uniQure (NASDAQ:QURE) reported third-quarter financial results on Wednesday. The transcript from the company’s third-quarter earnings call has been provided below.
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Summary
uniQure reported a significant milestone with the FDA accepting its biologics license application for AMT-130, a treatment for Huntington’s disease, with a priority review and a PDUFA date set for May 1, 2027.
The company reported Q3 2026 revenue of $5.2 million, up from $3.7 million in the same period last year, with cash reserves expected to fund operations into 2030.
uniQure is actively preparing for a potential U.S. launch of AMT-130 in 2027, focusing on commercial readiness, including training treatment centers and engaging with payers.
The company continues to advance its clinical pipeline, with progress in trials for AMT-260 for epilepsy and AMT-191 for Fabry disease, and expects new data in the first half of 2027.
Management emphasized its commitment to executing the confirmatory study for AMT-130 and expressed confidence in its ability to meet regulatory requirements and timelines.
Full Transcript
OPERATOR
Hello and thank you for standing by. My name is John and I will be your conference operator today. At this time I would like to welcome everyone to the uniQure third quarter 2026 earnings call. All lines have been placed on mute to prevent any background noise. After the speakers’ remarks, there will be a question-and-answer session. If you would like to ask a question during this time, simply press star followed by the number one on your telephone keypad.
If you would like to withdraw your question, press star one again. Thank you. I would like to turn the call over to Kiara Russo, Senior Director of Investor Relations.
Kiara Russo (Senior Director of Investor Relations)
Thank you. Good morning, and thank you for joining us for uniQure’s third quarter 2026 earnings call. Earlier this morning, uniQure released its financial results for the third quarter of 2026, and our press release is available on the investors and media section of our website at uniQure.com. Our 10-Q was also filed with the SEC earlier today. Joining me on the call this morning are Matt Kapusta, Chief Executive Officer, Dr. Walid Abi-Saab, Chief Medical Officer, Kylie O’Keefe, Chief Customer and Strategy Officer, and Christian Klemt, Chief Financial Officer.
After our formal remarks, we’ll open up for an analyst Q&A. Before we begin, please note that we will be making forward-looking statements during this investor call. All statements other than statements of historical fact 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 actual 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 information becomes available in the future. Now let me introduce Matt Kapusta, uniQure’s CEO.
Matthew Kapusta (Chief Executive Officer)
Thanks, Kiara. Good morning, and thank you for joining us on today’s call. For the families who have waited generations for treatment for Huntington’s disease, this quarter brought meaningful progress. As we announced Monday, the FDA has accepted our biologics license application for AMT-130 and granted priority review with a target PDUFA date of May 1, 2027. In the United Kingdom, the MHRA has also validated our marketing authorization application.
These are the first such marketing applications for a disease-modifying therapy for Huntington’s disease, and it brings patients and families one step closer to a treatment option. We greatly appreciate the engagement of both the FDA and the MHRA and their focus on the critical need for new therapies in Huntington’s disease. I also want to acknowledge the commitment of our clinical, regulatory and CMC teams who prepared and submitted both applications in record time, and to every HD patient, family member and investigator who has trusted us with their hope.
Thank you. In September we shared additional data from our ongoing Phase 1/2 studies, including updated 36-month data from all 27 evaluable patients across the low and high dose cohorts and, for the first time, 48 months of follow-up after a single administration of AMT-130. These data continue to show meaningful, dose-dependent slowing of disease progression and reinforce our strong belief that AMT-130 has the potential to deliver treatment benefit to patients.
We are also pleased to have aligned with the FDA on the key elements of the confirmatory study design, and we are making significant progress getting the trial up and running. We are fully committed to the timely execution of this study, and our priority is to have it well underway at the time of an approval decision. Waleed will provide more detail on the confirmatory study later in the call. Regarding commercial preparations, we are making excellent progress ahead of a potential U.S. launch in 2027. We continue to build out our field medical, market access and strategic account teams in a disciplined way, and our financial strength provides the runway needed to execute this build-out. Kylie and Christian will provide more details on our commercial readiness and financial position later on the call.
