- UniQure N.V. (NASDAQ:QURE) announced safety and biomarker data from 10 patients enrolled in the low-dose cohort of the ongoing Phase 1/2 trial of AMT-130 gene therapy for Huntington's disease.
- Six of the ten enrolled patients received AMT-130, and four received an imitation surgical procedure in this randomized, blinded clinical trial in the U.S.
- AMT-130 was generally well-tolerated in treated patients at the lower dose of 6x1012 vector genomes.
- No serious adverse events related to AMT-130 were reported in these patients.
- Related: Mizuho Comments On UniQure's Upcoming Low-Dose Huntington's Gene Therapy Trial Data.
- Measurements of CSF NfL, a key biomarker of neuronal damage, increased as expected following the AMT-130 surgical procedure and approached baseline at 12 months.
- Measurements of mHTT protein in the CSF of evaluable treated patients showed decreases compared to baseline through 12 months.
- Biomarker and clinical data, including 24-month follow-up in the low-dose U.S. cohort and 12-month follow-up in the high-dose U.S. cohort expected in 1H of 2023.
- Price Action: QURE shares are 24.40% at $19.15 during the market session on the last check Thursday.
