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Date

March 2, 2026, 8:30 a.m. ET

Call participants

  • Chief Executive Officer — Matthew Kapusta
  • Chief Financial Officer — Christian Klemt
  • Chief Medical Officer — Walid Abi-Saab
  • Chief Commercial Officer — Kylie O'Keefe

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Takeaways

  • Revenue -- $16.1 million, a $11.0 million decrease attributed mainly to a $10.7 million reduction in collaboration revenue and a $6.1 million decline in contract manufacturing revenues, partially offset by a $5.8 million increase in license revenue.
  • Cost of contract manufacturing revenue -- $0, following the 2024 Lexington facility divestiture; prior year cost was $17.1 million, now recorded net within other expenses.
  • Research and development (R&D) expenses -- $140.7 million, down $3.1 million from the prior year, with $26.0 million less in other R&D expenses largely due to $25.0 million of lower employee, contractor, severance, and facility costs related to restructuring and the Lexington divestment, offset by a $22.9 million increase in direct R&D, including $19.4 million for AMT-130 regulatory preparation.
  • Selling, general, and administrative (SG&A) expenses -- $65.5 million, reflecting a $12.8 million increase driven by $9.4 million higher professional fees, including $6.5 million for AMT-130 U.S. commercialization activities, a $3.6 million rise in employee and contractor costs, and a $2.8 million uptick in other expenses, offset by an aggregate $3.0 million reduction in share-based compensation and severance.
  • Cash, cash equivalents, and investment securities -- $622.5 million at year-end, up from $367.5 million, primarily from $404.2 million raised via public offerings of ordinary shares and prefunded warrants.
  • Cash runway -- Management projects adequate capital resources to fund operations into 2029.
  • AMT-130 regulatory status -- Company is preparing protocol amendments for a four-year data analysis to be submitted to the FDA, with plans to present data from 12 patients at four years as well as all eligible patients with three-year follow-up.
  • Phase 3 trial requirements -- FDA has strongly recommended a double-blind, sham-controlled trial that management acknowledges must be "adequately powered," with ongoing dialogue about flexible approaches using extensive natural history data from the Enroll-HD database.
  • Clinical safety -- No disease-related clinical safety events have been observed with AMT-130 since December 2022, though procedure-related safety events have occurred; "Those are not associated with any clinical consequences as we have seen."
  • Epilepsy program (AMT-260) -- An update in Q2 is expected to present six-month data from the first six patients in the initial dose cohort, with data focused on seizure frequency, safety, and pharmacodynamic effects.
  • Ex-U.S. regulatory strategy -- Management is actively pursuing discussions with the MHRA in the UK and the EMA in Europe regarding named-patient and early access programs, while evaluating additional opportunities in other jurisdictions.
  • Patient engagement and advocacy -- "a tremendous amount of advocacy on behalf of the patient community." and increased engagement from sites following data publication in September have been observed, supporting future trial enrollment efforts.
  • Partnering and commercialization -- No immediate partnering decisions for AMT-130 will be made until the Phase 3 design, timeline, and investment are better defined; the company's longstanding intent is to commercialize independently in the U.S.
  • Potential accelerated approval -- The possibility of using interim analysis of surrogate endpoints such as NfL for an accelerated path was verbally communicated previously by the FDA and could remain an option pending Phase 3 design discussions.

Summary

uniQure (QURE 3.53%) reported a significant reduction in annual revenue and higher SG&A expense attributed to strategic investments, while strengthening its cash position through successful capital raises supporting a multiyear operating runway. The company confirmed a revised regulatory plan for AMT-130, including protocol amendments for comprehensive four-year durability data and commitment to ongoing FDA discussions regarding trial design flexibility. Developments outside the U.S. include advancing AMT-130 and other pipeline assets through planned regulatory interactions with European agencies, while the epilepsy clinical program prepares for upcoming initial data disclosure. The dialogue with regulatory bodies centers on the necessity, feasibility, and ethics of sham-controlled trials versus leveraging robust natural history data, directly impacting the prospective path and timeline toward potential product approval. Management's responses reinforce focus on patient engagement, cross-border access strategies, and fiscal discipline as they navigate late-stage development challenges in a competitive gene therapy environment.

