uniQure (QURE) investor relations material

Executive summary

• Advanced AMT-130 for Huntington's disease, with a Type B FDA meeting scheduled and UK MAA submission planned for Q3 2026 following positive MHRA feedback; 36-month data showed significant slowing of disease progression, but FDA requires further study for BLA submission.

• Pipeline progress includes AMT-260 for refractory mesial temporal lobe epilepsy, with phase I/II-A data expected in Q2/Q3 2026, and AMT-191 for Fabry disease showing sustained enzyme activity and all 11 patients discontinuing enzyme replacement therapy.

• Discontinued AMT-162 for SOD1-ALS after safety review due to dose-limiting toxicity; ongoing safety follow-up for dosed patients.

• Collaboration with CSL Behring for HEMGENIX® supply transitioned to Genezen, with related supply agreements terminated in April 2026.

• Implementation of a new ERP system in January 2026, with updates to internal controls.

Financial highlights

• Q1 2026 revenue was $3.6 million, up from $1.6 million in Q1 2025, primarily from increased license revenue and CSL Behring royalties.

• Net loss for Q1 2026 was $53.5 million ($0.85 per share), compared to $43.6 million ($0.82 per share) in Q1 2025.

• R&D expenses decreased to $29.2 million from $36.1 million year-over-year, mainly due to lower program, personnel, and facility costs.

• SG&A expenses rose to $20.1 million from $10.9 million, mainly due to increased staffing, commercial planning, and professional fees.

• Cash, cash equivalents, and investment securities totaled $586.6 million as of March 31, 2026, down from $622.5 million at year-end 2025, with a projected runway into the second half of 2029.

Outlook and guidance

• Key 2026 milestones: Type B FDA meeting and AMT-260 clinical update in Q2/Q3, four-year AMT-130 data and UK MAA submission in Q3.

• Expects to engage with FDA on AMT-191 dosing and pathway in the second half of 2026.

• Cash runway expected to fund operations into the second half of 2029, but additional funding may be required depending on regulatory outcomes and Phase III trial timing.

• Commercial preparations underway in the UK and other geographies, focusing on treatment center readiness, patient engagement, and payer access.