uniQure’s Gene Therapy AMT-130 Significantly Slows Huntington’s Progression in Pivotal Trial

uniQure's gene therapy AMT-130, delivered via AAV5 viral vector brain surgery, was shown in a Phase I/II trial to significantly slow the progression of Huntington's disease symptoms123.

The therapy met its primary endpoint, marking the first time any drug has altered the course of Huntington's disease in a human clinical trial123.

High-dose participants experienced a 75% slower progression on the composite HD scale (cUHDRS) and approximately 60% slowing of decline in daily functional capacity compared to controls13.

Biomarker analysis also revealed favorable changes, including reductions in neurofilament light, which indicates less neuronal damage1.

These results were observed in a small sample size (fewer than 30 participants), and comparisons were made to external controls, so further confirmation is needed in larger trials1.

uniQure plans to meet with the FDA in late 2025 and file for approval in early 2026; the therapy has been granted special regulatory designations that could expedite review13.

The therapy targets both expanded and unexpanded forms of the huntingtin protein, aiming for a potentially one-time treatment effect14.

This development provides substantial hope to the Huntington's disease community and supports huntingtin-lowering as a viable therapeutic strategy13.

Sources:

1. https://en.hdbuzz.net/the-first-domino-falls-amt-130-gene-therapy-slows-huntingtons-in-landmark-trial/

2. https://firstwordpharma.com/story/6095418

3. https://www.biopharmadive.com/news/uniqure-huntington-gene-therapy-study-results-fda/760987/

4. https://www.uniqure.com/programs-pipeline/huntingtons-disease