  • Klemt stated, "We expect cash, cash equivalents, and investment securities will be sufficient to fund operations into 2029," clarifying capital allocation horizon and signaling balance sheet stability.
  • Abi-Saab disclosed, "presenting the data of the 12 patients at four years, but also all the patients who by then would have reached three years as well," emphasizing data completeness in future regulatory filings.
  • Kapusta noted that for ex-U.S. registrations, "there seems to be real interest from regulatory authorities," supporting potential international expansion without relying solely on U.S. pathways.
  • Management described extensive use of Enroll-HD data, exceeding 30,000 participants, as a strategic asset for trial control methodology, contrasting the regulatory preference for sham-controlled studies.
  • Plans call for a Type B meeting with the FDA in the second quarter specifically to discuss Phase 3 trial design, not to present additional efficacy or biomarker analyses prior to protocol updates.

Industry glossary

  • Enroll-HD: A global clinical research platform and observational study in Huntington's disease tracking 30,000+ participants, serving as a natural history and trial control data resource.
  • BLA: Biologics License Application; the regulatory submission to the FDA seeking authorization to market a biologic product in the United States.
  • MHRA: Medicines and Healthcare products Regulatory Agency; the UK’s regulatory authority for medicines and devices.
  • EMA: European Medicines Agency; responsible for the evaluation and supervision of medicinal products in the European Union.
  • NfL: Neurofilament light chain; a biomarker of neuronal injury often used in neurodegenerative disease clinical trials.
  • Sham-controlled trial: A study design where the control group receives a simulated medical intervention, mimicking the active procedure without delivering the experimental therapy, to measure true treatment effects while minimizing bias.
  • SAP: Statistical Analysis Plan; a document specifying statistical methodologies and data handling for clinical trial analyses.

Full Conference Call Transcript

Kylie O'Keefe: Load epilepsy, where a substantial proportion of patients remain drug resistant despite multiple anti-seizure medications, and continue to face ongoing unpredictable seizures that drive injury risk, cognitive decline, psychiatric comorbidities, and reduced quality of life. Even with surgical resection or neuromodulation, many patients are not eligible or fail to achieve durable seizure reduction, underscoring the need for innovative disease-modifying approaches that can address the underlying epileptic genetic focus and provide sustained benefit from a one-time intervention. Similarly, for AMT-191 in Fabry disease, a one-time gene therapy has the potential to address the underlying enzyme deficiency and meaningfully reduce lifelong treatment burden, positioning it to compete in a market currently defined by chronic enzyme replacement therapy and other long-term therapies.

Importantly, enzyme replacement therapies require regular lifelong infusions, may be associated with infusion-related reactions and antidrug antibodies, and often provide incomplete tissue penetration, highlighting the potential advantage of a durable one-time genetic approach. Overall, our customer-facing team remains intensely focused on disciplined execution today, while thoughtfully building the capabilities, partnerships, and evidence base required to drive long-term success across our full portfolio. I will now turn the call over to Christian Klemt for a financial update. Christian, thank you.

Christian Klemt: I will be sharing the financial highlights of the full year of 2025. Please refer to the earnings press release issued this morning and our quarterly filing with the SEC for additional details. Revenue for the year ended 12/31/2025 was $16.1 million compared to $27.1 million in 2024. The decrease of $11.0 million was primarily driven by a $10.7 million decrease in collaboration revenue and a $6.1 million decrease in contract manufacturing revenues, offset by a $5.8 million increase in license revenues. Cost of contract manufacturing revenues was nil for the year ended 12/31/2025 compared to $17.1 million in 2024.

