RE:RE:RE:Finally listened to call Very helpful and I hope those moaning about incompetent management and kicking the can and all that stupid stuff can use your posts to educate themselves on what an early stage Trial entails especially with a disruptive technology never tested in humans before this Phase 1 triial
I think it should be repeated also that based on publically available data in Phase 1 there is absolutely NO guarantee of success!!However based on comments made by Paul and Marsolais it would be surprising that this ends up being a nothingburger!! But that does not mean either that it will be a Big Mac or a Whopper
qwerty22 wrote:
The other thought that struck me recently which we've ignored given the focus on efficacy is that we are still very early in the process for safety. Only 13 patients in 1a recieved 300/420 mg doses. We really should be considering that the breaks on enrolment aren't off yet from a safety perspective. It's noticeable that they are timelining 10 months for enrolment and there are 10 patients in each cancer. It's possible the fda has set a max enrolment rate, say one patient for each cancer completing a cycle before the next can be enrolled. This would protect greater numbers of patients from being exposed to unknown risks.
If there is a safety component to the enrolment rate (which I imagine there should be at this very early stage) then probably enrolment is neither fast or slow but proceeding steadily as they indicated yesterday. It would mean that no one individual cancer type could race ahead and get across the finishing line faster. It might explain why for now they are comfortable with just 6 centres enrolling. My original thought, which would be wrong if this is true, was that if they could aggressively recruit one particular cancer type that could get us to a go/no-go signal earlier but if we are still looking at very controlled enrolment for safety reasons then that's not possible. A good reason why they still aren't reporting efficacy yet. It's possible some efficacy signals are popping up in different cancer but nothing has got across the finishing line. That might put them at about 5 enrolled patients in each cancer type atm. Remember five enrolled steadily in each cancer would break down as say 2 with 2 scans, 2 with 1 scan and 1 yet to be scanned. You wouldnt expect that to get you across the line for a go signal.
It's possible they do cross the line from an efficacy point of view before the end of Q1/23. If the drug is good it probably should happen in the next 90 days as you say. Whether from a safety perspective they are still tied to a steady enrolment rate due to safety after that IDK, it would make sense that they are.
I'm imagining those centres waiting in the wings in the US, Europe and Canada are for when the breaks are eased a little more on enrolment. It would be useful if anybody has some solid info on how fda safety concerns is likely to be controlling enrolment rates at this point. That might be the elephant in the room we haven't considered. BTW I'm not saying there is a particular safety issue with th1902 but this is how the fda always proceeds.
Inthink if enrolment is steady and controlled and across the range of cancer types then basically we are just too early for any one cancer to have reached the go signal for expansion. And if that's what they are waiting for for public announcements then we were all too aggressive in our expectations for this Q. The go signal is not so high that we will be waiting forever (although it might seem like that) but now is just too early.
PWIB123 wrote: You and Scarlett picked up on the ssame thing I did after I listened to the call myself. It was around the 35 min mark where the lady filling in for Ed Nash started asking questions about the basket trial and when we could expect information on data to be shared. I thought Christian's comments were extremely positive and provided insight into what his true thoughts were and the best and worse case timelines. He said he believes we will see signs of eficacy in some (plural) of those tumor types. He reiterated they are looking for 2 responses or more in at least 1 tumor type. He said they expect to be able to announce signs of efficacy by end of quarter, and then he backed up and said at least by end of first quarter next year, wihtin next 90 days. Christian was as direct and confident as anyone could expect. I thought this was hugely positive!
I also picked up on Paul making a statement that they were seeing a shift in the industry toward administration of Trogarzo in pharmacies rather than clinics and that the IMF push would benefit there.
I thought the team expressed additional confidence in Egrifta being a viable solution in spite of not being an easy to administer pill.
All in all, while not overly energetic, I thought the call was mostly postured with positive overtones. I still wanted more, and Paul and the board have got to accept the fact that they have to communicate differently in the future, but I didn't walk away thinking TH-1902 was a dud and the program would be scrapped. It's just a long-time in the making of what will be an overnight success story. Hopefully they build on it.
qwerty22 wrote: I was going on people reaction here before.
I thought the call was fine apart from no ongoing trial update.
There was one nugget when Christian seems to suggest that all the main cancers in the trial had over 85% Sortilin expression. He seemed to suggest that was what the up and coming microarray poster would say. If that's true then that is great, in fact fantastic. The worry that they are enrolling significant SORT- patients is really off the table now. It also makes screening for Sortilin less of a priority for now. That's great news.
The other good point for me is Christian clear said the protocol going forward is see two responders from upto 10 patients, expand to 25 and then move forward to the next phase if they have the data to support that.
They obviously made a prior decision not to discuss the ongoing trial. My take is it is hitting that 2 responders from 10 that triggers the next update especially if that triggers a quick expansion to 25. So this is what we should be expecting. Should that have happened by now? I don't think so necessarily. If no one cancer type has aggressively enrolled then they could have a mish mash of results where none of those quite reach that go signal benchmark. I guess we have to accept they aren't going to dribble out results on every responder leading up to that point.
I can sort of even understand why they don't want to talk about enrolment rates. If you say it's slow, then that's a negative. If you say it's fast and aren't releasing details on responders then it looks like the drug is a dud.
I think the one conclusion we can say is the probably haven't hit the go signal yet. I'm not too bothered (yet) about the trial being a dud. I'm putting this down to our expectations being too high about was is possible yet. You sort of have to admire their discipline is saying nothing about the ongoing trial. It sucks but they clearly decided to say nothing and they did it well.
On other things. I thought they contextualized the NASH situation the way I understand it. I also thought they inspired a bit more hope that the Trogarzo management plan could yield some potential benefits (eventually).