uniQure Reports Positive Data for Huntington's Gene Therapy, Stock Surges 80%

Jul 9, 2024, 08:42 PM

uniQure announced positive interim data from its Phase I/II trials of AMT-130, a gene therapy for Huntington's disease. The data demonstrated an 80% slowing of disease progression in patients receiving a high dose of AMT-130. This significant result was measured using the composite Unified Huntington's Disease Rating Scale (cUHDRS). The announcement led to a substantial surge in uniQure's stock, which saw an increase of up to 80%, with pre-market gains of 27.0% and an overall rise of 70% to 76%. The company believes this is the first clinical trial to show potential long-term clinical benefits and a reduction in a key marker of neurodegeneration for Huntington's disease. The promising results have raised hopes for a faster regulatory path with the FDA.