uniQure Q1 2026 Earnings Report

uniQure EPS Results

• Actual EPS: -$0.85
• Consensus EPS: -$0.88
• Beat/Miss: Beat by +$0.03
• One Year Ago EPS: N/A

uniQure Revenue Results

• Actual Revenue: $3.56 million
• Expected Revenue: $5.21 million
• Beat/Miss: Missed by -$1.64 million
• YoY Revenue Growth: N/A

uniQure Announcement Details

• Quarter: Q1 2026
• Date: 5/5/2026
• Time: Before Market Opens
• Conference Call Date: Tuesday, May 5, 2026
• Conference Call Time: 8:30AM ET

uniQure (NASDAQ:QURE) is a biotechnology company focused on the development and commercialization of gene therapies for patients with severe medical needs. Using its proprietary adeno‐associated viral (AAV) vector platform, the company designs single‐dose treatments aimed at addressing the underlying genetic causes of disease rather than solely managing symptoms. Its most advanced program, Hemgenix® (etranacogene dezaparvovec), received regulatory approval in the United States and Europe for adult patients with hemophilia B, marking one of the first gene therapies for a bleeding disorder to reach the market.

Beyond hemophilia B, uniQure’s pipeline includes preclinical and clinical-stage candidates targeting rare and debilitating conditions such as aromatic l-amino acid decarboxylase (AADC) deficiency, Huntington’s disease, and Parkinson’s disease. The company leverages its scalable manufacturing capabilities and in-house process development teams to optimize vector production and ensure supply chain robustness. Through strategic partnerships and collaborations, uniQure also explores ex vivo approaches for oncology and the engineering of next-generation AAV capsids to broaden its therapeutic reach.

Founded in 1998 as a spin-off from academic research in the Netherlands, uniQure has built a transatlantic presence with its corporate headquarters in Lexington, Massachusetts, and its European research and manufacturing operations in Amsterdam. This geographic footprint enables the company to engage with leading clinical centers and regulatory authorities across North America and Europe, while advancing global registration strategies for its therapies.

The company’s leadership team is led by President and Chief Executive Officer Jeroen Louwerse, who brings extensive experience in gene and cell therapy development. Under his guidance, uniQure continues to invest in research and manufacturing infrastructure and to expand its scientific collaborations. The company remains committed to delivering durable, one-time treatments that have the potential to transform the standard of care for patients with serious genetic disorders.

uniQure Q1 2026 Earnings Call Transcript

Key Takeaways

• Positive Sentiment: uniQure has a scheduled Type B meeting with the FDA later this quarter to discuss the statistical analysis plan for the 4‑year AMT‑130 data and design elements of a new study, and plans a MAA submission in the U.K. in Q3 based on 3‑year data, creating near‑term regulatory catalysts.
• Positive Sentiment: Enrollment in the Phase I/II‑A study of AMT‑260 for refractory mesial temporal lobe epilepsy is on track, with safety, tolerability and seizure‑frequency results from the first 6‑patient cohort (≥6 months) expected in Q2 and the company targeting a meaningful seizure‑reduction signal to guide dose selection.
• Neutral Sentiment: AMT‑191 (Fabry) showed dose‑dependent, durable increases in alpha‑gal A and all 11 dosed patients have discontinued enzyme replacement therapy, but dosing at mid/high cohorts was paused after two asymptomatic grade‑3 liver enzyme elevations and the program is continuing follow‑up while dosing at the low dose.
• Negative Sentiment: The company has discontinued development of AMT‑162 for SOD1‑ALS after a serious adverse event of dorsal root ganglia toxicity in one patient, and will continue safety follow‑up for the five dosed patients.
• Positive Sentiment: uniQure ended Q1 with $586.6 million in cash, equivalents and investments and states this is sufficient to fund operations into H2 2029, though management noted future prioritization will be required to preserve the runway.

Presentation

[Operator]

Thank you for standing by. My name is Liz, and I'll be your conference operator today. At this time, I would like to welcome everyone to the uniQure first quarter 2026 earnings call. All lines have been placed on mute to prevent any background noise. After the speaker's 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 now like to turn the call over to Chiara Russo, Senior Director of Investor Relations. Please go ahead.

[Chiara Russo, Senior Director of Investor Relations at uniQure]

Good morning, thank you for joining us for uniQure's first quarter of 2026 earnings call. Earlier this morning, uniQure released its financial results for the first 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 the call up for Q&A. Before we begin, please know that we will be making forward-looking statements during this investor call. All statements other than statements of historical fact are forward-looking statements.

[Chiara Russo, Senior Director of Investor Relations at uniQure]

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 new information becomes available in the future. Now, let me introduce Matt Kapusta, uniQure's CEO.

