AMT-130 Regulatory Progress
AMT-130 received breakthrough therapy designation from the FDA in April and is on track for a planned BLA submission. The company has had multiple productive interactions with the FDA, focusing on preparing for this submission.
Pipeline Expansion
uniQure expanded its clinical pipeline with the initiation of three additional clinical studies in refractory temporal lobe epilepsy, Fabry disease, and SOD1-ALS.
Strong Financial Position
uniQure has more than $400 million in cash as of March 31, 2025, providing financial flexibility to advance its pipeline, including the planned BLA submission and launch of AMT-130.
Reduced Expenses
Research and development expenses decreased to $36.1 million from $40.7 million year-over-year, and selling, general, and administrative expenses decreased to $10.9 million from $13.9 million in the same period in 2024.