Prepared remarks
Hello, and thank you for standing by. My name is Joy, and I will be your conference operator today. At this time, I would like to welcome everyone to the uniQure Second Quarter 2026 Earnings Call. I would now like to turn the call over to Chiara Russo, Senior Director of Investor Relations. Please go ahead.
Good morning, and thank you for joining us for uniQure's second quarter of 2026 earnings call. Earlier this morning, uniQure released financial results for the second 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, our Chief Financial Officer. After our formal remarks, we'll open up the call for Q&A. Before we begin, please note that we will be making forward-looking statements during this investor call. All statements other than statements of historical facts are forward-looking statements. They are based on management's beliefs and assumptions and on information available to management only as of the date of this conference call. Our 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.
Thanks, Chiara. Good morning, and thank you for joining us this morning. The second quarter was an important one for uniQure. We received guidance from both the FDA and MHRA, our near-term regulatory pathways for AMT-130, announced promising early data from our Fabry disease and refractory temporal lobe epilepsy programs and strengthened our balance sheet into 2030 through a follow-on offering. Taken together, these developments meaningfully advance our ability to deliver transformative therapies to patients with serious unmet needs. On today's call, I will provide a brief overview of the quarter before turning to Walid for an update on our clinical programs, Kylie on commercial readiness and Christian on the financials. I will then offer some closing remarks before opening the call to analyst questions. I want to start with AMT-130 and the progress we have made with our FDA interactions. In June 2026, we held a Type B meeting with the FDA during which we reached alignment with the FDA that a BLA submission under the accelerated approval pathway for AMT-130 based on the 3-year data is reasonable. This alignment was later confirmed in the final meeting minutes we recently received. The FDA asked to align the confirmatory study design prior to the BLA submission, including the consideration of a randomized standard of care design instead of a sham procedure. Additionally, consistent with the agency's January 2025 draft published guidance for accelerated approvals, the FDA stated that the confirmatory study should be feasible to conduct within a reasonable time frame and be well underway and potentially fully enrolled at the time of accelerated approval. We are fully committed to initiating the confirmatory trial as soon as possible after alignment has been reached. The FDA recognizes that HD is a serious disease with a high unmet need for safe and effective therapies, and we are working collaboratively with them on a confirmatory study design. We are on track to submit the BLA this quarter, and Walid will provide additional details later in the call. In parallel, after a successful presubmission meeting with the Medicines and Healthcare products Regulatory Agency, or MHRA, earlier this year, our U.K. regulatory submission is also on track as planned for the third quarter. Also in the third quarter, we expect to conduct our 4-year AMT-130 data analyses from the Phase I/II studies. We look forward to presenting the 4-year data results in September. Beyond AMT-130, we are encouraged by the progress across our broader pipeline. Early data from our Phase I/IIa study of AMT-260 in refractory mesial temporal lobe epilepsy and Phase I/II study of AMT-191 in Fabry disease continue to support the potential of both programs, and we look forward to sharing further updates in the first half of next year. As we prepare for potential commercialization of AMT-130, our team has intensified its focus and execution. We are deeply engaged with the Huntington's disease centers of excellence, payers and the patient community, and we are working diligently to put in place the infrastructure to support what we hope will be a timely and successful product launch. In summary, we are entering the second half of the year with strong momentum, clear regulatory pathways for AMT-130 in the U.S. and U.K., advancing pipeline programs and a customer-focused commercial organization prepared to deliver. We are grateful to the FDA for their continued engagement and collaboration, to the MHRA for their constructive interactions and above all, to the patients, families, investigators and advocates in the Huntington's disease community whose resilience and trust continue to inspire us every day. With that, I will turn the call over to Walid to provide additional detail on AMT-130 and our broader pipeline. Walid?
