uniQure Posts $622.5M Cash, FDA Requires Prospective AMT-130 Study
uniQure reported $622.5 million cash reserves at December 31, 2025, projected to fund operations into H2 2029, following Q4 and full-year 2025 results. The FDA concluded that AMT-130 Phase I/II external-control data are insufficient for approval, recommending a prospective randomized study; uniQure plans a Type B meeting in Q2 2026.
1. Financial Position
As of December 31, 2025, uniQure held approximately $622.5 million in cash, cash equivalents and investment securities, providing funding for operations into the second half of 2029.
2. AMT-130 36-Month Efficacy Data
High-dose AMT-130 showed a 75% slowing of disease progression on cUHDRS (p=0.003), a 60% slowing on Total Functional Capacity (p=0.033), and an 8.2% mean reduction in cerebrospinal fluid NfL at 36 months, with a manageable safety profile and no new serious adverse events.
3. FDA Feedback on AMT-130
The FDA concluded that AMT-130 Phase I/II studies compared to an external control are insufficient for approval and strongly recommended a prospective, randomized, double-blind, sham surgery-controlled trial; uniQure plans a follow-up Type B meeting in Q2 2026 to refine the Phase III design.
4. Pipeline Progress
uniQure completed enrollment of the first six-patient cohort in the Phase I/IIa AMT-260 study for mesial temporal lobe epilepsy, with updated data expected in the first half of 2026, while AMT-191 in Fabry disease achieved durable, dose-dependent α-Gal A activity increases up to 312.52-fold across three dose levels.