uniQure (QURE) investor relations material

Executive summary

• Advanced regulatory engagement for AMT-130 in 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 before BLA submission.

• Pipeline progress included 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 patients discontinuing enzyme replacement therapy; dosing paused in some cohorts due to liver enzyme elevations.

• Discontinued development of AMT-162 for SOD1-ALS after dose-limiting toxicity and safety review, reflecting disciplined capital allocation.

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

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

Financial highlights

• Q1 2026 revenue was $3.6 million, up from $1.6 million in Q1 2025, driven by 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, primarily due to increased staffing for commercial planning, professional fees, and IT costs.

• Cash, cash equivalents, and investment securities totaled $586.6 million as of March 31, 2026, expected to fund operations into H2 2029.

Outlook and guidance

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

• Cash runway supports ongoing clinical and operational projects through 2026 and is expected to fund operations into H2 2029; additional funding may be required depending on regulatory outcomes and Phase III trial timing.