Home 
Today's Top Picks 
Top Performers 
Watchlist 
My Robo Portfolios 
ETF Zone 
Help & Tutorial 
Cancel anytime
- Chart
- Upturn Summary
- Highlights
- Valuation
- Analyst Ratings
Upturn AI SWOT - About
RARE 
Ultragenyx (RARE)
- BUY Advisory
- SELL Advisory (Profit)
- SELL Advisory (Loss)
- Profit
- Loss
- Pass (Skip investing)
Stock price based on last close (see disclosures)
- ALL
- YEAR
- MONTH
- WEEK
Upturn Advisory Summary
06/27/2025: RARE (2-star) is currently NOT-A-BUY. Pass it for now.
Upturn Star Rating
Below Average Performance
These Stocks/ETFs, based on Upturn Advisory, often underperform the market, warranting careful consideration before investing.
Number of Analysts
20 Analysts rated it
These Stocks/ETFs, based on Upturn Advisory, have historically outperformed the market, making them a top-tier choice for investors.
Year Target Price $89.84
Year Target Price $89.84
| 13 | Strong Buy |
| 6 | Buy |
| 1 | Hold |
| 0 | Under performing |
| 0 | Sell |
Analysis of Past Performance
Type Stock | Historic Profit 34.1% | Avg. Invested days 39 | Today’s Advisory PASS |
Upturn Star Rating | Upturn Advisory Performance  4.0 | Stock Returns Performance  3.0 |
| Profits based on simulation | Last Close 06/27/2025 |
Key Highlights
Company Size Mid-Cap Stock | Market Capitalization 3.45B USD | Price to earnings Ratio - | 1Y Target Price 89.84 |
Price to earnings Ratio - | 1Y Target Price 89.84 | ||
Volume (30-day avg) - | Beta 0.24 | 52 Weeks Range 29.59 - 60.37 | Updated Date 06/30/2025 |
52 Weeks Range 29.59 - 60.37 | Updated Date 06/30/2025 | ||
Dividends yield (FY) - | Basic EPS (TTM) -5.83 |
Earnings Date
Report Date - | When - | Estimate - | Actual - |
Profitability
Profit Margin -93.04% | Operating Margin (TTM) -102.62% |
Management Effectiveness
Return on Assets (TTM) -24.51% | Return on Equity (TTM) -377.06% |
Valuation
Trailing PE - | Forward PE - | Enterprise Value 2995562697 | Price to Sales(TTM) 5.85 |
Enterprise Value 2995562697 | Price to Sales(TTM) 5.85 | ||
Enterprise Value to Revenue 5.07 | Enterprise Value to EBITDA -6.46 | Shares Outstanding 94542000 | Shares Floating 90977303 |
Shares Outstanding 94542000 | Shares Floating 90977303 | ||
Percent Insiders 3.46 | Percent Institutions 98.71 |
Analyst Ratings
Rating 4.6 | Target Price 89.84 | Buy 6 | Strong Buy 13 |
Buy 6 | Strong Buy 13 | ||
Hold 1 | Sell - | Strong Sell - | |
Strong Sell - |
Upturn AI SWOT
Ultragenyx
Company Overview
History and Background
Ultragenyx Pharmaceutical Inc. was founded in 2010. It focuses on developing treatments for rare and ultra-rare genetic diseases. The company has grown through drug development, acquisitions, and partnerships.
Core Business Areas
- Commercial Products: This segment focuses on the sales and marketing of approved therapies, including Crysvita, Dojolvi, Mepsevii, and Burokinumab.
- Research and Development: This segment focuses on the development of treatments for rare genetic diseases.
Leadership and Structure
Emil D. Kakkis, M.D., Ph.D., is the CEO and President. The company has a typical corporate structure with various departments headed by VPs reporting to the CEO.
Top Products and Market Share
Key Offerings
- Crysvita (burosumab): Crysvita treats X-linked hypophosphatemia (XLH) and tumor-induced osteomalacia (TIO). Revenue in 2023 was $773.4 million. Competitors include conventional phosphate and vitamin D therapies. Kyowa Kirin is a partner on this drug.
- Dojolvi (triheptanoin): Dojolvi treats long-chain fatty acid oxidation disorders (LC-FAOD). Revenue in 2023 was $101.7 million. There are currently no direct competitors.
- Mepsevii (vestronidase alfa): Mepsevii treats Mucopolysaccharidosis VII (MPS VII). Mepsevii revenue in 2023 was $46.6 million. There are currently no direct competitors.
- Burokinumab: Burokinumab is in Phase 3 development for uncontrolled gout. No revenue until approval.
