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RARE.O NASDAQ Pharmaceuticals

Ultragenyx Pharmaceutical Inc

$25,03
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1D5D1M3M6MYTD1Y5YMax
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Mcap
P/E
EV / Rev
Div yield
Op margin
-83,8 %
ROE
761,2 %
Net margin
-90,9 %
Debt / equity
Beta
52w range
Volume
Day range
Prev close
$34,61
Open
$34,71
Next earnings
2026-10-28
Ex-dividend
TR 1Y
About

Ultragenyx Pharmaceutical Inc is a biotechnology company focused on the discovery, development, and commercialization of transformative therapies for rare genetic diseases.

Business. Ultragenyx Pharmaceutical Inc (RARE.O) is a healthcare company operating in the pharmaceuticals industry. The firm is headquartered in the United States and is primarily listed on the NASDAQ stock exchange. As segment and geographic breakdowns are not provided, the company is described at the industry level as a participant in the pharmaceuticals sector.

Classification66 %
SectorHealthcare
Business sectorPharmaceuticals & Medical Research
Industry groupPharmaceuticals
Generated · model-assisted
Sell-side consensus
BUY19 analysts
17 buy2 hold0 sell
Avg 12m price target50,94

Analyst recommendations

19 analysts · consensus Buy
Buy17
Hold2
Sell0
12-month price target
50,94
Consensus of sell-side coverage.
Upcoming events
  • EarningsQ3 2026 earnings (expected)2026-10-28 · estimated
See all catalysts →

At a glance

Score
not yet scored
Valuation
valuation pending
Analysts
Buy
19 analysts · indicative
Ownership
AQR Capital Management, LLC
largest disclosed fund holder
Profitability
761,2 %
return on equity
Quality
not yet scored

What drives this business

The watch-list the newsroom runs for this company — derived from its sector path, sharpened layer by layer. Not investment advice.

— missing data

News & coverage

0
  • No recent newsroom coverage mentioning RARE.
  • Sector rotation

    Sector1D1Mvs mkt
    Materials+2,3 %+6,6 %+1,7 %
    Communication Services+2,2 %−5,5 %+1,6 %
    Energy+0,6 %+3,3 %+0,0 %
    Health Care · THIS SECTOR+0,6 %−0,8 %+0,0 %
    Information Technology+0,6 %+6,7 %+0,0 %
    Consumer Discretionary+0,4 %+7,6 %−0,2 %
    Financials−0,3 %−2,8 %−0,9 %
    Consumer Staples−0,4 %+2,5 %−1,0 %
    Real Estate−0,7 %+10,9 %−1,3 %
    Industrials−1,9 %−3,1 %−2,5 %
    Utilities−1,9 %+28,2 %−2,5 %

    Developing storylines

    No tracked sagas currently linked to RARE.O. Browse all sagas →

    Analysis

    AI analysis
    Generated · analysis pipeline · tier hybrid · as of 2026-08-04 ↑ At a glance

    Opportunity

    — missing data

    Upcoming catalysts

    Scheduled public events. Informational only — not investment advice.

    • Company
    • EarningsQ3 2026 earnings (expected)2026-10-28 · estimated
    • Macro
    • Rate decisionBank of England rate decision (press conf.)2026-08-06 · GB
    • Rate decisionReserve Bank of Australia rate decision (press conf.)2026-08-12 · AU
    • Rate decisionNorges Bank rate decision (press conf.)2026-08-20 · NO
    • Rate decisionSveriges Riksbank rate decision (press conf.)2026-09-03 · SE
    • Rate decisionBank of Canada rate decision (press conf.)2026-09-09 · CA
    • Rate decisionEuropean Central Bank rate decision (press conf.)2026-09-10 · EU
    • Macro & political
    • ElectionSE Swedish Election2026-09-14 · SE
    • ElectionUS U.S. Midterms2026-11-03 · US
    • ElectionFR French Legislative2027-06-01 · FR

