uniQure (QURE) Q1 2026 earnings summary

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; positive 36-month data showed significant slowing of disease progression, but FDA requires more data for BLA submission.

• Progressed AMT-260 for temporal lobe epilepsy, with enrollment on track and first cohort data to be presented in June 2026.

• AMT-191 Fabry disease program showed sustained enzyme activity and all patients discontinued enzyme replacement therapy; dosing paused at higher doses due to liver enzyme elevations and dose-limiting toxicities.

• Discontinued development of AMT-162 for SOD1-ALS after safety review and dose-limiting toxicity.

• 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, 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, with a projected runway into the second half of 2029.

Outlook and guidance

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