Replies to post #366730 on Anavex Life Sciences Corp (AVXL)

[Steady_T]

The Rare disease push had multiple benefits. First, was the small number of patients required which made it a relatively inexpensive trial. Second, is the SOC for the rare disease chosen is almost nonexistent which made the odds of success considerable higher in getting approved. Third, getting approved validates the MOA and increases the impact of a successful AD P2/3 trial in terms of negotiations for partnership. Forth, the pediatric Rett trial would yield a voucher worth a quick $100 Mil.

Alas the plan was laid out and working to schedule until the FDA said it wanted more subjects in the Excellence trial. At that point the schedule went down the tubes.

Now it looks like the voucher is unlikely and either Adult Rett or AD will be the first NDA filed.

[Gator328]

You use the rare disease to get the pediatric voucher to accelerate the profitable diseases. That's the focus point. Otherwise Anavex submits to the FDA and the application just languishes for months and months.

[Investor2014]

Instant MBA type gratification is rarely lasting.

There are plenty of a great things that can be achieved addressing the rare disease space, especially if the Anavex drug(s) work as hoped and eluded to. Nice space for Anavex to potentially own a good slice of all to themselves (and investors).

The challenge now is that the Paediatric Voucher, if we get it, is over a year way now that the EXCELLENCE study completion date has at last been updated to reflect the realistic timing end Dec 2022.

With DB lock, analysis etc. topline will be about end Q1 2023. Packing all the data, incl. co-analysis along with the previous adult Rett studies data will take another good few months.

Probably a Rett all ages NDA filing, like Neuren/ACADIA's just filed, can be around mid 2023. Add six months of FDA processing makes a potential approval and voucher be end 2023 at best to Q1 2024.

Will Anavex file an NDA for AD before then...and forfeit the voucher? Or will sweet F'(da)all happen until the voucher is insight?