uniQure N.V. announced on September 5, 2025, initial safety and exploratory efficacy data from the first cohort of its Phase I/IIa trial of AMT-191, an investigational gene therapy for Fabry disease. The preliminary data, presented at the International Congress of Inborn Errors of Metabolism, showed promising results for the one-time intravenously administered therapy.
All four patients in the first cohort (Cohort A, 6x10^13 gc/kg) achieved substantial increases in α-Gal A enzyme activity, ranging from 27- to 208-fold above the mean normal level. These elevated levels were sustained for as long as 45 weeks post-treatment for the first patient. Crucially, all four patients were able to discontinue enzyme replacement therapy (ERT) and maintained stable plasma lyso-Gb3 levels.
AMT-191 demonstrated a manageable safety profile at the highest dose. Two related serious adverse events (chest pain, increased troponin) and one possibly related serious adverse event (leptomeningeal enhancement) were observed in two patients. One patient experienced an asymptomatic Grade 3 liver enzyme elevation that resolved with corticosteroid therapy. Enrollment was completed in a second, lower dose cohort (Cohort B, 2x10^13 gc/kg), with no serious adverse events reported to date in that cohort. Updated clinical results are expected in the first half of 2026.
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