uniQure N.V. (QURE)
NASDAQ:QURE
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uniQure
(NASDAQ:QURE)
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Rating:45Neutral
Price Target:
$14.50
▼(-15.50% Downside)
Action:ReiteratedDate:03/10/26
The score is held down primarily by weak financial performance (persistent losses and cash burn) and a bearish technical setup (below key moving averages, negative MACD). The earnings call provides some offset via strong clinical efficacy signals and improved liquidity, but the FDA’s Phase III requirement and program safety pauses materially raise execution and timeline risk; valuation remains difficult to support with negative earnings and no dividend.
Positive Factors
AMT-130 clinical efficacy
Robust multi-year AMT-130 efficacy versus a large external comparator demonstrates potential for a durable, disease-modifying one-time therapy. Such clear clinical signal strengthens uniQure’s scientific differentiation and long-term commercial relevance if regulatory pathways can be secured.
Negative Factors
Major FDA regulatory hurdle
The FDA’s view that external‑control Phase I/II data are insufficient raises a material structural barrier: sham‑controlled Phase IIIs are costly, ethically complex and lengthen timelines. This increases execution risk, capital requirements and the probability of protracted regulatory dialogue.
Read all positive and negative factors
Positive Factors
Negative Factors
AMT-130 clinical efficacy
Robust multi-year AMT-130 efficacy versus a large external comparator demonstrates potential for a durable, disease-modifying one-time therapy. Such clear clinical signal strengthens uniQure’s scientific differentiation and long-term commercial relevance if regulatory pathways can be secured.
Read all positive factors
uniQure (QURE) vs. SPDR S&P 500 ETF (SPY)
Market Cap
$1.06B
Dividend YieldN/A
Average Volume (3M)4.73M
Price to Earnings (P/E)―
Beta (1Y)1.48
Revenue Growth-44.90%
EPS Growth11.49%
CountryUS
Employees209
SectorHealthcare
Sector Strength45
IndustryBiotechnology
Share Statistics
EPS (TTM)-0.57
Shares Outstanding61,603,770
10 Day Avg. Volume1,966,010
30 Day Avg. Volume4,733,432
Financial Highlights & Ratios
PEG Ratio0.23
Price to Book (P/B)6.92
Price to Sales (P/S)85.48
P/FCF Ratio-7.73
Enterprise Value/Market Cap1.43
Enterprise Value/Revenue94.02
Enterprise Value/Gross Profit132.27
Enterprise Value/Ebitda-10.63
Forecast
1Y Price Target
$32.00Price Target Upside86.48% Upside
Rating ConsensusModerate Buy
Number of Analyst Covering12
EPS Forecast (FY)-3.61
Revenue Forecast (FY)$34.70M
uniQure Business Overview & Revenue Model
Company Description
uniQure 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 ...
How the Company Makes Money
uniQure makes money primarily through (1) collaboration and license-related revenue and (2) product-related economics from Hemgenix via partnerships. For Hemgenix, uniQure entered into an arrangement with CSL (CSL Behring) under which CSL leads co...
uniQure Key Performance Indicators (KPIs)
Any
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.
Highlights income from different business areas, offering insight into which segments drive growth and profitability, and where there might be opportunities or challenges.
Data provided by:
The Fly
The FlyuniQure Earnings Call Summary
Earnings Call Date:Mar 02, 2026
(Q4-2025)
| % Change Since: |
Next Earnings Date:May 12, 2026
Earnings Call Sentiment Neutral
The call communicated major positive clinical advances (notably strong 3‑year AMT‑130 efficacy, supportive Fabry and TLE data) and a strengthened balance sheet, but these were tempered by significant regulatory resistance from the FDA (recommendation for a sham‑controlled Phase III), safety‑related pauses/holds in two programs (AMT‑191 dosing pause and AMT‑162 hold), and a substantial year‑over‑year revenue decline. Management emphasized active regulatory engagement, continued data generation (4‑year AMT‑130 analysis) and pursuit of ex‑U.S. pathways and early access options, while acknowledging material uncertainty around Phase III design, cost and feasibility.Positive Updates
AMT-130: Robust 3‑Year Clinical Efficacy
Phase I/II 3‑year data demonstrated a statistically significant 75% slowing of disease progression on the composite UHDRS and a 60% slowing on Total Functional Capacity versus an external Enroll HD comparator; reduction in neurofilament light and supportive trends across other clinically meaningful endpoints. Company plans a 4‑year analysis in Q3 2026 and intends to amend the SAP and submit the analysis to regulators.
