uniQure N.V. SEC 10-Q Report
uniQure N.V., a leading gene therapy company, has released its Form 10-Q report for the second quarter of 2025. The report provides a comprehensive overview of the company's financial performance and key business developments, highlighting significant progress in its clinical programs and strategic initiatives.
Financial Highlights
Total Revenues: $5.3 million, a decrease of $5.9 million compared to the same period in 2024, primarily due to the absence of contract manufacturing and collaboration revenues.
Net Loss: $37.7 million, an improvement of $18.6 million compared to the same period in 2024, driven by reduced operating expenses and increased license revenues.
Basic and Diluted Net Loss Per Ordinary Share: $0.69, reflecting a decrease in net loss per share compared to $1.16 in the same period in 2024.
Business Highlights
Huntington’s Disease Program (AMT-130)
The company announced that the FDA granted Breakthrough Therapy designation to AMT-130 for the treatment of Huntington’s disease, supported by clinical data from ongoing Phase I/II trials. The company plans to present 3-year follow-up data in the third quarter of 2025 to support a potential Biologics License Application (BLA) submission.
The FDA agreed that data from ongoing Phase I/II studies of AMT-130, compared to a natural history external control, may serve as the primary basis for a BLA submission under the FDA’s accelerated approval pathway. The company submitted the final statistical analysis plan to the FDA in July 2025.
The company plans to initiate a fourth cohort in the third quarter of 2025 as part of the ongoing clinical development of AMT-130, evaluating the safety of high-dose administration in patients with lower striatal volumes.
Temporal Lobe Epilepsy Program (AMT-260)
The company is conducting a Phase I/IIa clinical trial (GenTLE) for AMT-260 in the United States. Initial safety and exploratory efficacy data from the first treated patient showed a 92% reduction in seizure frequency with no serious safety events.
Fabry Disease Program (AMT-191)
The company is conducting a Phase I/IIa clinical trial for AMT-191, with the FDA granting Orphan Drug and Fast Track Designations. The independent data monitoring committee recommended proceeding with enrollment in the second cohort after reviewing safety data.
Amyotrophic Lateral Sclerosis (AMT-162)
The company is conducting a Phase I/II trial (EPISOD1) for AMT-162 in the United States. The independent data monitoring committee recommended proceeding with enrollment of the second cohort after reviewing safety data from the first cohort.
New Leadership Appointment
In June 2025, the company appointed Kylie O’Keefe as Chief Customer and Strategy Officer, responsible for leading all commercial and medical affairs.
Geographical Performance
The company’s operations are primarily focused in the United States, with significant regulatory interactions and clinical trials being conducted in this region.
Sales Units and Production
Following the divestment of commercial manufacturing activities in Lexington, MA, the company now relies on third-party suppliers for GMP material used in clinical programs.
SEC Filing: uniQure N.V. [ QURE ] - 10-Q - Jul. 29, 2025