uniQure Announces Completion of Enrollment in the First Cohort and Favorable Recommendation from the Independent Data Monitoring Committee for its Phase I/IIa Clinical Trial of AMT-191 for the Treatment of Fabry Disease

Core Insights - The Independent Data Monitoring Committee (IDMC) has recommended proceeding with the enrollment of the second cohort in the Phase I/IIa trial of AMT-191 after reviewing safety data from the first cohort [1][2] - The company expects to initiate enrollment of the second dose cohort in the first quarter of 2025 [1] Company Overview - uniQure N.V. is a leading gene therapy company focused on developing transformative therapies for patients with severe medical needs [1][6] - The company has a pipeline of proprietary gene therapies targeting various severe diseases, including Huntington's disease, refractory temporal lobe epilepsy, ALS, and Fabry disease [6] Clinical Trial Details - The Phase I/IIa clinical trial of AMT-191 is a multi-center, open-label study in the U.S. involving two dosing cohorts of up to six adult male patients each [3] - Patients will receive intravenous infusions of AMT-191 while continuing their regular enzyme replacement therapy for a period of 24 months [3] - The trial aims to explore safety, tolerability, and early signs of efficacy by measuring the expression of the lysosomal enzyme α-galactosidase A (aGLA-A) [3] Disease Context - Fabry disease is an X-linked genetic disorder caused by a deficiency of the GLA enzyme, leading to lipid accumulation in various cell types, including kidney and heart cells [2][5] - The prevalence of Fabry disease is estimated to be between one in 40,000 and one in 117,000 individuals [5] - Current standard care involves bi-weekly infusions of enzyme replacement therapy, which has limited effectiveness due to poor cross-correction [5] Regulatory Status - AMT-191 has received Orphan Drug status and Fast Track designation from the U.S. Food and Drug Administration [4]