My CC questions/observations:
Wonder what the inclusion/exclusion criteria are and whether they will be revealed at CTAD or at trial commencement. (No mention of the more pertinent biological factors either.) Otherwise this whole exercise still sounds very conceptual and an incomplete exercise after a year of waiting.
Will the same inc/exc criteria be applied for all trials?
Impressive to see them mucking around with 80! different explanatory variables and sussing out 83! rules. Can't believe they said they have "additional data to be incorporated" after all this time and all before start of trials.
Seems like dose response is likely to be the big driver around which KEM is modeling. I would think they have exercised any ability to escalate dosage going into Part C of the trial. There's a bunch in the bottom left quadrant that stand to move diagonally up to the top right and make the overall scatterplot a successful story to tell.
Am I wildly off in thinking, they have just 2 data-points/patients to try and figure out what biological factors may have allowed for an MMSE improvement at a low dose?
Wonder why they chose MMSE slope from baseline as y-axis of scatterplot rather than MMSE delta from baseline (like they did for ADCS-ADL.)
Have INDs been filed/accepted with the FDA at this point for Rett & Fragile-X? I'm guessing they have been filed unannounced as they insist on starting the trials this year.
Overall, I'm optimistic - this may be simplistic thinking but if you were to draw a diagonal on the scatterplot, running from the bottom-left corner to the top-right corner, most of the points on the scatterplot reside on the top-left side of that diagonal. To me they are the super-responders and ones that offer promise. Just 4 patients exist clearly to the bottom-right of that diagonal that may be considered as unlikely to see any benefit.