QURE

A month has gone by since the last earnings report for Spyre Therapeutics (SYRE). Shares have lost about 16.6% in that time frame, underperforming the S&P 500. But investors have to be wondering, will the recent negative trend continue leading up to its next earnings release, or is Spyre Therapeutics due for a breakout? Before we dive into how investors and analysts have reacted as of late, let's take a quick look at its latest earnings report in order to get a better handle on the important catalysts. Q2 Loss Narrower than Expected, Pipeline Progresses Spyre incurred a loss of 36 cents per share in the second quarter of 2026, narrower than the Zacks Consensus Estimate of a loss of 71 cents. In the year-ago quarter, the company reported a loss of 49 cents per share.Spyre has no marketed products in its portfolio and hence did not generate any revenues in the quarter.Total operating expenses were $41.6 million in the second quarter, slightly below $41.9 million in the year-ago period. The latest figure reflected a $40 million gain related to the previously completed sale of an in-process research and development asset, pegzilarginase. The gain was triggered by Immedica Pharma’s sale of a priority review voucher.The gain reduced the impact of elevated clinical development spending on quarterly results. It followed a $30 million gain recognized in the first quarter of 2026 from milestones connected with the same legacy asset.Research and development expenses totaled $65.5 million in the second quarter, up 63.2% year over year. The increase was primarily due to higher manufacturing and clinical study expenses, along with increased headcount costs.General and administrative expenses increased 36.9% year over year to $16.1 million, driven by higher headcount costs.Spyre ended the quarter with cash, cash equivalents and marketable securities of $1.15 billion compared with $741.5 million as of March 31, 2026. In April 2026, the company raised $435.2 million in net proceeds through a public offering of common stock. Management expects the current cash position to fund operations into the second half of 2029. Since the earnings release, investors have witnessed a downward trend in estimates revision. The consensus estimate has shifted -10.44% due to these changes. Currently, Spyre Therapeutics has a poor Growth Score of F, however its Momentum Score is doing a lot better with an A. However, the stock has a grade of F on the value side, putting it in the lowest quintile for this investment strategy. Overall, the stock has an aggregate VGM Score of F. If you aren't focused on one strategy, this score is the one you should be interested in. Estimates have been broadly trending downward for the stock, and the magnitude of these revisions indicates a downward shift. Notably, Spyre Therapeutics has a Zacks Rank #3 (Hold). We expect an in-line return from the stock in the next few months. Spyre Therapeutics belongs to the Zacks Medical - Biomedical and Genetics industry. Another stock from the same industry, uniQure (QURE), has gained 4.5% over the past month. More than a month has passed since the company reported results for the quarter ended June 2026. uniQure reported revenues of $5.84 million in the last reported quarter, representing a year-over-year change of +11%. EPS of -$1.22 for the same period compares with -$0.69 a year ago. uniQure is expected to post a loss of $0.87 per share for the current quarter, representing a year-over-year change of +37%. Over the last 30 days, the Zacks Consensus Estimate remained unchanged. uniQure has a Zacks Rank #3 (Hold) based on the overall direction and magnitude of estimate revisions. Additionally, the stock has a VGM Score of F. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Spyre Therapeutics, Inc. (SYRE) : Free Stock Analysis Report uniQure N.V. (QURE) : Free Stock Analysis Report This article originally published on Zacks Investment Research (zacks.com). Zacks Investment Research

A month has gone by since the last earnings report for uniQure (QURE). Shares have added about 17.5% in that time frame, outperforming the S&P 500. But investors have to be wondering, will the recent positive trend continue leading up to its next earnings release, or is uniQure due for a pullback? Well, first let's take a quick look at its most recent earnings report in order to get a better handle on the recent catalysts for uniQure N.V. before we dive into how investors and analysts have reacted as of late. QURE Q2 Earnings Miss Estimates on Higher Costs, Revenues Rise Y/Y uniQure incurred a second-quarter 2026 loss of $1.22 per share, wider than the Zacks Consensus Estimate of a loss of 81 cents. The loss widened 76.8% year over year, primarily due to higher non-operating and other expenses. Revenues rose 11% year over year to $5.8 million, driven primarily by higher license revenues. However, the metric missed the Zacks Consensus Estimate of $7 million. The company did not record any product sales during the quarter. Its revenue base continues to depend substantially on licensing arrangements, including economics associated with Hemgenix under its collaboration with CSL Behring. QURE’s Quarter in Detail Research and development (R&D) expenses declined 4% year over year to $34 million, primarily due to a $3.2 million reduction in other R&D expenses, partially offset by a $1.8 million increase in direct R&D spending. Selling, general and administrative expenses increased 28.6% year over year to $17.4 million. The increase primarily reflected higher employee-related costs to support the potential commercial launch of AMT-130, along with increased intellectual property and information technology costs, partially offset by lower professional fees. Other expenses surged 264.3% to $8 million, mainly due to a $6 million rise in costs associated with supplying Hemgenix to CSL Behring. uniQure ended the quarter with cash, cash equivalents and investments totaling $810.3 million compared with $586.6 million as of March 31, 2026. Management expects its current cash resources to fund operations into 2030. QURE’s Key Pipeline Updates uniQure continues to make significant regulatory progress with AMT-130, its investigational gene therapy for Huntington's disease. Following a Type B meeting in June 2026, the FDA agreed that a biologics license application (BLA) seeking accelerated approval based on the existing three-year phase I/II clinical data is reasonable, while requesting alignment on the confirmatory study design before submission. The company remains on track to submit the BLA in the third quarter of 2026 and plans to initiate the confirmatory study thereafter. In September 2026, uniQure expects to present updated data from its ongoing phase I/II studies on AMT-130 for the treatment of Huntington's disease, including four-year follow-up results from the first two patient cohorts. The company is advancing regulatory plans in the United Kingdom, with a regulatory submission scheduled for the third quarter of 2026. Apart from AMT-130, QURE’s pipeline comprises a few other candidates that are currently in development.  The company is evaluating AMT-260 for the treatment of refractory mesial temporal lobe epilepsy in the phase I/IIa study. The other candidate in the pipeline is AMT-191 for the treatment of Fabry disease. In June, the company announced encouraging preliminary six-month data from its phase I/IIa study, which demonstrated meaningful reductions in disabling seizures in half of the patients enrolled in the first low-dose cohort. uniQure expects to complete enrollment in the second dose cohort in the third quarter of 2026, with updated clinical data anticipated in the first half of 2027. In the past month, investors have witnessed a upward trend in estimates revision. The consensus estimate has shifted 6.37% due to these changes. At this time, uniQure has a poor Growth Score of F, a grade with the same score on the momentum front. Following the exact same course, the stock has a grade of F on the value side, putting it in the lowest quintile for this investment strategy. Overall, the stock has an aggregate VGM Score of F. If you aren't focused on one strategy, this score is the one you should be interested in. Estimates have been broadly trending upward for the stock, and the magnitude of these revisions looks promising. Notably, uniQure has a Zacks Rank #3 (Hold). We expect an in-line return from the stock in the next few months. uniQure belongs to the Zacks Medical - Biomedical and Genetics industry. Another stock from the same industry, Incyte (INCY), has gained 3.9% over the past month. More than a month has passed since the company reported results for the quarter ended June 2026. Incyte reported revenues of $1.67 billion in the last reported quarter, representing a year-over-year change of +37.7%. EPS of $3.09 for the same period compares with $1.57 a year ago. Incyte is expected to post a loss of $3.31 per share for the current quarter, representing a year-over-year change of -246.5%. Over the last 30 days, the Zacks Consensus Estimate has changed -90.4%. Incyte has a Zacks Rank #3 (Hold) based on the overall direction and magnitude of estimate revisions. Additionally, the stock has a VGM Score of B. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report uniQure N.V. (QURE) : Free Stock Analysis Report Incyte Corporation (INCY) : Free Stock Analysis Report This article originally published on Zacks Investment Research (zacks.com). Zacks Investment Research

