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And that was them and I was talking about Anavex. We already know that Anavex doesn't get the love we think it should.
My point was that once there is an approval people will have to sit up and take notice. Right now Anavex is not taken very seriously. You can use whatever reasons you want to explain why that is the case.
Seems to me that Missling was describing how the market values drugs with some similarities to 2-73. Not an immediate value but rather an ultimate value.
It will take an approval for an indication to realize that valuation. Once there is an approval that buyout indicates what 2-73 may be worth. Probably setting a floor value since 2-73 has a real possibility of additional indications.
IMHO
Based on the wording of his previous comments where he started to say published on the date of and then changed his wording, I think he has been told when the paper will be published. He is not saying when that will be. I don't know if that is a publisher restriction or he just doesn't want to communicate that date and let it be a surprise.
Missling will continue to raise cash as needed via the same mechanism as used previously.
More likely they have been doing the ground work for several of the trials and that is already part of the current expenditures.
Setting up the trial documents, signing up testing sites, initiating site training are no high Dollar items compared to doing the patient screening and the genetic lab work.
You might add "This is a drill. No live ammunition will be fired. This is a drill."
I agree with that. I wish there was a way to make the emojis bigger so I tell what some of them are. Even if it was just being able to put the mouse pointer on them and click and have them enlarge just while you hold the button down.
There is one emoji that I have seen a poster use that I can not find in the library. I can't quite tell what it is.
I am perfectly able to make my own evaluation of the various posters. Name calling serves no one. It is repetitive and clutters up the board with meaningless posts.
Sorry, but that isn't true. I can't speak for others on the board but I am heartily sick of the FUD name calling and the pumper name calling.
Send it to IR with MA as the addressee.
Said the kettle...
I think it is because they don't see it as complete failure. The statement that they would meet with regulators to get guidance on going forward is the key.
The TLD is not the final word on the trial.
Yeah.... Like this is respectful of others. "Good licks and DOG bless,"
Gimme a break.
Is there some reason we are discussing other posters instead of the company, the FDA, the TGA, The EMA, 2-73, 3-71, Statistical analysis, Rett syndrome, Alzheimer's disease, homeostasis, gene expression, RSBQ variability, share price, short position, and whether pigs have wings?
Why are you posting that to me? BSIG is your made up thing.
Don't know about BSIG but the algorithms seem to like AVXL today. + 5.41% as I type.
I picked up some more Jan 2025 $10 options today.
I agree with what you are saying.
Anavex has to demonstrate that those changes are clinically meaningful for specific indications. That will require additional trials.
What Anavex needs right now is an approval for ANY indication.
The genetic data supports the idea that 2-73 has a measurable impact on genetic expression. It further specifies some specific genes that are increased in expression.
What it doesn't do is show that those changes are directly related to any change in the symptoms of Rett.
Those gene expression changes may become biomarkers in the future. The question right now is what are they biomarkers of.
What part of the word suspect do you not understand? A suspicion is not a declaration of fact. A suspicion does not automatically exclude other possibilities rather in general is expands the list of possibilities. Turns out there can be more than one factor involved.
Walter E Kaufmann, MD, Chief Scientific Officer of Anavex commented, “We believe that a high placebo response may have masked the therapeutic effect of this innovative orally available molecule. High placebo responses are well documented especially in pediatric clinical studies. Although data analysis is ongoing, the early conclusion is that the placebo rate could have been higher in the study due to a slight imbalance in disease severity at baseline, across the treatment arms, and the 2 to 1 drug to placebo randomization ratio. We intend to further assess the collective results and discuss with the regulatory authorities next steps.”
The words may and could are used. In line with my suspicion that another factor may be involved. Gosh, who knows, it might not be a factor. I suspect that it is.
I suspect the placebo effect had little to do with the missed P value.
It looks to me the it is the high variability of the RSBQ measure as shown by the very high SE values.
The variability happened to be high enough to swamp the response in the noise. RSBQ is an imprecise tool.
McFarland made a comment as a site manager for an AD trial that there were people that had headaches, if I remember correctly, and that indicated that they were on the drug.
