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 ...

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 ...

uniQure (QURE) Q1 2025 Earnings Call May 09, 2025 08:30 AM ET Speaker0 Good day and thank you for standing by. Welcome to the UniCure First Quarter twenty twenty five Earnings Conference Call. At this time, all 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'll need to press 11 on your telephone. You will then hear an automated message advising your hand is raised. To answer your question, press ...

UniQure (QURE) came out with a quarterly loss of $0.82 per share versus the Zacks Consensus Estimate of a loss of $1.07. This compares to loss of $1.36 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of 23.36%. A quarter ago, it was expected that this human gene therapy company would post a loss of $0.44 per share when it actually produced a loss of $1.50, delivering a surprise of -240.91%.Over the last four quarters, the company ...

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 March 31, 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 (Sta ...

Exhibit 99.1 uniQure Announces First Quarter 2025 Financial Results and Highlights of Recent Company Progress ~ AMT-130 granted Breakthrough Therapy designation by FDA~ ~ Initial safety data from third cohort of Phase I/II study show AMT-130 continues to be generally well-tolerated, with no treatment-related serious adverse events ~ ~ Held Type B FDA meetings in the first and second quarters of 2025 to advance BLA preparations for AMT- 130; regulatory update expected in the second quarter of 2025 ~ ~ AMT-26 ...

~ AMT-130 granted Breakthrough Therapy designation by FDA ~ ~ Initial safety data from third cohort of Phase I/II study show AMT-130 continues to be generally well-tolerated, with no treatment-related serious adverse events ~ ~ Held Type B FDA meetings in the first and second quarters of 2025 to advance BLA preparations for AMT-130; regulatory update expected in the second quarter of 2025 ~ ~ AMT-260 clinical data from first patient to be presented at Epilepsy Therapies & Diagnostics Development Symposium o ...