uniQure (QURE) 2026 年第三季財報電話會議：AMT-130 優先審查與 2030 年現金可用期

重點摘要

• 美國 FDA 已受理 uniQure 的 AMT-130 生物製品許可申請 (BLA) 並給予優先審查，PDUFA 目標完成日期為 2027 年 5 月 1 日。英國 MHRA 也已通過其上市許可申請的驗證。
• 在 36 個月時，與傾向評分配對的外部對照組相比，高劑量 AMT-130 在 cUHDRS 上延緩了 75% 的亨丁頓舞蹈症病程進展，在 TFC 上延緩了 60%。兩者結果均具備統計顯著性。
• 預計進行的確認性研究將招募約 200 名患者，以 2:1 的比例隨機分配至高劑量 AMT-130 組與標準治療組。主要終點為 36 個月時的 TFC，預計患者篩選將於 2026 年底前啟動。
• 受惠於授權收入增加，2026 年第三季營收年增 150 萬美元至 520 萬美元。研發 (R&D) 費用為 3,550 萬美元，而銷售及管理 (SG&A) 費用因商業化準備擴展而增加至 2,390 萬美元。
• 截至 2026 年 9 月 30 日，現金、現金等價物與投資證券總計 7.684 億美元。管理層預期這些資金足以支持營運至 2030 年。
• 針對潛在的美國上市規劃，uniQure 預計初期將有超過 15 家治療中心準備就緒，最終數量可能達到 25 至 30 家。管理層預估早期普及率將主要受限於治療中心的容量，而非需求不足。

關鍵財務數據

研發成本中包含 AMT-130 製程驗證費用減少 410 萬美元，但部分被 AMT-260 費用增加 150 萬美元以及前臨床與技術創新活動費用增加 170 萬美元所抵銷。

業務與營運表現

AMT-130 監管與臨床進展

AMT-130 的美國 BLA 正處於積極的優先審查階段。FDA 已初步表示可能會在諮詢委員會會議上討論該申請，但會議時間安排仍由 FDA 裁量。

監管申報資料係基於 AMT-130 的 36 個月分析數據。在整個 I/II 期研究中，高劑量組患者的年均 TFC 下降程度低於 0.1 分，且該效果在治療後維持達四年。管理層承認該數據集涉及的患者人數相對較少。

uniQure 已就確認性試驗的關鍵要素與 FDA 達成共識。這項全球研究將涵蓋北美、歐洲和拉丁美洲的試驗中心。在 36 個月後，接受標準治療的受試者將可透過另一項開放標籤研究獲得 AMT-130 治療。

商業化準備

公司正因應潛在的 2027 年美國上市規劃，組建醫療、市場准入、戰略客戶及外勤團隊。其治療中心策略聚焦於兼具亨丁頓舞蹈症專業知識、影像引導神經外科手術能力，以及產品處理與後續追蹤所需基礎設施的醫療機構。

管理層表示，各中心處置能力差異極大，從每週約兩次手術到每月一次不等。機構核准、付費方准入（保險給付）及中心準備狀況均可能影響初期治療推進的速度。

在英國，uniQure 已開始與 NICE 及 NHS England 展開接洽。在獲得 MHRA 的潛在批准後，醫療給付需經由 NICE 審查並在英國相關醫療體系內完成委任流程。

其他研發管線計畫

用於治療難治性內側顳葉癲癇的 AMT-260 I/IIa 期研究中，包含 7 名患者的高劑量佇列已完成招募。管理層預計在 2027 年上半年公布兩個劑量佇列的最新結果。在包含 6 名患者的低劑量佇列中，約有一半展現強勁療效，而其餘患者的反應較不理想。

用於法布瑞氏症的 AMT-191 研究中，中劑量與高劑量佇列的招募已於 8 月恢復。先前因出現無症狀肝酵素升高而依據試驗方案暫停給藥，該狀況在接受免疫抑制治療後已完全消退。預計將於 2027 年上半年公布所有三個劑量佇列的最新數據。

管理層指引

• 預計現金、現金等價物及投資證券將支持營運至 2030 年，包含臨床計畫及 AMT-130 的上市準備。
• 隨著公司打造商業化能力，銷售及管理支出預計將增加，但管理層並未提供具體的費用指引。
• 核心 AMT-130 上市團隊預計將在 2027 年初準備就緒，早於 2027 年 5 月 1 日的 PDUFA 目標日期。
• AMT-260 及 AMT-191 的最新臨床數據預計將於 2027 年上半年公布。

