Last Update 01 Nov 25

uniQure's analyst price targets have been raised substantially, with increases ranging from $24 to as high as $80 per share. Analysts cite strong three-year Huntington's disease trial data and growing confidence in the approval and impact of AMT-130.

Analyst Commentary

Analyst coverage of uniQure has intensified following the release of positive three-year data from AMT-130 in Huntington's disease. Investors and Street observers are weighing both the potential upsides and the remaining risks as the company approaches key regulatory milestones.

Bullish Takeaways

Bullish analysts have raised price targets substantially, with some suggesting shares could more than double based on the transformative nature of the Huntington's disease data and management's growing confidence in regulatory approval.
There is consensus that AMT-130's statistically significant slowing of progression, as observed across multiple functional and cognitive measures, sets a new clinical benchmark. This could support rapid regulatory uptake and commercial adoption.
The probability of approval for AMT-130 has increased significantly in both the U.S. and European markets. Considerable excitement has been noted among patient advocacy communities and early manifest Huntington's disease populations.
Some analysts see AMT-130 as a leading candidate in the gene therapy space for Huntington's, with the potential for substantial long-term value creation. Peak sales projections reach as high as $2.5 billion by 2035.

Bearish Takeaways

Cautious analysts emphasize that despite robust data, some patients and providers may opt for a wait-and-see approach until full regulatory approval is secured. This could potentially delay near-term uptake.
There are concerns about external factors such as competition, including whether rival therapies will be able to meet the high efficacy bar set by AMT-130 or trigger increased scrutiny from regulators.
The gene therapy field continues to face safety concerns, particularly regarding adverse events like acute liver failure seen in other AAV-based products. However, AMT-130's administration route may mitigate this specific risk.
Uncertainty around the timing of major data releases and regulatory filing timelines could introduce volatility in share performance leading up to key inflection points.

What's in the News

uniQure announced positive topline data from its pivotal Phase I/II study of AMT-130 for Huntington's disease, meeting the primary endpoint and demonstrating statistically significant slowing of disease progression at 36 months (Key Developments).
AMT-130 received both Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations from the U.S. FDA, enhancing its potential path to approval (Key Developments).
uniQure completed a follow-on equity offering of $299.9 million, issuing ordinary shares and pre-funded warrants (Key Developments).
Certain options, restricted stock, common stock, and performance stock held by directors and executive officers are subject to a lock-up agreement, which ends on November 25, 2025 (Key Developments).

Valuation Changes

Fair Value Estimate has remained stable at $75.09 per share, with no material change since the prior assessment.
Discount Rate has increased slightly, moving from 6.70% to 6.76%. This reflects a marginally higher perceived risk or cost of capital.
Revenue Growth Forecast remains unchanged, projecting a robust 223.39% annual increase.
Net Profit Margin Estimate is stable at 20.13%, with no recent adjustments.
Future Price/Earnings (P/E) Ratio is essentially unchanged, rising modestly from 62.61x to 62.72x.

Accelerated approval of AMT-130 for Huntington's could significantly boost future revenues as it would be a pioneering treatment.
Strategic financial actions and strong cash reserves provide resilience and flexibility, supporting pipeline advancement and likely improving margins.
Reliance on AMT-130’s success and regulatory outcomes, amid revenue challenges and manufacturing changes, heightens exposure to product development and supply chain risks.

Catalysts

About uniQure

Develops treatments for patients suffering from rare and other devastating diseases in the United States.What are the underlying business or industry changes driving this perspective?

The potential accelerated approval for AMT-130 in treating Huntington's disease could significantly boost future revenues as it would be one of the first disease-modifying treatments available for this condition.
Expansion of the clinical pipeline with new studies in refractory temporal lobe epilepsy, Fabry disease, and SOD1-ALS could lead to additional revenue streams if these treatments are successful and commercialized.
The company’s strategic financial moves, including reducing cash burn and strengthening the balance sheet through a targeted offering, provide financial resilience and flexibility to support pipeline advancement and potentially improve net margins.
With strong cash reserves of over $400 million, uniQure is well-positioned to fund operations through the launch of AMT-130 and support other key data readouts, likely impacting earnings positively.
Impressive clinical results and newly granted breakthrough therapy designation for AMT-130 might enhance investor confidence, potentially driving stock value through anticipated increases in revenue and investor sentiment.

uniQure Future Earnings and Revenue Growth

Assumptions

How have these above catalysts been quantified?

