uniQure N.V. Q4 FY2025 earnings call

Matt Capista discussed Unicure's mission to deliver transformative medicines, including progress with AMT130 for Huntington's disease, feedback from the FDA regarding Phase 3 study recommendation, and plans to engage constructively with the FDA. Waleed Abisab spoke about AMT130's data, differences in interpretation of results with the FDA, progress with Fabry disease's AMT-191, AMT-260 for mesiotemporal epilepsy, and pipeline updates. Kylie O'Keefe talked about engagement with the Huntington's disease patient community, market opportunities for AMT260 and AMT191, and building global strategy. Christian Klempt provided financial highlights for 2025.

Revenue for the year ended December 31st, 2025 was $16.1 million compared to $27.1 million in 2024. The decrease was primarily driven by a $10.7 million decrease in collaboration revenue and a $6.1 million decrease in contract manufacturing revenues offset by a $5.8 million increase in license revenues. Cost of contract manufacturing revenues was nil for 2025 compared to $17.1 million in 2024. Research and development expenses were $140.7 million in 2025 compared to $143.8 million in 2024, with a decrease due to various factors. Selling, general, and administrative expenses were $65.5 million in 2025 compared to $52.7 million in 2024, with an increase due to several factors. Cash, cash columns, and investment securities totaled $622.5 million as of December 31, 2025, compared with $367.5 million as of December 31, 2024.

Matt Capista mentioned priorities for 2026 include constructively engaging with regulatory authorities, advancing pipeline programs, and generating high-quality data. They are focused on defining the path forward for AMT130, advancing other programs, and have a strong balance sheet to execute priorities.

Potential differences between actual results and forward-looking statements, factors described in Unicure's most recent SDC filings could cause material differences. Concerns about the burden and risks of a multi-year sham-controlled study for AMT130 in Huntington's disease, including potential ethical issues and impact on patients.

Q: Are there any paths to push agenda beyond traditional FDA channels and about four-year data?

A: Other avenues include outside US, leveraging patient community advocacy. Informed FDA of amending SAP for four-year analysis and will present totality of data.

Q: Concern about morbidity from burr hole procedures and strategy for Type B meeting?

A: Addressed patient safety, educated FDA on safety profile. Type B meeting goal is to discuss Phase 3 trial designs and leverage EnrollHD database.

Q: Thoughts on Phase 3 design enrollment and partnering?

A: Premature to talk about enrollment logistics, focus is on defining design. Partnering considered later when design and investment clear.

Q: Color on potential Phase 3 scenarios and cost?

A: Premature to detail, FDA recommended double-blind sham-controlled trial, cash runway considered but details too early.

Q: Regions for XUS discussions and epilepsy program update?

A: Looking at multiple jurisdictions, considering factors like epidemiology. Epilepsy program will update on six patients in first cohort with six months follow-up, phase one study so no set expectation yet.

Q: XUS commercial opportunity?

A: Premature on commercial opportunity, but will take learnings from Roche and others.

Q: Totality of sticking points with FDA and why sham controlled required?

A: FDA raised post hoc analysis and lack of signal in 12-month data. Sham control is gold standard but Unicure believes in leveraging natural history data.

Q: Why FDA requires sham study and commitment to move forward?

A: FDA seeks to minimize bias. Unicure is committed to moving forward if feasible and ethical.

Q: Discussions with FDA on biomarkers and Phase 3 design?

A: Premature, will provide details after aligning with FDA in second quarter.

Q: Elaboration on avoiding Phase 3 design too difficult for patients?

A: Concerns about length of sham study, risk to patients, and desire to work with FDA for flexible design.

Q: Biggest pushback from FDA and dedication to sham control trial?

A: FDA seeks maximum bias reduction. Unicure will consider feasibility and patient support to move forward with sham control trial if feasible.