風險與關注事項

• FDA 可能會召開諮詢委員會來審查 AMT-130。委員會成員、時間安排及討論議題尚未確定。
• 如果 FDA 將要求提供 AMT-130 四年數據歸類為重大修正，可能會推遲 PDUFA 日期。管理層表示，截至電話會議舉行時，該機構尚未要求提供該等數據。
• 商業化上市可能會增加確認性研究中標準治療組的脫落風險。uniQure 計劃開展全球招募，包括在試驗期間 AMT-130 可能尚未商業化上市的地區，並已針對潛在的人數流失調整了樣本量與分析計畫。
• AMT-130 的最終標籤說明書仍有待 FDA 審查。管理層目前對可服務市場的規劃係以紋狀體體積要求為前提。
• 早期商業化吞吐量（治療量）可能會受到神經外科手術能力、醫療機構核准、保險給付以及治療中心投入營運所需時間的限制。

分析師問答亮點

• 確認性研究執行：管理層未提供 PDUFA 日期前患者招募的具體目標，但表示屆時研究應已順利開展。公司初期將優先在美國招募，之後再擴展至全球各試驗中心。
• 研究設計與差異性：合格標準預計將與 I/II 期計畫相似，包含紋狀體體積要求。試驗中心培訓以及與亨丁頓研究小組 (Huntington Study Group) 的合作旨在使 TFC 評估標準化。
• 製造生產：管理層預計產能不會限制上市推進。AMT-130 每劑輸注約使用 500 微升，與靜脈給藥療法相比，每批次可治療更多患者。
• 治療流程：公司正在評估可以在獲批後縮短神經外科手術時間並降低複雜性的流程調整。
• 潛在未獲批准情況：管理層表示，即使產品未能在 PDUFA 日期前獲得批准，uniQure 仍致力於完成 AMT-130 研究；在這種情況下，該試驗可作為 III 期註冊性研究，而非上市後確認性研究。

完整法說會逐字稿

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完整財報電話會議逐字稿

管理層陳述

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. [Operator Instructions]

I would now like to turn the call over to Chiara Russo, Senior Director of Investor Relations.

Chiara Russo

Thank you. Good morning and thank you for joining us for uniQure's third quarter of 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 unicure.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'll 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

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 on 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 I/II 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. Walid will provide more detail on the confirmatory study later in the call.

Regarding commercial preparations, we are making excellent progress ahead of 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 I/IIa study of AMT-260 in refractory mesial temporal lobe epilepsy and have resumed enrollment in the Phase I/II 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 Walid to provide additional detail on AMT-130 and our pipeline. Walid?

Walid Abi-Saab

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 the 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 function 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 AdCom. The FDA indicated that they are tentatively planning to discuss our application at an AdCom. As you're aware, scheduling an AdCom is within the sole discretion of the agency.

In September 2026, we presented detailed analysis of 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 2:1 ratio of AMT-130 high dose to standard of care. It will include approximately 200 patients. The primary endpoint is TFC at 36-month. After 36-month, 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 2 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 TSC as the endpoint they find most meaningful when talking with patients.

Across our Phase I/II studies, TFC declined by less than 1/10 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 4 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 6-month data of the first low-dose cohort of 6 patients, which was presented in June, early in this quarter, we completed enrollment of the second cohort of 7 higher dose patients. We expect to present updated results from both cohorts in the Phase I/IIa 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 has been paused per protocol in these 2 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 3 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?

Kylie O'Keefe

Thanks, Walid. With a 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 AMT-130, 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 U.K. 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 Walid 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. AMT 130 is a one-time administered therapy through a neurosurgical procedure. Each treatment center, therefore, needs 3 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 U.S. and the U.K. Our readiness work at these centers covers 3 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 preapproval information exchange. These initial conversations cover the burden of Huntington's disease, the clinical data package and the economic profile of a onetime 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 AMT-130.

In the U.K., we have begun engaging with NICE 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 summarize, we are preparing with urgency and discipline. Our goal is that if AMT-130 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

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 3 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 3 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 $3.6 million increase in employee 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 AMT-130, a $1.5 million increase in expenses related to AMT-260 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 3 months ended September 30, 2026, compared to $19.4 million during the same period 2025. The $4.5 million increase was primarily related to a $3 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 AMT-130.

