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To paraphrase your long post it essentially says, if everything goes to hell in a hand basket the company might not survive.
I bought 1500 of those shares today @ $5.60
So there were no BUYS today?
I mistook the acronym MAA to mean it was an EMA application not a UK application. I didn't grasp the MHRA signifying the UK.
The UK does in some few circumstances count business days but for the MAA application they count calendar days.
The EMA counts business days.
My bad.
The FDA will be the judge of that. If the FDA considers the trial a failure it is likely that the FDA will require an additional pediatric Rett trial.
My point, which you seem to be overlooking, is that the Rett program is not dead that is what the poster I was responding to said.
You are willing to put a stake thru the heart of Rett at this time. I suggest that is a mistake. The status of Rett is not known until the FDA renders an opinion.
If the FDA says go ahead and file an NDA then things are back on track. If the FDA says run another trial then AD should be full on.
Even so, Anavex has the resources to run multiple trials at the same time.
At this point AD is at the NDA preparation process stage, not running trials stage so the cost is manageable. Running the 3-71 trial along with an additional Rett trial would not be a huge cost. Fragile X trial will not be a long trial and will likely not require a very large number of subjects so its costs will also be manageable.
Two things will determine Missling's ability to raise money at a reasonable cost. The FDA Rett decision and the contents of the AD peer review paper.
Until those items are known Anavex and the SP is in a holding pattern IMHO.
To use your argument, the heightened expectations of the placebo group should be the same for the treatment group. After all, it is unlikely that the subjects knew which arm they were in. If you claim that the kids and care givers in the treatment group were able to figure out that they were in the treatment arm then it would seem that they had lowered expectations compared to the folks that had not figured out which group they were in i.e. the placebo group.
The reason given by the company for the strong placebo response ascribed it to different levels of disease severity between the two arms. The random selection process when selecting small numbers from a larger population can sometimes result is a less representative group being selected. A larger selected group tends to generate a more representative group of the larger population.
Validated biomarkers for Rett would be very helpful in this regard, as you say. Unfortunately the genetic changes found between the treatment group and the placebo group are not yet validated biomarkers. At this point they sure are suggestive.
To quote Elon Musk GFY.
I think the technical term for what you propose is "a miracle."
If you keep slamming the stocks you are invested in, it is not a surprise. That is not in your best interest.
I get serious discussion of a company's strengths and weakness. Calling your investment shit on a regular basis seems more than a little counter productive.
But if whining publicly makes you feel better I guess you might as well, since I am told what is posted on message boards seems to have little effect on the share price
Be aware that the days listed in the EMA MAA schedule are not calendar days they are work days i.e. Monday thru Friday less holidays.
Good grief. I understand how to compute effect size etc. I also understand the consequences of high variability in scores resulting in a large standard deviation.
And there is a real possibility that a larger n in the placebo arm might have reduced the standard deviation and perhaps the mean.
Random selection has some issues especially when the n of the selected population is small. The smaller the n the likelihood that the selected population is less representative of the overall population. 30 is considered to be the smallest n that is normally used with the common statistical tools. An n less that 30 usually require the use of different tools.
There are both type 1 and type 2 errors.
Yes I understand that. As I said, I should have used absolute effect or, perhaps even better, the magnitude of change in the treatment arms.
I am posting on a message board not writing a scientific paper for publication.
Did I say that the difference between the drug response and the placebo didn't matter? I don't think so.
I was pointing out something else. You can agree with the point I was making or not as suits your fancy.
Frankly Scarlet...
Please reread my response.
Again, there is something inconsistent about your posting.
You seem to be be saying "I'll either lose big or win big." Do I have that right?
Yet your posting is 90% negative towards the company. Seems like you are rooting to lose big.
I did. I am discussing the absolute change in the treatment arm between 2-73 and Trofinetide.
Doc's discussion was about effect size in comparison to the placebo within each trial. Different things being discussed.
I probably should have used the words absolute effect size which would have been clearer.
If your cost basis is $4.01 sell now and take about a 50% profit. Most investors would be very pleased with that. After all if this is going to croak as you seem to expect you can buy back later if you change your mind about where Anavex is going.
There is something inconsistent about your posting.
The Rett trial showed a large effect size. What it didn't do was show statistical significance compared to the placebo.
Recruiting will take the longest amount of time. I would expect the trial size to be larger and probably a bit longer. Both of those changes would mitigate against a strong placebo effect.
