uniQure (QURE) Q2 2025 Earnings Call July 29, 2025 08:30 AM ET Speaker0Good day and thank you for standing by. Welcome to the UniCure Second Quarter twenty twenty five Earnings Conference Call. At this time, participants are in a listen only mode. After the speakers' presentation, there will be a question and answer session. To ask a question during the session, you will need to press 11 on your telephone.You will then hear an automated message advising your hand is raised. To withdraw your question, please ...

uniQure (QURE) came out with a quarterly loss of $0.69 per share versus the Zacks Consensus Estimate of a loss of $0.89. This compares to a loss of $1.16 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of +22.47%. A quarter ago, it was expected that this human gene therapy company would post a loss of $1.07 per share when it actually produced a loss of $0.82, delivering a surprise of +23.36%.Over the last four quarters, the compa ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _______ to _______ Commission file number: 001-36294 uniQure N.V. (Exact name of Registrant as specified in its charter) The Netherlands (Stat ...

Exhibit 99.1 uniQure Announces Second Quarter 2025 Financial Results and Highlights of Recent Company Progress ~ Achieved alignment with the FDA on the AMT-130 statistical analysis plan and CMC requirements to support a planned BLA submission in the first quarter of 2026; ~ Company expects to present AMT-130 topline three-year data in September 2025 ~ ~ Presented case study from first participant treated with AMT-260 for refractory mesial temporal lobe epilepsy showing 92% seizure reduction with no serious ...

~ Achieved alignment with the FDA on the AMT-130 statistical analysis plan and CMC requirements to support a planned BLA submission in the first quarter of 2026; ~ Company expects to present AMT-130 topline three-year data in September 2025 ~ ~ Presented case study from first participant treated with AMT-260 for refractory mesial temporal lobe epilepsy showing 92% seizure reduction with no serious adverse events through first five months of follow up ~ ~ Appointment of Kylie O’Keefe as Chief Customer and S ...

uniQure to host earnings call on Tuesday, July 29, 2025 at 8:30 a.m. ETLEXINGTON, Mass. and AMSTERDAM, July 22, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs will report second quarter 2025 financial results before market open on Tuesday, July 29, 2025. Management will then host a conference call at 8:30 a.m. ET. The event will be webcast under the Events & Presentations section of uniQure’s webs ...

~ Proven biotech executive to lead commercialization of AMT-130 in Huntington’s disease ~ LEXINGTON, Mass. and AMSTERDAM, June 11, 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 appointment of Kylie O’Keefe as Chief Customer and Strategy Officer, effective June 6, 2025. In this role, Ms. O’Keefe will lead the development and execution of uniQure’s global commercialization stra ...

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.

~ Alignment with FDA continues to support Accelerated Approval pathway ~ ~ BLA submission planned for first quarter of 2026 ~ ~ Conference call today at 8:30 a.m. ET ~ LEXINGTON, Mass. and AMSTERDAM, June 02, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today provided a regulatory update on AMT-130, its investigational gene therapy for the treatment of Huntington’s disease. Following recent Typ ...

~ 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 ...