[Matt Kapusta, CEO at uniQure]

Thanks, Chiara. Good morning, thank you for joining us today. During the first quarter of 2026, uniQure remained focused on advancing AMT-130 to patients while continuing to execute across our broader pipeline. Following our Type A meeting with the FDA in January, we acknowledged the agency's feedback and remain focused on engaging constructively to find a feasible path forward. We have since been granted a Type B meeting with the FDA later this quarter, where we plan to discuss our proposed statistical analysis plan for the data expected in the third quarter and key elements of a new clinical study. In parallel, we are progressing toward a potential regulatory submission in the U.K. Following a successful pre-submission meeting with the U.K. MHRA, we are preparing to submit a marketing authorization application in the third quarter based on the three-year data.

[Matt Kapusta, CEO at uniQure]

Taken together, these efforts reflect our commitment to advancing AMT-130 globally with urgency. Beyond Huntington's disease, we continue to make progress across our pipeline. For AMT-260 in refractory mesial temporal lobe epilepsy, enrollment in our phase I/II-A study is on track, and we expect to report data from the first cohort in the second quarter. In Fabry disease, updated data from our AMT-191 program showed sustained and dose-dependent increases in alpha gal A activity, stable lyso-Gb3 levels, and the discontinuation of enzyme replacement therapy in 11 patients, supporting the potential of AMT-191 as a meaningful treatment option. Regarding AMT-162 in SOD1-ALS, we announced our decision to discontinue development following a comprehensive review of the available data, reflecting our disciplined data-driven approach to capital allocation.

[Matt Kapusta, CEO at uniQure]

Looking ahead, key milestones include our Type B FDA meeting later in the second quarter, clinical update from AMT-260 in the second quarter, the four-year AMT-130 data analysis in the third quarter, and the planned MAA submission for AMT-130 in the U.K. in the third quarter. We believe these milestones represent important opportunities to advance our programs and demonstrate the potential of our platform. In summary, we are executing with focus, advancing our lead program through important regulatory interactions and managing our strong balance sheet to support our long-term strategy. We remain committed to delivering on the promise of gene therapy for patients and creating durable value for shareholders. With that, I'll turn the call over to Walid to provide more information on the pipeline.

[Walid Abi-Saab, Chief Medical Officer at uniQure]

Thank you, Matt. Good morning and good afternoon, everyone. I'll start with AMT-130 in Huntington's disease. As Matt noted, we continue to engage with the FDA and have a Type B meeting scheduled for later in the second quarter. Our goal is to work through the key design considerations for a potential new clinical study, addressing the agency's concern for an adequate and well-controlled trial while also ensuring the approach is practical, feasible, and appropriate in a rare, slow-progressing neurodegenerative disease. Huntington's disease is supported by one of the most robust natural history resources in rare diseases. Enroll-HD includes more than 30,000 participants and provides high-quality longitudinal clinical data collected over many years through the extraordinary efforts of the Huntington's disease community.

[Walid Abi-Saab, Chief Medical Officer at uniQure]

We believe this body of real-world evidence can inform efficient and statistically rigorous study designs, and it should be considered as we evaluate with the agency the appropriate design of a one-time administered therapy. Additionally, we plan to solicit feedback on our statistical analysis plan for the phase I, II study data expected in the third quarter. Turning now to our ex-U.S. regulatory efforts, we held a successful pre-submission meeting with the U.K. MHRA earlier this quarter. Based on this interaction, we plan to submit a marketing authorization application for AMT-130 in the third quarter of this year, supported by our three-year clinical data analysis. This is an exciting potential milestone for uniQure and the Huntington's disease community as we look to bring AMT-130 to patients around the world.

[Walid Abi-Saab, Chief Medical Officer at uniQure]

We have also started engaging with regulatory authorities in Europe and are evaluating additional opportunities internationally to potentially bring AMT-130 to patients as quickly and efficiently as possible. Finally, we expect a manuscript for our complete three-year analysis to be submitted in a peer-reviewed medical journal this year. Moving on to the rest of our clinical stage pipeline, starting with AMT-260 for temporal lobe epilepsy. We continue to collect data on the fully enrolled first dose cohort, which included those with both non-dominant and dominant hemisphere MTLE, and we plan to provide an update later in the second quarter on all six treated patients with at least six months of safety, tolerability, and seizure frequency outcomes. These are expected to be presented at the Epilepsy Foundation Pipeline Conference in Leesburg, Virginia in June of this year. Turning to AMT-191 for the treatment of Fabry disease.

[Walid Abi-Saab, Chief Medical Officer at uniQure]

In February, we reported preliminary safety and exploratory efficacy data from 11 patients in the ongoing phase I/II trial of AMT-191. As of January 8th of this year, the study cutoff date, all 11 patients across three dose cohorts demonstrated elevated alpha-galactosidase A enzyme activity that was dose-dependent and durable over the observed follow-up period, ranging from more than one year in the longest follow-up patient at the high dose to four months in a patient treated at the mid dose. Stable plasma lyso-Gb3 levels were maintained post-dose across all dose cohorts regardless of enzyme replacement therapy status. As of February 18th of this year, all 11 dosed patients have discontinued ERT. On safety, as previously reported, two patients in the mid-dose cohort experienced asymptomatic grade 3 liver enzyme elevations.

