Replies to post #455298 on Anavex Life Sciences Corp (AVXL)

[Joseph_K]

We're pretty much in sync on all this. I agree that the new (draft) guidance indicates a path for a later trial relying on biomarker endpoint(s) in Stage 1 subjects with no cognitive or functional endpoints at all. I read this document also to be aimed squarely at trials for Stage 2 and 3 subjects, which will greatly help an NDA based on the P2b/3 since it eliminates the need to satisfy any functional endpoints.

I entirely agree that the draft is "a call to the medical establishment to find and validate new AD biomarkers and [that] it indicated a willingness of the FDA to consider new AD biomarkers."

Maybe the blood-based and CSF measurements used by Anavex will get validated. In any event, this draft seems to give a boost to the FDA at least giving them more weight as surrogate biomarkers. There are journal articles that ought to incline the FDA to validate brain-region volumes, e.g., https://link.springer.com/article/10.1007/s13760-023-02235-9, which concludes,

Volumetric differences of hippocampus and amygdala were at a finer subfields scale, and the volumes of right basal nucleus, left parasubiculum, left medial nucleus, left GC-ML-DG, left hippocampal fissure, and right fimbria can be employed as neuroimaging biomarkers to assist the clinical diagnosis of MCI and AD.