Good day, everyone, and welcome to today's Neurocrine Biosciences Update. At this time, 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 and 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. So 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, and 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 School. 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, of the dopaminergic system. Second that, postsynaptic D2 antagonist, the anti psychotics, have a very nice effect in the, end of disease. And third, that are our earlier clinical trials, particularly T Forcegreen gave us an indication that by reaching higher blood levels, in the kids with Tourette, that our drug, had, had given us a good signal on efficacy, and then 4th.
And, 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 you just must accept the data readout that you that you have. Now granted, this is very top line data. This is a phase 2b study.
So there's a lot more data for us to go through. So we're only going to be able to comment on just really the, the, the very top line data. But one has to accept the fact that, the drug just did not show, a large, effect on the primary endpoint video global scale. We did see a consistent treatment effect, but it was smaller than what we expected. Airy is going to go over this now in a little more detail for you.
She has some opening statements. So I'll leave it to Airy 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 commitments, 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 tick 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, but as Kevin mentioned, unfortunately, the treatment effect seems that valbenazine was less than we expected.
Which is what led to us missing the primary endpoint for the study. Also, 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, and in particular, we're disappointed for the children adolescent suffering with Tourette Syndrome, given their great needs 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, and we will need to complete those analyses in order to better found this lack of treatment effect. After we review the data, we will then determine the next steps for valbenazine and Tourette Syndrome.
Thank you again for your time today. And I'll now turn it back
to Kevin. Yeah. And just one 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 with Valbenazine, in Tourette Syndrome. And so, we're gonna need some time to go through that but I really want to be straightforward.
We did not see, the robust effect that we were looking for on the primary endpoint. And the simplest explanation is is that the drug, does not show, that kind of effect in Tourette's syndrome first. So for those of you, who have Tourette in your model, which some of you do and some of you don't, might message you to be removed Tourette from the model going forward. We have a lot of exciting things going on here in Neurocrine, and that I believe that, we have a robust company obviously going forward, with TD and with the other drugs in our pipeline. So with that, I'd like to
press star 1 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 forward looking one. Was the failure of T4S Goal 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 Ira here. The primary analysis of the study is the total valbenazine group, compared to Placebo. And, 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, tick control and dose But at this time, we failed to meet the, the primary endpoint on the combined, dosing
And, Jeff, before you go on, one one thing I'd like to add is that, in this study design, we did push the dose as far as we believe we could. And and So dose and exposure, we probably don't think are a factor here. We, 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 an exposure exposure response model on this big data set, but we did push the dose in the study.
Okay. And then just as a follow-up for the for the ongoing platinum study, what implications does do you have for that? Do you anticipate, maybe changing the size or the power calculation or adding a different dose to that? Or should we just assume that 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, this study in more detail. And then as Kevin mentioned, we'll be doing that full analysis over the coming weeks and then working with our investigators and pence in the remainder of the program to understand the next steps.
Thank you. We'll take our next question from Paul Matteis of Stifel.
Great. Thanks so much for taking the questions. Just too. 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 still has a shot and it's still worth investing in?
What would be a kind of a good outcome of your additional research?
Yeah, Paul. I'm just going to say that I think one of the keys that we have to do, although I have, 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 ocean response model. So, iris 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.
But that's that's really the one piece of data that that we would look for here. Irene,
I think that's absolutely right, Kevin. And, I would reiterate, though, what Kevin said earlier, this was a very well designed study, well conducted study in this study, we did push the dose. And so using, this study design, I think it's, we we clearly would need to learn from that if there were any additional learnings from the exposure response model.
Yeah. And and Paul, I would say that, you know, we have a good group of people here from research all the way through, basically, since all the way through to, to clinical. And, we just need a little more time to live with this. We have a, we have a bunch of high hypothesis theories and everything. You always learn from me data.
In the past, I've said from phase 2As you learn from them in order to design the phase 2b and ultimate phase 3. That's not what I'm saying here this morning. I'm saying that that, there's some biology here that we don't fully, appreciate, and this is this is data. And just like any experiment, we're going to learn from this data and moving forward, maybe, you know, both for Tourette, but certainly also for, the many other indications, that we, that we look at as being areas that we want to explore, particularly with our, VINAT 2 backup. Compound.
So this is, it's not trying to always look on the bright side, but we've got data now. We've got a really good data here and it's going to talk to us.
Okay. No, I definitely appreciate that. Thanks. And then on the other ongoing study, I believe it's a randomized withdrawal study, which in, 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?
And to that point, Is that study a well designed study that, you know, 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. And and we believe that is a well designed study and is currently ongoing. As I mentioned earlier, we need to learn, at least, on this data set to understand the next steps for both platinum and for the remainder of our direct syndrome program, but it's just too early given where we are with the data today to make any definitive statements about the, the remainder of the program. And, Paul,
I would add that, in the event, and I think that you would have to put low probability of success. On this for platinum. But in the event that it that it was positive, that certainly wouldn't be sufficient for an sNDA filing. We don't believe that there would have to be another basically double blind placebo controlled randomized study that would need to be done. And currently, we don't we don't have any insights on how we would, design a better study than what T Force Gold Plus.