    Pre-earnings brief

    Briefing · model-assisted

    This is the first analysis for Ultragenyx Pharmaceutical Inc (RARE.O), establishing a baseline with no prior data for delta computation. Consequently, there are no material changes to report relative to a previous period, and the company currently has zero analyst coverage, zero index memberships, and zero reported officers in the available dataset. The most significant activity detected involves a major holder change for investor ID 587. On June 24, 2026, this investor’s position was modified to reflect 11,045 shares, valued at $0.231 million, representing a portfolio weight of 0.000134% as of March 31, 2026. This update followed an earlier detection on June 21, 2026, which recorded the position at 5 shares, valued at $0.000105 million, with a negligible weight of 0.00000006%. These conflicting signals regarding the same investor’s holdings on consecutive days highlight a data correction or reporting adjustment rather than a clear directional shift in ownership. The discrepancy between the 5-share and 11,045-share figures for the same reporting period (March 31, 2026) suggests volatility in the reporting of this specific stakeholder’s position. Ultragenyx maintains a broad base of 29 top holders, indicating distributed ownership despite the lack of analyst or index presence. With no cross-source signals or sagas identified, the current financial profile is defined by this initial data capture and the specific holder adjustment, providing a foundational view of the company’s shareholder structure.

    Signals & dispatch

    peak dispatch · —

    Composite-score breakdown

    Synthesis

    Business

    Ultragenyx Pharmaceutical Inc (RARE.O) is a healthcare company operating in the pharmaceuticals industry. The firm is headquartered in the United States and is primarily listed on the NASDAQ stock exchange. As segment and geographic breakdowns are not provided, the company is described at the industry level as a participant in the pharmaceuticals sector.

    Classification66 %
    SectorHealthcare
    Business sectorPharmaceuticals & Medical Research
    Industry groupPharmaceuticals
    AI synthesis
    GENERATED

    Ultragenyx operates with a negative equity position of -80 million USD against total liabilities of 1.605 billion USD and total assets of 1.532 billion USD. The balance sheet reflects a debt-to-equity ratio of -0.0, indicating that the negative equity base distorts traditional leverage metrics. Liquidity is supported by cash and equivalents of 421 million USD, which stands against an operating cash flow burn of 466 million USD. The company reports low liquidity risk and low dilution risk according to internal assessments, with no immediate filing-based flags detected. Capital expenditures remain minimal at 6 million USD, suggesting limited heavy asset investment requirements typical of small-molecule or biologic developers in clinical or early commercial phases.

    Profitability metrics are deeply negative, with a net income of -575 million USD and an operating income of -535 million USD on revenues of 673 million USD. The return on assets is -0.3975, reflecting the significant cash burn relative to the asset base. The reported return on equity of 7.6115 is a mathematical artifact of the negative equity denominator and does not represent positive economic returns. Without cohort median data provided for comparison, the absolute losses indicate a pre-profitability stage common in biotechnology firms with high research and development costs and limited commercial scale.

    Revenue concentration and segment details are not explicitly broken down in the provided data, but the total revenue of 673 million USD suggests a reliance on a limited number of approved therapies. Geographic exposure is not detailed in the input. The absence of segment data prevents a granular analysis of revenue drivers, but the scale of revenue relative to the cash burn indicates that commercial operations are active but not yet sufficient to cover operating expenses.

    Growth trajectory analysis is constrained by the absence of historical period data in the input. The current revenue of 673 million USD serves as the baseline for recent performance. Without multi-year revenue or net income history, long-term growth rates cannot be calculated. The company’s financial profile suggests a transition phase where commercial revenues are growing but have not yet offset the substantial operating costs associated with R&D and commercialization.

    Risk factors are characterized by low liquidity and dilution risks according to the assessment. The negative equity position presents a structural risk, but the substantial cash reserve of 421 million USD provides a buffer against immediate solvency concerns. The key flags section notes no immediate filing-based liquidity or dilution flags, suggesting that the current capital structure is stable in the short term. The primary risk remains the ability to generate sufficient cash flow from operations to sustain the business without further equity or debt financing.

    Recent events are reflected in analyst sentiment, with a mean price target of 50.94 USD and a median of 47.50 USD. The mean recommendation of 1.79 indicates a strong buy consensus, supported by 6 strong buy and 11 buy ratings versus only 2 holds. The high price target of 96.00 USD versus the low of 25.00 USD suggests significant divergence in analyst expectations regarding the company’s future commercial success and pipeline valuation. No specific filing, news, or transcript observations were provided in the input data.