Negative Updates
Regulatory Setback: FDA Recommends Sham‑Controlled Phase III
At a Type A meeting the FDA stated Phase I/II data compared to an external control are unlikely to provide primary evidence for a BLA and strongly recommended a randomized double‑blind sham surgery‑controlled Phase III trial, creating a material regulatory and feasibility hurdle and potential need for a large, costly, and ethically sensitive study.
Read all updates
Q4-2025 Updates
Positive
Negative
AMT-130: Robust 3‑Year Clinical Efficacy
Phase I/II 3‑year data demonstrated a statistically significant 75% slowing of disease progression on the composite UHDRS and a 60% slowing on Total Functional Capacity versus an external Enroll HD comparator; reduction in neurofilament light and supportive trends across other clinically meaningful endpoints. Company plans a 4‑year analysis in Q3 2026 and intends to amend the SAP and submit the analysis to regulators.
Read all positive updates
Company Guidance
The key regulatory guidance from the call was that the FDA reaffirmed it is unlikely to accept Phase I/II data versus an external control as primary evidence and strongly recommended a randomized, double‑blind, sham surgery‑controlled Phase III study (discussed at an October 2025 pre‑BLA and a Type A meeting on Jan 30, 2026, with minutes received Feb 27), prompting uniQure to plan a Type B meeting in Q2 2026 to discuss Phase III design and to amend the Phase I/II SAP to include a 4‑year analysis expected in Q3 2026 (including 12 patients at 4 years and all patients reaching 3 years); uniQure noted its 3‑year AMT‑130 data showed a 75% slowing on the composite UHDRS and 60% slowing on Total Functional Capacity with reductions in neurofilament light versus an Enroll HD external comparator (>30,000 participants, 14 years of data), but the 12‑month sham‑controlled U.S. cohort showed no worsening at 12 months, which influenced FDA views; the company will pursue constructive FDA dialogue, ex‑U.S. regulator discussions (e.g., EMA/MHRA), and feasibility work (including patient burden/ethical considerations around multiyear sham surgery), while continuing program updates (AMT‑191: 11 patients, all off ERT, dose‑dependent alpha‑Gal A increases durable >1 year to 4 months with 2 mid‑dose Grade 3 LFTs leading to a dosing pause; AMT‑260: six patients in Cohort 1 with ≥6 months follow‑up and Cohort 2 enrollment expected complete by midyear) and citing a cash balance of $622.5M as of 12/31/25 (vs. $367.5M a year earlier, including ~$404.2M raised) with runway into H2 2026.uniQure Financial Statement Overview
Summary
Income Statement
22
Negative
Balance Sheet
55
Neutral
Cash Flow
30
Negative
| Breakdown | Dec 2025 | Dec 2024 | Dec 2023 | Dec 2022 | Dec 2021 |
|---|---|---|---|---|---|
Income Statement | |||||
| Total Revenue | 16.10M | 27.12M | 15.84M | 106.48M | 524.00M |
| Gross Profit | -347.00K | 8.79M | 2.21M | 103.14M | 499.03M |
| EBITDA | -178.58M | -160.75M | -253.10M | -108.02M | 319.00M |
| Net Income | -198.97M | -239.56M | -308.48M | -126.79M | 329.59M |
Balance Sheet | |||||
| Total Assets | 824.91M | 556.54M | 831.69M | 704.96M | 809.18M |
| Cash, Cash Equivalents and Short-Term Investments | 622.54M | 367.52M | 617.89M | 352.84M | 556.26M |
| Total Debt | 536.59M | 66.06M | 138.41M | 142.89M | 135.72M |
| Total Liabilities | 626.01M | 563.29M | 624.02M | 228.96M | 213.40M |
| Stockholders Equity | 198.90M | -6.75M | 207.67M | 476.01M | 595.78M |
