RE:RE:RE:RE:RE:RE:Best Case ScenarioAll credit goes to Christian, who has impressed me in many ways over the years but most especially with his willingness to help people like you and your wife. Hopefully, some worthwhile treatment option will open up soon for your wife, assuming the Lord does not pull something off to heal her before that. We continue to pray daily for her!
Yours is the second report I have heard about the "enrolling one patient at a time" approach TH is apparently using. I am not sure that tells us much though. This trial was never going to have a lot of patients anyway and they could have added one patient a week since the company last shared they had only 5 patients through mid-October and that would only add up to an additional 12 patients or so. So, there just are not a lot of spots open in the first place. Also, it is entirely possible the trial has been altered in some way based on the early data, although that would likely mean something really noteworthy had been seen early on. Bottom line - we have no clue about where the trial is right now other than it must be somewhere near the end. The last we knew, theyhad stepped up to the 420 mg level and had a soewhat bad response, but not enough to stop the trial or even stop dosing people at the 420 mg level. But that was a long time ago. As we have speculated previously, TH could now be testing at a higher level or at a lower level in their search for the MTD. I suppose the upside scenario is they are now the roughly 3x level of normal docetaxel and the downside scenario is they encountered more problems at the 2x dosage level, hen moved down to the 1.5x dosage level and encountered more problems than they did with the one patient when the dosage level was still ascending.
The company remaining silent for so long does not seem to be an optimal capital markets strategy. I expect to see a new corporate presentation by at least Monday morning as they most likely will use one in their meetings with large shareholders early next week. If there is not much new info in this version, that will be pretty disappointing.
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.