UniQure (UNQ) Reports AMT-191 Maintains Enzyme Activity in Fabry Trial; Shares Rise

UniQure (UNQ) reported in a Phase I/IIa trial that AMT-191, an enzyme replacement therapy for Fabry disease, maintained key enzyme activity at week 24 in treated patients, supporting continued disease control. The trial enrolled 12 patients with disease progression and showed sustained activity in primary endpoints. Data from the trial, published in the New England Journal of Medicine, indicate that AMT-191 dosing at 0.2 mg/kg every two weeks was well-tolerated and led to reductions in disease activity biomarkers. The positive results support a potential regulatory filing pathway and a potential approval timeline earlier than originally expected.