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drops mic.
Thanks Bear. Our A2-73 reality may be getting clearer to us but my guess is most biotech investors would scratch their head and wonder what we are doing? NO big FDA/self inflicted overhead? Impossible.
I have worked with a team to develop and apply AI driven business planning/strategic-product technology "What If" models in a previous life. Tools worked exactly as predicted and we kept growing, we added to our base portfolio/capability virtually every day. The humans in the room did not always like the responses given to 1000's of "what if" linked scenarios at first but they eventually figured out how to use and grow the technology as a complete success. Humans expected different solutions than what the system produced. Getting the right people to input their ideas/questions and thoughts was ultimately more difficult than building/running the many links to the inference engine.
One must be very precise on what is asked, which escalates quickly to getting/applying more facts. The array of knowing what we do not know can grow very quickly in such a learning process. A key will be deciding when enough, is enough.
The AVXL approach is based on addressing the CNS/AD root cause and not the symptom as has already been done for AD, as example. In fact all past AD trials and $$$billions spent by the best and the brightest have only produced evidence on what the root cause is NOT. Knowing what NOT TO DO is also critical of course But, it took them 30+ years or so to get there. Which I took as evidence they were not learning. You would think that after say 10 years and 95%+ failures some one would have asked why keep on this path? That will NOT HAPPEN with AI b/c the tool will give red flags up front and should get everyone to stop and rethink their path. We'll see. In any case this path and tool will facilitate the learning process big time.
When the thought occurred to apply the same tools and problem solving methods being used by the cancer treatment teams to CNS diseases a whole new universe was opened up. Now that Fireman's troops are on the job, new appropriate methods will be applied. They will emphasize knowledge/risk based thinking processes after/while the links are established with WW labs/clinics/academia/every petri dish and lab rat on the planet. The incentives to cure the curse of CNS will be WW before we know it, IMO.
Our Rett first client(Pts) are well chosen IMO. A rare CNS disease where patients should present rapid and convincing evidence of A2-73 efficacy (we all pray). This whole thing cannot start soon enough. Lets all hope for a financially and spiritually rewarding process outcome.
Like M. said the other day at the PCon...his decision to go w/Precision was a strategic decision for AVXL. I heard that as code for ....we cannot and should not use our limited resources to go head to head w/BP and other factors. BTW, he was/is totally correct....big trials is where little P's go to die.
The strategic choice also has to be based on how much do we have to spend and is there a better way? The entire FDA "Precision Medicine" (PM) initiative presented an opportunity to do what he was planning to do anyways. Did we just get lucky or was he synched up w/FDA Dr.G? Maybe a little bit of both. After decades of consecutive failures BP sees and took the advise/wisdom of walking away. Having a little insight into how the process works is helpful when mixed w/common sense. BP cannot afford to just abandon the field to AVXL and the POSSIBILITY Dr. M. could be right w/the Homeostasis work, including Regen and Repair. Can we imagine the nightmares they are having? BP are doing SOMETHING , even as we speak. These people are smart and tough. IMO, Beware the ides of March.
BTW, I can be bought for $500/ea.
Talon...agree completely. Thanks for the added value.
[quote I think we have a leg up with Drs Fadiran and Aisen working and advising us. I think we will see IND approval and trial start up for Rett Syndrome shortly with a possible Master Protocol "basket trial" aspect. Also. think there is a good chance to conduct our AZ P3 as one of the initial candidates supported by the ACTC. thus being one of the first "lead the force" targeted precision medicine AZ trials. Definitely Biogen is watching this very closely (as well as several other Pharma's).
][/quote]
[quote You have to know the target. How can you have precision medicine if you are not being "precise" in who exactly you giving the "medicine" too?? ][/quote] This is exactly correct thinking IMO and we are on the right path...totally.
He, WE AVXL are breaking/have broken the trial mold here. When ALL BP had their "come to Jesus" moment a while back and each found a way to cancel/walk away from the AD Plaque path, that was the signal. At the very least they recognized/or were told what the new path was/would be. IMO, they are all out retooling for the new approach and they will all be back. AVXL has a clock running and we better move it or we will get run over.
The discovery process here is pretty obvious. We have been given a one lap head start in 4 lap race. As people like Falconer would point out, we are going to cherry pick the first set of pts. When that works,(and it will b/c we get to define success) then the next subset will start but only after a proper assessment of the first trial, and so it will go until all are either addressed or somehow understood. That is 101 level iterative learning process which we can all see coming. Getting started is key, the clock will not be held for us to finish the race. When BP sees what they must do they will leave the starting line on a bicycle, then move to a motorcycle, etc. Life is just not fare. But, we have the start and now we have to act like we earned it and we expect to finish/win. I am not looking for the Nobel prize, just $500/share and I do not care if they buy us or we get it the hard way. Time is critical here, how we use it is what matters now.
Talon, we might agree that the process for getting the new FDA trial guidelines written is going to take a while. As positive as the change looks to everyone I am wondering/concerned about the timing.
I do not want to see A2-73 hold up trials any longer than absolutely needed to get things rolling. Dr. M. has (IMO) cleverly planned start trials in other venues, which will help to keep some pressure on. It has been noted that other CNS trials have been started since October 2017 using old FDA rule sets. AVXL has chosen not to do that, for reasons we agree with.
FYI...I am planning to ask for special recognition from Rome for all legitimate longs considering your spirit and your work done over past couple of years...I will keep you informed .....but , if you suddenly feel younger/faster/stronger/smarter/richer/happier....you will know it's working...hang in
As you have pointed out previously Dr. M.'s strength is finance and directing very clever ways for getting trial funds/sponsors. Rett/MS-Sweden-WSU studies/MJFF/AUS-small trials for FDA. I predict AVXL will get funds for AD from somewhere NIH Grants or elsewhere.