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. We expect that cash, cash equivalents and investment securities will be sufficient to fund operations into 2030.

I'll now turn the call back over to Matt.

Matthew Kapusta

Thank you, Christian. As you've heard today, the third quarter of 2026 was an important one for uniQure and for people impacted by Huntington's disease. We believe that AMT-130 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 AMT-130 to patients as soon as possible.

With that, operator, please go ahead and open the call for questions.

Operator

[Operator Instructions] So our first question comes from the line of Debjit Chattopadhyay with Guggenheim Securities.

分析師問答

Debjit Chattopadhyay

I have a couple. First on AMT-130, if it becomes commercially available in the U.S. 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?

Matthew Kapusta

Debjit, it's Matt here. Walid, do you want to take the first question on the confirmatory study?

Walid Abi-Saab

Sure. I'd love to. Yes. So Debjit, this is going to be a global clinical study. Early on, we're going to be prioritizing recruitment in the U.S. 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 be -- we will go to places where we will not be having AMT-130 on the market during the conduct of the study, and that will reduce this.

In addition to this, we're doing a number of simulation, and we have adjusted our sample size to match this. So 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

Yes, Debjit, on the second question, there is actually a significant amount of work that's going on now looking at procedural adaptations. And 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.

Operator

Next question comes from the line of Paul Matteis with Stifel.

Matthew Ryan Tan

This is Matthew on for Paul. Congrats on the progress. I guess maybe a question for Kylie. How quickly would you anticipate patients will be able to get therapy, assuming the 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 I/II, including the stage of the disease and maybe the striata volume requirements?

Kylie O'Keefe

Thank you very much for the first question. So with regards to what could potentially be gating factors, I think there's 2 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 AMT-130 data package to ensure that we're able to move as quick as possible post launch.

Walid Abi-Saab

So regarding the eligibility for the confirmatory study, that will be similar to the -- what we did report the Phase I/II, including the striatal volume as well.

Operator

Next question comes from the line of Joe Schwartz with Leerink Partners.

Joseph Schwartz

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 4 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 rearguing the 48-month primary analysis? And then any sense yet of timing or which committee?

Matthew Kapusta

Joe, thanks for the questions. Walid, do you want to take them one by one?

Walid Abi-Saab

Sure, happy to. So the FDA's fundamental intent for the confirmatory studies based on their January 25 guidance is that to make sure that they can be completed in a timely manner after approval. And we are confident we can demonstrate that. 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 when the agency is, to a great extent, worried about it 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 the 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-month, 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 AdCom, we are -- 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.

Operator

Next question comes from the line of Luca Issi with RBC Capital Markets.

Luca Issi

Congrats on the progress. Maybe, Walid 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 concerns 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, SG&A obviously went up a little bit versus last year, you obviously preparing for the launch. How should we think about the line item going forward maybe before and after the PDUFA?

Walid Abi-Saab

Yes. Thanks, Luca. Look, as we have indicated that since now, we are in active review process, we will not be providing any updates based on request 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 communicating externally.

In terms of usually 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.

Matthew Kapusta

Christian, do you want to answer the SG&A question?

Christian Klemt

Sure, happy to. Look, as you know, we don't give specific guidance. It shouldn't be surprised 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 products into 2030.

Operator

Next question comes from the line of Joon Lee with UBS.

Joon Lee

Upon approval in May, how many patients would you be ready to dose in the U.S. and U.K. depending on the approval in U.K., 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?

Matthew Kapusta

Kylie, do you want to answer the first question on launch?

Kylie O'Keefe

Yes, 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 U.K. 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 are not quite there yet to give specific patient estimates, I think a reasonable analog is other gene therapies that have launched recently.

Matthew Kapusta

Yes. And maybe I'll answer the next 2 questions. Yes, on manufacturing capacity, we do not expect that to be a constraint. Remember, these are relatively small volumes that we're administering in a targeted and precise way 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, 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 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 III registrational study.

Operator

Next question comes from the line of Peyton Bohnsack with TD Cowen.

John Peyton Bohnsack

This is Peyton on for Joe. I guess, can you talk a little bit about the Phase III 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 2 groups?

Matthew Kapusta

Sure. I assume you meant the confirmatory study, but Walid, why don't you answer that one?

Walid Abi-Saab

Yes. Thanks, Matt. Yes, I assume it was on the confirmatory study. So look, we take very good care to try and train the sites and do regular training to make sure that the measurement is really accurate. We're 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, because this is not like subjective as much 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 a bit much more consistent, I should say. And so we will be doing -- was there another part of the question that I'm missing?