Image source: The Motley Fool. Wednesday, July 29, 2026 at 8:30 a.m. ET Chief Executive Officer - Matthew Kapusta Chief Medical Officer - Walid Abi-Saab Chief Customer and Strategy Officer - Kylie O'Keefe Chief Financial Officer - Christian Klemt Senior Director of Investor Relations - Chiara Russo Operator: 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. [Operator Instructions] I would now like to turn the call over to Chiara Russo, Senior Director of Investor Relations. Please go ahead, ma'am. Chiara Russo: 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. Matthew Kapusta: 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 control 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? Walid Abi-Saab: 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 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 with a reasonable time line. 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 top line 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 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 or ERT 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 the course of immunosuppression. 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. Kylie? Kylie O'Keefe: 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 time line, our commercial preparations have taken on renewed focus and urgency across 3 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. Christian? Christian Klemt: 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 3 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 3 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 $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 3 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 $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. Matthew Kapusta: 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. Operator: Your first question comes from the line of Debjit Chattopadhyay with Guggenheim Securities. Debjit Chattopadhyay: 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? Matthew Kapusta: 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, 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 delighted to provide that to them, whether it's the context of the review or the advisory committee. Debjit Chattopadhyay: 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. Walid Abi-Saab: All right. So essentially, it's our belief that the fundamental intent of the FDA is that for all confirmatory studies is to ensure that they are completely or completed in a timely manner after approval, and we are confident we can demonstrate that. We talked about sample size. We haven't yet finalized this with the FDA. So it's kind of premature for us to do this. But suffice it to say that our team has been working on this and expecting a positive outcome from a discussion with the FDA. We started all of the preparatory activity for a global study that's going to be conducted. So the idea is that most of the 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 in a timely manner. And we believe that the way we are taking this approach with the global trial gives us an incredible path to get there. I'm sure there were a couple of other little things that you asked, Debjit. So I don't know if Matt or anybody wants to point me in the right direction or maybe I've answered all the questions so far. Operator: Your next question comes from the line of Joe Schwartz with Leerink Partners. Joseph Schwartz: 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 activated center? Kylie O'Keefe: Yes, absolutely. Thanks, Joe, for the question. I think one of the things that has been incredibly important while we move forward with regulatory discussions has been that the team has not stopped the preparation and discussions with treatment centers of excellence. And this has been incredibly important, as you said, to get them to understand these institutional nuances that occur across the different hospitals. And one of the things that we have learned is that no hospital is the same. And so what we have been doing is mapping each process across each institution, looking at neurology, neurosurgery and a number of other specialties that would be involved in a procedure like this. I will say that we don't see it as a bottleneck because we're doing everything that we can to work with these institutions ahead of a potential BLA approval to make sure that at the point of that BLA approval, we're able to move forward as quickly as possible. There are a number of centers that are available to do this, and we are working with what we think is the right number in the initial term, and then we will continue to build from there. From a capacity point of view, it's very challenging to give you one number, and we're still working through that because it depends on the number of neurosurgeons at a particular hospital. It depends on the number of intraoperative OR suites and other competing priorities. But we're working through this, and we will plan to share more details around that in the coming months. Operator: Your next question comes from the line of Paul Matteis with Stifel. Unknown Analyst: 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? Walid Abi-Saab: Sorry, Matt. So regarding the AdCom, I think our expectations is that we most likely will have one. We welcome it, and we are preparing for it. Regarding the low striatal volume cohort, that cohort has been fully recruited, but it's a bit early right now to share any of the efficacy data. We've recently shared some of the safety data. So this is moving forward, we will be updating you as the data mature. In terms of the shorter surgical, there's no -- we don't have an ongoing cohort focusing on that at this point, although this is a key element for us that we're going to be thinking about acutely as we're moving forward. Operator: Your next question comes from the line of Luca Issi with RBC Capital Markets. Unknown Analyst: 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 Capricor and Replimune did raise some pointed questions about the efficacy of both drugs. 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. Matthew Kapusta: Yes. Thanks for the question. I mean, obviously, we're aware of the AdComs going on this week, and appreciate that those are serious diseases. But it's not appropriate for us to comment on those particular AdComs. I mean we -- from our perspective, each program is evaluated on its own merits, on its own data and its own patient population. I think it's possible that the FDA may request an AdCom. This would be the first disease-modifying treatment for Huntington's disease. We continue to feel like our interactions with the FDA have been constructive and productive. And in the end, our view is that the data speaks for itself, and we would very much look forward to the extent that there's an AdCom in participating and having that discussion. Operator: Your next question comes from the line of Salveen Richter with Goldman Sachs. Lydia Erdman: This is Lydia on for Salveen. Just on the regulatory side, again, if you could provide any more kind of 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? Walid Abi-Saab: Sure. Thanks, Matt. Yes, I think the study design is straightforward. So patients would be randomized to either receive treatment or be on the standard of care arm, where they are allowed to receive whatever is the latest standard of care available to them in their geography. I think it's a fair question that you asked in terms of retention. We believe that in our case, there's going to be a couple of items that are going to be paying attention to. One is that people who would be randomized to standard of care will be eligible to receive AMT-130 at the -- after 3 years. They don't have to meet the inclusion criteria anymore. As long as it is safe to administer it to them, they will be able to receive it. Now of course, if the AMT-130 becomes available to them earlier because it's commercially available, that's going to also depend on our strategy, which I alluded to earlier. The earlier part of the study, we will be prioritizing the U.S. recruitment. But later in the study, we will be focusing on countries where AMT-130 would not be yet available by the time we complete the study so that we can minimize this. Last but not least, any long-term study will always have a risk of a dropout rate. And we will be using statistical techniques and in agreement with the agency about how we will deal with those dropouts. So overall, we do feel very confident that we'll be able to recruit the study and execute it in such a way that we can draw conclusions on it. And I think this is bolstered by the fact that patients with Huntington's disease actually are amazingly dedicated to being part of studies and actually generating these data, not just for them, but also for their family and their community. Thank you. Operator: Your next question comes from the line of Ellie Merle with Barclays. Unknown Analyst: 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? Matthew Kapusta: Yes. Thanks, Jasmine. I'll take the first part of that question on the priority review and then hand it over to Walid to talk about the confirmatory. So just as a reminder, AMT-130 has breakthrough therapy designation and RMAT designation and Fast Track designation. Normally, the priority review would be requested at the time of the BLA submission and the FDA would grant that or not at the time of the acceptance. But given the unmet need here, we think that there's a reasonable chance that, that would be accepted by the FDA. But ultimately, that's to the FDA's discretion. On to you, Walid. Walid Abi-Saab: Thanks, Matt. So regarding confidence in the study conduct, I think we prepared for this. We're working diligently with -- within ClinOps to be able to do that. And I'm very confident that we'll be able to recruit it in time. In terms of the size, it's premature to talk about it. As I mentioned, we are still in discussion with the FDA in terms of finalizing the study design, and that will also have, of course, an indication about the sample size. And so once we do that, we'll be able to communicate and we'll be able to give you a better idea about the time line it will take us to recruit this trial. Operator: Your next question comes from the line of Uy Ear with Mizuho. Uy Ear: Congrats on all the progress. So I guess on my first question, could you just clarify, I just want to make sure I understood correctly, 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 the accelerate -- or the application won't be approved or will be held until it's completed? And the second question is, could you maybe just provide -- walk us through the assumptions behind your 2030 cash way? Like what does that include exactly? Matthew Kapusta: Yes. Thanks for the questions. I'll handle the first question and then pass it over to Christian for the second question. So I mean, just to understand, the FDA put out draft guidance in January of 2025. That draft guidance is for all accelerated approvals and addresses confirmatory studies. And the FDA's intent for all confirmatory studies is really to make sure that they can be completed in a timely manner post approval, right? Accelerated approval is -- you don't have to complete the study to get accelerated approval. But the FDA wants to ensure that the study can be completed in a timely manner. And I think as Walid said, we feel very confident that we'll be able to do that. Number one, we feel confident we can operationalize