Beyond AMT-130, we continue to advance our broader pipeline. We have completed enrollment in the second dose cohort of our Phase 1/2a study of AMT-260 in refractory mesial temporal lobe epilepsy and have resumed enrollment in the Phase 1/2 study of AMT-191 in Fabry disease. For both programs, we expect to present new and updated data in the first half of 2027. In summary, we are heading into the end of 2026 with strong momentum. Our priorities are clear: working closely with regulators as the application reviews proceed, executing our confirmatory study, advancing our pipeline, and building the organization to prepare for the planned launch of AMT-130. With that, I will turn the call over to Waleed to provide additional detail on AMT-130 and our pipeline. Waleed.
Walid Abi-Saab (Chief Medical Officer)
Thank you, Matt. Good morning and good afternoon everyone. I’ll start with AMT-130 in Huntington’s disease, or HD. As Matt noted, the BLA is under priority review with a PDUFA date of May 1, 2027, and the MAA has been validated by the MHRA. Both submissions are based on the 36-month analysis we presented in September 2025. In that analysis, the high dose of AMT-130 showed a 75% slowing of disease progression on the composite Unified Huntington’s Disease Rating Scale, or cUHDRS, and a 60% slowing on Total Functional Capacity, or TFC, compared with the propensity score–matched external control.
Both results were statistically significant. With the BLA now accepted and under active review, going forward we will not be commenting on specific information requests made by the FDA, including questions regarding a potential outcome. The FDA indicated that they are tentatively planning to discuss our application in an AdCom. As you’re aware, scheduling an AdCom is within the sole discretion of the agency. In September 2026, we presented detailed analyses from a recent data cutoff. I will not repeat that today. Instead, I would like to focus on the progress we’ve made towards initiating the confirmatory study. Earlier this year we reached alignment with the FDA on key elements of the design. Specifically, this will be a randomized study with a 2-to-1 ratio of AMT-130 high dose to standard of care. It will include approximately 200 patients. The primary endpoint is TFC at 36 months.
After 36 months, all standard of care participants will be offered AMT-130 in a separate open-label study. uniQure is fully committed to completing the study in a timely manner. Preparations started at the beginning of this year and have accelerated following alignment with the FDA. This will be a global study with sites across North America, Europe and Latin America. The protocol has already been submitted to institutional review boards, or IRBs, in the U.S., and we expect to begin patient screening by the end of this year.
I would like to take a moment to talk about TFC, the primary endpoint in the confirmatory study. This is a clinical endpoint that is very important not only for regulators but also for people living with HD. Why is TFC important? TFC captures whether a person can keep working, manage their finances, handle household responsibilities, take care of themselves day to day, and live without full-time care. For patients and families, these are not abstract measures.
They are the difference between maintaining and losing independence, and they are directly related to quality of life. This is what patients and families care about. In fact, it is what we heard from HD specialists at the two most recent scientific meetings we attended, the International Parkinson and Movement Disorder Society meeting in Seoul and the European Huntington’s Disease Network meeting in Krakow. These specialists describe TFC as the endpoint they find most meaningful when talking with patients.
Across our Phase 1/2 studies, TFC declined by less than a tenth of a point per year in patients who received a high dose of AMT-130. This remarkably slow decline was consistent across all analyses and maintained through four years following treatment. Although these results come from a relatively small number of patients, they have never been seen before in HD. Moving on to our other clinical programs, starting with AMT-260 for refractory mesial temporal lobe epilepsy. Following the initial six-month data of the first low-dose cohort of six patients, which was presented in June, earlier this quarter we completed enrollment of the second cohort of seven higher-dose patients. We expect to present updated results from both cohorts in the Phase 1/2a study in the first half of 2027.
Turning to AMT-191 for Fabry disease, in August, enrollment resumed in the mid and high dose cohorts. As a reminder, dosing had been paused per protocol in these two cohorts after asymptomatic liver enzyme elevations, which fully resolved following a course of immunosuppression. We presented updated results in June from the March 15 cutoff, and we expect to share updated results from all three dosing cohorts in the first half of 2027. Now I will turn the call over to Kylie to discuss our recent patient advocacy work.
Kylie O’Keefe (Chief Customer and Strategy Officer)
Thanks, Waleed. With the known PDUFA date ahead of us, the question for our customer-facing team is no longer when, it’s how? How do we make sure every piece of this patient journey is in place and well supported by the PDUFA date? So if approval is granted, we are ready to launch for AMT130. That journey runs from identifying an eligible patient through evaluation, surgery, follow‑up, and reimbursement. It involves many critical stakeholders. Our job is to ensure a smooth process for patients, families, and the clinicians who care for them.
