uniQure (QURE) Leerink’s Global Healthcare Conference 2025 summary

Recent progress and strategic goals

• Four clinical programs are underway: Huntington's disease (lead), temporal lobe epilepsy, Fabry disease, and SOD1 ALS, all in active clinical development with ongoing dosing.

• Significant news flow is expected over the next 6–18 months, including initial data for Fabry in H2 2024 and for epilepsy and ALS in H1 2026.

• Cash reserves of $445 million are projected to fund operations through the launch of AMT-130 in the US and into late 2027.

Huntington's disease program and regulatory pathway

• AMT-130 received RMAT designation and showed an 80% slowing of disease progression at two years in a propensity-weighted analysis.

• FDA supports an accelerated approval pathway using external natural history controls and the Composite Unified Huntington's Disease Rating Scale as an endpoint.

• Upcoming FDA meetings in Q1 and Q2 2024 will address CMC requirements and the statistical analysis plan for BLA submission.

• Perioperative steroid cohort data will inform the immunosuppression regimen for the BLA, with 90-day safety data expected in time for submission.

• Launch strategy includes identifying 55+ US sites with neurosurgical expertise and a center of excellence model.

Commercial and competitive landscape

• Substantial demand is anticipated for the first disease-modifying Huntington's therapy due to high unmet need.

• Preparations include market education, KOL engagement, patient services, and reimbursement planning.

• The market is expected to support multiple therapeutic modalities, with payers likely to cover several options for this rare disease.