Fine, you have now come up with a different explanation.
I get that the RSBQ is highly variable. The CGI is supposed to be done by Doctors who are supposed to be less influenced by expectations. Of course I get the zoom argument. That also applies to the placebo group as well as the treatment arm.
Unfortunately that argument applies equally to both the treatment arm and the placebo arm. Unless you can find a way to explain why the placebo arm would be more encouraging than the treatment arm that is not the explanation.
That is the number for the 4 week RSBQ measure, not the end of the trial, i.e. total RSBQ number.
George. What was the P value of the Total RSBQ?
Let's take this a bit more carefully and discuss the assumptions in both the question and the answers.
My assumption is the data from the first trial is indeed accurate and representative. You may argue that and if fact I think that is what you are arguing. If I understand what you posted you are saying that another trial might come up with different results because it is a different trial. That argument is basically based in the p<0.05 having a possibility of coming up with a different answer from the 5% random sampling possibility.
I'm arguing that another trial with a larger n can detect a smaller change in the primary variable with statistical significance than a smaller trial can. In fact with a very large trial a very small change in the primary variable can be detected with statistical significance. This is generally described by the use of the descriptor the power of the trial.
If there is in fact a real signal in a small trial that is swamped out by the random variations in the placebo response, a sufficiently larger trial will show that signal real signal statistical significance.
If there is no there there, it doesn't matter how much larger the trial is, it will not show statistical significance.
I'm looking forward to the TLR and and a deeper dive into the data.
We saw the RWE seizure videos. If the trial data supports that reduction in a large number of patients then this trial has a good chance of success. That might result in a conditional approval requiring a follow up P4. We won't know until the full results are available.
Do we know for a fact that the CGI interviews were done via Zoom?
Actually increasing the size of a trial can turn a failed endpoint into a met endpoint. The larger the trial the smaller difference between the placebo and the drug group is required for statistical significance.
I believe you are confusing clinical efficacy with statistical significance in this case.
I'm inclined not to think that enthusiasm for 2-73 was a confounding factor. There is no reason to think that the placebo group was any more or less enthusiastic about taking 2-73 than the drug group. After all, the subjects don't know which treatment arm they are in. That is the point of a blinded trial. Each group is led to believe they COULD be taking the drug. So the expectations should be the same in each arm.
To me it seems more likely that there is a high variability in the various measures and if you look at the SE you can see that they are pretty large.
The random variability in the measures broke the wrong way in this trial or perhaps it is what Anavex suspects. Anavex thinks that the placebo arm had a different overall level of disease severity in the placebo arm vs the drug arm and that skewed the results.
For example that FDA discusses the ADAS-COG measures of AD as not being valid until they are taken over a three year period because of the high levels of variability in the measure. It takes a long time to capture the real decline in function.
People have good days and bad days and behavioral measures capture that. If you are taking measures every week or better several times a week then the good day, bad day issue tends to get washed out. But trials don't take measures that frequently. You can imagine the difficulty in getting a Rett girl in a wheel chair dressed and ready for a possibly long car trip to visit the Dr's office for a series of behavioral tests and observations every week. That is a big burden on the girls and the caretakers.
I agree with that. I hadn't looked at it from that perspective. Good thinking.
Kinda like evaluating your posts?
I suppose it depends on what your definition of long shot is. If by that you mean based on the current pediatric trial I'll agree. If you mean long term I'll disagree. I think worst case is the FDA requires an additional larger trial. My feeling is that trial would succeed.
It all depends on what the FDA says. On that we can agree.
George,
You quoted the document out of order. If you think that is what the PR said so be it.
That is not the way I read it and re read it and checked again after reading your post.
Actually Anavex won't pay fees for Rett. It will for AD.
They can use the existing trial design with minor tweaks.
The feedback from the FDA will take a few months as you say.
Recruitment will be the biggest time portion of the overall timeline I think.
What is with your "won't allow an NDA" ?
Anavex hasn't filed one. The FDA can't disallow an NDA that hasn't been filed.