uniQure (QURE) investor relations material

Key clinical data and trial outcomes

• Phase I/II pooled data for Huntington's showed 75% slowing of progression at three years, with significant results on both composite UHDRS and Total Functional Capacity endpoints, and reduction in neurofilament light, indicating slowed neurodegeneration.

• Safety profile for AMT-130 was favorable, with no significant adverse events attributed to the therapy over three years; most events were procedure-related and manageable.

• Early data for AMT-260 in temporal lobe epilepsy showed a 92% reduction in seizure frequency in the first patient, with six patients dosed in the first cohort and more data expected in Q2.

• Fabry program (AMT-191) demonstrated dose-dependent increases in alpha-galactosidase A, with all 11 patients off enzyme replacement therapy and well-managed lyso-Gb3 levels.

Regulatory and strategic updates

• FDA granted RMAT designation for AMT-130 in early 2024, but ultimately did not accept phase I/II data as the primary basis for BLA due to post hoc analysis concerns.

• Ongoing dialogue with FDA focuses on phase III trial design, with emphasis on ethical and feasible approaches for slow-progressing diseases and leveraging external control data.

• Ex-U.S. regulators (MHRA, EMA) have shown interest following September data; discussions are planned for 2024 to clarify submission pathways and potential for expedited review.

Community engagement and advocacy

• Strong support from patient and physician communities, with advocacy groups actively petitioning the FDA and engaging policymakers to highlight unmet needs and influence trial design.

• Expanded access requests are being considered, with careful attention to ethical considerations and equitable access.