uniQure N.V. filed a Current Report on Form 8-K on September 29, 2026, disclosing updated clinical data for its Huntington's disease program. The company presented an update on the Phase I/II clinical trials of AMT-130, also known as ifezuntirgene inilparvovec, and issued a press release summarizing the findings. The filing incorporates the presentation and press release as exhibits.
The update focuses on the high-dose cohort of the ongoing Phase I/II study. The data cut-off for the 36-month analysis was June 30, 2025, while the 48-month analysis was conducted as of June 30, 2026. The company compared the AMT-130 high-dose arm to a propensity score-matched external control derived from the updated Enroll-HD natural history data set.
According to the presentation, the 36-month analysis showed statistically significant slowing of disease progression. The company reported a 60% slowing of disease progression based on the Total Functional Capacity (TFC) primary endpoint (p=0.033) and a 75% slowing based on the composite Unified Huntington’s Disease Rating Scale (cUHDRS) primary endpoint (p=0.003).
The 48-month analysis, which included 12 patients with 48 months of follow-up, showed continued evidence of meaningful slowing. The company reported a 61% slowing of disease progression based on TFC (p=0.008) and a 44% slowing based on cUHDRS (p=0.144). The company also reported favorable trends in secondary endpoints, including a 113% slowing based on the Stroop Word Reading Test (SWRT) (p=0.006).
The company noted that the updated external control likely understates disease progression and the treatment effect at 48 months due to survivor bias and missingness in the data. The company stated that AMT-130 continues to be generally well-tolerated. The filing includes forward-looking statements regarding the timing of regulatory interactions, the potential for accelerated regulatory pathways, and the utility of neurofilament light (NfL) as a biomarker.
