uniQure N.V. (QURE) Earnings Call Transcript & Summary

Good day, ladies and gentlemen, and thank you for standing by. Welcome to the regulatory update on AMT-130 for Huntington's disease. [Operator Instructions] At this time, I would like to turn the conference over to Chiara Russo, Senior Director of Investor Relations. Ms. Russo, please begin.

Thank you. Good morning, and thank you for joining us. This morning, uniQure announced alignment with the Food and Drug Administration on key elements of the accelerated approval pathway for our investigational gene therapy AMT-130 for the treatment of Huntington's disease. On this call, we will walk through the key elements we have reached alignment on with the agency, as well as our anticipated next steps. Joining me for this webcast are Matt Kapusta, our Chief Executive Officer; and Dr. Walid Abi-Saab, our Chief Medical Officer. Please note that we will be making forward-looking statements during this investor call. All statements other than statements of historical facts are forward-looking statements. They are based on management's beliefs and assumptions and on information available to management only as of the date of this conference call. Our results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, the factors described in uniQure's most recent SEC filings. Given these risks, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these statements even if new information becomes available in the future. Now let me introduce Matt Kapusta, uniQure's CEO.

Thank you, Chiara, and good morning, everyone. We're truly thrilled today to announce that following our Type B meeting with the FDA in late November, we have reached alignment on key elements of an accelerated approval pathway for AMT-130, our wholly owned investigational gene therapy for the treatment of Huntington's disease. This milestone represents a significant step forward for the Huntington's disease community and an incredible achievement for uniQure. The FDA's feedback, which was based on a thorough review of our most recent clinical data and discussions with our medical team, underscores the potential for AMT-130 to address unmet medical needs for the treatment of Huntington's disease. We're very grateful for the agency's guidance and commitment towards helping those people who live with this debilitating disease. The accelerated approval pathway avoids the need for an additional lengthy and costly pre-submission study, thereby reducing our time to potential licensure by approximately 5 years. We've already begun BLA readiness activities, including accelerating our CMC preparations, and very much welcome the opportunity to bring this potentially transformative treatment to patients as quickly as possible. With approximately 70,000 people diagnosed in the U.S. and Europe and hundreds of thousands more at risk of inheriting this devastating disease, Huntington's represents one of the world's largest genetically defined unmet needs. As such, AMT-130 has generated significant interest from patients, clinicians, investors and strategic parties alike. We're confident that our existing cash resources, alongside the significant cost reduction initiatives implemented earlier this year, provide the resources needed to swiftly advance AMT-130 to a BLA submission, and we look forward to providing more granularity on time lines and updated financial guidance in the new year. Now I'd like to turn the call over to Dr. Walid Abi-Saab, who will provide more detail on the data presented, the regulatory path ahead and next steps for the program. Walid?

