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 -58%

Valuation becoming less expensive
P/S 6M Chg %Price/Sales change over 6 months. Declining P/S indicates valuation has become less expensive. is -73%

Megatrend and thematic drivers
Megatrends include Biotechnology & Genomics, and Precision Medicine. Themes include Gene Editing & Therapy, Targeted Therapies, Show more.

Weak multi-year price returns
3Y Excs Rtn is -89%

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 18%

Not profitable at operating income level
Op Inc LTMOperating Income, Last Twelve Months is -181 Mil, Op Mgn LTMOperating Margin = Operating Income / Revenue Reflects profitability before taxes and before impact of capital structure (interest payments). is -1126%

Weak revenue growth
Rev Chg LTMRevenue Change % Last Twelve Months (LTM) is -41%, Rev Chg 3Y AvgRevenue Change % averaged over trailing 3 years is -18%

Significant share based compensation
SBC/Rev LTMShare Based Compensation / Revenue (Sales), Last Twelve Months (LTM) is 110%

Not cash flow generative
CFO/Rev LTMCash Flow from Operations / Revenue (Sales), Last Twelve Months (LTM) is -1105%, FCF/Rev LTMFree Cash Flow / Revenue (Sales), Last Twelve Months (LTM) is -1109%

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 -25%

High stock price volatility
Vol 12M is 276%

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 -58%
1 Valuation becoming less expensive
P/S 6M Chg %Price/Sales change over 6 months. Declining P/S indicates valuation has become less expensive. is -73%
2 Megatrend and thematic drivers
Megatrends include Biotechnology & Genomics, and Precision Medicine. Themes include Gene Editing & Therapy, Targeted Therapies, Show more.
3 Weak multi-year price returns
3Y Excs Rtn is -89%
4 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 18%
5 Not profitable at operating income level
Op Inc LTMOperating Income, Last Twelve Months is -181 Mil, Op Mgn LTMOperating Margin = Operating Income / Revenue Reflects profitability before taxes and before impact of capital structure (interest payments). is -1126%
6 Weak revenue growth
Rev Chg LTMRevenue Change % Last Twelve Months (LTM) is -41%, Rev Chg 3Y AvgRevenue Change % averaged over trailing 3 years is -18%
7 Significant share based compensation
SBC/Rev LTMShare Based Compensation / Revenue (Sales), Last Twelve Months (LTM) is 110%
8 Not cash flow generative
CFO/Rev LTMCash Flow from Operations / Revenue (Sales), Last Twelve Months (LTM) is -1105%, FCF/Rev LTMFree Cash Flow / Revenue (Sales), Last Twelve Months (LTM) is -1109%
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 -25%
10 High stock price volatility
Vol 12M is 276%
11 Key risks
QURE key risks include [1] an uncertain regulatory pathway for its lead Huntington's disease therapy, Show more.

Valuation, Metrics & Events

Price Chart

Why The Stock Moved

Qualitative Assessment

AI Analysis | Feedback

uniQure (QURE) stock has lost about 35% since 12/31/2025 because of the following key factors:

1. FDA Rejection of AMT-130 for Huntington's Disease Necessitates Lengthy Phase III Trial.

uniQure's stock plummeted over 37% following its March 2, 2026, announcement that the U.S. Food and Drug Administration (FDA) disagreed with the sufficiency of its Phase I/II data for AMT-130, a gene therapy for Huntington's disease, to support a marketing application. The FDA "strongly recommended" uniQure conduct a prospective, randomized, double-blind, sham surgery-controlled Phase III study. This regulatory setback fundamentally alters the drug's approval timeline, increasing development risk and capital requirements, and contradicts earlier company communications suggesting alignment with the FDA for a BLA submission in the first quarter of 2026.

2. Increased Financial Strain from Mandated Phase III Development.

Despite reporting an earnings per share (EPS) beat of ($0.56) against an estimated ($0.93) for Q4 2025 and holding approximately $622.5 million in cash, cash equivalents, and current investment securities as of December 31, 2025, uniQure's financial position faces new challenges. The company's revenue for the full year 2025 was $16.1 million, a decrease from $27.1 million in 2024, and it reported a net loss of $199.0 million for 2025, indicating an annual cash burn of nearly $200 million. The unexpected requirement for a new, costly, and multi-year Phase III study for AMT-130 will place considerable strain on these resources, extending the timeline to potential profitability.

