Tearsheet

Investment Highlights Why It Matters Detailed financial logic regarding cash flow yields vs trend-riding momentum.


0 Cash is significant % of market cap
Net D/ENet Debt/Equity. Debt net of cash. Negative indicates net cash. Equity is taken as the Market Capitalization is -44%
Weak multi-year price returns
3Y Excs Rtn is -75%
Not profitable at operating income level
Op Inc LTMOperating Income, Last Twelve Months is -174 Mil, Op Mgn LTMOperating Margin = Operating Income / Revenue Reflects profitability before taxes and before impact of capital structure (interest payments). is -1102%
1 Megatrend and thematic drivers
Megatrends include Biotechnology & Genomics, and Precision Medicine. Themes include Gene Editing & Therapy, Targeted Therapies, Show more.
Meaningful short interest
Short Interest % of Basic SharesShort Interest % of Basic Shares = (Short Interest Quantity) / (Basic Shares Outstanding). A high fraction of short interest can indicate potential risk of a short squeeze. is 14%
Weak revenue growth
Rev Chg LTMRevenue Change % Last Twelve Months (LTM) is -45%, Rev Chg 3Y AvgRevenue Change % averaged over trailing 3 years is -12%
2   Significant share based compensation
SBC/Rev LTMShare Based Compensation / Revenue (Sales), Last Twelve Months (LTM) is 113%
3   Not cash flow generative
CFO/Rev LTMCash Flow from Operations / Revenue (Sales), Last Twelve Months (LTM) is -973%, FCF/Rev LTMFree Cash Flow / Revenue (Sales), Last Twelve Months (LTM) is -976%
4   Valuation getting more expensive
P/S 6M Chg %Price/Sales change over 6 months. Declining P/S indicates valuation has become less expensive. is 69%
5   Yield minus risk free rate is negative
ERPEquity Risk Premium (ERP) = Total Yield - Risk Free Rate, Reflects the premium above risk free assets offered by the investment. is -21%
6   High stock price volatility
Vol 12M is 269%
7   Key risks
QURE key risks include [1] an uncertain regulatory pathway for its lead Huntington's disease therapy, Show more.
0 Cash is significant % of market cap
Net D/ENet Debt/Equity. Debt net of cash. Negative indicates net cash. Equity is taken as the Market Capitalization is -44%
1 Megatrend and thematic drivers
Megatrends include Biotechnology & Genomics, and Precision Medicine. Themes include Gene Editing & Therapy, Targeted Therapies, Show more.
2 Weak multi-year price returns
3Y Excs Rtn is -75%
3 Meaningful short interest
Short Interest % of Basic SharesShort Interest % of Basic Shares = (Short Interest Quantity) / (Basic Shares Outstanding). A high fraction of short interest can indicate potential risk of a short squeeze. is 14%
4 Not profitable at operating income level
Op Inc LTMOperating Income, Last Twelve Months is -174 Mil, Op Mgn LTMOperating Margin = Operating Income / Revenue Reflects profitability before taxes and before impact of capital structure (interest payments). is -1102%
5 Weak revenue growth
Rev Chg LTMRevenue Change % Last Twelve Months (LTM) is -45%, Rev Chg 3Y AvgRevenue Change % averaged over trailing 3 years is -12%
6 Significant share based compensation
SBC/Rev LTMShare Based Compensation / Revenue (Sales), Last Twelve Months (LTM) is 113%
7 Not cash flow generative
CFO/Rev LTMCash Flow from Operations / Revenue (Sales), Last Twelve Months (LTM) is -973%, FCF/Rev LTMFree Cash Flow / Revenue (Sales), Last Twelve Months (LTM) is -976%
8 Valuation getting more expensive
P/S 6M Chg %Price/Sales change over 6 months. Declining P/S indicates valuation has become less expensive. is 69%
9 Yield minus risk free rate is negative
ERPEquity Risk Premium (ERP) = Total Yield - Risk Free Rate, Reflects the premium above risk free assets offered by the investment. is -21%
10 High stock price volatility
Vol 12M is 269%
11 Key risks
QURE key risks include [1] an uncertain regulatory pathway for its lead Huntington's disease therapy, Show more.