Thank you, Matt. Good morning, and good afternoon, everybody. As you can imagine, I'm extremely excited that we have reached alignment on an accelerated approval pathway with the FDA. This is excellent news for the Huntington's disease community, including investigators and advocates whose unwavering support have made this update possible. In fact, without the tremendous work that was done in this field for decades to further our understanding of the biology, as well as the clinical course of the disease and associated biomarkers and, most importantly, without the invaluable contributions, commitment and generosity of people living with Huntington's disease and their loved ones, we would not be where we are today. So to all of you, a deep heartfelt thank you. First, I will share a regulatory update on our Huntington's disease program. I'd like to briefly review the progress we've made this year with our ongoing Phase I/II studies of AMT-130 and the data they have produced. In May of this year, AMT-130 became the first Huntington's disease investigational drug to receive Regenerative Medicine Advanced Therapy, or RMAT, designation, which allows for more frequent interactions and discussions with regulators. In applying for the designation, we submitted promising results that we shared with you last December, which included data from 8 patients at 24 months of follow-up compared to an external natural history control using propensity score matching. Those analyses provided preliminary clinical evidence that AMT-130 has the potential to address unmet medical needs for the treatment of Huntington's disease. Thus, the designation was granted. In July, we expanded on this analysis with data from 21 patients at 24 months of follow-up, demonstrating for the first time a statistically significant dose-dependent and potential durable slowing of disease progression based on cUHDRS compared to an external propensity score-weighted natural history control. Additionally, the data showed a statistically significant reduction from baseline in cerebrospinal fluid neurofilament light chain levels at 24 months, further supporting AMT-130's potential therapeutic effects. Building on this progress, the uniQure team prepared a comprehensive briefing book with both substantial nonclinical, CMC and clinical data as well as specific questions for an initial multidisciplinary Type B meeting with the FDA. Additional data from 3 high-dose patients having crossed the 24-month duration were also included with the updated data from July. So in total, 24 subjects with 2-year data, 12 on the high dose and 12 on the low dose, were submitted in the briefing book. We went into the meeting looking for clarity on 2 important topics; one, that the potential therapeutic benefit of AMT-130 may be demonstrated relative to a natural history external control group in the ongoing Phase I/II studies; and two, that data from the ongoing Phase I/II studies may serve as the primary basis for an accelerated approval application of AMT-130. During this meeting, the FDA provided us with important guidance that allows us the opportunity to submit a BLA using the accelerated approval pathway for AMT-130. First, data from the ongoing Phase I/II studies compared to a natural history external control may serve as the primary basis for a BLA application for accelerated approval. This means that an additional registrational trial for accelerated approval will not be required. Second, the UHDRS may be used as an intermediate clinical endpoint to support an accelerated approval application. And lastly, reduction in CSF NfL levels may serve as supportive evidence of therapeutic benefit. We are very pleased that the agency provided us with this important clarity on a path forward. In terms of next steps, we plan to engage with the FDA in the first half of 2025 to finalize our statistical analysis plan and technical CMC requirements. In the meantime, enrollment in Cohort 3, which aims to evaluate the effects of immunosuppression on perioperative safety, is almost complete. We expect the last of the 12 patients to be dosed in the first quarter of '25, at which time a total of 45 patients will have been dosed with AMT-130. We expect to provide an initial safety update from Cohort 3 in the first half of 2025 and remain on track to deliver a 3-year update on 21 patients from Cohorts 1 and 2 in [ late ] 2025, similar to the analysis shared in July. With that, I'll turn the call back over to Matt.

Thanks, Walid. As we close out 2024, uniQure stands on a markedly different value-creating trajectory compared to when we began this year. We now have a clear and accelerated path to potential approval for AMT-130 in Huntington's disease, avoiding the need for an additional pre-submission study and significantly expediting the registration of this potentially first- and best-in-class therapy. Our decisive cost containment measures, combined with our strong financial position, position us to advance AMT-130 as aggressively as possible. At the same time, we've initiated 3 new clinical studies over the past 6 months, each of which provides additional opportunities to create value in 2025 and beyond. With that, we will open the call to take questions. Operator, please proceed.

[Operator Instructions] Our first question or comment comes from the line of Salveen Richter from Goldman Sachs.

This is Lydia on for Salveen. Just on the path forward, would you still consider exploring an external partnership now that you have an accelerated approval path?

Yes. Thanks for the question. Right now, we are focused on driving AMT-130 to a BLA submission [Audio Gap] that we have the financial resources and made the significant decisions to reduce our [indiscernible] earlier this year that really put us in a position to be aggressive with moving this program forward. So our first order of business is taking AMT-130, advancing it, and that's really what we're focused on right now.

Our next question or comment comes from the line of Debjit Chattopadhyay from Guggenheim.

Congrats on the update here. Matt, I just wanted to confirm one thing. The filing is on the 2-year data and the FDA does not require the 3-year follow-up for the submission? And if the FDA does require the 3-year follow-up, is there a threshold that -- on the UHDRS that the agency wants to see?

Debjit, there was something wrong with the audio. Can you just repeat that?

Yes. So just wondering if the filing is gated only on the 2-year data and you wouldn't need the 3-year follow-up data for the submission? And if you need the 3-year data, does the FDA have any threshold on the UHDRS?

Yes. Thanks, Debjit, for the question. So 2 things on this. One is keep in mind that just given the cadence of enrollment, right, we already have [Audio Gap] At point in time, we're going to have a bolus of patients that have 3 years of follow-up. In fact, next year [Audio Gap] [ treated ]. The other thing that I would mention is that we expect to have discussions specifically about the statistical analysis plan, which will be prespecified in the first half of next year. So we expect to provide some additional guidance in that regard early in the new year.

Our next question or comment comes from the line of Paul Matteis from Stifel.