Market Dynamics
Industry Overview
The rare disease market is growing due to increased awareness, advances in genetic testing, and regulatory incentives. It is characterized by high unmet needs and orphan drug designations.
Positioning
Ultragenyx is a leader in developing and commercializing therapies for rare and ultra-rare genetic diseases. Its competitive advantage lies in its expertise in rare disease drug development and its established commercial infrastructure.
Total Addressable Market (TAM)
The rare disease market is projected to reach hundreds of billions of dollars. Ultragenyx is positioned to capture a significant share with its diverse pipeline and approved therapies.
Upturn SWOT Analysis
Strengths
- Strong expertise in rare disease drug development
- Established commercial infrastructure
- Diverse product portfolio
- Robust pipeline of investigational therapies
Weaknesses
- Reliance on a small number of key products
- High R&D expenses
- Dependence on regulatory approvals
- Potential for competition from larger pharmaceutical companies
Opportunities
- Expanding into new rare disease indications
- Acquiring or licensing new therapies
- Partnering with other companies
- Geographic expansion
Threats
- Clinical trial failures
- Regulatory setbacks
- Competition from biosimilars
- Pricing pressures
Competitors and Market Share
Key Competitors
- BMY
- LLY
- VRTX
Competitive Landscape
Ultragenyx competes with large pharmaceutical companies and smaller biotech firms in the rare disease space. Its focus on ultra-rare diseases gives it a niche advantage.
Major Acquisitions
GeneTx Biotherapeutics
- Year: 2020
- Acquisition Price (USD millions): 76
- Strategic Rationale: Acquired GeneTx to gain access to its GTX-101 program for Angelman syndrome.
Growth Trajectory and Initiatives
Historical Growth: Ultragenyx has experienced significant revenue growth driven by the commercialization of its products.
Future Projections: Analysts expect continued revenue growth driven by existing products and new product launches. Profitability is anticipated but subject to R&D success.
Recent Initiatives: The company has focused on expanding the indications for its existing products, advancing its pipeline, and pursuing strategic acquisitions.
Summary
Ultragenyx is a growing biopharmaceutical company focused on rare diseases with increasing revenues from its commercial products. It faces challenges in achieving profitability and managing R&D expenses. Successful pipeline development and strategic acquisitions will be key for its future growth. Competition is high as is risk of drug development setbacks.
Peer Comparison
Sources and Disclaimers
Data Sources:
- Ultragenyx SEC Filings (10-K, 10-Q)
- Company Investor Relations
- Analyst Reports
- Company Website
Disclaimers:
The information provided is for informational purposes only and should not be considered investment advice. Market data and analysis are subject to change and may not be accurate. Conduct thorough due diligence before making any investment decisions.
AI Summarization is directionally correct and might not be accurate.
Summarized information shown could be a few years old and not current.
Fundamental Rating based on AI could be based on old data.
AI-generated summaries may have inaccuracies (hallucinations). Please verify the information before taking action.
About Ultragenyx
Exchange NASDAQ | Headquaters Novato, CA, United States | ||
IPO Launch date 2014-01-31 | Founder, President, CEO & Director Dr. Emil D. Kakkis M.D., Ph.D. | ||
Sector Healthcare | Industry Biotechnology | Full time employees 1294 | Website https://www.ultragenyx.com |
Full time employees 1294 | Website https://www.ultragenyx.com | ||
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, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific. 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 the treatment of long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidates that are in Phase 3 clinical trials include UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; 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; and GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome. It also develops UX701 that is in Phase 2 clinical trial for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; GeneTx; Mereo; University of Pennsylvania; Solid Biosciences Inc.; Regeneron; Abeona; and Arcturus Therapeutics Holdings Inc. The company was incorporated in 2010 and is headquartered in Novato, California.
Note: This website is maintained by Upturn Corporation, which is an investment adviser registered with the U.S. Securities and Exchange Commission. Such registration does not imply a certain level of skill or training. Investing in securities has risks. Past performance is no guarantee of future returns. No assurance is provided as to any particular investment return, and you may lose money using our services. You are strongly advised to consult appropriate counsel before making any investments in companies you learn about through our services. You should obtain appropriate legal, tax, investment, accounting, and other advice that takes into account your investment portfolio and overall financial situation. You are solely responsible for conducting due diligence on a potential investment. We do not affect trades for you. You will select your own broker through which to transact. Investments are not FDIC insured, they are not guaranteed, and they may lose value. Please see the Privacy Policy, Terms of Use, and Disclosure for more information.