Core Insights - uniQure reported a quarterly loss of $0.56 per share, outperforming the Zacks Consensus Estimate of a loss of $0.93, and showing improvement from a loss of $1.5 per share a year ago, resulting in an earnings surprise of +39.91% [1] - The company generated revenues of $5.57 million for the quarter ended December 2025, which was 21.2% below the Zacks Consensus Estimate, but an increase from $5.22 million in the same quarter last year [2] - The stock has underperformed, losing approximately 34.7% year-to-date compared to a 0.5% gain in the S&P 500 [3] Earnings Outlook - The earnings outlook for uniQure is mixed, with the current consensus EPS estimate for the upcoming quarter at -$0.90 on revenues of $10.35 million, and for the current fiscal year at -$3.32 on revenues of $62.48 million [7] - The company's Zacks Rank is currently 3 (Hold), indicating expected performance in line with the market in the near future [6] Industry Context - The Medical - Biomedical and Genetics industry, to which uniQure belongs, is currently ranked in the top 36% of over 250 Zacks industries, suggesting a favorable outlook compared to lower-ranked industries [8] - Empirical research indicates a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can be tracked by investors [5]

Financial Data and Key Metrics Changes - Revenue for the year ended December 31, 2025, was $16.1 million, a decrease from $27.1 million in 2024, primarily due to a $10.7 million decrease in collaboration revenue and a $6.1 million decrease in contract manufacturing revenues, offset by a $5.8 million increase in license revenues [27] - Research and development expenses were $140.7 million for 2025, down from $143.8 million in 2024, driven by a $26 million decrease in total other research and development expenses [28] - Cash, cash equivalents, and investment securities totaled $622.5 million as of December 31, 2025, compared to $367.5 million as of December 31, 2024, primarily due to proceeds of approximately $404.2 million raised from public offerings [30] Business Line Data and Key Metrics Changes - AMT-130 showed a statistically significant 75% slowing of disease progression in Huntington's disease as measured by the Composite Unified Huntington's Disease Rating Scale [7] - AMT-191 for Fabry disease reported preliminary safety and exploratory efficacy data from 11 patients, with all patients successfully withdrawn from enzyme replacement therapy [18] - AMT-260 for mesial temporal lobe epilepsy showed promising initial data with a reduction in seizure frequency over the first 6 months [21] Market Data and Key Metrics Changes - The company is actively assessing ex-U.S. opportunities, evaluating priority markets based on epidemiology, regulatory pathways, pricing, and reimbursement landscapes [24] - The potential market for AMT-191 in Fabry disease is positioned to compete against chronic enzyme replacement therapies, highlighting the advantages of a one-time gene therapy approach [25] Company Strategy and Development Direction - The company remains committed to engaging with regulatory authorities to define the most appropriate path forward for AMT-130 and advancing its pipeline programs with discipline [31] - The strategy includes pursuing named patient and early access program opportunities in rare diseases outside the U.S. to enable access to therapies ahead of formal reimbursement decisions [24] - The company aims to leverage the Enroll-HD database to strengthen study designs for phase III trials and minimize patient burden [48] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strength and durability of AMT-130 data and the urgency of unmet needs in the Huntington's disease community [10][31] - The company is focused on constructive engagement with the FDA to define a clear regulatory path forward, despite challenges faced in the approval process [9][31] - Management emphasized the importance of patient safety and the need for regulatory flexibility in the context of rare, progressive diseases [9][45] Other Important Information - The company plans to request a Type B meeting in the second quarter of 2026 to discuss potential phase III study design approaches [16] - The company is preparing to present a manuscript with the complete results of the three-year analysis of AMT-130 in a peer-reviewed medical journal later this year [17] Q&A Session Summary Question: Are there any paths that you can potentially pursue in order to push your agenda beyond just the traditional FDA channels? - The company mentioned that advocacy from the patient community is critical in educating elements outside of the FDA about the urgency of the situation [36] Question: Was the morbidity associated with procedures involving burr holes a major sticking point in the Type A meeting? - Management acknowledged that patient safety is a focus for the FDA and emphasized the strong safety profile observed with AMT-130 [43] Question: Can you give some more color on the different scenarios for a potential phase III design? - The company stated that it is premature to discuss specifics about the phase III design until after discussions with the FDA [58] Question: What are the key differences in the FDA's requirements for a sham control versus a prospective natural history comparator? - Management indicated that the FDA is seeking maximum reduction of potential bias, which is why a sham-controlled study is recommended [96]

