uniQure shares slide 41% after AMT-130 Huntington’s data miss significance at 48 months

AI Market Summary
uniQure's AMT-130 48-month update missed statistical significance on the key cUHDRS measure, with the implied slowing dropping from 75% at 36 months to 44%, triggering a sharp selloff and raising execution/regulatory uncertainty around its accelerated approval filing. While management and analysts highlighted comparator-data issues and a steady TFC secondary endpoint, near-term sentiment is pressured by weaker primary-readout optics and higher scrutiny of the evidence base.
Impact level
● Medium
Affected assets
NCSKCRDO2USD/USDT+4.27%
AI Insight · NCSKCRDO2USD/USDTAI Insight
▼ Bearish
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uniQure released 48-month clinical data for its Huntington’s disease gene therapy AMT-130 that did not significantly slow disease progression. The cUHDRS measure showed the slowdown easing to 44% at month 48 from 75% at month 36. The stock fell 41% at the open to $23.15. Analysts and company executives said they remain encouraged by the strength of a secondary endpoint and still see a potential path to approval.