uniQure (QURE) investor relations material

Executive summary

• Regulatory alignment with FDA and MHRA for AMT-130 enables BLA submission for Accelerated Approval in Q3 2026, with U.K. submission also planned; pivotal four-year data expected in September 2026.

• Early clinical data from Fabry disease (AMT-191) and refractory temporal lobe epilepsy (AMT-260) programs showed promising safety and efficacy, supporting continued development; dosing in some AMT-191 cohorts paused due to transient liver enzyme elevations.

• Financial position strengthened by follow-on offerings totaling up to $259 million, with cash, cash equivalents, and investment securities reaching $811.9 million as of June 30, 2026, funding operations into 2030.

• 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.

• Major financing event: $242.7 million raised in June 2026 via a follow-on public offering.

Financial highlights

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

• Research and development expenses decreased to $34.0 million from $35.4 million year-over-year, reflecting lower facility and personnel costs, partially offset by higher direct R&D on pipeline programs.

• Selling, general, and administrative expenses rose to $17.4 million from $13.5 million, mainly due to increased staffing, share-based compensation, and IP fees.

• 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 cash used in operating activities for H1 2026 was $66.8 million, down from $84.0 million in the prior year period.

Outlook and guidance

• BLA submission for AMT-130 on track for Q3 2026, with U.K. regulatory submission also planned and confirmatory study to begin 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 H1 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.