Following the divestment of the Lexington facility in 2024, cost of contract manufacturing revenues are recorded net of associated revenue within other expenses. Research and development expenses were $140.7 million for the year ended 12/31/2025, compared to $143.8 million in 2024. The decrease of $3.1 million was primarily driven by a $26.0 million decrease in total other research and development expenses, $25.0 million of which related to decreases in employee, contractor-related, and severance costs, as well as facility costs resulting from the 2024 divestiture of the company’s Lexington manufacturing operation and organizational restructuring in the same year.

This was offset by a $22.9 million increase in total direct research and development expenses, of which $19.4 million related to the preparation of a potential BLA submission for AMT-130. Selling, general, and administrative expenses were $65.5 million for the year ended 12/31/2025, compared to $52.7 million in 2024. The $12.8 million increase was primarily driven by a $9.4 million increase in professional fees, including $6.5 million incurred to support preparation of the planned commercialization of AMT-130 in the United States, as well as a $3.6 million increase in employee and contractor-related expenses, and a $2.8 million increase in other expenses.

This was offset by a $1.8 million decrease in share-based compensation expenses and a $1.2 million decrease in severance costs. Cash, cash equivalents, and investment securities totaled $622.5 million as of 12/31/2025, compared with $367.5 million as of 12/31/2024. The net increase was primarily attributable to proceeds of approximately $404.2 million raised with public offerings of ordinary shares and prefunded warrants. With this strong balance sheet, we believe uniQure N.V. is well positioned to execute clinical and operational priorities throughout the coming year. We expect cash, cash equivalents, and investment securities will be sufficient to fund operations into 2029. I will now turn the call back over to Matthew Kapusta. Thank you, Christian.

As we look ahead to 2026, our priorities are clear. We are focused on constructively engaging with regulatory authorities

Matthew Kapusta: for AMT-130 inside and outside the United States to define the most appropriate path forward, advancing our pipeline programs with discipline, and continuing to generate high-quality data across our portfolio. The strength and durability of our Huntington’s disease dataset, the progress in Fabry disease and MTLE, and our strong balance sheet position us well to execute on this strategy. Most importantly, we remain committed to the patients and families we serve. The urgency in these communities is real. We believe our gene therapy platform has the potential to meaningfully change the trajectory of devastating diseases. We look forward to updating you as we continue to advance our programs thoughtfully and responsibly.

With that, we will open the call to take questions from our research analysts. Operator, please proceed.

Operator: At this time, I would like to remind everyone in order to ask a question, press star then the number one on your telephone keypad. We request that you limit yourself to one question and one follow-up. You are welcome to requeue with additional questions. We will pause for just a moment to compile the Q&A roster. Your first question comes from the line of Paul Matteis with Stifel.

Julian (for Paul Matteis): Hi there. This is Julian on for Paul. Thanks so much for taking our question. I guess, primarily, are there any paths that you can potentially pursue in order to push your agenda beyond just the traditional FDA channels here, and, curious, what other levers you can pull to potentially garner support for registration based on either the existing data or the four-year data? And then for the four-year data, can you just confirm whether you plan on submitting that to the agency, and whether we can expect the additional handful of patients in the analysis in the three-year analysis as well, or if you just plan on sharing 12 patients’ data at four years?

Matthew Kapusta: I will maybe answer the first part and then Walid can answer the second part. The other avenues we can pursue are potentially outside the United States. Inside the United States, the avenues go through the FDA. Over the last several months we have seen a tremendous amount of advocacy on behalf of the patient community. In my view, that is a critical part of educating and informing elements outside of the FDA around the needs and the sense of urgency within the community. We have also heard from the scientific and clinical community that continue to believe that regulatory flexibility is absolutely required for genetically defined diseases like HD that are neurodegenerative and progress very slowly.

To me that is going to be an essential element of this, and then pursuing opportunities where we can bring AMT-130 to patients as soon as possible outside the United States, where there seems to be real interest from regulatory authorities. I think that is what we are going to pursue. On the four-year data, I can hand it over to Walid.

