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crescentmotor

02/11/24 1:36 AM

#451628 RE: Investor2014 #451624

What we are looking at is to likely a large extend a notoriously difficulty in measuring with any accuracy treatment effects in Rett as an indication without established objective biomarkers.



The only thing that makes sense to me is putting full stop on Rett until the AD situation firms up. It's fine to chit-chat with the regulators about things but no undertaking of expensive new Rett trials. AVXL's $146 million cash position does not provide a lot of attractive options and the hook is already set for advancing 3-71. Missling must choose wisely--he cannot just continue floating downstream wistfully waiting for a current to appear. The important unanswered question in my mind is if AVXL can move forward in AD and rope in a strong partner. Having to dilute the shareholders holding a sub $5 stock is a fool's errand. Right now Rett is dead in the water and should stay that way for a while--just my opinion.

Steady_T

02/11/24 2:13 AM

#451629 RE: Investor2014 #451624

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.