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uniQure Announces Completion of Enrollment in the First Cohort and Favorable Recommendation from the Independent Data Monitoring Committee for its Phase I/IIa Clinical Trial of AMT-191 for the Treatment of Fabry Disease
~ Independent Data Monitoring Committee recommends proceeding with dosing of second cohort after planned safety assessment ~ ~ Company expects to initiate
About this update from Uniqure N.v.
[{"type":"text","content":"~ Independent Data Monitoring Committee recommends proceeding with dosing of second cohort after planned safety assessment ~ ~ Company expects to initiate enrollment of second dose cohort in the first quarter of 2025 ~ LEXINGTON, Mass. and AMSTERDAM, Feb. 03, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the completion of enrollment in the first cohort of the Phase I/IIa trial of AMT-191, an investigational gene therapy for the treatment of Fabry disease. Additionally, the Independent Data Monitoring Committee (IDMC) reviewed safety data from the initial two patients enrolled in the first cohort. The IDMC’s review identified no significant safety concerns and recommended proceeding with enrollment in the second cohort. “Fabry is a debilitating disease that continues to represent a significant unmet medical need,” stated Walid Abi-Saab, M.D., chief medical officer of uniQure. “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.” AMT-191 is an investigational AAV5-based gene therapy that uses a proprietary, highly potent promoter to deliver a galactosidase alpha (GLA) transgene designed to target the liver to produce GLA protein. In patients with Fabry disease, a pathogenic variant in the GLA gene leads to α-galactosidase A (aGAL-A) enzyme deficiency, which in turn results in a progressive accumulation of lipids in multiple cell types, including kidney and heart cells, eventually resulting in a multi-system disorder. AMT-191 may offer a novel one-time intravenously administered approach to treating Fabry disease. 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 two dosing cohorts of up to six adult male patients each receiving an intravenous infusion of AMT-191. Patients will continue to receive their regular enzyme replacement therapy until the cri...