Valuation, Metrics & Events

QURE Stock


Why The Stock Moved


Qualitative Assessment

AI Analysis | Feedback

Here are the key points for why uniQure's stock moved by 48.1% during the approximate time period from August 31, 2025, to today:

1. Positive Topline Data for AMT-130 in Huntington's Disease: In September 2025, uniQure announced positive topline three-year data from the pivotal Phase I/II study of AMT-130, its gene therapy for Huntington's disease. The data demonstrated a statistically significant slowing of disease progression at 36 months, meeting both primary and key secondary endpoints.

2. FDA Regulatory Setback for AMT-130: Following a pre-Biologics License Application (BLA) meeting in October 2025, uniQure received preliminary feedback from the U.S. Food and Drug Administration (FDA). The FDA indicated that the Phase I/II data, which relied on an external control, might not be sufficient as the primary evidence to support a BLA submission, introducing uncertainty regarding the timing of the submission.

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Stock Movement Drivers

Fundamental Drivers

The 77.0% change in QURE stock from 9/23/2025 to 12/23/2025 was primarily driven by a 72.0% change in the company's P/S Multiple.
923202512232025Change
Stock Price ($)13.6624.1877.01%
Change Contribution ByLTMLTM
Total Revenues ($ Mil)14.3415.759.86%
P/S Multiple52.2289.8372.02%
Shares Outstanding (Mil)54.8158.52-6.77%
Cumulative Contribution76.20%

LTM = Last Twelve Months as of date shown

Market Drivers

9/23/2025 to 12/23/2025
ReturnCorrelation
QURE77.0% 
Market (SPY)3.7%-4.6%
Sector (XLV)13.2%-6.8%

Fundamental Drivers

The 70.0% change in QURE stock from 6/24/2025 to 12/23/2025 was primarily driven by a 140.3% change in the company's P/S Multiple.
624202512232025Change
Stock Price ($)14.2224.1870.04%
Change Contribution ByLTMLTM
Total Revenues ($ Mil)20.2015.75-22.03%
P/S Multiple37.3989.83140.28%
Shares Outstanding (Mil)53.1158.52-10.18%
Cumulative Contribution68.28%

LTM = Last Twelve Months as of date shown

Market Drivers

6/24/2025 to 12/23/2025
ReturnCorrelation
QURE70.0% 
Market (SPY)13.7%-3.6%
Sector (XLV)16.4%-1.8%

Fundamental Drivers

The 38.7% change in QURE stock from 12/23/2024 to 12/23/2025 was primarily driven by a 202.4% change in the company's P/S Multiple.
1223202412232025Change
Stock Price ($)17.4324.1838.73%
Change Contribution ByLTMLTM
Total Revenues ($ Mil)28.5915.75-44.90%
P/S Multiple29.7089.83202.41%
Shares Outstanding (Mil)48.7258.52-20.11%
Cumulative Contribution33.12%

LTM = Last Twelve Months as of date shown

Market Drivers

12/23/2024 to 12/23/2025
ReturnCorrelation
QURE38.7% 
Market (SPY)16.7%7.3%
Sector (XLV)13.2%4.3%

Fundamental Drivers

The 3.8% change in QURE stock from 12/24/2022 to 12/23/2025 was primarily driven by a 406.6% change in the company's P/S Multiple.
1224202212232025Change
Stock Price ($)23.2924.183.82%
Change Contribution ByLTMLTM
Total Revenues ($ Mil)61.4315.75-74.36%
P/S Multiple17.7389.83406.57%
Shares Outstanding (Mil)46.7758.52-25.11%
Cumulative Contribution-2.72%

LTM = Last Twelve Months as of date shown

Market Drivers

12/24/2023 to 12/23/2025
ReturnCorrelation
QURE282.6% 
Market (SPY)48.4%9.1%
Sector (XLV)18.2%6.4%

Return vs. Risk


Price Returns Compared

 202020212022202320242025Total [1]
Returns
QURE Return-50%-43%9%-70%161%41%-65%
Peers Return10%-26%17%4%-19%30%5%
S&P 500 Return16%27%-19%24%23%17%114%

Monthly Win Rates [3]
QURE Win Rate33%25%33%8%50%33% 
Peers Win Rate48%43%62%48%42%58% 
S&P 500 Win Rate58%75%42%67%75%73% 