[Walid Abi-Saab, Chief Medical Officer at uniQure]

These events met protocol-defined criteria for potential dose-limiting toxicity and were reviewed and confirmed as such by the independent data monitoring committee. Accordingly, dosing at the mid dose and high doses were paused per protocol. To date, no new AMT-191-related serious adverse events have been observed. The program continues to demonstrate a manageable safety profile. Lastly, there's AMT-162 for SOD1-ALS. As previously disclosed, the phase I/II EPISOD1 trial of AMT-162 for SOD1-ALS has been on voluntary recruitment pause based on an independent data monitoring committee recommendation after a serious adverse event of dorsal root ganglia toxicity in one patient in the second cohort. This event was determined to be related to AMT-162. Following review of the preliminary efficacy and safety data generated from EPISOD1, the decision was made to discontinue development of AMT-162.

[Walid Abi-Saab, Chief Medical Officer at uniQure]

We will continue to collect follow-up safety from the five patients dosed, consistent with applicable safety and regulatory requirements. Now I will turn the call over to Kylie to discuss our ongoing work with the HD community and our ex-US commercial efforts. Kylie?

[Kylie O'Keefe, Chief Customer and Strategy Officer at uniQure]

Thank you, Walid. Before I turn to AMT-130, I want to take a moment to acknowledge the Huntington's disease community, the patients, the families, and the caregivers who live with this disease every day, and the researchers, clinicians, and advocates who have spent decades refusing to accept the status quo. Their resilience and their trust in us is not something we take lightly. It is what holds us accountable to progress we are here to discuss today. We remain committed to continuing the efforts in the U.S. and globally to advance AMT-130 as responsibly and efficiently as possible. We are encouraged by the path ahead in the U.K. following our recent engagement with the MHRA and are advancing commercial preparations across several key geographies based on this progress. Our market preparation efforts have centered on three near-term priorities.

[Kylie O'Keefe, Chief Customer and Strategy Officer at uniQure]

First, ensuring treatment center capacity and readiness and working closely with the multidisciplinary care teams at the centers of excellence that will be critical to success. Secondly, in parallel, ongoing patient engagement is critical to maintain continuity across the care journey, supporting genetic testing and referral pathways. Thirdly, market access readiness is advancing, including proactive payer engagement and development of a clear evidence-based value proposition. This is underpinned by robust health economics and outcomes research, generating data to demonstrate long-term clinical benefit and broader societal impact to support pricing, access, and adoption. We believe the U.K. unlocks a meaningful opportunity for uniQure to deliver a potentially transformational therapy to patients with HD, a community with no approved disease-modifying therapies today. There are between 7,000-8,000 patients living with HD in the U.K., with approximately 30,000 at risk.

[Kylie O'Keefe, Chief Customer and Strategy Officer at uniQure]

The U.K. has world-renowned neurosurgical capabilities and several leading HD centers of excellence, which we believe will be instrumental partners in making this potential therapy available to patients. Importantly, an MHRA approval would not only enable access in the U.K. We believe it could also enable early access or name patient programs in other geographies, including the Gulf countries in the Middle East, Latin America, Commonwealth of Independent States, and Central and Eastern Europe, enabling access to therapies ahead of formal reimbursement decisions, offering hope to patients and families while local regulatory and broader market access processes continue. We believe this discipline approach helps drive building a scalable global strategy to maximize the long-term value of our program for all stakeholders. Moving to AMT-260. In mesial temporal lobe epilepsy, where many patients remain completely refractory to anti-seizure medications, cycling through treatment after treatment with no meaningful seizure control.

[Kylie O'Keefe, Chief Customer and Strategy Officer at uniQure]

For those who do progress to surgical intervention, the options currently available are at its core, a tissue-destructive procedure. We believe being able to deliver a precisely targeted gene therapy in MTLE without destroying healthy tissue may represent a new treatment paradigm. Lastly, on AMT-191. In Fabry disease patients, they face a relentless multi-system disease burden, all driven by a single genetic defect in GLA. The current standard of care, which is biweekly enzyme replacement therapy, requires lifelong intravenous infusions that are logistically burdensome and has an occurrence of high rates of neutralizing antibody development, which limits efficacy over time. A single administration therapy correcting the enzymatic deficiency at the genetic level in Fabry, we believe has the potential to meet this unmet need.

[Kylie O'Keefe, Chief Customer and Strategy Officer at uniQure]

Across our customer-facing functions, we're focused on delivering strong execution while being disciplined in scaling the infrastructure needed to support commercial activities with a focus on strengthening center of excellence relationships, refining the patient and provider journey, and continuing to build the evidence required for access and adoption. As we continue our efforts in the U.S., we're also energized by the potential opportunity ahead in the U.K. and other geographies and are advancing towards an expected MAA submission and the possibility of bringing the first potential disease-modifying treatment for this devastating disease. Now I'll turn the call over to Christian for a financial update. Christian.

[Christian Klemt, CFO at uniQure]

Thank you, Kylie. I'll be sharing the financial highlights for the first quarter of 2026. Please refer to the earnings press release issued this morning and our quarterly filing with the SEC for additional details. Revenue for the three months ended March 31st, 2026, was $3.6 million compared to $1.6 million in the same period, 2025. The increase of $2 million is due to an increase in license revenue. Research and development expenses were $29.2 million for the three months ended March 31st, 2026, compared to $36.1 million to the same period, 2025.