    This is the first analysis for Ultragenyx Pharmaceutical Inc (RARE.O), establishing a baseline with no prior data for delta computation. Consequently, there are no material changes to report relative to a previous period, and the company currently has zero analyst coverage, zero index memberships, and zero reported officers in the available dataset. The most significant activity detected involves a major holder change for investor ID 587. On June 24, 2026, this investor’s position was modified to reflect 11,045 shares, valued at $0.231 million, representing a portfolio weight of 0.000134% as of March 31, 2026. This update followed an earlier detection on June 21, 2026, which recorded the position at 5 shares, valued at $0.000105 million, with a negligible weight of 0.00000006%. These conflicting signals regarding the same investor’s holdings on consecutive days highlight a data correction or reporting adjustment rather than a clear directional shift in ownership. The discrepancy between the 5-share and 11,045-share figures for the same reporting period (March 31, 2026) suggests volatility in the reporting of this specific stakeholder’s position. Ultragenyx maintains a broad base of 29 top holders, indicating distributed ownership despite the lack of analyst or index presence. With no cross-source signals or sagas identified, the current financial profile is defined by this initial data capture and the specific holder adjustment, providing a foundational view of the company’s shareholder structure.

    Key takeaways
    • Negative equity of -80 million USD distorts leverage ratios, but 421 million USD in cash provides short-term liquidity.
    • Operating cash flow burn of 466 million USD exceeds annual revenue of 673 million USD, indicating a path to profitability is not yet achieved.
    • Analyst consensus is strongly positive with a mean recommendation of 1.79 and a mean price target of 50.94 USD.
    • Low dilution and liquidity risk flags suggest no immediate pressure for capital raising, despite the negative equity position.
    • Minimal capital expenditures of 6 million USD align with a biotechnology model focused on R&D rather than heavy infrastructure.

    Bull / Bear case

    Generated · model-assisted
    BULL CASE · 5

    Revenue grew 20.1% year-over-year to $673 million in FY2026, demonstrating strong top-line expansion momentum.

    Gross profit surged to $564 million in FY2026, indicating robust pricing power and efficient cost of goods sold.

    Return on equity of 7.61% ranks as best-in-class within the pharmaceutical cohort of 842 peers.

    Current ratio of 2.48 suggests adequate short-term liquidity to cover immediate operational obligations comfortably.

    Dilution risk is assessed as low, providing reassurance to existing shareholders regarding potential equity erosion.

    BEAR CASE · 4

    The company posted a net loss of $575 million in FY2026, reflecting persistent inability to achieve profitability.

    Free cash flow remained deeply negative at -$561 million in FY2026, highlighting significant ongoing cash burn.

    Long-term debt increased to $1.15 billion in FY2026, signaling rising leverage and potential refinancing risks.

    Credit risk is flagged as high, indicating potential difficulties in securing favorable financing terms or repayment.

    In focus — financials by report

    Quarterly
    Annual
    QUARTERLYFiled 2026-05-05
    Q1 2026 · Quarter highlights
    Open report →

    Revenue $136.0M, −2,4% YoY; Operating income −18,2% YoY.

    Revenue$136.0M−2,4 % YoY
    Operating income-$169.0M−18,2 % YoY
    Net income-$185.0M−22,4 % YoY
    Free cash flow-$183.0M−15,5 % YoY
    EPS
    Operating cash flow-$197.0M−18,3 % YoY
    Financials
    Income statement
    Revenue$136.0M
    Gross profit$106.0M
    Operating income-$169.0M
    Net income-$185.0M
    Margins
    Gross margin77.9%
    Operating margin-124.3%
    Net margin-136.0%
    FCF margin-134.6%
    Balance sheet
    Total assets$1.30B
    Total liabilities$1.53B
    Total equity-$236.0M
    Cash & equivalents$175.0M
    Long-term debt$1.15B
    Cash flow
    Operating cash flow-$197.0M
    CapEx-$6.0M
    Free cash flow-$183.0M
    SBC
    P&L flow · revenue → net income
    Revenue $136.0MOperating costs $305.0MTax $16.0MNet income $185.0M
    Highlights
    • Revenue $136.0M, −2,4% YoY
    • Operating income −18,2% YoY
    • Net income −22,4% YoY
    • Free cash flow −15,5% YoY
    • Net margin -136.0%

    Valuation TTM

    Market price
    $25,03
    Market cap
    Enterprise value
    P/E
    Non-GAAP P/E
    EV / Revenue
    EV / Op income
    EV / OCF
    P / B
    P / Tangible book
    Tangible book
    -$73.0M
    Net cash
    $421.0M
    Current ratio
    Debt / equity
    ROA
    -39.8%
    ROE
    7.6%
    Cash conversion
    77.0%
    CapEx / revenue
    -3.1%
    SBC / revenue
    Dilution ratio
    0.0%

