• Source: mezha.net

Therapy slows disease progression by 75% in mid-stage study. Company shares surged more than 250% after the announcement.

UniQure’s experimental gene therapy significantly slowed the progression of Huntington’s disease in a mid-stage clinical trial, the biotechnology company said Wednesday. The announcement sent shares of the company soaring, more than tripling in value during midday trading.

Trial results

The therapy, called AMT-130, reduced disease progression by 75% after 36 months in patients who received a high dose, UniQure reported. The results were measured using a widely accepted clinical scale for Huntington’s disease.

In addition to the primary outcome, the therapy slowed the decline of patients’ functional abilities by 60%, achieving a key secondary goal of the study.

“These groundbreaking 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,” said Sarah Tabrizi, director of the University College London Huntington’s Disease Center.

The company said AMT-130 was generally well tolerated. No new serious side effects have been reported since late 2022.

Next steps of the process

UniQure plans to submit a marketing application to the U.S. Food and Drug Administration (FDA) in early 2026. If approved, the company aims to launch the therapy later that year.

Huntington’s disease currently has no FDA-approved treatments. The condition is a rare inherited brain disorder that steadily worsens over time and typically leads to death 10 to 30 years after symptoms appear.

About 41,000 people in the United States are living with the disease, while more than 200,000 are at risk of inheriting it, according to the Huntington’s Disease Society of America.

Market impact

Following the trial results, UniQure’s stock jumped by about 250% in midday trading. The surge reflects strong investor confidence in the therapy’s commercial potential, given the lack of treatment options and the urgent demand within the Huntington’s disease community.

The company’s progress places it among a small group of biotechnology firms pursuing gene therapy solutions for neurological disorders. If successful, AMT-130 would represent a breakthrough in a field where decades of research have yielded few effective treatments.

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