[Christian Klemt, CFO at uniQure]

The $6.9 million decrease was driven by a $2.6 million decrease in fair value of continued consideration, a $1.2 million decrease in costs related to external program spend, a $1.6 million decrease in employee and contractor-related expenses, including share-based compensation, and a $1.6 million decrease in facilities and other expenses compared to the prior period. Selling General Administrative Expenses were $20.1 million for the three months ended March 31, 2026, compared to $10.9 million during the same period, 2025.

[Christian Klemt, CFO at uniQure]

The $9.2 million increase was primarily related to a $5.5 million increase in employee and contractor-related expenses, including share-based compensation, mainly as a result of employees record-recruited in 2025 to support commercial planning for AMT-130, $1.8 million increase in professional fees, $0.6 million increase in intellectual property fees, and $1.3 million increase in information technology costs and other expenses compared to the prior period. Cash, cash equivalents and investment securities totaled $586.6 million as of March 31, 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 projects through 2026.

[Christian Klemt, CFO at uniQure]

We expect that cash and cash equivalents and investment securities will be sufficient to fund operations into the second half of 2029. I'll now turn the call back over to Matt.

[Matt Kapusta, CEO at uniQure]

Thank you, Christian. To summarize, our top priority remains continued engagement in the U.S. and internationally to advance a clear and viable path forward for AMT-130. In parallel, we are executing across our pipeline and maintaining a strong focus on capital allocation to support long-term value creation. With several important milestones ahead in 2026, we look forward to updating you on our progress. With that, we will open the call to take questions from our research analysts. Operator, please proceed.

[Operator]

At this time, I'd like to remind everyone in order to ask a question, press star then the number one on your telephone keypad. We will pause for just a moment to compile the Q&A roster. Your first question comes from the line of Moritz Reiterer with Guggenheim Securities. Please go ahead.

[Moritz Reiterer, Analyst at Guggenheim Securities]

Hi, this is Moritz on for David. Thanks so much for taking our question. I have two. The first one is around AMT-260.

[Moritz Reiterer, Analyst at Guggenheim Securities]

Could you just give a little bit more sort of an overview of what the expectations are for the upcoming dataset in June? I have a follow-on question about AMT-130, namely around the competitor PTC data that was published last week. We noticed that their natural history control was an order of magnitude smaller than what you used for AMT-130. Just trying to understand a little bit better what was the rationale for choosing such a large control cohort, and what are the potential upsides and downsides of that choice? Thank you.

[Walid Abi-Saab, Chief Medical Officer at uniQure]

Thank you. On AMT-260, we are conducting a phase I/II trial. As you know, the primary objective of that trial is to evaluate safety. Of course, we're looking at efficacy endpoints, you know, particularly seizure frequencies as measured by a diary in addition to a number of other endpoints. What we're looking is to identify a dose that's safe and well-tolerated, based on these data, and we expect to see a signal on a reduction of seizure frequency. You know, we're targeting at this stage a maybe perhaps a 50% reduction in seizure frequency could be a good signal for us to follow up in subsequent well-controlled studies. In terms of AMT-130, it's really very difficult to essentially compare or interpret results from competitors.

[Walid Abi-Saab, Chief Medical Officer at uniQure]

We're not in a position to do that. We don't quite know the details of the analysis plan they've done in using Enroll-HD and why the control numbers are low. I will withhold any interpretation on these data.

[Moritz Reiterer, Analyst at Guggenheim Securities]

Thank you.

[Operator]

Your next question comes from the line of Paul Matteis with Stifel. Please go ahead.

[Analyst at Stifel]

Hi, this is Emily on for Paul. We wanted to ask a little bit more about the U.K. market dynamics, maybe if you could share any color. Another question would be like, of the 7,000 to 8,000 patients in the U.K., how many of those are treated at centers of excellence currently? Thank you.

[Kylie O'Keefe, Chief Customer and Strategy Officer at uniQure]

Absolutely. Thanks, Emily, for the question. Maybe starting with the second part of the question. The vast majority of the patients that we alluded to, the 7,000-8,000, are treated at specialized centers. There is a collection of those patients that are managed by the specialized centers around the U.K., that is the vast majority. Maybe just some other color from that perspective around U.K. market dynamics. I think once we are able to secure MHRA approval, we obviously work with NICE and a number of the access bodies, including NHS England, to work through managed access agreements to be able to bring the product to market. That's work that has already started and is ongoing and will continue through a potential MHRA approval to bring this product to market.

[Analyst at Stifel]

Great. Thank you so much.

[Operator]

Your next question comes from the line of Joe Schwartz with Leerink Partners. Please go ahead.

