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uniQure uniQure Announces Second Quarter 2021 Financial Results and Highlights Recent Company Progress July 26 , 2021 Expanded gene therapy pipeline with four new research programs and announced acquisition of Corlieve Therapeutics ~ - Presented positive 52 - week data from HOPE - B pivotal study demonstrating sustained increases in Factor IX ( FIX ) levels with mean FIX activity of 41.5 percent of normal and significant reductions in bleeding events ~ Completed its global commercialization and license transaction with CSL Behring for hemophilia B gene therapy program ~ ~ Initiated patient enrollment in second dose cohort of Phase I / II clinical trial of AMT - 130 for Huntington's disease , with initiation of a European Phase Ib / Il clinical trial expected in second half of 2021 ~ LEXINGTON , Mass . and AMSTERDAM , The Netherlands , July 26 , 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 second quarter of 2021 and highlighted recent progress across its business . " We had a highly productive second quarter of this year marked by several notable achievements , including the closing on our global commercial licensing agreement with CSL Behring in hemophilia B and the recent presentation of 52 - week data on all patients in our HOPE - B pivotal study showing durable and sustained increases in Factor IX levels within the non - hemophiliac range 1 , as well as significant reductions in both bleeding events and usage of replacement therapy after a one - time administration of etranacogene dezaparvovec , " stated Matt Kapusta , chief executive officer at uniQure . " Additionally , we continued to make strong progress in our U.S. Phase I / II study of AMT - 130 in Huntington's disease , including the completion of patient enrollment in the first dose cohort and the initiation of dosing in the second cohort . " " We also recently hosted a Research and Development Day where we disclosed four new promising research programs that complemented our expanding focus in neurological disease and larger patient populations , including the acquisition of Corlieve Therapeutics and its gene therapy program targeting temporal lobe epilepsy that builds on our expanding focus in serious neurological diseases , " he added . " These investments deliver on our commitment to leverage our strong cash position to invest in new research programs and enabling technologies with the potential to drive significant value . In the second half of 2021 , we expect to continue our progress across all programs , including availability of 78 - week data from the HOPE - B study , initiating patient enrollment for our open - label European study of AMT - 130 in Huntington's disease , and announcing initial data on the first four randomized Huntington's disease patients in the ongoing U.S. Phase I / II study . " 1 Srivastava et al , Haemophilia . 2020 ; 26 ( Suppl 6 ) : 1-158 . Recent Company Progress • Advancing late - stage development of etranacogene dezaparvovec ( AMT - 061 ) for the treatment of hemophilia B • In June 2021 , the Company announced initial 52 - week post - administration Factor IX data and annualized bleeding rates ( ABR ) in the HOPE - B pivotal trial of etranacogene dezaparvovec in severe to moderately severe hemophilia B patients . Sustained increase in FIX levels was observed with a mean FIX activity of 41.5 % of normal compared to 39.0 % at 26 - weeks post administration . During the 52 - week period after dosing , patients reported an 80 % reduction in total bleeding events requiring treatment , an 85 % reduction in spontaneous bleeding events requiring treatment , and a 96 % reduction in annualized usage of FIX replacement therapy . • The Company expects all patients in the HOPE - B clinical trial to complete their 78 - week follow - up visits by the end of the third quarter of 2021 , and expects the BLA will be submitted in the first quarter of 2022 . • 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 . • Advancing the clinical development of AMT - 130 for the treatment of Huntington's disease • In May 2021 , the Company announced the completion of patient enrollment of the first dose cohort of ten patients in its ongoing Phase I / II study of AMT - 130 in Huntington's disease . The U.S. study is a multi - center , double - blinded , randomized and controlled trial to assess the safety and efficacy of AMT - 130 . The Data Safety Monitoring Board