Matthew Kapusta

No. I think he asked about...

Walid Abi-Saab

[indiscernible] Yes. And 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.

Operator

Next question comes from the line of Salveen Richter with Goldman Sachs.

Salveen Richter

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 maybe you could just also speak to how you're thinking about the label and whether it will reflect the broad Phase I/II inclusion criteria or whether it's restricted to specific striatal volume thresholds?

Kylie O'Keefe

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 2 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 stratal volume requirements. And then obviously, if that's not the case, then we would adjust that accordingly.

Operator

Moving on to our next question. It comes from the line of Yanan Zhu with Wells Fargo.

Matthew Kapusta

I think we should go to the next question, operator.

Operator

Next question comes from the line Ellie Merle with Barclays.

Eliana Merle

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 follow-up question on kind of like additional submissions to the FDA. I guess, if the FDA asked for the 4-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?

Matthew Kapusta

Thanks, Ellie. Walid, do you want to answer those?

Walid Abi-Saab

Sure. Regarding the number of patients, we're -- 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's any patients that have been dosed in the confirmatory trial by that cutoff date, those would be included, should be any patient available by that time.

Regarding the 4-year data, there's been no discussion with the FDA to date regarding the 4-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 because during the process, unless something is material, we will not be communicating on this. Now as you mentioned, in the event they ask 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

Our next question comes from the line of Patrick Trucchio with H.C. Wainwright.

Luis Santos

Luis Santos in for Patrick. The question is focused on trying to understand a little bit what could happen in the U.K. and what steps need to happen assuming MHRA approval for fully funded?

And then 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 do you expect consistency in the patients that were treated with lower dose to inform the expectations for the higher dose?

Matthew Kapusta

Thanks for the questions. Do you want to answer the first question, Kylie, on the U.K.?

Kylie O'Keefe

Absolutely. Thanks, Matt. So with regard to your question around what happens from an access point of view to ensure that AMT-130 can be available for patients in the U.K. 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 U.K.

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

So for the AMT-260 question in MTLE, the really Phase I studies are there to learn. We dosed 6 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 7 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 I 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.

Operator

Next question comes from the line of Rudy Li with Wolfe Research.

Unknown Analyst

This is Devin on for Rudy. Just had a question on AMT-260. Can you talk about the differentiation versus Neurona and RTX-1001? And what is the bar for a no-go versus a go decision for the ongoing study?

Matthew Kapusta

Walid, do you want to take that?

Walid Abi-Saab

Sure. Well, again, we're early in development. So our goal right now is to evaluate the risk benefit and -- of this product and see whether there's clear evidence of activity. Our goal in the end would be to be best-in-class, and that will determine -- but that will be determined after an appropriately well-controlled trial that we can do. At this point, we are still in the signal detection mode. And as always, we are very careful in making a go/no-go decision. 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.

Operator

Next question comes from the line of Lachlan Hanbury-Brown with William Blair Chicago.

Lachlan Hanbury-Brown

I wanted to follow up on one of the prior questions about the confirmatory trial. You mentioned FDA guidance about having to be sort of well underway at the time of accelerated approval. But I feel like we've kind of seen 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 stand and what well underway means in this instance.

Matthew Kapusta

Yes. I think I'll maybe just -- I think it's really the same answer to the question that Walid has mentioned previously. The FDA does have a 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.

We -- they obviously are aware that we just are starting the confirmatory study. We've shared with them approximate time lines 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 and 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 the study that we can complete it in a timely manner and then generate the data that is sufficient to support full approval.

Operator

Now our final question comes from the line of Sushila Hernandez with VLK.

Sushila Hernandez

So for the confirmatory study, could you expand on the primary analysis being assessed at 36 months, what is supporting this time point?

And secondly, how many U.S. centers do you expect could administrate AMT-130 in the first 12 months after approval? How do you look at this development during the initial launch period?

Matthew Kapusta

Walid, do you want to take the first one? And then, Kylie, you can take the second?

Walid Abi-Saab

Sure. Yes. 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?

Kylie O'Keefe

Thanks, Walid. 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 1? And then ultimately, how do we build that up over the first 6 months, 12 months, 18 months and so on.

So we're going to be having a certain number 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.

Operator

So this concludes the question-and-answer session and our call today. Thank you all for joining. You may now disconnect.