uniQure Announces Positive Interim Data Update Demonstrating Slowing of Disease Progression in Phase I/II Trials of AMT-130 for Huntington’s Disease

1. Positive Interim Data Update: uniQure announced positive interim data from ongoing Phase I/II trials of AMT-130 for Huntington's disease, demonstrating a statistically significant slowing of disease progression and lowering of neurofilament light protein (NfL) levels in cerebrospinal fluid (CSF) over 24 months.
2. Dose-Dependent Slowing of Disease Progression: Patients receiving the high dose of AMT-130 showed an 80% slowing of disease progression in the composite Unified Huntington's Disease Rating Scale (cUHDRS) at 24 months compared to a propensity score-weighted external control.
3. Lowering of NfL Levels: Mean CSF NfL levels for both doses were below baseline at 24 months, indicating a potential therapeutic benefit.
4. RMAT Designation: uniQure was granted the first-ever Regenerative Medicine Advanced Therapy (RMAT) designation in Huntington's disease, which may lead to expedited clinical development discussions with the FDA.
5. Investor Conference Call and Webcast: uniQure management hosted an investor conference call and webcast to discuss the updated results.
6. Huntington's Disease Background: Huntington's disease is a rare, inherited neurodegenerative disorder affecting approximately 70,000 people in the U.S. and Europe, with no available therapies to treat, delay, or slow the progression of the disease.