This is James on for Paul. And I actually had a follow-up there on kind of the time lines here. I guess, is there going to be a kind of set time line, you think, in terms of when this analysis will be done in the sense of everyone will be at 3 years? Or is it going to be kind of a mix just given where patients are? And then, kind of two, on that kind of designing an SAP, just kind of curious what sort of effect size you think you're powered for, I guess, in the sense in terms of showing statistical significance. And in the 2-year cut, we saw a very large implied kind of effect size versus natural history. So kind of just curious how that informs what the SAP may ultimately look like?

Sorry, Paul, could you repeat that question?

Yes. This is James on for Paul. Can you hear me? Yes. I just kind of wanted to have a quick follow-up on sort of that time line question in the sense of do you think everyone needs to be at 3 years? Like is there kind of a set threshold in terms of when this analysis is going to be done? And -- or is it kind of just a mix of where patients are kind of just at in terms of their follow-up? And then, two, on the designing of the SAP, what sort of effect size do you think you are powered for in terms of showing statistical significance? Obviously, we saw statistical significance at 2 years. So just kind of curious what you think that -- how that informs kind of your designing of the SAP.

Okay. You're connected. Can you hear us?

Yes, we can hear you now.

You're back on.

Sorry. Paul, you'll have to repeat your question.

No worries. This is James on for Paul. Congrats on the alignment here. I did want to follow up on kind of that last question in terms of time lines in the sense of is there a certain threshold or certain time line that all patients need to get to in terms of doing this analysis, like could it be all 3-year data? And then two, in terms of kind of designing the SAP, I just kind of curious what sort of effect size you think you're powered for in terms of showing a statistical significant benefit. Obviously, we saw the 2-year cut a stat sig benefit and a pretty large implied effect size versus natural history. So just kind of curious how that informs how you're thinking about the SAP.

Yes. So -- apologies, by the way. We just had a major Internet crash, so we're back on at least. So just to answer those questions, at this juncture, there is no specific guidance that we've received in terms of a particular number of patients that have to reach a specific amount of follow-up. And we'll obviously provide more guidance as we have future discussions with the FDA. The second part of your question is, in the end, we demonstrated in our July data presentation that we had statistical significance even with 12 patients that had achieved 2 years of follow-up. So I think we feel really encouraged by the data that we have thus far and our ability to achieve a prospectively defined endpoint with the FDA.

Our next question or comment comes from the line of Joe Schwartz from Leerink Partners.

It's Jenny on for Joe. Congrats on the progress. Can you give us some more details on how [ you're ] accelerating your CMC activities? What still needs to be done? And how many doses do you think you'll be able to supply the market by the time the FDA completes the review?

Yes. So I mean, the major things that have to be done, which are all standard, is to finalize the process validation and to do our PPQ consistency runs. We think we're going to be in a very good position. There's no tech transfer that's going to be required. The process that we're utilizing is highly comparable with the process that we used for HEMGENIX and the material is being made in a facility that is already licensed by the FDA to produce gene therapy. So we feel like we're going to be in a good position in that regard to complete our CMC activities. In terms of the readiness for commercial launch, just keep in mind, this is not an IV-administered product. So the quantities that are actually administered are relatively small. So we feel like we'll be in an excellent position to produce the material that we need for a launch of the product.

Our next question or comment comes from the line of [ Lucas ] (sic) [ Luca ] Issi from RBC Capital.

Obviously, congrats on the alignment with the FDA here. Maybe 2 quick ones. Maybe, Walid, just kind of stepping back, can you just talk about endpoints here? I find it fascinating that you're talking about cUHDRS and NfL versus Wave is talking about imaging and PTC is talking about mutant huntingtin. Again, I appreciate that these are different approaches. And obviously, there's some complexities around CBER versus CDER. What's the best way to rationalize why is the FDA asking different companies different endpoints there? So again, any color there much appreciated. And then maybe, Matt, can you just talk about Europe? What is the next interaction with the EMA and how should we think about the path for approval there?