the study very quickly. Number two, we believe that we'll be able to focus on the U.S. and pre-approval and to ensure that we have representation from the U.S. Third, this is going to be a global study where we're going to have sites in a number of different countries where the products are not commercially available. So we feel very confident that we'll be able to complete the study in a timely manner to address the FDA's priorities as it relates to confirmatory studies. And with that, I will pass it on to Christian. Christian Klemt: Thanks, Matt. Yes, the guidance into 2030 includes a number of things. So first and foremost, enrolling the confirmatory trial, funding the confirmatory trial into 2030, funding commercial launches as well as the ongoing clinical trials as well as making potential investments to advance certain other pipeline candidates into late-stage development. Operator: Your next question comes from the line of Joseph Thome with TD Cowen. Joseph Thome: 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 kind of gating for that readout as well? Walid Abi-Saab: Yes. So the 3-year SAP has not changed since we submitted it to the FDA in July of 2025. Based on that SAP, we shared with you the data back in September of 2025. So that has not changed. Actually, there should be no reason to change it after the fact. With regard to the 4-year analysis, the time lines are generally similar to what we've done for the last year -- for last year. So we're on track to be able to share the results with you in September of this year. Joseph Thome: Great. Maybe just a related follow-up. I guess, has the FDA signed off on that SAP used last year in the most recent meeting? I guess what level of communication do they give you on, yes, this is the SAP we agree with? Or do they not comment to that level? Walid Abi-Saab: So just to get back to the history a little bit. So we met with the FDA back in April of 2025, a month after we submitted the briefing book for the SAP. And the FDA at the time provided comments to us, which we incorporated in the SAP that we ultimately finalized and submitted to the FDA in June of last year. There's been no formal communication with the FDA since on that SAP. And we would not expect it. And that's -- these are the data that form the basis of the analysis and the FDA is aware of those data. And in the recent discussion with the FDA in the recent Type B meeting, we aligned with them that the data from the 3-year analysis -- from the 3-year data cut supports the BLA filing. So that is what we're moving forward with. Operator: Your next question comes from the line of Yanan Zhu with Wells Fargo. Unknown Analyst: 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 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 for -- to support full approval? Walid Abi-Saab: Yes. Thank you. So in terms of the magnitude effect of TFC, indeed, as you saw in our top line from the 3-year data analysis last year, the TFC changes were 60%. In the confirmatory trial, we will be using that information. Actually, it will be complemented with the updated 4-year analysis because if you recall, we have 3 more patients that would have reached the 3 years in that analysis. So we will have a total of 15 patients that of the 12 that we reported on last year, and we'll be using those to fine-tune the powering. There's been no discussion, as Matt indicated, with the FDA yet on the details of that study and the powering specifically. We had a proposal, but it's premature for us to be able to talk about it at this point before we reach agreement with the FDA. Operator: Your next question comes from the line of Kristen Kluska with Cantor. Kristen Kluska: 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. Matthew Kapusta: Yes. I think -- I mean, this is not a surprise to us at all. And we disclosed back in 2024 that the FDA views the composite unified Huntington's disease rating scale as an intermediate clinical endpoint that is reasonably likely to predict efficacy. Our sense is that the FDA, just philosophically, they look at composites as a number that in and of itself has value, but it's not as valuable or as pure, for lack of better words, as a functional endpoint. Total functional capacity is a measure of independence. It has a lot of aspects that are quality of life associated. And our sense is that in discussions that the FDA had with us as well as other sponsors that they tend to lean more towards total functional capacity as a primary endpoint for a confirmatory study. But in terms of an accelerated approval, the FDA is comfortable that the composite UHDRS is an intermediate clinical endpoint that is reasonably likely to predict efficacy or therapeutic benefit. Operator: Your next question comes from the line of Suzanne van Voorthuizen with Kempen & Company. Suzanne van Voorthuizen: Maybe assuming approval looking at the commercial launch, it's a first of its kind potentially. So can you elaborate a bit higher level on some key characteristics of this upcoming launch that you believe we should consider when thinking of proxies or example launches, speaking of things like the features of the treatment modality, the specifics of the indication or the setup of care centers, et cetera? Kylie O'Keefe: Yes, absolutely. Thank you very much for the question. So I think some of the characteristics that are going to be key launch criteria is on ensuring that we have the right number of treatment centers set up and ready to go and able to treat patients. I think this is obviously going to be one of the key criteria, which is why we've spent so much time engaging with the treatment centers, understanding the specialties that will be relevant within and ensuring we understand the processes as we were discussing earlier. I think this is something that will be a key priority leading up to launch and then obviously post launch. I think in addition to that, making sure that we have the right engagement on a payer level, making sure they understand the unmet need in Huntington's and the value that AMT-130 can potentially bring and then also ensuring that we understand the patient care pathways, how they're referred and how they're managed and the patient journey in totality. Understanding these 3 components will be critical to how we see launch success. You asked a little bit about analogs and other ways that would be consistent from a modality point of view. I think as we think about a treatment that is completed through a hospital procedure, I think ZOLGENSMA is a reasonable analog. Also, if you look at ELEVIDYS from a general understanding of capacity point of view, I think they're reasonable analogs, but I will caution that no analog is perfect. And I think every disease and every treatment space is a little bit different in the way the dynamics work, and we're looking to really ensure that we have all of our I's dotted and our T's crossed when it comes to launch preparation to bring this therapy to Huntington's patients. Suzanne van Voorthuizen: Got it. And maybe just a small follow-up. I know that Huntington's is a core focus, but I'm wondering for epilepsy and Fabry, you've reported some encouraging data for both this year. Can you shed some color on how you balance your prime focus versus how you go about decision-making and resource allocation for the other pipeline programs? Matthew Kapusta: Yes. I think over the years, we've really made it a priority to be very disciplined in how we invest and really to make data-driven decisions. We don't view discontinuing or deprioritizing programs as a failure. We're -- we talk about truth-seeking, and we try to run the experiments to answer questions. And when the data supports moving a program forward and advancing it, we want to do that and focus on impeccable execution. When data does not support moving the program forward, we're also happy to discontinue or deprioritize. We recently did that with our SOD1-ALS program. We've done that in the past, and that's going to be how we continue to make capital allocation decisions going forward. Operator: [Operator Instructions] Your next question comes from the line of Patrick Trucchio with H.C. Wainwright. Arabella Caroline Ng: 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 then also, do you have any other outstanding CMC items to work on before you can submit the BLA? Matthew Kapusta: Yes. Maybe I'll answer the first question and hand it over to Walid, understanding that we have not completed our discussions with the FDA on the confirmatory study. But on the CMC, we feel confident that we've completed the activities that are required for the BLA submission. Obviously, there, we need to complete Module 3 for the BLA submission. But the fundamental activities around PPQ, validation of analytics and assays, that work, we feel very comfortable that we've done what's required to be ready for the BLA submission. Walid? Walid Abi-Saab: Okay. So regarding the prespecified interim, it's really premature to discuss this. We haven't gone to that level yet with the FDA. I think it's a consideration that we should -- it should be part of it, but we haven't yet finalized it, so I really cannot discuss more. So hang tight, more to come once we have that clarified. Operator: Your next question comes from the line of Rudy Li with Wolfe Research. Guofang Li: Given that you already reached agreement on the filing package, what do you think could be the key questions and the 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. Matthew Kapusta: Sure. I didn't catch the last part of that, but just -- yes. I mean, I wouldn't want to speculate on what's going to be the content or the FDA's position of an AdCom meeting that hasn't yet been requested. So I don't know how helpful that would be there. And then can you just repeat the second part of the question? Guofang Li: Yes. Second part is really about do you expect enrollment of the confirmatory study to be a key limiting factor to get approval? Matthew Kapusta: Well, I think, I'll repeat what I said before. I think the FDA -- accelerated approvals are conditional approvals. And I think the FDA considers that flexibility because of these critically high unmet needs for Huntington's disease and other indications. But the confirmatory studies are important. And as I said, their fundamental focus is ensuring that they can be completed in a timely manner. And -- but the reality is they have wide discretion to do that. I mean this is not the first time that the FDA has contemplated a confirmatory study. And I think we feel very confident that we can operationalize the study expeditiously that we can demonstrate to the FDA that it is well underway and demonstrate to the FDA that we've got the infrastructure to complete it in a timely manner. So I think it will be a factor, but I think we feel confident that we can get the FDA comfortable on those key elements. Guofang Li: Congrats on the progress. Matthew Kapusta: Thank you. Operator: This concludes the question-and-answer session and our call today. Thank you all for joining. You may now disconnect. Before you buy stock in uniQure, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the 10 best stocks for investors to buy now… and uniQure wasn’t one of them. The 10 stocks that made the cut are built for long-term growth and could produce monster returns in the coming years. Consider when Netflix made this list on December 17, 2004... if you invested $1,000 at the time of our recommendation, you’d have $397,405!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of our recommendation, you’d have $1,344,091!* That performance is why people listen. 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The Motley Fool has a disclosure policy. uniQure (QURE) Q2 2026 Earnings Call Transcript was originally published by The Motley Fool