Walid Abi-Saab: On the four-year data, we informed the FDA that we will be amending the protocol, the SAP specifically, to conduct such analysis and we will submit to them as well. We did not specifically discuss with them what that would mean. We do not believe that there is any reason we have today to believe that this will change the FDA’s position regarding the Phase 1/2 trials. I need to be clear on that. Having said that, what data will we be evaluating? It would be essentially presenting the data of the 12 patients at four years, but also all the patients who by then would have reached three years as well.

We will be presenting the totality of the data. I think those data are very important for the HD community to be able to continue to demonstrate the durability of the effect as well as a potentially even more evident treatment effect of AMT-130.

Paul Matteis: Thanks very much.

Operator: Your next question comes from the line of Joe Schwartz with Leerink Partners.

Joe Schwartz: Hi. Thank you. So in last week’s CNBC interview with Dr. McCary, he seemed concerned about the morbidity associated with procedures involving burr holes, which is what you use with AMT-130. So I am just wondering, was this a major sticking point? Did it come up in the Type A meeting? Have you done everything possible to educate the FDA on that front? And what is your strategy for the Type B meeting, and outside the United States too now? Thank you.

Matthew Kapusta: We will not comment directly on what Dr. McCary said. In terms of the interaction with the FDA, they are focused on patient safety. We have, in our view, quite a strong safety profile. We have not seen disease-related clinical safety events associated with AMT-130 since December 2022. We saw some safety events that were associated with the procedure. It is a surgical procedure. We have also disclosed previously that there are some volumetric changes that are as to be expected. Those are not associated with any clinical consequences as we have seen, and we see no increases in neurofilament light that would be associated to the extent that those volumetric changes were related to accelerating atrophy.

In our recent meetings with the FDA, we had experts on the call that have talked about volumetric MRI changes. We had clinical experts that have seen patients. We have done everything we can to educate the FDA in this regard.

Walid Abi-Saab: For the Type B meeting, our main goal is going to be to discuss with the agency designs for the Phase 3 trial. We believe that we are very fortunate in this space to have a very high-grade, ongoing, contemporaneous natural history, specifically I am talking about Enroll-HD with more than 30,000 participants. This treasure trove is generously provided to us by CHDI and through the hard work of many patients and their families and the whole HD community. We think that could be leveraged to help us strengthen study designs for Phase 3 and try to avoid designs that would be difficult and challenging to the patients. We are looking forward to working with the FDA on that.

We hope that they will work with us and acknowledge the flexibility they often talk about that should be afforded to rare diseases. That is going to be the key focus of that Type B meeting in the second quarter.

Operator: Your next question comes from the line of Joseph Thome with TD Cowen. Your line is open.

Peyton (for Joseph Thome): Hi, guys. This is Peyton on for Joe. Thanks for taking our questions. Real quickly, when talking about the Phase 3 design, how quickly do you think that you would be able to enroll it? Would you be able to use an 18-month endpoint similar to what has been seen elsewhere in the space? Then, has this changed your partnering decisions as you have? Thanks.

Walid Abi-Saab: Maybe I will take the first part and then turn it over to Matt for the partnering piece. It is premature for us to talk about the logistics and how easy it will be to recruit or not because, as you heard, we have not yet defined the design. The duration is one element. The duration often depends on the sample size as well, the level of control, how we are using it, whether we are leveraging also external control using deep patient statistics or other types of techniques. It is premature to do that. Having said that, we are very comfortable with the interest of patients.

We have seen that after we published the results back in September, and that interest has increased through a lot of the great work done over the past months by our externally facing group in dealing with various sites across the United States predominantly. I am not worried right now, but we cannot give you more details until we figure out more of the design.

Matthew Kapusta: On the partnering side, I also think it is a little too early. We need to understand what the Phase 3 study design and protocol is going to be, the number of years, and the investment that is required. We have a strong balance sheet, and our strategy has been to take this forward and commercialize it ourselves. We really believe in this product. It deserves to be taken forward. It needs to be brought to patients, and we are going to do everything we possibly can to do that. If partnering plays a role in it, then we will evaluate it at that time, but it is premature to weigh in on that right now.

Joseph Thome: Great. Thank you, guys. Hey. Thank you.

