- uniQure (NASDAQ:QURE) experienced a significant stock drop following a four-year update on its AMT-130 gene therapy for Huntington’s disease.
- The AMT-130 therapy’s effectiveness in slowing disease progression decreased from 80% at three years to 44% at four years, failing to achieve statistical significance.
- Despite mixed analyst reactions, uniQure plans to pursue regulatory approval with the U.S. Food and Drug Administration (FDA) based on its stronger three-year data.
uniQure (NASDAQ:QURE) is a gene therapy company that develops treatments for patients with severe genetic diseases. A key focus for the biopharmaceutical company is its experimental therapy, AMT-130, which is designed to treat Huntington’s disease, a rare and inherited brain disorder that causes the progressive breakdown of nerve cells.
On September 29, 2026, the analyst firm H.C. Wainwright confirmed its “Buy” rating for uniQure. This rating suggests the firm believes the gene therapy stock is a good investment. At the time the rating was published, the stock price was $24.59, following a significant single-day drop of 37.14%.
The stock’s sharp decline followed a four-year update on its AMT-130 gene therapy clinical trial. While the therapy initially showed an 80% slowdown in disease progression after three years, this effectiveness dropped to 44% at the four-year mark. This new data involved 12 patients who received a high dose of the treatment.
As highlighted by Reuters, the treatment failed to achieve statistical significance in the trial. This is a measure used to determine if the results are reliable and not just due to chance. Analysts offered mixed reactions, with some describing the results as “clearly mixed” but still potentially “supportive of approval” for the Huntington’s disease treatment.
The company stated that the four-year results might be understated due to missing data. It also emphasized that the three-year data, which shows an 80% slowing of disease progression, is the primary basis for its application for regulatory approval with the U.S. Food and Drug Administration (FDA).
