UniQure Shares Fall After Clinical Update From Huntington's Disease Gene Therapy Trial

UniQure NV QURE announced initial observations on the first four patients enrolled in the lower-dose cohort of Phase 1/2 trial of AMT-130 for Huntington's disease. 

• AMT-130 was generally well tolerated at the lower dose of 6x1012 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. 
• Price Action: QURE shares are down 29.30% at $20.34 during the market session on the last check Thursday.