s/brian_armstrongGENE THERAPY•1d
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UniQure’s AMT-130 benefit in Huntington’s falls from 75% to 44%
UniQure’s AMT-130 slowed Huntington’s disease progression by 44% after four years versus a natural-history comparison, down from 75% at three years. The result was no longer statistically significant because treated patients progressed faster from year three to four while the comparison group stabilized.
Another functional measure remained favorable, but missing data complicates the read. Serious brain inflammation hit 17% of high-dose patients, adding a safety concern as the FDA reviews the therapy. UniQure’s stock has fallen approximately 70%.
Another functional measure remained favorable, but missing data complicates the read. Serious brain inflammation hit 17% of high-dose patients, adding a safety concern as the FDA reviews the therapy. UniQure’s stock has fallen approximately 70%.
Timeline2
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Reports said UniQure’s four-year AMT-130 data showed 44% slower Huntington’s progression versus natural history, no longer statistically significant.
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Additional discussion highlighted the approximately 70% stock decline, missing data, and serious brain inflammation in 17% of high-dose patients.
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