Show more

Stock Movement Drivers

Fundamental Drivers

The -35.7% change in QURE stock from 12/31/2025 to 4/11/2026 was primarily driven by a -33.0% change in the company's P/S Multiple.
(LTM values as of)123120254112026Change
Stock Price ($)23.9315.38-35.7%
Change Contribution By: 
Total Revenues ($ Mil)16162.2%
P/S Multiple88.959.6-33.0%
Shares Outstanding (Mil)5962-6.2%
Cumulative Contribution-35.7%

LTM = Last Twelve Months as of date shown

Market Drivers

12/31/2025 to 4/11/2026
ReturnCorrelation
QURE-35.7% 
Market (SPY)-5.4%10.5%
Sector (XLV)-4.8%8.4%

Fundamental Drivers

The -73.7% change in QURE stock from 9/30/2025 to 4/11/2026 was primarily driven by a -73.3% change in the company's P/S Multiple.
(LTM values as of)93020254112026Change
Stock Price ($)58.3715.38-73.7%
Change Contribution By: 
Total Revenues ($ Mil)141612.3%
P/S Multiple223.159.6-73.3%
Shares Outstanding (Mil)5562-12.1%
Cumulative Contribution-73.7%

LTM = Last Twelve Months as of date shown

Market Drivers

9/30/2025 to 4/11/2026
ReturnCorrelation
QURE-73.7% 
Market (SPY)-2.9%7.9%
Sector (XLV)6.3%8.0%

Fundamental Drivers

The 45.1% change in QURE stock from 3/31/2025 to 4/11/2026 was primarily driven by a 212.0% change in the company's P/S Multiple.
(LTM values as of)33120254112026Change
Stock Price ($)10.6015.3845.1%
Change Contribution By: 
Total Revenues ($ Mil)2716-40.6%
P/S Multiple19.159.6212.0%
Shares Outstanding (Mil)4962-21.6%
Cumulative Contribution45.1%

LTM = Last Twelve Months as of date shown

Market Drivers

3/31/2025 to 4/11/2026
ReturnCorrelation
QURE45.1% 
Market (SPY)16.3%5.8%
Sector (XLV)2.3%5.1%

Fundamental Drivers

The -23.6% change in QURE stock from 3/31/2023 to 4/11/2026 was primarily driven by a -84.9% change in the company's Total Revenues ($ Mil).
(LTM values as of)33120234112026Change
Stock Price ($)20.1415.38-23.6%
Change Contribution By: 
Total Revenues ($ Mil)10616-84.9%
P/S Multiple8.959.6571.7%
Shares Outstanding (Mil)4762-24.8%
Cumulative Contribution-23.6%

LTM = Last Twelve Months as of date shown

Market Drivers

3/31/2023 to 4/11/2026
ReturnCorrelation
QURE-23.6% 
Market (SPY)63.3%9.9%
Sector (XLV)19.1%7.1%

Return vs. Risk

Price Returns Compared

 202120222023202420252026Total [1]
Returns
QURE Return-43%9%-70%161%36%-32%-55%
Peers Return-26%17%4%-19%27%-9%-15%
S&P 500 Return27%-19%24%23%16%-0%82%

Monthly Win Rates [3]
QURE Win Rate25%33%8%50%33%25% 
Peers Win Rate43%62%48%42%60%45% 
S&P 500 Win Rate75%42%67%75%67%50% 

Max Drawdowns [4]
QURE Max Drawdown-44%-37%-75%-44%-53%-62% 
Peers Max Drawdown-39%-18%-26%-27%-36%-20% 
S&P 500 Max Drawdown-1%-25%-1%-2%-15%-7% 


[1] Cumulative total returns since the beginning of 2021
[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] 2026 data is for the year up to 4/10/2026 (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 BMRN, SRPT, IONS, VRTX, RGNX

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

Analogy 1: Imagine **Moderna**, but instead of mRNA technology, uniQure focuses on developing groundbreaking gene therapies for genetic diseases.

Analogy 2: A **Vertex Pharmaceuticals** dedicated to creating gene therapies for severe genetic conditions like hemophilia and Huntington's disease.

