uniQure (QURE) Stifel 2024 Healthcare Conference summary

Strategic priorities and progress

• Advanced Huntington's disease (HD) program toward regulatory clarity, initiated three new clinical studies, and implemented capital allocation changes for a 40% reduction in cash burn rate.

• Achieved RMAT designation for HD program and presented two-year follow-up data showing meaningful slowing of disease progression in AMT-130.

• Initiated clinical studies in epilepsy, ALS, and Fabry disease, with all programs progressing as planned.

Clinical data highlights

• Two-year follow-up of 21 patients in AMT-130 showed statistically significant, dose-dependent slowing of disease progression using the Composite Unified Huntington's Disease Rating Scale.

• Statistically significant reduction in CSF neurofilament light chain (NfL), a validated neurodegeneration marker, observed at two years, contrasting with expected natural history increase.

Regulatory engagement and approval pathway

• RMAT designation secured; type B multidisciplinary FDA meeting scheduled for end of month to discuss clinical data, endpoints, and use of external comparators.

• Exploring accelerated approval using external comparators and long-term outcomes from phase 1/2 study, with focus on functional endpoints and robust natural history data.

• Multiple regulatory scenarios considered, including additional follow-up or prospectively defined endpoints, but not necessarily requiring new placebo-controlled studies.

• Expectation of frequent, expedited FDA interactions under RMAT, with significant progress anticipated in the first half of next year.