Thank you, Matt. Good morning and good afternoon, everyone. I'll start with AMT-130 in Huntington's disease. As Matt noted, the Type B meeting with the FDA was a pivotal moment for this program and the HD community. At this meeting, the FDA communicated that our 3-year Phase I/II data would be acceptable as the primary basis of a BLA for the accelerated approval of AMT-130. The FDA also requested that we align on the design of the confirmatory trial to support accelerated approval prior to BLA submission. We're working with the FDA to finalize the design of the confirmatory study. However, the critical point is that the FDA agreed that a randomized study using a sham control is no longer required. The agency recommended instead that we run a randomized standard of care controlled study with total functional capacity at 36 months as the primary endpoint. We are committed to conducting the global confirmatory study and will work diligently to ensure that the study is completed within a reasonable timeline. We remain on track for a third quarter BLA submission and look forward to potentially bringing this therapy to patients. On the ex-U.S. regulatory strategy, as Matt noted earlier, we are on track with our planned regulatory activities with the MHRA. With our near-term regulatory focus on the U.S. and the U.K., we expect to engage more fully with the European Medicines Agency in 2027 and remain committed to bringing AMT-130 to patients across Europe in due course. Finally, turning to our clinical progress, I'm very pleased to report that the AMT-130 clinical team is on track with data quality and database lock activities based on the June 30 cutoff date for the 4-year data, keeping us on schedule for the expected September update. We currently plan to disclose safety and tolerability data through 4 years of follow-up. The update will also include topline data from 12 high- and 12 low-dose patients at 4 years with an additional 3 patients at the high dose for a total of 15 patients now with 3 years of follow-up. Clinical data will include cUHDRS and its components such as TFC compared to a propensity score–matched natural history control derived from the Enroll-HD database. We continue to believe Enroll-HD provides a robust and contemporaneous comparator, and we are pleased that CHDI has afforded us the opportunity to incorporate the latest iteration of the Enroll-HD database into the 4-year analysis, which has been recently updated with approximately 6,000 additional HD participants for a total of approximately 26,000 participants to draw from. We also plan on providing CSF NfL change from baseline at 4 years. Moving on to AMT-260 for refractory mesial temporal lobe epilepsy. This quarter brought the first cohort-level readout from the Phase I/II study, which we presented at a medical conference in June. As of the May 29, 2026 data cutoff, 3 of 6 patients in the first low-dose cohort achieved meaningful reductions in disabling seizures during months 4 through 6, ranging from 79% to 100% below baseline. The remaining 3 patients showed variable outcomes over the same period, ranging from a 33% decrease to a 36% increase from baseline. Variability and response at this early stage is not unexpected, and we believe longer-term follow-up and the higher-dose cohort data will be important to understanding potential dose response and patient selection. On safety, as of the presentation date, there were no serious adverse events related to AMT-260 or the surgical procedure. All adverse events in the low-dose cohort were mild or moderate, most commonly headache in 2 patients, and no immunosuppression was required. We view this tolerability profile, combined with early signals of biological activity, as supportive of continued evaluation at the higher dose. Enrollment in the second higher-dose cohort is expected to complete imminently. Updated results for both cohorts are expected in the first half of 2027. Lastly, I will cover AMT-191 for Fabry disease. In June of this year, we presented updated preliminary safety and exploratory efficacy data from the Phase I/II study with the March 15, 2026 cutoff date. Patient follow-up ranged from 3 months to more than 18 months. Consistent with our disclosure in February, dose-dependent elevations of alpha-Gal A activity were observed in all 11 patients across 3 dose levels, plasma lyso-Gb3 levels remained stable post dose across all cohorts regardless of enzyme replacement therapy status, and all 11 dosed patients remained withdrawn from ERT. On safety, AMT-191 continued to show a manageable safety profile at all dose levels. Per protocol, additional dosing in the mid- and high-dose cohorts remains paused, pending agreement with the FDA on a monitoring and management plan following the Grade 3 liver enzyme elevations reported in 2 patients from the mid-dose cohort. These events were reviewed and confirmed as dose-limiting toxicity by the Independent Data Monitoring Committee. As of the end of May 2026, these LFT elevations have all resolved following a course of immunosuppression. If there are additional questions, Matt or others can address them. Now I will turn the call over to Kylie to discuss our ongoing efforts with the HD community and our U.S. and ex-U.S. commercial efforts.
