Replies to post #350455 on Anavex Life Sciences Corp (AVXL)

[sab63090]

Investor:

Makes sense to me...

So, it seems to suggest a waiting game and gives times for the competition to outperform unless:

A surprise approval from Australia, UK, etc.

[boi568]

I understand your argument to wait until EXCELLENCE and I follow your point about expanded access under an IND.

What I don't follow is how expanded access under an IND can occur if Anavex is still going to wait to submit an IND following EXCELLENCE results. Do we know if a rolling submission, pre-EXCELLENCE, would be sufficient for an expanded access program?

[LBSR TO DA MOON]

Not quite sure how anyone can argue with your statement below. It certainly seems reasonable to me. Approval of A2-73 for Rett (both adult and pediatric) and the issuance of the voucher (possible value in excess of $100M) within the next 12 months? I'll take it! Of course, any earlier surprises would be more than welcomed...

An obvious way of putting any doubt to bed would be to wait for data from the much larger EXCELLENCE trial and look for even better response grouped by age and using the exact same primary CGI-I Anchored RSBQ AUC endpoint measure. Those score being well excellent in a 3rd parallel trial should put any statistical, clinical meaningfulness and questionnaire based scoring reliability concerns to bed and lead to approval of A2-73 for Rett in all age and the issuance of a the paediatric voucher.

[Boopka]

Investor, given the fact that the Avatar coherts probably had more advanced cases of Rett as compared to Excellence my question is not as much as waiting for confirmatory results from Excellence to back Avatar results. Instead, shouldn't the FDA look at Avatar independently given the fact that that is what the FDA wanted Anavex to do in the first place? If the FDA just looked at Avatar results alone and the safety and benefit it provides these terribly afflicted women don't you think the evidence is enough to stand for approval by itself? I do. Peter's breakdown sure seems to provide further confirmation as well. Just my 2 cents.

[Gernee20]

doubt Anavex would actually apply to recover costs and probably just serve the Rett community in the U.S., Canada, Australia and the UK assuming they invoke their respective versions of Compassionate Use. Upon approval, Anavex can work to set the actual charge for the drug appropriately without having established any precedence and expectations at that time with a Rett community already at least to some extend using blarcamesine daily.

Great post, I agree 100%

[jimmy_mcyoloswag]

How high/low do you gauge the impact of the "Rett Families meet the FDA" online meeting March 11th on the Rett approval timeline?

https://rettsyndromenews.com/2022/02/09/rett-community-perspectives-shared-online-fda-meeting/

[Jonjones325]

Not sure why Missling wouldn't just go for approval for adults.

I guess the voucher is more important than revenue.

or

He doesn't think he can get it and will settle for expanded access to give patients much needed help and to build up the customer base.


I believe the approval is more important if he can get it.

[end2war]

Of course the drug clearly appears to work, but still using the exact same measure and zero drug PK exposure, 38.5% of placebo patients are Responders and this is quite high. Higher I would think than most of us would have expected especially given the more stringent endpoint measurement.

One thought that I have about the high false positives is that it shows strong support from the caregivers/family which think their patient is improving, and that bodes well for A2-73 to get support if an application is filed with the agencies. Having good support for the drug that has some subjectivity will be a positive force for approval, and I think the parents will be influential.

Another thing about his false positive score is that there will be some key placebos to watch in the 48-week extension study. How do the false positives do with the real drug over 48 weeks; and how do the placebos do that were rated non-responders? Do the switch to clear responders with big effects?