Bourbon, these are all excellent questions and I hope MissinginAction does a better job of answering them than the cringe-worthy response he gave in the last call when he refused to even state that the P2/3 results would be released in 2H23. Here is my guess of what he'll say if he properly prepares for the meeting.
How long will it take to file an NDA for Rett after the trial is done? Will he be filed by Sept. 30?
Response: “Once we complete this final trial, we will very careful assimilate all the data into a comprehensive filing that clearly shows the overwhelming efficacy of Blarcamesine. Because of Covid, timelines have been so elongated that we don't know how long it will take the FDA to approve the NDA.”
Translation: Rather than get pinned to when AVXL decides to actually file an NDA, let's muddy the situation by alluding to the possible length of time it will take the FDA to approve the NDA.
Why were only one of the 6 stated goals for 2022 achieved?
Response: expect to hear the same answer as in the last call. “The company was very aggressive in it's stated goals and we will be much more focused in 2023.”
Translation: We over-promised and under-delivered but now we're going to be extra careful to use vague reassurances so that we aren't held accountable for our words or lack of actions.
When will we get the 50mg topline data? Why is this taking so long?
Response: “Originally this was designed as a 3 arm study, but because we are able to use precision medicine, we titrated both up and down. This gave us a very robust data set that needs to be very carefully analyzed to determine the exact parameters of our precision medicine approach. The ability to precisely define the super responders from the very good responders from the above average responders will be a crucial component of the data that we will use when we meet with the FDA to discuss the next steps in getting 2-73 approved.”
Translation: We're doing our best to massage the numbers to provide some sort of justification for our claim that we met our endpoints. We have no intention of wasting our money in filing for an NDA because we know that the FDA is going to require a phase 3 trial. We are alluding to next steps so our faithful followers will think we are very close to getting FDA approval for AD when in fact we're at least 2 years away. The “data set will be reviewed” and published is turning out to be a very successful smokescreen.
