uniQure Shares Plunge 64% After Huntington's Trial Data Miss
uniQure shares fell 64% after 48-month data for its Huntington's disease treatment failed to reach statistical significance on a primary composite scale.
uniQure N.V. saw its shares drop 64% on Tuesday following the release of 48-month clinical data for its experimental Huntington’s disease treatment, ifezuntirgene inilparvovec (AMT-130). The sell-off occurred after the treatment failed to reach statistical significance on the composite Unified Huntington’s Disease Rating Scale (cUHDRS), showing a 44% slowing of disease progression with a p-value of 0.144.
The company attributed the result to survivor bias, stating that 53% of the external control group dropped out by the 48th month, which left a healthier control population. However, the 48-month data did show a statistically significant 61% slowing of functional decline on the Total Functional Capacity (TFC) metric.
Despite the recent miss, uniQure maintains that its regulatory strategy remains intact. The U.S. Food and Drug Administration previously agreed that 36-month data, which demonstrated an 80% slowing of disease progression (p=0.005), would serve as the primary basis for the Biologics License Application already submitted under the accelerated approval pathway.
