Business
uniQure Announces Third Quarter 2020 Financial Results and Highlights Recent Company Progress
~ Enrolled First Four Patients in Phase I/II Clinical Trial of AMT-130 in Huntington’s Disease ~ ~ Top-Line Data from HOPE-B Pivotal Trial in Hemophilia B
About this update from Uniqure N.v.
[{"type":"text","content":"~ Enrolled First Four Patients in Phase I/II Clinical Trial of AMT-130 in Huntington’s Disease ~\n ~ Top-Line Data from HOPE-B Pivotal Trial in Hemophilia B Expected Before Year End ~ ~ Initiated IND-enabling Studies for AMT-150 in Spinocerebellar Ataxia Type 3 (SCA3) ~ LEXINGTON, Mass. and AMSTERDAM, Oct. 27, 2020 (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 third quarter of 2020 and highlighted recent progress across its business. “We made excellent progress during the third quarter in our Phase I/II gene therapy clinical trial of AMT-130 in Huntington’s disease as the first four patients have now been enrolled in the study, and we look forward to completing enrollment of our first dose cohort by mid-2021,” stated Matt Kapusta, chief executive officer at uniQure. “Regarding our hemophilia B gene therapy program, all 54 patients in the HOPE-B pivotal trial of etranacogene dezaparvovec have now completed their 26-week follow-up visits, and we remain on track to announce top-line data from this registration-enabling study before the end of this year. This is expected to be the first data from a pivotal study of a hemophilia B gene therapy, as well as the first clinical data from a large-scale study evaluating gene therapy in patients irrespective of their preexisting AAV neutralizing antibodies. We also continue to work toward the regulatory approval and closing of our landmark licensing agreement for etranacogene dezaparvovec with CSL Behring.” “I’d like to specially thank all our employees for their tireless focus and dedication during these challenging times,” he added. “We’ve made very strong progress thus far in 2020, and I look forward to ending the year on a strong note.” Recent Company Progress Advancing late-stage development of etranacogene dezaparvovec (AMT-061) for the treatment of hemophilia B Patient dosing was completed in the HOPE-B pivotal trial of etranacogene dezaparvovec in March, 2020, and 26-week follow up on all patients has now been achieved. The Company remains on track to report 26-week Factor IX data for all 54 patients enrolled in the global clinical trial before the end of this year.In June 2020, the Company and CSL Behring entered into a licensing ...