Max Drawdowns [4]
QURE Max Drawdown-51%-44%-37%-75%-44%-53% 
Peers Max Drawdown-28%-39%-18%-26%-27%-36% 
S&P 500 Max Drawdown-31%-1%-25%-1%-2%-15% 


[1] Cumulative total returns since the beginning of 2020
[2] Peers: BMRN, SRPT, IONS, VRTX, RGNX. See QURE Returns vs. Peers.
[3] Win Rate = % of calendar months in which monthly returns were positive
[4] Max drawdown represents maximum peak-to-trough decline within a year
[5] 2025 data is for the year up to 12/23/2025 (YTD)

How Low Can It Go

Unique KeyEventQURES&P 500
2022 Inflation Shock2022 Inflation Shock  
2022 Inflation Shock% Loss% Loss-86.6%-25.4%
2022 Inflation Shock% Gain to Breakeven% Gain to Breakeven646.5%34.1%
2022 Inflation ShockTime to BreakevenTime to Breakeven698 days464 days
2020 Covid Pandemic2020 Covid Pandemic  
2020 Covid Pandemic% Loss% Loss-52.2%-33.9%
2020 Covid Pandemic% Gain to Breakeven% Gain to Breakeven109.3%51.3%
2020 Covid PandemicTime to BreakevenTime to BreakevenNot Fully Recovered days148 days
2018 Correction2018 Correction  
2018 Correction% Loss% Loss-54.8%-19.8%
2018 Correction% Gain to Breakeven% Gain to Breakeven121.4%24.7%
2018 CorrectionTime to BreakevenTime to BreakevenNot Fully Recovered days120 days

Compare to ACT, ALKS, LIVN, PRGO, PCRX

In The Past

uniQure's stock fell -86.6% during the 2022 Inflation Shock from a high on 1/7/2021. A -86.6% loss requires a 646.5% gain to breakeven.

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About uniQure (QURE)

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 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.

AI Analysis | Feedback

Here are 1-3 brief analogies for uniQure (QURE):

  • Like Moderna, but focused on developing one-time gene therapies for genetic diseases rather than mRNA vaccines.
  • Like Vertex Pharmaceuticals, but specifically using gene therapy to provide potentially curative treatments for genetic diseases like hemophilia.

AI Analysis | Feedback

  • HEMGENIX® (etranacogene dezaparvovec): A one-time gene therapy approved for the treatment of adults with severe to moderately severe hemophilia B.
  • AMT-130: An investigational gene therapy currently in clinical trials for the treatment of Huntington's disease.

AI Analysis | Feedback

uniQure (QURE) is a biotechnology company focused on gene therapies. Its business model primarily involves the development and licensing of its therapeutic products to other pharmaceutical companies for commercialization.

Therefore, uniQure's major customer is another company:

  • CSL Behring (a subsidiary of CSL Limited, ASX: CSL)

CSL Behring is uniQure's partner for HEMGENIX® (etranacogene dezaparvovec), a gene therapy for severe hemophilia B. uniQure licensed the global commercialization rights for HEMGENIX to CSL Behring, and its revenue from this product largely consists of milestone payments and royalties received from CSL Behring.

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  • Lonza Group AG (SIX: LONN)

AI Analysis | Feedback

Matthew Kapusta, Chief Executive Officer

Mr. Kapusta has served as uniQure's Chief Executive Officer since December 2016 and is also a member of the company's Board of Directors. He initially joined uniQure as Chief Financial Officer in January 2015. Prior to uniQure, he was Senior Vice President at AngioDynamics from 2011 to 2014, where he was responsible for corporate development, strategic planning, and national accounts. Before AngioDynamics, he served as Vice President, Finance for Smith & Nephew Orthopaedics. Mr. Kapusta's career also includes over a decade of investment banking experience focused on emerging life sciences companies, having held positions at Collins Stewart as Managing Director, Healthcare Investment Banking, and at Wells Fargo Securities, Robertson Stephens, and PaineWebber. He has served as a director of Decibel Therapeutics (Nasdaq: DBTX) since March 2023.

