Replies to post #429237 on Anavex Life Sciences Corp (AVXL)

[abew4me]

The other option is to wait until Rett is approved and repurpose the drug for AD.

Submitting the P2b/3 trial results with a FDA-approved drug should be sufficient to substitute the need for a second P3/4 trial.

Remember, the FDA can approve an NDA (New Drug Application) with one successful P3 trial and the repurposing of an FDA-approved drug.

So, if the FDA approves A2-73 for Rett, that would qualify for the first half of our Alzheimer's application. The other half would be our P2b/3 AD trial.

IMO, this is the main reason why Anavex wants the FDA to approve Rett first. Once it is approved, they can expedite the entire process for our Alzheimer's application by simply repurposing A2-73. (Qualifying for the $100M voucher is a bonus)

I'm not worried at all because our FDA guys know what they're doing...especially Dr. Jin.

[frrol]

Meeting those OR endpoints didn't mean anything, unfortunately, given the missing data. ORs over 1 or 2 in those circumstances do not confirm efficacy. Math. If the missing data for those ORs are strong, not only will some subgroup ORs be better but, uncoincidentally, so will some sub-group mean changes. Math. It's the latter that would confirm (or not) efficacy, as shown in the past. Regulators will also pay attention to pTau and AB biomarkers, as shown more recently.

The way our AD trial would not lead to AA is firstly inadequate efficacy or biomarker support. If good but not great, our relatively small n will likely then be an issue for regulators. Some might not care, some might.

Unfortunately, looks like the company is struggling with getting clear dose sub-grouping. This is due to tolerance issues first surfaced by the 2a. (Noted by a couple investors here in 2016.) S1 status might be fruitful. PDD had S1 subgrouping but the Rett trials didn't. So S1 is not certain. Plasma concentrations is a possibility. Also pTau and AB clearance levels.