main focus is indeed on the central nervous system.
TWST: Did you want to say anything further on partnerships?
Dr. Missling: For larger markets like Alzheimer’s disease, partnerships for commercialization are very common. This is something that no small company can market by itself and, for that reason, at some point in time, there will be certainly discussions in that direction.
TWST: When might that be happening? Perhaps pre-Phase 3 or afterwards?
Dr. Missling: Our goal is always to create the highest possible value for shareholders. When you enter into such a collaboration or partnership for a larger indication, we will always try to aim for improving the outcome for the shareholders.
TWST: Are there any significant management or operational changes likely to take place in the next year and, if so, can you elaborate on what they might be and what they are for?
Dr. Missling: We are consistently growing in a thoughtful manner and we are adding people selectively because we are still relatively small. So this will be at an incremental and carefully considered pace. We intend to have several clinical trials up and running next year. One will likely be an orphan indication. All those trials will be double-blind placebo-controlled studies.
TWST: As a CEO, what is your chief challenge right now? And what are you doing to address it?
Dr. Missling: The chief challenge is really to keep in mind that drug development has inherent risks, and we would like to address those in order to reduce those risks to the extent possible. So we try to always do everything to the best of our knowledge and ability to minimize clinical trial risks in particular. That is the most important task for us and for me.
TWST: What do you want a potential investor in Anavex to know today?
Dr. Missling: We are a dedicated team and most of us have big pharmaceutical backgrounds. We want to help patients in indications like Alzheimer’s and Parkinson’s, but also those other devastating diseases like Rett syndrome and infantile spasm for which there are no cures for people today.
TWST: Is there anything you wanted to add that we haven't touched upon?
Dr. Missling: We believe the results we have demonstrated to date with ANAVEX 2-73 and other compounds both clinically and pre-clinically are quite encouraging. We would encourage investors to visit our website at www.anavex.com to learn more.
TWST: Thank you.
Dr. Missling: Thank you.
So what would you suggest? Stop reporting?
The clues point to a positive CTAD presentation of clinical results. Would not be surprised if some sort of commercial agreement is announced before then. GLTA longs.
NEW YORK, NY – July 29, 2016 – Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), confirms the information in the July 27, 2016 press release announcing positive results from the Phase 2a Alzheimer’s trial for ANAVEX 2-73.
Dr. Norman Relkin, MD, PhD, an Alzheimer clinical trialist and an advisor to Anavex, commented: “To interpret the ANAVEX 2-73 results presented at the 2016 AAIC meeting, it is important to keep in mind the stated goals of this first in Alzheimer’s patients study. This was a Phase 2a study, primarily designed to determine which ANAVEX 2-73 dosages are safe to administer to mild to moderate stage Alzheimer’s patients. The study was successful in establishing the maximum tolerated dose and in revealing the range of ANAVEX 2-73 doses that are well-tolerated by Alzheimer patients. It also provided encouraging evidence that previously reported positive trends in certain cognitive and biologic measures persisted over a period of approximately 31 weeks. However, this analysis was based on pooled data from a relatively small number of subjects receiving a variety of doses. It is therefore unlikely that these findings reflect the full potential ANAVEX 2-73 in treating Alzheimer’s disease. It is unreasonable to draw conclusions about any limits to the long-term efficacy of ANAVEX 2-73 based on the interim Phase 2a findings, especially since no statistically significant decline from baseline was reported, which is impressive. Detailed pre-planned analysis of the pharmacodynamic results is in progress, which is one of the key factors of relevance for regulatory agencies and which will also determine the optimal dose for future studies.”
Anavex confirms that there has been no change with regards to its science, data or the fundamentals of the Company. Anavex remains dedicated to advancing ANAVEX 2-73 and will be reporting new data to investors as it becomes available.
Mike, thanks for posting the link. For those browser challenged-:)
A CASE STUDY OF RETTSYNDROME.ORG’S SCOUT PROGRAM
Research with Results
October 14, 2016
Executive Summary
Rettsyndrome.org presents a case study of the successful partnership between our organization and a biopharmaceutical company (Anavex Life Sciences Corp.). The study helps to identify potential new treatments for Rett syndrome. This study also serves as an example of how a non-profit organization can encourage the pharmaceutical industry to test compounds and drugs. These drugs are in their pipeline for preclinical testing and can support a non-profit organization. In this case study, we cover the positive testing of the compound, ANAVEX 2-73, in a mouse model of Rett syndrome. It also outlines Anavex’s plan to move ahead into a human clinical study.