Financial Data and Key Metrics Changes - Revenue for the year ended December 31, 2025, was $16.1 million, a decrease from $27.1 million in 2024, primarily due to a $10.7 million decrease in collaboration revenue and a $6.1 million decrease in contract manufacturing revenues, offset by a $5.8 million increase in license revenues [27] - Research and development expenses were $140.7 million for 2025, down from $143.8 million in 2024, driven by a $26 million decrease in total other research and development expenses [28] - Cash, cash equivalents, and investment securities totaled $622.5 million as of December 31, 2025, compared to $367.5 million as of December 31, 2024, primarily due to proceeds of approximately $404.2 million raised from public offerings [30] Business Line Data and Key Metrics Changes - AMT-130 showed a statistically significant 75% slowing of disease progression in Huntington's disease as measured by the Composite Unified Huntington's Disease Rating Scale [7] - AMT-191 for Fabry disease reported that all 11 patients in the Phase 1/2 trial exhibited elevated alpha-Gal A enzyme activity, with 6 patients successfully withdrawn from enzyme replacement therapy [18] - AMT-260 for mesial temporal lobe epilepsy showed promising initial data with a reduction in seizure frequency over the first 6 months [21] Market Data and Key Metrics Changes - The company is actively assessing ex-U.S. opportunities, evaluating priority markets based on epidemiology, regulatory pathways, pricing, and reimbursement landscapes [24] - The market for AMT-191 in Fabry disease is positioned to compete against chronic enzyme replacement therapies, highlighting the potential advantage of a one-time genetic approach [25] Company Strategy and Development Direction - The company remains focused on engaging with regulatory authorities to define the most appropriate path forward for AMT-130 and advancing pipeline programs with discipline [31] - The strategy includes pursuing named patient and early access program opportunities in rare diseases outside of the U.S. to enable access to therapies ahead of formal reimbursement decisions [24] - The company aims to leverage the Enroll-HD database to strengthen study designs for Phase 3 trials and minimize patient burden [48] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strength and durability of AMT-130 data and the commitment to the Huntington's disease community [10] - The urgency in the Huntington's disease community is acknowledged, with a strong belief that gene therapy can meaningfully change disease trajectories [32] - Management is committed to working with the FDA to explore regulatory flexibility for rare diseases [9] Other Important Information - The company plans to request a Type B meeting in the second quarter of 2026 to discuss potential Phase 3 study design approaches [16] - The company is preparing to present a manuscript with the complete results of the three-year analysis in a peer-reviewed medical journal later this year [17] Q&A Session Summary Question: Are there any paths to push your agenda beyond traditional FDA channels? - The company is exploring opportunities outside the U.S. and believes advocacy from the patient community is critical in educating elements outside the FDA [36] Question: Was morbidity associated with procedures involving burr holes a major sticking point? - The company has a strong safety profile and has not seen disease or clinical safety events associated with AMT-130 since December 2022 [44] Question: What are the different scenarios for a potential Phase III design? - It is premature to discuss specifics, as the design is still being defined in discussions with the FDA [58] Question: Why is the FDA requiring a sham control trial? - The FDA seeks to minimize potential bias, and while the company believes in the robustness of the natural history data, they acknowledge the FDA's preference for sham-controlled studies [96]

Financial Data and Key Metrics Changes - Revenue for the year ended December 31, 2025, was $16.1 million, a decrease from $27.1 million in 2024, primarily due to a $10.7 million decrease in collaboration revenue and a $6.1 million decrease in contract manufacturing revenues, offset by a $5.8 million increase in license revenues [24] - Research and development expenses were $140.7 million for 2025, down from $143.8 million in 2024, driven by a $26 million decrease in total other research and development expenses [25][26] - Cash, cash equivalents, and investment securities totaled $622.5 million as of December 31, 2025, compared to $367.5 million as of December 31, 2024, primarily due to proceeds of approximately $404.2 million raised from public offerings [28] Business Line Data and Key Metrics Changes - AMT-130 showed a statistically significant 75% slowing of disease progression in Huntington's disease as measured by the Composite Unified Huntington's Disease Rating Scale, and a 60% slowing as measured by Total Functional Capacity [5] - AMT-191 for Fabry disease reported that all 11 patients in the Phase 1/2 trial exhibited elevated alpha-Gal A enzyme activity, with 6 patients successfully withdrawn from enzyme replacement therapy [16] - AMT-260 for mesial temporal lobe epilepsy showed promising initial data with a reduction in seizure frequency over the first 6 months, with no serious adverse events reported [18] Market Data and Key Metrics Changes - The company is actively assessing ex-U.S. opportunities, evaluating priority markets based on epidemiology, regulatory pathways, pricing, and reimbursement landscapes [21] - The potential market for AMT-191 in Fabry disease is positioned to compete against chronic enzyme replacement therapies, highlighting the advantages of a one-time gene therapy approach [22] Company Strategy and Development Direction - The company remains focused on engaging with regulatory authorities to define the most appropriate path forward for AMT-130, while advancing pipeline programs with discipline [29] - The strategy includes pursuing named patient and early access program opportunities in rare diseases outside the U.S. to enable access to therapies ahead of formal reimbursement decisions [21] - The company aims to leverage the Enroll-HD database to strengthen study designs for Phase 3 trials and minimize patient burden [45] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strength and durability of AMT-130 data, emphasizing the urgency of unmet needs in the Huntington's disease community [29] - The company is committed to constructive engagement with the FDA to explore regulatory flexibility for rare diseases, particularly in light of the challenges faced in the approval process [7][10] Other Important Information - The company plans to request a Type B meeting in the second quarter of 2026 to discuss potential Phase 3 study design approaches [14] - The company has initiated regulatory discussions with several agencies outside the U.S. following unsolicited outreach after the 3-year data disclosure [14] Q&A Session Summary Question: Are there any paths to push your agenda beyond traditional FDA channels? - The company is exploring opportunities outside the U.S. and believes that advocacy from the patient community is critical in educating elements outside the FDA about the urgency of the situation [33] Question: Can you confirm the plan for the four-year data submission? - The company plans to amend the protocol to include a four-year analysis and will present data from all patients who have reached three years, along with the 12 patients at four years [35] Question: What is the strategy for the Type B meeting? - The main goal is to discuss designs for the Phase 3 trial, leveraging the Enroll-HD database to strengthen study designs and minimize patient burden [44] Question: What are the sticking points with the FDA regarding the sham control requirement? - The FDA has raised concerns about the absence of treatment effects relative to sham subjects in the U.S. Phase I/II study after 12 months, leading to the recommendation for a sham-controlled study [10][70] Question: Is the company committed to moving forward with a sham study? - The company is dedicated to pursuing AMT-130 and will consider the feasibility and ethical implications of a sham-controlled study based on patient community support [78]

