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UniQure Says Additional AMT-130 Data Show Weaker Results at 4 Years; Shares Tumble

UniQure Says Additional AMT-130 Data Show Weaker Results at 4 Years; Shares Tumble

MT newswireMT newswire2026/09/29 12:26
08:26 AM EDT, 09/29/2026 (MT Newswires) -- UniQure (QURE) said Tuesday additional data from its ongoing phase 1/2 studies of AMT-130 in Huntington's disease showed less pronounced benefits at 48 months. The company said the primary endpoint of composite Unified Huntington's Disease Rating Scale showed 44% slowing of disease progression in 12 high-dose patients at 48 months, lower than the updated 36-month data of 80% slowing of disease progression in 15 high-dose patients. Total functional capacity delivered a 61% slowing of disease progression in 12 high-dose patients at 48 months, compared with the updated 36-month data of 67%, the company said. The company's shares fell 52.5% in premarket activity Tuesday.
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