uniQure(US:QURE)2025-11-10 14:30Financial Data and Key Metrics Changes - Revenue for Q3 2025 was $3.7 million, an increase from $2.3 million in Q3 2024, resulting in a $1.5 million increase in license revenues and a slight decrease in collaboration revenues [17] - Research and development expenses rose to $34.4 million in Q3 2025 from $30.6 million in Q3 2024, driven by a $10.1 million increase in direct R&D expenses, primarily for BLA submission preparation [18] - Selling, general, and administrative expenses increased to $19.4 million in Q3 2025 from $11.6 million in Q3 2024, mainly due to higher employee-related expenses and professional fees [18] - Cash, cash equivalents, and investment securities totaled $649.2 million as of September 30, 2025, up from $376.5 million at the end of 2024, primarily due to net proceeds from public offerings [19] Business Line Data and Key Metrics Changes - AMT-130 for Huntington's disease showed a statistically significant 75% slowing of disease progression at three years compared to an external control, meeting the pivotal study's primary endpoint [9] - AMT-260 for mesial temporal lobe epilepsy has activated 17 recruiting sites in the U.S. and completed enrollment of the first three patients in the first cohort, with updated data expected in the first half of 2026 [11] - AMT-191 for Fabry disease demonstrated supra-physiological alpha-galA enzyme activity in treated patients, with all successfully withdrawn from enzyme replacement therapy [12] Market Data and Key Metrics Changes - The company is focusing on building a foundational strategy for the U.S. market for AMT-130 while also exploring potential markets in the European Union and the United Kingdom [16] Company Strategy and Development Direction - The company remains committed to working with the FDA to clarify next steps for AMT-130 and is also planning discussions with other regulatory agencies [20] - The management emphasizes the importance of stakeholder engagement and education to prepare for a potential launch of AMT-130 [15] Management's Comments on Operating Environment and Future Outlook - Management expressed disappointment regarding the FDA's feedback on the BLA submission for AMT-130 but remains confident in the data and its potential to benefit patients [5][10] - The company plans to engage urgently with the FDA to discuss the next steps and remains committed to transparency and collaboration [6][11] Other Important Information - Enrollment in the phase I-II trial of AMT-162 for SOD1 ALS has been paused due to safety concerns, with ongoing data collection from treated patients [13] Q&A Session Summary Question: Treatment effect and external control arm construction - The company conducted rigorous propensity score matching with Enroll-HD and performed various sensitivity analyses to ensure robustness of findings [27] Question: Details on AMT-162 and dose differences - The middle dose of AMT-162 was about three-fold higher than the low dose, and the company is monitoring data to determine next steps [31] Question: Expectations from final meeting minutes with the FDA - The company hopes the minutes will outline FDA concerns and provide guidance on addressing those in future meetings [34] Question: Confirmation of prior meeting minutes regarding accelerated approval - The company confirmed that previous meetings indicated the data could support a BLA submission and that the composite UHDRS was an acceptable endpoint [37] Question: Future investment in Huntington's program - The company is committed to collaborating with the FDA to find an expedited path for BLA submission, emphasizing the urgency of patient needs [41] Question: EMA or MHRA feedback on AMT-130 - The company has not yet engaged with EMA or MHRA but plans to prioritize discussions with the FDA first [47]