Core Insights - uniQure N.V. has achieved alignment with the FDA on the statistical analysis plan and CMC requirements for AMT-130, supporting a planned BLA submission in Q1 2026 [1][4] - The company expects to present topline three-year data for AMT-130 in September 2025 [1][4] - Early clinical data for AMT-260 shows a 92% reduction in seizures for the first patient treated, with no serious adverse events reported [1][4] - The appointment of Kylie O'Keefe as Chief Customer and Strategy Officer is aimed at strengthening the leadership team [1][4] - As of June 30, 2025, the company has approximately $377.0 million in cash and equivalents, expected to fund operations into the second half of 2027 [1][5] Recent Developments - The FDA's alignment supports an Accelerated Approval pathway for AMT-130, with a focus on the three-year change in the composite Unified Huntington's Disease Rating Scale [4] - The final statistical analysis plan was submitted to the FDA in July 2025, and a PPQ campaign has been initiated [4] - AMT-130 has received Breakthrough Therapy designation from the FDA based on Phase I/II trial evidence [4] Financial Performance - Revenue for Q2 2025 was $5.3 million, a decrease from $11.1 million in Q2 2024, attributed to changes in license and collaboration revenue [6] - Research and development expenses increased to $35.4 million in Q2 2025 from $33.7 million in Q2 2024, driven by external program spending [9] - Selling, general and administrative expenses decreased to $13.5 million in Q2 2025 from $15.8 million in Q2 2024 [10] - The net loss for Q2 2025 was $37.7 million, compared to a net loss of $56.3 million in the same period in 2024 [13][21] Clinical Programs - AMT-260 is in clinical trials for refractory mesial temporal lobe epilepsy, with initial data showing significant seizure reduction [7] - AMT-191 for Fabry disease and AMT-162 for ALS are also in development, with initial data presentations expected in September 2025 and the first half of 2026, respectively [7] Leadership Changes - Kylie O'Keefe's appointment as Chief Customer and Strategy Officer is part of the company's strategy to enhance its commercial and medical affairs [1][4]

Core Viewpoint - uniQure N.V. is set to report its second quarter 2025 financial results on July 29, 2025, and will host a conference call to discuss these results [1]. Company Overview - uniQure is a leading gene therapy company focused on developing transformative therapies for patients with severe medical needs, including a pipeline for treating Huntington's disease, refractory temporal lobe epilepsy, ALS, Fabry disease, and other severe diseases [3]. - The company achieved a significant milestone with the approval of its gene therapy for hemophilia B, marking a historic achievement in genomic medicine after over a decade of research and clinical development [3]. Event Details - The earnings call will be available via webcast on uniQure's website, with a replay archived for 90 days [2]. - Participants wishing to join by phone must register online to receive dial-in details and a personal PIN number [2].

Core Insights - uniQure N.V. has appointed Kylie O'Keefe as Chief Customer and Strategy Officer to lead the commercialization of AMT-130 for Huntington's disease, effective June 6, 2025 [1][2] - The company is preparing for a potential U.S. commercial launch of AMT-130 in 2026, leveraging O'Keefe's extensive experience in rare diseases and gene therapy [2][3] - AMT-130 is positioned as a potential first disease-modifying treatment for Huntington's disease, with a clear path towards accelerated approval in the U.S. [3] Company Overview - uniQure is a leading gene therapy company focused on developing transformative therapies for patients with severe medical needs, including Huntington's disease, ALS, and Fabry disease [4] - The company has achieved significant milestones, including the approval of its gene therapy for hemophilia B, marking a major advancement in genomic medicine [4] Leadership Background - Kylie O'Keefe has a strong background in biopharmaceuticals, having previously served as Chief Commercial Officer at PTC Therapeutics, where she managed global commercial strategy for rare neurology and metabolic products across over 50 countries [2] - O'Keefe's experience includes leading significant commercial launches and developing reimbursement strategies, which will be crucial for uniQure's commercialization efforts [2]

