uniQure says AMT-130 data show 80% slowing on cUHDRS at 36 months in high-dose patients
QURE•uniQure said Phase I/II data showed 80% slowing on cUHDRS at 36 months in high-dose Huntington’s patients, with follow-up results extending through 48 months.
1. Follow-up results
New follow-up data from ongoing Phase I/II studies of AMT-130 showed continued slowing of disease progression in high-dose patients versus an external natural-history comparator, with functional benefit maintained over four years. The results also suggested a dose-dependent effect, with high-dose patients performing better than low-dose patients on overall disease and daily-function measures.
2. Application and safety
The data were not included in uniQure’s already submitted accelerated-approval application, and the company indicated the findings will be presented at a future scientific meeting. Safety remained broadly manageable, with earlier treatment-related central nervous system inflammation events reported as resolved.
