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uniQure Announces Initial AMT-191 Phase I/IIa Data Showing Sustained Increases in α-Gal A Enzyme Activity in Patients with Fabry Disease
~ All patients in the first cohort achieved between 27- to 208-fold increases in α-Gal A activity relative to mean normal level ~ ~ All patients in first
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[{"type":"text","content":"~ All patients in the first cohort achieved between 27- to 208-fold increases in α-Gal A activity relative to mean normal level ~ ~ All patients in first cohort discontinued enzyme replacement therapy ~ ~ Preliminary data show AMT-191 has a manageable safety profile at the highest dose ~ ~ Updated clinical results expected in the first half of 2026 ~ LEXINGTON, Mass. and AMSTERDAM, Sept. 05, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced initial safety and exploratory efficacy data from the first cohort of its Phase I/IIa trial of AMT-191, an investigational gene therapy for the treatment of Fabry disease. The preliminary data were presented at the International Congress of Inborn Errors of Metabolism (ICIEM) in Kyoto, Japan. Fabry disease is a genetic lysosomal storage disorder caused by a deficiency of the α-galactosidase A (α-Gal A) enzyme, leading to toxic accumulation of globotriaosylsphingosine (lyso-Gb3) that can damage the kidneys, heart, nervous system, eyes, gut and skin. AMT-191 is designed as a one-time, intravenously administered, investigational AAV5-based gene therapy that incorporates a proprietary, highly potent promoter designed to achieve supraphysiological α-Gal A expression. As of the July 24, 2025 study cutoff date, all four patients in the first cohort, Cohort A (6x1013 genome copies/kilogram (gc/kg)), showed substantial increases in α-Gal A activity, ranging from 27- to 208-fold above the mean normal level1. Sustained elevated levels were observed in all four patients in the first cohort as of the study cutoff date, which was as long as 45 weeks post-treatment for the first treated patient and 12 weeks post-treatment for the most recently treated patient. All four patients were withdrawn from enzyme replacement therapy (ERT) and maintained stable plasma lyso-Gb3 levels through the cutoff date. “These initial findings from the first cohort are encouraging, with all patients showing robust increases in α-Gal A activity and an ability to withdraw from ERT,” stated Walid Abi-Saab, M.D., chief medical officer of uniQure. “The early data highlight the potential of AMT-191 as a transformative one-time treatment option for people living with Fabry disease. I look forward to sharing addi...