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uniQure
(NASDAQ:QURE)
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Rating:53Neutral
Price Target:
$22.00
▼(-56.90% Downside)
Action:Reiterated
Date:09/30/26
QURE’s score is held down primarily by weak financial performance (very small revenue, extreme losses, and sustained cash burn) and bearish technical trends (price far below major moving averages with negative MACD). Offsetting these are a constructive earnings-call outlook and positive corporate milestones centered on AMT-130 (clearer regulatory path, major submissions, supportive longer-term data) plus a strengthened cash runway that reduces near-term financing pressure.
Positive Factors
AMT-130 regulatory progress
Submitting AMT-130 applications in both the U.S. and U.K. marks a major transition from clinical development toward potential commercialization. The therapy’s Breakthrough, RMAT and Fast Track designations may support regulatory interaction and strengthen uniQure’s position in central nervous system gene therapy.
Negative Factors
Persistent cash burn and deep losses
The company remains far from self-funded operations, with substantial operating and free-cash-flow losses relative to its small revenue base. Continued investment in clinical programs and commercialization therefore depends on existing capital, future financing or collaboration proceeds, leaving execution sensitive to spending and timing.
Read all positive and negative factors
Positive Factors
Negative Factors
AMT-130 regulatory progress
Submitting AMT-130 applications in both the U.S. and U.K. marks a major transition from clinical development toward potential commercialization. The therapy’s Breakthrough, RMAT and Fast Track designations may support regulatory interaction and strengthen uniQure’s position in central nervous system gene therapy.
Read all positive factors
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 (QURE) vs. SPDR S&P 500 ETF (SPY)
Market Cap
$1.75B
Dividend YieldN/A
Average Volume (3M)2.88M
Price to Earnings (P/E)―
Beta (1Y)1.74
Revenue Growth30.24%
EPS Growth-3.25%
CountryUS
Employees221
SectorHealthcare
Sector Strength45
IndustryBiotechnology
Share Statistics
EPS (TTM)-4.04
Shares Outstanding69,366,410
10 Day Avg. Volume1,299,252
30 Day Avg. Volume2,884,500
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 Cap1.59
Enterprise Value/Revenue149.58
Enterprise Value/Gross Profit656.41
Enterprise Value/Ebitda-15.43
Forecast
1Y Price Target
$51.67Price Target Upside1.21% Upside
Rating ConsensusStrong Buy
Number of Analyst Covering9
EPS Forecast (FY)-3.88
Revenue Forecast (FY)$23.45M
uniQure Business Overview & Revenue Model
Company Description
uniQure N.V. develops treatments for patients suffering from rare and other devastating diseases in the United States. The company offers HEMGENIX that allows people living with hemophilia B to produce factor IX, which can lower the risk of bleedi...
How the Company Makes Money
uniQure primarily makes money through (1) collaboration and licensing revenue and (2) product revenue from commercialized therapies, supplemented at times by contract/manufacturing-related income and other collaboration-related payments.
1) Colla...
uniQure Earnings Call Summary
Earnings Call Date:Jul 29, 2026
(Q2-2026)
| % Change Since: |
Next Earnings Date:Nov 04, 2026
Earnings Call Sentiment Positive
Overall the call conveyed strong positive momentum: regulatory progress (FDA alignment on using 3-year data as the basis for an accelerated BLA), an on-track BLA/MAA submission timeline, upcoming 4-year AMT-130 data, encouraging early efficacy and biological signals in AMT-260 and AMT-191, and a materially strengthened balance sheet with ~$810M cash supporting operations into 2030. Notable challenges include a pause in higher-dose AMT-191 dosing after Grade 3 LFTs (dose-limiting toxicity), mixed early efficacy in the AMT-260 low-dose cohort, increased SG&A to fund commercialization (near-term burn), and the operational/regulatory requirement to have the confirmatory trial well underway at the time of any accelerated approval. Taken together, the positives (regulatory clarity, data momentum, cash runway, early signals) outweigh the manageable clinical and execution risks identified.Positive Updates
Regulatory alignment for AMT-130 and near-term submission timeline
FDA Type B meeting confirmed that the 3-year Phase I/II AMT-130 data are acceptable as the primary basis for a BLA under the accelerated approval pathway; company is on track to submit the BLA in Q3 2026 and expects a parallel MHRA (U.K.) submission in Q3 2026.
