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uniQure N.V. (QURE)
NASDAQ:QURE
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uniQure (QURE) AI Stock Analysis

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QURE

uniQure

(NASDAQ:QURE)

Rating:54Neutral
Price Target:
$14.50
▲(9.19% Upside)
The overall stock score of 54 reflects the significant financial challenges faced by uniQure, which weigh heavily on the assessment. However, positive sentiment from recent earnings call achievements and corporate events, particularly in advancing clinical programs, provide a balanced outlook. The technical and valuation aspects contribute neutrally, neither strongly enhancing nor detracting from the score.
Positive Factors
Clinical Advancement
Management remains optimistic about AMT-130's success due to fundamental differences in their program, such as the use of a clinical endpoint for efficacy and clean safety data in patients dosed.
Financial Position
Backed by $377M in cash and funding into the second half of 2027, uniQure remains an HCW Top Pick for 2025 in the coverage universe.
Regulatory Alignment
UniQure has reached regulatory alignment with the FDA on their statistical analysis plan and CMC requirements for AMT-130, viewed as a significant de-risking event.
Negative Factors
Leadership Concerns
The announcement of Dr. Prasad as the head of CBER put pressure on the stock due to his outspoken views on accelerated approval and gene therapy.
Market Pressure
Several gene therapy programs have recently received clinical holds or complete response letters, which has placed some pressure on QURE shares.
Regulatory Risk
Regulatory risk is a key focus for investors, especially with tumultuous news flow from CBER.

uniQure (QURE) vs. SPDR S&P 500 ETF (SPY)

uniQure Business Overview & Revenue Model

Company DescriptionuniQure N.V., a gene therapy company, engages in the development of treatments for patients suffering from genetic and other devastating diseases. Its lead program is Etranacogene dezaparvovec (AMT-061), which is in Phase III HOPE-B pivotal trial for the treatment of hemophilia B. The company also engages in developing AMT-130, a gene therapy that is in Phase I/II clinical study for the treatment of Huntington's disease; AMT-060, which is in Phase I/II clinical trial for the treatment of hemophilia B; AMT-210, a product candidate for the treatment of Parkinson's disease; AMT-260 for temporal lobe epilepsy; AMT-240, a preclinical product candidate for the treatment of autosomal dominant Alzheimer's disease; and AMT-161 for the treatment of amyotrophic lateral sclerosis. uniQure N.V. was founded in 1998 and is headquartered in Amsterdam, the Netherlands.
How the Company Makes MoneyuniQure generates revenue primarily through the commercialization of its gene therapy products, which include marketed products like Zynteglo for beta-thalassemia and other pipeline therapies in late-stage clinical development. The company also earns revenue from research collaborations and licensing agreements with pharmaceutical and biotechnology companies. These partnerships may include upfront payments, milestone payments based on the achievement of specific developmental or regulatory goals, and royalties on future sales of licensed products. Additionally, uniQure may benefit financially from government grants and funding aimed at supporting innovative research in gene therapy.

uniQure Key Performance Indicators (KPIs)

Any
Any
Revenue by Segment
Revenue by Segment
Highlights income from different business areas, offering insight into which segments drive growth and profitability, and where there might be opportunities or challenges.
Chart InsightsuniQure's revenue has experienced significant volatility, with sharp declines in collaboration and contract manufacturing revenues leading to a notable drop in Q1 2025. Despite this, the company is strategically positioned with a strong cash reserve and reduced expenses, allowing it to focus on advancing its gene therapy pipeline. The breakthrough therapy designation for AMT-130 and ongoing clinical trials highlight potential future growth, although current revenue challenges and adverse events in trials present risks. Investors should watch for regulatory updates and initial data from upcoming trials as potential catalysts.
Data provided by:Main Street Data