AI Analysis | Feedback

  • Etranacogene dezaparvovec (AMT-061): A gene therapy in Phase III clinical trial for the treatment of hemophilia B.
  • AMT-130: A gene therapy in Phase I/II clinical study for the treatment of Huntington's disease.
  • AMT-060: A gene therapy 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: A product candidate for the treatment of temporal lobe epilepsy.
  • AMT-240: A preclinical product candidate for the treatment of autosomal dominant Alzheimer's disease.
  • AMT-161: A product candidate for the treatment of amyotrophic lateral sclerosis.

AI Analysis | Feedback

uniQure N.V. (QURE) is a gene therapy company primarily engaged in the research and development of treatments for genetic and other devastating diseases. As a biopharmaceutical company with most of its pipeline in clinical development stages, uniQure does not typically sell directly to individual patients. Instead, its "customers" or key partners for commercialized products are other pharmaceutical companies or healthcare systems. For its lead program, Etranacogene dezaparvovec (AMT-061), which is now marketed under the brand name HEMGENIX® for hemophilia B, uniQure has licensed the global commercialization rights. Therefore, its major customer/partner for this commercialized product is: * **CSL Behring**, a subsidiary of **CSL Limited** (ASX: CSL). CSL Behring holds the exclusive global license for the commercialization of HEMGENIX®. For its other pipeline product candidates, such as AMT-130 for Huntington's disease, AMT-210 for Parkinson's disease, and others, these are still in various stages of clinical trials or preclinical development. Consequently, uniQure currently does not have commercial customers for these investigational therapies. If these programs are successfully developed and approved, uniQure may commercialize them independently, partner with other pharmaceutical companies, or license them out, similar to the arrangement for HEMGENIX®.

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  • Bristol Myers Squibb Company (BMY)
  • Catalent, Inc. (CTLT)
  • Lonza Group Ltd (LONN)

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Matt Kapusta, Chief Executive Officer
Matt Kapusta has served as Chief Executive Officer of uniQure since December 2016 and is also an Executive Director on the Board. He previously held the role of Chief Financial Officer for uniQure from January 2015 until June 2021. Prior to joining uniQure, Mr. Kapusta held executive positions at AngioDynamics from 2011 to 2015 and Smith & Nephew from 2009 to 2011. His career also includes more than a decade of investment banking experience focused on emerging life-sciences companies, including serving as Managing Director, Healthcare Investment Banking at Collins Stewart, and various roles at Wells Fargo Securities, Robertson Stephens, and PaineWebber.

Christian Klemt, Chief Financial Officer
Christian Klemt was appointed Chief Financial Officer of uniQure in June 2021. Prior to this role, he served as uniQure's Chief Accounting Officer from August 2017 to June 2021. Mr. Klemt also continues to serve as the General Manager of uniQure's Amsterdam site.

Walid Abi-Saab, M.D., Chief Medical Officer
Walid Abi-Saab joined uniQure as Chief Medical Officer in June 2023. In this role, he is responsible for leading clinical research and development, regulatory affairs, medical affairs, and program management. Before uniQure, Dr. Abi-Saab served as Chief Medical Officer at Galapagos from 2017 through 2022, where he expanded the company's development organization and led the successful completion of Phase 3 studies for filgotinib. He has also held leadership positions at companies such as Novartis, Abbott Laboratories, Pfizer, and served as Group Vice President, Global Clinical Development at Shire.

Richard Porter, Ph.D., Chief Business and Scientific Officer
Richard Porter assumed responsibilities for research, non-clinical, and vector development in his role as Chief Business and Scientific Officer following a strategic reorganization in October 2023. He also continues to oversee business development and product planning. Dr. Porter joined uniQure in June 2021 through the acquisition of Corlieve Therapeutics, where he was the founder and Chief Executive Officer. He possesses over 25 years of neuroscience leadership experience in the biopharma industry.

Kylie O'Keefe, Chief Customer and Strategy Officer
Kylie O'Keefe was appointed Chief Customer and Strategy Officer in June 2025, where she is responsible for leading the development and execution of uniQure's global commercialization strategy for AMT-130, including all commercial functions and medical affairs. Prior to joining uniQure, Ms. O'Keefe served as Chief Commercial Officer at PTC Therapeutics, overseeing global commercial strategy, operations, and portfolio management for multiple rare neurology and metabolic commercial products across more than 50 countries. Her experience includes leading several strategically significant commercial launches and supporting corporate strategy and pipeline development for both small molecules and gene therapies.