Operator: Your next question comes from the line of Ellie Merle with Barclays. Your line is open.

Jasmine (for Ellie Merle): This is Jasmine on for Ellie. Thank you for taking our question. First, can you give some more color on the different scenarios for a potential Phase 3 design? What are you going to the FDA with and proposing, and has the FDA so far given any guidance on the length of the study or endpoints? Secondly, across these different scenarios, what would the cost of a Huntington’s Phase 3 program look like, and does your cash runway include this? Thanks.

Walid Abi-Saab: Thanks, Jasmine. It is premature to get into those details. Whatever I say now would likely be different after we talk with the FDA. There have been no specific discussions on the length of the trial. The FDA was clear about their strong recommendation to do a double-blind, sham-controlled trial that is adequately powered, which is the right thing to do to be able to evaluate this, and the adequately powered part is key. We need to discuss with them whether there is openness to use other designs or how we can leverage the external control. Whatever I say now is likely to change, so I would not like to go down that path. What is the other question?

Oh, for Christian regarding the runway.

Christian Klemt: Yeah. With all the uncertainties around the investments into the various late-stage opportunities we have, we have run scenario analyses. We have built in development spend, but it is too early to comment on specifically how much of that would relate to AMT-130 vis-à-vis AMT-260 or AMT-191.

Operator: Okay. Thank you. Your next question comes from the line of Suzanne Van Voorthuizen with Kempen. Your line is open.

Suzanne Van Voorthuizen: Hi, team. This is Suzanne from Kempen. Thanks for taking my question. Maybe one clarifying question on the Endicas program about other jurisdictions. What regions are you talking about, and what is the status of your discussions with regulators outside of the United States? And then I have one for the epilepsy program. Did I catch correctly that the update in Q2 will be on six patients from the first dose cohort with six months’ follow-up, or will there also be some early data from the second dose cohort? And perhaps for seizures specifically, can you give a sense of what reduction in the seizure frequency we should consider as a good or what level would be a great result?

Thank you.

Kylie O'Keefe: Absolutely. On the first question around ex-U.S., we are looking at a number of different jurisdictions at the moment. We are taking into consideration a number of factors. We are looking at epidemiology, regulatory pathways, and pricing and reimbursement, looking, as I mentioned, at named-patient programs and early access programs that are applicable to rare diseases, and taking that into assessment as we think about the strategy moving forward for ex-U.S. regions. We have mentioned that we are going to be in discussions with both MHRA in the UK and EMA from a European perspective, and then we are going to be looking at what other opportunities that affords us outside those two jurisdictions.

That is on the first question, and then handing over to Walid on the epilepsy question.

Walid Abi-Saab: On epilepsy, we will be presenting the data on exactly what you said: the first six patients, six months, seizure frequency. This is a Phase 1 study, so we have not yet set an expectation. We are trying to figure out overall safety, tolerability, and evidence of pharmacodynamic effect. It is a learning process. More to come once we share the data.

Operator: Your next question comes from the line of Luca Issi with RBC Capital Markets. Your line is open.

Luca Issi: Great. Thanks so much for taking my question. Maybe if you can circle back with the ex-U.S. opportunity here, Matt and Kylie. How should I think about the overall commercial opportunity here? I believe Roche was able to generate close to $100 million a quarter from selling Elevidys ex-U.S. before the drug ran into safety issues. Is that the right comp for us to think about, or would you advise against us?

Kylie O'Keefe: Absolutely. It is a little premature to be talking about commercial opportunity because we are truly in the planning and strategy phase around thinking through what opportunities we would be going after. We are bringing into the thinking exactly that Roche comp around how they have gone after certain regions through named-patient and early access programs, as well as other cell and gene therapies that have walked this path ahead of us, and we will be taking those learnings on board. A bit premature on commercial opportunity, but we will be looking at Roche and other companies for best practices.

Luca Issi: Yep. Got it. Thanks so much. Got it.

Operator: Your next question comes from the line of Salveen Richter with Goldman Sachs. Good morning. Thanks for taking my questions.

