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[quoteMissling could have made a crappy MTA with Biogen that gave them the first right of refusal][/quote]
A. BIIB has a dilemma, they have seen a peek at A2-73 and have studied the results from multiple MS researchers, all of which seem to indicate clinical promise unlike anything they have. Partnering to develop any A2-73 trials would likely lead to killing their golden geese. So, they will not move unless timing/PR/FDA and other conditions force them to. It is all easily understood.
B.We all know, No Micro Biotec's like AVXL can afford to sponsor the massive trials being suggested based on past FDA practices....so 21st century precision medicine CA must be applied w/smaller trials based on TBD guidance. This status quo has resulted in the previous ineffective FDA processes and zero pharma NP innovation...brilliant. So....we wait it out while FDA grinds on w/funding/staffing issues of their own.
C. We have a kind of showdown while BIIB continues to get BIG $$$ for MS, patients endure and AVXL is in this long Queue awaiting some kind of god like intervention, IMO.
Them who has the gold makes the rules.
Scott...yes, pls post the pR..thx
[quote he put out a very nice PR this morning then tweeted more PR's coming over the next 7-10 days][/quote]
Scott, looks like we need a partner w/an amount of investment $$$ to get this on track. Considering the unmet need, what are chances of him getting hooked up w/such?
Walker, thanks for these important posts, including #137743.
https://endpts.com/fda-commissioner-scott-gottlieb-just-broke-a-public-promise-on-publishing-crls-and-yes-it-matters/?utm_medium=email&utm_campaign=Wednesday%20January%2017%202018&utm_content=Wednesday%20January%2017%202018+CID_cb81e7923614fa6a45ef2512143a8622&utm_source=ENDPOINTS%20emails&utm_term=FDA%20commissioner%20Scott%20Gottlieb%20just%20broke%20a%20public%20promise%20on%20publishing%20CRLs%20%20and%20yes%20it%20matters
Many have said and I agree that, " the first step in solving a problem is admitting that one exists". These posts present evidence of CHANGE happening in AD researchers thinking. Very important. The system FINALLY worked. They admitted they failed. When NIH, FDA and BP admit they have been beaten by AD then they can move on and finally start over.
NOW, if someone will please find the A2-73 trial super responders and analyze (the shat out of) their results as in RWE they can start to learn, instead of dismissing them b/c there were statistically insignificant. Hey, maybe that is what Dr. M. has done. It is called reverse engineering and in instances like this it is now the only game in town. AVXL probably has an important piece to this puzzle, let's see if we get to say so.
Also, my guess is the BIIB of the world are in shock right now. Eventually, they all have to step up like Phzr did.
Bio, appreciate the thought.
Wondering if the PR today was intended to keep the OL CNS-Restore-Regen CNS Homeostasis theme in front of us. We do not know what the guy is thinking but the R&R BIG picture is always worth a shot.
IMO, BIIB has got to be twitching w/only a 3 lb. pull....then boom. We need to hire a car starter/food sampler for Dr. M.
Agree.....RWE-Real World evidence, comes to mind as a leverage point.
Yes the new release says that and also ...is this an opportunity to select and apply some of the new FDA trials rules...very low/no risk w/implied strong benefit...slam dunk option.
see post #137220 report..
[quoteNow it alleviates hypertension?][/quote]
Standby for the A2-73/Viagra happy meal.
21st CCA FDA work plan, this is NOT NEW.
https://www.fda.gov/downloads/advisorycommittees/committeesmeetingmaterials/scienceboardtothefoodanddrugadministration/ucm556618.pdf
We see the PLAN is the process metric/budget cycle for FDA, suggests how work is done by Gov. This is a process. CCA authorized 12/13/16. Helpful words on RWE and restore-regen (sound familiar) pg 7-9. Trying to get progress in context is tough, no matter how important the results might be.
I think we see Dr. M. trying to get work done outside the govt. plan (see MS Sweden). My guess is he is looking to generate SOME KIND OF TRIAL EVIDENCE which can be used to leverage the PROCESS. IMO, that is the reality of the current situation.
Not trying to pick an issue for contention by this but if anyone familiar w/how the FDA budget/work process can add insights then great.
https://finance.yahoo.com/news/2018-biogen-inc-apos-best-131800182.html
Interesting. MS is massive part of BIIB revenue. Does A2-73 present a threat? The dynamics of this competitive scenario alone suggest a set of conditions that cannot last long.(boom) It is a one or a zero. I have a family member w/MS. She deserves an effective and fairly priced treatment.
Well, MY, MY , MY...what have we got here...?
The new FDA guidelines allow lots of smart things to happen...if one gets creative...IMO, this will require a detailed understanding of new trial results. On the other hand, it looks like things can happen quickly when trial evidence presents the appropriate case. In fact, the new rules seem to say ...FDA expects and will support such action.
