Replies to post #316375 on Anavex Life Sciences Corp (AVXL)

[Steady_T]

The PR described this as the biomarker...

Data showed that ANAVEX2-73 treatment resulted in a significant increase in the expression of the SIGMAR1 mRNA biomarker that significantly correlated with improvements in the two primary clinical efficacy endpoints - Rett Syndrome Behaviour Questionnaire (RSBQ) and CGI-I (assessment scale for determining the effects of mental health treatment)

So the data showed the biomarker improvement AND showed how the biomarker correlated with clinical improvements. That is something that Adu** did not do.

Seems to me that 2-73 has a much stronger case for approval.

[Steady_T]

I did a little deeper research on Accelerated Approval and found this.

Apparently a surrogate endpoint is not a requirement but is one of two possible criteria. The other one is a clinical endpoint as described below.

Looks like 2-73 achieves them both. Bolding is mine.

? A drug that treats a serious
condition AND generally
provides a meaningful
advantage over available
therapies AND
demonstrates an effect on a
surrogate endpoint that is
reasonably likely to predict
clinical benefit or on a
clinical endpoint that can be
measured earlier than
irreversible morbidity or
mortality (IMM) that is
reasonably likely to predict
an effect on IMM or other
clinical benefit (i.e., an
intermediate clinical
endpoint)

Accelerated Approval would require an P4 trial to be set up and run. Regular approval process would not require a P4, just the usual monitoring program for SAE and AE.

Priority Review can be requested at NDA submission time. Priority Reivew designation results in an FDA decision on the NDA in 6 months as opposed to the usual 10 months.