uniQure’s shares rise on positive data for Huntington’s gene therapy

UniQure's stock price has surged over 100% following positive interim results from Phase I/II trials of its gene therapy for Huntington’s disease. In the high-dose cohort of AMT-130, disease progression, measured by the Unified Huntington’s Disease Rating Scale (cUHDRS), slowed by 80% compared to controls. The lower dose group saw a 30% slowdown.

Neurofilament light protein (NfL), a neurodegeneration biomarker, decreased by 11% across all treated patients, boosting investor confidence. UniQure's shares rose from $3.78 pre-announcement to $8.71 on July 12.

UniQure plans to meet with the FDA in late 2024 to discuss advancing AMT-130, administered via specialized brain surgery into the brain’s ventricles to lower huntingtin, a key Huntington’s biomarker.

Despite setbacks in 2022, including a dosing pause due to severe side effects, trials resumed with safety measures. AMT-130's RMAT designation in June 2023 underscores its potential as the first gene therapy for Huntington’s disease, a condition lacking approved disease-modifying treatments.