uniQure’s Gene Therapy AMT-130 Shows Strong Efficacy in Huntington’s Disease – Positive Phase I/II Results
uniQure N.V. (NASDAQ: QURE) has announced positive topline results from its pivotal Phase I/II study of AMT-130, an investigational one-time gene therapy for Huntington’s disease (HD).
The high-dose AMT-130 arm met its primary endpoint, showing a statistically significant 75% slowing of disease progression at 36 months, as measured by the composite Unified Huntington’s Disease Rating Scale (cUHDRS), compared to a propensity score-matched external control group.
Key Highlights from the Study
- Primary endpoint met: 75% slowing of disease progression on cUHDRS (p=0.003).
- Secondary endpoint achieved: 60% slowing on Total Functional Capacity (TFC) (p=0.033).
- Supportive measures: Favorable trends in motor and cognitive function tests (SDMT, SWRT, TMS).
- Biomarker data: Mean cerebrospinal fluid neurofilament light (NfL) levels were below baseline at 36 months, suggesting reduced neurodegeneration.
- Safety: AMT-130 was generally well-tolerated with a manageable safety profile.
Dr. Sarah Tabrizi of University College London described the results as “the most convincing in the field to date,” underscoring AMT-130’s potential as the first disease-modifying therapy for HD.
What’s Next?
- Regulatory path: uniQure plans to submit a Biologics License Application (BLA) in Q1 2026.
- Potential U.S. launch: Later in 2026, pending FDA approval.
- Designations: AMT-130 has already received Breakthrough Therapy and RMAT status from the FDA.
Why It Matters
Huntington’s disease is a rare, inherited neurodegenerative condition affecting ~75,000 people across the U.S., EU, and UK, with no approved therapies that slow disease progression. If approved, AMT-130 could represent the first true disease-modifying treatment — offering hope to patients and families impacted by this devastating disorder.
🔗 Full details: uniQure Press Release
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