~ No Significant Safety Concerns Observed Across a Total of 14 Completed Procedures ~
~ Full Study Enrollment Expected to be Completed by Mid-2022 ~
LEXINGTON, Mass. and AMSTERDAM, The Netherlands, Aug. 30, 2021 (GLOBE NEWSWIRE) — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that two additional patient procedures have been completed in the second, higher-dose cohort in the Phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease. These latest procedures follow a positive recommendation by the independent Data Safety Monitoring Board (DSMB) after reviewing available follow-up data from all participants in the trial. A total of 14 blinded administration procedures have now been completed, including four patients in the second dose cohort. In the study to date, eight patients have been treated with AMT-130, and six patients received imitation surgery.
In the fourth quarter of 2021, the Company expects the DSMB to review the 1-month safety data from the two most recently enrolled patients in the higher dose cohort, as well as the 3-month data from the first two patients. Upon receiving a positive recommendation from the DSMB, the Company will commence enrollment of the final 12 patients in the second dose cohort.
“We continue to be very pleased with the enrollment of this important study and look forward to having the trial fully enrolled by the middle of next year,” said David Cooper, M.D., vice president of clinical development at uniQure. “We also look forward to sharing preliminary imaging and biomarker data from initial patients in the U.S. clinical trial before the end of the year and initiating a separate open-label study of AMT-130 in Europe later this year.”