uniQure N.V. Q2 FY2025 earnings call

• Clinical Advancement: AMT-130 is advancing towards being a disease-modifying therapy for Huntington's disease, with productive interactions with the FDA, including breakthrough therapy designation and regulatory alignment. Broader pipeline includes progress in AMT-191 for Fabry disease, AMT-260 for mesial temporal lobe epilepsy, and AMT-162 for SOD1-ALS.
• Regulatory Alignment: AMT-130 has breakthrough therapy designation, and alignment with the FDA on statistical analysis plan and CMC requirements for a planned BLA submission in Q1 2026.
• BLA Readiness: Manufacturing of 2-scale pre-PPQ GMP batches complete, formal PPQ campaign initiated, and final statistical analysis plan submitted to the FDA.
• Commercial Launch Preparation: Appointed Chief Customer and Strategy Officer, recruiting key roles across various critical areas, and developing an integrated launch strategy.

Revenue for the second quarter of 2025 was $5.3 million, down from $11.1 million in the same period in 2024. The decrease in revenue resulted from a $3.4 million increase in license revenue, a $7.1 million decrease from collaboration revenue, and a $2.1 million decrease from contract manufacturing of HEMGENIX for CSL Behring. Research and development expenses were $35.4 million for the three months ended June 30, 2025, compared to $33.7 million in the same period in 2024. Selling, general and administrative expenses were $13.5 million for the three months ended June 30, 2025, compared to $15.8 million in the same period in 2024. Cash, cash equivalents and investment securities totaled $377 million as of June 30, 2025.

• Anticipate top line data from AMT-130 in September. Planned BLA submission for AMT-130 in Q1 2026, with potential 2026 launch.
• Broader pipeline: Initial Fabry data expected at ICIEM conference in September, and AMT-162 safety and biomarker data in H1 2026.

• Uncertainty in regulatory outcomes due to varying interpretations of clinical data.
• Potential differences in trial results and variability in how data is perceived by regulatory bodies.

Q: Does the FDA expect a minimum threshold for clinical benefit versus the ENROLL-HD or cUHDRS?

A: In discussions with the FDA, a minimum clinical effect has not been requested nor included in the SAP plan. The 3-year data is expected to show substantial evidence supporting accelerated approval after FDA review.

Q: Could you walk us through the AMT-130 procedure and what this would look like from a patient's perspective?

A: The procedure is technically not complicated, involving an MRI for planning, neurosurgeon insertion of probes, infusion of the product, with patients typically admitted for 24 hours and recovering quickly. Eligible patients are Stage II and III Huntington's disease patients.

Q: Can you give any color on what you plan to talk about and learn from the pre-BLA meeting with the FDA in 4Q?

A: We will meet with the FDA to share top line results from AMT-130's Phase III data, CMC activities, and discuss logistics. Feedback from the meeting will be communicated to the market.

Q: On the Huntington's commercial opportunity, how many sites in the U.S. are capable of doing the administration procedure?

A: There are approximately 50-55 sites in the U.S. capable of the procedure. Estimated prevalence of eligible patients in the U.S. is based on diagnosed Huntington's disease patients, with many thousands potentially eligible.