[Joe Schwartz, Analyst at Leerink Partners]

Thanks. Congrats on your progress and persistence. I'd like to ask a question each about your ex-U.S. and U.S. aspirations. First, how aligned do you expect the U.K. and broader EMA review processes to be, and what is your assessment of your ability to receive adequate reimbursement in these territories outside the U.S., which may be more constructive on approval at this point? Second, what specific feedback from the FDA are you hoping to clarify or potentially challenge in your Type B meeting? How are you preparing your briefing package to make your case?

[Matt Kapusta, CEO at uniQure]

Okay. Do you wanna start with the first one, Kylie?

[Kylie O'Keefe, Chief Customer and Strategy Officer at uniQure]

Yeah, absolutely. Just talking through ability to secure reimbursement in markets outside of the U.S., Joe, I think the aim is to start with named patient and early access programs that give us an ability to be able to secure patients very rapidly post-approval. We will also in parallel progress with formal pricing and reimbursement negotiations. I think one of the things that we're definitely seeing is the U.K. in particular is a market at an inflection point. They've really tried to look at how to bring advanced therapies to market, and they've tried to shift their thinking. For example, bringing in the Highly Specialized Technology route and other aspects of raising the QALY and ICER considerations. This is really trying to ensure that they're bringing advanced therapies to patients in the U.K. and not being left behind.

[Kylie O'Keefe, Chief Customer and Strategy Officer at uniQure]

Outside of the U.K., we'll be taking a very specialized approach. We'll be assessing markets on a market-by-market basis, looking at funding pathways, looking at access to therapies, and ensuring we're doing this in a step-by-step approach rather than more of a simultaneous approach. That will be taking reimbursement as a primary consideration into focus.

[Joe Schwartz, Analyst at Leerink Partners]

Thanks.

[Walid Abi-Saab, Chief Medical Officer at uniQure]

This is Walid. I'll take the second question. Thanks, Joe. In terms of the meeting with the FDA, we view this as a technical meeting. Our hope is to gain some clarity on key design elements of an additional new study to evaluate the efficacy of AMT-130 and also to get feedback on the statistical analysis plan for the data.

[Joe Schwartz, Analyst at Leerink Partners]

Thank you.

[Operator]

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

[Salveen Richter, Analyst at Goldman Sachs]

Good morning. Thanks for taking my questions. Can you speak to your base case assumption for the phase III study design of AMT-130 and whether Novartis' recent study is a precedent here? Separately, just frame expectations for the four-year data in the third quarter and what sensitivity analyses these might include. Thank you.

[Walid Abi-Saab, Chief Medical Officer at uniQure]

Thanks. In terms of design elements of a new study to evaluate clinical efficacy, we can't really go into details because it depends on these discussions with the agency. We genuinely want to have a constructive discussion with the agency. Our position is that in this rare slowly progressing disease, where we have a one-time therapy administration and when there is a treasure trove of natural history data that we could use, we should be looking at potential flexibility in trying to utilize these resources to minimize the burden on the patient and make these studies rigorous but still feasible. That is truly our goal in the meeting.

[Walid Abi-Saab, Chief Medical Officer at uniQure]

Regarding the analysis of the four-year data, in broad terms, they're actually going to be generally similar to the three-year analysis, with the addition, of course, of one more year of follow-up, which will bring the total number of patients at four years to 12 at the high dose and 12 at the low dose. In addition, there will be three patients at the high dose who would have completed three years, so making 15 patients who have reached the three-year analysis at the high dose. We are discussing with the agency whether there could be additional potential analyses that they would want to see in order to increase the level of confidence.

[Walid Abi-Saab, Chief Medical Officer at uniQure]

Our expectation are that with time, treatment effects will become more and more evident, and the absolute difference between those treated with AMT-130, particularly on the high dose, is gonna become much more evident when compared to well-matched external controls.

[Operator]

Your next question comes from the line of Yanan Zhu with Wells Fargo. Please go ahead.

[Analyst at Wells Fargo]

Hi. Thanks for taking our question. This is Quan on for Yanan. Also on Huntington's disease, you mentioned that in the Type meeting you'll talk about, design of the new study and data statistical plan. Can you talk about, will you also cover the potential of an alternative regulatory path? Is there still a possibility to file without starting a new study? Thank you.

[Walid Abi-Saab, Chief Medical Officer at uniQure]

The purpose of the meeting, as I said previously, is technical in nature to discuss elements of the design for a new additional study to evaluate the efficacy and also the analysis. We do not intend to have a specific discussion about a regulatory path to filing at this point.

[Analyst at Wells Fargo]

Got it. Thank you.

[Operator]

Your next question comes from the line of Peyton Bohnsack with TD Cowen. Please go ahead.

[Peyton Bohnsack, Analyst at TD Cowen]

Hi. This is Peyton on for Joe. Thanks for taking our questions. I guess I'm kind of looking at the U.K. commercial opportunity. Can you talk about the number of centers that you've identified in the U.K. that are equipped to do the MRI-guided stereotactic surgery? Are there any changed, planned changes to the surgical procedure in a potential commercial product, specifically any changes in the length of the time of the procedure? Does anything need to be done to validate or approve the cannulas that is used? Thanks.

