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Ultragenyx Pharmaceutical NasdaqGS:RARE Stock Report

Last Price


Market Cap







30 Sep, 2022


Company Financials +
RARE fundamental analysis
Snowflake Score
Future Growth2/6
Past Performance0/6
Financial Health6/6

RARE Stock Overview

Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Europe, and internationally.

Ultragenyx Pharmaceutical Competitors

Price History & Performance

Summary of all time highs, changes and price drops for Ultragenyx Pharmaceutical
Historical stock prices
Current Share PriceUS$41.41
52 Week HighUS$89.72
52 Week LowUS$39.55
1 Month Change-13.17%
3 Month Change-33.90%
1 Year Change-52.84%
3 Year Change-6.99%
5 Year Change-23.75%
Change since IPO-1.99%

Recent News & Updates

Sep 02

Ultragenyx Pharmaceuticals: Downward Trajectory Likely To Continue

Summary Ultragenyx has built a remunerative, albeit not yet profitable, business around treating patients with orphan and ultra-orphan diseases. The problem from an investor's viewpoint is its outsized expense profile. Ultragenyx has an impressive pipeline and strong liquidity. This is my first look at Ultragenyx Pharmaceuticals (RARE), a little biotech with a long name and an active business. Its specific niche of orphan and ultra-orphan diseases presents extra challenges for the company and its shareholders as I will hereafter discuss. Ultragenyx has significant reliable revenue streams The initial challenge presented by Ultragenyx is to wrap one's head around its area of specialization. The following excerpt from the company profile describing its therapies is illustrative: Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii [VESTRONIDASE ALFA], an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII [Sly Syndrome] and; Dojolvi [UX007/TRIHEPTANOIN] for treating long-chain fatty acid oxidation disorders [LC-FAOD]... The FDA first approved Mepsevii in 11/2017. Less than 100 cases have been reported in the United States; worldwide prevalence is estimated at ~1:250,000 births. The FDA awarded it with a Rare Pediatric Disease Priority Review Voucher [PRV], which it promptly sold to Novartis (NVS) for $130 million. Shortly thereafter, Crysvita was first approved by the FDA in 04/2018 to treat X-linked hypophosphatemia [XLH]. XLH is a rare (prevalence ~1:20,000) inherited form of rickets for which vitamin D therapy is ineffective. In its latest 10-Q it estimates that there are ~48,000 patients with XLH in the developed world. Later in 06/2022, the FDA approved Crysvita to treat tumor-induced osteomalacia [TIO]. TIO is exceptionally rare. One article notes that ~1,000 cases of TIO have been reported worldwide. Its extreme rarity contributes to the likelihood of misdiagnosis. The FDA approved Dojolvi in treatment of LC-FAOD in 06/2020. Ultragenyx estimates that it afflicts between 2,000-3,500 people in the US. Ultragenyx's Q2, 2022 10-Q lists the following disaggregated revenues during its 3 and 6 month periods ending 06/30/2021 and 2022: Ultragenyx's interest in Crysvita (KRN23) arose out of a 2013 collaboration with Kyowa Hakko Kirin Co., Ltd. (KYKOY) for it to develop and commercialize KRN23. At the time Kyowa Hakko Kirin was completing a Phase 1/2 study in adults with XLH in the US and Canada. During its Q2, 2022 earnings call (the "Call"), CCO Harris advised that Crysvita revenues to date gave it confidence to guide for full year 2022 Crysvita revenues of $250 to $260 million in Ultragenyx territories. He further confirmed prior guidance for $55 to $65 million in Dojolvi revenues. He offered no Mepsevii guidance. Ultragenyx expenses far outstrip its revenues. Ultragenyx's balance between its revenue from customers and its operating expenses is sadly askew. Its quarterly expenses are shown below: Just a glance shows how its nice quarterly revenues of ~$89 million discussed above are totally overmatched by operating expenses of ~$231 million. All three categories of expenses shot up in Q2, 2022 compared to Q2, 2021, cost of sales by ~164%, R&D by ~36%, SG&A by ~28%. In the aggregate expenses were up by ~36%. One cannot go to the grocery store without lamenting about inflation; the problem seems to have hit Ultragenyx with extra force. CFO Dier's presentation during the Call provides helpful color on the expected shape of future expenses, although she does not provide an expense guidance. She noted: ... 2022 is a peak burn year for us as we have initiated multiple late-stage clinical programs in-licensed Evkeeza [Evinacumab-dgnb] completed the acquisition of [GeneTx] and are completing the build-out of our gene therapy manufacturing facility. In 2023, we don't anticipate additional one-time events of this nature or large capital expenditures and we anticipate SG&A will decrease compared to 2022 as we transition US and Canadian commercialization responsibilities for Crysvita to KKC. We will continue to invest in our clinical and preclinical programs as discussed, and the overall net effect across the company then will be a decrease in net cash burn. The referenced Evkeeza in-license refers to its 01/2022 deal with Regeneron (REGN) to clinically develop, commercialize and distribute Evkeeza (evinacumab-dgnb) ex-US. The deal called for $30 million upfront with $63 million in milestones. The GeneTx acquisition refers to Ultragenyx's 07/2022 $75 million upfront exercise of its option to acquire its partner GeneTx; GeneTx had just reported encouraging interim data from its open-label, dose-escalating Phase 1/2 study of GTX-102 for the treatment of Angelman syndrome [AS]. During the Call there were a variety of questions showing great interest in the AS program. CEO Kakkis minced no words in expressing support for this program. He stated that Ultragenyx was "all in" on it. He enthused that:

Shareholder Returns

RAREUS BiotechsUS Market

Return vs Industry: RARE underperformed the US Biotechs industry which returned -25.8% over the past year.

Return vs Market: RARE underperformed the US Market which returned -21.5% over the past year.

Price Volatility

Is RARE's price volatile compared to industry and market?
RARE volatility
RARE Average Weekly Movement7.1%
Biotechs Industry Average Movement11.1%
Market Average Movement6.9%
10% most volatile stocks in US Market15.6%
10% least volatile stocks in US Market2.8%

Stable Share Price: RARE is not significantly more volatile than the rest of US stocks over the past 3 months, typically moving +/- 7% a week.

Volatility Over Time: RARE's weekly volatility (7%) has been stable over the past year.

About the Company

20101,119Emil Kakkis

Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Europe, and internationally. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for treating long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company’s products candidatures include DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome; UX701, for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III.

Ultragenyx Pharmaceutical Fundamentals Summary

How do Ultragenyx Pharmaceutical's earnings and revenue compare to its market cap?
RARE fundamental statistics
Market CapUS$2.90b
Earnings (TTM)-US$505.94m
Revenue (TTM)US$334.31m


P/S Ratio


P/E Ratio

Earnings & Revenue

Key profitability statistics from the latest earnings report
RARE income statement (TTM)
Cost of RevenueUS$527.96m
Gross Profit-US$193.65m
Other ExpensesUS$312.29m

Last Reported Earnings

Jun 30, 2022

Next Earnings Date


Earnings per share (EPS)-7.22
Gross Margin-57.92%
Net Profit Margin-151.34%
Debt/Equity Ratio0%

How did RARE perform over the long term?

See historical performance and comparison
We’ve recently updated our valuation analysis.


Is RARE undervalued compared to its fair value, analyst forecasts and its price relative to the market?

Valuation Score


Valuation Score 2/6

  • Price-To-Sales vs Peers

  • Price-To-Sales vs Industry

  • Price-To-Sales vs Fair Ratio

  • Below Fair Value

  • Significantly Below Fair Value

  • Analyst Forecast

Key Valuation Metric

Which metric is best to use when looking at relative valuation for RARE?

Other financial metrics that can be useful for relative valuation.

RARE key valuation metrics and ratios. From Price to Earnings, Price to Sales and Price to Book to Price to Earnings Growth Ratio, Enterprise Value and EBITDA.
Key Statistics
Enterprise Value/Revenue6.7x
Enterprise Value/EBITDA-4.9x
PEG Ration/a

Price to Sales Ratio vs Peers

How does RARE's PS Ratio compare to its peers?

RARE PS Ratio vs Peers
The above table shows the PS ratio for RARE vs its peers. Here we also display the market cap and forecasted growth for additional consideration.
CompanyPSEstimated GrowthMarket Cap
Peer Average14x
TWST Twist Bioscience
CYTK Cytokinetics
FOLD Amicus Therapeutics
PTCT PTC Therapeutics
RARE Ultragenyx Pharmaceutical

Price-To-Sales vs Peers: RARE is good value based on its Price-To-Sales Ratio (8.7x) compared to the peer average (14x).

Price to Earnings Ratio vs Industry

How does RARE's PE Ratio compare vs other companies in the US Biotechs Industry?