The other good news is he has developed a rationale around precision medicine which fits well w/technical strengths and smaller trial populations. I still believe the RETT trial will become the poster child on structure and results.
The other news is, when you are a small fish...you are a small fish. If we keep alive, we will be taken shortly, IMO. If A2-73 and platform is anywhere near as good as claimed then being taken seems certain.
Classic problem solving tree shaping up here.
Agree, this should be considered his major strength and value adder to AVXL.
call tells me he is reading here from time to time...good MS still alive
Is it audio only? According to the link provided byF1ash 1 @11.27 here it is audio only...call 1-866-939-3921 @4:30 Eastern
Penny, BIIB has it's issues, IMO. MS-A2-73 can piggy back on Rett and other data . At some point, the holder of the AI data tool will call the shots. Would like to think you are right. Why the MS silence? Must be an explanation. If they were out of the game, they would say so.
You could be right.
CNS trial decisions were made w/o using any new FDA precision medicine guidance (see today's BIIB withdrawl as a possible example). A search provided by investor shows continued use of existing trials. There will be a lot of "What If" being done before this is over. Being first is never easy.
Bear also consider the PR today from BIIB on their most recent trial failure for a stroke treatment...using MS drug.OOOOOPS, Never Mind. I would like to believe we will all look back of this example and shiver like we do when we see photos of civil war operating room practices. "what were they thinking?"
Can we take the following as an example of "Never Again"?. Will ALL BP ex. BIIB be forced to reconsider their practices? Will the FDA learn how to play hardball when the next trial is presented (before guidance on Precision medicine) are published. Is AVXL really a beta site for new trails or are they on their own? Clearly, once the new FDA/Dr.M. trials protocols are adapted, there will be no going back. The future of the AVXL investment will be driven by the acceptance of RWE and early trials actions to approve uses. Could this explode when investors understand or will that take years also?
https://www.fda.gov/downloads/AboutFDA/ReportsManualsForms/Reports/UCM592001.pdf
Keeping my fingers crossed but have limited insight into just how fast the BP/WS crowd will buy in, or IF they will at all. They have to choose. Keep spending massive amounts of money and getting high risk lousy results or "STOP THE TRAIN" and reinvest in new science/technology/AI/other. These are the kinds of choices that companies make to either survive or disappear. Many, many examples to point to. The rest of the world has begun to understand that .."It's ALL about risk. Understanding RISK and limiting/mitigating it. or die.
From PR by BIIB:
Feb 7 (Reuters) - Biogen Inc said on Wednesday it would stop developing its multiple sclerosis drug Tysabri to treat patients with acute ischemic stroke after it failed a mid-stage study.
The treatment did not improve patients' self-dependence and their ability to perform daily activities when compared with a placebo, the drugmaker said.
Ischemic strokes require immediate medical attention and occur when blood supply is cut off to part of the brain.
Analysts have called Biogen's move to develop Tysabri for ischemic strokes as "high risk."
"Stroke is very challenging and even if it works, (Wall Street) may be hesitant to ascribe too much value as Biogen would need to run a larger trial and the commercial opportunity is still far off," Jefferies analyst Michael Yee said last month.
Bear...I love this kind of talk. Way overdue. Puts a lot of pressure up front. from FDA...
Exactly what I thought. Here we are 1 year later and ???. There has to be more to this. Just what process has been slowing/stopping us in past yr? The $$$ was there, pts, etc?
https://www.marketwatch.com/story/anavex-awarded-grant-to-fund-clinical-trial-of-rett-syndrome-drug-2017-01-19
Flashback to about a year ago. Note the trial size was predicted to be small and short then as now. Having 3 dear little granddaughters myself I cannot imagine how important such research must be. God bless them. Best
Anyone know of ANY new CNS trials being started (by anyone) in the past couple of months? Yeah, we all know who it wasn't but just curious if anything did happen. Also, any thoughts on what caused the BP to walk away from their ongoing AD trials (more or less) at the same time. Except for BIIB of course who seem to be in a different time/space continuum.
Maybe we will learn more on Wed. about what REALLY did happen in Q4, but I am not counting on it. It is promising to see Dr.M. joined at the hip w/Rett admin/leader.
What will it take to get RGIN to $2.00?
Judging from these topical bullets they are speaking to .."How to get things moving/done."
AB-E-NORMAL
IMO, all we need is the SLIGHTEST HINT of AVXL technical validation and this puppy will launch. This going on while the Reset of world ( ROW) has decided to stand down, it's a vacuum out there.
YUP, thinking along the same lines. And, IMO this may serve as a template for similar rare disease trial protocols.
[quoteThis Wed. will bring me a good chance to re-evaluate Missling from my perspective. Hopefully he brings a better answer than silence or rehash.
][/quote]
Just tell the truth.
Eventually we will be able to connect all the dots simply based on the trail of facts. Like: Numerous Academic pubs which all support the Restore & Regenerate (R&R) trials/science. MJFF funding. Rett orphan. Aus trials, people are still alive and stable, which has never been done anywhere. Did that REALLY happen or not? Are they all liars? The Biib MS MTA mystery, gone or not gone? Many other facts as Bear often points out.
Then...."Starting 3 trials ASAP". Then...."Never Mind". WTF?
Self declared experts have said we retailers are just dumb cult buyers. Like the crook looking down the barrel of Dirty Harry's 44 Magnum said..."We just Got's to know".
My favorite Irish expression..."Unconstrained by knowledge, we moved on".