Business
uniQure Announces Second Quarter 2023 Financial Results and Highlights Recent Company Progress
~ Announced encouraging interim analysis data from ongoing Phase I/II clinical trial of AMT-130 in Huntington’s disease showing preservation of function
About this update from Uniqure N.v.
[{"type":"text","content":"~ Announced encouraging interim analysis data from ongoing Phase I/II clinical trial of AMT-130 in Huntington’s disease showing preservation of function compared to baseline and clinical benefits relative to natural history; both dose cohorts of AMT-130 continue to be generally well-tolerated ~ ~ Preparations underway to initiate two Phase I/II clinical trials in refractory temporal lobe epilepsy and SOD1-ALS later this year; IND for Fabry disease expected to be submitted in the second half of 2023 ~ ~ Strong cash position of $628.6 million as of June 30, 2023, which excludes a $100 million milestone related to first commercial sale of HEMGENIX® in the U.S. received in July 2023 ~ ~ Announced sale of royalty interest in HEMGENIX® for up to $400 million ~ ~ Appointed Walid Abi-Saab, M.D., as Chief Medical Officer; Jeannette Potts as Chief Legal Officer ~ LEXINGTON, Mass. and AMSTERDAM, Aug. 01, 2023 (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 2023 and highlighted recent progress across its business. “In the second quarter of the year, we continued to make strong progress advancing our programs, including announcing encouraging data from the interim analysis of our Phase I/II study of AMT-130 in Huntington’s disease,” stated Matt Kapusta, chief executive officer of uniQure. “These data show that patients treated with AMT-130 appear to have generally preserved clinical and motor function and neurofilament light chain (NfL) levels suggesting a stable to improving neurodegenerative profile. We believe that, collectively, the data suggest early and promising evidence of clinical benefit in patients treated with AMT-130 compared to a patient-matched natural history data set. We look forward to providing another update in the fourth quarter of 2023 that will include data from our European study, as well as preparing for regulatory interactions in the first quarter of 2024 to discuss the data and potential late-stage development of AMT-130.” “We are also working towards the initiation of clinical trials for our gene therapy candidates in refractory temporal lobe epilepsy and SOD1-ALS later this year, as well as the submission of an IND for our Fabry disease gene ther...