uniQure reports longer Huntington’s follow-up, with mixed statistical results

uniQure ($QURE) released additional Phase I/II results for AMT-130 in Huntington’s disease. Among 15 high-dose patients followed for 36 months, the company reported 80% slower decline on the composite disease-rating scale against an external natural-history control. At 48 months, 12 high-dose patients showed 44% slowing on that scale, but the comparison was not statistically significant, with p=0.144. Functional-capacity results remained nominally significant.

The longer follow-up informs the durability question for the gene therapy, but small patient numbers, missing control data and reliance on external comparisons limit interpretation. Previously disclosed treatment-related serious inflammatory events in five high-dose participants had resolved. The new findings were not part of the already submitted biologics application. They neither establish regulatory approval nor remove the need for confirmatory evidence.

Sources:

• uniQure GlobeNewswire release, reproduced by StockTitan