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uniQure Announces Clinical Update on First Patients in Phase I/II Clinical Trial of AMT-130 Gene Therapy for the Treatment of Huntington’s Disease
~ Treatment was well tolerated with no significant safety issues related to AMT-130 in first two treated patients through one year of follow-up ~ ~
About this update from Uniqure N.v.
[{"type":"text","content":"~ Treatment was well tolerated with no significant safety issues related to AMT-130 in first two treated patients through one year of follow-up ~ ~ Neurofilament Light Chain (NfL) rose as expected immediately following surgery and returned to baseline in treated patients ~ ~ A total of 19 patient procedures have been performed in the U.S. Phase I/II clinical trial, with higher-dose cohort enrollment expected to be completed by mid-2022 ~ ~ Screening initiated in European open-label Phase I/II study ~ ~ Conference call today at 8:30 a.m. ET ~ LEXINGTON, Mass. and AMSTERDAM, The Netherlands, Dec. 16, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced initial observations on the first four patients enrolled in the lower-dose cohort of its ongoing Phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease. Two of the four enrolled patients received AMT-130, and two patients experienced an imitation (sham) surgery in this randomized, blinded clinical trial being conducted in the United States. “We are pleased with these data that support the tolerability of AMT-130, a potential one-time gene-therapy approach for Huntington’s disease,” stated Ricardo Dolmetsch, Ph.D., president of research and development at uniQure. “Neurofilament light chain, a key biomarker of injury in the brain, trended downward and returned to baseline levels in the treated patients, and structural MRI data are consistent with the safety profile of AMT-130. Based on recommendations of the DSMB and steering committee, we will disclose efficacy data including volumetric MRI changes once all patients in the first two cohorts are unblinded. We look forward to evaluating safety, biomarker and functional data on a larger number of patients and over a longer period during the ongoing study.” “AMT-130 appears to be generally well tolerated both immediately after surgical administration and at one year of follow up in these four patients,” stated Jody Corey-Bloom, M.D., PhD, professor of neurosciences at University of California, San Diego and chairperson of the Data Safety Monitoring Board (DSMB) for AMT-130. “We are encouraged by the early data in two treated patients and will continue to provide comprehensive safety oversight...