uniQure QURE incurred a second-quarter 2026 loss of $1.22 per share, wider than the Zacks Consensus Estimate of a loss of 81 cents. The loss widened 76.8% year over year, primarily due to higher non-operating and other expenses. Revenues rose 11% year over year to $5.8 million, driven primarily by higher license revenues. However, the metric missed the Zacks Consensus Estimate of $7 million. The company did not record any product sales during the quarter. Its revenue base continues to depend substantially on licensing arrangements, including economics associated with Hemgenix under its collaboration with CSL Behring. Year to date, uniQure's shares have surged 67.4% compared with the industry’s 4.4% growth. Image Source: Zacks Investment Research Research and development (R&D) expenses declined 4% year over year to $34 million, primarily due to a $3.2 million reduction in other R&D expenses, partially offset by a $1.8 million increase in direct R&D spending. Selling, general and administrative expenses increased 28.6% year over year to $17.4 million. The increase primarily reflected higher employee-related costs to support the potential commercial launch of AMT-130, along with increased intellectual property and information technology costs, partially offset by lower professional fees. Other expenses surged 264.3% to $8 million, mainly due to a $6 million rise in costs associated with supplying Hemgenix to CSL Behring. uniQure ended the quarter with cash, cash equivalents and investments totaling $810.3 million, compared with $586.6 million as of March 31, 2026. Management expects its current cash resources to fund operations into 2030. uniQure continues to make significant regulatory progress with AMT-130, its investigational gene therapy for Huntington's disease. Following a Type B meeting in June 2026, the FDA agreed that a biologics license application (BLA) seeking accelerated approval based on the existing three-year phase I/II clinical data is reasonable, while requesting alignment on the confirmatory study design before submission. The company remains on track to submit the BLA in the third quarter of 2026 and plans to initiate the confirmatory study thereafter. In September 2026, uniQure expects to present updated data from its ongoing phase I/II studieson AMT-130 for the treatment of Huntington's disease, including four-year follow-up results from the first two patient cohorts. The company is advancing regulatory plans in the United Kingdom, with a regulatory submission scheduled for the third quarter of 2026. Apart from AMT-130, QURE’s pipeline comprises a few other candidates that are currently in development.  The company is evaluating AMT-260 for the treatment of refractory mesial temporal lobe epilepsy in the phase I/IIa study. The other candidate in the pipeline is AMT-191 for the treatment of Fabry disease. In June, the company announced encouraging preliminary six-month data from the phase I/IIa study which demonstrated meaningful reductions in disabling seizures in half of the patients enrolled in the first low-dose cohort. uniQure expects to complete enrollment in the second dose cohort in the third quarter of 2026, with updated clinical data anticipated in the first half of 2027. uniQure N.V. price-consensus-eps-surprise-chart | uniQure N.V. Quote uniQure currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the biotech sector are Harmony Biosciences HRMY and Liquidia Corporation LQDA, each currently sporting a Zacks Rank #1 (Strong Buy) and Neurocrine Biosciences NBIX which carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here. Over the past 90 days, earnings per share estimates for Harmony Biosciences have decreased from $3.34 to $3.30 for 2026. Over the same period, estimates for earnings per share increased from $3.79 to $3.87 for 2027. HRMY shares have lost 4.2% year to date. Harmony Biosciences missed on earnings in each of the trailing four quarters, delivering an average negative surprise of 25.16%. Over the past 90 days, estimates for Liquidia’s 2026 earnings per share have increased to $3.02 from $1.50. Over the same period, EPS estimates for 2027 have risen to $5.31 from $2.91. LQDA shares have gained 151.7% year to date. Liquidia’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 54.40%. Over the past 90 days, estimates for Neurocrine Biosciences’ 2026 earnings per share have risen from $8.00 to $9.09. Over the same period, EPS estimates for 2027 have increased from $9.48 to $10.81. NBIX shares have gained 27.5% year to date. Neurocrine Biosciences’ earnings beat estimates in three of the trailing four quarters and missed in the remaining one, the average surprise being 9.08%. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report uniQure N.V. (QURE) : Free Stock Analysis Report Neurocrine Biosciences, Inc. (NBIX) : Free Stock Analysis Report Liquidia Corporation (LQDA) : Free Stock Analysis Report Harmony Biosciences Holdings, Inc. (HRMY) : Free Stock Analysis Report This article originally published on Zacks Investment Research (zacks.com). Zacks Investment Research

This article first appeared on GuruFocus. Revenue: $5.8 million for the three months ended June 30, 2026, compared to $5.3 million in the same period in 2025. Research and Development Expenses: $34 million for the three months ended June 30, 2026, compared to $35.4 million during the same period in 2025. Selling, General and Administrative Expenses: $17.4 million for the three months ended June 30, 2026, compared to $13.5 million during the same period of 2025. Cash Equivalents and Investment Securities: $810.3 million as of June 30, 2026, compared to $622.5 million as of December 31, 2025. Warning! GuruFocus has detected 5 Warning Signs with QURE. Is QURE fairly valued? Test your thesis with our free DCF calculator. Release Date: July 29, 2026 For the complete transcript of the earnings call, please refer to the full earnings call transcript. uniQure NV (NASDAQ:QURE) received guidance from both the FDA and MHRA, advancing regulatory pathways for AMT-130. Promising early data from Fabry disease and refractory temporal lobe epilepsy programs were announced. The company strengthened its balance sheet into 2030 through a follow-on offering. uniQure NV (NASDAQ:QURE) is on track to submit a BLA for AMT-130 under the accelerated approval pathway. The company has engaged deeply with Huntington's Disease Centers of Excellence, payers, and the patient community to prepare for potential commercialization. Research and development expenses remain high, with $34 million reported for the quarter. There is a need to align the confirmatory study design with the FDA, which could impact timelines. The FDA requires the confirmatory study to be well underway at the time of accelerated approval, posing operational challenges. AMT191 for Fabry disease faced dose-limiting toxicity issues, leading to a pause in additional dosing. Selling, general, and administrative expenses increased significantly, driven by higher employee-related costs. Q: Given the strength of the three-year data, what would you consider to be the best outcome for the four-year data? And what role do you think the four-year data are going to play in any potential outcome for a first dental therapy for Huntington's? A: As we're in a quiet period, we can't comment on the four-year data. The FDA will decide on the outcome. The package for submission is based on the three-year data, but if the FDA requests the four-year data, we are ready to provide it. Q: 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? A: The FDA's intent is to ensure confirmatory studies are completed timely post-approval. We are confident in our ability to demonstrate this. While we haven't finalized the sample size with the FDA, we are preparing for a global study to ensure timely completion. Q: As you prepare for launch, what have you learned from trial sites about operational bottlenecks? A: We've learned that each hospital is unique, so we're mapping processes across institutions. We're working with treatment centers ahead of potential BLA approval to ensure readiness. Capacity depends on factors like the number of neurosurgeons and OR suites, and we'll share more details soon. Q: Are you expecting an adcom meeting for this BLA submission? And what's the progress on studies for patients' post rate of volume and potentially shorter neurosurgical administration? A: We expect an adcom and are preparing for it. The low strider volume cohort is fully recruited, but it's early to share efficacy data. We will update as data matures. We don't have an ongoing cohort focusing on short-term surgical administration. Q: Does the tone of recent FDA briefing documents give you any pause that the FDA could push back on AMT-130, even with the three-year data agreed upon as the primary basis for the BLA? A: Each program is evaluated on its own merits. The FDA may request an adcom as this would be the first disease-modifying treatment for Huntington's. Our interactions with the FDA have been constructive, and we believe the data speaks for itself. For the complete transcript of the earnings call, please refer to the full earnings call transcript.

