uniQure N.V. announced updated interim data including up to 30 months of follow-up from 39 patients enrolled in the ongoing U.S. and European Phase I/II clinical trials of AMT-130 for the treatment of Huntington?s disease. In the multi-center, Phase I/II clinical trial of AMT-130 being conducted in the U.S., a total of 26 patients with early-manifest Huntington?s disease have been enrolled, including an initial 10-patient low-dose cohort (6 treated, 4 control) with up to 30 months of follow-up and a subsequent 16-patient high-dose cohort (10 treated, 6 control) with up to 18 months of follow-up. Patients were randomized to treatment with AMT-130 or an imitation (sham) surgery.

The U.S. study consists of a blinded 12-month core study period followed by unblinded long-term follow-up of five years for treated patients. Four of the six control patients in the high-dose cohort were crossed over to treatment and the remaining two patients failed to meet the study?s inclusion criteria. The multi-center, open-label, Phase I/II clinical trial of AMT-130 being conducted in Europe and the UK enrolled a total of 13 patients with the same early manifest criteria for Huntington?s disease as the U.S. study.

Six patients were treated with AMT-130 in the initial low-dose cohort and seven patients were treated in the subsequent high-dose cohort. The combined U.S. and European data presented in this release are subject to a September 30, 2023 cut-off date and do not include efficacy and biomarker data from the control patients who crossed over to treatment.