AI Analysis | Feedback

uniQure (NASDAQ: QURE) faces several significant business risks, primarily stemming from the challenging regulatory landscape and the inherent uncertainties of gene therapy development. The most prominent risk centers on **regulatory and legal challenges for its Huntington's disease gene therapy candidate, AMT-130**. The U.S. Food and Drug Administration (FDA) has "strongly recommended" that uniQure conduct a new, costly, and lengthy Phase III sham-controlled study for AMT-130, rejecting the company's current data package for accelerated approval. This decision significantly alters the drug's path to market, increasing both development risks and capital requirements. Further exacerbating this risk, an unnamed senior FDA official has publicly criticized uniQure, accusing the company of presenting "distorted" and "manipulated" clinical data for a "failed" therapy and of misleading behavior in its interactions with federal health authorities. These accusations have led to a substantial drop in uniQure's share price and have prompted multiple law firms to file securities fraud class-action lawsuits against the company, alleging misleading statements about FDA alignment and timelines for AMT-130. Such legal actions expose uniQure to significant financial penalties and reputational damage. A second key risk arises from the **inherent complexities and uncertainties of gene therapy development**. Developing gene therapies is a costly endeavor with a high risk of clinical trial failure. While uniQure has programs in various clinical phases, there are ongoing concerns regarding long-term safety, including potential delayed adverse effects, immune responses, and unintended off-target effects. Manufacturing these advanced therapeutic medicinal products also presents significant challenges related to quality control, scalability, and standardization. Even if a therapy gains regulatory approval, commercialization hurdles such as high treatment costs and challenges in market acceptance and reimbursement pose additional risks. Finally, uniQure's **financial health and cash burn** present a significant business risk. The company has experienced a substantial revenue growth decline and a deeply negative net margin over the past three years. Although uniQure currently holds a strong cash position, its considerable annual cash burn rate, estimated at nearly $200 million, means that the newly recommended, extended Phase III trial for AMT-130 will severely test its financial resilience. The company's Altman Z-Score, which indicates financial distress, is also low, implying a potential risk of bankruptcy in the near future.

AI Analysis | Feedback

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AI Analysis | Feedback

uniQure N.V. (symbol: QURE) operates in the gene therapy space, developing treatments for genetic and other devastating diseases. The addressable markets for their main products are as follows:

Etranacogene dezaparvovec (HEMGENIX) for Hemophilia B

HEMGENIX (etranacogene dezaparvovec) is uniQure's approved gene therapy for hemophilia B. The global market for hemophilia B gene therapy was valued at approximately USD 0.51 billion in 2023 and is projected to reach USD 3.43 billion by 2032, growing at a compound annual growth rate (CAGR) of roughly 23.60% between 2024 and 2032. The broader global hemophilia B market was valued at US$ 2,956.0 million in 2024 and is estimated to grow to US$ 4,362.0 million by 2030. In the United States, the hemophilia B market size was approximately USD 2 billion in 2023.

AMT-130 for Huntington's Disease

Huntington's disease impacts approximately 75,000 people across the U.S., Europe, and the UK. The global Huntington's disease treatment market was valued at USD 1.2 billion in 2024 and is projected to reach USD 3.7 billion by 2034, exhibiting an 8.9% CAGR. Another estimate places the global market at USD 578.9 million in 2024, expected to reach USD 2,443 million by 2033 with a CAGR of 17.4% from 2025-2033. The North American market for Huntington's disease treatment held the leading regional position in 2024, with an estimated value of approximately USD 0.5 billion.

AMT-210 for Parkinson's Disease

uniQure announced in October 2023 its decision to discontinue the AMT-210 program for Parkinson's disease as part of a strategic reorganization. Therefore, there is no addressable market size for this specific product from uniQure's portfolio.

AI Analysis | Feedback

uniQure N.V. (QURE) is expected to drive future revenue growth over the next 2-3 years primarily through the commercialization of its gene therapies. The key drivers include:
  • Commercialization and Market Penetration of Hemgenix (etranacogene dezaparvovec): uniQure's approved gene therapy for hemophilia B, Hemgenix, is a current source of revenue, and its continued rollout and increasing adoption in the market are anticipated to contribute to revenue growth.
  • Potential Regulatory Approval and Commercial Launch of AMT-130 for Huntington's Disease: Despite recent regulatory setbacks and the U.S. FDA recommending a Phase III sham-controlled study, AMT-130 remains a significant potential revenue driver. Analysts project substantial annual revenue if approved, with a potential commercial launch in 2027 or 2028.
  • Expansion into International Markets: The company is actively engaging with regulatory authorities outside the U.S. for its pipeline assets, including AMT-130. This strategic focus on international markets presents an opportunity for geographical expansion and increased revenue from existing or newly approved products.

