QURE Stock Jumped A Whopping 161% Pre-Market – Here’s What Happened

uniQure N.V. (QURE) on Wednesday said that a high dose of its experimental gene therapy AMT-130 demonstrated a statistically significant slowing of disease progression in Huntington’s disease in an early-to-mid stage trial.

Shares of the company rose a whopping 161% in the pre-market session at the time of writing.

Huntington’s disease is a rare, inherited neurodegenerative disorder that leads to motor symptoms including chorea, behavioral abnormalities, and cognitive decline. The company stated that high-dose AMT-130 demonstrated 75% disease slowing at 36 months, as measured by the composite Unified Huntington's Disease Rating Scale (cUHDRS), a comprehensive assessment tool used in clinical trials for Huntington's disease. The company also identified a statistically significant 60% slowing of disease progression as measured by Total Functional Capacity (TFC) at 36 months.

On Stocktwits, retail sentiment around QURE stock jumped from ‘neutral’ to ‘extremely bullish’ territory over the past 24 hours, while message volume rose from ‘normal’ to ‘extremely high’ levels.

uniQure on Wednesday said that the therapy was generally well-tolerated in the trial with a manageable safety profile. No new drug-related serious adverse events have been observed from December 2022 to the end of June 2025, it added.

Chief Medical Officer Walid Abi-Saab said that the company is looking forward to discussing the data with the U.S. Food and Drug Administration at a meeting later this year. The company plans to submit an application for approval of AMT-130 to the FDA in the first quarter of 2026 and anticipates launching it in the U.S. later in the same year.

According to the company, there are currently no approved therapies to slow the progression of Huntington’s disease. The disease affects approximately 75,000 people in the U.S., EU, and the U.K., it added.

QURE stock is down by 23% this year but has risen approximately 166% over the past 12 months. 

Read also: Acadia Pharmaceuticals’ Investigational Drug For Rare Genetic Disorder Fails In Late-Stage Study

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