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Ultragenyx Pharmaceutical Inc. (RARE)

Ultragenyx Pharmaceutical Inc. is a biopharmaceutical firm dedicated to discovering, developing, and commercializing innovative treatments for rare and ultra-rare genetic conditions. Its operations span North America, Europe, and other international markets. The company's portfolio of marketed biologic products addresses several serious diseases. This includes Crysvita (burosumab), an antibody that targets fibroblast growth factor 23, used to treat X-linked hypophosphatemia and tumor-induced osteomalacia. Mepsevii offers enzyme replacement therapy for both pediatric and adult patients suffering from Mucopolysaccharidosis VII. Dojolvi is available for individuals with long-chain fatty acid oxidation disorders, while Evkeeza (evinacumab) provides a treatment option for homozygous familial hypercholesterolemia. Ultragenyx also boasts a robust pipeline of investigational therapies. Notable candidates include DTX401, an adeno-associated virus 8 (AAV8) gene therapy for glycogen storage disease type Ia, and DTX301, another AAV8 gene therapy aimed at ornithine transcarbamylase deficiency. Other promising developments feature UX143, a human monoclonal antibody for osteogenesis imperfecta; GTX-102, an antisense oligonucleotide designed for Angelman syndrome; UX701, targeting Wilson disease; and UX053, in development for glycogen storage disease type III. To advance its research and development efforts, Ultragenyx Pharmaceutical Inc. maintains strategic collaboration and licensing agreements with numerous partners, such as Kyowa Kirin Co., Ltd., Saint Louis University, REGENXBIO Inc., Bayer Healthcare LLC, GeneTx, Mereo, University of Pennsylvania, Arcturus Therapeutics Holdings Inc., Solid Biosciences Inc., and Daiichi Sankyo Co., Ltd. The company was founded in 2010 and operates from its headquarters in Novato, California.

Sector: Pharmaceutical Preparations

Industry: Biotechnology

Ultragenyx Pharmaceutical Inc. logo

Ultragenyx Pharmaceutical Inc.

NASDAQ:RAREHealthcare
-4.05%$16.10
AH$16.10+0.00%vs close
Mkt Cap$1.59B
Float93.53M
O/S98.59M
Vol
$ Vol
RVOL
Inst Own-
SI21.5%
Earnings
Dilution
Financials Summary
Revenue$673M
Net Income-$575M
Total Assets$1.53B
Free Cash Flow-$472M
Debt & Equity
Debt 107%
Total Debt$1.28B
Total Equity-$80M
Shares Outstanding
Short Interest
Split History

No splits on record

Nothing in RARE's public trading history: no forward or reverse split has affected the share count.

