RE:RE:RE:RE:RE:RE:RE:RE:RE:Best Case ScenarioI believe you have been told that, but if they enroll only one patient per month at a given dose, it could be much longer for them to find the MTD. Maybe it's one patient per month per center. I don't know, but only one patient per month overall makes no sense et this stage, even for results on MTD in March.
juniper88 wrote: I only wrote that they are enrolling 1 patient at a time. Nothing about a dosage. Specifically I was told a patient had just been enrolled and that the next one would be in 1 month, but because of a wait list there would be no guarantee that my wife would be picked from the pool. I was also told that they did not know how patients were selected as "this sponsor is not very transparent". Maybe my wife can wait a month but there is no guarantee and my wife does need treatment. One site has offered a different trial which we will try to have her go on. If it doesn't work out then maybe we can try again when Thera starts the 1b part.
jfm1330 wrote: I have a hard time to believe that they are still enrolling one patient per dose level, maybe they are enrolling them sequentially at the same dose, but if you look at their diagram in the Credit Suisse presentation, they should be well at doses requiring many patients. I am not putting in doubt what juniper88 said he was told, but if they are still at one patient per dose level, we will have the results of this thing next summer.
longterm56 wrote: Wow, Juniper88, that really brings some reality to this board, thanks for sharing. It also reminds me that this is much more than an investment ... it may be a lifesaving drug to many people. I hope something changes soon that would allow your wife access to a trial.
A question to the board ... if they are only accepting one patient at a time, doesn't this mean they have not yet reached a dosage that has caused problems? Once they do, they back down and give a lower dose to several ... correct?
-LT
juniper88 wrote: As most on this board probably know my wife is battling Ovarian cancer. Through the help of Rusty who connected me to Christian Marsolais my wife was referred to 2 different clinical sites. Unfortunately, my will not be able to get onto the trial at this time because there is a waiting list to get on, so I was told. I was also told that they are still enrolling 1 patient at a time. It seems to me, and I'm just speculating, that patients are on a waiting list because doctors have seen some efficacy.
Wino115 wrote: Yes, I borrowed this from the science guys here -- Recommended Phase 2 Dose (RP2D), although I think this is a term they use in trials. In our case, it will also be for Phase 1b, although they may play around with some other dosage schedules around size and timing if they learned something over 1a that's intriguing.
juniper88 wrote: The dose to be used in the Phase II trial.
SPCEO1 wrote: I am likely going to say "Oh, of course!" but what is RP2D?
Wino115 wrote: Somewhat implied in your Best Case, but I would add a few signals around that final group of 6 patients that took the MTD to make it the RP2D:
--- All six are still on the drug and longest on the trial is at 4 months of progression free survival and ongoing.
--- Of those 6, at least 2 of 6 have had an objective response of some kind (recall, ORRs of 30% are considered huge in this class of patient. ORRs would be partial most likely, which is at least a 30% reduction in tumor).
SPCEO1 wrote: Here is how I would define it:
1.) Meaningful tumor regression in all sortilin overexpressing patients who survived long enough to get meaningful measurements in the phase 1a trial.
2.) MTD established at roughly three times the normal docetaxel dosage
3.) Minimal safety issues in the phase 1a. No worrying issues in the trial at all.
4.) FDA grants TH-1902 Breakthrough Therapy status
5.) US cancer analysts rush to pick up coverage of TH
6.) TH holds another cancer KOL presentation to update the market on the latest findings with Dr Rothenberg as one of the main speakers. The presentation includes not only more detail on the phase 1a but also addresses a now expanded phase 1b trial that will look at as many as 8 dfferent sortilin overexpressing tumor types and will also delve into where the Sort1+ platform goes from this initial start.
7.) These developments are all completed by the end of March
What am I missing?
Obviously, the reality of the situation is likely to be much more complicated. But sketching out a Best Case Scenario can help us judge how good the reality is when it finally becomes reality, which hopefully will be quite soon. Since I am an optimist, I will leave the Worst Case Scenario to anyone else who might want to out it together.