Interested in uniQure N.V.? Here are five stocks we like better. AMT-130 regulatory progress: uniQure said the FDA is aligned with a potential accelerated-approval pathway based on three-year Phase I/II data, with a biologics license application targeted for the third quarter of 2026. The company plans to present four-year data in September and is preparing for a potential FDA advisory committee review. Pipeline updates were mixed: AMT-260 epilepsy data showed seizure reductions of up to 100% in some low-dose patients, while AMT-191 Fabry disease dosing remains paused after dose-limiting liver enzyme elevations in two patients. Financial position strengthened: Second-quarter revenue rose to $5.8 million, while cash and investments reached $810.3 million as of June 30. Management said existing resources are expected to fund operations into 2030, including confirmatory trials and potential AMT-130 commercialization. Breakout Momentum Plays You Need to Know About uniQure (NASDAQ:QURE) said it has aligned with the U.S. Food and Drug Administration on a potential accelerated-approval pathway for AMT-130, its investigational gene therapy for Huntington's disease, and remains on track to submit a biologics license application in the third quarter of 2026. Chief Executive Officer Matt Kapusta said the company held a Type B meeting with the FDA in June, during which the agency indicated that three-year Phase I/II data could serve as the primary basis for an accelerated-approval application. The company said it subsequently received final meeting minutes confirming that alignment. → This Tiny AI Supplier Could Be More Important Than the Chipmakers QURE: Why Analysts See Up to 63% Upside After 250% Single-Day Pop The FDA has asked uniQure to reach agreement on the design of a confirmatory study before the BLA submission. The agency recommended a randomized study using a standard-of-care control rather than a sham procedure, with Total Functional Capacity, or TFC, at 36 months as the primary endpoint, according to Chief Medical Officer Walid Abi-Saab. Abi-Saab said the company is working with the FDA to finalize the confirmatory trial design and has begun preparatory work for a global study. While uniQure did not disclose a proposed enrollment target or timeline, management said it believes it can initiate and complete the study within a reasonable period after a potential approval. → Refiner Stocks Are Near Record Highs—Can Iran-Driven Margins Keep Them There? Patients randomized to standard care in the proposed confirmatory study would be eligible to receive AMT-130 after three years, provided treatment is safe for them, Abi-Saab said. The company also expects to use sites in countries where AMT-130 is not yet commercially available to support enrollment and trial completion. AMT-130 has received Breakthrough Therapy, Regenerative Medicine Advanced Therapy and Fast Track designations from the FDA. Kapusta said uniQure expects to request priority review when it submits the BLA, though the decision remains at the FDA's discretion. Management said it believes an FDA advisory committee meeting is likely and is preparing for that possibility. → Innovative ETF Strategies That Are Paying Off This Summer The company also plans to submit a marketing authorization application to the U.K. Medicines and Healthcare products Regulatory Agency in the third quarter. uniQure expects to engage more fully with the European Medicines Agency in 2027. UniQure plans to present four-year AMT-130 data in September. The analysis will include safety and tolerability data through four years, as well as top-line clinical data from 12 high-dose and 12 low-dose patients with four years of follow-up. An additional three high-dose patients will have reached three years of follow-up. The analysis is expected to include cUHDRS, TFC and other clinical measures compared with a propensity-score-matched natural-history control from the Enroll-HD database. The updated Enroll-HD database includes about 26,000 Huntington's disease participants, including roughly 6,000 added participants, the company said. UniQure also plans to report changes from baseline in cerebrospinal fluid neurofilament light chain at four years. Kapusta said the company is in a quiet period regarding the four-year findings and will not provide further commentary before the September presentation. He added that the three-year statistical analysis plan has not changed since its submission to the FDA in 2025. UniQure also reported early clinical findings for AMT-260, its investigational therapy for refractory mesial temporal lobe epilepsy. As of a May 29 data cutoff, three of six patients in the first low-dose cohort experienced reductions in disabling seizures of 79% to 100% from baseline during months four through six. The other three patients had outcomes ranging from a 33% reduction to a 36% increase in disabling seizures over the same period. There were no serious adverse events related to AMT-260 or the surgical procedure as of the presentation date, the company said. Reported adverse events in the low-dose cohort were mild or moderate, with headache reported in two patients. No immunosuppression was required. Enrollment in a second, higher-dose cohort is expected to be completed imminently, and uniQure expects updated results from both cohorts in the first half of 2027. For AMT-191 in Fabry disease, uniQure said all 11 dosed patients across three dose levels showed dose-dependent elevations in alpha-Gal A activity. Plasma lyso-Gb3 levels remained stable after dosing across cohorts, regardless of enzyme replacement therapy status, and all 11 patients remained off enzyme replacement therapy. Additional dosing in the mid- and high-dose cohorts remains paused while the company works with the FDA on a monitoring and management plan following grade 3 liver enzyme elevations in two mid-dose patients. An independent data monitoring committee classified the events as dose-limiting toxicities. UniQure said the liver-function-test elevations had resolved following immunosuppression as of the end of May. Chief Customer and Strategy Officer Kylie O'Keefe said uniQure is preparing for a potential AMT-130 launch by working with Huntington's disease centers of excellence in the United States and United Kingdom. The company is mapping institutional processes involving neurology, neurosurgery and other specialties, while also engaging with payers and patient communities. O'Keefe said treatment-center capacity will vary by institution based on factors including the number of neurosurgeons, operating-room availability and competing priorities. She cited Zolgensma and Elevidys as potential commercial analogs for a hospital-administered therapy, while cautioning that no launch comparison is exact. Second-quarter revenue was $5.8 million, compared with $5.3 million a year earlier, driven by higher license revenue. Research and development expense declined to $34 million from $35.4 million in the prior-year period. Selling, general and administrative expense rose to $17.4 million from $13.5 million, reflecting hiring and other preparation for potential AMT-130 commercialization. Cash, cash equivalents and investment securities totaled $810.3 million as of June 30, up from $622.5 million at the end of 2025. Chief Financial Officer Christian Klemt said uniQure expects its cash resources to fund operations into 2030. The estimate includes the confirmatory trial, potential commercial launches, ongoing clinical trials and possible investments to advance other pipeline candidates into late-stage development. uniQure N.V. 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. This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to [email protected]. The article "uniQure Q2 Earnings Call Highlights" was originally published by MarketBeat. View MarketBeat's top stocks for July 2026.

~ U.S. and U.K. regulatory submissions for AMT-130 for Huntington’s disease on track as planned for the third quarter of 2026 ~ ~ Topline four-year data from the Phase I/II study of AMT-130 expected in September 2026 ~ ~ Reported data from the first cohort in the Phase I/IIa trial of AMT-260 for refractory mesial temporal lobe epilepsy showed early biological signals of therapeutic activity and a favorable safety profile ~ ~ Strengthened financial position with $259 million follow-on offering, extending cash runway into 2030 and funding the anticipated commercial launch of AMT-130 and continued pipeline investment ~ ~ uniQure to host earnings call at 8:30 a.m. ET ~ LEXINGTON, Mass. and AMSTERDAM, July 29, 2026 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the second quarter of 2026 and highlighted recent progress across its business. “This has been a defining quarter – not just for uniQure, but for the Huntington's disease community,” said Matthew Kapusta, chief executive officer at uniQure. “Following a productive Type B meeting with the FDA, we remain on track to submit our BLA for AMT-130 in the third quarter — a milestone that reflects years of rigorous science, disciplined execution, and an unwavering commitment to the patients and families living with this devastating disease. With four-year data expected in September and the U.K. regulatory activities progressing as planned, we enter the second half of 2026 with real momentum and a clear line of sight to potentially bringing this therapy to the people who need it.” “Beyond AMT-130, we continue to execute across our broader pipeline,” Mr. Kapusta continued. “With a strong balance sheet, we believe we are well-positioned for what will be a transformative period for uniQure and the patients we serve.” Recent Company Developments and Updates Advancing AMT-130 for the treatment of Huntington’s disease In June 2026, the Company held a Type B meeting with the U.S. Food and Drug Administration (FDA). Official meeting minutes received in July 2026 confirmed that the FDA and the Company reached alignment that a Biologics License Application (BLA) submission under the accelerated approval pathway for AMT-130, based on the existing clinical data, is reasonable. In addition, the FDA seeks to align on the confirmatory study design prior to the BLA submission, including consideration of a randomized standard-of-care control design instead of a sham