pril 27, 2018 meeting notes 4
Data read where do we go from there”
Linda: the range of possibilities is the range one would always have. If we happen to have the happy circumstance that the range of patients are still alive, that will help us make a decision about which way we will go.....What we are trying to get to, and all of you should want tdo see, is what is the full performance of this product, what is the full performance of this technology? And we've had discussions with our scientific board, wonderful, expert opinions in the field. One of the concerns of our advisors is don't unblind too early. There are multiple examples in the past unblinded too early. The pressures to unblind are enormous, enormous. We totally understand and how much ..Shareholders want to know the information. We want to know the information. It's like waiting to open your presents on Christmas morning. The companies that have unblinded too early have really hurt themselves and their shareholders, because once you unblind, you will never know what was the full performance of your product....As an immunotherapy, what everyone wants to see is that long tail of survival. The medians are important, the early events, that's important. But in the world of immunotherapy, the big focus and the big excitement is what is that tail? On the other hand, we're not going to go forever, we're not going to wait til the last patient is gone, we're in a continuous advise mode, and we'll continue to get advise from our investigators and our scientific board. Now, I can't give you all the specifics, except to tell you that is what we want to achieve. We want to see the full picture.
We don't have a formal or fixed plan, but given the process takes 3-4 months. There's nothing to say we can't turn around and do another data collection in the fall. It's a big burden on the CRO and expenses, because it's such a big exercise, but in terms of going through this exercise about once a year, is giving us the periodic update that's been helpful for us.
Question: Linda Liau about a year ago mentioned the long tail was about 20 to 25% of the 331? Has she repeated it.
Linda: I wasn't aware that she used that number in the video.
Question: If a patient dies, are they required to tell you?
Linda: They don't tell us, they tell the CRO. And that information goes to the statisticion.
Question: It's logical you could have a speaker get up around June 1st as ASCO and give the new data?
Linda: I can't comment on that one way or another. It's technically a possibility, but I can't comment on whether that fits this year's timeframe.
Les: and you can't cherrypick it.
Linda: We haven't said that, and right now we're particularly focused on finishing the process of the publication of last year's data. We want everybody to get a full picture of what already exists.
Question: Wouldn't the pub want to have this year's data?
Les: There's a time frame: scrubbing, and studying and peer reviews. If we got it tomorrow, it wouldn't be in a publication in a couple weeks. It'd be like a year.
Linda: Once you choose to go down the path of one of these scientific publications, you're in a long-term timeframe. There's nothing unusual about where we are today with the data set or this publication. It's only 6 months since our first submission step. That's absolutely par for the course in the academic world as far as I understand. So, that's where we are.
Me: Endpoints and what the FDA would accept or not accept?
Are you in contact with the FDA and this is an adaptive clinical trial?
Linda: I don't believe the level of adaptive clinical design is applicable. Dr. Bosch is the one to talk to about trial design. (shortened)
Me, shortened: Can ou address some of the potential complications with the endpoints? Has there been problems with the whole pseudoprogression caused by our treatment? The second endpoint, the crossover, has it complicated OS. In your dealings with the FDA have you talked about it, have those two endpoints been compromised?
Linda: In the whelm of brain cancer pseudoprogression has been an extremely well-recognized phenominum. Over the recent years, not only has it been recognized as a regular phenoninum, as many as 30% of brain cancer patients have pseudoprogression, and methodoligies have developed in the field at looking at pseudoprogression. I think there's a growing understanding about the whole subject of pseudoprogression. I can't say sitting here today what the FDA's perspective on a particular aspect of pseudoprogression on any particulator trial, ours or anyone elses, would be. That's going to be down the road. I can only say I wouldn't personally describe them as complications. They are just part of the brain tumor landscape and treatment landscape. Whatever discussions we've had with the FDA or we may in the future, I wouldn't be able to discuss them publicly, it will ultimately come out in the process. All of us are going to have to wait for that stage.
Me: PFS progression, did we find people who had not really progressed but because of pseudoprogression, were put down as having progressed?
Linda: I'm not sure where you're getting that from ...us going through this or that process.
We haven't said anything publicly about PFS or progression.We haven't talked about having gone through this process or that process. We haven't spoken about the subject yet. That's something will be... that lies ahead, that we will be speaking about in the future. But not that I'm aware of, we haven't spoken about it yet. So you have to stay tuned.