Balance Sheet Details
Assets$1.53B
Liabilities$1.6B
Accounts Receivable$159M
Accounts Payable$31M
Inventory$52M
PP&E (Net)$267M
Goodwill$44M
Intangible Assets$176M
Income Statement
PeriodFY 2025FY 2024FY 2023FY 2022FY 2021FY 2020FY 2019FY 2018FY 2017FY 2016FY 2015FY 2014FY 2013FY 2012FY 2011
CurrencyUSDUSDUSDUSDUSDUSDUSDUSDUSDUSDUSDUSDUSDUSDUSD
Period Ending2025-12-312024-12-312023-12-312022-12-312021-12-312020-12-312019-12-312018-12-312017-12-312016-12-312015-12-312014-12-312013-12-312012-12-312011-12-31
Revenue$673M$560.23M$434.25M$363.33M$351.41M$271.03M$103.71M$51.49M$2.61M$133K$0$0$0$0$0
YoY Growth20.1%29.0%19.5%3.4%29.7%161.3%101.4%1871.5%1863.9%------
Cost of Revenue$109M$76.73M$45.21M$28.32M$16.01M$6.13M$9.01M$1.15M$1K$183.2M$1.38M$0$0$0$0
Gross Profit$564M$483.5M$389.04M$335.01M$335.4M$264.9M$94.71M$50.35M$2.61M-$183.07M-$1.38M$0$0$0$0
Gross Margin83.8%86.3%89.6%92.2%95.4%97.7%91.3%97.8%100.0%-137647.4%-----
Research and Development$750M$697.87M$648.45M$705.79M$497.15M$412.08M$357.36M$294M$231.64M$183.2M$114.74M$45.97M$27.83M$12.64M$4.72M
Selling, General, and Administrative$349M$321.61M$309.8M$278.14M$219.98M$182.93M$161.52M$127.72M$99.91M$64.94M$33M$10.81M$4.45M$3.34M$1.84M
Other$0$0$0$0$0$0$0$3.95M$0$2.17M-$200K$0$0$0-$22K
Total Operating Expenses$1.1B$1.02B$958.25M$983.93M$717.13M$595.02M$518.88M$421.72M$331.55M$248.14M$147.74M$56.78M$32.28M$15.98M$6.56M
Operating Income (EBIT)-$535M-$535.97M-$569.21M-$648.92M-$381.74M-$330.12M-$424.17M-$371.37M-$328.94M-$248.01M-$147.74M-$56.78M-$32.28M-$15.98M-$6.56M
Operating Margin-79.5%-95.7%-131.1%-178.6%-108.6%-121.8%-409.0%-721.2%-12593.5%-186471.4%-----
Interest Expense$62M$63.04M$66M$43.02M$29.42M$33.29M$1.14M$0$0$0$0$0$0$0$270K
Interest Income$25M$36.51M$26.69M$11.07M$1.93M$7.04M$13.24M$9.54M$4.07M$3.79M$2.32M$608K$216K$1K$4K
Other Income (Expense)-$36M-$31.61M-$39.26M-$52.81M-$71.24M$144.76M$24.73M$174.28M$10.6M$2.17M$2.12M-$3.02M-$2.79M-$349K-$288K
Income Before Tax (EBT)-$571M-$567.59M-$608.46M-$701.73M-$452.98M-$185.36M-$399.44M-$197.1M-$318.34M-$245.84M-$145.62M-$59.8M-$35.07M-$16.33M-$6.85M
Income Tax Expense$4M$1.6M-$1.82M$5.7M$1.04M$1.21M$3.28M$514K-$16.2M$35K$0$0$0$0$0
Net Income-$575M-$569.18M-$606.64M-$707.42M-$454.02M-$186.57M-$402.73M-$197.61M-$302.14M-$245.87M-$145.62M-$59.8M-$35.07M-$16.33M-$6.85M
Net Income Margin-85.4%-101.6%-139.7%-194.7%-129.2%-68.8%-388.3%-383.7%-11567.3%-184867.7%-----
Supplementary Data
EPS-5.83-6.29-8.25-10.12-6.70-3.07-7.12-3.97-7.12-6.21-3.96-2.08-1.21-0.71-0.30
Diluted EPS-5.83-6.29-8.25-10.12-6.70-3.07-7.12-3.97-7.12-6.21-3.96-2.08-1.21-0.71-0.30
Weighted Average OS98.60M90.54M73.54M69.91M67.80M60.85M56.58M49.78M42.45M39.59M36.78M28.76M29.06M23.06M23.06M