Negative Updates
AMT-191 dosing paused after Grade 3 liver enzyme elevations
Additional dosing in the mid- and high-dose AMT-191 cohorts is paused pending an FDA-agreed monitoring and management plan after two patients in the mid-dose cohort experienced Grade 3 LFT elevations; the IDMC characterized these as dose-limiting toxicities. The events resolved after immunosuppression as of end-May 2026.
Read all updates
Q2-2026 Updates
Positive
Negative
Regulatory alignment for AMT-130 and near-term submission timeline
FDA Type B meeting confirmed that the 3-year Phase I/II AMT-130 data are acceptable as the primary basis for a BLA under the accelerated approval pathway; company is on track to submit the BLA in Q3 2026 and expects a parallel MHRA (U.K.) submission in Q3 2026.
Read all positive updates
Company Guidance
The company provided clear near-term regulatory and programmatic guidance: uniQure is on track to submit a BLA for AMT‑130 in Q3 2026 (and an MAA to the MHRA in Q3), with the FDA agreeing that the 3‑year Phase I/II data can serve as the primary basis for accelerated approval but asking that the confirmatory randomized, standard‑of‑care‑controlled trial (with total functional capacity at 36 months as the proposed primary endpoint) be aligned and well underway—or potentially fully enrolled—around the time of approval; uniQure intends to initiate that global confirmatory study ASAP. Key data milestones include a June 30, 2026 data cutoff for the planned 4‑year AMT‑130 analysis to be presented in September (analysis to include 12 high‑ and 12 low‑dose patients at 4 years plus 3 additional high‑dose patients with 3‑year follow‑up, cUHDRS/TFC comparisons vs a ~26,000‑participant Enroll‑HD natural history pool, and CSF NfL change). For other programs, AMT‑260 low‑dose cohort data (May 29, 2026 cutoff) showed 3 of 6 patients with 79–100% reductions in disabling seizures in months 4–6 (others −33% to +36%), no related SAEs, and higher‑dose cohort enrollment expected to complete imminently with updates in H1 2027; AMT‑191 (March 15, 2026 cutoff) has 11 dosed patients across 3 dose levels with dose‑dependent alpha‑Gal A increases, stable plasma lyso‑Gb3, all 11 off ERT, but 2 Grade‑3 LFTs in the mid‑dose cohort confirmed as DLTs (resolved with immunosuppression by end‑May 2026) and mid/high dosing paused pending an FDA monitoring/management plan. Financially, Q2 revenue was $5.8M (vs. $5.3M YoY), R&D expense $34.0M (vs. $35.4M), SG&A $17.4M (vs. $13.5M), and cash/investments were $810.3M as of June 30, 2026 (up from $622.5M at 12/31/25), which the company expects to fund operations into 2030; AMT‑130 holds Breakthrough, RMAT and Fast Track designations and priority review will be requested at submission.uniQure Financial Statement Overview
Summary
Income Statement
22
Negative
Balance Sheet
55
Neutral
Cash Flow
28
Negative
| Breakdown | TTM | Dec 2025 | Dec 2024 | Dec 2023 | Dec 2022 | Dec 2021 |
|---|---|---|---|---|---|---|
Income Statement | ||||||
| Total Revenue | 18.67M | 16.10M | 27.12M | 15.84M | 106.48M | 524.00M |
| Gross Profit | 4.25M | 14.41M | 8.79M | 2.21M | 103.14M | 499.03M |
| EBITDA | -181.06M | -116.59M | -160.75M | -253.10M | -108.02M | 319.00M |
