Positive interim data shown in phase 1/2 trials of Huntington’s disease therapy
Click Here to Manage Email Alerts
A gene therapy company has announced updated interim data from ongoing United States and European phase 1/2 clinical trials of AMT-130 to treat Huntington’s disease.
According to a press release from uniQure, the combined data have a Sept. 30 cutoff date and do not include efficacy and biomarker data from controls who crossed over to treatment.
The U.S. cohort includes 26 patients with early manifest HD. An initial 10-patient low-dose cohort (six treated, four control) has up to 30 months of follow-up, and a subsequent 16-patient high-dose cohort (10 treated, six control) has up to 18 months of follow-up. Participants were randomized to treatment with AMT-130 or sham surgery.
The U.S. study consists of a blinded 12-month core study period followed by unblinded long-term follow-up of 5 years for treated patients, while the multicenter, open label study in the U.K. and Europe enrolled 13 patients with the same early manifest criteria for HD as the U.S. study. Six patients were treated with AMT-130 in the initial low-dose cohort, and seven others were treated in the subsequent high-dose cohort.
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, according to the release.
Treatment with AMT-130 showed a difference in Composite Unified Huntington’s Disease Rating Scale of 0.39 points at 30 months and 1.24 points at 18 months for the low- and high-dose cohorts, respectively. For Total Functional Capacity, AMT-130 demonstrated a favorable difference of 0.95 points at 30 months in the low-dose and 0.49 points at 18 months in the high-dose cohort while also recording a favorable difference in Total Motor Score of 2.8 points at 30 months in the low-dose and 1.7 points in the high-dose at 18 months, per the release.
“The clinical assessment trends in the ongoing studies of AMT-130 look very promising and continue to show disease stability in Huntington’s disease patients treated with this one-time administered gene therapy, several of whom have now been followed more than 2 years,” Walid Abi-Saab, MD, uniQure chief medical officer, said in the release. “We are observing favorable trends in evaluation of motor skills, functional independence and composite rating scores as compared to a non-concurrent criteria-matched natural history cohort.”
UniQure expects to present another clinical update from the ongoing clinical trials of AMT-130, including additional follow-up data from the treated patients in the U.S. and European cohorts, in mid-2024.