Price-To-Sales vs Industry: RARE is good value based on its Price-To-Sales Ratio (8.7x) compared to the US Biotechs industry average (13.2x)

Price to Sales Ratio vs Fair Ratio

What is RARE's PS Ratio compared to its Fair PS Ratio? This is the expected PS Ratio taking into account the company's forecast earnings growth, profit margins and other risk factors.

RARE PS Ratio vs Fair Ratio.
Fair Ratio
Current PS Ratio8.7x
Fair PS Ratio2.2x

Price-To-Sales vs Fair Ratio: RARE is expensive based on its Price-To-Sales Ratio (8.7x) compared to the estimated Fair Price-To-Sales Ratio (2.2x).

Share Price vs Fair Value

What is the Fair Price of RARE when looking at its future cash flows? For this estimate we use a Discounted Cash Flow model.

Below Fair Value: Insufficient data to calculate RARE's fair value for valuation analysis.

Significantly Below Fair Value: Insufficient data to calculate RARE's fair value for valuation analysis.

Analyst Price Targets

What is the analyst 12-month forecast and do we have any statistical confidence in the consensus price target?

Analyst Forecast: Target price is more than 20% higher than the current share price, but analysts are not within a statistically confident range of agreement.

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Future Growth

How is Ultragenyx Pharmaceutical forecast to perform in the next 1 to 3 years based on estimates from 16 analysts?

Future Growth Score


Future Growth Score 2/6

  • Earnings vs Savings Rate

  • Earnings vs Market

  • High Growth Earnings

  • Revenue vs Market

  • High Growth Revenue

  • Future ROE


Forecasted annual earnings growth

Earnings and Revenue Growth Forecasts

Analyst Future Growth Forecasts

Earnings vs Savings Rate: RARE is forecast to remain unprofitable over the next 3 years.

Earnings vs Market: RARE is forecast to remain unprofitable over the next 3 years.

High Growth Earnings: RARE is forecast to remain unprofitable over the next 3 years.

Revenue vs Market: RARE's revenue (26.6% per year) is forecast to grow faster than the US market (7.6% per year).

High Growth Revenue: RARE's revenue (26.6% per year) is forecast to grow faster than 20% per year.

Earnings per Share Growth Forecasts

Future Return on Equity

Future ROE: RARE is forecast to be unprofitable in 3 years.

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Past Performance

How has Ultragenyx Pharmaceutical performed over the past 5 years?

Past Performance Score


Past Performance Score 0/6

  • Quality Earnings

  • Growing Profit Margin

  • Earnings Trend

  • Accelerating Growth

  • Earnings vs Industry

  • High ROE


Historical annual earnings growth

Earnings and Revenue History

Quality Earnings: RARE is currently unprofitable.

Growing Profit Margin: RARE is currently unprofitable.

Past Earnings Growth Analysis

Earnings Trend: RARE is unprofitable, and losses have increased over the past 5 years at a rate of 11.3% per year.

Accelerating Growth: Unable to compare RARE's earnings growth over the past year to its 5-year average as it is currently unprofitable

Earnings vs Industry: RARE is unprofitable, making it difficult to compare its past year earnings growth to the Biotechs industry (29.2%).

Return on Equity

High ROE: RARE has a negative Return on Equity (-74.61%), as it is currently unprofitable.

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Financial Health

How is Ultragenyx Pharmaceutical's financial position?

Financial Health Score


Financial Health Score 6/6

  • Short Term Liabilities

  • Long Term Liabilities

  • Debt Level

  • Reducing Debt

  • Stable Cash Runway

  • Forecast Cash Runway

Financial Position Analysis

Short Term Liabilities: RARE's short term assets ($678.5M) exceed its short term liabilities ($220.6M).

Long Term Liabilities: RARE's short term assets ($678.5M) exceed its long term liabilities ($421.9M).

Debt to Equity History and Analysis

Debt Level: RARE is debt free.

Reducing Debt: RARE has not had any debt for past 5 years.

Balance Sheet

Cash Runway Analysis

For companies that have on average been loss making in the past we assess whether they have at least 1 year of cash runway.

Stable Cash Runway: RARE has sufficient cash runway for more than a year based on its current free cash flow.

Forecast Cash Runway: RARE has sufficient cash runway for 1.5 years if free cash flow continues to reduce at historical rates of 12.2% each year.

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What is Ultragenyx Pharmaceutical current dividend yield, its reliability and sustainability?