Background - Scout Program
In June of 2013, the Rettsyndrome.org Board of Directors gave ¬final approval to launch the Scout Program, a Drug Discovery Screen in a mouse model of Rett syndrome. Since then, $1.2M has been put into this program that puts Rettsyndrome.org in a unique position to proactively accelerate Rett syndrome research. Over 14 compounds have been tested, and 3 compounds are going into human clinical trial planning.
Steve Kaminsky, PhD, Chief Science Officer of Rettsyndrome.org, determined that a standardized testing bed for preclinical testing was lacking for Rett syndrome, and the Scout program was developed. Participants in a workshop recommended important guidelines that the community of Rett researchers can implement to ensure more standardized study design and transparent reporting. Therefore, the Scout program was designed and focused on the state of the art in animal studies for Rett syndrome.
The Scout program has been developed to provide a rigorous platform for the drug companies to use for either new or repurposed compounds they believe might be useful in treating Rett syndrome. This aspect of the Scout program provides an incentive to the drug company to make Rett syndrome a priority in their clinical research portfolio because Rettsyndrome.org provides the testing environment through our established partnership with a Contract Research Organization (CRO) and does not take any intellectual property away from the company. The program is designed to facilitate drug discovery for industrial partners and allows them the opportunity to help those with Rett syndrome.
The Scout program testing bed is performed under contract with the CRO PsychoGenics, Inc. in Tarrytown, New York.
ANAVEX 2-73
Anavex has a vision of bringing Precision Medicine to patients suffering from a wide range of Neurodevelopmental and Neurodegenerative Diseases, including Rett syndrome, Parkinson’s and Alzheimer’s. To achieve this goal, Anavex is dedicated to developing Effective Targeted Therapies.
ANAVEX 2-73 is a small molecule in clinical development. It activates the Sigma-1 receptor, targeting cellular homeostasis by reducing protein misfolding, oxidative stress, mitochondrial dysfunction, inflammation and cellular stress, relevant in the pathophysiology of a wide spectrum of neurodegenerative and neurodevelopmental diseases. Given the converging beneficial effects of ANAVEX 2-73 on seizures, cognition and anxiety, confirmed prior in independent studies, Anavex was committed to exploring an indication with a very high unmet need, Rett syndrome, which might require the combined amelioration of those individual ailments.
Partnerships
ANAVEX was informed that Rettsyndrome.org offered the Scout program to accelerate Rett syndrome research. The program could test ANAVEX 2-73 in a validated MECP2 deficient mouse model that causes neurological symptoms that mimic Rett syndrome. ANAVEX 2-73 was added to the priority list for testing in the Scout program by the independent CRO. The encouraging results demonstrated dose related significant improvements in an array of behavioral and gait paradigms in the Rett syndrome model. The data was subsequently presented by Christopher Missling, PhD, President and Chief Executive Officer of Anavex at the 14th Rett syndrome Research Symposium in June 2016.
Results
Given the interest by the Rett syndrome community, Anavex is now planning a clinical study with Rett syndrome. With the data generated from the Scout Program, Anavex received the FDA orphan drug status for ANAVEX 2-73 in May 2016. Now, Anavex is committed to continue to work very closely with the Rett syndrome community which builds the case that Rettsyndrome.org and its partnerships are key to bringing forth potential new options for the treatment of Rett syndrome and other neurodevelopmental and neurodegenerative diseases.
Page 15
8.45 a.m OC59 - 9-Months and 12-Months Safety and Exploratory Efficacy Data of ANAVEX 2-73 in a Phase 2a Study
in Mild-to-Moderate Alzheimer’s Disease Patients
Stephen Macfarlane, MD1
, Marco Cecchi, PhD2
, Paul Maruff, PhD3
, Kristina M Kapiak4
, Christopher U Missling, PhD4
(1) Caulfield Hospital, Melbourne, Australia, (2) Neuronetrix, Louisville, KY, USA, (3) Cogstate Ltd., Melbourne, Australia, (4) Anavex Life Sciences Corp., New York, NY,
http://www.ctad-alzheimer.com/sites/ctad.prod/files/files/PROGRAMprel_CTAD2016_7oct.pdf
I posted this a couple of months ago. I am reposting it so we can take a look back and assess the credibility of what I was told by IR. I underlined the points that I believe have been on target; others remain TBD.
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IR called me today.
Some of you may have read my post regarding an email that I sent to IR last week expressing my concerns about how the company communicates with its shareholders.
