QURE Stock Clocks Worst Day In Over 10 Months: Four-Year Huntington’s Data Spooks Investors Despite Stronger Three-Year Filing Data

• UniQure filed an application with the FDA seeking approval for the gene therapy earlier this month. 
• Stifel said the overall case for AMT-130 looks unchanged heading into regulatory review.
• Wells Fargo argued the four-year composite reading is a secondary measure distorted by dropouts in the external control group and does not undo the three-year result that underpins the filing. 

Shares of uniQure (QURE) collapsed 37% on Tuesday after four-year results from its Huntington’s gene therapy study missed a key statistical test, even as the company said the treatment still slowed decline, and analysts largely kept bullish ratings.

Shares clocked their worst day since Nov. 2025 and closed Monday at $24.51, down from Monday’s closing price of $39.11.

What The QURE Trial Showed

uniQure said its one-time gene therapy, AMT-130, continued to slow Huntington’s disease in ongoing early- and mid-stage studies. Huntington’s is an inherited brain disorder with no approved treatment that changes how the disease unfolds.

Patients received a single dose delivered into the brain. Their results were compared with similar patients from a large long-term disease database, not a conventional placebo group.

The U.S. application filed earlier this month rests on three-year results. That snapshot covers 15 high-dose patients. On a broad score of thinking, movement and daily function, decline slowed by 80% following treatment. On a narrower score of everyday independence, it slowed decline by 67%. The company said both differences stood out in its analysis. The treatment was generally well tolerated. Most side effects were linked to the brain surgery used to deliver the therapy and later resolved.

The first four-year look, limited to 12 high-dose patients, was weaker. Slowing on the broad score fell to 44% and missed statistical significance. Everyday independence still showed a 61% slowing. uniQure said the later comparison likely understated the benefit because many people in the control database dropped out, leaving a group that appeared to decline more slowly than typical Huntington’s patients.

Analysts Stay Constructive On QURE

Stifel kept a Buy rating. It said the expanded three-year results still look robust and that four-year data continue to support a drug effect, even if the composite score faded. Daily-function results held up, the firm said, and the overall case looks unchanged heading into regulatory review.

H.C. Wainwright also kept a Buy rating and an $80 target. It called the broader three-year update encouraging but said the four-year composite result complicates the durability argument. Those later results were not part of the filed application. The next milestone, the firm said, is whether the FDA accepts the filing for review.

RBC Capital’s Luca Issi called the package mixed. The four-year composite miss and the apparent fade from year three were not what the firm wanted to see, though daily-function benefit stayed consistent and comparisons across time points are imperfect. RBC still thinks approval is possible in a disease with a sharply elevated suicide risk and said the stock move looks overdone. It kept an Outperform rating.

Wells Fargo reiterated an Overweight rating and a $75 target. It argued the four-year composite reading is a secondary measure distorted by dropouts in the external control group and does not undo the three-year result that underpins the filing. The firm called the selloff out of proportion and said the weakness may create a buying opportunity before the next regulatory update.

How Did QURE Retail Traders React?

On Stocktwits, retail sentiment around QURE stock stayed within the extremely bullish territory over the past 24 hours, while message volume rose from high to extremely high levels.

A Stocktwits user opined that despite the data announced on Tuesday, AMT-130 is “still the best hope for Huntington.”

Another dismissed the selloff on Tuesday as an overreaction.

QURE stock has gained 2% year-to-date after Tuesday’s selloff. 

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