Dividend Score


Dividend Score 0/6

  • Notable Dividend

  • High Dividend

  • Stable Dividend

  • Growing Dividend

  • Earnings Coverage

  • Cash Flow Coverage

Dividend Yield vs Market

Ultragenyx Pharmaceutical Dividend Yield vs Market
How does Ultragenyx Pharmaceutical dividend yield compare to the market?
SegmentDividend Yield
Company (Ultragenyx Pharmaceutical)n/a
Market Bottom 25% (US)1.7%
Market Top 25% (US)4.7%
Industry Average (Biotechs)2.8%
Analyst forecast in 3 Years (Ultragenyx Pharmaceutical)0%

Notable Dividend: Unable to evaluate RARE's dividend yield against the bottom 25% of dividend payers, as the company has not reported any recent payouts.

High Dividend: Unable to evaluate RARE's dividend yield against the top 25% of dividend payers, as the company has not reported any recent payouts.

Stability and Growth of Payments

Stable Dividend: Insufficient data to determine if RARE's dividends per share have been stable in the past.

Growing Dividend: Insufficient data to determine if RARE's dividend payments have been increasing.

Earnings Payout to Shareholders

Earnings Coverage: Insufficient data to calculate payout ratio to determine if its dividend payments are covered by earnings.

Cash Payout to Shareholders

Cash Flow Coverage: Unable to calculate sustainability of dividends as RARE has not reported any payouts.

Discover strong dividend paying companies


How experienced are the management team and are they aligned to shareholders interests?


Average management tenure


Emil Kakkis (62 yo)





Dr. Emil D. Kakkis, MD, Ph.D. is Founder for Ultragenyx Pharmaceutical Inc. He has been the Chief Executive Officer and President of Ultragenyx Pharmaceutical Inc. since April 2010. Dr. Kakkis founded Ultr...

CEO Compensation Analysis

Emil Kakkis's Compensation vs Ultragenyx Pharmaceutical Earnings
How has Emil Kakkis's remuneration changed compared to Ultragenyx Pharmaceutical's earnings?
DateTotal Comp.SalaryCompany Earnings
Jun 30 2022n/an/a


Mar 31 2022n/an/a


Dec 31 2021US$10mUS$769k


Sep 30 2021n/an/a


Jun 30 2021n/an/a


Mar 31 2021n/an/a


Dec 31 2020US$5mUS$759k


Sep 30 2020n/an/a


Jun 30 2020n/an/a


Mar 31 2020n/an/a


Dec 31 2019US$7mUS$714k


Sep 30 2019n/an/a


Jun 30 2019n/an/a


Mar 31 2019n/an/a


Dec 31 2018US$5mUS$680k


Sep 30 2018n/an/a


Jun 30 2018n/an/a


Mar 31 2018n/an/a


Dec 31 2017US$7mUS$638k


Sep 30 2017n/an/a


Jun 30 2017n/an/a


Mar 31 2017n/an/a


Dec 31 2016US$4mUS$567k


Sep 30 2016n/an/a


Jun 30 2016n/an/a


Mar 31 2016n/an/a


Dec 31 2015US$5mUS$535k


Compensation vs Market: Emil's total compensation ($USD9.52M) is above average for companies of similar size in the US market ($USD6.85M).

Compensation vs Earnings: Emil's compensation has increased whilst the company is unprofitable.

Leadership Team

Experienced Management: RARE's management team is seasoned and experienced (7.2 years average tenure).

Board Members

Experienced Board: RARE's board of directors are considered experienced (6.5 years average tenure).


Who are the major shareholders and have insiders been buying or selling?

Insider Trading Volume

Insider Buying: Insufficient data to determine if insiders have bought more shares than they have sold in the past 3 months.

