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UniQure Shares Are Up After Low-Dose Cohort Data From Huntington's Gene Therapy Trial

  • UniQure N.V. 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 6×1012 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.

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