Thanks for adding clarification and context to the process.
The transition from RCT to OLE is an important important step of which I don't know the details, but I do know the parents of the patients know the facts of the RWE and if the drug created the changes.
My guesses are that Rett will go to regular way approval with no further trials after the current P3, and a NDA filled in early 2024.
AD on the other hand has a few potential paths depending on the strength of the current data from the P2/3 and acceptance of new biomarkers. From the last 10q, to me this sounds like Anavex will be seeking Accelerated Approval via this new biomarker. "Alzheimer’s disease: Full data ANAVEX®2-73-AD-004, including newly available preliminary results of surrogate biomarkers of pivotal Phase 2b/3 clinical trial. The Company intends to discuss these findings with regulatory authorities in the context of the ongoing clinical development of ANAVEX®2-73 in this indication"
For the PD/PDD indications, the trials are finalizing their designs and these trials will also involve newly discovered biomarkers and methods announced this year by the MJFF.
And I wanted to comment on this too...
All of this also ethically linked to P4 commercial post Accelerated Approvals, where really patients are participating in a clinical trial, but must pay (through insurance and/or privately). This versus running e.g. P3 confirmatory trial where patients do not pay, but may need to wait for the OLE to know they actually get drug/treatment.
Regarding patient costs in a P4 scenario for AD, it should be noted the cost to the patient for blarcamesine will cost a fraction of the costs of the anti-amyloid drugs allowing for robust participation regardless of costs.
