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Bas...agree completely with your thought. When(if) we ever get to understand the whole story behind this set of trials we will see politics, money, science, medical factors and (yes)of course patients are being considered. IMO, W/O Gottlieb/Dr.M. this would not be happening and A2-73 might never see the light of day. BUT, Said different, if BP had A2-73 it would be headlines in all media. IMO.
Dado...thx for your comment. Coming from you this is a really positive input. Best.
roy...helpful post...thx. We know the bias against progress/risk taking has prevailed. Details matter when medical/political demons are under foot.
raja..
Investor..nicely done....an old street expression comes to mind while reading it..."If you think I did not cut you, try moving your head."
T 38...First of all thanks for your service to the country.
It seems from your posts that you at very well informed on the people driving the bus and the mission they are on. We all appreciate your insights. My own read is from a process standpoint. They are driving for changes to a massive system which (IMO) has not served the country well over past decades and which urgently needs change if we are to ever get out of the woods.
I also agree that AVXL and FDA leadership are positioned to accomplish a breakthrough (provided A2-73 does as advertised-Homeostasis in CNS). We sense that this is a first step in a true leadership initiative. I also sense there is more than just inertia holding them back. We ref here how long it took for medical science to understand and apply the benefits of penicillin as an anti biotic compound. IMO, this is a useful analogy to apply to A2-73. Gottlieb and Woodcock have their work cut out for them but as you indicate, they are on a mission of massive urgency and importance. Now that is time well spent.
BTW, U.S. Navy here VS-28 S2, many night traps as A/C. We will be turning into the wind shortly. Stand by.
Just in case you wondered...source
http://www.sciencemag.org/news/2017/04/congress-and-fda-nominee-heap-love-adaptive-trials
reader/User friendly article on Adaptive trials...IMO.dated may be repeat
Anybody else recall the FDA news release about a month+ ago that talked to the new rules and the fact that 4 tests already in the queue? That news said 4 trials test cases had already been approved and were ready to go w/new trials protocol. I have gone back and cannot find it but am certain of what I saw. Anyone else recall? thx
Gotta like this:
You folks take good care, we are with you. A2-73 will help many brave people.
If they only knew...best selling drug will be...1.3 trillion$ mkt w/o A2-73
https://www.fool.com/investing/2017/09/16/which-of-these-5-will-be-the-best-selling-drug-wor.aspx?yptr=yahoo
You nailed it..thx
F'ing brilliant
Huh. Good point. Maybe that answer involves understanding why we have had silence (other than to declare dates/goals). The events can only be explained if one considers that the plans are/were completed and they include AVXL as one of the poster children for the FDA rollout. Then the pieces start to fit together.
[quote Just relax][/quote]
The comment is relevant and should not be shut down, as it addresses context. The FDA rules establish what, when, how, importance and in so doing the context for all trials. This automatically establishes risk profile/evaluation criteria. We know there will always be some residual risk. Simply stated risk in this context is, what could go wrong and if it did...then what?. This entire exercise (trials) is an exercise in risk mitigation. We get that medicine is tricky and that Biotech is a crap shoot. We also get that the antidote for risk is information w/knowledge applied in some context. Understanding risk is the opposite of FUD. Got it. BTW, we are just about all on the same page here and appreciate all points of view, just about.
Trader, I think you have explained the AVXL position well. We know how pissed he was b/c he had to spend time/money on the phony legal action last year.
Like this wording:
FDARA builds upon the goals outlined in previous user fee agreements and in the 21st Century Cures Act and will help us continue the essential work we are doing in many of our priority areas. The new law provides critical support for important FDA activities related to medical product regulation, including:
Enhancing our ability to capture the patient voice in drug development.
Allowing FDA flexibility to inspect medical device facilities based on risk, enabling the Agency to focus its resources where they are most needed, while providing greater predictability and transparency to the inspection process.