    Revenue by segment

    Market share

    — missing data

    Business relationships

    Business relationships16 disclosed relationships · 2 types · extracted from filings & disclosures
    Subsidiaries15
    EX21:0a546fcadf1eEX21:0a546fcadf1eSubsidiarie97%
    EX21:1173df31ed24EX21:1173df31ed24Subsidiarie97%
    EX21:1fd3f799d852EX21:1fd3f799d852Subsidiarie97%
    EX21:2e5f43bfc36bEX21:2e5f43bfc36bSubsidiarie97%
    EX21:30cc3da8f58cEX21:30cc3da8f58cSubsidiarie97%
    EX21:3ed442bae098EX21:3ed442bae098Subsidiarie97%
    EX21:429346a47211EX21:429346a47211Subsidiarie97%
    EX21:7d4333fc4d15EX21:7d4333fc4d15Subsidiarie97%
    EX21:8908690bf842EX21:8908690bf842Subsidiarie97%
    EX21:9a9682d0afc7EX21:9a9682d0afc7Subsidiarie97%
    EX21:a3222889c7f0EX21:a3222889c7f0Subsidiarie97%
    EX21:a5e9f3e83503EX21:a5e9f3e83503Subsidiarie97%
    EX21:a9a4be89f9b7EX21:a9a4be89f9b7Subsidiarie97%
    EX21:b7f3e3580accEX21:b7f3e3580accSubsidiarie97%
    EX21:c910489eecc9EX21:c910489eecc9Subsidiarie97%

    Supply chain

    Vendors → RARE ↔ Partners → Customers · click any node to drill
    VENDORSPARTNERSCUSTOMERSRARERAREUltragenyx Pharmaceutical Inc16 entitiesREGENERON PHARMACEUTICDiversified PharmaceuticUS
    1 mention in 10-K0 from corporate websites0 from news dispatches
    Updated 2026-06-24

    Peer comparison

    — missing data

    Market position

    Stress test

    — missing data

    Predictor forecast

    Next quarternear-term
    Earnings · next quarterconf 45 %
    EPS
    Consensus EPS
    -4,63
    Predicted surprise
    -0,00
    Beat probability
    45 %
    Analysts
    19
    Other metrics
    Revenue
    no estimate
    Segment revenue
    no estimate
    Margin
    no estimate
    Segment margin
    no estimate
    as of 2026-07-10 · Earnings Surprise V1
    Period note: consensus is not fiscal-period-aligned at source — read as consensus vs the last reported actual, not a calibrated same-quarter surprise.
    Full fiscal year~1 year ahead
    Full fiscal year · our forecast vs guidance vs consensus
    MetricOur forecastGuidanceConsensus
    EPSno estimateno estimate-4,63
    Revenueno estimateno estimate742,0M USD
    Operating incomeno estimateno estimate-442,7M USD
    Full-year consensus mean (period as reported by source) · consensus in USD. Company-level full-year forecast and management guidance are not yet modelled at scale — shown as "no estimate", never inferred.
    Corporate eventM&A horizon
    M&A probabilityconf 3 %
    Probability
    3 %
    Base rate
    3 %
    Band
    180-day horizon · target 2027-01-19 · 1,0× base rate
    No acquirer candidates identified by the signal model.
    Probabilistic model output — not investment advice. · generated 2026-08-04

    Options

    — missing data

    Short squeeze

    — missing data

    Earnings-call key lines

    — missing data

    Consensus distribution

    sell-side coverage
    Recommendation distribution19 analysts
    Strong buy6
    Buy11
    Hold2
    Sell0
    Strong sell0
    12-month price target$50,94 · Median $47,50
    Low $25,00High $96,00
    Operating income · consensus-442,7M USD
    EPS surprise
    −25,9 %
    reported vs consensus · miss
    Revenue surprise
    −9,3 %
    reported vs consensus · miss

    Estimate revisions

    consensus EPS · 26-week trend
    — missing data

    Sell-side observations

    Low$25,00
    Mean$50,94
    Median$47,50
    High$96,00
    Spot$25,03
    +103.5 %implied to mean12-month sell-side price targets · ▲ spot

    Themes

    — missing data

    ESG

    — missing data

    Risk factors

    Dilution riskLow
    Liquidity riskLow
    Filing-based flags
    • No immediate filing-based liquidity or dilution flags were detected.

