QURE: Why Analysts See Up to 63% Upside After 250% Single-Day Pop

Key Points

• Shares of biotech stock uniQure absolutely exploded on Sept. 24, more than tripling in value.
• The company's Huntington's disease trial results were extremely impressive, setting the company up to potentially become a huge player in this market.
• Multiple analysts are eyeing over 50% more upside in shares. If uniQure's drug hits sales estimates, the long-term opportunity could be much larger.

In one of the most incredible single-day moves this year, biotechnology stock uniQure NASDAQ: QURE surged by 284% on Sept. 24. This came as the firm released fantastic clinical results for its developmental medicine, AMT-130.

With such a massive spike, uniQure is turning the heads of Wall Street analysts. Most still see upside, implying a high-risk, high-reward opportunity for investors.

So, what exactly does uniQure do, and why are markets so excited about this stock? Additionally, what are the prospects of uniQure experiencing further appreciation? Let’s dive into these questions below.

Huntington’s Results Send QURE to the Moon

uniQure develops gene therapies, with Huntington’s disease being its most immediate area of focus. That’s where its treatment, AMT-130, comes in. On Sept. 24, the company provided Phase I/II Food and Drug Administration trial data that blew analysts away.

At 36 months, AMT-130 met its primary endpoint, showing a statistically significant 75% slowing of Huntington’s disease progression among high-dose patients. Researchers measured this using the composite Unified Huntington’s Disease Rating Scale (cUHDRS) compared to historical data. The composite provides a holistic view of disease progression, combining functional, cognitive, and motor tests.

The trial also met its secondary endpoint, showing a 60% slowing of disease progression based on Total Functional Capacity (TFC). TFC measures how well a patient can perform daily tasks without assistance. Within medical research, endpoints that measure the quality of patients' lives are particularly coveted. They provide clear evidence that patients will actually be better off and that the cost of the treatment is worth it. The strong results and the nature of the data bode well for AMT-130’s ability to receive FDA approval. Approval would translate into significant revenue for uniQure and support further gains in its share price.

Analysts Give Glowing Reviews of AMT-130 Data, Targets Indicate Upside

On the data release call, analysts noted that the AMT-130 results were “amazing” and “super encouraging." This highlights how the AMT-130 data surpassed expectations substantially. With the results, AMT-130 has a strong chance to become the first disease-modifying treatment for Huntington’s. This could be a golden ticket for the company, as AMT-130 works to slow disease progression rather than simply mitigate symptoms.

Overall, the MarketBeat consensus price target on uniQure is just over $67. However, excluding HC Wainwright’s $52 target, which it has not updated since 2024, the average moves up to $73. This target suggests shares could rise by 25%, a substantial improvement over the consensus.

The lowest updated target of $56 comes from Goldman Sachs. Even though it is the most bearish forecast, it still only implies around 4% downside in shares. On the other hand, highly bullish targets of $95 come from both Guggenheim and UBS. Achieving this level would require an additional gain in shares of around 63%.

AMT-130’s Path to Approval Looks Promising, But Biotech Risk Still Looms

To achieve this bullish target, it is likely that uniQure would need to gain FDA approval for AMT-130. uniQure plans to submit the drug for approval in Q1 2026. In Q2 2025, the company secured regulatory alignment with the FDA. The FDA said it would use data from the Phase I/II trial to assess the accelerated approval of AMT-130. This essentially bypassed the need for a Phase III trial, somewhat de-risking AMT-130’s path to approval.

Analysts appear bullish on AMT-130’s ability to gain FDA approval. Leerink Partners says that the current results support approval, creating limited regulatory risk around uniQure. Despite their low 12-month price target, Goldman Sachs sees a 90% probability of AMT-130 reaching peak annual sales of $2.5 billion. Currently, U.S. large-cap biotech and pharma stocks have a median forward price-to-sales (P/S) ratio of around 5x. Thus, if AMT-130 realizes Goldman’s peak sales forecast, there could be a lot of room for uniQure to surge past its current $4.3 billion market cap in the long term.

Overall, uniQure looks like it has a significant chance to gain FDA approval for AMT-130. This would be a key catalyst for shares to ride higher. Still, biotech stocks are some of the riskiest in the market, and none of these analysts have a crystal ball. If AMT-130 is not approved, the stock could give back a huge amount of the gain it has achieved. The Congressional Budget Office estimates that the FDA only approves 12% of drug candidates. This highlights the steep odds uniQure is up against and the riskiness of the biotech industry.