Christian Klemt, Chief Financial Officer

Mr. Klemt was appointed Chief Financial Officer of uniQure in June 2021. He has been with uniQure since 2015 and previously served as Chief Accounting Officer starting in August 2017 and as General Manager of the Amsterdam site in 2020. During his tenure, he has transformed the finance and accounting functions, established financial planning and analysis, led tax and treasury initiatives, and overseen IT development. Before joining uniQure, Mr. Klemt was Finance Director at CGGVeritas from 2009 to 2015. He also served as Group Finance Manager of Basell Polyolefins N.V. from 2005 to 2007 and played a role in acquisition projects following its merger with Lyondell Inc. in 2007. Mr. Klemt qualified as a German Certified Public Accountant and Tax Advisor while working at KPMG from 1999 to 2005.

Walid Abi-Saab, M.D., Chief Medical Officer

Dr. Abi-Saab joined uniQure as Chief Medical Officer in June 2023, overseeing clinical research and development, regulatory affairs, and program management. Prior to uniQure, he was Chief Medical Officer at Galapagos from 2017 to 2022, where he expanded the development organization and led successful Phase 3 studies for filgotinib. He has also held leadership positions at Shire (now part of Takeda Pharmaceutical Co.), Novartis, Abbott Laboratories, and Pfizer. Dr. Abi-Saab also served as a resident, research fellow, and assistant professor at Yale University School of Medicine.

Richard Porter, Ph.D., Chief Business and Scientific Officer

Dr. Porter was named Chief Business and Scientific Officer in October 2023. In this role, he is responsible for research, non-clinical and vector development, business development, and product planning. Dr. Porter joined uniQure as General Manager of the Corlieve subsidiary following uniQure's acquisition of Corlieve Therapeutics in July 2021. He was the founder and CEO of Corlieve Therapeutics, which was founded in November 2019. He previously served as Chief Operating Officer of Therachon until its acquisition by Pfizer. Dr. Porter's experience also includes roles at Roche as Global Head of Scientific Business Strategy and Operations for Neuroscience Ophthalmology and Rare Diseases, and as Product General Manager of the Emerging Business Unit at Shire Pharmaceuticals.

Jeannette Potts, Ph.D., J.D., Chief Legal & Compliance Officer

Dr. Potts joined uniQure as Chief Legal and Compliance Officer in May 2023. She brings over 25 years of global legal, management, and transactional experience in the biopharmaceutical industry. Most recently, she was Senior Vice President, General Counsel & Corporate Secretary at Forma Therapeutics, which was acquired by Novo Nordisk in 2022. At Forma, she built and led the legal, IP, and compliance team and played key roles in corporate financings and business development transactions. Before Forma, Dr. Potts served as Vice President, Legal and Head Counsel, Research and Development, at Takeda Pharmaceuticals, and prior to that, Vice President, Legal at Millennium Pharmaceuticals before its acquisition by Takeda.

AI Analysis | Feedback

The key risks to uniQure's business include:
  1. Regulatory Uncertainty for AMT-130: The most significant risk stems from the regulatory pathway for AMT-130, its investigational gene therapy for Huntington's disease. The U.S. Food and Drug Administration (FDA) recently indicated that current Phase I/II study data for AMT-130 may not be sufficient to support a Biologics License Application (BLA) submission for accelerated approval, creating uncertainty around the timing of a potential submission and approval. This regulatory setback has led to a substantial decline in uniQure's stock price and is considered a critical "binary event" given the importance of AMT-130 to the company's future.
  2. Financial Health and Funding: uniQure continues to face significant financial challenges, reporting deepening losses, high research and development (R&D) costs, and negative operating and net margins. Despite recent financing activities that have improved its cash position, the company's Altman Z-Score indicates financial distress and a potential risk of bankruptcy within two years. The ability to fund ongoing operations and potentially raise additional capital if regulatory timelines are delayed is a persistent concern.
  3. Intense Competition and Market Volatility: The biotechnology sector, particularly gene therapy, is characterized by rapid technological advancements, intense competition, and high market volatility. uniQure operates in a highly dynamic environment where new players and innovations can quickly alter market dynamics, requiring continuous innovation to maintain a competitive edge. The company's stock price has experienced significant fluctuations, which can impact investor confidence and its ability to raise capital in the future.

