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uniQure Announces Positive Recommendation to Advance Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease
Independent Data Safety Monitoring Board Recommends Proceeding with Second, Higher-Dose Cohort LEXINGTON, Mass. and AMSTERDAM, The Netherlands, May 27, 2021
About this update from Uniqure N.v.
[{"type":"text","content":"Independent Data Safety Monitoring Board Recommends Proceeding with Second, Higher-Dose Cohort\nLEXINGTON, Mass. and AMSTERDAM, The Netherlands, May 27, 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 the independent Data Safety Monitoring Board (DSMB) overseeing the Phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease has met and reviewed safety data for the fully enrolled first cohort of ten patients. This data set included nine-month safety data from the first two enrolled patients, six-month safety data from the next two enrolled patients, and 30-day safety data on remaining six patients in the trial. The DSMB recommended continued dosing in the study, and uniQure will now begin to enroll patients in the higher-dose cohort of the trial. The Phase I/II study is a double-blind, randomized clinical trial being conducted in the United States. To date, six patients have been treated with AMT-130, and four patients received the imitation surgery. “We are very pleased with the progress of the study and with the recommendations of the DSMB. We look forward to proceeding with dose-escalation in the second cohort of patients,” said Ricardo Dolmetsch, Ph.D., president of research and development at uniQure. “We remain highly focused on progressing this therapy given the significant unmet need in the Huntington’s disease community and are committed to initiating clinical development of AMT-130 in Europe in the second half of this year and to sharing initial imaging and biomarker data from 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 will explore the safety, tolerability, and efficacy signals in 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 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. Patients will receive a single administration of AMT-130 through MRI-guided, convection-enhanced stereotacti...