Anavex Life Sciences Corp (AVXL)

[Doc328]

From the publication you referenced

In AD trials, target engagement biomarkers demonstrate whether a pharmacologic agent has engaged the specific target of therapy. Pharmacodynamic biomarkers are also used as trial outcomes to determine whether an agent has a disease-modifying impact on AD. The lowering of amyloid plaque burden, as shown on amyloid PET, is regarded by the US FDA as a pharmacodynamic biomarker likely to predict a cognitive outcome. Plaque reduction was used in the accelerated approval of aducanumab [33]. Amyloid and tau biomarkers may function as either target engagement biomarkers, showing that the agent has directly or indirectly impacted Aß- or tau-related processes, or as biomarkers providing evidence in support of disease modification. Their interpretation depends on the COU defined for the biomarker prior to the initiation of the trial.

The FDA has draft guidance on how to get a biomarker qualified for clinical trials. A don't see a final guidance so there could eventually be some changes. Amyloid PET was not done so that biomarker is out. We know CSF was collected (though unclear if on every patient or a smaller group). I think either CSF Aß42/ptau or Aß40/Aß40 rations could potentially be used as a biomarker as there is fairly good correlation to amyloid PET. Additionally, the lecanemab trial had some correlative CSF data with efficacy. Similar serum tests are being developed but are less correlative with amyloid PET at this time and are not ready for prime time yet. I don't know if Anavex or other companies have done the process to get the CSF biomarkers qualified.