Thank you, Walid. I want to begin, as always, by acknowledging the Huntington's disease community—the patients, the families and the caregivers who live with this disease every day—the clinicians who care for them and the advocates who have tirelessly pushed for regulatory flexibility and access. Your trust in us is what drives our sense of urgency and our recent accomplishments are a direct reflection of the work we have all done together. The FDA's communication that our 3-year Phase I/II data would be acceptable as the primary basis for a BLA submission represents the regulatory clarity our commercial team has been preparing for. With the BLA submission planned for the third quarter and an MAA submission to the U.K. MHRA on the same timeline, our commercial preparations have taken on renewed focus and urgency across three key priorities. First, treatment center readiness. We have maintained deep and ongoing engagement with Huntington's disease centers of excellence across the United States and the United Kingdom, working closely with the multidisciplinary neurosurgical, neurology and care teams that we believe are critical to a successful launch. The feedback we continue to receive from the community on the AMT-130 data set and its potential to meaningfully slow disease progression has been consistently strong and continues to reinforce our conviction to have a successful launch. Second, community engagement and education. Ensuring continuity across the care journey, understanding genetic testing and referral pathways and scientific education and communications remains a core focus. We are actively working to ensure that potentially eligible patients and the providers who care for them remain informed as we continue our commercial preparations. Third, market access readiness. Payer engagement is advancing in both the U.S. and the U.K., underpinned by a robust health economics and outcomes research program that continues to build the evidence base for the potential long-term clinical and societal value of AMT-130. Potential approval in either the U.S. or the U.K. would also unlock the potential for named patient and early access programs in additional geographies, including the Middle East, Latin America and Central and Eastern Europe, extending our potential reach to patients ahead of formal reimbursement decisions locally. Turning to AMT-260 in refractory temporal lobe epilepsy and AMT-191 in Fabry disease, our teams continue to deepen center of excellence relationships, refine the patient and provider journey and build the evidence base needed to support potential future development decisions in both indications. We remain energized by the early clinical signals from both programs and are laying the strategic groundwork in parallel with clinical development. I'll close by saying that we believe the opportunity to potentially deliver the first disease-modifying therapy to patients with Huntington's disease is closer than it has ever been. We are energized by the potential of AMT-130. And as we continue to engage with treatment centers, build pathways for patients and interact with payers, our organization will be ready as the HD community has waited long enough. Now I will turn the call over to Christian for a financial update.
Thank you, Kylie. I'll be sharing the financial highlights of the second 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 June 30, 2026, was $5.8 million compared to $5.3 million in the same period in 2025. The increase of $0.5 million is due to the increase in license revenue compared to the prior period. Research and development expenses were $34 million for the three months ended June 30, 2026, compared to $35.4 million during the same period in 2025. The $1.4 million decrease was driven by a $3.2 million decrease in other research and development expenses, partially offset by a $1.8 million increase in direct research and development expenses. The decrease in our research and development expenses primarily reflected a $1.5 million decrease in facility expenses, a $1.3 million decrease in employee and contractor-related expenses, including share-based compensation, and a $1 million decrease in the fair value of contingent consideration, partially offset by a $0.6 million increase in information technology costs. The increase in direct research and development expenses reflected higher spend on the AMT-260, AMT-162 and AMT-191 programs, partially offset by lower spend on AMT-130 compared to the prior period. Selling, general and administrative expenses were $17.4 million for the three months ended June 30, 2026, compared to $13.5 million during the same period of 2025. The $3.9 million increase was primarily related to a $4.3 million increase in employee and contractor-related expenses, including share-based compensation, mainly as a result of a higher number of employees recruited in the second half of 2025 to support the potential commercial launches of AMT-130, a $0.7 million increase in intellectual property fees and a $0.7 million increase in information technology costs and other expenses. This was partially offset by a $1.8 million decrease in professional fees, primarily as a result of lower costs incurred in support of the potential commercial launches of AMT-130 compared to the prior period. Cash, cash equivalents and investment securities totaled $810.3 million as of June 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 through 2026. 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.
