Thanks TP! I posted this as part of a larger overv
Post# of 72440
Thanks TP! I posted this as part of a larger overview 6 months ago. Some is still relevant today. Very exciting to see this trial develop!
small "s" (safety) indications are primary, and we don't even have the first pk data, only very early indications of low "t" (toxicity). After enough small s occurrences, escalating doses will achieve and quantify MTD (maximum tolerable dose). Other markers are likely known with the first pk analysis, but as yet undisclosed. One would look for these first labs to show "something" related to "e" (efficacy) even before MTD is achieved. In the absence of data, continued cohorts and dose escalation are good signs.
Clearly, statistical significance (robust N) becomes crucial to a big "E" disclosure, but many will be looking carefully at even the smallest cohort sizes, and regardless of N (number of subjects) whatever the first two pk labs samples show will carry some significant implications (of possible small "e" evidence).
What level of data from labs/scans will be disclosed and when? We don't know... this is up to DF (not publications/journals). However, once evidence from cumulative trials positively correlates activated p23 with MRI scans showing decreased tumor size-- then we have arrived, i.e. efficacy with a capital E.
As an investor, if I wait for such positive efficacy evidence to be disclosed, I will no doubt have missed a significant SP move based on a series of prior small "e' evidence. But here's the caveat: one or two, or even a series of small positive markers (e) does not necessarily add up to a Big E. Hence, I fear ongoing vulnerability to whiplash, head-fakes and churn as sequential data points are released.
In sum: small "s" is a necessary but not a sufficient condition for "e"... but glimmers of "e" should be observed even before bigger S arrives with MTD. Clearly, the more signs of little s's and e's the better. I have my magnifying glass out, but its more difficult being many time zones away.
Best to all longs here.