Replies to post #349307 on Anavex Life Sciences Corp (AVXL)

[ImGettingBetter]

Red - Great Job!! Get this post documented in a Yellow Sticky at a minimum. Plus you are hired to get the Weekly Report started as I posted earlier... LOL.

Loved to be able to do a one shop of info like this that is kept current for this board.

Appreciate you!!

[end2war]

Thanks Red,

That conversation with IR help confirm my understanding of what they are saying, and I don't think anything wrong has occurred to break the required blinding.

AFs comments have been fully and creditably rebutted in my opinion.

The only real issues remaining are whether the FDA determines the AVATAR study is strong enough to support a NDA, or if the FDA will advise it needs more evidence for a pivotal study to be considered.

[Investor2014]

Thanks for that recap Red.

This bit is causing me bib bib bib, does not compute trouble.

Q: You are confirming that the FDA directly requested that Anavex change the trial to a Phase 3 and that the endpoints be changed?
A: Yes I can confirm that.

It is inconsistent with the official 8-K files PR as quoted below.

Following the successful completion of U.S. Phase 2 Rett syndrome study (ANAVEX®2-73-RS-001) as announced in December 2020, and the knowledge gained from it, the AVATAR study (ANAVEX®2-73-RS-002) appropriately updated endpoints according to ICH guidelines were approved by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) and in Australia by the Human Research Ethics Committees (HREC), where the AVATAR study was conducted. Subsequently the AVATAR study was updated from a Phase 2 to a Phase 3 study. The January 2022 update to the trial description in ‘clinicaltrials.gov’ was not a real-time communication and may have given the wrong impression of a late change of trial endpoints / phase of the study, which is not the case.


Based on the results of the AVATAR Phase 3 study (ANAVEX®2-73-RS-002) and the prior successful U.S. Phase 2 (ANAVEX®2-73-RS-001) study in adult patients with Rett syndrome, Anavex is planning to meet with the FDA to discuss the approval pathway. There are no FDA-approved drugs for Rett syndrome. ANAVEX®2-73 has Fast Track designation, Rare Pediatric Disease designation and Orphan Drug designation from the FDA for the treatment of Rett syndrome.

According to above the changes made to the AVATAR trial were NOT approved by the FDA. It does of course not mean that the FDA might disagree if or rather when Anavex plan "to meet with the FDA to discuss the approval pathway". When is that meeting planned for; soon, just around the corner or year-end after the EXCELLENCE readout, that is THE question?

I don't believe the FDA would proactively reach out to Anavex, or any company, with advice to change the primary endpoint and upgrade the trial from P2 to P3. Guidance from the FDA would come during a formal meeting appointment. Just like I am sure Anavex had planned meetings with the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) and in Australia by the Human Research Ethics Committees (HREC).

The only time FDA will reach out directly is to halt a trial for safety based on a DSMB/DMC trial report.