uniQure Earnings Call Summary

Earnings Call Date:Jul 29, 2025
(Q2-2025)
|
% Change Since: -11.35%|
Next Earnings Date:Oct 29, 2025
Earnings Call Sentiment Positive
The earnings call highlighted significant progress in uniQure's clinical pipeline, especially with AMT-130 for Huntington's disease, and a strong financial position. However, the company faced challenges with decreased revenue and trial recruitment issues in epilepsy. Overall, the highlights outweigh the lowlights, suggesting a positive outlook.
Q2-2025 Updates
Positive Updates
Advancement of AMT-130 for Huntington's Disease
AMT-130 has received breakthrough therapy designation from the FDA, indicating its potential as the first disease-modifying therapy for Huntington's disease. The company achieved regulatory alignment with the FDA on the statistical analysis plan and CMC requirements for a planned BLA submission in early 2026.
Encouraging Data for AMT-260 in Epilepsy
The first patient treated with AMT-260 for mesial temporal lobe epilepsy experienced a 92% reduction in seizure frequency over five months with no significant adverse events.
Strong Financial Position
Cash, cash equivalents, and investment securities totaled $377 million as of June 30, 2025, providing a solid foundation to fund operations into the second half of 2027.
Progress in Broader Clinical Pipeline
Advancements were made in clinical programs for AMT-191 for Fabry disease and AMT-162 for SOD1-ALS, with initial data expected in September 2025 and first half of 2026, respectively.
Negative Updates
Decrease in Revenue
Revenue for Q2 2025 was $5.3 million, down from $11.1 million in Q2 2024. The decrease was primarily due to reduced collaboration and contract manufacturing revenue.
Challenges in Trial Recruitment
Trial recruitment for AMT-260 in epilepsy has been challenging, though recent data has sparked increased interest and screening activity.
Company Guidance
During the second quarter of 2025, uniQure made significant progress in advancing its clinical programs, notably with AMT-130 for Huntington's disease, which received breakthrough therapy designation from the FDA. The company achieved regulatory alignment on its statistical analysis plan and CMC requirements, targeting a BLA submission in Q1 2026. The primary efficacy analysis will compare a 3-year change in cUHDRS in high-dose patients against a propensity score-matched external control arm from the ENROLL-HD study. Additionally, uniQure reported a 92% reduction in seizure frequency in its AMT-260 program for mesial temporal lobe epilepsy and is preparing for the commercial launch of AMT-130 in 2026. Financially, uniQure reported Q2 revenue of $5.3 million, with a strong cash position of $377 million, expected to fund operations into H2 2027.

uniQure Financial Statement Overview

Summary
uniQure faces significant financial challenges with declining revenues, persistent losses, and a weakened balance sheet. Despite some positive cash flow from investing activities, the overall financial health is concerning, with risks of insolvency due to negative equity and ongoing cash burn. Strategic financial restructuring and revenue growth are critical for future stability.
Income Statement
35
Negative
uniQure's income statement shows significant volatility in revenue and profitability. The company experienced a dramatic revenue decline from 2021 to 2023, with a slight recovery in 2024. Gross profit margin is low, and net profit margins are negative, indicating ongoing losses. The EBIT and EBITDA margins are also negative, reflecting operational challenges.
Balance Sheet
40
Negative
The balance sheet reveals a concerning financial structure with negative stockholders' equity in 2024, indicating potential insolvency risks. The debt-to-equity ratio is not calculable due to negative equity, but the company has managed to maintain a reasonable cash position. The equity ratio has deteriorated significantly, highlighting financial instability.
Cash Flow
45
Neutral
Cash flow analysis shows negative free cash flow and operating cash flow, indicating cash burn issues. The free cash flow to net income ratio is negative, which is concerning. However, the company has managed to generate positive cash flow from investing activities in 2024, providing some liquidity relief.
BreakdownDec 2024Dec 2023Dec 2022Dec 2021Dec 2020
Income Statement
Total Revenue27.12M15.84M106.48M524.00M37.51M
Gross Profit8.79M2.21M103.14M499.03M-84.89M
EBITDA-160.75M-253.10M-108.02M319.00M-126.97M
Net Income-239.56M-308.48M-126.79M329.59M-125.02M
Balance Sheet
Total Assets556.54M831.69M704.96M809.18M340.39M
Cash, Cash Equivalents and Short-Term Investments367.52M617.89M352.84M556.26M244.93M
Total Debt66.06M138.41M142.89M135.72M71.54M
Total Liabilities563.29M624.02M228.96M213.40M96.49M
Stockholders Equity-6.75M207.67M476.01M595.78M243.91M
Cash Flow
Free Cash Flow-186.10M-153.08M-162.75M270.52M-144.31M
Operating Cash Flow-182.73M-145.93M-145.06M287.96M-134.83M
Investing Cash Flow162.97M-205.69M-182.73M-67.39M-9.48M
Financing Cash Flow-59.49M362.72M1.45M94.86M7.44M

uniQure Technical Analysis

Technical Analysis Sentiment
Negative
Last Price13.28
Price Trends
50DMA
14.69
Negative
100DMA
13.70
Negative
200DMA
12.76
Positive
Market Momentum
MACD
-0.30
Positive
RSI
40.10
Neutral
STOCH
27.22
Neutral
Evaluating momentum and price trends is crucial in stock analysis to make informed investment decisions. For QURE, the sentiment is Negative. The current price of 13.28 is below the 20-day moving average (MA) of 14.31, below the 50-day MA of 14.69, and above the 200-day MA of 12.76, indicating a neutral trend. The MACD of -0.30 indicates Positive momentum. The RSI at 40.10 is Neutral, neither overbought nor oversold. The STOCH value of 27.22 is Neutral, not indicating any strong overbought or oversold conditions. Overall, these indicators collectively point to a Negative sentiment for QURE.

