uniQure Shares Up After an Update on Low-Dose Cohort in Phase I/II Clinical Trial of Gene Therapy for the Treatment of Huntington’s Disease

Jun 23, 2022 By MarketDepth

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uniQure N.V. (NASDAQ: QURE) share up more than 15% after the Company announced on Thursday new 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. 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.”

Ricardo Dolmetsch, Ph.D., president of research and development at uniQure

All 26 patients have been enrolled in the U.S. clinical trial, including the 10 patients in the low-dose cohort and 16 patients in the high-dose cohort. In the open-label European trial, all 6 patients in the low-dose cohort and 4 of 9 patients in the high-dose cohort have received AMT-130.

“We have made excellent progress in our clinical investigation of AMT-130 and now have a total of 36 patients enrolled across our two Phase I/II clinical studies in the U.S. and Europe. Enrollment in the high-dose cohort of our open-label European study is well underway and is expected to be completed by the end of this year. We look forward to continued collaboration with the Huntington’s disease community to advance AMT-130 as a potential treatment for those living with Huntington’s disease.”

Matt Kapusta, chief executive officer of uniQure