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Saturday, 01/06/2024 11:23:56 AM

Saturday, January 06, 2024 11:23:56 AM

Post# of 459684
From Piotr...

https://www.patreon.com/file?h=95835549&i=17186530

Conclusion
1. The bias present in the RS-003 placebo arm toward seeing improvement in girls was excessive and unpredictable.
2. There is a question why there was such excessive bias toward improvement in RS-003 placebo arm? Is there any plausible explanation? Could be networking among the families of girls?
3. This reminds for the failure of BioVie, due to no fault of the drug or the company.
4. It is very likely that $AVXL might reach out to regulatory agencies with the available data for special treatment. All due to the case of a better drug failing trial due to misfortune placebo arm.


https://www.patreon.com/file?h=95739679&i=17191263

Conclusion
1. The bias present in the placebo arm toward seeing improvement in the girls was excessive
and unpredictable.
2. The inherent dispersion of the RSBQ scores prevented reaching statistical significance
together with the biased placebo arm.
3. Effect Size Cohen’s d is a better measurement to approve drugs than p-values.
4. Blarcamesine should be approved for Rett syndrome


https://www.patreon.com/file?h=95875572&i=17193703

Conclusion
1. The study was correctly powered and had ample margin based on the experience in trial RS-001 and its analysis.
2. The outsized improvement in placebo arm, both unpredictable and probably false caused the study to fail reaching status of statistically significant.
3. The statistical significance was missed by a small margin due to large therapeutic effect still present in spite of the disastrous placebo effect.

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