AI Analysis | Feedback

The most clear emerging threat to uniQure (QURE) is the imminent market entry of **Pfizer's gene therapy for hemophilia B, fidanacogene elaparvovec (SPK-9001/PF-06838435)**.

uniQure's Hemgenix is currently the only FDA-approved gene therapy for hemophilia B. However, Pfizer's fidanacogene elaparvovec is a direct competitor, also an AAV-based gene therapy delivering the factor IX gene. Pfizer announced positive Phase 3 results (BENEGENE-2 study) in 2022 and, in December 2023, confirmed that the FDA had accepted its Biologics License Application (BLA) for the investigational gene therapy. This positions Pfizer's product to potentially launch in the market in the near future, directly challenging uniQure's first-mover advantage and market share in the hemophilia B gene therapy space.

AI Analysis | Feedback

uniQure's main products target Hemophilia B and Huntington's disease.

HEMGENIX (etranacogene dezaparvovec) for Hemophilia B

The addressable market for Hemophilia B gene therapy is substantial. The global Hemophilia B gene therapy market size was valued at approximately USD 0.51 billion in 2023 and is projected to reach USD 3.43 billion by 2032, with a compound annual growth rate (CAGR) of around 23.60% from 2024 to 2032. Another estimate for the global Hemophilia B gene therapy market projects a valuation of USD 5.94 billion by 2032, with a CAGR of 34.1% from 2023. The broader Hemophilia B treatment market across the 7 Major Markets (7MM), which includes the United States, Germany, Spain, Italy, France, the United Kingdom, and Japan, was approximately USD 3.4 billion in 2023, with the United States being the largest contributor. The global Hemophilia B treatment market is estimated to reach a valuation of approximately USD 4.29 billion in 2024 and is expected to grow at a CAGR of 7.6% during the forecast period of 2024 to 2034.

AMT-130 for Huntington's Disease

The addressable market for Huntington's disease treatments is also significant. The global Huntington's disease treatment market size was estimated at USD 500 million in 2024 and is projected to reach USD 1.87 billion by 2030, growing at a CAGR of 23.8% from 2025 to 2030. Another source states the global market size was USD 578.9 million in 2024 and is estimated to reach USD 2.44 billion by 2033, exhibiting a CAGR of 17.4% from 2025 to 2033. North America currently holds the largest share of this market, accounting for approximately 40.0% in 2024. The Huntington's disease treatment market size was valued at USD 658.31 million in 2025 and is likely to surpass USD 1.69 billion by 2035, growing at a CAGR of more than 9.9% between 2026 and 2035.

Other Products

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AI Analysis | Feedback

uniQure (QURE) is expected to drive future revenue growth over the next 2-3 years through several key initiatives:

  1. Commercial Launch and Sales of AMT-130 for Huntington's Disease: The most significant anticipated driver is the potential commercial launch of AMT-130, a gene therapy for Huntington's disease (HD). uniQure plans to submit a Biologics License Application (BLA) to the FDA in the first quarter of 2026, following positive top-line data from its Phase 1/2 study, which demonstrated a statistically significant slowing of disease progression. Analysts project peak revenues for AMT-130 could reach approximately $3 billion, positioning it as a potentially first-in-class disease-modifying therapy for HD, addressing a high unmet medical need.
  2. Advancement and Potential Commercialization of Other Pipeline Assets: uniQure is progressing a focused pipeline of innovative gene therapies beyond Huntington's disease. Key programs include AMT-260 for refractory mesial temporal lobe epilepsy, which has shown encouraging early clinical signals, and AMT-191 for Fabry disease, for which initial safety and efficacy data are expected. Further clinical development, potential regulatory approvals, and eventual commercialization of these and other early-stage candidates like AMT-162 for SOD1-ALS and AMT-240 for autosomal dominant Alzheimer's disease would contribute to future revenue diversification.
  3. Milestone Payments from Hemgenix: Although uniQure previously sold a portion of its royalty rights from Hemgenix (etranacogene dezaparvovec-drbl) net sales for hemophilia B, the company retains eligibility for substantial contractual milestone payments from CSL Behring, the licensee. These milestones, potentially totaling up to $1.5 billion, are contingent on the ongoing commercial and regulatory progress of Hemgenix in various territories.

