Business
uniQure Announces 2024 Financial Results and Highlights Recent Company Progress
~ Announced alignment with the U.S. Food and Drug Administration (FDA) on key elements of the Accelerated Approval pathway for AMT-130 in Huntington’s
About this update from Uniqure N.v.
[{"type":"text","content":"~ Announced alignment with the U.S. Food and Drug Administration (FDA) on key elements of the Accelerated Approval pathway for AMT-130 in Huntington’s disease; Initiated preparations for a potential Biologics License Application (BLA) submission ~ ~ Completed patient enrollment in the third cohort of the Phase I/II study of AMT-130 ~ ~ Initiated dosing of the Phase I/II study of AMT-260 in mesial temporal lobe epilepsy (mTLE); Implementing protocol changes to expand study inclusion criteria ~ ~ Completed enrollment of the first cohorts in the Phase I/II studies of AMT-191 in Fabry disease and AMT-162 in SOD1-ALS; Received favorable recommendations from the respective Independent Data Monitoring Committees (IDMC) to proceed with dosing the second cohorts ~ ~ Cash and cash equivalents of approximately $367.5 million as of December 31, 2024, combined with $80.7 million in net proceeds from the recently completed financing, are expected to fund operations into the second half of 2027 ~ LEXINGTON, Mass. and AMSTERDAM, Feb. 27, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the fourth quarter and full year of 2024 and highlighted recent progress across its business. “This past year was transformative for uniQure, marked by significant clinical and operational progress,” stated Matt Kapusta, chief executive officer of uniQure. “On the clinical front, we made great strides advancing AMT-130 for Huntington’s disease, including securing alignment with the FDA on key elements of the Accelerated Approval pathway, a major milestone that brings us closer to delivering the first potentially disease-modifying treatment for this devastating condition. With this regulatory clarity, we have initiated BLA-readiness activities and look forward to further engagement with the FDA throughout the first half of 2025. Our RMAT designation has enabled a productive and expedited dialogue with the FDA, which we will continue leveraging to advance AMT-130 through the regulatory process as rapidly as possible. This is welcome news for patients awaiting further development of our groundbreaking therapies.” Mr. Kapusta continued, “Beyond AMT-130, we continue to advance our broader pipeline of investigational ge...