    Benchmarks vs cohort

    Op Margin-83,8 %Bottom quartile
    Net Margin-90,9 %Bottom quartile
    ROE761,2 %Best in class
    Capex / Rev-3,1 %Above median
    D/E-0,00Above P75
    Cash Conv0,77Below median

    Corporate actions / M&A

    M&A probabilityconf —
    Probability
    3 %
    Base rate
    3 %
    Band
    Low
    180-day horizon · target 2027-01-19 · 1,0× base rate
    No acquirer candidates identified by the signal model.
    Corporate actionsNo corporate-action source connected yet — splits, dividends and buyback events are not tracked for this company. Honest-dim (no data fabricated).

    FX exposure

    — missing data

    Comparable transactions

    — missing data

    Derivatives & instruments

    Derivative transactions

    Net acquired 618 143 sh15 grants15 insiders
    • Fust Matthew KDirector · Stock Option (Right to Buy) → Common StockAcquired 14 058 @ $25,80grant · 2026-05-14
    • Ray AmritDirector · Stock Option (Right to Buy) → Common StockAcquired 14 058 @ $25,80grant · 2026-05-14
    • SULIMAN SHEHNAAZDirector · Stock Option (Right to Buy) → Common StockAcquired 14 058 @ $25,80grant · 2026-05-14
    • NARACHI MICHAELDirector · Stock Option (Right to Buy) → Common StockAcquired 14 058 @ $25,80grant · 2026-05-14
    • WELCH DANIEL GDirector · Stock Option (Right to Buy) → Common StockAcquired 14 058 @ $25,80grant · 2026-05-14
    • Dunsire DeborahDirector · Stock Option (Right to Buy) → Common StockAcquired 14 058 @ $25,80grant · 2026-05-14
    • Sanders Corazon (Corsee) D.Director · Stock Option (Right to Buy) → Common StockAcquired 14 058 @ $25,80grant · 2026-05-14
    • Huizenga Theodore AlanSVP, Chief Accounting Officer · Stock Option (Right to Buy) → Common StockAcquired 7 348 @ $24,61grant · 2026-04-22
    • Parschauer Karah HerdmanEVP and Chief Legal Officer · Stock Option (Right to Buy) → Common StockAcquired 49 960 @ $24,52grant · 2026-04-16
    • KAKKIS EMIL DDirector, President & CEO · Stock Option (Right to Buy) → Common StockAcquired 132 693 @ $24,52grant · 2026-04-16
    • Huang Dennis KarlSee Remarks · Stock Option (Right to Buy) → Common StockAcquired 79 936 @ $24,52grant · 2026-04-16
    • Pinion John RichardSee Remarks · Stock Option (Right to Buy) → Common StockAcquired 79 936 @ $24,52grant · 2026-04-16
    • Harris ErikEVP & Chief Commercial Officer · Stock Option (Right to Buy) → Common StockAcquired 49 960 @ $24,52grant · 2026-04-16
    • Crombez EricEVP and Chief Medical Officer · Stock Option (Right to Buy) → Common StockAcquired 59 952 @ $24,52grant · 2026-04-16
    • Horn HowardChief Financial Officer · Stock Option (Right to Buy) → Common StockAcquired 59 952 @ $24,52grant · 2026-04-16

    Actions

    Ask Handelsavisen

    — missing data
    Data sources
    • Market data
    • Market data cache
    • Issuer disclosures
    • Public news
    • Earnings transcripts
    • Consensus estimates
    • ESG data
    • Reference data
    How metrics are computed
    • Debt To Equity
      (short_term_debt + long_term_debt) / total_equity
    • Cash Conversion Ratio
      operating_cash_flow / net_income
    • Dilution Ratio
      (shares_outstanding_diluted - shares_outstanding_basic) / shares_outstanding_basic
    • Net Cash
      cash_and_equivalents + short_term_investments - short_term_debt - long_term_debt
    • Return On Assets
      net_income / total_assets
    • Return On Equity
      net_income / total_equity
    Source documents
    • ULTRAGENYX PHARMACEUTICAL INC. Market data — financials · 2026-07-10
    • Ultragenyx Pharmaceutical Inc Market data — analyst estimates · 2026-07-10
    • Ultragenyx Pharmaceutical Inc Market data — ESG · 2026-07-10
    • Ultragenyx Pharmaceutical Inc HA canonical relationships · 2026-07-10