For many of these families, the patient journey already runs through a familiar place, a specialty HD clinic they have relied on for years. That is an important feature of this market. As HD care is concentrated, a large share of diagnosed patients are seen at a number of specialty centers, many of them HDSA Centers of Excellence. That concentration will allow for a focused launch footprint. We continue to see strong enthusiasm from the physician and patient community.
This quarter our team spent significant time with the Huntington’s disease clinical and patient communities that included the International Congress of Parkinson’s Disease and Movement Disorders, or MDS, and the EHDN Clinical Research Congress. These meetings came shortly after we submitted our BLA and UK MAA and shared our recent data. That gave us a timely chance to hear directly from the physicians who care for HD patients every day. Our ongoing interactions tell us that physicians and families place great weight on preserving function and independence, and that is why the total functional capacity data Waleed described resonates so strongly.
We expect uptake to be shaped mainly by treatment center capacity in the early launch period, not by demand. That’s why center readiness is our top operational priority and preparing to allow them to be operational as quickly as possible after a potential approval. Moving on to identifying treatment centers, AMT130 is a one‑time administered therapy through a neurosurgical procedure. Each treatment center therefore needs three capabilities working together: HD neurology expertise to identify and evaluate patients, neurosurgical teams experienced in image‑guided delivery, and the ancillary infrastructure to support the procedure and product handling. We have identified priority centers in the US and the UK. Our readiness work at these centers covers three key areas: first, neurosurgical training and procedural support; second, product handling; and third, patient evaluation and referral pathways. Our goal is for a meaningful number of centers to be ready to treat eligible patients soon after a potential approval.
Finally, access and our ongoing work with payers. We have been engaging with access stakeholders through appropriate pre‑approval information exchange. These initial conversations cover the burden of Huntington’s disease, the clinical data package, and the economic profile of a one‑time therapy. From early discussions, payers recognize the high unmet need in HD and the lack of any approved disease‑modifying therapies, and there is a strong interest in learning more about AMT130.
In the UK, we have begun engaging with NICE and NHS England and are planning for health technology assessments so that access can follow as closely as possible after any potential MHRA decision. Finally, we continue to build the team that will make this launch a success. We expect to have our core team fully in place by early next year ahead of the PDUFA date. We’re hiring with care, bringing in people with deep experience in rare disease, gene therapy, and neurology launches. To summarise, we are preparing with urgency and discipline. Our goal is that if AMT130 is approved, patients can move from referral to treatment as efficiently as possible.
With that, I’ll turn the call over to Christian for a financial update. Christian?
Christian Klemt (Chief Financial Officer)
Thank you, Kylie. I’ll now be sharing the financial highlights of the third quarter of 2026. Please refer to the earnings press release issued this morning and our quarterly filings with the SEC for additional detail. Revenue for the three months ended September 30, 2026 was $5.2 million, compared to $3.7 million in the same period in 2025. The increase of $1.5 million is due to an increase in license revenue compared to the prior period. Research and development expenses were $35.5 million for the three months ended September 30, 2026, compared to $34.4 million during the same period in 2025. The $1.1 million increase was primarily driven by a $3.6 million increase in employee and contractor‑related expenses, including share‑based compensation, which was partially offset by a $2.3 million decrease in direct research and development expenses.
The decrease in direct research and development expenses resulted from a $4.1 million reduction in process validation expenses related to AMT130, a $1.5 million increase in expenses related to AMT260, and a $1.7 million increase in expenses related to preclinical activities and technology innovation projects. Selling, general and administrative expenses were $23.9 million for the three months ended September 30, 2026, compared to $19.4 million during the same period in 2025. The $4.5 million increase was primarily related to a $3.0 million increase in employee and contractor‑related expenses, including share‑based compensation, mainly as a result of the recruitment efforts over the last 12 to 15 months, a $0.7 million increase in professional fees, and a $0.7 million increase in information technology costs to support the potential commercial launches of AMT130.
Cash, cash equivalents and investment securities totaled $768.4 million as of September 30, 2026, compared to $622.5 million as of December 31, 2025. We believe that uniQure continues to be well positioned to execute on its clinical and operational priorities and expect that cash, cash equivalents and investment securities will be sufficient to fund operations into 2030. I now turn the call back over to Matt.