AI Analysis | Feedback

Share Issuance

  • In September 2025, uniQure completed an upsized public offering, generating approximately $345 million in gross proceeds from the sale of 6,736,841 ordinary shares and pre-funded warrants for 526,316 shares.
  • In September 2025, the company announced the pricing of a $300 million public offering, which included 5,789,473 ordinary shares and pre-funded warrants for 526,316 shares.
  • During January and February 2025, uniQure raised $75 million through a public offering of 4.4 million shares at $17 per share, and an additional $10.6 million in net proceeds from underwriters' option exercise for 0.7 million shares.

Inbound Investments

  • In September 2025, uniQure secured a $175 million non-dilutive senior secured term loan facility from Hercules Capital, Inc., which included refinancing an existing $50 million debt and providing access to an additional $125 million.
  • The additional $125 million in non-dilutive funding consists of a $100 million tranche contingent on regulatory and financial milestones for AMT-130, and a $25 million tranche subject to Hercules' approval.
  • As of November 10, 2025, the liability from the HEMGENIX royalty financing stood at $469.1 million.

Capital Expenditures

  • For the trailing twelve months ended June 2025, uniQure reported capital expenditures of approximately $-9.13 million.
  • For the three months ended June 2025, capital expenditures were approximately $-0.26 million.
  • These expenditures are primarily focused on acquiring or upgrading physical assets like property, industrial buildings, or equipment.

Better Bets than uniQure (QURE)

Trade Ideas

Select ideas related to QURE. For more, see Trefis Trade Ideas.

Unique KeyDateTickerCompanyCategoryTrade Strategy6M Fwd Rtn12M Fwd Rtn12M Max DD
CRL_11142025_Dip_Buyer_FCFYield11142025CRLCharles River Laboratories InternationalDip BuyDB | FCFY OPMDip Buy with High FCF Yield and High Margin
Buying dips for companies with high FCF yield and meaningfully high operating margin
20.9%20.9%-3.7%
GDRX_11142025_Dip_Buyer_High_CFO_Margins_ExInd_DE11142025GDRXGoodRxDip BuyDB | CFO/Rev | Low D/EDip Buy with High Cash Flow Margins
Buying dips for companies with significant cash flows from operations and reasonable debt / market cap
-7.4%-7.4%-11.8%
ASTH_11142025_Dip_Buyer_High_FCF_Yield_ExInd_DE_RevG11142025ASTHAstrana HealthDip BuyDB | FCF Yield | Low D/EDip Buy with High Free Cash Flow Yield
Buying dips for companies with significant free cash flow yield (FCF / Market Cap) and reasonable debt / market cap
13.5%13.5%-5.5%
SGRY_11142025_Dip_Buyer_High_FCF_Yield_ExInd_DE_RevG11142025SGRYSurgery PartnersDip BuyDB | FCF Yield | Low D/EDip Buy with High Free Cash Flow Yield
Buying dips for companies with significant free cash flow yield (FCF / Market Cap) and reasonable debt / market cap
1.6%1.6%-1.4%
TFX_11072025_Dip_Buyer_FCFYield11072025TFXTeleflexDip BuyDB | FCFY OPMDip Buy with High FCF Yield and High Margin
Buying dips for companies with high FCF yield and meaningfully high operating margin
11.8%11.8%-5.1%

Recent Active Movers

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Peer Comparisons for uniQure

Peers to compare with:

Financials

QUREBMRNSRPTIONSVRTXRGNXMedian
NameuniQure BioMarin.Sarepta .Ionis Ph.Vertex P.Regenxbio 
Mkt Price24.1861.1422.0080.33459.2114.3542.66
Mkt Cap1.411.72.212.8117.40.77.0
Rev LTM163,0942,41496711,7231611,690
Op Inc LTM-174615-86-278-92-155-123
FCF LTM-154826-389-3093,337-106-130
FCF 3Y Avg-169386-495-4202,064-174-172
CFO LTM-153914-245-2473,718-103-128
CFO 3Y Avg-161489-379-3802,419-168-164

