uniQure's Q2 2025: Navigating Contradictions in AMT-130 Strategy, Regulatory Path, and Market Expansion

Regulatory path and strategy for AMT-130, data analysis methodology for natural history comparator, market expansion strategy, company strategy, and regulatory strategy and timeline are the key contradictions discussed in uniQure's latest 2025Q2 earnings call.



AMT-130 Pivotal Data and Regulatory Alignment:
- uniQure's AMT-130 is expected to have pivotal top-line data in September, with a BLA submission planned in Q1 2026 for potential U.S. commercial launch later that year.
- The company has achieved regulatory alignment with FDA, including Breakthrough Therapy and RMAT designations, and alignment on statistical analysis plans and manufacturing processes.

Advancements in Clinical Pipeline:
- Encouraging early data from AMT-260 for mesial temporal lobe epilepsy showed a 92% reduction in seizure frequency, and initial Fabry data is expected at the ICIEM conference in September.
- Progression in clinical trials reflects improvements in manufacturing and regulatory collaboration, positioning the company for significant data updates in the second half of 2025.

Financial Performance and Cash Position:
- Revenue for Q2 2025 was $5.3 million, down from $11.1 million in the same period of 2024.
- The decrease was primarily due to a decrease in collaboration revenue and contract manufacturing of HEMGENIX. The company's cash position remains strong at $377 million, supported by a successful follow-on offering.

Preparation for Commercial Launch:
- uniQure has appointed an experienced leader for commercial planning and is actively recruiting key roles in medical affairs and operations.
- The company is making disciplined investments in preparation for a potential 2026 launch of AMT-130, with an integrated launch strategy under development.