All right. Thanks, [ Lucas ]. This is Walid. I'll take both questions actually. On the first one, on the endpoints, I mean, I think it's very difficult to speculate. The FDA feedback is often given based on the questions that are asked, honestly, and it's very difficult to speculate how these discussions with other companies are turning out. We've always indicated that changes in, for example, mutant huntingtin is unlikely to serve a basis because, simply, there's a very large trial with tominersen that showed dose-dependent reductions at the same time patients did not get better. If anything, they got worse, unfortunately, in that trial. So what I can comment is, on our discussions with the FDA, we made a case that cUHDRS is a likely endpoint to be able to predict potential for benefit and as such, could serve as an intermediate clinical endpoint. It is something that's been actually used often. There's a lot of new data now have accumulated, to a great extent also thanks to the tominersen data set that is very large. And I think the agency is feeling comfortable with it to serve as an intermediate clinical endpoint, which is reasonably likely to predict the future potential benefit. On the NfL, I think it's also -- there's increasing evidence that NfL, although not specific for Huntington's disease, is very specific for neuronal injury and actually leaks out of the cells when they are injured or degenerating. And for us to have data that we've shared with you previously that robustly show a reduction from baseline at 2 years, when you would expect a significant increase from baseline as the disease progresses, also serves as a very solid foundation for this. So we we're very happy. We feel the FDA is actually reacting to data that we presented. This data is getting more and more mature, as Matt said. What we included in the briefing book are data from 24 subjects who have crossed the 2-year time point, 12 on the low dose and 12 on the high dose. But again, at that time, we also had a number of subjects, particularly on the low dose, that have been -- for whom we have more data. On the EMA, the plan is to engage with the EMA with a series of meetings starting early in 2025. So we're -- our aim is to align with them on a path which we hope would be very much in line with what the FDA is chartering for us and agreeing for us on a way forward. So we'll keep you posted.

Our next question or comment comes from the line of Ellie Merle from UBS.

Congratulations on the update. Just want to clarify a little bit. What's your expectation for when you plan to file the BLA? Is it that you're waiting for the 3-year data and the safety from Cohort 3 and then you could file towards the end of next year? Or just help us think through that. And then just a second question, in terms of the clinical endpoints, I guess, should -- like what does the FDA view as a clinically meaningful difference on cUHDRS? And I guess, should we interpret this as the FDA viewing your data reported as already showing a meaningful difference, since they're reaching sort of alignment with you on that endpoint?

Yes. Ellie, I guess I could take the first question and Walid can take the second question. What I can tell you is internally, we are going to be aggressively moving forward to put us in a position to submit a BLA as quickly as possible. We've had one meeting with the FDA, and it was an extremely productive meeting that we're very excited about. We expect to have some additional discussions with the FDA beginning in the first quarter of next year. And once we have those discussions, we'll provide more color on the specific timing of a BLA submission.

Thanks. And I'll take the second question. Again, I think it's also same color as what Matt said. This was the first meeting whose purpose was actually to have a high-level agreement with the FDA on the development plan across multiple disciplines. Now in addition, we asked specific questions for which we got specific answers and -- as we shared in the PR and also in the call earlier today. There's not been any more detailed discussions. The question of clinically meaningful difference did not come up at all in our discussions. So that doesn't mean it's not -- it's on the table, but it didn't come up. And I think the purpose of the subsequent meetings, as Matt said, would be to nail down from a clinical perspective the statistical analysis plan, the protocol for the external cohorts and how we're going to be comparing those. But the meaningful clinical difference did not come up. And to me, I'm not necessarily worried about it. I think the agency has seen the initial data as supportive for a potential accelerated approval and engaged with us in a very constructive discussion to help us achieve that goal, which is also their goal, to bring medicines to patients as quickly as possible in a safe way.

Our next question or comment comes from the line of Danielle Brill from Raymond James.

Congrats on the progress here. I guess I'm just curious why you can't pursue a traditional approval with cUHDRS versus natural history? What additional evidence does the agency want, or how much additional follow-up are they looking for to confirm benefit? And then as a follow-up, what are your expectations at this point for confirmatory trial requirements?

Yes. Maybe I'll take a stab at that, Walid, you can jump in. I think that the -- I mean, just keep in mind the progress that's being made from a regulatory perspective. I think what we're excited is that the agency has recognized cUHDRS as an intermediate clinical endpoint. And this is not just in Huntington's disease, but I think the agency often takes a skeptical approach of composites because composites are a mixture of different variables. But there's been a tremendous amount of independent data that has shown that the composite UHDRS is actually highly correlated with progression of disease and, if anything, more sensitive than some of its individual components. The FDA does seem to be focused on total functional capacity, which is a strong measure of the quality of life and the impact of the disease on patients. So that's something that the agency perhaps may be looking at in thinking about full approval. But it's not clear. In all honesty, the requirements for full approval are still on the table, and even for what would constitute confirmatory evidence are still on the table and whether or not a new study would even be required. So those are things that we need to discuss further with the agency in the first half of next year, and we'll provide input or feedback once we have those conversations.

