Clinical Research
uniQure shares four-year data from ongoing studies of AMT-130
New four-year results from uniQure provide additional insight into AMT-130, an investigational gene therapy being studied as a potential treatment for Huntington disease (HD).
The latest results update on two commonly studied clinical measures in HD:
– Total Functional Capacity (TFC): At 48 months, participants who received the high dose of AMT-130 showed a 61% slower decline compared with an external control group.
– cUHDRS: The analysis showed a 44% slowing of progression at 48 months compared with the external control group. This result did not reach statistical significance.
TFC and cUHDRS measure different aspects of HD progression. TFC focuses on functional abilities and independence, while cUHDRS combines several clinical measures to provide a broader assessment of disease progression.
The 48-month results need to be interpreted with caution. The number of participants in the study remains relatively small, and the analysis relies on comparisons with an external control group. uniQure has also noted that substantial missing data and potential survivor bias in the updated external control group may have affected the results. AMT-130 is still an investigational treatment, and these results do not yet establish whether it will ultimately be approved or how it may be used in clinical practice. The HD treatment landscape continues to evolve, and HSC will continue following AMT-130 and other clinical developments closely. We’ll share updates as more information becomes available.