Thank you, Christian. To summarize, we entered the second half of 2026 with clarity on our regulatory pathway for AMT-130, both in the U.S. and U.K. With the submission of multiple license applications for AMT-130 and the anticipated release of 4-year data, the coming months represent potentially transformational milestones for uniQure and for the HD community we are committed to serving. In parallel, we continue to execute across our pipeline with disciplined capital allocation, supported by a strong balance sheet that we expect to fund operations into 2030. Before we open to questions, I want to note that with the June 30 data cutoff passed, we are in a quiet period on the AMT-130 4-year data and will not be providing further commentary ahead of our September readout. We very much look forward to sharing those results with you then. Finally, I want to take a minute to sincerely thank my leadership, regulatory and clinical teams. I am truly motivated by their perseverance and unwavering commitment to the patients and families for whom we aim to deliver potentially life-changing therapies. With that, operator, please open up the call to questions. Thank you.
Questions and answers
Your first question comes from the line of Debjit Chattopadhyay with Guggenheim Securities.
Given the strength of the 3-year data, what would you consider to be the best outcome for the 4-year data? And what role do you think the 4-year data are going to play in any potential AdCom that one should expect for a first-in-class therapy for Huntington's?
Debjit, it's Matt. Thanks for the question. As I mentioned on the call, given that we're in a quiet period, we're not able to comment on the 4-year data. Obviously, with respect to an AdCom, that will be at the discretion of the FDA. The package that we're going to be submitting, if there's alignment, that package is going to be based on the 3-year data. The package is considered complete and self-contained. Of course, if the FDA requests the 4-year data, we're prepared to provide that to them, whether it's in the context of the review or an advisory committee.
Got it. And just one more follow-up here. Given that the confirmatory study needs to be nearly fully enrolled at the time of any accelerated approval, how quickly can the team operationalize this? And any clarity on the number of patients you are likely to enroll in the study would be helpful. And good luck going forward.
All right. So essentially, it's our belief that the fundamental intent of the FDA for all confirmatory studies is to ensure that they can be completed in a timely manner after approval, and we are confident we can demonstrate that. We haven't yet finalized the sample size with the FDA, so it's premature to provide exact numbers. We have, however, started preparatory activity for a global study. The plan is that most recruitment post approval will occur in countries before the drug becomes available in those countries such that we can complete the study on time. We are confident that we will be able to conduct the study and complete it on a reasonable timeline. This global approach gives us an effective path to get there. If there are additional points, others on the team can add, but that's the current position.
Your next question comes from the line of Joe Schwartz with Leerink Partners.
Congratulations on the impressive ascent here. In speaking with functional neurosurgeons, our takeaway is that AMT-130 delivery is very feasible at expert centers, but commercial uptake might depend less on surgeon willingness and technical ability and more on institutional workflow. As you prepare for launch, what have you learned from trial sites about operational bottlenecks? What are you doing to help address them? And how should investors think about realistic year 1 throughput for an activated center?
Yes, absolutely. Thanks, Joe, for the question. One of the things that has been important as we proceed with regulatory discussions is that we've continued preparation and discussions with treatment centers of excellence. We've been mapping each institution's process—neurology, neurosurgery and other specialties involved in a procedure like this—because no hospital is the same. We don't view these differences as insurmountable bottlenecks because we're working with institutions ahead of a potential BLA approval to make sure that, at the point of approval, we can move forward quickly. There are a number of centers ready to treat patients, and we are working with what we believe is the right initial number and will build from there. From a capacity perspective, it's challenging to give a single number because it depends on the number of neurosurgeons at a hospital, the number of intraoperative OR suites and other competing priorities. We're working through this and will plan to share more details in the coming months.
