UniQure stock catapulted more than 200% Wednesday after the company said its experimental gene therapy successfully treated Huntington's disease.
Huntington's disease is a neurological condition that slowly destroys brain cells, robbing patients of the ability to move and reason. But a high dose of UniQure's AMT-130 slowed disease progression by 75% after three years.
Dr. Sarah Tabrizi, director of the University College London Huntington's Disease Center, called the results "groundbreaking."
"These data are the most convincing in the field to date and underscore potential disease-modifying effects in Huntington's disease, where an urgent need persists," she said in a statement. "These data indicate that AMT-130 has the potential to meaningfully slow disease progression — offering long-awaited hope to individuals and families impacted by this devastating disease."
UniQure stock launched 247.7% to 47.50. Shares touched a nearly five-year high.
'Game-Changing' Results
UniQure said patients who received the gene therapy showed a statistically significant improvement on the composite Unified Huntington's Disease Rating Scale, or cUHDRS. The scale was created to assess disease progression in early-to-moderate Huntington's disease.
Further, patients showed decreased levels of neurofilament light chain in their cerebrospinal fluid after 36 months. Neurofilament light chain, or NfL, is a biomarker that indicates damage to the central nervous system. The gene therapy also continues to be well tolerated, UniQure said.
The company is now planning to file an application for Food and Drug Administration approval in the first quarter of 2026.
Leerink Partners analyst Joseph Schwartz raised his price target on UniQure stock to 68 from 48 after the company held a call with Huntington's disease experts. He sees the data as "definitive" and the gene therapy as "clearly making a difference on progression."
"We think we will rapidly see excitement grow as one of the key leaders in HD described AMT-130 data on the call as 'game-changing,' offering a 'beacon of hope' for patients and their families, and representing a 'significant step toward delivering a licensed disease-modifying therapy for HD,' " he said.
Schwartz now sees an 85% chance AMT-130 wins FDA approval, and a 50% chance European regulators sign off on it.
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