Matthew Kapusta (Chief Executive Officer)
Thank you, Christian. As you heard today, the third quarter of 2026 was an important one for uniQure and for people impacted by Huntington’s disease. We believe that AMT130 has demonstrated a meaningful slowing of disease progression and has the potential to provide treatment benefit for people living with Huntington’s disease. We now look forward to working with the regulatory agencies to bring AMT130 to patients as soon as possible. With that, operator, please go ahead and open the call for questions.
Thank you.
OPERATOR
We will now begin the question‑and‑answer session. If you’re dialed in and would like to ask a question, simply press star, then the number one on your telephone keypad to raise your hand and enter the queue. Okay, so our first question comes from the line of Debjit Chattopadhyay with Guggenheim Securities. Your line is now open.
Debjit Chattopadhyay (Analyst)
Hey, good morning, and thank you for taking my questions. I have a couple. First, on AMT130, if it becomes commercially available in the US next year, how are you modeling potential dropouts from the standard‑of‑care cohort in the confirmatory study? And number two, are you evaluating any alternative delivery approaches to potentially expedite the procedure times? Thank you so much.
Matthew Kapusta (Chief Executive Officer)
Hey, Debjit, it’s Matt here. Walid, do you want to take the first question on the confirmatory study?
Walid Abi-Saab (Chief Medical Officer)
Sure, I’d love to, yeah. So, Debjit, this is going to be a global clinical study. Early on we’re going to be prioritizing recruitment in the US to get us up and running and be meaningfully recruited. But the reach will be global such that during the conduct of the study, we will go to places where we will not be having AMT130 on the market during the conduct of the study, and that will reduce this. In addition to this, we are doing a number of simulations, and we have adjusted our sample size to match this eventuality as well, as well as sort of analysis plans to deal with dropouts whenever they happen in the SoC.
I turn it over to you, Matt.
Matthew Kapusta (Chief Executive Officer)
Yeah, Debjit, on the second question, there is actually a significant amount of work that’s going on now looking at procedural adaptations. And, you know, these would be things that would potentially be implemented post‑approval, but a number of these things have the potential to meaningfully reduce procedural time and complexity.
Debjit Chattopadhyay (Analyst)
Got it, thank you. And looking forward to the outcome next year. Congrats.
Matthew Kapusta (Chief Executive Officer)
Thank you.
OPERATOR
Okay, next question comes from the line of Paul Matthews with Stifel. Your line is now open.
Matthew (Analyst at Stifel)
Hi, this is Matthew on for Paul. Thank you so much for taking our question, and congrats on the progress. I guess maybe a question for Kylie: how quickly would you anticipate patients would be able to get therapy, assuming a positive outcome from the PDUFA? And what would be the gating factor there? Would it be access? And then another quick follow‑up for the confirmatory study: would the eligibility criteria be similar to the Phase 1/2, including the stage of the disease and maybe the striatal volume requirements?
Thank you so much.
Kylie O’Keefe (Chief Customer and Strategy Officer)
Thank you very much for the first question. So with regards to what could potentially be gating factors, I think there’s two elements to this. Access is, of course, one of them, as well as ensuring that we have P&T approvals in the individual institutions to ensure that patients can get access to the therapy and that the institutions can get paid for that. However, what I would say, while these are traditional gating factors, the team is working very hard to ensure that we do as much preparation as possible ahead of a potential launch to ensure that this is streamlined in the best possible way.
So there’s a lot of work going on in all the institutions to map the P&T processes, understand how often do they meet, what are the requirements that are needed to secure approval, and ensure that we’re working with the institutions to provide as much information in advance so that it’s streamlined at the point of launch. In addition to that, as I mentioned in the prepared remarks, the team is also working with payers on educating across both the unmet need in Huntington’s disease, as well as the AMT130 data package, to ensure that we’re able to move as quickly as possible.
Walid Abi-Saab (Chief Medical Officer)
So regarding the eligibility for the confirmatory study, that will be similar to what we did for the Phase 1/2, including the striatal volume as well.
Matthew (Analyst at Stifel)
Thank you.
OPERATOR
Okay, thank you. Next question comes from the line of Joel Schwartz with Leerink Partners.
Joel Schwartz (Analyst)
Thanks. Congrats on all the progress. Given the FDA expects the confirmatory study to be well underway and potentially fully enrolled at the time of accelerated approval, and screening starts by year‑end and the decision date is about four months later on May 1, we were wondering how many patients do you expect to have randomized or dosed by then? And then, for the advisory committee, how do you keep the discussion on the 36‑month analysis, which the FDA agreed supports the BLA, rather than re‑arguing the 48‑month primary analysis?