Salveen Richter: Can you just help us understand the totality of the sticking points with the FDA here and why a sham-controlled is required versus a prospective natural history comparator? Thank you.

Walid Abi-Saab: Thanks, Salveen. At the pre-BLA meeting, the FDA raised the point that those studies were designed as hypothesis-generating studies and any such analyses after we collected the data and looked at them would be considered post hoc. Recently, they reverted to start looking at the double-blind part of the U.S. study, raising questions around absence of any clinical or biomarker signal in that smaller U.S. study, which was sham-controlled. We do not have the same interpretation as the FDA. In this type of rare disease where there is slow progression, and we are taking people early in their disease, it is difficult to detect a meaningful and reliable change after one year in a Phase 1/2 study such as ours.

You need to start looking at data from a subsequent time point, and with AMT-130, every time we looked at the data, the signal became more evident. We believe that this warrants evaluation compared to an external control, which is the kind of regulatory flexibility that should be afforded to diseases such as Huntington’s, which are monogenetic, progressive, and rare, and also considering the procedure we do, which is a one-time administered gene therapy. These are the things that we are going to be discussing with the FDA. We will continue that dialogue because fundamentally, AMT-130 is doing what we have been expecting it to do, and that effect continues to be stronger.

We are going to keep analyzing these data and accumulating more data, and we are hopeful that we are going to be able to align with the FDA on a study design that would allow us to confirm these findings. Then we will take this one step at a time as we start getting more clarity with them.

Operator: Your next question comes from the line of Uy Ear with Mizuho. Your line is open.

Uy Ear: Hey, guys. Thanks for taking our question. I guess I still do not quite understand why the FDA is requiring a sham study. I understand the objection the FDA had previously; it did not sound like the FDA was objecting to natural history in your Phase 1/2 study. This time around, is there anything about the natural history database that they objected to, or the kind of data or the kind of statistical plan that would be involved with using natural history that they are not comfortable with? That is the first question. The second question is, Matt, are you committed to taking this forward even with a sham study? Thanks.

Matthew Kapusta: We disclosed back in November 2024 that the FDA had stated in writing that we may use the data from the Phase 1/2 study in comparison to an external control as the primary basis for a BLA submission. I do not think they have had any criticisms of the Enroll-HD database. This is a database with more than 30,000 participants. It has been collected over the last 14 years, and it is a clinical-grade natural history. It is effectively a clinical trial. Moreover, part of the comparison was contemporaneous with the patients that we enrolled. There are a lot of checkboxes there.

It is puzzling to us, other than the fact that a sham-controlled study is certainly gold-standard science, why with such a plethora and treasure trove of natural history we would not be able to leverage that in a way for a registrational pathway. That would be disappointing. With respect to your second question, I believe in my soul that AMT-130 can benefit patients with HD. Over these years, I have gotten to know these patients and their families, and I understand the urgency of this unmet need. If there is a study that we believe is feasible and ethical, we are going to do everything we can to drive AMT-130 forward.

Operator: Thanks. Your next question comes from the line of Patrick Trucchio with H.C. Wainwright. Your line is open.

Luis Santos (for Patrick Trucchio): Hi, everyone. This is Luis Santos in for Patrick. I just wanted to ask if there was anything in the FDA’s feedback that precluded a potential accelerated path for this supposed Phase 3 study, based on an interim analysis, say, of surrogates including NfL.

Walid Abi-Saab: There was no discussion on this with the FDA, but there is no reason to think that. It was verbally communicated in a previous time that this would be possible as well. I do think that could be an option if we go down that path. Let us first discuss what that Phase 3 design would look like and then what potential accelerated approval or full approval pathway that would be.

Operator: Your next question comes from the line of Kristen Kluska with Cantor. Your line is open.

Kristen Kluska: Hi, everyone. Was part of your discussions with the FDA around a lack of biomarker data, and is there going to be an expectation that you will be able to show some of this in a sham-controlled study?