uniQure Risk Analysis

uniQure disclosed 65 risk factors in its most recent earnings report. uniQure reported the most risks in the "Tech & Innovation" category.
Finance & Corporate - Financial and accounting risks. Risks related to the execution of corporate activity and strategy
Latest Risks Added 0 New Risks

uniQure Peers Comparison

Overall Rating
UnderperformOutperform
Sector (60)
Financial Indicators
Name
Overall Rating
Market Cap
P/E Ratio
ROE
Dividend Yield
Revenue Growth
EPS Growth
76
Outperform
$1.22B5.9934.46%799.36%
60
Neutral
HK$18.08B4.77-4.00%3.35%9.59%-23.62%
57
Neutral
$1.16B-56.95%14.99%14.42%
54
Neutral
$728.63M-427.47%-48.25%33.77%
50
Neutral
$624.83M-24.75%2190.50%52.85%
43
Neutral
$915.68M2.31-41.00%-429.68%
40
Underperform
$308.61M-69.65%10.36%
* Healthcare Sector Average
Performance Comparison
Ticker
Company Name
Price
Change
% Change
QURE
uniQure
13.28
6.06
83.93%
RCKT
Rocket Pharmaceuticals
2.86
-16.09
-84.91%
SYRE
Spyre Therapeutics
15.16
-9.15
-37.64%
NTLA
Intellia Therapeutics
10.79
-10.98
-50.44%
CVAC
CureVac
5.44
2.19
67.38%
DAWN
Day One Biopharmaceuticals
6.10
-7.39
-54.78%

uniQure Corporate Events

Private Placements and FinancingBusiness Operations and Strategy
uniQure Announces $200 Million Sales Agreement
Positive
Jul 29, 2025

On July 29, 2025, uniQure N.V. announced a sales agreement with Leerink Partners LLC for an at-the-market offering program, allowing the company to sell its ordinary shares worth up to $200 million. This strategic move provides uniQure with flexibility in raising capital, potentially enhancing its financial position and supporting its ongoing operations in the competitive gene therapy market.

The most recent analyst rating on (QURE) stock is a Buy with a $25.00 price target. To see the full list of analyst forecasts on uniQure stock, see the QURE Stock Forecast page.

Executive/Board ChangesShareholder Meetings
uniQure Shareholders Approve Key Proposals at Annual Meeting
Neutral
Jun 13, 2025

On June 11, 2025, uniQure held its Annual Meeting where shareholders voted on several key proposals. The meeting resulted in the re-election of Matthew Kapusta as executive director and Dr. Robert Gut and Dr. Jeremy Springhorn as non-executive directors, each to serve until the 2028 annual general meeting. Additionally, all other proposals, including the adoption of the 2024 Dutch statutory annual accounts, discharge of board members’ liabilities, and various authorizations related to shares and compensation, were approved.

The most recent analyst rating on (QURE) stock is a Buy with a $20.00 price target. To see the full list of analyst forecasts on uniQure stock, see the QURE Stock Forecast page.

Product-Related AnnouncementsBusiness Operations and StrategyRegulatory Filings and Compliance
uniQure Aligns with FDA for AMT-130 BLA Approval
Positive
Jun 2, 2025

On June 2, 2025, uniQure announced alignment with the FDA on key components necessary for a Biologics License Application (BLA) for AMT-130, a gene therapy for Huntington’s disease. This alignment supports an accelerated approval pathway, with a BLA submission planned for early 2026. The company is leveraging extensive clinical data and FDA guidance to pursue the first disease-modifying treatment for Huntington’s disease, reflecting a significant step forward in addressing this unmet medical need.

The most recent analyst rating on (QURE) stock is a Buy with a $20.00 price target. To see the full list of analyst forecasts on uniQure stock, see the QURE Stock Forecast page.

Glossary
BuyA stock rated as a "Buy" is expected to perform better than the overall market or a specific benchmark over the near-to-medium term. This rating suggests the stock is likely to deliver higher returns compared to other stocks in the same sector or market index. Note: This is not investment advice; please consult a financial advisor before making investment decisions.
HoldA stock rated as a "Hold" is expected to perform in line with the overall market or a specific benchmark. This rating indicates that the stock is neither particularly compelling nor unfavorable for investment. Note: This is not investment advice; please consult a financial advisor before making investment decisions.
SellA stock rated as a "Sell" is expected to perform worse than the overall market or a specific benchmark over the near-to-medium term. This rating suggests the stock may deliver lower returns compared to other stocks in the same sector or market index. Note: This is not investment advice; please consult a financial advisor before making investment decisions.

Disclaimer

This AI Analyst Stock Report is automatically generated by our AI systems using advanced algorithms and publicly available financial, technical, and market data. While the information provided aims to be accurate and insightful, it is intended for informational purposes only and should not be considered financial advice. Any content created by an AI (Artificial Intelligence) system may contain inaccuracies and/or contain errors. Investing in stocks carries inherent risks, and past performance is not indicative of future results. This report does not account for your personal financial circumstances, objectives, or risk tolerance. Always conduct your own research or consult with a qualified financial advisor before making investment decisions. The analysis and recommendations provided are based on historical and current data and may not fully reflect future market conditions or unexpected developments. Neither the creators of this report nor its affiliated entities guarantee the accuracy, completeness, or reliability of the information presented. Use this report at your own discretion and risk.Date of analysis: Jul 31, 2025