procedure. The FDA also stated that, in accordance with the FDA’s draft public guidance for accelerated approvals, the confirmatory study should be feasible to conduct within a reasonable timeline and be well underway, and potentially fully enrolled, at the time of accelerated approval. Discussions with the FDA to align on the confirmatory study design and analysis are underway and the Company expects to submit a BLA in the third quarter of 2026. The Company plans to initiate the confirmatory study as expeditiously as possible after aligning with the FDA on the trial design. In September 2026, the Company plans to present data from its ongoing Phase I/II studies of AMT-130. The update is expected to include follow-up data on all patients treated with AMT-130 in the first two cohorts, including four years of follow-up on 24 patients (12 patients at the high-dose and 12 patients at the low dose). In March 2026, the Company held a successful pre-submission meeting with the United Kingdom’s (U.K.) Medicines and Healthcare products Regulatory Agency (MHRA) and the regulatory submission is progressing as planned for the third quarter of 2026. Continued clinical progress in pipeline programs AMT-260 for the treatment of refractory mesial temporal lobe epilepsy (MTLE) In June 2026, the Company announced preliminary six-month follow-up data on the first, low dose cohort of six patients in the ongoing Phase I/IIa study. The Company expects to complete enrollment in the second dose cohort in the Phase I/IIa study in the third quarter of 2026. The Company expects to present updated results from the Phase I/IIa study in the first half of 2027. AMT-191 for the treatment of Fabry disease In June 2026, the Company presented new data from the Phase I/II study of AMT-191 in Fabry disease. The data, with a cutoff date of March 15, 2026, included patient follow-up ranging from three months to more than 18 months and consisted of the following: Per protocol, additional dosing in the mid- and high-dose cohorts remains paused, pending further evaluation of asymptomatic liver enzyme elevations reported in two patients from the mid-dose cohort, which were confirmed as dose-limiting toxicities. These elevations resolved as of the end of May 2026 following a course of immunosuppression as per the study protocol. Focused execution and strong financial position In June 2026, the Company closed an upsized underwritten public offering of 5,686,813 ordinary shares at a public offering price of $45.50 per share, including the full exercise of the underwriters’ option to purchase additional shares. The aggregate gross proceeds to uniQure from the offering, before deducting the underwriting discounts and commissions and offering expenses payable by uniQure, were $259 million. As of June 30, 2026, the Company had cash, cash equivalents and current investment securities of $810.3 million. The Company expects that these resources will be sufficient to fund projected operating expenses into 2030. Financial Highlights Cash Position: As of June 30, 2026, the Company held $810.3 million in cash, cash equivalents and current investment securities, compared to $622.5 million as of December 31, 2025. The Company expects that these resources will be sufficient to fund our projected operating expenses into 2030. Revenues: 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 an increase in license revenue, compared to the prior period. R&D Expenses: Research and development expenses were $34.0 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 other 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 $1.0 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. SG&A Expenses: 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 in 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. Other Expense: Other expense increased to $8.0 million for the three months ended June 30, 2026 from $2.2 million during the same period in 2025, primarily due to a $6.0 million increase in costs associated with the supply of HEMGENIX® to CSL Behring. Other non-Operating Items, net: Other non-operating items, net was an expense of $27.0 million for the three months ended June 30, 2026, compared to a gain of $6.6 million during the same period in 2025. The $33.6 million increase in net non-operating expense was primarily related to a $20.4 million unfavorable change in net foreign currency, from an $18.6 million gain to a $1.7 million loss, and a $16.0 million loss resulting from changes in the fair value of the liability related to pre-funded warrants, compared to nil in the prior period. This was partially offset by a $1.5 million increase in interest income and a $1.2 million decrease in interest expense, compared to the prior period. Net loss: The net loss for the three months ending June 30, 2026, was $81.1 million, or $1.22 basic and diluted loss per ordinary share, compared to a $37.7 million net loss for the comparative period in 2025, or $0.69 basic and diluted loss per ordinary share. Upcoming investor events: 2026 Biotech Summer Summit, August 10 -12 – Newport, RI Investor Conference Call and Webcast Information uniQure management will host an investor conference call and webcast today, Wednesday, July 29th at 8:30 a.m. ET. The event will be webcast under the Events & Presentations section of uniQure’s website at https://www.uniqure.com/investors-media/events-presentations, and following the event a replay will be archived for 90 days. Analysts wishing to participate in the question and answer session should access the live call by dialing (646) 307-1963 or toll-free (800) 715-9871 and entering conference ID 5075555. If you are joining the conference call, please join 15 minutes before the start time. About uniQure uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. The approvals of uniQure’s gene therapy for hemophilia B – an historic achievement based on more than a decade of research and clinical development – represent a major milestone in the field of genomic medicine and ushers in a new treatment approach for patients living with hemophilia. uniQure is now advancing a pipeline of proprietary gene therapies for the treatment of patients with Huntington's disease, refractory temporal lobe epilepsy, Fabry disease, and other severe diseases. www.uniQure.com uniQure Forward-Looking Statements This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," “establish,” "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," “seek,” "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Examples of these forward-looking statements include, but are not limited to, statements concerning: the Company’s cash runway and its ability to fund its operations into 2030; the Company’s ability and plans to strategically advance its programs, including the potential commercialization of AMT-130; the Company’s plans and timing with respect to future interactions with regulatory authorities and regulatory updates related to AMT-130; the Company’s plans to conduct a confirmatory study for AMT-130, including the design, timing, endpoints and control arm of such study, and to align with the FDA on such study prior to BLA submission as well as the potential use of a concurrent standard-of-care control arm rather than a sham procedure; expectations regarding the timing of a BLA submission and receiving accelerated approval of AMT-130; the Company’s plans to complete enrollment in the second dose cohort in the Phase I/IIa study for MTLE in the third quarter of 2026; the Company’s plans to provide further clinical updates, including plans to announce topline four-year data from the Company’s AMT-130 program in September 2026 and updated results from the Phase I/IIa study in MTLE in the first half of 2027; and the Company’s plans to attend upcoming investor events. The Company’s actual results could differ materially from those anticipated in these forward-looking statements for many reasons. These risks and uncertainties include, among others: risks associated with the clinical results and the development and timing of the Company’s programs, including the risk that clinical results will be unable to demonstrate data sufficient to support further clinical development or regulatory approval in any country where approval is pursued; the risk that the FDA ultimately concludes that the Phase I/II trial data for AMT-130 are not sufficient to support a BLA or accelerated approval; the risk that more patient data become available that results in a different interpretation than the one derived from preliminary, interim or topline data; the Company’s interactions with regulatory authorities, including the FDA and MHRA, which may affect the initiation, timing and progress of clinical trials and pathways and timing for regulatory approval; whether the measurements that the Company is evaluating are viewed as robust and sensitive measurements of disease progression suitable for regulatory approval; the Company’s ability to conduct and fund any required confirmatory study for AMT-130; the Company’s ability to successfully complete any required confirmatory study for AMT-130; the risk that accelerated approval, if granted, may be subject to post-approval requirements that are difficult or costly to satisfy; the Company’s ability to continue to build and maintain the infrastructure and personnel needed to achieve its goals; the Company’s effectiveness in managing current and future clinical trials and regulatory processes; the continued development and acceptance of gene therapies; the Company’s ability to demonstrate the therapeutic benefits of its gene therapy candidates in clinical trials; the Company’s ability to obtain, maintain and protect intellectual property; and the Company’s ability to fund its operations. These risks and uncertainties are more fully described under the heading "Risk Factors" in the Company’s periodic filings with the U.S. Securities & Exchange Commission (“SEC”), including the Company’s Annual Reports on Form 10-K and Quarterly Reports on Form 10-Q, and in other filings that the Company makes with the SEC from time to time. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and, except as required by law, the Company assumes no obligation to update these forward-looking statements, even if new information becomes available in the future.