AI Analysis | Feedback

Share Issuance

  • In 2025, uniQure received aggregate net proceeds of $404.2 million from follow-on public offerings, which included the issuance of 11.8 million ordinary shares and pre-funded warrants to purchase 0.5 million ordinary shares.
  • An underwritten public offering in September 2025 generated approximately $345 million in gross proceeds from the sale of 6,736,841 ordinary shares and 526,316 pre-funded warrants.
  • Net proceeds of $80.5 million were raised in January and February 2025 through a follow-on offering.

Inbound Investments

  • uniQure refinanced its existing $50 million debt in the third quarter of 2025, extending the term to October 2030 and reducing its cost of capital.
  • An additional term loan tranche of $100 million is available, subject to a pre-defined regulatory milestone for AMT-130, with a third tranche of $25 million also available upon lender approval.
  • The company's cash and investments grew to $622.5 million as of December 31, 2025, primarily due to proceeds from public offerings, up from $367.5 million at the end of 2024.

Capital Expenditures

  • Research and development expenses totaled $140.7 million in 2025, a decrease from $143.8 million in 2024.
  • Selling, general and administrative expenses were $65.5 million in 2025, increasing from $52.7 million in 2024, partly due to $6.5 million incurred for the planned commercialization of AMT-130 in the United States.
  • In the third quarter of 2025, direct research and development expenses increased by $10.1 million, including $6.6 million related to the preparation for the Biologics License Application submission for AMT-130.

Better Bets vs. uniQure (QURE)

Latest Trefis Analyses

Trade Ideas

Select ideas related to QURE.

Unique KeyDateTickerCompanyCategoryTrade Strategy6M Fwd Rtn12M Fwd Rtn12M Max DD
PGNY_3312026_Dip_Buyer_High_FCF_Yield_ExInd_DE_RevG03312026PGNYProgynyDip 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
0.0%0.0%0.0%
CNC_3272026_Dip_Buyer_High_FCF_Yield_ExInd_DE_RevG03272026CNCCenteneDip 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
2.3%2.3%-0.6%
OSCR_3272026_Dip_Buyer_High_FCF_Yield_ExInd_DE_RevG03272026OSCROscar 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
3.0%3.0%-2.6%
WAT_3202026_Monopoly_xInd_xCD_Getting_Cheaper03202026WATWatersMonopolyMY | Getting CheaperMonopoly-Like with P/S Decline
Large cap with monopoly-like margins or cash flow generation and getting cheaper based on P/S multiple
-0.4%-0.4%-3.3%
GILD_3202026_Quality_Momentum_RoomToRun_10%03202026GILDGilead SciencesQualityQ | Momentum | UpsideQuality Stocks with Momentum and Upside
Buying quality stocks with strong momentum but still having room to run
1.6%1.6%-2.2%

Recent Active Movers

Peer Comparisons

Peers to compare with:

Financials

QUREBMRNSRPTIONSVRTXRGNXMedian
NameuniQure BioMarin.Sarepta .Ionis Ph.Vertex P.Regenxbio 
Mkt Price15.3854.5321.1875.17436.278.8037.86
Mkt Cap1.010.52.212.2110.60.56.4
Rev LTM163,2212,19894412,0011701,571
Op Inc LTM-181409-658-3824,554-161-171
FCF LTM-178717-315-3263,194-126-152
FCF 3Y Avg-175415-419-4051,832-177-176
CFO LTM-178828-205-2693,631-124-151
CFO 3Y Avg-169520-304-3592,225-172-170