Your next question comes from the line of Paul Matteis with Stifel.
This is Matthew on for Paul. Congrats on all the progress. I guess based on your interactions with the FDA so far, are you expecting an AdCom meeting for this BLA submission? And then separately, I understand you had some studies on patients' low striatal volume and potentially shorter neurosurgical administration. What's the progress on those? And when might we see data from those cohorts?
Regarding an AdCom, our expectation is that we most likely will have one. We welcome it and are preparing accordingly. Regarding the low striatal volume cohort, that cohort has been fully recruited, but it's a bit early to share any efficacy data; we've recently shared some safety data. That cohort is moving forward and we will update as data mature. There is no ongoing cohort focused on a shorter surgical administration at this point, although that remains a key consideration for us as we move forward.
Your next question comes from the line of Luca Issi with RBC Capital Markets.
Team, this is Shelby on for Luca. Maybe on the regulatory setup for Huntington's. I appreciate different indications, but the recent FDA briefing documents ahead of AdComs for other companies did raise some pointed questions about drug efficacy. Does the tone of those documents give you any pause that the FDA could still push back on AMT-130 even with the 3-year data agreed upon as the primary basis for the BLA? Any color there, much appreciated.
Yes. Thanks for the question. We're aware of the AdComs occurring this week. It's not appropriate for us to comment on other companies' AdComs. From our perspective, each program is evaluated on its own merits, data and patient population. It's possible the FDA may request an AdCom; that will be their decision. We've had constructive and productive interactions with the FDA, and our view is that the data speaks for itself. Should there be an AdCom, we would welcome the opportunity to participate in that discussion.
Your next question comes from the line of Salveen Richter with Goldman Sachs.
This is Lydia on for Salveen. Just on the regulatory side, again, if you could provide any more color on the ongoing discussions, particularly around the standard of care control arm and how that might impact recruitment and retention in the study given the somewhat open-label nature of that?
Sure. The study design is straightforward: patients would be randomized to either receive treatment or be in the standard of care arm, where they are allowed to receive whatever is the latest standard of care available in their geography. Concerning retention, patients randomized to standard of care would be eligible to receive AMT-130 after 3 years, provided it is safe to administer to them. If AMT-130 becomes commercially available earlier in some countries, our strategy will account for that. Early recruitment will prioritize the U.S., and later recruitment will focus on countries where AMT-130 would not yet be available by the time we complete the study to minimize crossover that could affect the study. We will also use agreed statistical techniques to address dropouts. Overall, we are confident we can recruit and execute the study and draw meaningful conclusions. Patients with Huntington's disease are highly dedicated to participating in studies to help their families and their community.
Your next question comes from the line of Ellie Merle with Barclays.
This is Jasmine on for Ellie. So is your current expectation still that you will get priority review? And just following up on this, can you give some more detail on the ways that you're preparing to move quickly to have the confirmatory trial well underway at the time of approval? And how long would you potentially expect enrollment in the confirmatory trial to take?
Thanks, Jasmine. I'll take the first part on priority review. AMT-130 has Breakthrough Therapy designation, RMAT designation and Fast Track designation. Priority review is normally requested at the time of BLA submission and granted at the FDA's discretion upon acceptance. Given the unmet need here, we think there's a reasonable chance for priority review, but ultimately that is the FDA's decision. I'll hand the rest of the question to Walid about the confirmatory trial.
Regarding confidence in study conduct, we've been preparing and working within Clinical Operations to recruit and run a global trial. I'm confident we'll be able to recruit in time. In terms of size, it's premature to discuss sample size; we are still in discussion with the FDA to finalize the study design, which will determine powering and duration. Once agreed, we'll communicate timelines and provide a clearer idea about how long recruitment will take.
