Positive Top-Line Data for AMT-130 in Huntington's Disease
uniQure announced positive top-line data from their pivotal Phase 1/2 study of AMT-130, showing statistically significant 75% slowing of disease progression at three years compared to a control group. A 60% slowing was observed in a key secondary endpoint.
Financial Growth
uniQure's revenue for Q3 2025 was $3.7 million, up from $2.3 million in Q3 2024, marking an increase of $1.4 million. The increase was due to $1.5 million in license revenues.
Strong Cash Position
uniQure reported cash, cash equivalents, and investment securities totaling $649.2 million as of September 30, 2025, up from $376.5 million at the end of 2024, attributed to net proceeds from public offerings.
Encouraging Initial Data for AMT-260 in Epilepsy
Initial data from AMT-260 for mesial temporal lobe epilepsy showed a promising reduction in seizure frequency with no serious adverse events. Recruitment has expanded following a favorable review.
Fabry Disease Therapy Progress
Phase 1/2a trial of AMT-191 showed encouraging results, with one patient successfully withdrawing from enzyme replacement therapy while maintaining stable levels, indicating potential as a one-time gene therapy.