Replies to post #328089 on Anavex Life Sciences Corp (AVXL)

[Steady_T]

All P3 trials are a big risk. The larger the trial the more sensitive the trial is to statistically valid effects. In a very large trial the effect can be quite small and still be statistically significant.

Because an effect is statistically significant does not mean that the effect is clinically significant.

Many drugs have an effect on 30 to 50% of the population. That drags down the effect size in the stat analysis because of the people that the drug does not affect are lumped into the analysis. One of the advantages of precision medicine is that it identifies the sub population of the general population that the a drug will affect. That allows the statistical analysis to be done on just that population without the dilutive effects of the non affected population.

P3 trials do have an element of safety trail involved. The numbers in P3 trials are larger and are more likely to discover low frequency significant adverse events that a smaller n trial might not see.

[boi568]

You have responded on two points. The first relates to the process of achieving regulatory proof for a drug's safety and efficacy. The first post in this chain was blarcamesine-specific, but since the issue it raised was a more general question my response was not blarcamesine-specific. That's because Anavex doesn't get special treatment from regulators. So you have misread my comment in that respect.

I agree that 2-73 is safe. There was just a safety peek that confirmed this. However, the general point I made remains intact, that there is a higher price attached to cutting short the drug approval process than might be gained in the short term by more quickly marketing drugs that ultimately will be found approvable.

You have made two errors here: first, misconstruing the context of my remark and second, making the as yet unproven assumption that 2-73 meets FDA efficacy standards.

As to your Australian argument, you are incorrectly characterizing the 225 participants there as constituting a separate trial arm. Not so -- there are three trial arms based on dosages and placebo, not on geography. The Australians may be in their own time zone, but they are not in their own trial arm. And what if they were? How do you justify stopping one trial arm ahead of the others when in a placeboed trial they must remain linked?

[Investor2014]

History of Changes for Study: NCT03790709 ANAVEX2-73 for Treatment of Early Alzheimer's Disease

When you follow this link you will see a side-by-side comparison of the original and latest updated records of the AD P2b/3 trial.

The trial always anticipated to enroll 450 patients. There is no such thing as a separate 225 person Australian trial, just continuous enrolment with the first sites initiated in Australia followed overtime by Europe and North America.