uniQure N.V. delivered strong top-line results from its pivotal Phase 1/2 study of AMT-130, marking a significant milestone in Huntington's disease treatment, although FDA feedback introduces uncertainty regarding the expected BLA submission timeline.

• AMT-130 demonstrated a statistically significant 75% slowing of disease progression over three years, meeting pivotal study endpoints.
• The FDA's recent feedback has raised concerns over the adequacy of AMT-130's evidence for a BLA submission, leading to uncertainty regarding the submission timeline.
• In addition to Huntington's, uniQure is advancing AMT-260 for epilepsy, with active recruitment of clinical sites and initial positive data expected next year.
• Results from the Phase 1/2a trial of AMT-191 for Fabry disease show promising enzyme activity, with one patient successfully withdrawing from enzyme replacement therapy.
• uniQure remains committed to engaging with the FDA to expedite the path forward for AMT-130 despite the recent setbacks.