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# uniQure Reports Favorable Data in Huntington’s Trial
- URL: https://www.fdaweb.com/uniqure-reports-favorable-data-in-huntingtons-trial/
- Published: 2025-09-24T12:00:00.000Z
- Updated: 2026-09-14T15:22:30.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5159932

uniQure says its investigational gene therapy AMT-130 significantly slowed disease progression in Huntington’s disease patients, which the company calls the most convincing evidence to date of a disease-modifying effect in the fatal neurodegenerative disorder. In a pivotal Phase 1-2 study, patients who received a high dose of AMT-130 experienced a 75% slowing of disease progression over three years, measured by the composite Unified Huntington’s Disease Rating Scale, compared with matched external controls. The therapy also met a key secondary endpoint, showing a 60% slowing of decline in “total functional capacity.”

The study followed 29 patients treated with either high or low doses of AMT-130, with outcomes compared against more than 1,500 patients in the Enroll-HD natural history dataset. At the 36-month mark, those given the high dose not only showed slowing of motor and cognitive decline on multiple measures, but also a mean reduction of cerebrospinal neurofilament light protein, a biomarker of neurodegeneration, the company says.

uniQure plans to meet with FDA later this year ahead of a BLA submission in the first quarter of 2026\. AMT-130 already holds a breakthrough therapy and regenerative medicine designations.