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

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 (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.