UniQure says AMT-130 slowed Huntington’s disease progression 44% at four years, missing statistical significance
UniQure reported 4-year follow-up data for Huntington's gene therapy AMT-130 showing a 44% slowing in disease progression in the high-dose cohort versus an external control, but the effect was not statistically significant and appears to have waned versus last year's analysis. With the FDA beginning review of the marketing application, durability and evidentiary strength become near-term focal points for biotech risk appetite.
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UniQure reported four-year follow-up data showing its gene therapy AMT-130 reduced Huntington’s disease progression by 44% in a high-dose group, though the result was not statistically significant. The size of the benefit was smaller than in a comparable analysis conducted one year earlier, prompting renewed questions about durability. The update comes as the U.S. Food and Drug Administration begins reviewing UniQure’s marketing application.