Financial Performance - The company reported a net loss for the years ended December 31, 2025, and 2024, and anticipates continued losses in the future[17] - The company has incurred net losses in the years ended December 31, 2025 and 2024, and expects to continue incurring losses in the future, indicating a risk of never achieving profitability[17] Product Development and Regulatory Challenges - The lead product candidate, AMT-130, is critical for the company's success in treating Huntington's disease, with potential regulatory approval challenges[15] - The company may leverage specialized regulatory pathways such as the FDA's accelerated approval pathway and Breakthrough Therapy designation for product candidates[15] - The company acknowledges risks associated with regulatory authorities that could affect clinical trial progress and regulatory submissions[11] - The company may face delays in clinical trials and challenges in demonstrating safety and efficacy, which could adversely affect its business[15] - The company may leverage specialized regulatory pathways such as the FDA's accelerated approval pathway, but may not maintain the benefits associated with such designations[15] - Negative public opinion and increased regulatory scrutiny of gene therapy could damage the public perception of the company's product candidates[15] Market and Competition - The company faces significant competition in the gene therapy market, which may impact its ability to commercialize products successfully[17] - The company faces substantial competition, with the risk that others may develop or commercialize competing products more successfully[17] Manufacturing and Operational Risks - The company relies on third-party manufacturers for its product candidates, which may lead to production challenges and delays[17] - The company relies on third parties for manufacturing its product candidates, which may not perform satisfactorily or comply with regulations, leading to potential delays[17] Strategic Partnerships and Personnel - The company is actively seeking partnerships to support the development and commercialization of its product candidates[17] - The company emphasizes the importance of retaining key personnel to achieve its strategic goals and maintain operational effectiveness[17] Stock Market and Investor Risks - The market price of the company's ordinary shares has been volatile and may fluctuate significantly in the future[17] - The market price of the company's ordinary shares has been volatile and may fluctuate substantially, posing risks to investors[17] Future Outlook - Future changes in legal and regulatory requirements may materially impact the company's results of operations[17]

Group 1 - The FDA has informed UniQure that the data from its early-to-mid stage trial is insufficient to support a marketing application for its gene therapy aimed at treating brain disorders [1] - UniQure's gene therapy is currently under scrutiny, which may impact its future development and commercialization plans [1] Group 2 - BlackRock's Global Infrastructure Partners and EQT AB have agreed to acquire AES Corp for a total of $33.4 billion, including debt [6] - This acquisition reflects ongoing consolidation trends in the energy sector, highlighting the interest of investment firms in utility companies [6]

Exhibit 99.1 uniQure Announces 2025 Financial Results and Provides Recent Company Updates ~ Held Type A meeting with FDA to discuss AMT-130 for Huntington's disease; Company evaluating Phase III development considerations and plans to request follow-up Type B meeting in the second quarter of 2026 ~ ~ Completed enrollment of the first cohort in the Phase I/IIa study of AMT-260 in refractory mesial temporal lobe epilepsy, with additional clinical data expected in the first half of 2026~ ~ Presented updated Ph ...

