uniQure shares fall 48% after Huntington's therapy misses trial threshold

uniQure's shares fell 48% to $20.50 in premarket trading Tuesday after new 48-month results for its experimental Huntington's disease gene therapy missed a statistical significance threshold. Among 12 high-dose patients, AMT-130 showed a 44% reduction in disease progression against an external control group, but the result did not meet that threshold. The company said attrition left the control group weighted toward patients whose disease progressed more slowly. Its application to the FDA for accelerated approval, filed earlier this month, relies instead on 36-month results from all 15 high-dose recipients, which the company says were statistically significant. The later analysis weakens a key comparison for the therapy while the application awaits a decision.