If these new rules are now effective or close then (IMO) it is no wonder that BP trials which were going no where have been dropped. My guess is that since Dr.M has been a principal sponsor for many of these initiatives that AVXL trials will be accordingly structured. Lots of moving parts here but the days of massive trail and error are gone...finally. And, the BP R&D structures will be(and are) being reconfigured to support more AI/tools and fewer petri dishes.
[quote Implement new streamlined orphan designation review template and expand orphan opportunities to new areas
• Modernize the Office of New Drugs to ensure policies are rooted in the best science and management principles, and staff have the support and tools to achieve the public health mission
• Develop a series of new, disease-focused medical product guidance documents to update and modernize approaches to clinical trial design and other aspects of the development of drugs targeting unmet medical needs. This will include a new suite of guidances and policies focused on the development of drugs targeting a group of serious neurodegenerative disorders
• Update and modernize FDA’s approach to applying evidentiary standards for establishing safety and effectiveness for new drugs to more clearly define the role of real-world data and evidence, especially when it comes to evaluating post-market safety
• Develop patient-focused drug development guidance to facilitate a more systematic approach to gathering and using patient perspectives to inform regulatory decision-making
• Advance the use of in silico techniques to develop novel methods for creating models of virtual patient outcomes and modernizing FDA’s evaluation of patient benefit and risk
• Develop and advance recommendations for an OTC monograph user fee program, to implement structural reforms to streamline and improve the timeliness of review activities, and foster innovation of OTC products
• Advance a new regulatory framework (NSURE) to enable more drugs to be made available in OTC forms through the use of technology (such as medical apps) that can help patients better selfselect when a product is appropriate
• Explore the development of a policy framework under which an accelerated approval approach could be used to support marketing of a drug that demonstrates a survival benefit early in clinical development. The goal is to expedite availability of a therapy while the magnitude of the benefit it provides is being confirmed
• Advance the use of new drug development tools and mobile technology for better capturing clinical trial data and the measurement of safety and benefit in pre- and post-market settings
][/quote]
https://www.fda.gov/downloads/AboutFDA/ReportsManualsForms/Reports/UCM592001.pdf...
Check out pg 13. The new rules are written to allow discretionary authority to FDA when interim trial results warrant such. Very cool. IMO.
Bear...nicely done
Brilliant...
Finally, most of the flat earth people have declared "Das Ende"...done, we give up, it don't work, let's all just move on...after a long time and much discussion they agree. And, IMO, they have declared an end to whatever scientific method got this result....I hope. But, I do expect there will still be people who insist that(even if it does not work you MUST have thousands of people in meaningless trials to prove it, b/c I cannot change my mental model on trials...)....NAH, NA ,NA, NAH NAHHH,....GOOD BYE..
Page 13 ref :
https://www.rettsyndrome.org/research/clinical-trials
not on banner? but still shown as taking applications for trial...
rocalinda...thank you for your excellent report-your effort-your reporting credibility and your skills in describing this scenario.
At some level this kind of information assures us that AVXL staff are doing everything humanly possible to get this science applied in the correct manner at the correct time.
We may all see something in the development process controlled by FDA has stopped working. We do not know why but we can review the evidence on CNS trials clearly says the process is broken and has been for a long time, essentially it has stopped. FDA have not declared and emergency but it is obvious they have a problem, not only AVXL is being impacted.
I do believe that AVXL is not scamming shareholders but would admit this is a trust issue which each owner has to resolve for themselves. I trust that if anything material had changed I would be told or facts would be made clear enough for consideration.
Back in the day there was a brutal process called the "parking lot exercise", it usually was done during layoffs. This feels a little bit like one of those. If you had it to do all over again, would you?
Just as long as I do not have to be 15 again...
Have new FDA CNS trials guidance docs been released? If N, then only a fool would be publicly defining/starting a new CNS trial/endpoints. Lots of moving parts here. We have a dilemma. Recent anecdotal information about other companies walking away from CNS/ AD R&D resources and evidence of trials being dropped may be telling us something.
On our best day (not today) any story (+/_) we put together has big holes.
[quoteshareholders entitled to know WTF is going on with participants in the AD trial extension---where we stand with the Rett IND--what steps are being taken in the PD trial-][/quote]
Agree totally w/this
Finally, a solid explanation...dilly, dilly
[quote suspect that the delay has to do with the poor understanding of the reasons that the blood concentrations of 2-73 do not correlate strongly with dosage.
Without understanding what the issues are controlling blood levels ALL trials are at a much higher risk of failure. That includes the Rett trial][/quote]
Why are we only discussing this now? Game changer? or Not? First I recall anyone suggesting this issue?
Who is responsible to get an AD solution? Now that FDA is resetting it's CNS processes and BP are closing tents or reconfiguring resources, who is in charge. The old free enterprise model of "build it and they will come" seems to have run out of steam. Clueless. The silence is deafening.