Growth & Margins

QUREBMRNSRPTIONSVRTXRGNXMedian
NameuniQure BioMarin.Sarepta .Ionis Ph.Vertex P.Regenxbio 
Rev Chg LTM-44.9%12.4%47.1%20.4%10.3%91.3%16.4%
Rev Chg 3Y Avg-12.4%15.5%40.6%7.1%10.5%-1.0%8.8%
Rev Chg Q61.8%4.1%-14.5%17.1%11.0%22.9%14.1%
QoQ Delta Rev Chg LTM9.9%1.0%-2.7%2.4%2.7%3.6%2.5%
Op Mgn LTM-1,102.1%19.9%-3.6%-28.8%-0.8%-96.0%-16.2%
Op Mgn 3Y Avg-658.9%13.5%-11.6%-55.1%26.2%-216.3%-33.3%
QoQ Delta Op Mgn LTM42.1%-5.4%-3.5%-0.5%1.0%9.6%0.2%
CFO/Rev LTM-972.9%29.5%-10.1%-25.5%31.7%-64.0%-17.8%
CFO/Rev 3Y Avg-578.5%16.9%-26.8%-52.4%23.1%-166.2%-39.6%
FCF/Rev LTM-975.7%26.7%-16.1%-32.0%28.5%-65.9%-24.1%
FCF/Rev 3Y Avg-598.4%13.0%-33.7%-57.4%19.8%-172.6%-45.6%

Valuation

QUREBMRNSRPTIONSVRTXRGNXMedian
NameuniQure BioMarin.Sarepta .Ionis Ph.Vertex P.Regenxbio 
Mkt Cap1.411.72.212.8117.40.77.0
P/S89.83.80.913.310.04.67.3
P/EBIT-8.717.0-9.6-73.726.3-5.5-7.1
P/E-6.022.6-8.1-50.131.9-4.2-5.1
P/CFO-9.212.8-9.0-52.031.6-7.2-8.1
Total Yield-16.6%4.4%-12.3%-2.0%3.1%-24.0%-7.2%
Dividend Yield0.0%0.0%0.0%0.0%0.0%0.0%0.0%
FCF Yield 3Y Avg-45.1%3.5%-10.0%-6.0%2.3%-29.3%-8.0%
D/E0.00.10.60.10.00.10.1
Net D/E-0.4-0.10.2-0.1-0.0-0.3-0.1

Returns

QUREBMRNSRPTIONSVRTXRGNXMedian
NameuniQure BioMarin.Sarepta .Ionis Ph.Vertex P.Regenxbio 
1M Rtn-13.1%11.8%24.4%5.6%7.6%21.6%9.7%
3M Rtn77.0%14.5%22.7%28.4%20.9%60.7%25.5%
6M Rtn70.0%11.8%15.9%102.6%2.0%67.6%41.8%
12M Rtn38.7%-7.7%-81.8%122.6%13.3%94.4%26.0%
3Y Rtn3.8%-41.4%-83.1%110.5%58.5%-35.5%-15.8%
1M Excs Rtn-17.7%7.1%19.7%0.9%3.0%17.0%5.1%
3M Excs Rtn-53.2%9.6%16.4%21.5%17.8%46.3%17.1%
6M Excs Rtn55.3%-0.5%12.6%87.4%-10.4%54.6%33.6%
12M Excs Rtn38.5%-23.7%-99.4%107.9%-2.0%75.9%18.3%
3Y Excs Rtn-74.7%-123.1%-161.6%28.4%-28.7%-117.6%-96.2%

Financials

Segment Financials

Revenue by Segment
$ Mil20242023202220212020
Discovery and development of innovative gene therapies16    
Collaboration revenues 5702
Contract manufacturing revenues 2   
License revenues 100517375
Total16106524387


Price Behavior

Price Behavior
Market Price$24.18 
Market Cap ($ Bil)1.3 
First Trading Date02/05/2014 
Distance from 52W High-65.7% 
   50 Days200 Days
DMA Price$36.50$22.71
DMA Trendupdown
Distance from DMA-33.7%6.5%
 3M1YR
Volatility510.2%270.7%
Downside Capture-762.36-0.47
Upside Capture-335.5731.90
Correlation (SPY)-4.1%7.4%
QURE Betas & Captures as of 11/30/2025

 1M2M3M6M1Y3Y
Beta-0.820.87-2.16-1.031.011.26
Up Beta4.615.074.182.401.131.07
Down Beta-0.381.209.534.143.312.14
Up Capture-691%-264%-196%-62%95%132%
Bmk +ve Days13263974142427
Stock +ve Days6172759119336
Down Capture110%118%-1499%-772%-124%106%
Bmk -ve Days7162452107323
Stock -ve Days14253666128402

