Sept 29 (Reuters) – UniQure said its gene therapy for Huntington’s disease fell short of statistical significance on the main goal of slowing disease progression in a four-year analysis, sending its U.S.-listed shares plummeting 55% in premarket trading on Tuesday.
In the study with 12 high-dose patients, AMT-130 showed 44% slowing of disease progression at 48 months when compared with an external control, but the data was not statistically significant.
Huntington’s disease is a rare inherited brain disorder that causes movement difficulties and triggers behavioural changes and cognitive decline. No approved drugs exist that help slow progression.
(Reporting by Christy Santhosh in Bengaluru; Editing by Sahal Muhammed)
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