QURE Stock Fades After AMT-130 filing — But William Blair Sees 58% Upside

• William Blair noted AMT-130 will potentially be the first approved therapy to treat the underlying cause of Huntington's in the first half of 2027.
• It views the biologics license application acceptance as an incremental catalyst for the stock, saying it will further reduce regulatory risk.
• The company has also applied for approval of the gene therapy in the UK.

Shares of uniQure (QURE) swung sharply on Wednesday after the company said it had filed for U.S. and U.K. approval of its Huntington’s disease gene therapy, AMT-130, while William Blair resumed coverage of uniQure with an Outperform rating and a $73 fair value estimate.  

The stock opened up about 6% but eventually closed the day down 3%, likely because investors had already priced in the application filing.

QURE Seeks AMT-130 Approval

Before the market opened, uniQure announced it had submitted a Biologics License Application to the FDA seeking accelerated approval of ifezuntirgene inilparvovec (AMT-130). It also filed a Marketing Authorization Application with the U.K.’s MHRA. Both filings are based on three-year trial results showing the disease progressed more slowly with AMT-130 than in similar untreated patients. The company asked the FDA for priority review, which could cut the review clock to six months after the agency’s filing review period. Four-year data from the same study are still due later this month.

Investors had already priced in a third-quarter filing after uniQure said in June that the FDA agreed the existing three-year dataset could support an accelerated-approval BLA.

Why The Filing Matters

Huntington’s is a rare inherited brain disease that causes motor problems, behavioral changes, and cognitive decline. About 75,000 people live with it in the U.S., EU, and U.K. There is still no approved therapy that delays onset or slows progression. AMT-130 is designed to silence the gene responsible for the condition. It is the first investigational Huntington’s treatment to receive both Breakthrough Therapy and RMAT designations from the FDA.

If approved, AMT-130 would become uniQure’s second marketed gene therapy after Hemgenix for hemophilia B. Accelerated approval would still require a confirmatory study.

The rest of uniQure’s pipeline is in earlier stages of development. AMT-260 is in early-to-mid stage trials for hard-to-treat temporal lobe epilepsy. AMT-191 is in a similar stage for Fabry disease, making AMT-130 the company’s only near-term catalyst.

Wall Street Weighs In

Earlier on Wednesday, William Blair resumed coverage of uniQure with an Outperform rating and a $73 fair value estimate. The firm said it sees a "clearer pathway" to FDA approval for AMT-130 in Huntington's disease and noted AMT-130 will potentially be the first approved therapy to treat the underlying cause of Huntington's in the first half of 2027. It views the biologics license application acceptance as an incremental catalyst for the stock, saying it will further reduce regulatory risk.

William Blair’s fair value estimate implies a potential upside of about 58% from the stock’s closing price on Wednesday.

How Did QURE Retail Traders React?

On Stocktwits, retail sentiment around QURE stock rose from ‘neutral’ to ‘bullish’ over the past 24 hours, while message volume increased from ‘low’ to ‘normal’ levels.

QURE stock has gained 94% year-to-date. 

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