uniQure (NASDAQ:QURE) has announced the completion of enrollment for its Phase 1/2a trial of AMT-191 in patients with Fabry disease. The company also reports receiving a favorable recommendation from the Independent Data Monitoring Committee (IDMC) for the trial.
uniQure expects to initiate enrollment of the trial’s second dose cohort in the first quarter of 2025. AMT-191 is an investigational gene therapy designed to treat Fabry disease, an X-linked genetic disorder caused by a mutation in the alpha-galactosidase A enzyme (GLA gene).
Walid Abi-Saab, MD, CMO of uniQure, stated, “Fabry is a debilitating disease that continues to represent a significant unmet medical need. We are encouraged by the initial pharmacodynamics, biomarkers, and safety profile observed to date for AMT-191 as well as the positive outcome of the IDMC review. This strengthens our confidence in the potential of AMT-191 to make a meaningful difference in the lives of patients with Fabry disease. We look forward to advancing to the second cohort in this important clinical program.”