Summary of Conference Call on AMT-130 for Huntington's Disease Company and Industry - **Company**: UniCure - **Industry**: Biotechnology, specifically focused on gene therapy for Huntington's disease Key Points and Arguments 1. **Regulatory Milestone**: UniCure announced alignment with the FDA on key components of the primary statistical analysis plan and CMC requirements for AMT-130, supporting a BLA submission planned for Q1 2026 [6][12][17] 2. **FDA Meetings**: Two Type B meetings with the FDA confirmed that validation of the AMT-130 manufacturing process is feasible using prior knowledge from Hemgenics, streamlining the path towards BLA submission [7][13] 3. **Efficacy Analysis**: The primary efficacy analysis will compare the three-year change in CUHDRS in high-dose AMT-130 patients to a propensity score adjusted external control group from the ENROL HD database [8][14] 4. **Unmet Medical Need**: Huntington's disease is a rare neurodegenerative condition affecting tens of thousands in the US and Europe, with AMT-130 positioned as a potential one-time treatment targeting the disease's root cause [9] 5. **Commercial Readiness**: UniCure is advancing its commercial readiness in phases to support the planned launch of AMT-130 in 2026 [10] 6. **Statistical Methodology**: The FDA supports the use of CUHDRS as an intermediate clinical endpoint, and the analysis will include sensitivity analyses using various datasets to ensure robustness [12][14][70] 7. **Patient Data**: Over 33,000 patients are enrolled in the ENROL HD study, providing a comprehensive dataset for analysis, which is expected to enhance the robustness of the propensity score model [15][36] 8. **Manufacturing Process**: The AMT-130 manufacturing process is similar to that of Hemgenics, with expectations for a process performance qualification to take approximately four to five months [48][49] 9. **Pre-BLA Meeting**: A pre-BLA meeting with the FDA is expected in Q4 2025 to align on submission requirements and confirmatory trial needs [92] 10. **International Strategy**: While prioritizing the FDA, UniCure is also engaging with the EMA regarding next steps for potential approval in Europe [99] Additional Important Content 1. **Forward-Looking Statements**: The call included forward-looking statements that are subject to risks, and actual results may differ materially from those anticipated [4] 2. **Community Engagement**: The support from the Huntington's disease community is acknowledged as instrumental in advancing AMT-130 [18] 3. **Statistical Analysis**: There is no specific P value threshold required by the FDA, but formal statistical analyses will be conducted [58][63] 4. **Sensitivity Analyses**: The consistency of results across various datasets is crucial for regulatory approval, and sensitivity analyses will be performed to validate findings [31][70] 5. **Patient Population**: The patient population for the study includes those with early-stage Huntington's disease, and any slowing of disease progression is considered significant for quality of life [63] This summary encapsulates the critical aspects of the conference call regarding UniCure's AMT-130 and its regulatory journey, highlighting the company's strategic focus and the importance of community and stakeholder engagement.

Core Viewpoint - uniQure N.V. is advancing its investigational gene therapy AMT-130 for Huntington's disease, with a Biologics License Application (BLA) submission planned for the first quarter of 2026, following alignment with the FDA on key components of the statistical analysis plan and Chemistry, Manufacturing and Controls (CMC) information [1][2][8] Regulatory Update - The company has engaged in productive discussions with the FDA, confirming the use of the composite Unified Huntington's Disease Rating Scale (cUHDRS) as an acceptable clinical endpoint for accelerated approval [3][4] - The FDA has agreed to the use of external control data from the ENROLL-HD dataset, which includes approximately 33,000 patients, to enhance the robustness of the statistical analysis plan [4][5] Statistical Analysis Plan - The primary efficacy analysis for the BLA will evaluate the 3-year change in cUHDRS in high-dose AMT-130 patients compared to a propensity score-adjusted external control arm [3] - The company plans to submit an updated statistical analysis plan to the FDA in the second quarter of 2025 [5] Chemistry, Manufacturing and Controls (CMC) - The FDA has agreed that the validation of the AMT-130 manufacturing process can leverage prior knowledge from the etranacogene dezaparvovec-drlb (HEMGENIX) process [6][7] - The proposed drug product release testing plan, including the potency assay, is pending completion of qualification activities [7] Next Steps - Key upcoming milestones include submitting the updated statistical analysis plan in Q2 2025, presenting topline Phase I/II data in Q3 2025, holding a pre-BLA meeting in Q4 2025, and submitting the BLA in Q1 2026 [8][15] Clinical Program Overview - uniQure is conducting two multi-center, dose-escalating, Phase I/II clinical studies to assess the safety and efficacy of AMT-130 in patients with early manifest Huntington's disease [10][11] - AMT-130 has received the FDA's Regenerative Medicine Advanced Therapy (RMAT) designation and Breakthrough Therapy designation, marking it as the first therapy for Huntington's disease to achieve RMAT designation [11] Huntington's Disease Context - Huntington's disease is a rare neurodegenerative disorder affecting approximately 70,000 diagnosed individuals in the U.S. and Europe, with no approved therapies currently available to slow its progression [12] Company Background - uniQure is focused on gene therapy, with a pipeline that includes treatments for Huntington's disease, refractory temporal lobe epilepsy, ALS, Fabry disease, and other severe conditions [13]

~ No serious adverse events and 92% reduction in seizure frequency observed in the first trial participant through first five months of follow up ~ ~ Data to be presented today at Epilepsy Therapies & Diagnostics Development Symposium (ETDD) ~ LEXINGTON, Mass. and AMSTERDAM, May 29, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the presentation of a clinical case study of the fir ...

