uniQure gene therapy slows disease progression in Huntington’s trial

Amsterdam-based uniQure’s gene therapy candidate, AMT-130, met its primary endpoint as well as a key secondary endpoint in a pivotal phase 1/2 trial for Huntington’s disease.

In the study, uniQure analyzed clinical outcomes for 29 patients treated with AMT-130 (n=17 high dose; n=12 low dose) of which 12 patients per dose group had attained 36 months of follow up.

The study met its prespecified primary endpoint, with high-dose AMT-130 demonstrating a statistically significant 75% slowing of disease progression as measured by the composite Unified Huntington’s Disease Rating Scale (cUHDRS) at 36 months compared to a propensity score-matched external control. The study also met a key secondary endpoint by achieving statistically significant 60% slowing of disease progression as measured by Total Functional Capacity (TFC) at 36 months compared to a propensity score-matched external control.

AMT-130 consists of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene, leveraging the company’s proprietary miQURE silencing technology. The therapeutic goal is to inhibit the production of the mutant protein. AMT-130 has been granted breakthrough therapy designation and regenerative medicine advanced therapy (RMAT) designation from the FDA.

Huntington’s disease, a rare, inherited neurodegenerative disorder for which there are currently no disease-modifying therapies available. The disease is an autosomal dominant condition with a disease-causing CAG repeat expansion in the first exon of the huntingtin gene that leads to the production and aggregation of abnormal protein in the brain. 

uniQure will discuss the new data with the FDA in a pre-BLA meeting expected later this year, with the goal of submitting a BLA in the first quarter of 2026.