Business
uniQure Announces First Quarter 2021 Financial Results and Highlights Recent Company Progress
~ 52-week follow-up data from HOPE-B pivotal study expected to be presented later this quarter ~ ~ Announced closing of the global commercialization and
About this update from Uniqure N.v.
[{"type":"text","content":"~ 52-week follow-up data from HOPE-B pivotal study expected to be presented later this quarter ~ ~ Announced closing of the global commercialization and license agreement with CSL Behring for hemophilia B gene therapy ~ ~ Completed enrollment of first dose cohort of U.S. Phase I/II clinical trial of AMT-130 for Huntington’s disease, with initiation of second dose cohort and European Phase Ib/II expected in second half of 2021 ~ ~ Pierre Caloz to be appointed Chief Operating Officer ~ ~ Research & Development Day to be held Tuesday, June 22, 2021 ~ LEXINGTON, Mass. and AMSTERDAM, the Netherlands, May 10, 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 the first quarter of 2021 and highlighted recent progress across its business. “We have made great strides with our clinical trial execution across both our hemophilia and Huntington’s disease programs, with all patients in the HOPE-B pivotal trial completing their 52-week follow-up visits and all ten patients in the first dose cohort of our U.S. clinical trial in Huntington’s disease completing their procedures,” stated Matt Kapusta, chief executive officer at uniQure. “Looking at important near-terms catalysts, we look forward to announcing top-line, 52-week follow-up data from the HOPE-B pivotal trial, initiating our collaboration with CSL Behring and holding an R&D Day to provide updates on the research pipeline, including new product candidates,” he added. “In the second half of the year, we expect to begin patient enrollment in the second cohort of AMT-130 and to initiate our open-label European study of AMT-130. Later this year, we expect to submit the BLA for etranacogene dezaparvovec and announce early data on the first four patients in the Phase I/II study of AMT-130.” Recent Company Progress Advancing late-stage development of etranacogene dezaparvovec (AMT-061) for the treatment of hemophilia B The Company expects to announce top-line data during the second quarter of 2021 related to additional clinical trial endpoints in the HOPE-B pivotal trial of etranacogene dezaparvovec in severe to moderately severe hemophilia B patients, including 52-week annualized bleeding rates and FIX activity.The Company announced the closing...