uniQure(US:QURE)2025-06-02 13:30Summary 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.