Business
uniQure Announces Second Quarter 2025 Financial Results and Highlights of Recent Company Progress
~ Achieved alignment with the FDA on the AMT-130 statistical analysis plan and CMC requirements to support a planned BLA submission in the first quarter of
About this update from Uniqure N.v.
[{"type":"text","content":"~ Achieved alignment with the FDA on the AMT-130 statistical analysis plan and CMC requirements to support a planned BLA submission in the first quarter of 2026; ~ Company expects to present AMT-130 topline three-year data in September 2025 ~ ~ Presented case study from first participant treated with AMT-260 for refractory mesial temporal lobe epilepsy showing 92% seizure reduction with no serious adverse events through first five months of follow up ~ ~ Appointment of Kylie O’Keefe as Chief Customer and Strategy Officer ~ ~ Cash, cash equivalents and current investment securities of approximately $377.0 million as of June 30, 2025 are expected to fund operations into second half of 2027 ~ ~ uniQure to host earnings call at 8:30 a.m. ET ~ LEXINGTON, Mass. and AMSTERDAM, July 29, 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 second quarter of 2025 and highlighted recent progress across its business. “We delivered tremendous progress across our business in the first half of 2025, setting the stage for a transformative period ahead for uniQure,” said Matt Kapusta, chief executive officer of uniQure. “With alignment from the FDA on a biological license application (BLA) pathway and pivotal topline data expected in September, AMT-130 is well-positioned to potentially become the first disease-modifying therapy for people living with Huntington’s disease.” “At the same time, we’re encouraged by early clinical signals from the first patient treated with AMT-260 in refractory mesial temporal lobe epilepsy and the continued advancement of our Fabry and ALS programs,” continued Mr. Kapusta. “Backed by a strong balance sheet and an exceptional team, we believe that we are well prepared to execute on the opportunities ahead, including the planned commercialization of AMT-130, and delivering lasting value to patients and shareholders.” Recent Company Developments and Updates Alignment with FDA continues to support Accelerated Approval pathway for AMT-130 In June 2025, the Company announced its alignment with the FDA on the statistical analysis plan (SAP) and Chemistry, Manufacturing and Controls (CMC) requirements to support a planned Biologics License Application (BLA) submission for A...