AMT-130 was generally well tolerated at the lower dose of 6×1012 vector genomes (vg).
There have been no serious adverse events (SAEs) related to AMT-130.
Related Link:DSMB Recommends UniQure To Continue Huntington’s Gene Therapy Trial Unchanged.
NfL increased as expected following the AMT-130 surgical procedure and returned to baseline. NfL remained relatively constant in the two untreated control patients.
Structural magnetic resonance imaging did not reveal any clinically meaningful safety findings at one year of follow-up.
Measurements of total and mutant HTT protein in the cerebral spinal fluid of the four patients were highly variable and inconclusive.
A clinical update on the low-dose cohort of ten patients, primarily focused on safety, is expected in Q2 of 2022.
Full safety and efficacy data from the first two cohorts are expected in 1H of 2023 after all patients in the higher-dose cohort have achieved one year of follow-up.
The U.S. Phase 1/2 trial of AMT-130 in a planned 26 patients with early manifest Huntington’s disease, split into a ten patient, low-dose cohort followed by a 16 patient, higher-dose cohort.
The multi-center trial consists of a blinded 12-month core study period followed by unblinded long-term follow-up for five years.
UniQure also plans to initiate a third cohort in 2H of 2022 to explore alternative stereotactic navigation systems to simplify the placement of catheters for infusions of AMT-130.
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