Business
uniQure Announces Third Quarter 2022 Financial Results and Highlights Recent Company Progress
~ Patient enrollment at the higher dose of AMT-130 to resume in European Phase Ib/II study following Data Safety Monitoring Board recommendation ~~ U.S. and
About this update from Uniqure N.v.
[{"type":"text","content":"~ Patient enrollment at the higher dose of AMT-130 to resume in European Phase Ib/II study following Data Safety Monitoring Board recommendation ~~ U.S. and European pre-approval inspections of Company’s Lexington, MA manufacturing site completed with EMA issuing Good Manufacturing Certificate ~~ Virtual investor event focused on next pipeline program, AMT-260 investigational gene therapy for refractory temporal lobe epilepsy, to be held on Tuesday, November 29, 2022 ~ LEXINGTON, Mass. and AMSTERDAM, Nov. 02, 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 the third quarter of 2022 and highlighted recent progress across its business. “We are pleased that following a comprehensive review of all available safety, biomarker and imaging data, the Data Safety Monitoring Board (DSMB) has recommended that we resume patient enrollment at the higher dose in the ongoing Phase Ib/II study evaluating AMT-130 in Huntington’s disease,” stated Matt Kapusta, chief executive officer of uniQure. “Patient safety will always be our utmost priority, and we are grateful for the close collaboration between our clinical team, study investigators and most importantly, the patients, all of whom were instrumental in completing our comprehensive investigations. We believe that AMT-130 has the potential to provide a positive impact for patients with this devastating disease for which there is no currently approved treatment. We look forward to finishing patient enrollment in the higher-dose cohort of the European study in the first half of 2023 and remain on track to announce data from the U.S. Phase I/II study in the second quarter of 2023.” “In the third quarter, the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) completed site inspections of our Lexington, Massachusetts gene therapy manufacturing facility, and we have since received good manufacturing practice (GMP) certification from the European authorities. This is an important milestone as we seek to establish industry-leading commercial gene therapy manufacturing capabilities. Our partner, CSL Behring, continues to interact with U.S. and European health authorities as they complete their review of the marketing appli...