uniQure (QURE) 2024 Wells Fargo Healthcare Conference summary

Platform and pipeline overview

• Pioneering AAV gene therapy with over 25 years of experience and two approved products, focusing on CNS and liver-directed therapies.

• Four clinical programs are active, with Huntington's disease as the lead.

Corporate strategy and capital management

• Recent sale of manufacturing facility and headcount reduction aim to conserve capital and streamline operations.

• Focus on maintaining flexibility for expedited development of the Huntington's program.

• No traditional equity raise since 2019, prioritizing capital efficiency and value creation.

Huntington's disease program and regulatory strategy

• Utilizes propensity-matched external controls to reduce bias and variability in clinical outcome comparisons.

• RMAT designation enables earlier and more frequent FDA interactions, with a kickoff meeting expected in the second half of the year.

• Long-term clinical outcomes data, rather than surrogate biomarkers, are emphasized for potential accelerated approval.

• FDA openness to natural history comparators increases confidence in expedited approval discussions.

• Multiple regulatory paths are possible, ranging from expedited approval based on phase I/II data to a full phase III trial.