Okay.
All right. Thank you Kevin and I really 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 Ira, I'm sorry if I missed this in the in the opening comments, but were there any imbalances in the baseline characteristics in in T4Sure worth noting? Thanks so much.
No. This was a very well designed, well controlled study. There were no significant imbalances on any of the is really in the baseline characteristics. And, 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, but 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 albenazine although present and present consistently was smaller than we had anticipated.
Yeah. And also, I would just, I would just add again, both the things that you would normally look at and maybe you could hang, why, why you didn't get the result that you anticipated I already talked about, placebo effect, or, you know, dropouts or, or more variability than what you see. There's none of that. This was a this was a very a very clean study. The the study gave us a, a very, direct answer to the hypothesis that we were testing, in here.
Thanks so much for
Thank you. We'll move next to Jay Olson of Oppenheimer. Your line is open.
Hey, guys. Thanks for taking the questions. I know you've commented on the primary endpoint for, T Force Gold. Were there any subcategories of the, total tick score such as motor or sonic ticks that showed greater separation from Placebo.
We're obviously still analyzing the full data set. We just have top line data right now, but what I can say is that the effect size of Albenazine was pretty consistent across the, sub elements of the, Yale global tick score.
Okay. Thank you. And then maybe just as a follow-up, I know you've mentioned previously interested in pursuing new indications either for valbenazine or the next gen VMAT2 inhibitor in a certain undisclosed movement disorders does the lack of efficacy in Tourette Syndrome change your views on the potential, for additional studies in in those other movement disorders?
It's a good question. I'll take 1st crack at it. I think we're still very enthusiastic, about the about the mechanism of modulating, presynaptic levels, of dopamine And there are, 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 it adds a it adds a what I think is gonna be a very good and useful, piece of information for us after we've had the, 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, but I still, very much believe that, this is an important mechanism in in CNS and in movement disorders.
So I would agree completely. I think we know that Valdera's income produce a profound efficacy for patients with tardive dyskinesia and that's certainly a validation of this mechanism, in, in that patient population. And as Kevin mentioned, we have a broad number of different indications that we're interested in, and 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'll take that
question from Alan Carr of Needham And Company.
Hi. Thanks for taking my questions. Kevin, you sound a little pessimistic around platinum outcome. But I'm wondering if you could remind us that the the design of that one, maybe powering around that. Is there, any chance that, a size of it also?
Any chance that, the effect size might, that that the outcome of that might might still be favorable. And then also if, in the end, you do, drop Trent syndrome, does it what sort of impact would that have on your overall business development strategy, if anything. Thanks.
Yeah. So I'll, I'll let Ira handle the first question. I'll take the second question.
So, T Force platinum design is such that, this is a randomized withdrawal study. The patients receive open label treatment of to an optimized dose of valbenazine for the 1st 6 weeks of the study. And then that treatment is continued till week 12. And then patients who are responding are randomized to either placebo or, to continue active treatment with valbenazine. This study answers a different from a parallel randomized control study.
And, as such, I I think it is an independent study in terms of its likely outcome. But 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. So we still need to learn from the current data to, determine if there is any, of any next step in this indication, and we'll be doing that over the coming weeks.
Ma'am, Alan, I'm gonna use, I'm gonna use the the the last part of your question to actually get my summary, remarks here before we sign off. I don't want to minimize the, the disappointment we have here and, and, and also the fact that, we're, we're very, we're very surprised and, about the outcome that we had here But honestly, when it comes to the company itself, it it is a it is a setback, but it is a far bigger setback. For the patient suffering, from Tourette's. And so that's why we're going to be very thoughtful, in our approach to valbenazine and other therapies that we have, that that we're gonna explore in Tourette. This is a this is a very high unmet medical need area.
And and and this is where the real, the real setback is, is for the patients, for the company. We have a number of, of very strong drivers of growth taking us into the future and Accenture still looks very, very good, for Neurocrine. We're we're fortunate that we're not a one product company. And we're fortunate that, that the compounds we've discovered and developed commercialize on our own commercialize with our partner AbbVie they have a lot of runway, that they're going through. There's there's, the drugs are very good.
They bring tremendous benefit in tardec dyskinesia and endometriosis. And I would say soon, that you'll see the filing that AbbVie will be 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 a pick up on, that we're going to be filing in Q2. And then we have CAH, which we're gonna look forward to sharing the data with you in, 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 2 that, that hit, this year, and we look for more next year. And then on the business development side, we are, we are more active than what we've ever been before. We, we price a high emphasis on this. There's no need for Neurocrine dependent care in any situation.
It doesn't change our mission. It doesn't change our strategy. And we're going to keep moving forward. And, and that mission is to bring hope, to patients. And that's exactly what we're going to do through all facets of the company.
And so 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, and everyone have a great day.