uniQure stock crashes as Huntington’s gene therapy data weaken

Key Points:

• uniQure’s gene therapy AMT-130 for Huntington’s disease failed to show a statistically significant slowing of disease progression at 48 months, causing the company’s stock to drop 41% at market open.
• Despite missing the primary endpoint, uniQure and analysts remain optimistic due to positive trends in the data and a strong secondary endpoint, total functional capacity (TFC), which showed a consistent 61% slowing of disease progression.
• The primary endpoint analysis was complicated by missing external control data and potential methodological flaws, including an outlier that disproportionately affected results, leading to concerns that the treatment benefit may be understated.
• uniQure’s CEO emphasized the importance of TFC as a critical measure of patient quality of life, capturing abilities like employment and independent living, and this endpoint is the focus of their confirmatory trial.
• The company has filed for accelerated FDA approval based on earlier data, but it remains uncertain whether the FDA will require reanalysis with the updated 48-month data.