Replies to post #401402 on Anavex Life Sciences Corp (AVXL)

[boi568]

I find it very hard to believe that the FDA will require an entirely new Phase 3 AD trial if the 2b/3 shows it met its efficacy endpoints, as announced, and is safe. The FDA's mission is to promote and protect the public health, and putting off for several years a safe and effective AD medication that outperforms the SOC is destructive to that mission and, I'd argue, a violation of medical ethics as well.

More likely would be an AA with a Phase 4, or possibly a decisional delay until the OLE reports out in 2024.

[Investor2014]

The next trial may also be P3 Precision Medicine trial.

Just for the sake of completeness of course.

[TempePhil]

This FDA link describes all 4 designations.

FDAs four designations- link

As I see it, for Alz, Blarc easily qualifies for Priority Review and Fast Track designations.
Blarc may not qualify for Breakthrough Therapy and Accelerated approval, as they are generally linked to accepted “surrogate endpoints”. As you point out, Anavex does not seem to have any designated surrogates in any trial. (FYI it was the Accelerated Approval pathway that Biogen was able to use based on “reduction in brain amyloid as measured by PET”. Where amyloid was an accepted surrogate.

On the other hand, Blarc may quailfy for Breakthrough Therapy, meeting the requirement of “A significantly improved safety profile compared to available therapy, with evidence of similar efficacy.”

[Gernee20]

Did Lilly use pet scans/plaque removal as a surrogate?