Growth & Margins

QUREBMRNSRPTIONSVRTXRGNXMedian
NameuniQure BioMarin.Sarepta .Ionis Ph.Vertex P.Regenxbio 
Rev Chg LTM-40.6%12.9%15.6%33.8%8.9%104.5%14.2%
Rev Chg 3Y Avg-18.2%15.4%33.9%19.2%10.4%25.6%17.3%
Rev Chg Q6.6%17.0%-32.7%-10.3%9.5%43.0%8.1%
QoQ Delta Rev Chg LTM2.2%4.1%-8.9%-2.4%2.4%5.7%2.3%
Op Mgn LTM-1,125.9%12.7%-29.9%-40.5%37.9%-94.6%-35.2%
Op Mgn 3Y Avg-1,193.2%12.9%-13.3%-50.9%24.7%-223.1%-32.1%
QoQ Delta Op Mgn LTM-23.8%-7.2%-26.4%-11.7%38.7%1.5%-9.4%
CFO/Rev LTM-1,105.5%25.7%-9.3%-28.5%30.3%-72.7%-18.9%
CFO/Rev 3Y Avg-900.1%17.5%-20.2%-46.2%20.5%-174.2%-33.2%
FCF/Rev LTM-1,108.6%22.3%-14.3%-34.5%26.6%-74.1%-24.4%
FCF/Rev 3Y Avg-930.6%13.7%-26.7%-51.9%17.0%-179.3%-39.3%

Valuation

QUREBMRNSRPTIONSVRTXRGNXMedian
NameuniQure BioMarin.Sarepta .Ionis Ph.Vertex P.Regenxbio 
Mkt Cap1.010.52.212.2110.60.56.4
P/S59.63.31.012.99.22.76.2
P/EBIT-7.321.2-3.3-41.323.8-3.1-3.2
P/E-4.830.0-3.1-32.028.0-2.3-2.7
P/CFO-5.412.7-10.8-45.430.5-3.7-4.5
Total Yield-20.7%3.3%-32.1%-3.1%3.6%-42.6%-11.9%
Dividend Yield0.0%0.0%0.0%0.0%0.0%0.0%0.0%
FCF Yield 3Y Avg-27.3%3.5%-7.6%-5.7%1.7%-30.2%-6.7%
D/E0.10.10.50.20.00.20.1
Net D/E-0.6-0.10.0-0.1-0.0-0.3-0.1

Returns

QUREBMRNSRPTIONSVRTXRGNXMedian
NameuniQure BioMarin.Sarepta .Ionis Ph.Vertex P.Regenxbio 
1M Rtn-9.2%-7.7%24.3%3.9%-8.8%-3.1%-5.4%
3M Rtn-39.3%-7.5%-11.1%-6.4%-5.9%-39.7%-9.3%
6M Rtn-75.0%4.2%-4.2%7.4%7.0%-19.4%0.0%
12M Rtn72.2%-4.0%-58.5%169.4%-9.9%49.4%22.7%
3Y Rtn-16.7%-44.3%-84.6%105.4%33.7%-50.3%-30.5%
1M Excs Rtn-13.0%-10.2%19.9%-0.1%-12.0%-6.8%-8.5%
3M Excs Rtn-32.1%-8.1%-7.0%-6.3%-5.6%-39.8%-7.6%
6M Excs Rtn-75.3%-1.2%-10.3%6.5%2.7%-28.9%-5.8%
12M Excs Rtn47.6%-39.2%-93.8%157.9%-44.5%28.9%-5.2%
3Y Excs Rtn-88.7%-109.1%-151.0%46.4%-27.5%-120.2%-98.9%

Comparison Analyses

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Financials

Segment Financials

Revenue by Segment
$ Mil20252024202320222021
Discovery and development of innovative gene therapies2716   
Collaboration revenues  570
Contract manufacturing revenues  2  
License revenues  10051737
Total271610652438


Price Behavior

Price Behavior
Market Price$15.38 
Market Cap ($ Bil)1.0 
First Trading Date02/05/2014 
Distance from 52W High-78.2% 
   50 Days200 Days
DMA Price$18.70$25.19
DMA Trendindeterminatedown
Distance from DMA-17.8%-39.0%
 3M1YR
Volatility158.3%278.2%
Downside Capture1.38-0.34
Upside Capture32.9919.96
Correlation (SPY)11.6%2.0%
QURE Betas & Captures as of 3/31/2026

 1M2M3M6M1Y3Y
Beta0.561.681.300.890.821.25
Up Beta30.229.847.146.311.281.32
Down Beta-1.98-2.56-1.31-0.162.121.98
Up Capture-197%67%39%-118%9%88%
Bmk +ve Days7162765139424
Stock +ve Days10192955117339
Down Capture-97%238%205%141%-17%106%
Bmk -ve Days12233358110323
Stock -ve Days12233471133401

