uniQure follow-up AMT-130 Phase I/II data show 80% slowing at 36 months in high-dose patients
uniQure said follow-up Phase I/II results for AMT-130 in Huntington’s extended to 48 months, with high-dose patients showing 80% slowing at 36 months versus an external natural-history comparator; functional benefit was maintained over four years.
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11:45:28 AM UTC
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* US-listed shares of gene therapy developer uniQure fall 62% to $14.83 premarket * Co says its experimental gene therapy for Huntington's disease, an inherited brain disorder, continued to slow disease progression in an early- to mid-stage study * Updated 36-month data showed 80% slowing of disease progression on cUHDRS and 67% on Total Functional Capacity, the main measure in its confirmatory study * At 48 months, cUHDRS, the primary endpoint, showed 44% slowing but missed statistical significance; Total Functional Capacity showed 61% slowing * FDA previously accepted 36-month data as the basis for the company's accelerated approval application * Stock up 63% YTD, as of last close (Reporting by Sahil Pandey in Bengaluru) ((mailto:Sahil.Pandey@thomsonreuters.com)