uniQure (QURE) Stock Plunges 48% After Huntington’s Drug Data Miss

TLDR

• uniQure stock fell 48% to $20.50 in premarket trading on Tuesday.
• The drop followed a new 48-month analysis of the Huntington’s disease therapy AMT-130.
• AMT-130 showed a 44% slowing of disease progression at 48 months, but the result did not clear the statistical bar.
• uniQure says its FDA filing still rests on strong 36-month data covering all 15 high-dose patients.
• The company submitted a Biologics License Application to the FDA earlier this month under the accelerated pathway.

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uniQure stock dropped 48% to $20.50 in premarket trading on Tuesday. The move came after the Dutch biotech released fresh data on its Huntington’s disease therapy.

uniQure N.V., QURE

The therapy is called AMT-130. It’s designed to slow the progression of the fatal neurological disorder by cutting levels of a toxic protein in the brain.

In a 48-month analysis of 12 high-dose patients, AMT-130 showed a 44% slowing of disease progression. The result did not clear the statistical bar researchers had set.

That’s a sharp turn from June, when uniQure said it would seek regulatory approval for the therapy. The stock jumped on that news at the time.

What the New Data Showed

uniQure compared trial patients against an outside control group. Over time, more people dropped out of that control group.

That left a comparison group made up mostly of patients whose disease happened to be progressing more slowly on its own. uniQure said this may have skewed the 48-month numbers.

The company pointed to a separate 36-month analysis as the more reliable dataset. That version covered all 15 high-dose patients and came back statistically strong.

A different 36-month look at just 12 high-dose patients showed an 80% decline in disease progression. That figure was based on data collected through June 30, 2026.

uniQure also said patients tolerated the treatment well at both doses tested. Safety was not cited as a reason for Tuesday’s drop.

The FDA Timeline

Earlier this month, uniQure filed a Biologics License Application with the FDA. The submission sought accelerated approval for AMT-130, also known as ifezuntirgene inilparvovec.

That filing was built on the 36-month data, not the newer 48-month numbers. uniQure said the FDA agreed in a June meeting that the 36-month results from 12 high-dose patients would be enough to support the application.

The agency had pushed back on the therapy before. Last year the FDA said Phase I and II data weren’t enough to support a filing, and it suggested another study as recently as March.

Huntington’s disease is inherited and always fatal. There is currently no approved treatment that changes how the disease progresses, only options that manage symptoms.

uniQure has worked on AMT-130 for nearly a decade. It remains the central program in the company’s pipeline.

uniQure said Tuesday’s update is still meaningful for patients given the lack of other disease-modifying options. The company plans to present the full findings at a future medical meeting.

Tuesday’s decline was on pace to be uniQure’s largest single-day percentage drop since November, according to Dow Jones Market Data.