    Ownership & reference

    Top holders

    • AQR Capital Management, LLCInvestment Managers · as of 2026-03-310,04 %$96M
    • Wellington Management Company, LLPInvestment Managers · as of 2026-03-310,01 %$55M
    • BlackRock Institutional Trust Company, N.A.Investment Managers · as of 2024-06-300,01 %$230M
    • Nuveen LLCInvestment Managers · as of 2024-12-310,00 %$16M
    • State Street Investment Management (US)Investment Managers · as of 2026-03-310,00 %$105M
    • Vanguard Capital Management, LLCInvestment Managers · as of 2025-12-310,00 %$236M
    • UBS Financial Services, Inc.Investment Managers · as of 2026-03-310,00 %$17M
    • Geode Capital Management, L.L.C.Investment Managers · as of 2026-03-310,00 %$39M
    • T. Rowe Price Associates, Inc.Investment Managers · as of 2026-03-310,00 %$0M
    • Morgan Stanley Smith Barney LLCInvestment Managers · as of 2026-03-310,00 %$31M
    • MASSACHUSETTS FINANCIAL SERVICES CO /MA/Institutional Investor · as of 2026-03-310,00 %$5M
    • Charles Schwab Investment Management, Inc.Investment Managers · as of 2026-03-310,00 %$12M
    • Franklin Advisers, Inc.Investment Managers · as of 2026-03-310,00 %$7M
    • Northern Trust Investments, Inc.Investment Managers · as of 2026-03-310,00 %$11M
    • Bank of New York Mellon CorpInstitutional Investor · as of 2026-03-310,00 %$7M
    • Goldman Sachs Asset Management, L.P.Investment Managers · as of 2026-03-310,00 %$7M
    • Invesco QQQ TrustFunds · as of 2026-03-310,00 %$6M
    • JANUS HENDERSON GROUP PLCInstitutional Investor · as of 2026-03-310,00 %$1M
    • FMR LLCInstitutional Investor · as of 2026-03-310,00 %$6M
    • Wells Fargo AdvisorsBrokerage Firms · as of 2026-03-310,00 %$1M
    • Victory Capital Management Inc.Investment Managers · as of 2026-03-310,00 %$0M
    • American Century Investment Management, Inc.Investment Managers · as of 2026-03-310,00 %$0M
    • First Trust Advisors L.P.Investment Managers · as of 2026-03-310,00 %$0M
    • AllianceBernstein L.P.Investment Managers · as of 2026-03-310,00 %$1M
    • AMERIPRISE FINANCIAL INCInstitutional Investor · as of 2026-03-310,00 %$1M
    • Raymond James & Associates, Inc.Brokerage Firms · as of 2026-03-310,00 %$1M
    • JONES FINANCIAL COMPANIES LLLPInstitutional Investor · as of 2026-03-310,00 %$0M
    • PNC Investments LLCInvestment Managers · as of 2026-03-310,00 %$0M
    • PNC Investments LLCInvestment Managers · as of 2026-03-310,00 %$0M

    Insider activity

    Net selling0 buys · 13 sellsnet -$2,6M15 insiders · last 365d
    • Horn HowardChief Financial Officer · Common StockSold 4 698 @ $33,13$156K · 2026-07-01
    • Sanders Corazon (Corsee) D.Director · Common StockSold 2 000 @ $25,05$50K · 2026-06-15
    • Parschauer Karah HerdmanEVP and Chief Legal Officer · Common StockSold 1 899 @ $24,62$47K · 2026-06-15
    • Horn HowardChief Financial Officer · Common StockSold 4 653 @ $23,77$111K · 2026-06-01
    • SULIMAN SHEHNAAZDirector · Common StockSold 5 740 @ $25,12$144K · 2026-05-18
    • SULIMAN SHEHNAAZDirector · Common StockOther 7 751 · 2026-05-14
    • Fust Matthew KDirector · Common StockOther 7 751 · 2026-05-14
    • Ray AmritDirector · Common StockOther 7 751 · 2026-05-14
    • Sanders Corazon (Corsee) D.Director · Common StockOther 7 751 · 2026-05-14
    • NARACHI MICHAELDirector · Common StockOther 7 751 · 2026-05-14
    • WELCH DANIEL GDirector · Common StockOther 7 751 · 2026-05-14
    • Dunsire DeborahDirector · Common StockOther 7 751 · 2026-05-14
    • Crombez EricEVP and Chief Medical Officer · Common StockSold 344 @ $24,96$9K · 2026-05-05
    • Horn HowardChief Financial Officer · Common StockSold 4 683 @ $24,90$117K · 2026-05-01
    • Huizenga Theodore AlanSVP, Chief Accounting Officer · Common StockOther 20 241 · 2026-04-22
    • Pinion John RichardSee Remarks · Common StockOther 23 116 · 2026-04-16
    • KAKKIS EMIL DDirector, President & CEO · Common StockOther 76 745 · 2026-04-16
    • Huang Dennis KarlSee Remarks · Common StockOther 23 116 · 2026-04-16
    • Parschauer Karah HerdmanEVP and Chief Legal Officer · Common StockOther 28 895 · 2026-04-16
    • Harris ErikEVP & Chief Commercial Officer · Common StockOther 28 895 · 2026-04-16
    • Crombez EricEVP and Chief Medical Officer · Common StockOther 34 674 · 2026-04-16
    • Horn HowardChief Financial Officer · Common StockOther 34 674 · 2026-04-16
    • Horn HowardChief Financial Officer · Common StockSold 4 683 @ $20,97$98K · 2026-04-01
    • Parschauer Karah HerdmanEVP and Chief Legal Officer · Common StockSold 3 508 @ $22,67$80K · 2026-03-10
    • Parschauer Karah HerdmanEVP and Chief Legal Officer · Common StockSold 6 018 @ $21,40$129K · 2026-03-06