[Kylie O'Keefe, Chief Customer and Strategy Officer at uniQure]

Thanks, Peyton. I'll unpack. There's a few elements to answer there. Maybe just starting with the number of specialized centers in the U.K. I think probably you do know this, but we had a number of centers that were incorporated into our European clinical trial. There are a number of centers that have already treated AMT-130 patients. This is just a small handful of the number of centers that exist in the U.K. that have neurosurgical stereotactic capabilities. We have already identified a number of those and have engaged with them to start to really plan the market in the U.K. The second aspect of the question was related to changes in the procedure.

[Kylie O'Keefe, Chief Customer and Strategy Officer at uniQure]

From that perspective, we don't anticipate any changes in the procedure, transitioning from a clinical program into a commercial program. We expect it to be consistent with what was done in the clinical trials. From that perspective, no changes there. The third part of the question was whether or not we see any challenges in getting the cannula into the U.K., and no challenges there. The cannula has been shipped to a number of different countries around the world, and the U.K. is no issue there, including through clinical trials and through commercial aspects. We are not the only company that utilizes the cannula, and so there are already commercial companies that are utilizing the cannula in the U.K.

[Peyton Bohnsack, Analyst at TD Cowen]

Great. Thanks.

[Operator]

Your next question comes from the line of Luca Issi with RBC Capital Markets. Please go ahead.

[Luca Issi, Analyst at RBC Capital Markets]

Oh, great. Thanks so much for taking my questions. maybe Matt or Walid, you know, kind of bigger picture, can you just maybe compare and contrast the Type A meeting you had in January, the FDA, versus the pre-submission meeting you had with the MHRA in the U.K.? Again, appreciate that these are different jurisdictions and different regulatory bodies, but why did the U.K. found your data persuasive versus the FDA did not? Is that because they're more willing to compare data with single arm data with, like, historical control? Is that because they have a better appreciation for the unmet medical need? What's driving that dichotomy there? I think any color there much appreciated. Thanks so much.

[Matt Kapusta, CEO at uniQure]

Luca, it's obviously hard to get into the mind's eye of, you know, each of the regulatory authorities. You know, we've been saying this now for, you know, for 6+ months that we strongly believe in the strength of our data. You know, we've achieved 75% slowing of disease with high statistical significance out to three years on the composite UHDRS. We achieved statistical significance in a slowing of disease on Total Functional Capacity. We see favorable trends across other clinical measures. We see neurofilament light below baseline. There's a tremendous unmet need here where there's no disease-modifying treatments for these patients. I think, you know, based on the discussion that we had with the U.K., I think they recognized these elements. Walid, do you wanna chime in?

[Walid Abi-Saab, Chief Medical Officer at uniQure]

Yeah, I just wanna chime in one piece. I think one of the things that also might be a bit different in the U.K. now is that focus on rare disease has been a policy for the current government. I think this actually meets with a certain agreement within the overall policy of the government there and actually allows for more flexibility to be afforded in rare diseases like this. We hope that this could also be used in other countries as well, of course, in the U.S., and we continue to work constructively with the FDA to achieve that. You know, at one point, there were more openness and flexibility with us. More recently, it's been a bit more difficult.

[Walid Abi-Saab, Chief Medical Officer at uniQure]

Again, we continue to work with the FDA, and at the end of the day, the data that we're gonna be generating will help, hopefully, to get us to where we need to go.

[Luca Issi, Analyst at RBC Capital Markets]

Got it. Thanks so much, guys.

[Operator]

Your next question comes from the line of Ellie Merle with Barclays. Please go ahead.

[Ellie Merle, Analyst at Barclays]

Can you elaborate a bit on the range of outcomes for what we could learn from the Type B meeting? Then a second question, do you plan to request another meeting with the FDA after the data to pursue accelerated approval again based on the data? Thanks.

[Matt Kapusta, CEO at uniQure]

Yeah. I think at this juncture, just with the Type B meeting scheduled, we won't speculate on the range of meetings and on the range of outcomes. Then, you know, based on the discussion that we have, we'll ascertain whether there's a need for another follow-up meeting with the FDA, and we'll certainly provide that as part of our update, once we receive the minutes.

[Ellie Merle, Analyst at Barclays]

Understood. Thanks.

[Operator]

Your next question comes from the line of Suzanne van Voorthuizen with Kempen. Please go ahead.

[Suzanne van Voorthuizen, Analyst at Kempen]

For the ex-U.S. strategy, can you elaborate on your current thinking on the commercial launch in Europe beyond the U.K., assuming you're also targeting a potential EU approval? For example, for the sequential rollout, which countries are most likely first to target, and what are dynamics that you see that are similar or different from the U.S. which we should consider when launching 130 for Huntington's? A small one on your cash runway guidance into H2 2029. Can you remind us what parts of your business plan are or are not included in that guidance? Thank you.

[Walid Abi-Saab, Chief Medical Officer at uniQure]

This is Walid. I'll start with regulatory strategy beyond the U.K. and the U.S. We are evaluating, and we've actually started the process of engaging a number of regulatory authorities, including Europe and outside the U.S. We expect to have an update for you in the second half of the year. That's as far as regulatory. I'll turn it over to Kylie to talk about the commercial strategy.