EBITDA-$474M-$469M-$516.45M-$640.49M-$410.32M-$139.81M-$389.77M-$177.56M-$312.51M-$244.58M-$146.35M-$56.09M-$34.63M-$16.02M-$6.54M
EBITDA Margin-70.4%-83.7%-118.9%-176.3%-116.8%-51.6%-375.8%-344.8%-11964.5%-183897.0%-----
Balance Sheet
PeriodFY 2025FY 2024FY 2023FY 2022FY 2021FY 2020FY 2019FY 2018FY 2017FY 2016FY 2015FY 2014FY 2013FY 2012FY 2011
CurrencyUSDUSDUSDUSDUSDUSDUSDUSDUSDUSDUSDUSDUSDUSDUSD
Period Ending2025-12-312024-12-312023-12-312022-12-312021-12-312020-12-312019-12-312018-12-312017-12-312016-12-312015-12-312014-12-312013-12-312012-12-312011-12-31
Assets
Current Assets
Cash and Cash Equivalents$434M$173.73M$213.58M$132.94M$307.58M$713.53M$433.58M$113.43M$100.49M$161.12M$93.57M$24.32M$7.43M$86.19M$10.64M
Short-Term Investments$259M$436.3M$363.63M$614.82M$432.61M$488.01M$321.65M$346.27M$134M$219.03M$343.43M$163.16M$45.95M$0$0
Cash and Short-Term Investments$693M$610.02M$577.21M$747.76M$740.2M$1.2B$755.23M$459.71M$244.47M$498.11M$437M$187.49M$53.38M$86.19M$10.64M
Accounts Receivable$159M$121.8M$73.39M$40.45M$44.43M$23.09M$32.84M$12.74M$5.17M$0$0$0$0$0$103K
Inventory$52M$45.01M$33.97M$26.77M$16.23M$13.05M$11.55M$7.07M$757K$0$0$0$0$0$0
Other Current Assets$47M$40.29M$47.62M$68.93M$55.74M$0$0$0$0$1.41M$150K$5.93M$0$0$0
Total Current Assets$951M$817.12M$732.18M$883.9M$856.6M$1.3B$851.02M$522.37M$244.47M$498.11M$450.21M$187.49M$55.23M$86.44M$10.95M
Long-Term Assets
Property, Plant & Equipment Net$267M$265.93M$290.57M$259.73M$176.18M$114.04M$74.68M$20.05M$21.84M$17.05M$7.37M$3.03M$1.32M$1.36M$759K
Goodwill$44M$44.41M$44.41M$44.41M$44.41M$44.41M$44.41M$44.41M$44.41M$0$0$0$0$0$0
Intangible Assets$176M$178.31M$166.27M$160.1M$130.79M$131.11M$129M$129.22M$141.54M$0$0$0$0$0$0
Long-Term Investments$59M$135M$199.9M$148.97M$293.86M$165.88M$32.93M$1.82M$9.97M$117.96M$99.26M$0$0$0$0
Deferred Tax Assets$0$0$0$0$0$0$0-$1.82M$25.11M$0$0$0$0$0$0
Other Long-Term Assets$35M$62.68M$57.69M$48.34M$20.56M$8.81M$3.47M$3.51M$3.41M-$92.5M$2.73M$7.45M$3.1M$509K$417K
Total Long-Term Assets$581M$686.33M$758.83M$661.54M$665.79M$464.25M$284.48M$197.19M$246.28M$42.52M$109.36M$10.48M$4.42M$1.87M$1.18M
Other Assets$0$0$0$0$0$0$0$0$0$0$0$0$0$0$0
Total Assets$1.53B$1.5B$1.49B$1.55B$1.52B$1.76B$1.14B$719.56M$490.75M$540.63M$559.57M$197.97M$59.65M$88.32M$12.13M
Liabilities
Current Liabilities
Accounts Payable$31M$38.76M$42.11M$43.27M$17.14M$12.92M$12.87M$12.28M$8.89M$5.36M$2.94M$4.86M$1.44M$1.2M$342K
Short-Term Debt$81M$0$0$0$0$0$0$0$0$0$0$0$0$0$0
Current Income Tax Payable$0$0$0$0$0$0$0$0$0$0$0$0$0$0$0
Current Unearned Revenue$0$0$0$1.48M$7.61M$59.22M$0$0$5.99M$0$0$0$0$0$0
Other Current Liabilities$183M$87.47M$142.79M$0$0$0$0$0$0$37.67M$15.55M$85K$78K$75K$0
Total Current Liabilities$384M$344.15M$280.44M$261.21M$181.37M$189.61M$103.3M$74.72M$71.01M$60.26M$27.92M$12.52M$5.92M$3.19M$999K
Long-Term Liabilities
