uniQure CEO Defends Huntington’s Therapy Data, Sees Opportunity Ahead of FDA Decision

Key Points

• uniQure CEO Matt Kapusta defended AMT-130’s four-year data analysis, saying the newer natural-history dataset was scientifically appropriate and pre-specified. He highlighted high-dose patients’ average annual loss of less than 0.1 Total Functional Capacity points, compared with more than one point among untreated patients; the FDA will review the application using the original dataset.
• The company is finalizing plans for a 200-patient confirmatory study measuring Total Functional Capacity at 36 months. It expects patient eligibility to closely match the earlier trial and aims to show the study is underway ahead of the FDA decision.
• AMT-130 remains under regulatory review, with the interviewer citing May 1 as the target date for an FDA decision. uniQure is also preparing for a potential launch, identifying about 60 U.S. Huntington’s disease centers of excellence equipped for treatment.

uniQure NASDAQ: QURE Chief Executive Matt Kapusta said investors are focused on regulatory risk surrounding its experimental Huntington’s disease therapy, AMT-130, but may be underestimating the need for treatment and the potential commercial opportunity.

Speaking at Guggenheim’s 3rd Annual Healthcare Innovation Conference, Kapusta said the company plans to discuss its commercial strategy further as it approaches the end of the year and early the following year. The conference interviewer cited a May 1 target date for the U.S. Food and Drug Administration’s decision on the therapy.

Kapusta also defended uniQure’s use of an updated natural-history dataset in a four-year analysis, discussed plans for a confirmatory study and outlined preparations for a potential launch. AMT-130 remains under regulatory review.

CEO Defends Data Comparison

Kapusta said the FDA will review data in uniQure’s biologics license application based on the original Enroll-HD dataset, referred to as the A cut, which the company presented in September 2025. The interviewer noted that a subsequent analysis using the B cohort produced a statistically non-significant result on the composite Unified Huntington’s Disease Rating Scale, or cUHDRS.

The CEO said using the newer dataset was scientifically appropriate because it contained an additional two and a half years of follow-up on existing patients and approximately 6,000 newly recruited patients. The company pre-specified its use of the latest data cut, he said, after the FDA raised concerns about a lack of pre-specification in the proposed four-year analysis.

Kapusta said missing data provided important context for interpreting the newer analysis. He also pointed to Total Functional Capacity, or TFC, a measure of how the disease affects daily activities and the primary endpoint for the planned confirmatory study.

According to Kapusta, high-dose AMT-130 patients lost less than 0.1 TFC points per year on average over 48 months, while untreated patients lost more than one point. He said other components of cUHDRS involve performance-based tests that can be more variable, and cautioned against over-interpreting changes in a single year.

Kapusta said the high-dose group performed more than one point better on cUHDRS at 48 months than the low-dose group. The company believes the low dose has a minimal clinical effect, he said.

Confirmatory Study Plans

The interviewer described the planned confirmatory study as enrolling 200 patients and measuring TFC at 36 months. Kapusta said uniQure has not disclosed its statistical powering assumptions and is still finalizing the analysis plan. The company did not simply apply the assumptions from its smaller phase 1/2 study, he said.

Patient eligibility criteria are expected to be nearly identical, if not identical, to those in the earlier study. Kapusta said he expects demand for the trial despite the need for MRI screening. The company has previously disclosed a screening failure rate of 50% to 60% in the phase 1/2 study, he added.

Kapusta said uniQure has no specific U.S. enrollment target before the FDA decision date, but wants to demonstrate that the study is well underway through open sites, patients in screening and enrolled participants. If the product is approved, the company expects to emphasize enrollment outside the U.S., he said.

Regulatory Review and Potential Launch

An FDA advisory committee meeting remains at the agency’s discretion, Kapusta said. If one occurs, he expects the Huntington’s disease community’s voice to be represented, though he declined to say whether trial participants would attend.

Kapusta said the U.K. review process differs from the FDA’s and could take more than three additional months. Unlike the U.S. process, he said, the U.K. review has no specific decision date and can include pauses while the sponsor answers regulators’ questions.

For a potential launch, Kapusta said treatment-center capacity will vary, with some sites potentially performing one procedure a week and others one a month. He said uniQure has identified about 60 Huntington’s disease centers of excellence with ClearPoint equipment and roughly 100 U.S. sites overall with the equipment.

If the eventual product label requires volumetric MRI, Kapusta said uniQure would work with treatment centers to make it available and prevent it from becoming an obstacle for patients.

About uniQure (NASDAQ:QURE)

uniQure N.V. is a biotechnology company that develops gene therapies for serious genetic and other diseases. The company uses adeno-associated virus (AAV) vectors to deliver genetic material to patients' cells, with research and development operations in the United States and the Netherlands. Its programs have targeted conditions including Huntington's disease, epilepsy and hemophilia B. uniQure developed the gene therapy now known as Hemgenix for hemophilia B; it is commercialized by CSL Behring under a partnership.

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