And then any sense yet of timing or which committee? Thanks.
Matthew Kapusta (Chief Executive Officer)
Hey Joel, thanks for the questions. Walid, do you want to take them one by one?
Walid Abi‐Saab (Chief Medical Officer)
Sure. Happy to. So the FDA’s fundamental intent for the confirmatory studies based on their January 25th guidance is to make sure that they can be completed in a timely manner after approval. And we are confident we can demonstrate that. You know, we’ve been starting, we started to prepare for the study before we engaged fully with the FDA and after we aligned with them, we accelerated that. Our goal is to run the study globally, as I indicated, across the U.S., Canada, North America, Latin America and Europe. And we intend also to run it in places where there will not be access during the trial so that there will be lower risk of dropout, which is what the agency is to a great extent worried about if we cannot complete the study. So with all of these elements in place and making sure that we demonstrate to the agency the full engagement and all these sites across the world that we’ve engaged with and the momentum for enrollment, we believe that we can present a credible path that would satisfy the agency regarding the ADCOM.
And 48 versus 36 months, you know, it’s premature for us to position this. We will be ready to discuss any action, but to a great extent, that will be depending on the agency’s briefing book. As you know, in preparation for the outcome, we will be preparing for any possible scenario. And we will have to gauge this based on what our specific questions are going to be raising for the ADCOM and the briefing book supporting that. So we will get a chance to adjust in due course.
And regarding the committee, I do not have any more information on this. Definitely not from the FDA, but I would imagine it will be the typical committee that reviews cell and gene therapy and advanced therapies. Thank you.
OPERATOR
Thank you. Next question comes from the line of Luca Issey with RBC Capital Markets. Your line is now open.
Luca Issey (Analyst)
Oh, great. Thanks so much for taking my question. Congrats on the progress. Maybe Waleed, can you confirm that you have yet to receive the Day 74 letter? And then maybe bigger picture, can you just maybe educate us on what is usually included in that letter? Do you think the FDA will share some of their preliminary concern in the context of the letter? I guess what I’m trying to ask here is whether there could be anything material in the letter that you need to disclose to the street as well or not.
And then maybe super quickly, you know, SG&A obviously went up a little bit versus last year. You’re obviously preparing for the launch. How should we think about the line item going forward? Maybe before and after the PDUFA? Thanks so much.
Matthew Kapusta (Chief Executive Officer)
Yeah, thanks, Luca. Look, as we have indicated, since now we are in an active review process, we will not be providing any updates, you know, based on requests for information or any other communication with the FDA. Of course, we will respond to them in due course, but unless things are material, we will not be, you know, communicating externally. In terms of usually what comes at Day 74, that would be one example. If the agency has, they list their areas of concern and areas that they’re focused on, that’s usually part of that Day 74.
Christian, you want to answer the SG&A question?
Christian Klemt (Chief Financial Officer)
Sure. Happy to. Look, as you know, we don’t give specific guidance. It shouldn’t be a surprise that SG&A spend will go up, but we’ll maintain our disciplined approach. And it’s all built into the runway guidance that we are capable of funding all our, prior to 2030.
Luca Issey (Analyst)
Got it. Thanks so much.
OPERATOR
Next question comes from the line of Jun Lee with UBS. Your line is now open.
Jun Lee (Analyst)
Hi. Thanks for taking our question. Upon approval in May, how many patients would you be ready to dose in the U.S. and the UK, depending on the approval in the UK, obviously? And what is your manufacturing capacity? And last question, in the unlikely scenario where the FDA does not end up approving AMT-130, are you still obligated to complete the confirmatory trial? Thank you.
Matthew Kapusta (Chief Executive Officer)
Kylie, do you want to answer the first question on launch?
Kylie O’Keefe (Chief Customer and Strategy Officer)
Yeah, absolutely. I think across the board, from a patient perspective, it’s a bit premature to provide specific patient estimates. I think what the team is working on extensively is ensuring that we’re able to understand what the capacity is at each of the different centers, both in the U.S. and in the UK, to understand what we think is a reasonable number of centers that need to be operationalized at launch to be able to try and meet the demand as quickly as possible.
