uniQure — Third Quarter 2026 Earnings Summary

Financial Performance:

• Revenue for the three months ended September 30, 2026, was $5.2 million, compared to $3.7 million in the same period in 2025, driven by an increase in license revenue.
• Research and development expenses were $35.5 million for the quarter, up from $34.4 million in 2025; the increase was primarily due to a $3.6 million rise in employee and contractor-related expenses (including share-based compensation), partially offset by a $2.3 million decrease in direct R&D expenses.
• Selling, general, and administrative (SG&A) expenses were $23.9 million for the quarter, an increase from $19.4 million in 2025, primarily attributed to a $3.0 million increase in employee and contractor-related expenses related to recruitment, plus increases in professional fees and IT costs.
• Non-operating items, net, were an expense of $7.6 million for the quarter, a decrease from $20.9 million in 2025, driven by a $6.3 million reduction in pre-funded warrant expenses and a $6.8 million improvement in net interest income.
• Net loss for the three months ended September 30, 2026, was $61.2 million ($0.88 per share), compared to a net loss of $80.5 million ($1.38 per share) in the prior year period.
• Cash, cash equivalents, and investment securities totaled $768.4 million as of September 30, 2026, compared to $622.5 million as of December 31, 2025.

Guidance and Future Outlook:

• The Company expects its current cash resources of $768.4 million will be sufficient to fund projected operating expenses into 2030.
• The FDA granted Priority Review for the BLA of ifezuntirgene inilparvovec (AMT-130) for Huntington's disease, with a PDUFA target action date of May 1, 2027.
• The Company and the FDA have aligned on key elements of the confirmatory study design for ifezuntirgene inilparvovec, with U.S. patient screening expected to begin by the end of 2026.
• The Company plans to initiate patient screening for the confirmatory study in the U.S. by the end of 2026.

Business Segments and Product Lines:

• ifezuntirgene inilparvovec (AMT-130): The BLA was submitted under the Accelerated Approval pathway and supported by 36-month data from 12 high-dose patients compared with a propensity score-matched external control. In the 12 high-dose patients with 48 months of follow-up, the primary endpoint (cUHDRS) showed a 44% slowing of disease progression versus external control (non-significant p=0.144), while Total Functional Capacity (TFC) showed a 61% slowing (nominal p=0.008). An updated 36-month analysis of all 15 high-dose patients showed an 80% slowing on cUHDRS (nominal p=0.005) and a 67% slowing on TFC (nominal p=0.011). The therapy was generally well tolerated across 51 treated patients.
• AMT-260: Enrollment in the second dose cohort of seven patients for refractory mesial temporal lobe epilepsy (MTLE) was completed in the third quarter of 2026, with updated results expected in the first half of 2027.
• AMT-191: Enrollment resumed in August 2026 for mid- and high-dose cohorts in the Phase I/II study for Fabry disease following a pause caused by asymptomatic liver enzyme elevations in two mid-dose patients, which resolved after immunosuppression. Updated results from all dosing cohorts are expected in the first half of 2027.
• The U.K. Medicines and Healthcare products Regulatory Agency (MHRA) validated the Marketing Authorization Application (MAA) for ifezuntirgene inilparvovec in the third quarter of 2026.
• The Company is advancing launch readiness activities, including building field medical, market access, and strategic account teams, and engaging with U.S. Huntington's disease treatment centers.

Market and Competitive Landscape:

• The confirmatory study for ifezuntirgene inilparvovec is designed as a randomized, standard-of-care-controlled trial enrolling approximately 200 patients in a 2:1 ratio to treatment or standard of care, with TFC as the primary endpoint at 36 months.

Risks and Challenges:

• Forward-looking statements highlight risks including the possibility that clinical results may not demonstrate data sufficient to support further development or regulatory approval.
• There is a risk that the FDA may conclude that Phase I/II trial data are insufficient to support approval of ifezuntirgene inilparvovec.
• Accelerated approval, if granted, may be subject to post-approval requirements that are difficult or costly to satisfy.
• Risks include the ability to successfully complete any required confirmatory study, the ability to fund operations, and the continued development and acceptance of gene therapies.

Management Commentary and Tone:

• CEO Matthew Kapusta stated the Company has made significant progress toward delivering the first disease-modifying therapy for Huntington's disease and strongly believes AMT-130 has the potential to deliver meaningful benefit.
• Management expressed confidence in the financial strength providing a runway to deliver on plans and is energized by the build-out of commercial capabilities in preparation for a potential launch.
• The Company highlighted gratitude to patients, families, and investigators for enabling the progress made.

Other Key Points:

• The Company will host an investor conference call and webcast on November 4, 2026, at 8:30 a.m. ET.
• Management plans to attend the Guggenheim 3rd Annual Healthcare Conference (November 10), Stifel 2026 Healthcare Conference (November 11), and UBS Global Biopharma Conference 2026 (November 16).
• The liability related to pre-funded warrants was reduced to zero as of September 30, 2026, down from $12.595 million as of December 31, 2025.
• Total shareholders' equity increased to $294.7 million as of September 30, 2026, from $198.9 million as of December 31, 2025.
• Long-term debt increased slightly to $50.3 million as of September 30, 2026, from $49.7 million as of December 31, 2025.