A leading gene therapy company, uniQure, that advances transformative therapies for patients with severe medical needs, announced positive topline data from the pivotal Phase I/II study of AMT-130 for the treatment of Huntington’s disease.
The uniQure study met its prespecified primary endpoint, with high-dose AMT130 demonstrating a statistically significant slowing of disease progression as measured by the composite Uni-fied Huntington’s Disease Rating Scale at 36 months compared to a propensity score-matched external control.
The study also met a key secondary endpoint by achieving statistically significant slowing of disease progression as measured by Total Functional Capacity (TFC) at 36 months compared to a propensity score-matched external control.
In accordance with the prospectively defined statistical analysis plan aligned with, and submitted to, the U.S. Food and Drug Administration (FDA) for the pivotal Phase I/II study, uniQure analyzed clinical outcomes for 29 patients treated with AMT-130 of which 12 patients per dose group had attained 36 months of follow up and were evaluated.
Topline 36-month efficacy results for patients receiving high-dose AMT-130 had a statistically significant slowing of disease progression and affected motor and cognitive function.
The company believes that the consistently favorable results in functional, motor and cognitive endpoints at 36 months observed in the study group show hope for treating Huntington’s Disease.
AMT-130 was generally well-tolerated, with a manageable safety profile. As of June 30, 2025, no new drug-related serious adverse events have been observed since December 2022. The most common adverse events in the treatment groups were related to the administration procedure, which all resolved.
AMT-130 has been granted Breakthrough Therapy designation and Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. Huntington’s disease is a rare, inherited neurodegenerative disorder that leads to motor symptoms including chorea, behavioral abnormalities and cognitive decline resulting in progressive physical and mental deterioration. Approximately 75,000 people have Huntington’s disease in the U.S. and Europe, with hundreds of thousands of others at risk of inheriting the disease. Up to now, there have been no approved therapies to delay the onset or to slow the disease’s progression.