Good day everyone, welcome to today's Neurocrine Biosciences update. At this time, all participants are on a listen-only mode. Later you'll have the opportunity to ask questions during the question-and-answer session. You may register to ask a question by pressing the star one on your touchtone phone. You may withdraw yourself from the queue by pressing the pound key. Please note this call may be recorded. I'll be standing by if you should need any assistance. It is my pleasure to turn the program over to Kevin Gorman, CEO.
Thank you very much, thank you everyone for joining us this morning on a call that is a difficult one for us and disappointing. I'm joined with Eiry Roberts, our Chief Medical Officer, and Matt Abernethy, our Chief Financial Officer. Before we get started, we will be making forward-looking statements, I'd like Jane to read our safe harbor.
Certain statements made in the course of this conference call that are not historical statements may be forward-looking statements, which are subject to risks and uncertainties. Information concerning factors that could cause actual results to differ materially from those contained in or implied by the forward-looking statements is contained in the company's SEC filings, including but not limited to the company's most recent quarterly report on Form 10-Q and in today's press release. Copies may be obtained by visiting the investor relations page on the company's website. Any forward-looking statements are made only as of today's date, we disclaim any obligation to update these forward-looking statements. Kevin?
Thank you very much. Suffice it to say that we're very disappointed with the outcome from T-Force GOLD. You all know that I've been very confident when talking about my expectations for T-Force GOLD. That confidence was driven by a number of factors. First, that the mechanism as we know it that's involved in Tourette syndrome is a dysregulation of the dopaminergic system. Second, that postsynaptic D2 antagonists, the antipsychotics, have a very nice effect in the disease. Third, that our earlier clinical trials, particularly T-Force GREEN, gave us an indication that by reaching higher blood levels in the kids with Tourette, that our drug had given us a good signal on efficacy.
Fourth, and probably even most of all, is that the robust and often profound efficacy that we see with INGREZZA in our TD trials and in the marketplace, was one that led us to believe that we were going to see a robust and potentially profound efficacy in these kids. At the end of the day, I think what you have to do when you have a trial that was as well designed and conducted as this one, and the data hangs together real well, you just must accept the data readout that you have. Now granted, this is very top-line data. This is a phase II-B study, there's a lot more data for us to go through. We're only going to be able to comment on just really the very top-line data.
One has to accept the fact that the drug just did not show a large effect on the primary endpoint of the Yale Global scale. We did see a consistent treatment effect, but it was smaller than what we expected. Eiry's going to go over this now in a little more detail for you. She has some opening statements, I'll leave it to Eiry right now.
Thanks, Kevin, and thanks to the participants joining our call today. First I'd like to take a moment to thank all of the investigators, patients, and caregivers who participated in the T-Force GOLD trial. With their participation and commitment, we were able to complete a study that provided further insight into the potential efficacy of valbenazine in children and adolescents with Tourette syndrome. The investigators did a really good job identifying appropriate subjects for the trial and assessing the Yale Global Tic Severity scores for patients, which is evidence of a well-designed and well-conducted study. The placebo effect in the study was as we anticipated, as Kevin mentioned, unfortunately the treatment effect seen with valbenazine was less than we expected, which is what led to us missing the primary endpoint for the study.
I can say that the types of treatment-emergent adverse events observed in the trial were consistent with those seen in other valbenazine studies. We're very disappointed with the outcome of this study, in particular, we're disappointed for the children and adolescents suffering with Tourette syndrome, given their great need for new treatment options. Today we're sharing the top-line data only from the study. We don't have all of the data analyzed yet since we just blocked the database very recently, we will need to complete those analyses in order to better understand this lack of treatment effect. After we review the data, we will determine the next steps for valbenazine in Tourette syndrome. Thank you again for your time today, I'll now turn it back to Kevin.
Just one last statement that I would like to make before we open it up to your questions, is that we do have a lot of data to go through before we look at next steps here with valbenazine in Tourette syndrome. We're going to need some time to go through that. I really want to be straightforward. We did not see the robust effect that we were looking for in the primary endpoint. The simplest explanation is that the drug does not show that kind of effect in Tourette syndrome. For those of you who have Tourette in your model, which some of you do and some of you don't, my message to you would to be remove Tourette from the model going forward.
We have a lot of exciting things going on here in Neurocrine, I believe that we have a robust company obviously going forward with TD and with the other drugs in our pipeline. With that, I'd like to open it up for questions.
At this time, if you would like to ask a question, press star one now on your touch-tone telephone. To withdraw yourself from the queue, you may press the pound key. We'll take our first question from Geoff Meacham of Barclays. Your line is open.
Hi, guys. I just had a clarification question and then a forward-looking one. Was the failure of T-Force GOLD the aggregate of all doses versus placebo? In other words, did you see doses that did have an effect, and some that did not? i.e., is it similar to the outcome with T-Force GREEN?
