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
|Historical stock prices|
|Current Share Price||US$41.41|
|52 Week High||US$89.72|
|52 Week Low||US$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
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: seekingalpha.com 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: seekingalpha.com seekingalpha.com 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:
|RARE||US Biotechs||US 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.
|RARE Average Weekly Movement||7.1%|
|Biotechs Industry Average Movement||11.1%|
|Market Average Movement||6.9%|
|10% most volatile stocks in US Market||15.6%|
|10% least volatile stocks in US Market||2.8%|
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
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
|RARE fundamental statistics|
Is RARE overvalued?See Fair Value and valuation analysis
Earnings & Revenue
|RARE income statement (TTM)|
|Cost of Revenue||US$527.96m|
Last Reported Earnings
Jun 30, 2022
Next Earnings Date
|Earnings per share (EPS)||-7.22|
|Net Profit Margin||-151.34%|
How did RARE perform over the long term?See historical performance and comparison
Is RARE undervalued compared to its fair value, analyst forecasts and its price relative to the market?
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
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.
|What is RARE's n/a Ratio?|
Price to Sales Ratio vs Peers
How does RARE's PS Ratio compare to its peers?
|RARE PS Ratio vs Peers|
|Company||PS||Estimated Growth||Market Cap|
TWST Twist Bioscience
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.
|Current PS Ratio||8.7x|
|Fair PS Ratio||2.2x|
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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How is Ultragenyx Pharmaceutical forecast to perform in the next 1 to 3 years based on estimates from 16 analysts?
Future Growth Score2/6
Future Growth Score 2/6
Earnings vs Savings Rate
Earnings vs Market
High Growth Earnings
Revenue vs Market
High Growth Revenue
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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How has Ultragenyx Pharmaceutical performed over the past 5 years?
Past Performance Score0/6
Past Performance Score 0/6
Growing Profit Margin
Earnings vs Industry
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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How is Ultragenyx Pharmaceutical's financial position?
Financial Health Score6/6
Financial Health Score 6/6
Short Term Liabilities
Long Term Liabilities
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.
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 0/6
Cash Flow Coverage
Dividend Yield vs Market
|Ultragenyx Pharmaceutical Dividend Yield vs Market|
|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.
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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|
|Date||Total Comp.||Salary||Company Earnings|
|Jun 30 2022||n/a||n/a|
|Mar 31 2022||n/a||n/a|
|Dec 31 2021||US$10m||US$769k|
|Sep 30 2021||n/a||n/a|
|Jun 30 2021||n/a||n/a|
|Mar 31 2021||n/a||n/a|
|Dec 31 2020||US$5m||US$759k|
|Sep 30 2020||n/a||n/a|
|Jun 30 2020||n/a||n/a|
|Mar 31 2020||n/a||n/a|
|Dec 31 2019||US$7m||US$714k|
|Sep 30 2019||n/a||n/a|
|Jun 30 2019||n/a||n/a|
|Mar 31 2019||n/a||n/a|
|Dec 31 2018||US$5m||US$680k|
|Sep 30 2018||n/a||n/a|
|Jun 30 2018||n/a||n/a|
|Mar 31 2018||n/a||n/a|
|Dec 31 2017||US$7m||US$638k|
|Sep 30 2017||n/a||n/a|
|Jun 30 2017||n/a||n/a|
|Mar 31 2017||n/a||n/a|
|Dec 31 2016||US$4m||US$567k|
|Sep 30 2016||n/a||n/a|
|Jun 30 2016||n/a||n/a|
|Mar 31 2016||n/a||n/a|
|Dec 31 2015||US$5m||US$535k|
Compensation vs Market: Emil's total compensation ($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.
Experienced Management: RARE's management team is seasoned and experienced (7.2 years average tenure).
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
|19 Jun 22||SellUS$16,160||Erik Harris||Individual||295||US$54.78|
|16 Apr 22||SellUS$11,161||Theodore Huizenga||Individual||132||US$84.55|
|11 Mar 22||SellUS$688,930||Thomas Kassberg||Individual||10,281||US$67.01|
|07 Mar 22||SellUS$323,563||Karah Parschauer||Individual||5,140||US$62.95|
|01 Mar 22||SellUS$158,156||Thomas Kassberg||Individual||2,374||US$66.62|
|01 Mar 22||SellUS$56,161||Theodore Huizenga||Individual||843||US$66.62|
|01 Mar 22||SellUS$35,575||Mardi Dier||Individual||534||US$66.62|
|01 Mar 22||SellUS$187,935||Camille Bedrosian||Individual||2,821||US$66.62|
|01 Mar 22||SellUS$192,265||Karah Parschauer||Individual||2,886||US$66.62|
|01 Mar 22||SellUS$215,449||Dennis Huang||Individual||3,234||US$66.62|
|01 Mar 22||SellUS$299,657||John Pinion||Individual||4,498||US$66.62|
|01 Mar 22||SellUS$180,740||Erik Harris||Individual||2,713||US$66.62|
|30 Jan 22||SellUS$109,859||Camille Bedrosian||Individual||1,657||US$66.30|
|14 Oct 21||SellUS$29,048||John Pinion||Individual||358||US$81.14|
|14 Oct 21||SellUS$25,153||Karah Parschauer||Individual||310||US$81.14|
|14 Oct 21||SellUS$23,206||Thomas Kassberg||Individual||286||US$81.14|
|14 Oct 21||SellUS$24,585||Erik Harris||Individual||303||US$81.14|
|14 Oct 21||SellUS$29,129||Camille Bedrosian||Individual||359||US$81.14|
|14 Oct 21||SellUS$11,360||Theodore Huizenga||Individual||140||US$81.14|
|14 Oct 21||SellUS$29,048||Dennis Huang||Individual||358||US$81.14|
|12 Oct 21||SellUS$287,861||Mardi Dier||Individual||3,477||US$82.79|
|Owner Type||Number of Shares||Ownership Percentage|
|State or Government||28,243||0.04%|
Dilution of Shares: Shareholders have been diluted in the past year, with total shares outstanding growing by 3.2%.
|Ownership||Name||Shares||Current Value||Change %||Portfolio %|
Ultragenyx Pharmaceutical Inc.'s employee growth, exchange listings and data sources
- 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: https://www.ultragenyx.com
Number of Employees
- Ultragenyx Pharmaceutical Inc.
- 60 Leveroni Court
- United States
|Ticker||Exchange||Primary Security||Security Type||Country||Currency||Listed on|
|RARE||NasdaqGS (Nasdaq Global Select)||Yes||Common Stock||US||USD||Jan 2014|
|UP0||DB (Deutsche Boerse AG)||Yes||Common Stock||DE||EUR||Jan 2014|
|0LIF||LSE (London Stock Exchange)||Yes||Common Stock||GB||USD||Jan 2014|
|RARE *||BMV (Bolsa Mexicana de Valores)||Yes||Common Stock||MX||MXN||Jan 2014|
|R2AR34||BOVESPA (Bolsa de Valores de Sao Paulo)||BDR EACH 10 REPR 1 COM||BR||BRL||Jan 2022|
Company Analysis and Financial Data Status
|Data||Last Updated (UTC time)|
|Company Analysis||2022/09/30 00:00|
|End of Day Share Price||2022/09/30 00:00|
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.