Business
uniQure Announces First Quarter 2022 Financial Results and Highlights Recent Company Progress
~ Partner CSL Behring submitted Marketing Authorization Application (MAA) in Europe foretranacogene dezaparvovec in hemophilia B; MAA validated by the
About this update from Uniqure N.v.
[{"type":"text","content":"~ Partner CSL Behring submitted Marketing Authorization Application (MAA) in Europe foretranacogene dezaparvovec in hemophilia B; MAA validated by the European Medicines Agency (EMA)and granted accelerated assessment ~ ~ Received $55 million in milestone payments associated with global submissions of marketing and license applications ~ ~ Completed patient enrollment of higher dose cohort of U.S. Phase I/II study of AMT-130 and lower dose cohort of European Phase Ib/II study; total of 32 patient procedures completed to date ~ ~ One year safety and biomarker data from all 10 patients in lower dose cohort of U.S. Phase I/II study of AMT-130 in Huntington’s disease, including mHTT and NfL biomarkers, expected in second quarter 2022 ~ ~ Multiple preclinical data presentations to be featured at American Society of Gene and Cell Therapy (ASGCT) Hybrid Congress ~ ~ Appointed Erin Boyer as Chief People and Culture Officer and Richard Porter, previously founder and CEO of Corlieve as Chief Business Officer ~ LEXINGTON, Mass. and AMSTERDAM, May 02, 2022 (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 first quarter of 2022 and highlighted recent progress across its business. “uniQure is off to a strong start in 2022, with important advancements across our key programs and earlier-stage research activities, as well as the strengthening of our executive leadership team,” stated Matt Kapusta, chief executive officer at uniQure. “In the first quarter of the year, our partner CSL Behring made important progress with global regulatory submissions for etranacogene dezaparvovec, including the granting of accelerated assessment by the EMA in Europe.” “We also made significant advancements enrolling patients in our Phase I/II studies for AMT-130 in Huntington’s disease,” he added. “To date, we have completed 32 patient procedures across both studies and look forward to presenting in the second quarter of 2022 one-year safety and biomarker data from the 10-patient lower dose cohort of the U.S. randomized, controlled Phase I/II trial. We continue to advance our pipeline of earlier-stage gene therapy candidates and are pleased to have recently initiated a dose-ranging IND-enabling study for AMT-260 in r...