    Short positioning

    13.4Mshares short-17.2% vs prior
    6.94days to cover
    28.4%short of daily vol
    1fails-to-deliver
    as of 2026-07-15 · short-interest report (free)

    Geographic breakdown

    — missing data
    Listings · one canonical issuer all listings resolve to the canonical
    RARE.OCanonical
    NASDAQ · USD

    Intel & risk

    PredictorBeat prob45 %Surprise-0,00M&A prob3 %Full forecast →
    peak dispatch · —
    OSINT findings
    Dilution riskLow
    Liquidity riskLow
    No immediate filing-based liquidity or dilution flags were detected.
    Relationship graph
    RAREREGN.OEX21:0a5EX21:117EX21:1fdEX21:2e5EX21:30cEX21:3edEX21:429EX21:7d4EX21:890Pharmaceutical
    This companySectorSuppliersSubsidiaries

    Evidence & claims

    From filings & derived data
    • Current ratio (FY 2025-12-31): 2.48xDerived (calculated)
    • Total liabilities (YoY) (2025-12-31 vs 2024-12-31): 29.3%Derived (calculated)
    • Total assets (YoY) (2025-12-31 vs 2024-12-31): 1.9%Derived (calculated)
    • Shareholders' equity (YoY) (2025-12-31 vs 2024-12-31): -131.4%Derived (calculated)
    • Revenue (YoY) (2025-12-31 vs 2024-12-31): 20.2%Derived (calculated)
    • Operating cash flow (YoY) (2025-12-31 vs 2024-12-31): -12.6%Derived (calculated)
    • Net margin (FY 2025-12-31): -85.4%Derived (calculated)
    • Return on equity (FY 2025-12-31): 718.8%Derived (calculated)
    • Return on assets (FY 2025-12-31): -37.5%Derived (calculated)
    • Debt-to-equity (FY 2025-12-31): -20.06xDerived (calculated)
    • Operating income (YoY) (2025-12-31 vs 2024-12-31): 0.2%Derived (calculated)
    • Capex (YoY) (2025-12-31 vs 2024-12-31): -14.3%Derived (calculated)
    • Net income (YoY) (2025-12-31 vs 2024-12-31): -1.1%Derived (calculated)
    • Cash & equivalents (YoY) (2025-12-31 vs 2024-12-31): 142.0%Derived (calculated)
    • R&D expense (YoY) (2025-12-31 vs 2024-12-31): 7.4%Derived (calculated)
    • EPS (basic) (YoY) (2025-12-31 vs 2024-12-31): 7.3%Derived (calculated)
    • EPS (diluted) (YoY) (2025-12-31 vs 2024-12-31): 7.3%Derived (calculated)
    • Current assets (annual): USD 951MSEC XBRL filing
    • Revenue (annual): USD 673MSEC XBRL filing
    • EPS (basic) (annual): USD-PER-SHARES -6SEC XBRL filing
    • Operating cash flow (annual): USD -466MSEC XBRL filing
    • Shares outstanding (annual): 96.6MSEC XBRL filing
    • Total assets (annual): USD 1.53BSEC XBRL filing
    • Current liabilities (annual): USD 384MSEC XBRL filing
    Showing 24 of 60 surfaced claims.
    Atomic claims from regulatory filings and derived calculations. Provenance shown as source kind only.

