UniQure (QURE) Reports Positive Phase I/IIa AMT-191 Data for Fabry Disease, Shows Sustained Enzyme Activity

SelfRugger · 2026-03-23T13:27:02+00:00

UniQure's Phase I/IIa trial of AMT-191 for Fabry disease showed increased enzyme activity in all participants, with some able to stop regular treatments. However, dosing has paused due to safety concerns, including liver enzyme abnormalities.

SelfRugger

2026-03-23 13:27:02

Generación de resúmenes en curso

UniQure (QURE) Reports Positive Phase I/IIa AMT-191 Data for Fabry Disease, Shows Sustained Enzyme Activity

Maham Fatima

Sat, February 14, 2026 at 3:28 PM GMT+9 2 min read

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UniQure (NASDAQ:QURE) is one of the most promising low-cost stocks to buy now. On February 6, UniQure reported positive updated data from its Phase I/IIa trial of AMT-191, which is a gene therapy for Fabry disease. All 11 patients across three different dose levels showed increased enzyme activity, with the longest-treated patient maintaining these high levels for over a year. Notably, the enzyme levels were significantly above normal ranges, showing a clear relationship between the dose received and the amount of enzyme produced.

The treatment allowed 6 out of the 11 patients to stop their regular enzyme replacement therapy/ERT after meeting specific clinical criteria. Even after stopping ERT, patients maintained stable levels of lyso-Gb3, the toxic substance that typically builds up in Fabry disease. This suggests that the one-time gene therapy could potentially replace the need for lifelong, bi-weekly infusions for some patients.

While the therapy shows promise, UniQure has paused new dosing in the mid- and high-dose groups to evaluate some safety findings. Two patients in the middle dose group experienced high liver enzyme levels that were classified as dose-limiting toxicities, though both responded to steroid treatment. Other previously reported serious events in the high-dose group included chest pain and a stroke, though some were determined to be unrelated to the study drug.

UniQure (QURE) Reports Positive Phase I/IIa AMT-191 Data for Fabry Disease, Shows Sustained Enzyme Activity

UniQure (NASDAQ:QURE) develops treatments for patients suffering from rare and other devastating diseases in the US.

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Disclosure: None. This article is originally published at Insider Monkey.

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