Business
uniQure Announces Second Quarter 2022 Financial Results and Highlights Recent Company Progress
~ Announced 12-month data on the lower-dose cohort of AMT-130 in Huntington’s disease showed the investigative gene therapy was generally well tolerated at
About this update from Uniqure N.v.
[{"type":"text","content":"~ Announced 12-month data on the lower-dose cohort of AMT-130 in Huntington’s disease showed the investigative gene therapy was generally well tolerated at this dose with a mean reduction of 53.8% of mutant Huntingtin protein (mHTT) observed in cerebral spinal fluid (CSF) ~ ~ Announced postponement of AMT-130 higher-dose procedures due to recent suspected unexpected severe adverse reactions at this dose; Lower-dose procedures are not affected and no impact is expected on anticipated data readouts in 2023 ~ ~ Advancing regulatory reviews of the U.S. and European marketing applications for etranacogene dezaparvovec in hemophilia B ~ ~ Investor conference call and webcast today at 8:30 a.m. ET ~ LEXINGTON, Mass. and AMSTERDAM, Aug. 08, 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 second quarter of 2022 and highlighted recent progress across its business. “We continued to make strong progress across all of our programs highlighted by encouraging data from the lower-dose cohort in our Phase I/II study of AMT-130 and the advancement of the U.S. and European regulatory reviews for the marketing applications for etranacogene dezaparvovec in hemophilia B,” stated Matt Kapusta, chief executive officer at uniQure. “Our manufacturing, CMC and Quality teams are working tirelessly to produce commercial launch supplies and also to ensure successful pre-approval site inspections, which are expected to be completed in the third quarter of this year. We also are very pleased to have recently received notification that GMP certification will be provided to our Lexington manufacturing facility by the European authorities.” “In July, we reported to the health authorities suspected unexpected severe adverse reactions in three of the 14 patients treated with the higher dose of AMT-130,” he continued. “While these patients have fully or substantially recovered, and no clear root cause has yet been identified, we believe it’s prudent to temporarily delay additional higher-dose procedures until we put additional monitoring and treatment plans in place and complete our safety review early in the fourth quarter of 2022. The delay is not expected to impact any future lower-dose procedures, as no seri...