uniQure Advances AAV Gene Therapy AMT-130 for Huntington’s Disease

LEXINGTON, MA – July 29, 2025 – uniQure N.V. a leading AAV gene therapy company, today announced its financial results for the second quarter of 2025 and highlighted significant pipeline advancements.

“We delivered tremendous progress across our business in the first half of 2025,” stated Matt Kapusta, uniQure CEO. “With FDA alignment on a BLA pathway and pivotal topline data expected in September, AMT-130 is well-positioned to potentially become the first disease-modifying AAV gene therapy for Huntington’s disease.”

The company reported a narrowed net loss of $37.7 million for Q2 2025 and ended the quarter with $377.0 million in cash, expected to fund operations into the second half of 2027.

Key Developments for AMT-130 (Huntington’s Disease):

• FDA Alignment: In June 2025, uniQure aligned with the FDA on the statistical analysis plan (SAP) and CMC requirements for a planned BLA submission in Q1 2026, leveraging its proprietary AAV5 vector technology.
• Breakthrough Therapy Designation: FDA granted Breakthrough Therapy designation in April 2025 based on Phase I/II evidence.
• Upcoming Milestones: Topline, three-year data from Phase I/II trials expected in September 2025, with BLA submission targeted for Q1 2026.

Advancing Additional Clinical Programs (All leveraging AAV technology):

• AMT-260 (Refractory MTLE): Initial data showed a 92% reduction in seizure frequency from the first patient treated with this AAV9 gene therapy.
• AMT-191 (Fabry Disease): Initial data for this AAV5 gene therapy expected September 5, 2025.
• AMT-162 (SOD1 ALS): Initial data for this AAVrh10 gene therapy expected in H1 2026.