The S&P 500 Index ($SPX) (SPY) today is down -0.15%, the Dow Jones Industrial Average ($DOWI) (DIA) is up +0.23%, and the Nasdaq 100 Index ($IUXX) (QQQ) is up +0.30%.  June E-mini S&P futures (ESM26) are down -0.17%, and June E-mini Nasdaq futures (NQM26) are up +0.24%. Stock indexes are mixed today, with the Dow Jones Industrials posting a new all-time high.  Strength in chipmakers is leading the overall market higher.  Stocks also garnered support on better-than-expected US economic reports on US May retail sales, a sign of resilient consumer demand, and May pending home sales.  Weakness in telecommunication and trucking stocks is limiting gains in the overall market. Rocket Lab vs. Redwire: 1 Stock Has the Stronger Growth Story for the Next Decade Dear SpaceX Stock Fans, Mark Your Calendars for June 16 Dear Western Digital Stock Fans, Mark Your Calendars for June 22 Our exclusive Barchart Brief newsletter is your FREE midday guide to what's moving stocks, sectors, and investor sentiment - delivered right when you need the info most. Subscribe today! Stocks also have carryover support from Monday after the US and Iran agreed to end their war and reopen the Strait of Hormuz, knocking crude oil prices down to a 3.5-month low and stoking risk-on sentiment in asset markets. The market’s focus will be on the conclusion of today’s 2-day FOMC meeting, the first under the leadership of new Fed Chair Kevin Warsh. While the Fed is expected to keep interest rates unchanged, the spotlight will be on how Mr. Warsh navigates the post-meeting press conference and the outlook for inflation. US MBA mortgage applications fell -3.8% in the week ended June 12, with the purchase mortgage sub-index down -3.4% and the refinancing mortgage sub-index down -4.5%.  The average 30-year fixed rate mortgage was unchanged from last week at 6.60%. US May retail sales rose +0.9% m/m, stronger than expectations of +0.6% m/m.  Also, May retail sales ex-autos rose +0.8% m/m, stronger than expectations of +0.6% m/m. US May pending home sales rose +3.8% m/m, stronger than expectations of +0.9% m/m and the biggest increase in 20 months. WTI crude oil prices (CLN26) recovered from a 3.5-month low today and are moving higher as prices consolidate following this week’s plunge.  The eventual resumption of vessel traffic through the Strait of Hormuz could lead to the release of more than 100 laden ships carrying oil from Middle Eastern countries other than Iran that are stuck in the Persian Gulf, effectively releasing stockpiles into the market.  Goldman Sachs on Tuesday cut its price forecast on Brent crude to $80 a barrel in Q4 of this year, down from $90 a barrel, and said it expects Persian Gulf crude exports to return to pre-war levels by the end of July, one month earlier than previously expected. The markets are discounting a 5% chance of a +25 bp rate hike at the conclusion of today’s FOMC meeting. Overseas stock markets are higher today.  The Euro Stoxx 50 rallied to a new record high and is up +0.62%.  China's Shanghai Composite rose to a 2.5-week high and closed up +0.40%.  Japan’s Nikkei-225 Stock Average climbed to a new all-time high and closed up +0.72%. Interest Rates September 10-year T-notes (ZNU6) today are down -3 ticks, and the 10-year T-note yield is up +1.2 bp to 4.434%.  Sep T-notes are moving lower today after US reports showed May retail sales and May pending home sales rose more than expected, a hawkish factor for Fed policy.  Also, today’s stock strength has reduced safe-haven demand for T-notes.  Losses in T-notes are limited in hopes for a less hawkish FOMC meeting today, given that oil prices should decline over time if the Strait of Hormuz reopens as expected. European government bond yields are moving lower today.  The 10-year German bund yield fell to a 1.75-month low of 2.914% and is down -0.3 bp to 2.927%.  The 10-year UK gilt yield fell to a 2-month low of 4.734% and is down -3.3 bp to 4.755%. Eurozone May core CPI was revised upward to 2.6% y/y from the previously reported 2.5% y/y, the strongest pace of increase in 13 months. ECB Governing Council member Gediminas Simkus said that the "pass-through of the increase in energy and other raw material prices to the market has already occurred," and "at least one more rate increase is certainly more likely than not." UK May CPI rose 2.8% y/y, weaker than expectations of 3.0% y/y. May core CPI rose 2.6% y/y, weaker than expectations of 2.7% y/y. Swaps are discounting a 15% chance of a +25 bp ECB rate hike at its next policy meeting on July 23. US Stock Movers Chipmakers are rebounding today, recovering some of Tuesday’s sharp losses.  Applied Materials (AMAT) is up more than +8% to lead gainers in the S&P 500 and Nasdaq 100, and ASML Holding NV (ASML), ARM Holdings Plc (ARM), and Lam Research (LRCX) are up more than +5%.  Also, Broadcom (AVGO) and Marvell Technology (MRVL) are up more than +4%, and KLA Corp (KLAC) and (INTC) are up more than +3%.  In addition, Advanced Micro Devices (AMD) is up more than +2%, and Analog Devices (ADI), Micron Technology (MU), NXP Semiconductors NV (NXPI), and Qualcomm (QCOM) are up more than +1%. The Magnificent Seven technology stocks are moving lower today, weighing on the broader market.  Meta Platforms (META) is down more than -3% to lead losers in the Nasdaq 100, and Alphabet (GOOGL) and Amazon.com (AMZN) are down more than-2%.  Also, Microsoft (MSFT) and Tesla (TSLA) are down more than -1%.  In addition, Apple (AAPL) is down -0.64% and Nvidia (NVDA) is down -0.50%. Telecommunication stocks are under pressure today, a negative factor for the overall market.  Charter Communications (CHTR), Verizon Communications (VZ), and AT&T (T) are down more than -2%.  Also, Comcast Corp (CMCSA) is down more than -1%. Trucking stocks are sliding today for a third day after Citigroup on Monday warned that the recent rally in trucking and logistics stocks had been overdone.  RXO Inc (RXO) is down more than -4%, and Old Dominion Freight Line (ODFL), ArcBest (ARCB), and XPO Inc (XPO) are down more than -3%.  Also, Knight-Swift Transportation Holdings (KNX) and FedEx Freight Holdings (FDXF) are down more than -2%. UniQure NV (QURE) is up more than +77% after saying the FDA allowed 3-year data from its Phase I/II study of AMT-130 for Huntington’s disease to be acceptable as the primary basis for an application for accelerated approval. Wabash National Corp (WNC) is up more than +21% after D.A. Davidson upgraded the stock to buy from neutral with a price target of $20. La-Z-Boy (LZB) is up more than +15% after reporting Q4 adjusted EPS of $1.26, stronger than the consensus of 83 cents. Aehr Test Systems (AEHR) is up more than +11% after saying it received a follow-on production order for a fully automated FOX-XP wafer-level burn-in (WLBI) system. Credicorp Ltd (BAP) is up more than +7% after Morgan Stanley upgraded the stock to overweight from equal weight with a price target of $480. Figma Inc (FIG) is up more than +5% after Citigroup initiated coverage on the stock with a recommendation of buy and a price target of $36. Jabil (JBL) is up more than +4% after reporting Q3 net revenue of $8.80 billion, better than the consensus of $8.54 billion, and raised its full-year net revenue forecast to $35 billion from $34 billion, stronger than the consensus of $34.24 billion. Charles River Laboratories International (CRL) is up more than +2% after Morgan Stanley upgraded the stock to overweight from equal weight with a price target of $220. Ormat Technologies (ORA) is down more than -4% after Bernstein initiated coverage on the stock with a recommendation of underperform and a price target of $115. CME Group (CME) is down more than -3% after announcing that CEO Terry Duffy is stepping down and CFO Lynne Fitzpatrick will replace him on March 1, 2027. Rexford Industrial Realty (REXR) is down more than -2% after JPMorgan Chase downgraded the stock to underweight from neutral with a price target of $25. Leidos Holdings (LDOS) is down more than -2% after Bank of America Global Research downgraded the stock to neutral from buy. ResMed (RMD) is down more than -2% after Morgan Stanley downgraded the stock to equal weight from overweight. Earnings Reports(6/17/2026) CarMax Inc (KMX), Jabil Inc (JBL), Safe Bulkers Inc (SB), Smith & Wesson Brands Inc (SWBI). On the date of publication, Rich Asplund did not have (either directly or indirectly) positions in any of the securities mentioned in this article. All information and data in this article is solely for informational purposes. This article was originally published on Barchart.com

