Business
uniQure Announces Third Quarter 2021 Financial Results and Highlights Recent Company Progress
~ Completed 78-week follow-up for hemophilia B pivotal study, with top-line data expected by year-end 2021 ~ ~ Enrolled 14 patients to date in Phase 1/2
About this update from Uniqure N.v.
[{"type":"text","content":"~ Completed 78-week follow-up for hemophilia B pivotal study, with top-line data expected by year-end 2021 ~ ~ Enrolled 14 patients to date in Phase 1/2 clinical trial of AMT-130 in Huntington’s Disease, with no significant safety concerns observed ~ ~ Preliminary data on first four patients in ongoing Phase I/II clinical trial of AMT-130 and initiation of European Phase Ib/II clinical trial expected by year-end 2021 ~ ~ Closed acquisition of Corlieve Therapeutics adding preclinical program in temporal lobe epilepsy to proprietary research pipeline ~ LEXINGTON, Mass. and AMSTERDAM, The Netherlands, Oct. 25, 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 third quarter of 2021 and highlighted recent progress across its business. “We continue to make strong progress across our clinical trials and research programs, and CSL Behring is making significant strides towards a BLA submission for etranacogene dezaparvovec in hemophilia B, which is expected in the first half of 2022,” stated Matt Kapusta, chief executive officer at uniQure. “Before the end of this year, we look forward to announcing clinical data from our Huntington’s disease gene therapy program, including preliminary data on the first four patients in our ongoing U.S. Phase I/II study of AMT-130. Additionally, top-line 78-week follow-up data on all HOPE-B pivotal study patients is expected to be announced by CSL Behring in late 4Q2021.” Advancing late-stage development of etranacogene dezaparvovec (AMT-061) for the treatment of hemophilia B – licensed to CSL Behring In September 2021, all patients in the HOPE-B pivotal trial of etranacogene dezaparvovec in severe to moderately severe hemophilia B completed their 78-week follow-up visits. Along with our global partner, CSL Behring, the Company expects to announce top-line data from the HOPE-B trial in the late fourth quarter of 2021.The Company announced initial 52-week follow-up data from the HOPE-B pivotal trial in June 2021. Data showed that after a single dose of etranacogene dezaparvovec, patients continued to demonstrate durable, sustained increases in FIX activity with a mean FIX activity of 41.5% of normal at 52 weeks of follow-up, compared to a mean of 39.0% ...