Long-Term Debt$1.17B$0$0$0$0$0$0$0$0$0$0$0$0$0$0
Long-Term Unearned Revenue$0$0$0$0$1.47M$7.35M$0$0$0$0$0$0$0$0$0
Long-Term Deferred Tax Liabilities$30M$30.06M$30.06M$31.67M$33.31M$33.31M$33.31M$31.17M$31.17M$0$0$0$0$0$0
Other Long-Term Liabilities-$4M$836.91M$874.53M$880.26M$352.79M$335.67M$315.37M$4.76M$5.12M$6.39M$561K$505K$3.62M$788K$487K
Total Long-Term Liabilities$1.22B$897.01M$935.16M$931.74M$418.47M$415.57M$378.43M$35.92M$36.28M$6.39M$561K$505K$3.62M$788K$487K
Other Liabilities$0$0$0$0$0$0$0$0$0$0$0$0$0$0$0
Total Liabilities$1.6B$1.24B$1.22B$1.19B$599.84M$605.18M$481.73M$110.65M$107.3M$66.65M$28.48M$13.02M$9.54M$3.98M$1.49M
Stockholders' Equity
Preferred Stock$0$0$0$0$0$0$0$0$0$95K$0$0$124.93M$111.39M$18.6M
Common Stock$0$92K$82K$70K$69K$67K$58K$51K$44K$41K$39K$32K$4K$3K$3K
Retained Earnings-$4.53B-$3.96B-$3.39B-$2.78B-$2.07B-$1.62B-$1.43B-$1.03B-$832.67M-$530.53M-$284.66M-$139.04M-$74.84M-$27.06M-$8.15M
Comprehensive Income & Other$1M-$643K$647K-$6.57M-$1.4M$689K-$147K-$633K-$5.68M$905K-$868K-$174K$11K-$353K-$40K
Other Stockholders' Equity$4.45B$4.21B$3.66B$3.13B$3B$2.77B$2.09B$1.64B$1.22B$1B$815.71M$323.95M$124.94M$111.39M$18.8M
Total Equity-$80M$255.3M$275.41M$352.49M$922.56M$1.15B$653.76M$608.91M$383.45M$473.97M$531.09M$184.94M$50.11M$84.34M$10.64M
Minority Interest$7M$7M$0$0$0$0$0$0$0$0$0$0$0$0$0
Total Liabilities and Equity$1.52B$1.5B$1.49B$1.55B$1.52B$1.76B$1.14B$719.56M$490.75M$540.63M$559.57M$197.97M$59.65M$88.32M$12.13M
Supplementary Data
Total Debt$1.28B$40.34M$43.17M$31.59M$41.97M$48.23M$36.99M$0$0$0$0$0$0$0$0
Net Debt$842M-$133.39M-$170.41M-$101.35M-$265.61M-$665.3M-$396.59M-$113.43M-$100.49M-$161.12M-$93.57M-$24.32M-$7.43M-$86.19M-$10.64M
Cash Flow Statement
PeriodFY 2025FY 2024FY 2023FY 2022FY 2021FY 2020FY 2019FY 2018FY 2017FY 2016FY 2015FY 2014FY 2013FY 2012FY 2011
CurrencyUSDUSDUSDUSDUSDUSDUSDUSDUSDUSDUSDUSDUSDUSDUSD
Period Ending2025-12-312024-12-312023-12-312022-12-312021-12-312020-12-312019-12-312018-12-312017-12-312016-12-312015-12-312014-12-312013-12-312012-12-312011-12-31
Operating Activities
Net Income-$575M-$569.18M-$606.64M-$707.42M-$454.02M-$186.57M-$402.73M-$197.61M-$302.14M-$245.87M-$145.62M-$59.8M-$35.07M-$16.33M-$6.85M
Depreciation & Amortization$35M$35.54M$26.01M$18.22M$13.24M$12.26M$8.54M$19.54M$5.83M$3.42M$1.38M$684K$444K$313K$34K
Deferred Income Tax$0$0-$1.62M$0$48.67M-$169.78M-$18.94M-$167.65M-$16.25M$6.16M$0$0$0$0$13K
Stock-Based Compensation$153M$158.03M$135.21M$130.38M$104.95M$85.73M$82M$80.11M$68.01M$48.31M$24.88M$5.39M$657K$891K$254K
Changes in Working Capital:
  Accounts Receivable-$31M-$33.6M-$22.78M-$12.07M-$5.43M$9.84M-$20.1M-$7.58M-$5.17M$0$0$0$0$0$0
  Inventory-$6M-$11.21M-$6.93M-$9.7M-$3.12M-$1.35M-$4.45M-$5.28M-$757K$0$0$0$0$0$0
