uniQure releases promising data from phase 1/2 trial of Huntington’s disease gene therapy
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Gene therapy company uniQure has announced safety and biomarker data from 10 patients enrolled in the low-dose cohort of the ongoing phase 1/2 clinical trial of AMT-130 for treatment of Huntington’s disease.
According to a company release, six patients enrolled in the study received AMT-130 and four underwent an imitation surgical procedure. At the 12-month follow-up, measurements of cerebrospinal fluid NfL, a key biomarker of neuronal damage, increased as expected following the AMT-130 surgical procedure and approached baseline at 12 months.
In addition, measurements of mutant HTT protein in the CSF of evaluable treated patients showed decreases compared with baseline through 12 months, and AMT-130 was generally well-tolerated in treated patients at the lower dose of 6x1012 vector genomes.
“We are encouraged by this 12-month update on the patients enrolled in the low-dose cohort,” Ricardo Dolmetsch, PhD, president of research and development at uniQure, stated in the release. “Thus far in the clinical trial, AMT-130 has been well-tolerated, with no serious adverse events related to the gene therapy and NfL levels approaching baseline.”
The U.S.-based clinical trial of AMT-130 explored the safety, tolerability and efficacy signals in 26 patients with early manifest Huntington’s disease, of whom 10 were in a low-dose cohort and 16 in a high-dose cohort, the company stated in the release. Patients were randomized to treatment with either AMT-130 or sham surgery and participated in a blinded, 12-month core study period followed by an unblinded long-term follow-up for 5 years.
A total of 16 patients in the clinical trial received a single administration of AMT-130 through MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the dorsal portion of the striatum (caudate and putamen).
In an open-label European trial, six patients in the low-dose cohort and four of nine patients in the high-dose cohort have received AMT-130. Enrollment in the high-dose cohort is underway and expected to be completed by the end of 2022, the release stated.
“We have made excellent progress in our clinical investigation of AMT-130 and now have a total of 36 patients enrolled across our two phase 1/2 clinical studies in the U.S. and Europe,” Matt Kapusta, uniQure CEO, said in the release. “We look forward to continued collaboration with the Huntington’s disease community to advance AMT-130 as a potential treatment for those living with Huntington’s disease.”