Core Insights - uniQure N.V. reported updated Phase I/II data for AMT-191 in Fabry disease, showing durable, dose-dependent increases in α-Gal A enzyme activity [1] - The company has a strong financial position with approximately $622.5 million in cash and equivalents as of December 31, 2025, expected to fund operations into the second half of 2029 [1][13] - The CEO expressed confidence in AMT-130 as a potential first disease-modifying therapy for Huntington's disease, despite not yet aligning with the FDA on an approval pathway [2] Financial Performance - Revenue for the year ended December 31, 2025, was $16.1 million, a decrease from $27.1 million in 2024, primarily due to a $10.7 million drop in collaboration revenue [14] - Research and development expenses were $140.7 million for 2025, down from $143.8 million in 2024, mainly due to reduced costs following the divestment of the Lexington facility [16] - The net loss for the year ending December 31, 2025, was $199.0 million, or $3.46 per share, compared to a net loss of $239.6 million, or $4.92 per share, in 2024 [21][22] Clinical Developments - AMT-130 for Huntington's disease showed a statistically significant 75% slowing in disease progression at 36 months compared to an external control [5] - The company completed enrollment of the first cohort in the Phase I/IIa study of AMT-260 for refractory mesial temporal lobe epilepsy, with additional data expected in the first half of 2026 [4][11] - AMT-191 demonstrated dose-dependent elevations in α-Gal A activity, with six of 11 patients withdrawing from enzyme replacement therapy [11] Regulatory Engagement - The company held a Type A meeting with the FDA regarding AMT-130 and plans to request a follow-up Type B meeting in the second quarter of 2026 [4][6] - The FDA indicated that the data from Phase I/II studies compared to an external control may not be sufficient for a marketing application, recommending a prospective, randomized, double-blind study [6] Upcoming Events - uniQure will host an earnings call on March 2, 2026, to discuss its financial results and business updates [1][23] - The company is scheduled to participate in several investor conferences in March and April 2026 [26]

Core Viewpoint - A securities fraud class action lawsuit has been filed against uniQure N.V. for misleading investors regarding its gene therapy drug AMT-130 during the specified Class Period [2][4][5]. Company Overview - uniQure N.V. is a biotechnology company focused on developing gene therapies for rare diseases, particularly Huntington's disease (HD) [4]. - The company's leading drug candidate is AMT-130, aimed at slowing the progression of HD [4]. Lawsuit Details - The lawsuit, titled Scocco v. uniQure N.V., was filed in the United States District Court for the Southern District of New York [2]. - The Class Period for the lawsuit is defined as September 24, 2025, to October 31, 2025 [7]. - Investors who purchased shares during this period may seek to serve as lead plaintiffs by April 13, 2026 [3][9]. Allegations - The complaint alleges that uniQure made materially false and misleading statements regarding its Phase I/II clinical trials and the timeline for its Biologics License Application (BLA) submission to the FDA [5]. - Specific allegations include: 1. The design of the Pivotal Study was not fully approved by the FDA [5]. 2. The likelihood of needing to delay the BLA timeline was downplayed [5]. 3. Statements about the company's business and prospects lacked a reasonable basis [5]. Impact on Share Price - On November 3, 2025, uniQure's share price dropped by $33.40, or over 49%, from $67.69 to $34.29 following the revelation that the FDA did not agree with the adequacy of the data for BLA submission [6][8].

Core Viewpoint - Rosen Law Firm is reminding investors who purchased ordinary shares of uniQure N.V. between September 24, 2025, and October 31, 2025, about the upcoming lead plaintiff deadline on April 13, 2026, for a class action lawsuit [1]. Group 1: Class Action Details - Investors who bought uniQure shares during the specified period may be eligible for compensation without any out-of-pocket fees through a contingency fee arrangement [2]. - A class action lawsuit has already been filed, and interested parties can join by contacting Rosen Law Firm [3]. - To serve as lead plaintiff, individuals must file a motion with the Court by April 13, 2026 [3]. Group 2: Case Background - The lawsuit alleges that uniQure misrepresented and/or failed to disclose critical information regarding the design of its Pivotal Study for a drug candidate targeting Huntington's Disease, including the lack of full FDA approval [5]. - It is claimed that uniQure downplayed the likelihood of needing to delay its Biologics License Application (BLA) timeline due to the necessity of additional studies [5]. - As a result of these misrepresentations, the lawsuit asserts that the statements made by uniQure regarding its business and prospects lacked a reasonable basis, leading to investor damages when the truth was revealed [5]. Group 3: Rosen Law Firm's Credentials - Rosen Law Firm emphasizes the importance of selecting qualified legal counsel with a proven track record in securities class actions, highlighting its own success in this area [4]. - The firm has achieved significant settlements, including the largest securities class action settlement against a Chinese company, and has consistently ranked highly in terms of settlements since 2013 [4]. - In 2019, the firm secured over $438 million for investors, showcasing its capability in recovering funds for clients [4].