Your next question comes from the line of Uy Ear with Mizuho.
Congrats on all the progress. So I guess on my first question, could you just clarify whether the accelerated approval is dependent on completion of enrollment for the confirmatory study? If you don't complete, does that mean you don't get accelerated approval or will the application be held until it's completed? And the second question is, could you maybe just walk us through the assumptions behind your 2030 cash runway? Like what does that include exactly?
Thanks for the questions. I'll handle the first and then pass to Christian on the cash runway. The FDA's January 2025 draft guidance on accelerated approvals clarifies that accelerated approval does not require completion of the confirmatory study prior to approval. However, the FDA intends that confirmatory studies can be completed within a reasonable timeframe post approval. We do not need to have the trial completed to receive accelerated approval, but the FDA wants confidence that the study can be completed in a timely way. We feel very confident we can operationalize the study quickly, prioritize U.S. recruitment pre-approval and run a global study with sites in countries where AMT-130 will not yet be available, which will help minimize crossover and enable timely completion. I'll pass to Christian on the cash assumptions.
Thanks, Matt. Our guidance that cash, cash equivalents and investment securities will fund operations into 2030 includes assumptions such as funding the confirmatory trial, funding commercial launches, supporting ongoing clinical trials and making potential investments to advance other pipeline candidates into later-stage development. These assumptions reflect our current planning and capital allocation decisions.
Your next question comes from the line of Joseph Thome with TD Cowen.
Can you review with us maybe how the SAP for the 3-year data has changed at all over the past year since we saw the September data from last year and your level of alignment with the FDA on that for the final submission? And then maybe relatedly, with the June 30 cutoff date and the September data presentation for the 4-year data, is that just how long it takes to lock and clean the database? Or is there any SAP alignment that's gating that readout as well?
The 3-year SAP has not changed since we submitted it to the FDA in July 2025. Based on that SAP, we shared the data in September 2025, and there has been no reason to change it. Regarding the 4-year analysis, timelines are similar to last year; we're on track to share the results in September of this year. The timing reflects standard activities such as database lock and data cleaning, not any change in the SAP.
Great. Maybe just a related follow-up. Has the FDA signed off on that SAP used last year in the most recent meeting? What level of communication did they give you on that?
To provide context, we met with the FDA in April 2025, a month after we submitted the SAP briefing book. The FDA provided comments that we incorporated into the SAP, which we finalized and submitted in June 2025. There has been no formal further communication on that SAP, nor would we expect one. The FDA is aware of the data, and in the recent Type B meeting we aligned that the 3-year data support the BLA filing. That is what we are moving forward with.
Your next question comes from the line of Yanan Zhu with Wells Fargo.
This is Jeff on for Yanan. So following receipt of the Type B meeting minutes, how closely did the written feedback align with your interpretation of the discussions? Were there any areas of clarification or any points that differed from your initial takeaways? And separately, I believe I heard that total functional capacity at 3 years could serve as the primary endpoint for the confirmatory trial. Given that AMT-130 had about a 60% slowing of TFC in the Phase I/II study at 3 years, could you talk about the efficacy bar for the confirmatory trial? Is there any magnitude of TFC benefit that you believe could be required to support full approval?
The written meeting minutes aligned closely with our interpretation of the discussions. Regarding the magnitude of effect, our 3-year topline showed approximately a 60% slowing in TFC. The confirmatory trial will use that information, and the updated 4-year analysis (which includes additional patients) will further inform powering. We have a proposal but it is premature to discuss specific powering or exact efficacy thresholds until we reach agreement with the FDA on study details.
Your next question comes from the line of Kristen Kluska with Cantor.
Just to follow up on that point. Curious why the FDA is considering TFC as the primary endpoint over cUHDRS and if that's going to influence how they're going to review the package coming up while recognizing that you also had positive benefits on that endpoint.
