uniQure's Investigational Gene Therapy Shows Promising Efficacy With Manageable Safety Risks For Fabry Disease

uniQure N.V. (NASDAQ:QURE) on Friday released initial safety and exploratory efficacy data from the first cohort of its Phase 1/2a trial of AMT-191, an investigational gene therapy for Fabry disease.

Fabry disease is a genetic lysosomal storage disorder characterized by a deficiency of the enzyme that breaks down fats.

Fabry disease is 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.

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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 (6×1013 genome copies/kilogram (gc/kg)), showed substantial increases in α-Gal A activity, ranging from 27- to 208-fold above the mean normal level.

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All four patients were withdrawn from enzyme replacement therapy (ERT) and maintained stable plasma lyso-Gb3 levels through the cutoff date.

Based on data observed to date, AMT-191 showed a manageable safety profile.

At the 6×1013 gc/kg dose, two Serious Adverse Events (SAEs) unrelated to AMT-191 (stroke, diplopia), two related SAEs (chest pain, increased troponin), and one possibly related SAE (leptomeningeal enhancement) were observed in two patients.

Additionally, one patient experienced an asymptomatic Grade 3 liver enzyme elevation that resolved with corticosteroid therapy. This event, classified as a dose-limiting toxicity per protocol, was not considered serious and did not require hospitalization. No loss of α-Gal A expression was observed in this patient.

uniQure expects to present updated results from the Phase 1/2a trial in the first half of 2026.

Price Action: QURE stock is up 0.79% at $17.87 at the last check on Friday.

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