Recent Insider Transactions

NasdaqGS:RARE Recent Insider Transactions by Companies or Individuals
DateValueNameEntityRoleSharesMax Price
19 Jun 22SellUS$16,160Erik HarrisIndividual295US$54.78
16 Apr 22SellUS$11,161Theodore HuizengaIndividual132US$84.55
11 Mar 22SellUS$688,930Thomas KassbergIndividual10,281US$67.01
07 Mar 22SellUS$323,563Karah ParschauerIndividual5,140US$62.95
01 Mar 22SellUS$158,156Thomas KassbergIndividual2,374US$66.62
01 Mar 22SellUS$56,161Theodore HuizengaIndividual843US$66.62
01 Mar 22SellUS$35,575Mardi DierIndividual534US$66.62
01 Mar 22SellUS$187,935Camille BedrosianIndividual2,821US$66.62
01 Mar 22SellUS$192,265Karah ParschauerIndividual2,886US$66.62
01 Mar 22SellUS$215,449Dennis HuangIndividual3,234US$66.62
01 Mar 22SellUS$299,657John PinionIndividual4,498US$66.62
01 Mar 22SellUS$180,740Erik HarrisIndividual2,713US$66.62
30 Jan 22SellUS$109,859Camille BedrosianIndividual1,657US$66.30
14 Oct 21SellUS$29,048John PinionIndividual358US$81.14
14 Oct 21SellUS$25,153Karah ParschauerIndividual310US$81.14
14 Oct 21SellUS$23,206Thomas KassbergIndividual286US$81.14
14 Oct 21SellUS$24,585Erik HarrisIndividual303US$81.14
14 Oct 21SellUS$29,129Camille BedrosianIndividual359US$81.14
14 Oct 21SellUS$11,360Theodore HuizengaIndividual140US$81.14
14 Oct 21SellUS$29,048Dennis HuangIndividual358US$81.14
12 Oct 21SellUS$287,861Mardi DierIndividual3,477US$82.79

Ownership Breakdown

What is the ownership structure of RARE?
Owner TypeNumber of SharesOwnership Percentage
State or Government28,2430.04%
General Public3,254,4914.6%
Individual Insiders3,318,2304.7%

Dilution of Shares: Shareholders have been diluted in the past year, with total shares outstanding growing by 3.2%.

Top Shareholders

Top 25 shareholders own 75.46% of the company
OwnershipNameSharesCurrent ValueChange %Portfolio %
The Vanguard Group, Inc.
T. Rowe Price Group, Inc.
Capital Research and Management Company
Wellington Management Group LLP
Federated Hermes, Inc.
BlackRock, Inc.
3,597,584$149.0m4.56%no data
ClearBridge Investments, LLC
Emil Kakkis
2,764,926$114.5m0%no data
State Street Global Advisors, Inc.
Alkeon Capital Management, LLC
Avidity Partners Management, L.P.
RTW Investments, LP
AllianceBernstein L.P.
1,158,110$48.0m-7.75%no data
Macquarie Investment Management Business Trust
Rock Springs Capital Management LP
Citadel Advisors LLC
Geode Capital Management, LLC
908,713$37.6m0.64%no data
BNY Mellon Asset Management
Cadian Capital Management, LP
Avoro Capital Advisors LLC
Adage Capital Management, L.P.
Norges Bank Investment Management
621,576$25.7m0%no data
Goldman Sachs Asset Management, L.P.
First Trust Advisors LP

Company Information

Ultragenyx Pharmaceutical Inc.'s employee growth, exchange listings and data sources

Key Information

  • Name: Ultragenyx Pharmaceutical Inc.
  • Ticker: RARE
  • Exchange: NasdaqGS
  • Founded: 2010
  • Industry: Biotechnology
  • Sector: Pharmaceuticals & Biotech
  • Implied Market Cap: US$2.900b
  • Shares outstanding: 70.04m
  • Website:

Number of Employees


  • Ultragenyx Pharmaceutical Inc.
  • 60 Leveroni Court
  • Novato
  • California
  • 94949
  • United States


TickerExchangePrimary SecuritySecurity TypeCountryCurrencyListed on
RARENasdaqGS (Nasdaq Global Select)YesCommon StockUSUSDJan 2014
UP0DB (Deutsche Boerse AG)YesCommon StockDEEURJan 2014
0LIFLSE (London Stock Exchange)YesCommon StockGBUSDJan 2014
RARE *BMV (Bolsa Mexicana de Valores)YesCommon StockMXMXNJan 2014
R2AR34BOVESPA (Bolsa de Valores de Sao Paulo)BDR EACH 10 REPR 1 COMBRBRLJan 2022

Company Analysis and Financial Data Status

All financial data provided by Standard & Poor's Capital IQ.
DataLast Updated (UTC time)
Company Analysis2022/09/30 00:00
End of Day Share Price2022/09/30 00:00
Annual Earnings2021/12/31

Unless specified all financial data is based on a yearly period but updated quarterly. This is known as Trailing Twelve Month (TTM) or Last Twelve Month (LTM) Data. Learn more here.