Replies to post #417169 on Anavex Life Sciences Corp (AVXL)

[BAR123]

So can someone help explain something to me as a layman. With all the trials that we have completed for Alzheimer’s along with the success we have had with our primary and secondary endpoints, we are now looking for approval based on “other endpoints “ or biomarkers . That being the success of reducing the abeta and tau in the brain as well as or better than Biogen and Lilly.

Does that mean that our endpoints don’t matter anymore or just an added benefit that we improve cognitive performance? Do we have to be better than what is now approved with reducing abeta and tau or just close to them because we are just a pill and we don’t require an infusion every 2 weeks? It seems weird to me if this is accurate that we can get approval based on this rather than all the great results we have had on cognition and all the other side benefits of Blarcamisine.

[Joseph_K]

Sorry, but I'm not quite following. I can't tell if you're agreeing or disagreeing with Anshu when he wrote:

First assumption is that FDA will only give AA without any subgroup analysis, except for the dosage grouping. This assumption is extremely likely to be true.

With that assumption — a confirmation trial would need to show clinical benefit for the same population as in the P2b/P3 (else it wouldn’t be confirming the basis of AA).

His logic seems narrowly sound (that AA requires a confirmatory trial, and the trial wouldn't be confirmatory if the population has been changed), but in the big picture it feels wrong to me.

The part of your post I'm confused by is the first paragraph:

If Anavex gets AA based on biomarkers for the 2b/3, it will be a fairly broad-based approval -- without taking on any risk for label restrictions based on genetic status. That possibility exists in the event the FDA requires a second successful Phase 3 with Precision Medicine limitations (upon receipt of an NDA for final approval).

First off, when you say "in the event the FDA requires a second successful Phase 3," are you counting the P2b/3 as the first successful P3 or talking about two more trials? I'm guessing you're counting the P2b/3. Then it sounds like you're disagreeing with Anshu, but without directly addressing his point that a confirmatory trial by definition should be for the same population as the trial it's confirming.