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ClearPoint Neuro, Inc. Congratulates uniQure on Completion of Enrollment in First Cohort of Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease
IRVINE, Calif., April 06, 2021 (GLOBE NEWSWIRE) -- ClearPoint Neuro, Inc. (Nasdaq: CLPT) (the “Company”), a global therapy-enabling platform company providing
About this update from Clearpoint Neuro Inc.
[{"type":"text","content":"IRVINE, Calif., April 06, 2021 (GLOBE NEWSWIRE) -- ClearPoint Neuro, Inc. (Nasdaq: CLPT) (the “Company”), a global therapy-enabling platform company providing navigation and delivery to the brain, today congratulates uniQure N.V. on the early completion of patient enrollment in the first dose cohort of a randomized, double-blinded, Phase I/II clinical trial of AMT-130 being conducted in the United States for the treatment of early-stage Huntington’s disease. Six patients out of ten who were enrolled in the first cohort were randomized to receive AMT-130 gene therapy and underwent administration procedures using the ClearPoint® Neuro Navigation System and SmartFlow® MRI-safe neuro ventricular cannulae under MRI guidance. The Company’s clinical specialist team provided support during the administration and imitation surgery (sham) procedures to assist the study team in executing the clinical trial protocol. A second, higher-dose cohort in this study is expected to begin enrolling 16 patients in the second half of 2021. A separate, open-label Phase Ib/II clinical trial of AMT-130 for 15 patients at European trial sites is expected to be performed using the ClearPoint Neuro Navigation System and SmartFlow cannulae, with patient enrollment planned to begin later this year. Huntington’s disease is a rare, inherited neurodegenerative disorder impacting approximately 70,000 people in the U.S. and Europe.1 It affects motor function and leads to behavioral symptoms and cognitive decline in young adults, resulting in total physical and mental deterioration. It is caused by the expansion of the CAG trinucleotide in exon 1 of a multifunctional gene coding for a protein called huntingtin. Despite the clear etiology of Huntington’s disease, there are no currently approved therapies to delay the onset or to slow disease progression. uniQure’s gene therapy product candidate AMT-130 consists of an AAV5 vector carrying a gene that expresses a micro-RNA specifically tailored to block the production of huntingtin protein, including the toxic mutant variant. Using AAV vectors to deliver this treatment directly to affected areas in the brain under live MRI-guidance for non-selective knockdown of the huntingtin mRNA represents a highly innovative and promising approach to treating Huntington’s disease. “ClearPoint is proud to help advance potential tre...