[Kylie O'Keefe, Chief Customer and Strategy Officer at uniQure]

Absolutely. As mentioned, from a commercial strategy point of view, we're going to be taking each step as a sort of assessment of country by country. We're looking at countries that have named patient access and early access programs well-established, which there's a number of countries in Europe that would allow us to unlock treating patients early on in the process as we progress with formal pricing and reimbursement. Obviously, Germany has a well-established pathway with regards to free pricing in the first six months for a rare disease products. France also has the ATU program or the AAP program, which it has now evolved into. There's a number of other countries, Italy, for example, that has named patient and early access.

[Kylie O'Keefe, Chief Customer and Strategy Officer at uniQure]

We would look at this on a country-by-country basis and assess the funding pathways and the ability to bring this therapy to patients ahead of formal pricing and reimbursement and, take the steps from there.

[Christian Klemt, CFO at uniQure]

Yeah. Yeah. Quickly on the runway, it's kind of the same assumption as for a couple of cues that we complete the ongoing clinical trials for TLE, HD, and Fabry. To get into the second half of 2029 does not allow us to simultaneously take forward all the candidates in kind of the most expedited manner. There will need to be prioritization decisions if we wanna maintain the runway.

[Operator]

All right. Your next question comes from the line of Patrick Trucchio with H.C. Wainwright. Please go ahead.

[Patrick Trucchio, Analyst at H.C. Wainwright]

Thanks. Good morning. My questions are on AMT-191. I'm wondering what the gating criteria are to resume AMT-191 dosing or select a go-forward dose

[Patrick Trucchio, Analyst at H.C. Wainwright]

After the mid dose DLT. Specifically, I'm wondering what IDMC regulatory, you know, steroid prophylaxis or other follow-up criteria are necessary to move forward. Separately, now that we have all 11 AMT-191 patients off ERT, I'm wondering what duration and organ level follow-up are needed to define the next development step.

[Walid Abi-Saab, Chief Medical Officer at uniQure]

Thanks, Patrick. Great questions. The per protocol, anytime we see a grade 3 increase in the or grade 3 adverse event, that could be potentially a DLT. Per protocol, we stop dosing. There's been very close collaboration with the IDMC informing the FDA. The patients are followed very closely and treated with a steroid as well as steroid sparing therapies. One patient fully recovered. The other one is really within a very close statistically recovering. I'm very pleased with the process. Once that is done, we will submit these data to the FDA for their review and discuss resuming dosing at one of these two doses.

[Walid Abi-Saab, Chief Medical Officer at uniQure]

In the meantime, the study is ongoing, we continue to dose with our low dose of 2E13, and we continue to follow the patients. At the end of the day, this is a phase I/II trial, our goal in this trial is to be able to identify a dose that's safe and well-tolerated. We're going to be looking at the totality of the data. If we see these changes in LFTs as we have observed, to what degree we can monitor them and manage them with steroids, to what degree they're associated with any other types of, you know, data to suggest that there might be autoimmune in nature. We don't see that yet.

[Walid Abi-Saab, Chief Medical Officer at uniQure]

At the end of the day, we will pick a dose that is safe and well-tolerated based on these data, and that will generate a significant increase in alpha gal activity, especially when these patients are off steroids. We will be engaging with the FDA in the second half of the year, I should say, to better understand any potential pathway forward, specifically, essentially, a pathway that would be similar to what was afforded to Sangamo. Based on these, we will be then making a decision about next steps with this program in the, you know, next six to 12 months.

[Operator]

Your next question comes from the line of Kristen Kluska with Cantor. Please go ahead.

[Kristen Kluska, Analyst at Cantor]

Hi, good morning, everyone. Thanks for taking the questions. On AMT-130, of the 7,000-8,000 patients diagnosed today, what percent or number of them do you think would be potentially eligible for therapy at launch? Based on your timelines for submission and the fact that they really seem to be pressing with this policy for rare diseases, when would you ultimately expect an approval decision? Thank you.

[Kylie O'Keefe, Chief Customer and Strategy Officer at uniQure]

Hi, Kristen. I can take the first part of the question, and then Walid can take the second one. From a percent eligible perspective, I think it's a little bit premature for us to put a point on a specific percentage. I think we're working through that at the moment with an understanding of what we think the label could look like, because obviously that will have a key consideration of percent eligible. I think as we sort of understand the market in more detail, we'll be able to share more specifics, more to come on that.

[Walid Abi-Saab, Chief Medical Officer at uniQure]

In terms of timing, as you know, for the MHRA, the timing is not as clear as with the FDA in terms of PDUFA date. Though that depends because it's variable, that depends on how many rounds of questions you have and the clock stop, which is the time that it will take us to answer those questions. That also will depend on how many questions, how complicated they are to get answers to. We are, you know, we will be working, of course, very diligently to move this as quickly as possible. But it's very difficult to give you an exact timing because it all depends on the number, how many rounds of questions and as I mentioned.