[1] Upside and downside betas calculated using positive and negative benchmark daily returns respectively
Based On 1-Year Data
 Comparison of QURE With Other Asset Classes (Last 1Y)
 QURESector ETFEquityGoldCommoditiesReal EstateBitcoin
Annualized Return53.4%14.9%18.8%72.9%9.0%3.7%-11.4%
Annualized Volatility269.2%17.3%19.5%19.2%15.3%17.2%35.0%
Sharpe Ratio0.830.640.762.720.360.05-0.14
Correlation With Other Assets 4.6%7.5%-8.8%5.6%2.3%10.6%

ETFs used for asset classes: Sector ETF = XLV, Equity = SPY, Gold = GLD, Commodities = DBC, Real Estate = VNQ, and Bitcoin = BTCUSD
Smart multi-asset allocation framework can stack odds in your favor. Learn How
Based On 5-Year Data
 Comparison of QURE With Other Asset Classes (Last 5Y)
 QURESector ETFEquityGoldCommoditiesReal EstateBitcoin
Annualized Return-12.8%8.4%14.8%18.9%11.8%4.7%35.5%
Annualized Volatility149.6%14.5%17.1%15.5%18.7%18.9%48.9%
Sharpe Ratio0.350.400.700.980.510.160.62
Correlation With Other Assets 11.5%15.6%-0.7%1.3%9.7%10.1%

ETFs used for asset classes: Sector ETF = XLV, Equity = SPY, Gold = GLD, Commodities = DBC, Real Estate = VNQ, and Bitcoin = BTCUSD
Smart multi-asset allocation framework can stack odds in your favor. Learn How
Based On 10-Year Data
 Comparison of QURE With Other Asset Classes (Last 10Y)
 QURESector ETFEquityGoldCommoditiesReal EstateBitcoin
Annualized Return3.2%9.9%14.8%15.1%6.8%5.4%69.1%
Annualized Volatility117.6%16.6%18.0%14.8%17.6%20.8%55.8%
Sharpe Ratio0.410.490.710.850.310.230.90
Correlation With Other Assets 16.9%18.1%-0.3%3.9%10.7%9.0%

ETFs used for asset classes: Sector ETF = XLV, Equity = SPY, Gold = GLD, Commodities = DBC, Real Estate = VNQ, and Bitcoin = BTCUSD
Smart multi-asset allocation framework can stack odds in your favor. Learn How

Short Interest

Short Interest: As Of Date11282025
Short Interest: Shares Quantity8,335,603
Short Interest: % Change Since 11152025-0.7%
Average Daily Volume1,907,330
Days-to-Cover Short Interest4.37
Basic Shares Quantity58,516,415
Short % of Basic Shares14.2%

Earnings Returns History

Expand for More
 Forward Returns
Earnings Date1D Returns5D Returns21D Returns
11/10/2025-5.9%7.5%-27.5%
7/29/2025-8.7%-8.1%5.4%
5/9/20254.4%8.2%38.0%
2/27/20253.4%3.6%12.5%
11/5/20249.0%37.8%13.1%
8/1/2024-2.3%-7.0%-18.8%
2/28/2024-0.9%-25.3%-19.9%
11/7/202312.7%-1.8%15.4%
...
SUMMARY STATS   
# Positive12810
# Negative91311
Median Positive3.5%7.7%13.2%
Median Negative-3.3%-8.1%-17.8%
Max Positive12.7%37.8%38.0%
Max Negative-27.0%-25.3%-27.5%

SEC Filings

Expand for More
Report DateFiling DateFiling
93020251110202510-Q 9/30/2025
6302025729202510-Q 6/30/2025
3312025509202510-Q 3/31/2025
12312024227202510-K 12/31/2024
93020241105202410-Q 9/30/2024
6302024801202410-Q 6/30/2024
3312024507202410-Q 3/31/2024
12312023228202410-K 12/31/2023
93020231107202310-Q 9/30/2023
6302023801202310-Q 6/30/2023
3312023509202310-Q 3/31/2023
12312022227202310-K 12/31/2022
93020221102202210-Q 9/30/2022
6302022808202210-Q 6/30/2022
3312022502202210-Q 3/31/2022
12312021225202210-K 12/31/2021