This is not a surprise to us. We disclosed in 2024 that the FDA views the composite UHDRS as an intermediate clinical endpoint that is reasonably likely to predict efficacy. Our sense is that the FDA tends to favor functional endpoints, and total functional capacity is a direct measure of independence and has clear quality-of-life implications. While the FDA may lean toward TFC as a primary endpoint for a confirmatory study, the agency is comfortable that cUHDRS can serve as an intermediate clinical endpoint for accelerated approval.
Your next question comes from the line of Suzanne van Voorthuizen with Kempen & Company.
Maybe assuming approval looking at the commercial launch, it's a first-of-its-kind potential therapy. Can you elaborate a bit on some key characteristics of this upcoming launch that we should consider when thinking of proxies or example launches, speaking of features of the treatment modality, specifics of the indication or the setup of care centers, etc.?
Yes, absolutely. Key launch criteria include ensuring we have the right number of treatment centers set up and ready to treat patients, which is why we've spent significant time engaging with centers and understanding the specialties and internal processes involved. Another priority is payer engagement to ensure payers understand the unmet need in Huntington's disease and the potential value of AMT-130. Lastly, understanding patient care pathways, referral patterns and the overall patient journey is critical. In terms of analogs, a hospital-based procedure like ZOLGENSMA is a reasonable reference, and ELEVIDYS offers capacity perspectives, but no analog is perfect; each disease and treatment space has different dynamics. We're focused on ensuring operational readiness across centers, payers and patient pathways.
Got it. And maybe just a small follow-up. I know that Huntington's is a core focus, but you've reported encouraging data for epilepsy and Fabry this year. Can you shed some color on how you balance your prime focus versus decision-making and resource allocation for the other pipeline programs?
We prioritize disciplined, data-driven decisions. We run experiments to answer key questions. When data support advancing a program, we focus resources and execution on it. When data do not support continuation, we will deprioritize or discontinue programs. This approach helps us allocate capital effectively; we've used it in the past, for example with our SOD1-ALS program, and we'll continue to apply it.
Your next question comes from the line of Patrick Trucchio with H.C. Wainwright.
This is Arabella on for Patrick. I was just wondering, should we expect a prespecified interim analysis built into the confirmatory study? And if that was positive, say, at 2 years, could that support conversion to full approval prior to the 3-year primary? And also, do you have any other outstanding CMC items to work on before you can submit the BLA?
I'll answer the CMC point briefly. We feel confident we've completed activities required for BLA submission; Module 3 for the BLA must be completed, but the core activities around process performance qualification (PPQ), validation of analytics and assays are in place. We'll continue to finalize Module 3 as part of the submission. I'll hand the interim analysis question to Walid.
Regarding a prespecified interim analysis, it's premature to discuss specifics. It's a consideration that could be part of the study design, but we have not finalized this with the FDA, so I cannot provide details at this time. More to come once clarified.
Your next question comes from the line of Rudy Li with Wolfe Research.
Given that you already reached agreement on the filing package, what do you think could be the key questions and debates to be discussed at the upcoming AdCom meeting? Do you imagine any pushback from FDA? And secondly, it sounds like we don't expect enrollment of the confirmatory study to be a key limiting factor to get approval. So I just want to confirm.
I wouldn't want to speculate on the topics or positions at an AdCom that hasn't been requested; it's not helpful to predict what may happen. As for enrollment of the confirmatory study, accelerated approvals are conditional and the FDA's intent is to ensure confirmatory studies can be completed in a timely manner. We feel confident we can operationalize the study quickly, demonstrate it is well underway, and show the infrastructure to complete it. Recruitment should not be a limiting factor given our global approach and prioritization strategy.
Congrats on the progress.
Thank you.
This concludes the question-and-answer session and our call today. Thank you all for joining. You may now disconnect.