[Operator]

Your next question comes from a line of Daniil Gataulin with Chardan. Please go ahead.

[Daniil Gataulin, Analyst at Chardan]

Yes. Hi, good morning, guys. Thank you for taking my question. Quick one, 1:30 in the U.K., what at launch, if it's approved, what would you expect the capacity to be with all the centers that can administer the procedure? The second question is, are there countries that recognize the MHRA decision for their approval, and how many patients could that potentially add?

[Kylie O'Keefe, Chief Customer and Strategy Officer at uniQure]

Great. Thank you very much for the question. On the first part, which is capacity in the U.S., I think, ex-U.S., U.K., sorry. Capacity in the U.K. I think there's a number of centers, as I mentioned earlier, that have the capabilities to be able to do this treatment procedure. I think it depends on the process that we'll be ensuing from an access point of view. There is the Innovative Medicines Fund that allows you to secure early access and early revenue, and that would unlock some patients. Obviously we would need to move through the process with NICE and ultimately NHS England to secure a formal recommendation and manage access agreement to be able to open up a larger potential pool.

[Kylie O'Keefe, Chief Customer and Strategy Officer at uniQure]

In the near term, I think the capacity is very much where it needs to be. In the longer run, we'll be able to take a deeper look at what does that capacity look like. I think similarly to the U.S., the team is starting to look at the capacity and the pull-through there. I think from where we stand today, it looks like in the near term we're in a good position, and then we'll work through what else is needed over the longer run. From countries outside of the U.K. that reference an MHRA approval, there's a number of countries that do that open up main patient and early access programs. A couple just to highlight from the Gulf countries in the Middle East, Saudi Arabia, UAE as an example.

[Kylie O'Keefe, Chief Customer and Strategy Officer at uniQure]

There's a number of countries that we're able to move forward in Latin America, in Commonwealth of Independent States, and then also in non-EU Central and Eastern Europe. That's just to give you an example of some of the markets that are unlocked through a potential MHRA approval.

[Operator]

Thank you. Again, if you would like to ask a question, press star one on your telephone keypad. Your next question comes from the line of Rudy Li with Wolfe Research. Please go ahead.

[Rudy Li, Analyst at Wolfe Research]

Thanks for taking my question. I think you mentioned that you're now planning to discuss filing at the upcoming Type B meeting. Is it fair to say that the base case scenario will be running a new pivotal trial to support filing? Secondly, what do you think is the biggest pushback from FDA regarding natural history control? 'Cause I'm still confused why they would require a sham control phase III instead of a single arm pivotal trial. Thanks.

[Matt Kapusta, CEO at uniQure]

Yeah. I think, you know, what we know is that we have the guidance that we got from the FDA, right, previously, which is, you know, their recommendation that we conduct another study. You know, that's why obviously we wanna go into the FDA and have a discussion around those design elements. As I say, you know, this upcoming meeting is an interaction with the review team. You know, there are tactical and technical matters that we wanna get through, you know, that include not only what I just described, but also a review of the statistical analysis plan. We're gonna continue to follow these patients.

[Matt Kapusta, CEO at uniQure]

As I said, you know, we believe strongly in the therapeutic potential of AMT-130 and the potential that the data will continue to demonstrate that. I think once we have the data, then we can engage with the FDA and discuss what is the appropriate path forward. In terms of your question around natural history, again, that's a question for the FDA. I think what we would say is that, you know, there's probably no indication that I'm aware of in the rare disease space that has as much natural history data available to leverage. You know, on top of that, clinical grade quality longitudinal data.

[Matt Kapusta, CEO at uniQure]

You know, to the extent that single arm studies and external control comparisons are acceptable for intractable diseases with high unmet need, we think there's a strong rationale, particularly given the slow progressing nature of HD, the one-time administrative nature of AMT-130, and the surgical delivery of the product. I think that's what we would say in that regard.

[Rudy Li, Analyst at Wolfe Research]

Very helpful. Thanks.

[Operator]

There are no further questions at this time. Ladies and gentlemen, that concludes today's call. Thank you all for joining. You may now disconnect.

Participants

Executives

• Chiara Russo, Senior Director of Investor Relations
• Christian Klemt, CFO
• Kylie O'Keefe, Chief Customer and Strategy Officer
• Matt Kapusta, CEO
• Walid Abi-Saab, Chief Medical Officer

Analysts

• Daniil Gataulin, Analyst at Chardan
• Ellie Merle, Analyst at Barclays
• Joe Schwartz, Analyst at Leerink Partners
• Kristen Kluska, Analyst at Cantor
• Luca Issi, Analyst at RBC Capital Markets
• Moritz Reiterer, Analyst at Guggenheim Securities
• Patrick Trucchio, Analyst at H.C. Wainwright
• Peyton Bohnsack, Analyst at TD Cowen
• Rudy Li, Analyst at Wolfe Research
• Salveen Richter, Analyst at Goldman Sachs
• Suzanne van Voorthuizen, Analyst at Kempen
• Analyst at Stifel
• Analyst at Wells Fargo