UniQure’s Huntington’s gene therapy AMT-130 fails to slow disease progression significantly
UniQure said its gene therapy AMT-130 missed statistical significance on the main goal of slowing Huntington’s disease progression in a four-year analysis, with 44% slowing at 48 months in 12 high-dose patients versus an external control.
-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.
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