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uniQure Announces Latest Positive Recommendation from Data Safety Monitoring Board in Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease
~ No Significant Safety Concerns Observed in First Four Patients Enrolled in Higher-dose Cohort ~ ~ Enrollment Expected to be Completed by Mid-2022 ~
About this update from Uniqure N.v.
[{"type":"text","content":"~ No Significant Safety Concerns Observed in First Four Patients Enrolled in Higher-dose Cohort ~ ~ Enrollment Expected to be Completed by Mid-2022 ~ LEXINGTON, Mass. and AMSTERDAM, Nov. 02, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the positive recommendation by the independent Data Safety Monitoring Board (DSMB) following a review of safety data from the four patient procedures in the higher-dose cohort of the Phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease. With the positive recommendation, the final 12 patients in this second cohort are now cleared for enrollment. A total of 14 blinded administration procedures have been completed as of August 2021. In the study to date, eight patients have been treated with AMT-130, and six patients have received imitation surgery. Full enrollment of the second, higher-dose cohort is expected to be completed in mid-2022. “The AMT-130 program continues to advance at a steady pace, and with this positive recommendation from the DSMB, we are eager to enroll the final 12 patients in the higher-dose cohort,” said David Cooper, M.D., vice president of clinical development at uniQure. “We also look forward to expanding our efforts with the initiation of a separate open-label study of AMT-130 in Europe and to sharing preliminary imaging and biomarker data from the initial four patients in the U.S. clinical trial before the end of the year.” About the Phase I/II Clinical Program of AMT-130 The U.S. Phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease is exploring the safety, tolerability, and efficacy signals in a planned 26 total patients with early manifest Huntington’s disease split into a 10 patient, low-dose cohort followed by a 16 patient, higher-dose cohort; patients will be randomized to treatment with AMT-130 or an imitation (sham) surgery. The multi-center trial consists of a blinded 12-month core study period followed by unblinded long-term follow-up for five years. A total of 16 patients in the clinical trial will receive a single administration of AMT-130 through MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum (caudate and putamen). Additional details a...