Cash Flow | |||||
| Free Cash Flow | -177.96M | -186.10M | -153.08M | -162.75M | 270.52M |
| Operating Cash Flow | -177.96M | -182.73M | -145.93M | -145.06M | 287.96M |
| Investing Cash Flow | -321.62M | 162.97M | -205.69M | -182.73M | -67.39M |
| Financing Cash Flow | 415.40M | -59.49M | 362.72M | 1.45M | 94.86M |
uniQure Technical Analysis
Neutral
17.16
Price Trends
19.47
Negative
22.07
Negative
25.15
Negative
Market Momentum
-0.69
Negative
51.29
Neutral
74.21
Neutral
Evaluating momentum and price trends is crucial in stock analysis to make informed investment decisions. For QURE, the sentiment is Neutral. The current price of 17.16 is above the 20-day moving average (MA) of 16.06, below the 50-day MA of 19.47, and below the 200-day MA of 25.15, indicating a neutral trend. The MACD of -0.69 indicates Negative momentum. The RSI at 51.29 is Neutral, neither overbought nor oversold. The STOCH value of 74.21 is Neutral, not indicating any strong overbought or oversold conditions. Overall, these indicators collectively point to a Neutral sentiment for QURE.
uniQure Risk Analysis
uniQure disclosed 69 risk factors in its most recent earnings report. uniQure reported the most risks in the "Legal & Regulatory" 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
UnderperformOutperform
Sector (51)
Name | Overall Rating | Market Cap | P/E Ratio | ROE | Dividend Yield | Revenue Growth | EPS Growth |
|---|---|---|---|---|---|---|---|
70 Outperform | $2.26B | 6.20 | -76.45% | ― | 45.38% | 92.85% | |
56 Neutral | $1.83B | -8.91 | -67.11% | ― | ― | -20.08% | |
52 Neutral | $2.06B | -3.82 | -31.07% | ― | 36.26% | -158.98% | |
52 Neutral | $1.65B | -7.89 | -57.52% | ― | -100.34% | 52.85% | |
51 Neutral | $7.86B | -0.30 | -43.30% | 2.27% | 22.53% | -2.21% | |
46 Neutral | $2.47B | -8.83 | -57.61% | ― | -4.44% | 63.58% | |
45 Neutral | $1.06B | -6.92 | -174.03% | ― | -44.90% | 11.49% |
* Healthcare Sector Average
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uniQure
17.16
8.40
95.89%
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uniQure Corporate Events
Business Operations and StrategyFinancial DisclosuresPrivate Placements and FinancingRegulatory Filings and Compliance
uniQure Highlights 2025 Results and Gene Therapy Progress
Neutral
Mar 2, 2026
On March 2, 2026, uniQure reported its fourth-quarter and full-year 2025 financial results and highlighted progress in its gene therapy pipeline, led by AMT-130 for Huntington’s disease, which showed statistically significant 36‑month ...
Business Operations and StrategyProduct-Related AnnouncementsRegulatory Filings and Compliance
uniQure updates gene therapy pipeline and regulatory outlook
Positive
Jan 13, 2026
In January 2026, uniQure N.V. updated its corporate presentation to highlight its positioning as a leader in gene therapy, underscoring a robust clinical pipeline and substantial liquidity to fund development across multiple rare and serious neuro...
Business Operations and StrategyProduct-Related AnnouncementsRegulatory Filings and Compliance
uniQure prepares for FDA Type A meeting on AMT-130
Positive
Jan 9, 2026
On January 9, 2026, uniQure announced that the U.S. Food and Drug Administration has scheduled a Type A meeting with the company to discuss the Biologics License Application data package supporting a potential accelerated approval of AMT-130, its ...
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.