Thanks, Geoff. This is Eiry here. The primary analysis of the study is the total valbenazine group, compared to placebo. As we mentioned, we failed to achieve the primary endpoint on that. As part of the deeper data analysis that we'll do moving forward, we'll obviously be looking at both the relationship of exposure to tic control and dose. At this time, we failed to meet the primary endpoint on the combined dosing group.
Geoff, before you go on, one thing I'd like to add is that, in this study design, we did push the dose as far as we believed we could. So dose and exposure, we probably don't think are a factor here. We really do think that we got the exposures. Even though we don't know that for certain, we have sampling PK that tells us that. We're still waiting for all the PK to come in so we can do a full exposure response model on this big data set. We did push the dose in this study.
Okay. Then just as a follow-up, for the ongoing T-Force PLATINUM study, what implications do you have for that? Do you anticipate maybe changing the size or the power calculation or adding a different dose to that? Should we just assume that study may be winding down on the back of today's result?
It's too early to make any definitive comments about that. We really need to fully understand the data from this study in more detail. Then, as Kevin mentioned, we'll be doing that full analysis over the coming weeks, and then working with our investigators and participants in the remainder of the program to understand the next steps.
Okay. Thanks so much.
Thank you, Geoff.
Thank you. We'll take our next question from Paul Matteis of Stifel.
Great. Thanks so much for taking the questions. Just two. I appreciate you being so transparent this morning, at least on being pretty conservative and realistic going forward. I guess to that point, while you have a lot more data to analyze, can you help us understand what would Neurocrine need to see in the full analyses and the secondary endpoints to think that Tourette's still has a shot and is still worth investing in? What would be a good outcome of your additional research?
Paul, I'm just going to say that I think one of the keys that we have to do, although I have said here today that we have enough sampling PK that we feel we had adequate exposure, you really do need to run the full exposure response model. Eiry's nodding her head here, too. That's the one piece of data, and unfortunately, it's the one that takes actually quite a bit of time to go through. That's really the one piece of data that we would look for here. Eiry, you too.
I think that's absolutely right, Kevin. I would reiterate though, what Kevin said earlier. This was a very well-designed study, well-conducted study, and in this study, we did push the dose. Using this study design, I think we clearly would need to learn from that if there were any additional learnings from the exposure response model.
Paul, I would say that we have a good group of people here from research all the way through, basic research all the way through to clinical. We just need a little more time to live with this. We have a bunch of hypotheses, theories, and everything. You always learn from your data. In the past, I've said from phase II-A, you learn from them in order to design the phase II-B and ultimate phase III. That's not what I'm saying here this morning. I'm saying that there's some biology here that we don't fully appreciate, and this is data. Just like any experiment, we're going to learn from this data moving forward. Maybe both for Tourette, but certainly also for the many other indications that we look at as being areas that we want to explore, particularly with our VMAT2 backup compounds.
It's not trying to always look on the bright side, but we've got data now. We've got a really good data set here, and it's going to talk to us.
Okay. No, I definitely appreciate that. Thanks. On the other ongoing study, I believe it's a randomized withdrawal study, which in neuropsych, those designs are usually used as confirmatory studies of a positive signal. Is that the right way to think about the way you were contemplating that design, that it was a good design to follow up a positive result? To that point, is that study a well-designed study that if it did somehow work, it could actually lead to a possible approval?
I think your interpretation of the role of randomized withdrawal studies is correct, Paul. We believe that is a well-designed study and is currently ongoing. As I mentioned earlier, we need to learn fully from this data set to understand the next steps for both PLATINUM and for the remainder of our Tourette syndrome program. It's just too early, given where we are with the data today, to make any definitive statements about the remainder of the program.
Paul, I would add that in the event, and I think that you would have to put a low probability of success on this for PLATINUM. In the event that it was positive, that certainly wouldn't be sufficient for an sNDA filing, we don't believe. There would have to be another, basically, double-blind, placebo-controlled, randomized study that would need to be done. Currently, we don't have any insights on how we would design a better study than what T-Force GOLD was.
Okay. All right. Thank you, Kevin and Eiry. Appreciate it.
Thank you.
We'll take our next question from Anupam Rama of JP Morgan.
Hey, guys. Thanks so much for taking the question. Kevin and Eiry, I'm sorry if I missed this in the opening comments. Were there any imbalances in the baseline characteristics in T-Force GOLD worth noting? Thanks so much.
No. This was a very well-designed, well-controlled study. There were no significant imbalances on any of the parameters, really, in the baseline characteristics. Importantly, the placebo response was very well controlled. I know people had questions about the placebo response potentially for this study, given that it was a dose optimization study. We were very pleased with the outcome from the perspective of placebo. It was just very unfortunate that the magnitude of the treatment effect that we saw with valbenazine, although present and present consistently, was smaller than we had anticipated.
