uniQure Announces Initial AMT-191 Phase I/IIa Data Showing Sustained Increases in α-Gal A Enzyme Activity in Patients with Fabry Disease

Core Insights - The article discusses the promising initial results from the Phase I/IIa trial of AMT-191, a gene therapy for Fabry disease, highlighting significant increases in α-Gal A activity among patients [1][4][3] Company Overview - uniQure N.V. is a gene therapy company focused on developing transformative therapies for severe medical conditions, including Fabry disease [1][10] - The company has previously achieved a milestone with its gene therapy for hemophilia B, showcasing its commitment to advancing genomic medicine [10] Clinical Trial Details - The Phase I/IIa trial of AMT-191 includes multiple cohorts, with the first cohort (Cohort A) showing α-Gal A activity increases of 27- to 208-fold above normal levels [3][4] - All patients in the first cohort discontinued enzyme replacement therapy (ERT) and maintained stable plasma lyso-Gb3 levels [3][4] - A second cohort (Cohort B) has been enrolled with a lower dose, and no serious adverse events (SAEs) have been reported to date [5] Safety and Efficacy - Preliminary data indicate that AMT-191 has a manageable safety profile, with some SAEs observed that were unrelated to the treatment [4][5] - The trial aims to explore the safety, tolerability, and early efficacy of AMT-191, with updated results expected in the first half of 2026 [1][6] Disease Context - Fabry disease is a genetic disorder caused by α-Gal A enzyme deficiency, leading to harmful substrate accumulation affecting various organs [2][9] - The current standard treatment involves bi-weekly ERT, which has limitations in effectiveness [9] Regulatory Status - AMT-191 has received Orphan Drug and Fast Track designations from the U.S. FDA, indicating its potential significance in treating Fabry disease [7]