Anavex Life Sciences Corp (AVXL)

[boi568]

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).*

The challenge to confirm an AA should be met with a replication trial, although in that trial I would expect a bit longer duration (to strengthen clinical effects), and perhaps some modification on when doses are taken to lower dropout rates for dizziness, etc. There may or may not need to be a higher n under those conditions. And without a placebo group, i.e., a Phase 4, given the problematic ethics of failing to dose participants while 2-73 is otherwise commercially available.

This would be the most profitable outcome for Anavex, although I believe targeting the drug more carefully would be more scientifically sound. However, even that benefit would be offset by a years-long delay in getting the drug on the AD market in the first place -- at quite a significant social cost.

* I should reiterate I view this as unlikely, if only because the FDA's risk/benefit balancing test favors approving such a safe AD drug as blarcamesine.