I would just add again, the things that you would normally look at that maybe you could hang why you didn't get the result that you anticipated. Eiry talked about placebo effect or dropouts or more variability than what you see. There's none of that. This was a very clean study. The study gave us a very direct answer to the hypothesis that we were testing in here.
Thanks so much for taking the question.
Thank you. We'll move next to Jay Olson of Oppenheimer. Your line is open.
Well, hey, guys. Thanks for taking the question. I know you've commented on the primary endpoint for T-Force GOLD. Were there any subcategories of the Total Tic Score, such as motor or phonic tics, that showed greater separation from placebo?
We're obviously still analyzing the full data set. We just have top-line data right now. What I can say is that the effect size for valbenazine was pretty consistent across the sub-elements of the Yale Global Tic Score.
Okay. Thank you. Maybe just as a follow-up, I know you've mentioned previously interest in pursuing new indications, either for valbenazine or the next gen VMAT2 inhibitor in certain undisclosed movement disorders. Does the lack of efficacy in Tourette syndrome change your views on the potential for additional studies in those other movement disorders?
It's a good question. I'll take first crack at it. I think we're still very enthusiastic about the mechanism of modulating presynaptic levels of dopamine. We have a number of different indications that we're looking at to take either INGREZZA or the backup compounds into. What this does, as I said previously, it adds what I think is going to be a very good and useful piece of information for us after we've had the chance to go through all the data, reflect on it. I think it's going to be very useful in helping us prioritize what those indications are. I still very much believe that this is an important mechanism in CNS and in movement disorders. Eiry?
No, I would agree completely. I think we know that valbenazine can produce a profound efficacy for patients with tardive dyskinesia, that's certainly a validation of this mechanism in that patient population. As Kevin mentioned, we have a broad number of different indications that we're interested in, we will learn from this experience to prioritize those moving forward.
Great. Thanks for taking the question.
Operator, we have time for one more question. Very well. We will take that question from Alan Carr of Needham & Company.
Hi, thanks for taking my questions. Kevin, you sound a little pessimistic around PLATINUM outcome, I'm wondering if you could remind us of the design of that one and maybe powering around that. Is there any chance that the size of it also, any chance that the effect size might, that the outcome of that might still be favorable? Also, if in the end you do drop Tourette syndrome, what sort of impact would that have on your overall business development strategy, if any? Thanks.
Yeah. I will let Eiry handle the first question. I will take the second question.
T-Force PLATINUM design is such that this is a randomized withdrawal study. The patients receive open label treatment to an optimized dose of valbenazine for the first six weeks of the study. That treatment is continued till week 12, then patients who are responding are randomized to either placebo or to continue active treatment with valbenazine. This study answers a different question from a parallel randomized control study. As such, I think it is an independent study in terms of its likely outcome. Given what Kevin said earlier, the important factor is that this would be a supportive study of any submission, rather than a standalone single study for submission. We still need to learn from the current data to determine if there are any next steps in this indication, and we'll be doing that over the coming weeks.
Alan, I'm going to use the last part of your question to actually give my summary remarks here before we sign off. I don't want to minimize the disappointment we have here and also the fact that we're very surprised about the outcome that we had here. Honestly, when it comes to the company itself, it is a setback, but it is a far bigger setback for the patients suffering from Tourette's. That's why we're going to be very thoughtful in our approach to valbenazine and other therapies that we're going to explore in Tourette. This is a very high-end medical need area, and this is where the real setback is for the patients. For the company, we have a number of very strong drivers of growth taking us into the future. That future still looks very, very good for Neurocrine.
We're fortunate that we're not a one-product company, and we're fortunate that the compounds we've discovered and developed, commercialized on our own, commercialized with our partner, AbbVie, they have a lot of runway that they're going through. The drugs are very good. They bring tremendous benefit in tardive dyskinesia and endometriosis. I would say soon that you'll see the filing that AbbVie will do in uterine fibroids. Those lifts are going to be there, and they're going to be there for a number of years going on. We also have opicapone that we're going to be filing in Q2, and then we have CAH, which we're going to look forward to sharing the data with you later in Q1, as well as continuing to refresh our pipeline and add to it through internal research with a number of exciting programs.
We had two that hit this year, and we look for more next year. On the business development side, we are more active than what we've ever been before. We place a high emphasis on this. There's no need for Neurocrine to panic here in any situation. It doesn't change our mission, it doesn't change our strategy, and we're going to keep moving forward. That mission is to bring hope to patients, and that's exactly what we're going to do through all facets of the company. With that, I thank you all for your questions, and I'll be signing off now. Thank you.
This does conclude today's Neurocrine Biosciences update. You may now disconnect your lines. Everyone, have a great day.