Our next question or comment comes from the line of Uy Ear from Mizuho.

Congrats on the progress. So just a couple of quick questions. So you indicated in the press release that there were staffs and senior staff members at the FDA meeting. Just maybe provide some colors on who was there and what was sort of their -- your read of their impression on the data, the briefing books that you saw? And why is it -- I guess I'm just going back to a little earlier where you kind of told us not to get too excited because of other companies -- FDA were more favorable to biomarkers. And -- yes -- and essentially, you told us to not get too excited. Just wondering like what was different in terms of your expectations going into it and coming out of this with these alignments? And second question is on CMC, do you -- can you remind us whether the current product, I guess, do you need to do anything? Is there a bridging study to get to commercial products?

Yes. I mean maybe -- again, I can chime in, and Walid, I think you should also discuss the kind of tenor or tone of the meeting. But I think that the -- I'm not going to comment specifically on who attended the meeting. What I can tell you is that there were, I think, over 30 people. This was an in-person meeting, but also hybrid, of course, with people dialing in, but it was a multidisciplinary meeting with, I think, over 30 people that had attended, and there was senior FBR officials at the meeting. And that's obviously one of the benefits of being under RMAT designation. I think that, obviously, everybody's interactions on different programs are all nuanced and different. We have a lot of insight into other companies going through this process and understand that it was a journey to achieve alignment with the FDA. And so we were really pleasantly surprised. While we believed in the adequacy of an accelerated approval pathway, we're really pleasantly surprised by our ability to align in the initial RMAT meeting. But honestly, we think this is a testament to the strength of our data and the long-term outcomes that we have and the FDA, in my mind, leaning in to really try to work with sponsors to find expedited pathways of bringing these transformational therapies to patients. Just lastly on the CMC piece is we do not require any material changes to the process that we use to make the material that is being utilized in our Phase I/II study compared to what we expect would be utilized in the -- in commercially. So we don't expect there to be any bridging studies that are required because of the fact that the process is going to be largely consistent with what we have used and are clinically testing.

Our next question or comment comes from Sami Corwin from William Blair.

I guess I was curious, as you're kind of thinking in the future about labeling, what the inclusion -- included population might look like given the requirement for caudate size and disease severity in the trial and if you think you'll need to conduct additional studies in the future in order to expand this. And then I was wondering if you could also remind us how many centers or doctors are currently equipped to do the surgical procedure in the U.S.

Yes. Thanks, Sam. This is Walid. So I think this is really premature. There's been no specific discussion talking about labeling and indication. But very simply, we set out to test the hypothesis that blocking a mutant huntingtin protein, lowering it, and particularly lowering the HT1a, which is now becoming more and more clear that this plays an important role, is a way to be able to change the course of the disease. And I think now we're starting to generate data to show that, and that's why we are where we are today. So I think we will be discussing with the FDA what this study that we have would allow us in terms of label. But I would imagine if this is a therapy that will affect the fundamental underlying biology of the disease, I think there will be a lot of collaboration with the agency to see how best we make it available to the most appropriate patients and what additional work potentially could be done to enlarge this to even an earlier stage of the disease. So in terms of the centers, I think currently we have a number of centers in the U.S. and some in the U.K. But we are, of course, as part of our preparation for the future, would be planning to increase those. I mean, this would not be very different than any of the highly sophisticated type of therapies that you will get at very specific centers who are equipped to do these types of surgeries, equipped also with MRI-guided technology to be able to do this. As a onetime therapy, I think that will be also compatible with administering the therapy going forward. But again, we are in the early stages of this. We're all hands on deck preparing for this, and we will be providing more comments as we further articulate that story.

Our next question or comment comes from the line of Peyton Bunseit from TD Cowen.

This is Peyton on for Joe. First off, congratulations on what appears to be a very productive meeting. I guess, looking forward to the safety data from Cohort 3, would you need to have that before you would submit? And if you saw benefit from the perioperative immunosuppression, would you -- how would you incorporate that [ and roll ] into the BLA submission?

