uniQure reports 4-year data for Huntington’s disease gene therapy AMT-130

uniQure has released updated Phase I/II clinical data for AMT-130, its investigational gene therapy for Huntington’s disease. The company reported that the therapy continues to show a slowing of disease progression through the 4-year mark.

At the 36-month milestone, high-dose patients showed an 80% slowing of decline on the Composite Unified Huntington’s Disease Rating Scale (cUHDRS) and a 67% slowing on Total Functional Capacity (TFC) compared to an external control group. However, 48-month data for 12 high-dose patients showed a 44% slowing in cUHDRS decline, which did not reach statistical significance. Despite this, the TFC metric showed a 61% slowing at 48 months.

uniQure has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) and expects an acceptance decision in the fourth quarter. Additionally, its marketing authorization application has been validated by the U.K. Medicines and Healthcare products Regulatory Agency. The company plans to begin screening patients for a confirmatory study by the end of the year.