InvestorsHub Logo
icon url

Kentucky123

08/28/21 7:29 AM

#327954 RE: powerwalker #327953

[4) many people will go beyond a point in which blarcamesine might help them by waiting an additional 18 months./quote]

Please help me understand the above. thanks

icon url

tredenwater2

08/28/21 12:20 PM

#327994 RE: powerwalker #327953

Great points power! Alot of talk on one side or the other about “peekyboo” but another few scenarios that may in play are:

1) 2 very intensely studied peer reviewed papers are past due and are due any day.

2) Every Ph 2, in 3 different diseases will of had a peer review and went (and or most likely will “go”) into a pivotal Ph 3.

3) 2 more Rett partial readouts on top of this in the next 3-6 months

4) Initiation of a “Preventative dementia trial” in the next 6-12 months

4) Ph 3 Fragile X initiation in the next 6 months

5) Ph 3 for an Undisclosed rare disease. •’On a side note, this is the wildcard in play imo. Dr M mentioned recently that “ we have got to get this trial right, we have so many great pre clinicals to choose from, we need to pick the right/best one.” (Paraphrased)

6) “Highly enrolling/Enrolled OLE’s in every disease.

The list goes on and on and on….. what I see, if any, relating to the interim Ph3 Alzheimer’s trial peek is that Dr M would only consider looking early for a major benefit. One such benefit would be for it to further corroborate aaalllll the data to date. In other words, after the remaining pivotal Ph 2/3 start and Peer reviewed papers are out, it somehow, its results on say the first 225 in AU would seal the deal for one giant, extremely combed, data set package (some up to 6 yrs for safety) ends up being submitted for the rolling start to one giant Ph 4. Look at the evidence above, thats what it is! The snowball is rolling down hill, getting larger as it rolls picking up speed and many, many more will come all for one drug! Get ready folks! There is no coincidence that the trials were started in a carefully staggered stage, with “minor delays”/ accelerations, to end up with most if not all of each trials data coming together at one point in time.

“The front end of our work (proving cellular homeostasis) is/will be very time consuming and difficult but in the back end (after our master protocol/umbrella/Ph 4 “ preventative trial starts) we will see great benefit/ease from it (adding rolling pre clinicals to “it” as Ph 3’s ).” (Again paraphrased and I added all words in parenthesis)

In closing, this emergence or moreover the explosion of data to date as a slow volcanic eruption shooting high into the air, large safe clouds of symptomatic relief high into the air slowly encompassing all regions of the world!

We got this!

Tred
icon url

boi568

08/29/21 1:10 AM

#328039 RE: powerwalker #327953

"Anavex understands how well everyone is doing as over 95% signed up for the OLE, probably similar to PDD trial which showed significant numbers in a short period of time (12 weeks)"

Anavex surely has a lot of useful information out of the ongoing OLE, and the high enrollment rate is another data point. However, an OLE is an uncontrolled look, without a placebo arm, and therefore we cannot ascribe a p value to its results. That's what the trial itself is for.

"2) for an international study of 450 participants, it is strange that the first 225 were selected from only one country; this was obviously intentional with a specific, well-guarded reason for such action"

There's no reason why this wasn't based simply on logistics and/or financing. Australians have the same biology as everyone else.

"3) we don't know if Anavex is peeking or not as Dr. M does not give any hints of what Anavex is doing, other than offering a tidbit that it might peek"

Come on. There hasn't been a peek, there is no basis to believe there has been an efficacy peek, and even your own language above supports this conclusion.

"4) many people will go beyond a point in which blarcamesine might help them to recover/improve by waiting an additional 18 months for those final numbers."

That is true, but also incomplete. Regulators are measuring outcomes against an unlimited time horizon, not just the next 18 months or so. In that context, they need to know that the drugs they approve will be effective. Cutting short the proof process means that marketed drugs may not work at all, and this would clearly be at the expense of people paying for something useless (think Aduhelm as an example of a regulatory failure) or, worse, harmful (think Thalidomide in Europe in the 1960s). They require that extra time to get the decision right, and cutting out that time carries an even heavier price to public health.

"The numbers are the numbers whether a peek occurs or not."

Actually, the numbers aren't the same numbers as time goes along. First, we have numbers on one person, then two, then three, etc. Eventually we have the numbers on the n that the company and the regulators agree will provide a trustworthy answer. Ultimately, the drug may work or the drug may not work "whether a peek occurs or not." But we won't know for sure if it works unless it passes the necessary statistical tests for efficacy and safety, and for reasons that have recently been explained here, passage of the efficacy test can be imperiled by an interim peek.

"Also, has anyone provided the name of a trial and sponsor that took a peek early, which caused its number to be non-significant at the end? If so, please re-state."

I don't know where to look for that to do your research for you, but even so it would not lead to a scientific conclusion. The premise of your request is that anecdotes are proof, or a lack of anecdotes are proof. Neither is true, either in science or in other areas, such as social policy, no matter what people have been misled to believe over the last few decades.