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UniQure Soars on New Huntington's Disease Study Data

uniQure N.V. (NASDAQ: QURE) surged 76.5% on Jul 9 after it announced updated interim data, including up to 24 months of follow-up data, from 29 treated patients enrolled in the ongoing early to mid-stage studies of AMT-130 for Huntington's disease in the United States and EU. Results demonstrated a potential long-term, durable clinical benefit and reduction of a key marker of neurodegeneration upon treatment with the candidate. AMT-130, a one-time administered investigational gene therapy, is being developed to treat Huntington's disease. Huntington's disease is a genetic disorder that causes the progressive breakdown of nerve cells in the brain, which leads to a decline in cognitive and physical abilities, often resulting in movement, thinking and psychiatric problems. uniQure is simultaneously conducting two phase I/II studies of AMT-130, with 26 participants in the United States and 13 in the EU/UK to treat Huntington's disease. Among these, a total of 29 patients received either a low dose (12 patients) or a high dose (17 patients) of AMT-130, while 10 control patients underwent imitation surgery. As of Mar 31, 2024, follow-up data at 24 months were available for 21 patients, including 12 from the low-dose group and nine from the high-dose group. Please note that uniQure, for the first time, performed a statistical analysis of clinical outcomes at 24 months for the 21 treated patients. This analysis, compared with an expanded, propensity-weighted external control group of 154 patients, developed in collaboration with the Cure Huntington's Disease Initiative. The control data was derived from the TRACK-HD, TRACK-ON and PREDICT-HD natural history studies. Year to date, shares of QURE have lost 1.5% compared with the industry's 6.3% decline. Image Source: Zacks Investment Research Per the data readout from the phase I/II Huntington's disease studies, patients treated with the high ...