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uniQure Announces Update on Low-Dose Cohort in Phase I/II Clinical Trial of AMT-130 Gene Therapy for the Treatment of Huntington’s Disease
~ Treatment generally well-tolerated with no significant safety issues related to AMT-130 in treated patients through one year of follow-up ~ ~ A mean
About this update from Uniqure N.v.
[{"type":"text","content":"~ Treatment generally well-tolerated with no significant safety issues related to AMT-130 in treated patients through one year of follow-up ~ ~ A mean reduction of 53.8% of mutant HTT (mHTT) observed in cerebral spinal fluid (CSF) at 12 months in evaluable patients treated with AMT-130 ~ ~ Neurofilament Light Chain (NfL) in CSF near baseline at 12 months in patients treated with AMT-130 ~ ~ Biomarker and clinical data including 24-month follow-up in the low-dose U.S. cohort and 12-month follow up in the high-dose U.S. cohort expected in first half of 2023 ~ ~ Investor conference call and webcast today at 8:30 a.m. ET ~ LEXINGTON, Mass. and AMSTERDAM, June 23, 2022 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced safety and biomarker data from 10 patients enrolled in the low-dose cohort of the ongoing Phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease. Six of the 10 enrolled patients received AMT-130, and four patients received an imitation surgical procedure in this randomized, blinded clinical trial being conducted in the United States. “We are encouraged by this 12-month update on the patients enrolled in the low-dose cohort,” stated Ricardo Dolmetsch, Ph.D., president of research and development at uniQure. “Thus far in the clinical trial, AMT-130 has been well-tolerated, with no serious adverse events related to the gene therapy and NfL levels approaching baseline. We are also pleased to observe trends suggesting target engagement that are supported by the lowering of mHTT protein in evaluable patients receiving AMT-130. We look forward to presenting additional clinical data, including functional outcomes, on all patients from this important study next year.” One-Year Observations on Low-Dose Cohort of AMT-130 The 10 patients enrolled in the low-dose cohort were all clinically diagnosed with early-stage Huntington’s disease with CAG repeats between 40-44, baseline Total Functional Capacity scores of 10-13, and Total Motor Scores of 7-23. Four patients were male and six were female with ages ranging from 34-58 years old. Demographics of the six treated patients were generally consistent with the four control patients. Ten patients were evaluable for NfL measurements. Four treate...