~ Data from largest gene therapy study in hemophilia B showed sustained therapeutic effect at 18-months and statistical superiority in reducing annualized bleeding rate compared to baseline FIX prophylactic therapy; Submissions of marketing applications on track for the first half of 2022 ~

~ Data from first four patients in ongoing U.S. Phase I/II clinical trial in Huntington’s Disease showed AMT-130 was well tolerated, with no significant safety concerns observed ~

~ Additional clinical data from all patients in lower dose cohort of U.S. Phase I/II study of AMT-130, including mHTT and NfL biomarkers, expected to be presented in second quarter of 2022 ~

~ Initiated enrollment of European Phase Ib/II study of AMT-130 with first two patients dosed ~

~ Refractory temporal lobe epilepsy and Fabry programs advancing into IND-enabling studies in 2022 ~

~ Ended 2021 in strong financial position with $556 million in cash ~

LEXINGTON, Mass. and AMSTERDAM, Feb. 25, 2022 (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 2021 and highlighted recent progress across its business.

“We made tremendous progress in 2021 across all our strategic imperatives, including advancing our clinical-stage programs, expanding our research pipeline, and preparing for the submission of marketing applications planned for etranacogene dezaparvovec during the first half of 2022,” stated Matt Kapusta, chief executive officer at uniQure. “With positive data from the HOPE-B pivotal study we look forward to working closely with our partner, CSL Behring to bring this potentially life-changing gene therapy to patients living with hemophilia B.”

“During 2022, we are also keenly focused on maintaining our strong momentum in enrolling two ongoing Phase I/II studies in Huntington’s disease. Importantly, we look forward to sharing additional safety and biomarker data from all ten patients in the lower dose cohort, including mutant HTT protein (mHTT) and neurofilament light chain (NfL), during the second quarter of 2022,” he added. “We also are poised to advance our gene therapy product candidates for Fabry disease and refractory temporal lobe epilepsy into IND-enabling toxicology studies, and we expect to initiate at least two new gene therapy programs targeting the liver and CNS during the year. As we continue to advance and grow our pipeline, we are also expanding our manufacturing footprint with a second cGMP facility in Amsterdam, which we expect will come online in 2022.”

Recent Key Accomplishments

• Advancing the late-stage development of etranacogene dezaparvovec (AMT-061) for the treatment of hemophilia B
  • In December 2021, the Company and CSL Behring announced the achievement of primary and secondary endpoints from the HOPE-B pivotal trial of etranacogene dezaparvovec in severe and moderately severe hemophilia B patients. The primary endpoint of non-inferiority in annualized bleeding rate (ABR) 18-months following administration compared to baseline Factor IX (FIX) prophylactic therapy was achieved, as was a secondary superiority endpoint on ABR.
  • Data from the HOPE-B trial demonstrated that etranacogene dezaparvovec produced mean FIX activity of 39.0 percent of normal at six months and 36.9 percent of normal at 18 months post infusion. After the six-month lead-in period post-infusion, the adjusted annualized bleeding rate (ABR) (1.51) for all bleeds was reduced by 64 percent (p=0.0002) and all FIX-treated bleeds was reduced by 77 percent (3.65 to 0.83; p