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uniQure uniQure Announces First Quarter 2021 Financial Results and Highlights Recent Company Progress May 10 , 2021 ~ 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 lb / ll 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 of the transaction with CSL Behring for its hemophilia B gene therapy following the expiration of the waiting period under the Hart - Scott - Rodino Antitrust Improvements Act of 1976 on May 5 , 2021. In accordance with the agreement , the upfront payment of $ 450 million was paid to uniQure on May 7 , 2021 . • In April 2021 , the FDA removed the clinical hold on the Company's hemophilia B gene therapy program regarding a diagnosis of hepatocellular carcinoma ( HCC ) , a form of liver cancer , in one patient in the HOPE - B trial . This followed a review of data from a comprehensive investigation that found it was highly unlikely etranacogene dezaparvovec was the cause of the HCC in this patient . • Advancing the clinical development of AMT - 130 for the treatment of Huntington's disease • The ten - patient , first dose cohort of the Phase I / II clinical trial of AMT - 130 was fully enrolled in the second quarter of 2021. The second , higher - dose cohort is expected to begin enrollment in the third quarter of 2021 after a review by the independent Data Safety Monitoring Board ( DSMB ) . Early imaging and biomarker data from the first four enrolled patients in this first - in - human AAV - gene therapy trial in Huntington's disease are anticipated by year - end 2021 . • In February 2021 , the DSMB recommended proceeding with patient enrollment in the Phase I / II clinical trial of AMT - 130 after finding no significant safety concerns based on its review of the six - month data from the first two enrolled patients and the 90 - day data from the next two enrolled patients . • The Company also announced plans to initiate an open label , Phase Ib / II clinical study of AMT - 130 in Europe with enrollment beginning in the second half of 2021 . • Expanding the Pipeline and Strengthening the Platform