uniQure (QURE) investor relations material

Executive summary

• Achieved regulatory alignment with FDA and MHRA for AMT-130, enabling a BLA submission for Huntington's disease under the Accelerated Approval pathway based on three-year data, with regulatory submissions on track for Q3 2026 in both the U.S. and U.K.

• Early promising data reported for AMT-260 (refractory mesial temporal lobe epilepsy) and AMT-191 (Fabry disease), supporting continued development; AMT-191 showed dose-dependent enzyme activity increases and manageable safety, with all patients withdrawn from enzyme replacement therapy.

• Strengthened balance sheet through follow-on offerings totaling up to $259 million, extending operational runway and cash resources into 2030.

• Commercial readiness efforts intensified for AMT-130, with deep engagement at treatment centers and payers in the U.S. and U.K.

• Net loss for Q2 2026 was $81.1 million, up from $37.7 million in Q2 2025, driven by higher operating and non-operating expenses.

Financial highlights

• Q2 2026 revenue was $5.8 million, up from $5.3 million in Q2 2025, driven by increased license revenue, primarily from royalties on HEMGENIX® sales.

• Research and development expenses decreased to $34 million from $35.4 million year-over-year, reflecting lower facility and personnel costs, partially offset by higher direct R&D spend on pipeline programs.

• Selling, general, and administrative expenses rose to $17.4 million from $13.5 million, mainly due to increased staffing for commercial launch preparations and higher employee-related costs.

• Cash, cash equivalents, and investment securities totaled $811.9 million as of June 30, 2026, up from $622.5 million at year-end 2025.

• Net loss per share for Q2 2026 was $1.22, compared to $0.69 in Q2 2025.

Outlook and guidance

• BLA submission for AMT-130 on track for Q3 2026 in the U.S. and U.K.; confirmatory study to begin promptly after FDA alignment.

• Four-year AMT-130 data from Phase I/II studies to be presented in September 2026; updated results for AMT-260 and AMT-191 expected in the first half of 2027.

• Cash runway expected to fund operations, confirmatory trials, and commercial launches into 2030.

• Additional $100 million available under the Hercules loan facility, contingent on BLA approval for AMT-130.