| Net Income | -252.21M | -198.97M | -239.56M | -308.48M | -126.79M | 329.59M |
Balance Sheet | ||||||
| Total Assets | 985.10M | 824.91M | 556.54M | 831.69M | 704.96M | 809.18M |
| Cash, Cash Equivalents and Short-Term Investments | 810.34M | 622.54M | 367.52M | 617.89M | 352.84M | 556.26M |
| Total Debt | 62.83M | 536.59M | 66.06M | 138.41M | 142.89M | 135.72M |
| Total Liabilities | 661.05M | 626.01M | 563.29M | 624.02M | 228.96M | 213.40M |
| Stockholders Equity | 324.05M | 198.90M | -6.75M | 207.67M | 476.01M | 595.78M |
Cash Flow | ||||||
| Free Cash Flow | -161.01M | -177.96M | -186.10M | -153.08M | -162.75M | 270.52M |
| Operating Cash Flow | -160.79M | -177.96M | -182.73M | -145.93M | -145.06M | 287.96M |
| Investing Cash Flow | -278.57M | -321.62M | 162.97M | -205.69M | -182.73M | -67.39M |
| Financing Cash Flow | 578.33M | 415.40M | -59.49M | 362.72M | 1.45M | 94.86M |
uniQure Technical Analysis
Negative
51.05
Price Trends
35.04
Negative
38.44
Negative
29.64
Negative
Market Momentum
-2.79
Negative
44.01
Neutral
92.61
Negative
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 51.05 is above the 20-day moving average (MA) of 22.77, above the 50-day MA of 35.04, and above the 200-day MA of 29.64, indicating a neutral trend. The MACD of -2.79 indicates Negative momentum. The RSI at 44.01 is Neutral, neither overbought nor oversold. The STOCH value of 92.61 is Negative, not indicating any strong overbought or oversold conditions. Overall, these indicators collectively point to a Negative sentiment for QURE.
uniQure Risk Analysis
uniQure disclosed 70 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 |
|---|---|---|---|---|---|---|---|
61 Neutral | $3.60B | -20.34 | -52.92% | 5.64% | ― | 42.67% | |
58 Neutral | $2.18B | -5.92 | 101.55% | ― | 68.72% | -737.08% | |
58 Neutral | $2.35B | -11.11 | -61.24% | ― | ― | 9.42% | |
55 Neutral | $2.22B | -5.31 | -35.67% | ― | 140.58% | -161.11% | |
53 Neutral | $1.75B | -6.26 | -111.96% | ― | 30.24% | -3.25% | |
51 Neutral | $7.86B | -0.30 | -43.30% | 2.27% | 22.53% | -2.21% | |
47 Neutral | $2.16B | -7.20 | -50.56% | ― | -100.00% | 18.12% |
* Healthcare Sector Average
QURE
uniQure
24.83
-42.86
-63.32%
BCRX
BioCryst
8.47
1.15
15.71%
AGIO
Agios Pharma
30.55
-12.69
-29.35%
IMNM
Immunome
18.21
2.14
13.32%
PHVS
Pharvaris
33.53
11.31
50.90%
AMLX
Amylyx Pharmaceuticals Inc
28.86
14.93
107.18%
uniQure Corporate Events
Business Operations and StrategyProduct-Related AnnouncementsRegulatory Filings and Compliance
uniQure Highlights Long-Term AMT-130 Huntington’s Data
Positive
Sep 29, 2026
On September 29, 2026, uniQure reported updated Phase I/II data for its Huntington’s disease gene therapy AMT-130, showing the high-dose cohort maintained meaningful slowing of disease progression and a generally favorable tolerability profi...
Business Operations and StrategyProduct-Related AnnouncementsRegulatory Filings and Compliance
uniQure seeks accelerated approval for Huntington’s gene therapy
Positive
Sep 2, 2026
On September 2, 2026, uniQure submitted a Biologics License Application to the U.S. Food and Drug Administration seeking accelerated approval of its investigational gene therapy ifezuntirgene inilparvovec (AMT-130) for Huntington’s disease. ...
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.