uniQure Q3 Earnings Call Highlights

Key Points

• The FDA accepted uniQure’s AMT-130 application for Huntington’s disease and granted Priority Review, with a target decision date of May 1, 2027; the U.K. regulator also validated its marketing application. The filing cites 36-month results showing slower disease progression versus matched external controls, while longer-term data come from a relatively small patient group.
• uniQure plans a randomized, approximately 200-patient confirmatory trial, with screening expected to begin before the end of 2026. Ahead of a potential 2027 launch, it is preparing treatment centers for the one-time neurosurgical therapy, with more than 15 centers expected to be operational.
• Third-quarter revenue rose to $5.2 million from $3.7 million a year earlier, while R&D and SG&A expenses also increased. The company reported $768.4 million in cash, equivalents and investments and expects its funds to support operations into 2030.

uniQure NASDAQ: QURE said the FDA has accepted its Biologics License Application for AMT-130, an investigational treatment for Huntington’s disease, and granted the application Priority Review with a target action date of May 1, 2027. The U.K. Medicines and Healthcare products Regulatory Agency has also validated the company’s Marketing Authorization Application, according to management.

Chief Executive Officer Matt Kapusta said the submissions represent the first marketing applications for a disease-modifying therapy in Huntington’s disease. The company is simultaneously preparing a confirmatory study and building commercial capabilities ahead of a potential U.S. launch in 2027.

AMT-130 Review Based on Three-Year Data

Chief Medical Officer Walid Abi-Saab said the U.S. and U.K. applications are based on a 36-month analysis presented in September 2025. In that analysis, the high dose of AMT-130 showed a 75% slowing of disease progression on the composite Unified Huntington’s Disease Rating Scale, or cUHDRS, and a 60% slowing on Total Functional Capacity, or TFC, compared with a propensity score-matched external control. The company said both results were statistically significant.

Abi-Saab said uniQure has also reported follow-up data extending to 48 months after a single administration of AMT-130. Across the company’s Phase I/II studies, TFC declined by less than one-tenth of a point annually among high-dose recipients, a trend that was maintained through four years of follow-up, he said. He noted, however, that those results came from a relatively small patient group.

The FDA has tentatively indicated that it plans to discuss the application at an advisory committee meeting, although the timing and committee have not been determined. Abi-Saab said uniQure would not comment on specific FDA information requests while the BLA is under active review unless developments become material.

The company said it expects to provide a customary day-120 safety update to the FDA. If patients in the confirmatory study have been treated by the relevant cutoff date, their available data would be included in that update, Abi-Saab said.

Confirmatory Study Planned Across Multiple Regions

uniQure said it aligned with the FDA earlier this year on key elements of an AMT-130 confirmatory study. The randomized trial is expected to enroll about 200 patients and assign participants in a 2:1 ratio to high-dose AMT-130 or standard of care. Its primary endpoint will be TFC at 36 months.

After the 36-month period, participants assigned to standard of care will be offered AMT-130 in a separate open-label study. The protocol has been submitted to U.S. institutional review boards, and uniQure expects patient screening to begin before the end of 2026.

The global study is expected to include sites in North America, Europe and Latin America. Management said it plans to enroll in markets where AMT-130 may not be commercially available during the trial, an approach intended to reduce the risk that standard-of-care participants leave the study to seek treatment.

During the question-and-answer session, management said it has accounted for potential standard-of-care dropouts in its sample-size planning and analysis plans. The company also said it is working with the Huntington Study Group to help standardize TFC administration and is conducting site training intended to support consistent measurements.

Kapusta said the company expects to have the study “well underway” by the potential approval date and believes it can demonstrate a credible path to completing the trial in a timely manner. If AMT-130 were not approved, he said uniQure would assess the reasons and next steps, but added that the company believes it has an ethical commitment to continue the study for the Huntington’s disease community.

Commercial Preparation Focuses on Treatment-Center Capacity

Chief Customer and Strategy Officer Kylie O’Keefe said early commercial uptake for AMT-130 could be constrained more by treatment-center capacity than by demand. AMT-130 is designed as a one-time therapy delivered through a neurosurgical procedure, requiring Huntington’s disease neurology expertise, image-guided neurosurgical experience, and infrastructure for product handling and patient follow-up.

The company has identified priority treatment centers in the U.S. and U.K. and is focusing its readiness work on neurosurgical training, product handling, patient evaluation and referral pathways. O’Keefe said uniQure expects more than 15 centers to be operational at a potential launch, although the final number has not been set. The company is evaluating whether that figure could range from roughly 25 to 30 centers.

uniQure said it is prioritizing institutions that combine Huntington’s disease expertise with ClearPoint capabilities, which management said could help patients move more seamlessly from evaluation to surgery and follow-up care. O’Keefe said capacity can vary substantially by institution, ranging from approximately two procedures per week to one per month.

The company is also engaging payers and institutions before a potential approval. O’Keefe said the work includes mapping pharmacy and therapeutics committee processes and educating payers on Huntington’s disease, AMT-130’s clinical package and the economics of a one-time therapy. In the U.K., uniQure has begun scientific engagement with NICE and NHS England and expects to submit its dossier following any potential MHRA approval.

Other Pipeline Programs Advance

Outside Huntington’s disease, uniQure completed enrollment in the second, higher-dose cohort of its Phase I/IIa study of AMT-260 in refractory mesial temporal lobe epilepsy. The company previously presented six-month data from the first low-dose cohort of six patients and has now enrolled seven patients in the higher-dose cohort. Updated results from both cohorts are expected in the first half of 2027.

Enrollment also resumed in the mid- and high-dose cohorts of the Phase I/II study of AMT-191 in Fabry disease. Dosing in those cohorts had been paused under the protocol after asymptomatic liver enzyme elevations that resolved following immunosuppression. The company expects updated data across all three dosing cohorts in the first half of 2027.

Third-Quarter Financial Results

For the third quarter ended Sept. 30, uniQure reported revenue of $5.2 million, compared with $3.7 million in the same period of 2025. The increase was attributed to higher license revenue.

• Research and development expense was $35.5 million, compared with $34.4 million a year earlier.
• Selling, general and administrative expense was $23.9 million, compared with $19.4 million in the prior-year period.
• Cash, cash equivalents and investment securities totaled $768.4 million as of Sept. 30, up from $622.5 million at the end of 2025.

Chief Financial Officer Christian Klemt said higher SG&A spending reflected hiring, professional fees and information technology investments supporting potential commercial launches. He said the company expects spending to rise as it prepares for launch but intends to maintain discipline. uniQure expects its cash, cash equivalents and investment securities to fund operations into 2030.

About uniQure (NASDAQ:QURE)

uniQure N.V. is a biotechnology company focused on developing gene therapies for serious and life-threatening diseases. The company uses adeno-associated virus (AAV) vectors and other genetic medicine technologies to deliver therapeutic genes intended to address the underlying causes of disease.

uniQure's best-known product is HEMGENIX, a gene therapy for adults with hemophilia B caused by factor IX deficiency. HEMGENIX was developed by uniQure and is commercialized by CSL Behring under a global licensing agreement.

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