RE:RE:RE:RE:RE:RE:RE:RE:RE:RE:RE:RE:At least give us news about no news ... We are definitely in bum twitching territory. It took a long(er) time to get to the higher doses. We are definitely in that period now. You have all the random factors like Sortilin expression in play to enroll a potential responder, you need that sick individual to stay healthy long enough to develop a response and you need that technical 2/3 month period of waiting for all the scans in order to get to a confirmed response. It would make sense to me that they have info that points to efficacy now and that it could be reportable in January. That would fit with them doing the investor circuit late Jan.
They have to report on 1a. It should talk about dose, pK and safety. It will either have an efficacy signal or not. Either way it will likely mean they move to 1b regardless.
SPCEO1 wrote: Sorry, my bad - you had made this clear earlier and I just forgot.
So, in the preclinical work, TH saw tumor regression on low doses if I recall correctly. While we know the first patient passed away, we know nothing of the other early patients (4 of them). They should have had many cycles now, if they were fortunate enough to survive, and TH should have seen some sign of efficacy by now assuming half of them had sortilin over-expressing tumors. Are any of you concerned that they have not reported any preliminary signs of efficacy yet based on those assumptions and what we know about what the preclinical work suggested? Or do you think if TH saw such evidence they would wait until they thought best to share that info?
juniper88 wrote: Treatment is given every 3 weeks until progression or ifvthere is too much toxicity. Up to 18 months. Progression is determined by CT Scan.
jfm1330 wrote: I did not see any specific info on that, but my understanding is that they will continue to treat a patient with the drug as long as there is a percieved benefit. I think that ethically it is the way it must be done.
SPCEO1 wrote: Actually, I think we agree. You restated what I was saying as best I can tell. In phase 1a, do we know how many cycles they are giving the patients? I am not sure depsite having listened to everything very closely over time. But maybe I still missed it and they are doing at least three cycles. At times I thought that but I am no longer sure they are doing more than one or two. It would be nice to have that cleared up.
jfm1330 wrote: I don't agree. Preliminary indication of efficacy is after the first cycle of treatment. Efficacy, or confirmed efficacy, is after several cycles of treatment. The time factor is important when assessing efficacy. You need to see it over time. After a single treatment, the tumor shrinkage can be too small to be sure it's real efficacy. Radiologists will tell you that from scan to scan, some tumors can move, and just the angle of it can make it look smaller or bigger within a 10% range. That's why a partial response is considered to be 30% size reduction or more. You are unlikely to have 30% shrinkage after a single cycle of treatment.
- Partial response (PR): At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters.
- https://www.verywellhealth.com/partial-response-pr-2252162
SPCEO1 wrote: I
One thing I have been thinking is that we need to listen to their words closely. When they say "preliminary indications of efficacy" I think they are talking about phase 1a and when they just say "efficacy", I think they are talking about phase 1b.