So the team is working on that at the moment and we’re targeting a number of institutions that have both HDSA Centers of Excellence as well as ClearPoint in the same institution, to ensure that patients are able to be evaluated, move to surgery and then back to neurology very seamlessly. And that is the work that’s been ongoing. So while we’re not quite there yet to give specific patient estimates, I think a reasonable analog is other gene therapies that have launched recently.
Matthew Kapusta (Chief Executive Officer)
Yeah, and maybe I’ll answer the next two questions. Yeah. On manufacturing capacity, we do not expect that to be a constraint. You know, remember, you know, these are relatively small volumes that we’re administering in a targeted and precise way. You know, per infusion, this is approximately 500 microliters. So the yields that we get in terms of number of patients that can be treated per batch is meaningfully higher than an IV-administered medicine.
On the third question regarding the confirmatory study, you know, obviously if we’re not approved at the PDUFA date, we have to understand the reasons why and what our next steps are going to be. If you’re asking more about, you know, the obligations related to the confirmatory study, I mean, I certainly think we have a commitment to this patient community and there would be an ethical obligation to continue with that study. I don’t know if it’s a legal obligation, but we’re absolutely committed to the HD community and completing that study, whether or not the drug is approved and it’s a confirmatory study or if it’s a Phase 3 registrational study.
Thank you.
OPERATOR
Thank you. Next question comes from the line of Peyton Bonsack with TD Cowen. Your line is now open.
Peyton Bonsack (Analyst)
Hi, this is Peyton on for Jo. Thanks for taking our questions. I guess can you talk a little bit about the Phase 3 protocol or trial design and anything that’s included to decrease any variability in the TFC measure or make sure that there is the highest chance to detect the difference between the two groups? Thanks.
Matthew Kapusta (Chief Executive Officer)
Sure. I assume you meant the confirmatory study, but Wally, why don’t you answer that one?
Walid Abi‐Saab (Chief Medical Officer)
Yeah, thanks, Matt. Yeah, I assumed it was on the confirmatory study. So, look, we take, you know, very good care to try and train the sites and do regular training to make sure that the measurement is really accurate. We were collaborating with HSG where they are trying to standardize also the administration of the question for TFC. I mean, TFC tends to be a bit less noisy, I mean, you know, because this is not subjective as much, like in terms of, like, to what degree you have an abnormal movement in various parts of your body, like TMS, which has been shown to be excessively variable.
TFC talks about, like, really crude measures. Can you work full time? Can you work part time? Can you take care of yourself? And those tend to be, you know, a bit much more consistent, you know, I should say. And so we will be doing this. Was that another part of the question that I’m missing?
Matthew Kapusta (Chief Executive Officer)
I think he asked
Walid Abi‐Saab (Chief Medical Officer)
Yeah. Oh, I wanted to mention also, in powering the study, we also were careful. Of course, we don’t want to be skating too close to the edge. We base it on information that we have from our ongoing study and took the necessary precautions to make sure that we will have enough power, also considering potential dropout in the standard-of-care patients in the event that happens.
Peyton Bonsack (Analyst)
Great, thank you.
OPERATOR
Thank you. Next question comes from the line of Salvin Richter with Goldman Sachs. Your line is now open.
Salvin Richter (Analyst at Goldman Sachs)
Good morning. Thanks for taking my question. On the launch here, how many certified neurosurgical centers do you expect to be fully trained and operationally prepared to dose commercial patients at approval? And what is the target throughput capacity here? And then if you could just also speak to how you’re thinking about the label and whether it’ll reflect the broad Phase 1/2 inclusion criteria or whether it’s restricted to specific striatal volume thresholds.
Kylie O’Keefe (Chief Customer and Strategy Officer)
Absolutely. So starting with the first question on number of neurosurgical centers and capacity, I think one of the things that is important to note is neurosurgical capacity differs quite dramatically from center to center. And so again, this has been a lot of the work that the team has been doing to understand how many functional neurosurgeons would be able to perform the procedure, how many operating rooms are in place that could be used, and then calculating capacity based on those discussions with the centers.
And so you see sometimes something from two a week to one a month, and it varies center to center. So the team has been really thinking through what is the capacity at each institution. And as you look across the institutions, how does that scale up on a national level? We’re still working through the number that we think will be available at launch. We’re certainly going to be north of 15 centers. Could it be 30, could it be 25? We’re still working through that to understand what that could look like and how quickly we can operationalize these centers. But we’re looking at centers with the highest potential capacity initially and then also adding additional centers from there.
