uniQure Rises on Positive Fabry Gene Therapy Trial Data

Key Trial Results Boost uniQure Stock

uniQure (NASDAQ:QURE) stock increased 3.1% in premarket trading after the company announced positive preliminary data from its Phase I/IIa trial of AMT-191. This investigational gene therapy targets Fabry disease, a rare genetic disorder. The results indicate significant therapeutic potential, with all 11 treated patients showing elevated enzyme activity.

Overview of Clinical Findings

The trial demonstrated dose-dependent increases in α-galactosidase A (α-Gal A) activity across all patient cohorts. In the highest dose group, enzyme activity reached levels ranging from 27.7 to 223.7 times above normal. A significant milestone was achieved as six of the eleven patients successfully discontinued their standard enzyme replacement therapy (ERT) after meeting specific criteria.

Market Reaction and Safety Profile

The promising efficacy data fueled investor confidence, driving the stock's premarket gains. However, the company also reported that two patients in the mid-dose cohort experienced dose-limiting toxicities related to elevated liver enzymes. While manageable, this has led to a temporary pause in dosing for the mid- and high-dose groups pending further evaluation by the safety monitoring board.

Summary and Future Outlook

The preliminary success of AMT-191 reinforces its potential as a one-time treatment for Fabry disease. Investors will now focus on the resolution of the safety concerns and future data readouts, which are critical for the therapy's continued development and eventual path to market approval.

FAQ

Q: What is AMT-191?
A: AMT-191 is an investigational gene therapy developed by uniQure for the treatment of Fabry disease, a rare genetic disorder caused by a deficiency of the α-Gal A enzyme.

Q: Why did uniQure's stock price increase?
A: The stock price rose following the release of promising preliminary clinical trial data that showed AMT-191 significantly increased enzyme activity in all patients, with several able to stop their existing therapy.

Source: Investing.com