Company Insights - UBS has a positive outlook on uniQure (QURE.US) and PTC Therapeutics (PTCT.US), giving both companies a buy rating based on optimistic feedback from a neurologist regarding their treatments for Huntington's disease (HD) [1] - The neurologist treats approximately 100 HD patients, with 85% showing symptoms, and believes that if AMT-130 is available soon, about 25% of patients would be prescribed this gene therapy, while 40% would be suitable for PTC-518 [1][2] - The report highlights the importance of upcoming data updates from PTC in Q2 and uniQure in Q3, which are expected to demonstrate sustained efficacy [2] Industry Opportunities and Challenges - The Huntington's disease field presents significant opportunities for both PTC and uniQure due to high patient awareness and strong treatment willingness [2] - There remains a substantial unmet medical need in the HD treatment landscape, with disease-modifying therapies offering important opportunities, although the data released on May 5 is still early and complex [2] - Regulatory developments for uniQure later this quarter are seen as key catalysts that could enhance investor confidence, while the long-term follow-up data from uniQure has shown some supportive evidence despite historical challenges in HD research [2]

uniQure (QURE) closed the last trading session at $12.81, gaining 32.8% over the past four weeks, but there could be plenty of upside left in the stock if short-term price targets set by Wall Street analysts are any guide. The mean price target of $33.50 indicates a 161.5% upside potential.The average comprises 10 short-term price targets ranging from a low of $20 to a high of $70, with a standard deviation of $15.33. While the lowest estimate indicates an increase of 56.1% from the current price level, the ...

uniQure (QURE) Q1 2025 Earnings Call May 09, 2025 08:30 AM ET Company Participants Chiara Russo - Senior Director - IRMatt Kapusta - CEOWalid Abi-Saab - Chief Medical Officer & Member of Management BoardChristian Klemt - CFODebjit Chattopadhyay - Senior Managing DirectorPaul Matteis - Managing Director, Head of Therapeutics ResearchPatrick Trucchio - Managing DirectorJenny Gonzalez-Armenta - Equity Research AssociateUy Ear - Vice PresidentSuzanne van Voorthuizen - Head of Life Sciences Equity Research Confe ...

Financial Data and Key Metrics Changes - Revenue for Q1 2025 was $1.6 million, a decrease of $6.9 million compared to $8.5 million in Q1 2024, primarily due to a reduction in collaboration revenue and contract manufacturing revenue [17][18] - Research and development expenses were $36.1 million in Q1 2025, down from $40.7 million in the same period in 2024, mainly due to decreased employee-related and facility expenses [18] - Cash, cash equivalents, and investment securities totaled $409 million as of March 31, 2025, compared to $367.5 million as of December 31, 2024, reflecting the net proceeds from an $80.5 million follow-on offering [19][20] Business Line Data and Key Metrics Changes - The company is advancing its clinical pipeline with the initiation of three additional studies in refractory temporal lobe epilepsy, Fabry disease, and SOD1 ALS, while continuing enrollment in existing studies [8][9] - Enrollment for the SOD1 ALS trial's first two dose cohorts has been completed, and initial data from the Fabry disease study is expected in the second half of 2025 [9][16] Market Data and Key Metrics Changes - The FDA granted breakthrough therapy designation for AMT-130, highlighting the urgent need for treatments in Huntington's disease [8][12] - The company is preparing for a planned BLA submission and expects to provide a regulatory update later this quarter [21] Company Strategy and Development Direction - The company aims to submit a BLA for AMT-130 in 2025, which is seen as a transformational year with multiple milestones ahead [7][10] - The focus remains on delivering innovative therapies for Huntington's disease, with plans to engage with European regulators for potential commercialization [82] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the data supporting AMT-130 and its potential to slow disease progression, emphasizing the importance of clinical outcomes over surrogate endpoints [26][27] - The company remains optimistic about its interactions with the FDA and the path forward for its clinical programs [41][42] Other Important Information - The company has significantly reduced its cash burn through divestitures and restructuring, providing financial flexibility to advance its pipeline [9][10] - The management highlighted the importance of patient advocacy groups in the development of treatments for Huntington's disease [50][51] Q&A Session Summary Question: Confidence in three-year follow-up data on DUHDRS - Management is confident that the dose-dependent reduction in CUHDRS observed at two years will be maintained at three years [30][32] Question: Inclusion of propensity map scoring in third-quarter update - The analysis will be agreed upon with the FDA before locking the database, and top-line results will be shared accordingly [34] Question: Changes in key personnel after CMC meeting - No material changes in the review team have been noted, and the company remains encouraged about its pipeline [75] Question: Future confirmatory study discussions - The FDA is not ready to discuss confirmatory studies until the BLA submission data is reviewed, but the company does not expect this to delay the BLA filing [105]