Peter Marks, M.D., Ph.D., is Director of FDA’s Center for Biologics Evaluation and Research
Advancing and facilitating the development and timely approval of drugs and biologics for rare diseases, including diseases of children. In particular, FDARA provides the FDA with new authority to require a pediatric investigation into an adult cancer drug if that drug is directed at a molecular target that is relevant to a pediatric cancer.
Providing resources for the popular, highly successful, and resource intensive breakthrough therapies program for drugs.
Continuing to leverage the use of “real-world” health data to inform regulatory decision making, including enhancing the capabilities of FDA’s Sentinel System for drugs.
Strengthening our partnership with patients, by providing funding for the development of the National Evaluation System for health Technology (NEST) to help pay for a NEST Coordinating Center and pilot projects. NEST is intended to facilitate the use of real world evidence to support premarket activities.
Establishing a flexible and more efficient path to market for certain new medical device accessories, to enable new and innovative accessories to come to market more rapidly and enable accessories to be used with a wide range of devices – creating important options for patients.
Creating a category of over-the-counter hearing aids which will help lower costs and enable access for patients who greatly need these devices.
Providing new opportunities for early consultation on the use of new surrogate endpoints.
Streamlining combination product review to enhance coordination and transparency between FDA and industry.
Improving FDA hiring and retention efforts including a dedicated unit for scientific staff and continuous independent assessment of the Agency’s hiring and retention operations.
Insights on how FDA process guidelines get modified/published in a situation like this? What should we expect in terms of elapsed time between publication and effectiveness. For example, in this case I would expect that the new guidelines can be effective immediately, etc.
I have read FDA site pubs and have an idea of what should be going on but just in case we have any experts I wanted to get inputs on the process.
roc...agree w/your assessment...illustrates the changes we will see.
Agree...the poster content will likely be modified to present facts on trials which cannot be publicized at this point. W/luck trials will be 1/2 done by then.
Thx...any thoughts on comment "will start" poster being done in November?
Good, we will need them. This is a classy problem to have but it needs to be put on the table. Producing A2-73 to support WW requirements is a non trivial task, we are going to need a structure/system/etal.
If there were contradictory or simply non supportive factual information on the CNS restorative/regenerative possibilities of A2-73 I would be less positive. If we were having debates about one scientific school of thought vs another (possible/not possible) then I would be listening/concerned. But there is non of that. A lot of smart people would like to blow the whole thesis to bits but not a peep of factual argument. What we have are endless "he said-she said" BS trivia which is fundamentally pointless IMO.
We now see today that the new Director of the FDA is saying basically the same thing that Dr. M. has been saying for a long time...he is saying that is the direction the FDA are headed in. He did not say AVXL is a brilliant solution but he also did not say "More Plaque research is the answer" either. He did say it is possible there is another "bacteria" level of new world breakthrough out there and he is looking for it vs keep doing more of the same. He is ready to acknowledge it.
I am still connecting the dots as I see them. I see Red, Falcon, Jimmy, Iceberg, bear, dad and others who are pretty damn capable people (to the extent I am qualified to judge)biased possibly but not stupid enough to let that bias drive their investment. We want this to work and will keep connecting the dots. IMO, now that the FDA has come out showing some cards we will see things start to happen faster. As soon as some credible contradictory data presents on CNS homeostasis being a non possibility and possibly the thesis cannot be proven.
Last point would be to evaluate what has been done over the passed 20+ years with the benefit of hindsight. No AD patient ever stabilized or got better/recovered....ever, kids still have Rett, people still have PD, MS...etc. That is just wrong. More of the same would be wrong.
It is easy to become self concerned to exclusion of others when following a Bio. The real people who will benefit from A2-73 are the patients. We need to keep them in mind. They and their families have a lot at stake here.
We are basically up.30 then flat @t+50mins, BIIB is down slightly.
FWIW,,,quick scan for PR's shows that other organizations at conference have no related news so far either. Eventually we will have something, silence is not a sustainable comm. strategy.
https://www.gtcbio.com/conferences/cns-diseases-summit/?section=fullagenda