  Accounts Payable$24M$46.99M$2.38M$79.84M$2.81M$29.6M$20.65M$3.37M$3.46M$2.5M-$1.98M$3.18M$237K$858K$252K
  Other Working Capital-$10M$11.73M$12.23M$2.16M-$57.49M$66.57M-$12.04M-$15.3M-$23.69M$23.8M$9.72M-$1.01M-$1.78M$1.47M$201K
Total Working Capital Change-$23M$13.92M-$15.1M$60.23M-$63.24M$104.66M-$15.94M-$24.79M-$20.23M$26.3M$7.74M$2.17M-$1.54M$2.32M$453K
Other Non-Cash Items-$56M-$52.5M-$12.67M$118.12M$11.71M$21.46M$1.69M-$156K$10.93M$700K$5.64M$6.92M$4.31M$302K$270K
Net Cash from Operating-$466M-$414.19M-$474.81M-$380.46M-$338.69M-$132.22M-$345.38M-$290.57M-$253.84M-$160.97M-$105.98M-$44.63M-$31.2M-$12.5M-$5.83M
Investing Activities
Capital Expenditures-$6M-$7.49M-$44.27M-$116.12M-$73.09M-$43.91M-$24.83M-$4.08M-$2.79M-$10.19M-$4.96M-$2.15M-$407K-$1.09M-$548K
Acquisitions$0$0$0-$75.03M$0$0-$11.79M$0-$142.8M$0$0$0$0$0$0
Purchases of Investments-$186M-$408.61M-$526.38M-$614.74M-$1.01B-$850.3M-$707.16M-$509.8M-$230.49M-$442.49M-$624.23M-$208.97M-$63.95M$0$0
Sales of Investments$444M$413.27M$746.2M$545.08M$890.85M$720.64M$718.96M$310.22M$431.57M$543.79M$338.37M$87.97M$16.6M$0$0
Other Investing Activities-$16M-$14.94M-$7.55M-$30.84M-$942K-$5.55M$11.79M$170.32M$934K-$1.2M-$1.54M-$293K$25K-$100K-$376K
Net Cash from Investing$236M-$17.77M$168M-$291.65M-$195.37M-$179.12M-$13.04M-$33.33M$56.42M$89.92M-$292.35M-$123.44M-$47.73M-$1.19M-$924K
Financing Activities
Debt Repaid-$392M-------$4.94M-----$100K-$2.45M
Common Stock Issuance$86M$380.98M$379.75M$0$78.94M$511.21M$355.24M$309.02M$131.96M$79.49M$467.57M$189.32M$171K$131K$0
Common Stock Repurchases$0$0$0$0$0$0$0$0$0$0$0$0$0$0$0
Dividends Paid$0$0$0$0$0$0$0$0$0$0$0$0$0$0$0
Other Financing Activities$0$18.26M$8.39M$501.21M$39.61M$89.06M$324.06M$27.83M$9.25M$59.19M$6.44M$736K$0$0$0
Net Cash from Financing$478M$399.24M$388.14M$501.21M$118.55M$600.27M$679.31M$336.85M$136.27M$138.68M$467.57M$184.97M$171K$89.24M$17.33M
Forex Effect$5.46M-$2.52M$462K-$1.07M-$1.19M$1.12M-$165K-$472K$528K-$65K$0$0$0$0$0
Net Change in Cash$253.47M-$35.24M$81.8M-$171.98M-$416.71M$290.05M$320.72M$12.48M-$60.63M$67.55M$69.25M$16.9M-$78.76M$75.55M$10.58M
Supplementary Data
Operating Cash Flow-$466M-$414.19M-$474.81M-$380.46M-$338.69M-$132.22M-$345.38M-$290.57M-$253.84M-$160.97M-$105.98M-$44.63M-$31.2M-$12.5M-$5.83M
Capital Expenditure-$6M-$7.49M-$46.77M-$146.12M-$73.09M-$43.91M-$24.83M-$4.08M-$2.79M-$10.19M-$4.96M-$2.15M-$407K-$1.09M-$548K
Free Cash Flow-$472M-$421.68M-$521.57M-$526.59M-$411.79M-$176.13M-$370.21M-$294.64M-$256.64M-$171.16M-$110.93M-$46.78M-$31.61M-$13.6M-$6.37M
Cash at Beginning$184.16M$219.4M$137.6M$309.58M$726.29M$436.24M$115.53M$103.04M$161.12M$93.57M$24.32M$7.43M$86.19M$10.64M$65K
Cash at End$436M$184.16M$219.4M$137.6M$309.58M$726.29M$436.24M$115.53M$100.49M$161.12M$93.57M$24.32M$7.43M$86.19M$10.64M