With regards to the second part of the question associated with the label, I think it’s a bit premature for us to comment on that. We’re not at the stage of discussing the label with the FDA. However, the way we’ve thought about the addressable market is assuming striatal volume requirements. And then obviously if that’s not the case, then we would adjust that accordingly.
OPERATOR
Okay, moving on to our next question comes from the line of Yananjuk with Wells Fargo. Your line is now open.
Matthew Kapusta (Chief Executive Officer)
I think we should go to the next question. Operator.
OPERATOR
Okay, thank you. Next question comes from the line of Ellie Merle with Barclays. Your line is now open.
Ellie Merle (Analyst)
Hey guys, thank you so much for taking your question. Just a follow-up to the earlier question on the number of patients that you anticipate having dosed in the confirmatory trial at the time of, like, potential approval, I guess. Do you have plans to submit additional safety data to the FDA prior to accelerated approval, after some of the patients in the confirmatory study have received the initial procedures? And then just a few follow-up questions on kind of, like, additional submissions to the FDA, I guess.
If the FDA asked for the four-year data, would that potentially warrant a major amendment as part of the review, and how you’re thinking about that as a potential scenario? Thanks so much.
Matthew Kapusta (Chief Executive Officer)
Thanks, Ellie. Wally, do you want to answer those?
Walid Abi‐Saab (Chief Medical Officer)
Sure. Regarding the number of patients, we are confident that we will be well underway by the time we reach PDUFA. And as I mentioned before, we have a lot of measures in place to be able to complete the study in a timely manner across the world, so pretty comfortable with that. In terms of the safety data, we will be providing a Day 120 safety update, which is typical in this situation. And of course, if there are any patients that have been dosed in the confirmatory trial by that cutoff date, those will be included, should there be any patient available by that time.
Regarding the four‑year data, there’s been no discussion with the FDA to date regarding the four‑year data in terms of requesting it. But as I mentioned before, if this were to happen during the course of the review, we will not be communicating on this during the process unless something is material. Now, as you mentioned, in the event they asked for it, we submit it, and they consider that a major amendment and there will be a delay to PDUFA, of course that will become material and we will be sharing that information with you.
OPERATOR
Okay, thank you. Next question comes from the line of Patrick Lucilla with H.C. Wainwright. Your line is now open.
Luis Santos (Analyst)
Hello. Hi, this is Luis Santos. Thank you for taking our questions. The question is focused on trying to understand a little bit what could happen in the UK and what steps need to happen assuming an MHRA approval for fully funded. And I would like to also ask a question on your epilepsy data coming up, and I was thinking if you could tell us a little bit more about how you expect consistency in the patients that were treated with lower dose to inform the expectations for the higher dose? Thank you.
Matthew Kapusta (Chief Executive Officer)
Thanks for the questions. Do you want to answer the first question, Kylie, on the UK?
Kylie O’Keefe (Chief Customer and Strategy Officer)
Absolutely. Thanks, Matt. So with regards to your question around what happens from an access point of view to ensure that AMT‑130 can be available for patients in the UK, what would happen is we would undergo a NICE review and from there, assuming success, we would begin NHS commissioning—NHS England, NHS Wales, and so on and so forth. We would be commissioning with each of the individual countries in the UK. So what we have done is begun engaging from an early scientific point of view with both NICE and NHS England to ensure that we’re able to understand how they’re viewing Huntington’s disease, how they’re viewing the AMT‑130 package, and then ultimately we’ll be in a position next year to be submitting the dossier to ensure that we’re starting that process immediately upon approval.
Walid Abi‐Saab (Chief Medical Officer)
So for the AMT‑260 question on MTLE, the phase 1 studies are there to learn. With those six patients in the low‑dose cohort we had great results in probably half the patients, and the others did not respond as well. We continue to follow them and we will provide you also an update during the first half of next year on those patients. In addition, we now fully dosed seven more patients in the higher‑dose cohort and depending on the results, we will be able to interpret the data. One possibility, if we find very consistently positive results, then there would be one interpretation. If that’s not the case, then we need to figure out what would be the next step. So those would be part of the evaluation that we will do and we will be sharing that information with you in the first half of next year.
