Replies to post #256622 on Anavex Life Sciences Corp (AVXL)

[Investor2014]

Stat sig topline results are not necessary for approval.

Opinion stated as fact!!!

[nidan7500]

Xena

We are not dealing with typical symptomatic treatment here, the MOA addresses the root cause of the dysfunction, cellular homeostasis, so I expect benefit to be wide spread.

Agree with this line of reasoning. Since CNS diseases have never been effectively treated (aside from some symptom patches) how do we know the current pass/fail trial metrics are adequate or even how meaningful to the status of the entire CNS? We are doing work that has never been done, we must keep an open mind. Hate to see us pick the wrong turn here.

BTW, the note here about heating water prior to dosing was a very positive example (imo) on the level of detail monitoring and control being applied by AVXL staff. assume nothing. Very well done.

IMO, this recent paper provides some good insights.

https://www.knowablemagazine.org/article/living-world/2020/single-cell-studies?utm_source=twitter&utm_medium=post&utm_campaign=originals&fbclid=IwAR0XhoBZ7XaX5FkuyoTSixFfbRcalytpl0RTtpoz7Z7qdydU09Ozjg0vQ0g

“Cells don’t necessarily care about their history,” Klein says. “They may have to transition to one identity in one place and another identity in another.”

[frrol]

What we're looking for, and all biotechs, pharma, researchers, and regulators look for, is a therapeutic effect that is above control (historical SOC or placebo) and is statistically significant.

Make sure you understand therapeutic effect and statistical significant. And don't confuse the two.

An experimental drug can have a few results above 2 SD in a large trial. That is apparent effect. But it may not be stat sig. Another drug can have no such outcomes, but the results are stat sig (ie, the trial confidently showed there was no effect). Neither gets approved. But if a drug has enough response signal, in magnitude and volume (size of response and # of patients), it can be deemed therapeutically effective and stat sig. They're both necessary.

You're hoping we just need to have a few folks who did much better than placebo to get approval. That's incorrect. And it's called bad medicine and bad drug regulation and is something to be avoided. Don't let the fact that you own shares in something cloud your judgement. Be wary of confirmation bias.

[Steady_T]

Substantial relief where there is none for all patients will be adequate.

That is the definition of statistical significance.

The relief has to be one of the end points being measured, and the "all patients" guarantees the result will would be statistically significant.