Analysts are assuming uniQure's revenue will grow by 147.5% annually over the next 3 years.
Analysts are not forecasting that uniQure will become profitable in next 3 years. To represent the Analyst Price Target as a Future PE Valuation we will estimate uniQure's profit margin will increase from -1077.1% to the average US Biotechs industry of 10.5% in 3 years.
If uniQure's profit margin were to converge on the industry average, you could expect earnings to reach $32.3 million (and earnings per share of $0.48) by about July 2028, up from $-217.6 million today. However, there is a considerable amount of disagreement amongst the analysts with the most bullish expecting $439.8 million in earnings, and the most bearish expecting $-242.8 million.
In order for the above numbers to justify the analysts price target, the company would need to trade at a PE ratio of 90.8x on those 2028 earnings, up from -3.6x today. This future PE is greater than the current PE for the US Biotechs industry at 16.5x.
Analysts expect the number of shares outstanding to grow by 7.0% per year for the next 3 years.
To value all of this in today's terms, we will use a discount rate of 7.2%, as per the Simply Wall St company report.

uniQure Future Earnings Per Share Growth

Risks

What could happen that would invalidate this narrative?

The company's reliance on successful regulatory outcomes, specifically the BLA submission for AMT-130, coupled with uncertainties about FDA interactions and changes in FDA leadership, poses a risk that could impact revenue and projected market entry timelines.
The decrease in revenue from $8.5 million in Q1 2024 to $1.6 million in Q1 2025 highlights challenges in sustaining revenue streams, which may affect the company's short-term financial health.
The divestiture of the GMP manufacturing facility and reliance on external entities for manufacturing could impact cost efficiencies or lead to potential supply chain disruptions, affecting net margins.
The presence of serious adverse events related to immunosuppression in clinical trials indicates risks that may require additional resources to address or delay product timelines, potentially impacting future earnings.
The company's strong dependency on the success of AMT-130 and limited diversification of revenue-generating products increase their exposure to product development risks which might impact future revenue if any set targets are not met.

Valuation

How have all the factors above been brought together to estimate a fair value?

The analysts have a consensus price target of $35.514 for uniQure based on their expectations of its future earnings growth, profit margins and other risk factors. However, there is a degree of disagreement amongst analysts, with the most bullish reporting a price target of $69.56, and the most bearish reporting a price target of just $13.41.
In order for you to agree with the analyst's consensus, you'd need to believe that by 2028, revenues will be $306.4 million, earnings will come to $32.3 million, and it would be trading on a PE ratio of 90.8x, assuming you use a discount rate of 7.2%.
Given the current share price of $14.25, the analyst price target of $35.51 is 59.9% higher.
We always encourage you to reach your own conclusions though. So sense check these analyst numbers against your own assumptions and expectations based on your understanding of the business and what you believe is probable.

How well do narratives help inform your perspective?

Disclaimer

AnalystConsensusTarget is a tool utilizing a Large Language Model (LLM) that ingests data on consensus price targets, forecasted revenue and earnings figures, as well as the transcripts of earnings calls to produce qualitative analysis. The narratives produced by AnalystConsensusTarget are general in nature and are based solely on analyst data and publicly-available material published by the respective companies. These scenarios are not indicative of the company's future performance and are exploratory in nature. Simply Wall St has no position in the company(s) mentioned. Simply Wall St may provide the securities issuer or related entities with website advertising services for a fee, on an arm's length basis. These relationships have no impact on the way we conduct our business, the content we host, or how our content is served to users. The price targets and estimates used are consensus data, and do not constitute a recommendation to buy or sell any stock, and they do not take account of your objectives, or your financial situation. Note that AnalystConsensusTarget's analysis may not factor in the latest price-sensitive company announcements or qualitative material.

QURE: Pivotal Three-Year Huntington’s Data Will Drive Approval and Market Uptake