uniQure AMT-130 3-Year Data Show Disease Slowing, 2025 Revenue Falls 41%
uniQure reported 3-year phase 12 data for AMT-130 showing significant slowing of Huntington’s progression and holds $622.5 million in cash and securities. FDA recommended a phase 3 randomized double-blind sham-surgery study, while 2025 revenue fell to $16.1 million from $27.1 million and SG&A expenses rose by $12.8 million.
1. AMT-130 Phase 12 3-Year Data
uniQure’s phase 12 study of AMT-130 released 3-year data from 12 patients showing significant slowing of Huntington’s disease progression, reinforcing the therapy’s potential to modify disease course.
2. Revenue and Expense Trends
Revenue for 2025 declined by 41% to $16.1 million from $27.1 million in 2024, while SG&A expenses increased by $12.8 million, driven by higher development and commercialization costs. The company maintains a cash and securities balance of $622.5 million, providing runway for advancing its pipeline.
3. Regulatory Strategy and Next Steps
FDA recommended a phase 3 randomized double-blind sham-surgery controlled trial for AMT-130, which could extend the approval timeline. uniQure is engaging with international regulators to explore alternative pathways, preparing for a Type B meeting on trial design and leveraging external databases to optimize study parameters.