uniQure Announces Initial AMT-191 Phase I/IIa Data Showing Sustained Increases in α-Gal A Enzyme Activity in Patients with Fabry Disease - Seite 2

Based on data observed to date, AMT-191 showed a manageable safety profile.   At the 6x10¹³ gc/kg dose, two Serious Adverse Events (SAEs) unrelated to AMT-191 (stroke, diplopia), two related SAEs (chest pain, increased troponin), and one possibly related SAE (leptomeningeal enhancement) 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. No loss of α-Gal A expression was observed in this patient.

Enrollment was completed in a second, lower dose cohort, Cohort B (2x10¹³ gc/kg), consisting of three patients. As of the study cutoff date, all patients had less than three months of follow-up. 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.

About the Phase I/IIa Clinical Program of AMT-191

The Phase I/IIa clinical trial of AMT-191 is a multi-center, open-label trial being conducted in the United States consisting of three dosing cohorts of three or more adult male patients each receiving an intravenous infusion of AMT-191. Patients were not excluded from the trial based on pre-existing neutralizing anti-bodies to AAV5. Patients continue to receive their regular enzyme replacement therapy until meeting withdrawal criteria and will be followed for a period of 24 months. The trial will explore the safety, tolerability, and early signs of efficacy by measuring the expression of lysosomal enzyme α-Gal A. Additional details are available on www.clinicaltrials.gov (NCT06270316).

AMT-191 has been granted both Orphan Drug and Fast Track designation by the U.S. Food and Drug Administration.

About Fabry Disease

Fabry disease is an X-linked genetic disorder resulting from a deficiency of GLA. Based on a 2020 study published in the Journal of Therapeutics and Clinical Risk Management, the prevalence is estimated to be between one in 40,000 and one in 117,000 individuals. The current standard of care for Fabry disease is bi-weekly infusions of enzyme replacement therapy, a treatment with limited effectiveness in many patients due to poor cross-correction, with inefficient clearance of substrates in the target organs, in particular the kidney and the heart.