uniQure N.V. Q3 FY2025 earnings call

• Positive top-line data from pivotal Phase 1/2 study of AMT-130 in Huntington's disease, showing statistically significant slowing of disease progression. Met with FDA in late October, but FDA now has different view on adequacy of data for BLA submission. - AMT-260 for mesial temporal lobe epilepsy: activated 17 recruiting sites in US, completed enrollment of first three patients in first cohort, recruitment expanded, expect updated data in 2026. - AMT-191 for Fabry disease: encouraging results from Phase 1/2a trial, observed supraphysiological alpha-galactosidase A enzyme activity, enrollment in second lower dose cohort completed, third cohort enrolling, expect updated data in 2026. - Voluntarily paused enrollment in Phase 1/2 episode 1 trial of AMT-162 for SOD1 ALS due to dose-limiting toxicity in one patient, will continue to collect and evaluate data. - Committed to stakeholder engagement and education for potential launch of AMT-130, looking at additional potential markets outside US.

For the three months ended September 30, 2025, revenue was $3.7 million compared to $2.3 million in the same period in 2024, an increase of $1.4 million. The increase in revenue resulted from a $1.5 million increase in license revenues and a decrease of $100,000 from collaboration revenues. Cost manufacturing revenues were nil for the three months ended September 30, 2025, compared to $800,000 for the same period in 2024. Research and development expenses were $34.4 million for the three months ended September 30, 2025, compared to $30.6 million during the same period in 2024. Selling, general, and administrative expenses were $19.4 million for the three months ended September 30, 2025, compared to $11.6 million during the same period in 2024. Cash, cash equivalents, and investment securities totaled $649.2 million as of September 30, 2025, compared to $376.5 million as of December 31, 2024.

• Focus on working with FDA to clarify next steps and determine expeditious path to bring AMT-130 to US patients. - Plan to advance discussions with other regulatory agencies including EU and UK.

• Uncertainty introduced by FDA feedback on BLA submission for AMT-130. - Dose-limiting toxicity observed in one patient in AMT-162 trial, resulting in serious adverse event related to AMT-162.

Q: Joseph Thome from Leerink Partners asked about stress-testing the results of AMT-130 and construction of external control arm.

A: Walid Abi-Saab responded about rigorous propensity score matching with enroll-HD, sensitivity testing including different matching, weighting, and comparison to other analyses showing robustness of findings Q: Uy Ear from Mizuho asked about AMT-162, vector and dose difference.

A: Walid Abi-Saab said AMT-162 has different capsid and mode of administration, saw dorsal root ganglia toxicity at middle dose which is threefold higher than low dose Q: Lydia Erdman from Goldman Sachs asked about details to learn from final FDA meeting minutes.

A: Matthew Kapusta said they hope minutes will give sense of FDA concerns and outline for addressing them Q: Joseph Thome from TD Cowen asked about prior meeting minutes confirming ability to file for accelerated approval and detail in meeting minutes.

A: Matthew Kapusta said in November 2024 meeting FDA stated data could serve as primary basis for BLA submission and composite UHDRS could support accelerated approval, but minutes didn't get into specifics of statistical analysis plan Q: Luca Issi from RBC Capital Markets asked about what needs to happen in next few weeks to invest capital in Huntington's.

A: Matthew Kapusta said 100% committed to collaborating with FDA to determine expedited path to submit BLA, believes AMT-130 can benefit patients and will work with FDA to address concerns Q: Yanan Zhu from Wells Fargo asked about ALS program delivery and patient/doctor community motivation.

A: Walid Abi-Saab said ALS program is intrathecal delivery with dorsal root ganglia toxicity, Kylie O'Keefe said patient and doctor community is motivated with huge unmet need and working together to move forward Q: Arabella from H.C. Wainwright asked about EMA or MHRA preliminary feedback on AMT-130 and ex-US submission.

A: Walid Abi-Saab said not yet engaged with UK or EMA, but committed to working with FDA and other agencies to advance submission Q: Paul Matteis from Stifel asked about why FDA's view changed and specific data shared with FDA.

A: Matthew Kapusta said can't comment on details of specific meeting, but hope for clarity from minutes, and data submitted to FDA was consistent with publicly shared data