    The Thread

    Everything we know, in order
    2026-10-28EVENTUpcomingQ3 2026 earnings (expected) estimated date
    2026-08-04 00:00 UTCTRANSCRIPTEarnings call — Q3 2026
    2026-07-01INSIDERHorn Howard sold 4.7k sh — Chief Financial Officer ~$156k
    2026-06-30 03:38 UTCEARNINGSUpcomingForecast: insider_flow_signal (90d)
    2026-06-30 03:38 UTCEARNINGSUpcomingForecast: ma_probability (180d)
    2026-06-24 03:12 UTCHOLDER1 institutional position change flagged (1 major)
    2026-06-21 02:57 UTCHOLDER1 institutional position change flagged (1 major)
    2026-06-15 00:00 UTCINSIDER3 insider transactions — 2026-06 · net sell $207k
    2026-06-15INSIDERSanders Corazon (Corsee) D. sold 2.0k sh — Director ~$50k
    2026-06-15INSIDERParschauer Karah Herdman sold 1.9k sh — EVP and Chief Legal Officer ~$47k
    2026-06-01INSIDERHorn Howard sold 4.7k sh — Chief Financial Officer ~$111k
    2026-05-18 00:00 UTCINSIDER17 insider transactions — 2026-05 · net sell $269k 7 derivative
    2026-05-18FILING8-K filing →
    2026-05-18INSIDERSULIMAN SHEHNAAZ sold 5.7k sh — Director ~$144k
    2026-05-14INSIDERSULIMAN SHEHNAAZ transacted 7.8k sh — Director
    2026-05-14INSIDERFust Matthew K transacted 7.8k sh — Director
    2026-05-14INSIDERRay Amrit transacted 7.8k sh — Director
    2026-05-14INSIDERSanders Corazon (Corsee) D. transacted 7.8k sh — Director
    2026-05-14INSIDERNARACHI MICHAEL transacted 7.8k sh — Director
    2026-05-14INSIDERWELCH DANIEL G transacted 7.8k sh — Director
    2026-05-14INSIDERDunsire Deborah transacted 7.8k sh — Director
    2026-05-14INSIDERRay Amrit acquired 14.1k sh Stock Option (Right to Buy) — Director
    2026-05-14INSIDERSULIMAN SHEHNAAZ acquired 14.1k sh Stock Option (Right to Buy) — Director
    2026-05-14INSIDERFust Matthew K acquired 14.1k sh Stock Option (Right to Buy) — Director
    2026-05-14INSIDERNARACHI MICHAEL acquired 14.1k sh Stock Option (Right to Buy) — Director
    2026-05-14INSIDERWELCH DANIEL G acquired 14.1k sh Stock Option (Right to Buy) — Director
    2026-05-14INSIDERSanders Corazon (Corsee) D. acquired 14.1k sh Stock Option (Right to Buy) — Director
    2026-05-14INSIDERDunsire Deborah acquired 14.1k sh Stock Option (Right to Buy) — Director
    2026-05-06FILING10-Q filing →
    2026-05-05 16:01 UTCEARNINGSQuarterly results — Q1 2026 Revenue USD 136.0M · Net USD -185.0M
    2026-05-05FILING8-K filing →
    2026-05-05INSIDERCrombez Eric sold 344 sh — EVP and Chief Medical Officer ~$9k
    2026-05-01INSIDERHorn Howard sold 4.7k sh — Chief Financial Officer ~$117k
    2026-04-22 00:00 UTCINSIDER17 insider transactions — 2026-04 8 derivative
    2026-04-22INSIDERHuizenga Theodore Alan transacted 20.2k sh — SVP, Chief Accounting Officer
    2026-04-22INSIDERHuizenga Theodore Alan acquired 7.3k sh Stock Option (Right to Buy) — SVP, Chief Accounting Officer
    2026-04-16INSIDERPinion John Richard transacted 23.1k sh — See Remarks
    2026-04-16INSIDERKAKKIS EMIL D transacted 76.7k sh — Director, President & CEO
    2026-04-16INSIDERHuang Dennis Karl transacted 23.1k sh — See Remarks
    2026-04-16INSIDERParschauer Karah Herdman transacted 28.9k sh — EVP and Chief Legal Officer
    Showing the 40 most recent of 75 entries.
    The entity's full life in the product — typed, chronological, joined across Newspaper, Platform and Data. Our memory, made visible.
    Sources filings · IR · transcripts · market data · tier hybrid · as of 2026-08-04 Market data · Issuer disclosures · Public news · Earnings transcripts · Consensus estimates · ESG data · Reference data Premium coverage