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UniQure Says AMT-130 Slows Huntington's Disease Progression in Phase 1/2 Study
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UniQure Says AMT-130 Slows Huntington's Disease Progression in Phase 1/2 Study
Sep 24, 2025 5:30 AM

08:01 AM EDT, 09/24/2025 (MT Newswires) -- UniQure ( QURE ) said Wednesday that its phase 1/2 study in Huntington's disease met its primary endpoint, with high-dose AMT-130 showing statistically significant 75% slowing of disease at 36 months as measured by the composite Unified Huntington's Disease Rating Scale.

The study also met a key secondary endpoint of a statistically significant slowing of disease progression as measured by Total Functional Capacity at 36 months compared to a propensity score-matched external control, the company said.

AMT-130 was generally well-tolerated, with a manageable safety profile and no new drug-related serious adverse events, uniQure said.

UniQure ( QURE ) aims to submit a biologics license application for AMT-130 in Q1 of 2026, Chief Medical Officer Walid Abi-Saab said.

The company's shares were up more than 3% in recent premarket activity Wednesday.

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