Walid Abi-Saab: The FDA reverted back to looking at the 12-month data of our U.S. study because that is the only study that had the sham control in it. They raised challenges that they do not see biomarker data in that small sample size over one year. There was no specific discussion on three-year data or a requirement for what we need to show or not at this point. It is premature for me to get into that. We will provide more details on the Phase 3 and the FDA expectations after we align with them in the second quarter.

Operator: Your next question comes from the line of Yanan Zhu with Wells Fargo. Your line is open.

Kwan (for Yanan Zhu): Hi, thanks for taking our questions. This is Kwan on for Yanan. In previous questions, you mentioned that you were trying to avoid a Phase 3 design that will be too difficult or too challenging to the patient. Can you elaborate on that point? Are you talking about the length of study, and what would be considered too difficult? Is it a three-year or longer study? And I have a quick follow-up. Thank you.

Walid Abi-Saab: The concept of having a sham surgery where patients would be anesthetized for an extended period of time, 10 to 12 hours, where you have to cut through the skin and maybe superficially drill a hole on the skull without really going through the bone—these elements represent risk for these patients, especially if the length of the study is two or three years, and they would spend all this time not knowing whether they get a drug or not. Potentially, at the end of this period, they might have progressed enough that they cannot benefit from the drug or they will never get back that level of worsening.

This is where we find it difficult, particularly with the type of therapy that we provide. That is why we are keen to work with the FDA. We know that ultimately we have the same goal: we want to bring safe and effective medicine to patients. We share that, and we know that the FDA cares about patients. We just want to work with them and appeal to their flexibility to design scientifically sound studies and leverage the available data that exists now, so we can minimize the burden to the patient as much as possible.

Kwan (for Yanan Zhu): In the planned Type B meeting, is there any additional evidence that you plan to present to the FDA, or is it just a discussion on the Phase 3 design? Thank you.

Walid Abi-Saab: It would be only to discuss the Phase 3 design. We are not doing any additional analyses until we update the SAP, and the next time we share the data would be in the fourth quarter.

Yanan Zhu: Got it. Thank you for all the color. Thank you.

Operator: Next question comes from the line of Rudi Lee with Wolfe Research. Your line is open.

Rudi Lee: Hi. Thanks for taking my question. Just another quick follow-up to the trial design. What is the biggest pushback from the FDA? Why do they feel strongly that you need to run the sham-controlled trial? They seem open to single-arm trials for Stoke Therapeutics and Praxis. What are the key differences here? Thanks.

Matthew Kapusta: You are absolutely right. There is precedent for genetic diseases and one-time administered medicines to be approvable without doing a placebo-controlled study. There is a theoretical benefit—there is a reason why sham-controlled or placebo-controlled is gold standard, and that is because it addresses potential bias, whether that is selection bias or motivational bias. There is no disagreement that a placebo-controlled study is a higher level of robustness. In our view, it does not reflect regulatory flexibility given the urgency of unmet need here, nor does it necessarily take into consideration the tremendous amount of natural history data that can be leveraged in order to provide a very useful and meaningful comparator.

Based on our understanding, the FDA is seeking a maximum reduction of potential bias in recommending that we do a sham-controlled study.

Rudi Lee: Right. Just to be clear, if they really want a sham-controlled trial, are you still dedicated to move forward to the trial?

Matthew Kapusta: We are going to do some feasibility work. We did do a sham-controlled portion of our Phase 1/2 study; it was one year and a much smaller study. If we do our feasibility work and we think it is feasible and the patient community is supportive of it, we are seriously going to consider that. I think we need to; we have to. If this is feasible and the patient community supports it, we have a moral obligation, given the strength of our data, to continue to pursue this. I feel that very strongly. I understand these things cost money and take time, and that is something we can explore the best way to do.

I am here at this company because I want to bring therapies like AMT-130 to patient populations like Huntington’s disease patients. Given the strength of our data, this is an endeavor that we continue to be dedicated to.

Operator: Ladies and gentlemen, that concludes today’s call. Thank you for joining. Have a great day. You may now disconnect.