uniQure Announces Second Quarter 2025 Financial Results and Highlights of Recent Company Progress

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]