[1] Upside and downside betas calculated using positive and negative benchmark daily returns respectively
Based On 1-Year Data
Annualized
Return
Annualized
Volatility
Sharpe
Ratio
Correlation
with QURE
QURE84.5%277.4%0.95-
Sector ETF (XLV)12.3%16.8%0.523.5%
Equity (SPY)31.2%17.3%1.474.0%
Gold (GLD)60.1%27.8%1.69-3.1%
Commodities (DBC)29.8%16.6%1.582.6%
Real Estate (VNQ)21.3%15.2%1.07-3.3%
Bitcoin (BTCUSD)-4.3%43.7%0.024.9%

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Based On 5-Year Data
Annualized
Return
Annualized
Volatility
Sharpe
Ratio
Correlation
with QURE
QURE-15.6%153.1%0.36-
Sector ETF (XLV)6.3%14.6%0.2511.1%
Equity (SPY)11.1%17.0%0.5015.0%
Gold (GLD)22.1%17.8%1.020.4%
Commodities (DBC)11.8%18.8%0.520.5%
Real Estate (VNQ)3.7%18.8%0.108.5%
Bitcoin (BTCUSD)4.3%56.5%0.308.3%

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Based On 10-Year Data
Annualized
Return
Annualized
Volatility
Sharpe
Ratio
Correlation
with QURE
QURE1.9%119.5%0.41-
Sector ETF (XLV)9.8%16.5%0.4816.1%
Equity (SPY)13.8%17.9%0.6617.8%
Gold (GLD)14.2%15.9%0.740.9%
Commodities (DBC)8.6%17.6%0.413.1%
Real Estate (VNQ)5.1%20.7%0.2210.1%
Bitcoin (BTCUSD)67.6%66.9%1.077.7%

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Short Interest

Short Interest: As Of Date3312026
Short Interest: Shares Quantity11.0 Mil
Short Interest: % Change Since 3152026-13.7%
Average Daily Volume2.0 Mil
Days-to-Cover Short Interest5.5 days
Basic Shares Quantity62.4 Mil
Short % of Basic Shares17.6%

Earnings Returns History

Expand for More
 Forward Returns
Earnings Date1D Returns5D Returns21D Returns
3/2/2026-32.8%-8.7%-5.8%
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%
...
SUMMARY STATS   
# Positive11810
# Negative101311
Median Positive3.6%7.7%13.2%
Median Negative-4.3%-8.3%-17.8%
Max Positive12.7%37.8%38.0%
Max Negative-32.8%-25.3%-27.5%

SEC Filings

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Report DateFiling DateFiling
12/31/202503/02/202610-K
09/30/202511/10/202510-Q
06/30/202507/29/202510-Q
03/31/202505/09/202510-Q
12/31/202402/27/202510-K
09/30/202411/05/202410-Q
06/30/202408/01/202410-Q
03/31/202405/07/202410-Q
12/31/202302/28/202410-K
09/30/202311/07/202310-Q
06/30/202308/01/202310-Q
03/31/202305/09/202310-Q
12/31/202202/27/202310-K
09/30/202211/02/202210-Q
06/30/202208/08/202210-Q
03/31/202205/02/202210-Q

Recent Forward Guidance [BETA]

Latest: Q4 2025 Earnings Reported 3/2/2026

Forward GuidanceGuidance Change
MetricLowMidHigh% Chg% DeltaChangePrior
2029 Cash Runway     AffirmedGuidance: 2,029 for 2029

Prior: Q3 2025 Earnings Reported 11/10/2025

Forward GuidanceGuidance Change
MetricLowMidHigh% Chg% DeltaChangePrior
2025 Patient Dosing Completion 2,025    
2029 Cash Runway 2,029 0.7% RaisedGuidance: 2,027 for 2027

Insider Activity

Expand for More
#OwnerTitleHoldingActionFiling DatePriceSharesTransacted
Value
Value of
Held Shares
Form
1Gut, RobertDirectSell1110202527.2631,434856,8811,094,340Form
2Kaye, JackDirectSell1106202530.3438,8101,177,517620,131Form
3Kapusta, Matthew CCEO, Managing DirectorDirectSell926202541.46226,3169,382,47327,007,588Form
4Klemt, ChristianChief Financial OfficerDirectSell926202540.043,000120,1208,717,909Form
5Klemt, ChristianChief Financial OfficerDirectSell926202555.0015,000825,00011,975,150Form