Worst case for Rett is running another trial. Setback? Possibly. Dead? Not likely.
Since when is bringing a new patent application to the board a pump attempt?
The Rett data I am most currently interested is the seizure logs from the participants. The anecdotal responses we have heard about are dramatic and life changing for those that experienced them. If the reduction in seizures is seen in a significant number of subjects that will be a very important trial result and will guarantee Rett approval but not necessarily without an additional trial.
I suspect a peer reviewed paper is not cause for a meeting. Perhaps a supporting document in concert with other docs to discuss a specific agenda item.
Meetings with the FDA are not casual affairs. The documents and information have to be provided to the FDA well in advance of the meeting to allow the FDA people involved to review and come to the meeting informed with their own thoughts and questions. The guidance and recommendations that result from a meeting are considered to be the official position of the FDA. As I understand it those are not legally binding but one challenges the bureaucracy at one's own risk.
As I see it a peer reviewed paper would be included in an NDA as a supporting document.
Doc can weigh in on this. I see a peer reviewed paper as legitimizing the company's statements about a trial outcome. A more in depth review and analysis of a trial and the conclusion derived from the trial. It should have sufficient information to allow knowledgeable people to arrive at their own conclusions concerning the trial methodology and results.
A positive peer reviewed paper will be taken seriously by the biotech investing community far more than the company's pronouncements about a trail and the results.
There are clearly defined meeting types for meeting with the FDA. Each meeting type has its own requirements in terms of time frame, required submissions, subject matter areas etc.
The FDA has said that it likes to see peer reviewed papers. Is it a requirement? No. It does represent another review process from a different set of eyeballs.
My guess is that Anavex has been working on 2 or 3 different drug applications of late.
It seems likely that they were working hard on the Rett NDA with the expectation that the trail results would allow for immediate NDA application.
They also have been working on the MAA with the EMA as we have seen with the recent authorization to proceed on AD.
Now that the Rett process is up in the air I would suspect that Anavex is also working on the AD NDA for presentation to the FDA.
There is some commonality between the EMA MAA and the FDA NDA for AD and there are a lot of differences. The EMA requires drug labels and literature in 24 different languages for example although that is not due until 215 days into the CHMP review process.
The disease, the progression of the disease, and any biomarkers of cancer are much better understood than CNS diseases.
Probably the most important is the ability to see the tumors and cancer cells via diagnostics such as x-rays, MRI etc. CNS has nothing like that degree of clarity into diagnosis and disease progression or regression. Some CNS diseases are getting better in genetic diagnoses but in regards to treatment there are still questionnaires and patient history logs compared to shrinking tumor size for cancer.
Case in point , how variable the placebo response was in the recent Rett trial. ADAS-COG is known to be highly suspect over the short term as are most observational scales.
Another post that contributes nothing to the board. Why bother?
Just like Mayo, Anavex is the only stock you report on. For someone looking for outsized returns in a high-risk sector you have a very restricted portfolio in that sector.
Does anyone care that you noticed?
Actually the words he used were backup plan.
Remains to be seen what the FDA has to say about it.
The existing sites can be used if a new Rett trial is required. The sites will need an update on the protocol changes which won't be much.
I am disappointed that there was no new information about the FDA meetings and no mention of the TGA.
All in all it wasn't worth getting up early for.
That also explains why the shorts are after Anavex. History is on their side.
We will see if Dr. M can get Anavex to the other side of the valley of death to revenues.
Sorry. I have made my opinion clear. No need to discuss it further.
It's not like it matters who made the initial evaluation of the request for central marketing procedure. The point is the request was approved and now the MAA procedure has started.
No. I don't agree.
More likely the CHMP is the group that reviewed the initial request for centralized procedure. They are the group that has the expertise in that area for the EMA.
By the way, Anavex had no choice but to apply for the Centralized procedure. That is a requirement for CNS drugs.
Where did you see that sequence?
It would not surprise me if Missling did not PR the letter of intent. Making that public allows everyone to start a clock including the shorts.
I know there is an accelerated review process available with the EMA once the MAA is filed. I don't know if there is a possible path to shorten the time from letter of intent to MAA submission.
As early as possible means just that. We don't yet know exactly what is possible.
MAA filing is 7 months after the letter of intent. So far we haven't had any information on a letter of intent being filed.