uniQure Announces Update on Phase I/II Clinical Trials of AMT-130 Gene Therapy for the Treatment of Huntington’s Disease - Seite 2

Ongoing Phase I/II Trials of AMT-130 in 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.

Updated Interim Data

Exploratory Efficacy Data

Clinical and functional measurements for treated patients in each dose cohort were compared to baseline measurements, as well as to control patients (up to 12 months) and a non-concurrent criteria-matched natural history cohort. The natural history cohort was developed by uniQure in collaboration with the Cure Huntington’s Disease Initiative (CHDI) using the TRACK-HD natural history study of patients with early Huntington’s disease. The cohort includes 31 patients that met the uniQure clinical trial inclusion criteria of Total Functional Capacity, Diagnostic Classification Level and minimum striatal volumes.

• Updated clinical data through 30 months for the low-dose cohort and 18 months for the high-dose show ongoing evidence of potential dose-dependent clinical benefit relative to the non-concurrent criteria-matched natural history.
  
• For patients receiving the high dose, neurological function as measured by cUHDRS and each of its individual components was preserved or improved at 18 months compared to pre-treatment baseline measurements.