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uniQure Announces Completion of Patient Enrollment in the First Two Cohorts of its Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease
LEXINGTON, Mass. and AMSTERDAM, March 21, 2022 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative
About this update from Uniqure N.v.
[{"type":"text","content":"LEXINGTON, Mass. and AMSTERDAM, March 21, 2022 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the completion of patient enrollment in the first two cohorts of its randomized, double-blinded, Phase I/II clinical trial of AMT-130 for the treatment of early stage Huntington’s disease. “We are very pleased to have completed the enrollment of the first 26 patients in this ongoing clinical trial,” stated Ricardo Dolmetsch, Ph.D., president of research and development. “It’s a major milestone for the trial and an important achievement for our clinical operations team. Our investigators have done an exceptional job in enrolling this important study. We look forward to providing a clinical update in the second quarter of this year from the 12-month interim analysis of the 10 patients in the first cohort, including safety, mutant HTT protein (mHTT) and neurofilament light chain (NfL) data. We also remain on track with the enrollment of our European open-label clinical trial of AMT-130 and plan to begin a third patient cohort in the U.S. to explore an improved administration procedure.” The ongoing Phase I/II clinical trial of AMT-130 is a randomized, sham controlled, double-blinded study to explore the safety, tolerability, and proof of concept of AMT-130 in patients with early manifest Huntington’s disease. The study includes two dose cohorts of 26 patients randomized to either treatment with AMT-130 or to an imitation surgical procedure. The first dose cohort includes 10 patients, of which six patients received treatment with AMT-130 and four patients received imitation surgery. The second dose cohort includes 16 patients, of which ten patients received treatment with AMT-130 and six patients received imitation surgery. A third cohort exploring the use of an alternative stereotactic navigation system to simplify placement of infusion catheters will include up to 18 additional randomized patients. The Phase I/II clinical trial consists of a blinded 12-month period followed by unblinded long-term follow-up for five years. The treated patients have received a single administration of AMT-130 using MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum (caudate and putamen). The ...