uniQure announces initial AMT-191 Phase I/IIa data

uniQure (QURE) announced initial safety and exploratory efficacy data from the first cohort of its Phase I/IIa trial of AMT-191. The preliminary data were presented at the International Congress of Inborn Errors of Metabolism, ICIEM, in Kyoto, Japan. As of the July 24, 2025 study cutoff date, all four patients in the first cohort, Cohort A, showed substantial increases in alpha-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 All four patients were withdrawn from enzyme replacement therapy and maintained stable plasma lyso-Gb3 levels through the cutoff date.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 unrelated to AMT-191, two related SAEs, and one possibly related SAE 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. To date, no SAEs were reported in the second cohort. uniQure expects to present updated results from the Phase I/IIa clinical trial in the first half of 2026.