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Upturn AI SWOT - About
RARE 
Ultragenyx (RARE)
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Upturn Advisory Summary
10/21/2025: RARE (2-star) has a low Upturn Star Rating. Not recommended to BUY.
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
Well-followed company, solid analyst reports, reliable data for confident investing.
1 Year Target Price $86.1
1 Year Target Price $86.1
| 13 | Strong Buy |
| 6 | Buy |
| 1 | Hold |
| 0 | Sell |
| 0 | Strong Sell |
Analysis of Past Performance
Type Stock | Historic Profit 1.45% | Avg. Invested days 25 | Today’s Advisory Consider higher Upturn Star rating |
Upturn Star Rating | Upturn Advisory Performance  3.0 | Stock Returns Performance  1.0 |
| Profits based on simulation | Last Close 10/21/2025 |
Key Highlights
Company Size Mid-Cap Stock | Market Capitalization 3.29B USD | Price to earnings Ratio - | 1Y Target Price 86.1 |
Price to earnings Ratio - | 1Y Target Price 86.1 | ||
Volume (30-day avg) 20 | Beta 0.21 | 52 Weeks Range 25.81 - 56.38 | Updated Date 10/21/2025 |
52 Weeks Range 25.81 - 56.38 | Updated Date 10/21/2025 | ||
Dividends yield (FY) - | Basic EPS (TTM) -5.48 |
Analyzing Revenue: Products, Geography and Growth
Revenue by Products
Product revenue - Year on Year
Revenue by Geography
Geography revenue - Year on Year
Earnings Date
Report Date - | When - | Estimate - | Actual - |
Profitability
Profit Margin -87.34% | Operating Margin (TTM) -64.8% |
Management Effectiveness
Return on Assets (TTM) -21.59% | Return on Equity (TTM) -180.44% |
Valuation
Trailing PE - | Forward PE - | Enterprise Value 2536795768 | Price to Sales(TTM) 5.4 |
Enterprise Value 2536795768 | Price to Sales(TTM) 5.4 | ||
Enterprise Value to Revenue 4.16 | Enterprise Value to EBITDA -6.46 | Shares Outstanding 96371643 | Shares Floating 92783558 |
Shares Outstanding 96371643 | Shares Floating 92783558 | ||
Percent Insiders 3.13 | Percent Institutions 100.46 |
Upturn AI SWOT
Ultragenyx
Company Overview
History and Background
Ultragenyx Pharmaceutical Inc. was founded in 2010 by Emil Kakkis. It focuses on developing treatments for rare and ultra-rare genetic diseases. The company has grown through internal development, acquisitions, and strategic partnerships, aiming to address unmet medical needs.
Core Business Areas
- Biopharmaceutical Development: Focuses on the discovery, development, and commercialization of therapies for rare and ultra-rare genetic diseases.
- Commercialization: Responsible for the marketing and sales of Ultragenyx's approved products in various global markets.
Leadership and Structure
Ultragenyx is led by CEO Emil Kakkis. The company has a structured organizational framework with distinct departments for research and development, commercial operations, finance, and corporate affairs.
Top Products and Market Share
Key Offerings
- Crysvita (burosumab): A monoclonal antibody targeting fibroblast growth factor 23 (FGF23) for the treatment of X-linked hypophosphatemia (XLH) and tumor-induced osteomalacia (TIO). The market share varies by geography, but it is a significant revenue driver. Competitors include Kyowa Kirin (with similar anti-FGF23 therapies).
- Dojolvi (triheptanoin): An oral therapy for patients with long-chain fatty acid oxidation disorders (LC-FAOD). It commands a substantial share within its niche market. No direct competitors exist for this specific molecule.
- Mepsevii (vestronidase alfa): An enzyme replacement therapy for Mucopolysaccharidosis VII (MPS VII), also known as Sly syndrome. Given that MPS VII is an extremely rare disease, the market share is practically the entire treated patient population. BioMarin Pharmaceutical Inc. is a competitor in enzyme replacement therapies.
Market Dynamics
Industry Overview
The biopharmaceutical industry, particularly the segment focused on rare diseases, is growing. Driven by regulatory incentives (like orphan drug status), increasing awareness, and advancements in genetic therapies, there is significant investment and innovation.
Positioning
Ultragenyx is a key player in the rare disease space, specializing in enzyme replacement, gene, and antibody therapies. Its competitive advantage lies in its focused approach, expertise in rare diseases, and established commercial infrastructure.
Total Addressable Market (TAM)
The rare disease therapeutics market is estimated to be over $200 billion. Ultragenyx is positioned to capture a portion of this TAM by focusing on specific rare conditions with unmet medical needs and developing innovative therapies.
Upturn SWOT Analysis
Strengths
- Strong pipeline focused on rare and ultra-rare diseases
- Established expertise in developing and commercializing niche therapies
- Orphan drug designations providing market exclusivity and regulatory benefits
- Experienced management team
- Strategic partnerships
Weaknesses
- High research and development costs
- Dependence on successful clinical trial outcomes
- Reliance on a limited number of marketed products
- Potential competition from larger pharmaceutical companies
- Pricing and reimbursement pressures
Opportunities
- Expanding into new rare disease indications
- Acquiring or licensing promising rare disease therapies
- Geographic expansion into emerging markets
- Advancements in gene therapy and other innovative platforms
- Continued regulatory support for rare disease drug development
Threats
- Clinical trial failures
- Regulatory setbacks
- Competition from biosimilars or generic versions of existing drugs
- Changes in reimbursement policies
- Economic downturn impacting healthcare spending
Competitors and Market Share
Key Competitors
- BMRN
- SGEN
- AMGN
Competitive Landscape
Ultragenyx faces competition from established pharmaceutical companies with broader portfolios and resources, but it maintains a competitive edge through its focused approach, specialized expertise, and orphan drug designations. BioMarin competes directly in MPS therapies.
Major Acquisitions
GeneTx Biotherapeutics
- Year: 2020
- Acquisition Price (USD millions): 76
- Strategic Rationale: Expanded Ultragenyx's pipeline with a gene therapy program targeting Angelman syndrome, a rare neurogenetic disorder.
Growth Trajectory and Initiatives
Historical Growth: Ultragenyx has experienced significant revenue growth driven by the increasing adoption of its approved products. The company has also invested heavily in its research and development pipeline.
Future Projections: Analysts predict continued revenue growth for Ultragenyx, driven by further market penetration of its existing products and the potential approval of new therapies in its pipeline.
Recent Initiatives: Ultragenyx has recently focused on expanding its gene therapy programs and pursuing strategic collaborations to accelerate its research and development efforts.
Summary
Ultragenyx is a promising biopharmaceutical company focused on rare diseases with a solid pipeline and growing revenues. Its strong expertise and orphan drug designations provide a competitive advantage. However, it faces high R&D costs, reliance on clinical trial success, and competition from larger firms and needs to carefully manage these risks to maintain its growth trajectory and market position.
Peer Comparison
Sources and Disclaimers
Data Sources:
- Company Filings (SEC)
- Industry Reports
- Analyst Estimates
Disclaimers:
This analysis is for informational purposes only and does not constitute financial advice. Investment decisions should be made based on thorough research and consultation with a financial professional.
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.
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