Business
uniQure Announces 2020 Financial Results and Highlights Recent Company Progress
~ Six patient procedures now completed in Phase I/II for Huntington’s disease, including two procedures conducted in February 2021, with full enrollment of
About this update from Uniqure N.v.
[{"type":"text","content":"~ Six patient procedures now completed in Phase I/II for Huntington’s disease, including two procedures conducted in February 2021, with full enrollment of first cohort expected mid-2021 ~ ~ Last patient in HOPE-B pivotal study to complete 52-week follow-up visit before end of the first quarter of 2021, with top-line data to be presented in the second quarter of 2021 ~ ~ Analysis of the HOPE-B safety event expected to be completed and submitted to the FDA before end of the first quarter of 2021 ~ ~ Research & Development Day to be held mid-2021 to announce expanded pipeline and platform advancements ~ LEXINGTON, Mass. and AMSTERDAM, March 01, 2021 (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 2020 and highlighted recent progress across its business. “Despite unprecedented challenges in 2020, the uniQure team demonstrated extraordinary resiliency and dedication to the patients we serve, qualities which have defined our company for more than two decades,” stated Matt Kapusta, chief executive officer at uniQure. “I’m very proud of uniQure’s accomplishments, including completing patient dosing in the HOPE-B pivotal trial, presenting positive 26-week data on 54 patients that met the study’s first primary endpoint, and announcing a landmark transaction with CSL Behring for the global commercialization of etranacogene dezaparvovec, which is expected to close in the second quarter of 2021 following the U.S. antitrust review. We also became the first company to initiate the clinical investigation of an AAV gene therapy for Huntington’s disease and have now enrolled six patients in the study.” “Looking at 2021, we are focused on important catalysts as we prepare the filings for regulatory approval of etranacogene dezaparvovec and advance our Phase I/II study of AMT-130 in Huntington’s disease, with early biomarker data on the initial patients expected as early as the end of this year. We are also making investments to expand our pipeline of promising gene therapies and further strengthen our AAV manufacturing platform. We look forward to hosting a Research and Development Day mid-year during which we will unveil new research programs and other technology initiatives.” Recent Company Progress...