I received a call from IR (do not want to disclose the caller's name because I did not ask him specifically if I could). This was ~20 minute conversation with someone who seems to be well versed on the business of Anavex and the biotech industry in general. I asked him how comfortable he was with me sharing--obviously if he shared with me he should be comfortable sharing with others, but I just wanted to be sure-- within the forums that I exchange information with fellow investors. He was OK with it as long as he would not be miss-quoted or misconstrued, which I respect and understand. With that disclaimer and while paraphrasing, here are some of the key points from our discussion:
31 week data: Keep in mind that the results are from pooled data so it is difficult to ascertain anything conclusive. However, the main objective of this phase, to establish tolerance and safety dosage, was achieved and this encourages the company to move forward with the next steps in the process. Management is pleased about early efficacy signs but cautious about getting too exuberant; again, pooled data, relative and relatively small sample, nothing discouraging. Next step is to analyse the data at the individual level and continue to report.
Ph-3 AD trial:
• Likely to start in 2017.
• Reluctant to provide a more conclusive timeline as this is in the early planning phase.
• Will need to raise money or conclude partnership to execute.
• No immediate plan to raise cash.
• There have been discussions with potential partners. Unable to elaborate further.
My conclusions: nothing new here but confirmation that partnership seems to be the preferred avenue for funding while not discarding alternatives. Do not expect a trial to start in 2016. My prognostic is that Q2 is likely. The results disclosed last week should not be of concern for pursuing partnership. Actually, the company is encouraged by the results.
Parkinson’s -MJF study: Executing. Expect news on this in the not too distant future (weeks). I found this encouraging.
Orphan Designation indications: Rett is priority. Will proceed with Ph-2 study being that it will be of short duration (months) and relatively inexpensive, people and money, to execute. Expect news soon.
My take away: encouraging as this will give us a good indication as to where we stand in regards to not being a “one trick pony”. Revenue may start coming in sooner than expected.
SEC investigation and class action LS: nothing new to report. The company is not concerned in either situation. From his extensive experience with this type of situations, they happen a lot and most tend to quietly go away. He did mentioned that the company remains cautious about what/how to disclose information in light of the lack of conclusive resolution.
Comment was made referencing AF and other engaged in distorting the facts. These people get away with murder with no repercussions; regrettable and frustrating but just the reality we live. Agreed that the company can do a better job of, and I sensed that there is serious internal discussion as to the best way to do so, setting the record straight. He mentioned the PR last Friday. I commented that while positive, more of and with more emphasis on refuting the "lies" is needed. He agreed. He also commented that because of the complexity in analysing the results, it is easy to distort the interpretation, as it has been done.
He emphasized that the company acknowledges that it needs to do a more effective job of communicating to the public and will endeavour to do so going forward.
In summary: I was pleased to receive the call. I found the caller to be willing to tell as much as he could without getting in trouble.
How do I react? I believe the science is still viable, management continues to learn about dealing with the market place, limited resources (people/money) are a big hurdle that needs to be overcomed soon, I expect more/better communication going forward. In conclusion: I remain committed to my investment an will proceed cautiously but still optimistic that this will payoff for investors and those in need of relief from neurological disorders.
Cheers and good luck!-----------------
Ultimately, I think we are all in agreement that new cash will be needed within 12 months. Let's hope that a good partnership deal is the way to do it, albeit decretive to future revenues.
I believe and seems that you do too.
Cheers!
What partnership means most of the time is that future revenues will decrease accordingly, thus impacting EPS and subsequently PPS. So, technically you are right. However, in order to stain in business you need cash and without products the ways to get it is to borrow it, sell equity of find partners that are willing to give you upfront cash and assume costs. We already know that Anavex has done a good job of obtaining grants, the only reason that the burned rate has remained low, but we know that alone does not keep the doors open so at some point something else has to give. I hope is a partnership with upfront and milestone payments. Bottom line, I look for PPS to increase as some of the catalysts that are in play become reality and bottom line that is what matters most from an investment standpoint. I will be extremely happy to see AVXL in the 20's so I can take some off the table and then ride a good chunk of shares into what the ultimate price could be (100's)if the company delivers on the potential of its platform.
God luck and cheers!
Cash can also be raised by partnership with upfront an milestone payments from partner (i.e. BP). I still believe that is that avenue that Anavex is pursuing.
However, if executing the LP agreement or other ways, requiring issuing new shares, to raise cash is the way to go to sustain the development plan, then I can live with dilution. For me, what matters most is what the PPS will be in the future--and that will be dictated by trial results-- regardless of how many shares are floated. In other words, use whatever means there are to achieve the target of getting the compounds to market is the most important aspect of the execution plan. Obviously, my preference would be a good partnership deal which will result in a rapid PPS increase and perhaps an opportunity to take some profit. I am long for the long as long as the science continues to prove the case(s) and the Dr. continues to execute as he has. I still have faith on both.
Cheers!