Yes. What I would say is we believe, based on data from the Phase I/II study, in particular the first 2 cohorts, that both doses are generally safe and well tolerated with a manageable safety profile. So that's even with the initial immunosuppression regimen that we had. I think we view that the third cohort is really in order to optimize the immunosuppression, and we don't necessarily view it as a long pole in the tent or a prerequisite.

Our next question or comment comes from the line of Kristen Kluska from Cantor Fitzgerald.

Congrats on these updates. So originally, you had guided to the Street that it may take a few discussions to get some concrete feedback on the path forward. But it appears with today's update that one meeting alone gave you a really good sense. So I was hoping you can maybe speak on anything from a macro perspective that may have changed this? I know the FDA has been heavily involved in this. They recently attended an event hosted by HDSA. So curious if that or just anything from the macro perspective gave them the ability to give you more concrete feedback on this first meeting versus a series of them.

No. I mean I think I know what you mean when you talk about macro and did that create some kind of urgency, the political landscape. Honestly, we just don't buy that. We don't see that. We think that this is not -- the FDA is not acting as a political animal, the FDA is focused on data and focused on executing their mission. And if anything, our view is that the speed at which that we've been able to align with them is really more of a testament to the substantial amount of data and follow-up, including clinical outcomes, that we've been able to demonstrate and not something unrelated or macro or political in nature.

Our next question or comment comes from the line of Yanan Zhu from Wells Fargo Securities.

Congrats on the alignment. Three questions, if we may. So first, on the safety database for the original surgical procedure and route, is there a sense of the size of safety database required for the filing? And could that be more gating than the efficacy cohort size? And second question, on the size of the natural history control or the leeway of selecting those natural history patients, do you have a sense that FDA is granting more or less leeway of how to select those natural history patients compared with what you presented in the July presentation? And thirdly, on the magnitude of neurofilament light chain reduction at year 2 from baseline, do you think -- I think for the BLA, the number of patients from the high-dose cohort may not be the same as what you were presenting in July. So I was wondering, given that this will be a supportive endpoint, is there a requirement that a certain degree similar to what you presented has to be achieved at year 2? Or could it be even an increase potentially and still this could support approval? Sorry for the multitude of questions.

Okay. Thanks for the questions. Let me just try to tick them off. And again, I'll encourage Walid to join in. So in terms of the safety base, let me just repeat that very shortly we're going to have 45 patients in total treated with AMT-130, with the first patient achieving, I believe, 5 years of follow-up next year and an increasing number of patients with more than 3 years and everybody from the first 2 cohorts with at least 2 years of follow-up next year. So I mean, our view is that this represents a substantial safety database that I think should give the agency comfort that this is generally safe and well tolerated. Obviously, we need to have additional conversations with the FDA, but that's our perspective. On the natural history, again, the details have to be discussed at later meetings. I mean the most important point is that the FDA understands the difficulty in administering a properly internally controlled blinded study and has accepted that the natural history, given how much is available in Huntington's disease, can potentially provide accelerated approval or support of an accelerated approval. To me, that's the most important thing. And there's obviously some details that have to be discussed, but those we can get to in the next year or the new year. And then in terms of NfL reductions, I think the important thing here -- there was no conversation about specific requirements of what number it had to be at or what the flexibility was. I think what was clear is that the FDA understands NfL, understands that it is generally a very well-characterized marker for neurodegeneration and appreciates the fact that reductions in CSF NfL indicate lower levels of neurodegeneration happening, which is clearly not expected as Huntington's disease progresses. So I think all of these things are, I think, incredible progress that we've made in a singular meeting, and we'll provide some additional details as we have additional meetings with the FDA next year.

[Operator Instructions] I'm showing no additional questions in the queue at this time. I'd like to turn the conference back over to management for any closing remarks.

Okay. Thank you, everybody, for joining us today. I'd also like to give a special thanks to the uniQure team for their unwavering dedication to our mission and the Huntington's disease community for all of your support and partnership over the years. We deeply appreciate the FDA's recognition of the urgency to address the unmet medical need clearly present in Huntington's, and we very much look forward to providing additional program updates in the new year. Thank you very much, and have a great day.

Ladies and gentlemen, thank you for participating in today's conference. This concludes the program. You may now disconnect. Everyone, have a wonderful day. Speakers, stand by.