AMT-130 regulatory push: uniQure has a Type B meeting with the FDA later in Q2 to discuss a statistical analysis plan and design of a new study, and plans a U.K. MAA submission in Q3 supported by three‑year data that the company says shows ~75% slowing on the composite UHDRS at three years. Pipeline and safety updates: a four‑year AMT‑130 analysis is expected in Q3; early data for epilepsy candidate AMT‑260 from the first cohort are due in Q2; dosing for AMT‑191 continues at low dose after mid/high dose pauses for asymptomatic grade‑3 liver enzyme elevations, and AMT‑162 was discontinued following a dorsal root ganglia toxicity event. Financial position: Q1 revenue rose to $3.6M while R&D declined to $29.2M and SG&A increased to $20.1M for commercial hires, with cash, cash equivalents and securities of $586.6M—management expects runway into the second half of 2029. Interested in uniQure N.V.? Here are five stocks we like better. Breakout Momentum Plays You Need to Know About uniQure (NASDAQ:QURE) executives outlined progress and upcoming regulatory milestones for lead Huntington’s disease gene therapy candidate AMT-130 during the company’s first-quarter 2026 earnings call, while also providing updates across its epilepsy and Fabry programs and reviewing quarterly financial results. Chief Executive Officer Matt Kapusta said uniQure remains focused on advancing AMT-130 “globally with urgency,” while continuing discussions with U.S. regulators following a Type A meeting with the FDA in January. Kapusta said the company has been granted a Type B meeting with the FDA later in the second quarter, where uniQure plans to discuss a proposed statistical analysis plan for data expected in the third quarter, as well as “key elements of a new clinical study.” → Roblox Stock Slides to New Low as Safety Changes Weigh on Outlook QURE: Why Analysts See Up to 63% Upside After 250% Single-Day Pop Chief Medical Officer Dr. Walid Abi-Saab characterized the upcoming Type B meeting as “a technical meeting,” aimed at clarifying “key design elements of an additional new study to evaluate the efficacy of AMT-130” and obtaining FDA feedback on the statistical analysis plan for the phase I/II study data expected in the third quarter. When asked whether uniQure would discuss an alternative regulatory path that might avoid initiating a new study, Abi-Saab said, “We do not intend to have a specific discussion about a regulatory path to filing at this point.” In parallel, the company is advancing an ex-U.S. strategy. Abi-Saab said uniQure held a “successful pre-submission meeting” with the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) and plans to submit a marketing authorization application (MAA) for AMT-130 in the third quarter, supported by its three-year clinical data analysis. He added the company has begun engaging with European regulators and is “evaluating additional opportunities internationally” to pursue access efficiently. → The Real SpaceX Play: 5 Chip Stocks Powering the IPO Before It Launches Kapusta discussed the company’s view of the strength of its AMT-130 dataset when responding to a question about differences between the FDA and MHRA interactions. He said uniQure believes it has “achieved 75% slowing of disease with high statistical significance out to three years on the composite UHDRS,” along with “statistical significance in a slowing of disease on Total Functional Capacity,” favorable trends across other measures, and neurofilament light “below baseline.” Abi-Saab added that rare disease policy focus in the U.K. “allows for more flexibility to be afforded in rare diseases like this,” while noting that engagement with the FDA has become “a bit more difficult” more recently. Abi-Saab said the four-year analysis, expected in the third quarter, will generally mirror the three-year analysis with an additional year of follow-up. He said the dataset is expected to include 12 high-dose and 12 low-dose patients with four years of follow-up, plus three additional high-dose patients reaching three years (15 high-dose patients at the three-year timepoint). Abi-Saab said uniQure is discussing with the FDA whether additional analyses could “increase the level of confidence,” adding that the company expects treatment effects to become more apparent over time, particularly for high-dose patients compared with “well-matched external controls.” → 3 Emerging Markets ETFs to Maximize Exposure to High-Potential Countries Executives repeatedly emphasized the role of real-world evidence for trial design. Abi-Saab highlighted the Enroll-HD resource, which he said includes more than 30,000 participants and provides longitudinal clinical data that could inform efficient and statistically rigorous designs for a one-time therapy in a slow-progressing disease. Chief Customer and Strategy Officer Kylie O’Keefe said the company is advancing commercial preparations in several geographies based on progress with the MHRA. She described three near-term priorities: Ensuring treatment center capacity and readiness in collaboration with multidisciplinary care teams at centers of excellence. Maintaining continuity in the patient journey through engagement efforts that support genetic testing and referral pathways. Advancing market access readiness through proactive payer engagement and an evidence-based value proposition supported by health economics and outcomes research. O’Keefe said there are “between 7,000-8,000 patients living with HD in the U.K., with approximately 30,000 at risk,” and told analysts that “the vast majority” of diagnosed patients are treated at specialized centers. She said that following potential MHRA approval, uniQure expects to work with NICE and other access bodies, including NHS England, to pursue managed access agreements. When asked about U.K. capacity and procedural readiness, O’Keefe said uniQure does not anticipate changes to the AMT-130 surgical procedure when moving from clinical to commercial use. She also said the cannula used in the procedure has already been shipped to multiple countries and that the U.K. “is no issue,” adding that other companies already use the cannula commercially in the U.K. On broader ex-U.S. access, O’Keefe said uniQure aims to begin with named patient and early access programs and then pursue formal pricing and reimbursement. She cited the U.K.’s “Highly Specialized Technology” route and evolving cost-effectiveness considerations as signs of a shifting approach to advanced therapies. She also said the company is evaluating countries on a market-by-market basis, referencing Germany’s initial free-pricing period, France’s early access pathways, and named patient approaches in countries such as Italy. O’Keefe also said an MHRA approval could enable early access or named patient programs in other regions, including Gulf countries in the Middle East, Latin America, the Commonwealth of Independent States, and parts of Central and Eastern Europe. Beyond Huntington’s disease, Kapusta and Abi-Saab highlighted several pipeline updates. For AMT-260 in refractory mesial temporal lobe epilepsy, Kapusta said enrollment in the phase I/IIa study is on track, with data from the first cohort expected in the second quarter. Abi-Saab said uniQure plans to provide an update later in the second quarter on six treated patients with at least six months of safety, tolerability, and seizure frequency outcomes, with results expected to be presented at the Epilepsy Foundation Pipeline Conference in June. He said the trial’s primary objective is safety, while seizure frequency is a key exploratory endpoint; he added that the company would view “perhaps a 50% reduction in seizure frequency” as a meaningful signal to pursue in subsequent controlled studies. For AMT-191 in Fabry disease, executives reiterated previously disclosed data from 11 patients across three dose cohorts. Abi-Saab said patients demonstrated dose-dependent and durable increases in alpha-galactosidase A activity, with stable plasma lyso-Gb3 levels maintained post-dose regardless of enzyme replacement therapy (ERT) status. He also said that as of Feb. 18, all 11 dosed patients had discontinued ERT. On safety, Abi-Saab said two mid-dose patients experienced asymptomatic grade 3 liver enzyme elevations that met protocol-defined criteria for potential dose-limiting toxicity, which the independent data monitoring committee reviewed and confirmed. As a result, dosing at the mid and high doses was paused per protocol. He said no new AMT-191-related serious adverse events have been observed to date and described the program as demonstrating “a manageable safety profile.” Abi-Saab told analysts uniQure is continuing to dose at the low dose while following affected patients closely and plans to submit the liver enzyme data to the FDA and discuss resuming dosing at the mid or high dose. For AMT-162 in SOD1-ALS, Abi-Saab said uniQure discontinued development following a review of EPISOD1 trial data after a serious adverse event of dorsal root ganglia toxicity in one patient that was determined to be related to AMT-162. He said the company will continue safety follow-up for the five dosed patients consistent with regulatory requirements. Chief Financial Officer Christian Klemt reported first-quarter 2026 revenue of $3.6 million, up from $1.6 million in the same period of 2025, attributing the increase to higher license revenue. Research and development expenses were $29.2 million, down from $36.1 million a year earlier, driven by decreases including a $2.6 million reduction in fair value of contingent consideration, lower external program spend, reduced employee and contractor-related expenses (including share-based compensation), and lower facilities and other costs. Selling, general and administrative expenses rose to $20.1 million from $10.9 million, which Klemt said was primarily driven by higher employee and contractor-related expenses, including share-based compensation, “mainly as a result of employees recruited in 2025 to support commercial planning for AMT-130,” along with increases in professional fees, intellectual property fees, and information technology and other expenses. Cash, cash equivalents, and investment securities totaled $586.6 million as of March 31, 2026, compared with $622.5 million at the end of 2025. Klemt said uniQure expects its cash resources to be sufficient to fund operations into the second half of 2029. Abi-Saab later added that runway assumptions include completing ongoing clinical trials in temporal lobe epilepsy, Huntington’s disease, and Fabry disease, but that the plan does not support advancing “all the candidates in kind of the most expedited manner” simultaneously without prioritization decisions. Looking ahead, Kapusta highlighted 2026 milestones including the Type B FDA meeting later in the second quarter, an AMT-260 clinical update in the second quarter, the four-year AMT-130 data analysis in the third quarter, and the planned U.K. MAA submission for AMT-130 in the third quarter. uniQure N.V. 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 article "uniQure Q1 Earnings Call Highlights" was originally published by MarketBeat.