Another element we do in the phase 1 study is to look at safety, and in the first cohort that profile was fairly good at this stage of development. So we were encouraged by that and that allowed us to go to the higher‑dose cohort. And again, we will be sharing with you the results in the first half of the year.
Luis Santos (Analyst)
Thank you so much.
OPERATOR
Thank you. Next question comes from the line of Rudy Lee with Wolfe Research. Your line is now open.
Devin (Analyst at Wolfe Research)
Hi, this is Devin on for Rudy. Just had a question on AMT‑260. Can you talk about the differentiation versus Neurona NRTX‑1001, and what is the bar for a no‑go versus a go decision for the ongoing study? Thank you.
Matthew Kapusta (Chief Executive Officer)
Wally, do you want to take that?
Walid Abi‐Saab (Chief Medical Officer)
Sure. Look, again, we’re early in development, so our goal right now is to evaluate the risk‑benefit of this product and see whether there’s clear evidence of activity. Our goal in the end would be to be best in class, but that will be determined after an appropriately well‑controlled trial that we can do. At this point we’re still in the signal detection mode and, as always, we are very careful in making go/no‑go decisions. So we’re still gathering that information. We will share with you an update early next year, and based on that we will tell you which direction, based on these data, we will be going next. Thank you.
OPERATOR
Next question comes from the line of Lachlan Hanbury Brown with William Blair, Chicago.
Lachlan Hanbury Brown (Analyst at William Blair)
Hey, guys, thanks for taking the question. I wanted to follow up on one of the prior questions about the confirmatory trial. You mentioned FDA’s guidance about having to be sort of well underway at the time of accelerated approval, but I feel like we’ve kind of seen them apply that standard differently across programs. So I was just wondering if you had any sort of explicit discussions with them on how they’re thinking about that standard and what well underway means in this instance.
Matthew Kapusta (Chief Executive Officer)
Yeah, I think I’ll maybe just—it’s really the same answer to the question that Waleed has mentioned previously. The FDA does have wide discretion to determine what is meaningfully underway. The spirit of this, which applies to really all accelerated approvals, is that they want to ensure that the execution of confirmatory evidence generation is done in a timely manner. They obviously are aware that we are starting the confirmatory study. We’ve shared with them approximate timelines as part of our communication. The progress that we’re making in getting the study up and running is accelerating. We will have an initial focus on enrolling patients in the United States in centers that are already up and running, and we plan to have numerous centers outside the United States, assuming the product is approved. So we’re very confident and very committed to this study, that we can complete it in a timely manner and then generate the data that is sufficient to support full approval.
OPERATOR
Thank you. Now, our final question comes from the line of Sushaila Hernandez with VLK. Your line is now open.
Sushaila Hernandez (Analyst)
Yes, thank you for taking my questions. So, for the confirmatory study, could you expand on the primary analysis being assessed at 36 months? What’s supporting this time point? And secondly, how many U.S. centers do you expect could administer AMT‑130 in the first 12 months after approval? How do you look at this development during the initial launch period? Thank you.
Matthew Kapusta (Chief Executive Officer)
Wally, you want to take the first one, and then Kylie, you can take the second?
Walid Abi‐Saab (Chief Medical Officer)
Sure, yeah. The primary analysis is essentially at 36 months TFC, comparing our treated subjects to the standard of care control. Not sure if there’s any need to elaborate more. This would be an MMRM analysis, which is typical of these types of studies. Kylie, great.
Kylie O’Keefe (Chief Customer and Strategy Officer)
Thanks, Waleed. And with the second question, across the U.S. there’s more than 60 HDSA Centers of Excellence. And then when you layer over the top of that centers that also have ClearPoint capabilities, you get to north of 100. This is obviously not what we’re targeting in the first 12 months, and as I said earlier that number has not been locked down yet. We’re working through understanding the capacity at each of the different institutions in the U.S. and then trying to look at demand in the first 12 months and saying what do we think is needed to be ready to go on day one, and then ultimately how do we build that up over the first six months, 12 months, 18 months, and so on.
So we’re going to be having a certain amount of capacity at launch, which, as I said, is going to be north of 15 centers. Whether it’s 25, whether it’s 30—that number has not been defined yet—and then we will build from there.
Sushaila Hernandez (Analyst)
That’s clear. Thank you.
OPERATOR
Thank you. This concludes the question-and-answer session and our call today. Thank you all for joining. You may now disconnect.
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