RARE$33.53
$34.03
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Powered by LiveAIOverall Analysis
HoldUltragenyx Pharmaceutical Inc. presents a mixed investment profile. While its focus on rare diseases aligns with a strong thematic trend and its balance sheet appears manageable, the company is consistently unprofitable and faces significant near-term headwinds indicated by technicals. A 'Hold' rating is advised, pending improved profitability and clearer technical signals.
Thematic
Ultragenyx operates in the biopharmaceutical sector, with a specific focus on rare and ultra-rare genetic diseases. This niche addresses unmet medical needs and often commands premium pricing, presenting a strong thematic tailwind. The company's pipeline includes gene therapies and novel therapeutics for conditions like Sanfilippo syndrome and Angelman syndrome, which are areas of significant scientific and market interest.
Fundamental
Ultragenyx Pharmaceutical Inc. exhibits a weak fundamental profile characterized by consistent net losses and negative EPS, alongside significant operating expenses. While the company has substantial current assets and manageable debt levels, its profitability and cash flow generation remain a concern. The valuation metrics are also unfavorable given the lack of profitability.
Technical
Ultragenyx Pharmaceutical Inc. (RARE) is experiencing a significant downward trend, trading well below its longer-term moving averages and showing weak momentum across various timeframes. Key support levels are being tested, and technical indicators suggest a bearish outlook in the short to medium term.
| Factor | Score |
|---|---|
| Rare Disease Focus | 90 |
| Gene Therapy & Novel Therapeutics | 85 |
| Market Growth Potential | 70 |
| Regulatory Environment | 65 |
| Competitive Landscape | 70 |
| Factor | Score |
|---|---|
| Valuation | 45 |
| Profitability | 10 |
| Growth | 70 |
| Balance Sheet Health | 75 |
| Cash Flow | 20 |
| Factor | Score |
|---|---|
| Trend Analysis | 20 |
| Momentum | 40 |
| Volume Confirmation | 30 |
| Support & Resistance | 50 |
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Powered by LiveAIBullish Points (6)
Revenue Growth
Ultragenyx reported total revenue of $139 million for the third quarter of 2024, a 42% increase year-over-year. For the full year 2024, total revenue was $560 million, a 29% increase compared to the prior year. The company has provided 2025 financial guidance projecting total revenue between $640 million to $670 million, indicating expected continued revenue growth.
Positive Analyst Ratings and Price Targets
The average 12-month price target from analysts covering Ultragenyx Pharmaceutical is approximately $83.64 to $87.4, suggesting a significant potential upside of over 200% from current trading prices. The consensus rating among analysts is 'Moderate Buy' to 'Strong Buy', with a majority of analysts recommending a 'Buy'.
Bearish Points (7)
Consistent Net Losses
Ultragenyx Pharmaceutical has consistently reported net losses, with net loss for the year ended December 31, 2024, being $569 million. This ongoing unprofitability is typical for companies in the biotechnology sector focused on growth but presents a significant risk.
High Valuation Metrics for an Unprofitable Company
Despite consistent losses, the company's market capitalization is substantial. The Price-to-Sales (P/S) ratio of approximately 4.19, combined with the absence of positive earnings (indicated by a negative or non-existent P/E ratio), suggests a high valuation that is reliant on future growth and success of its pipeline.
Calendar
July 2025
30
Next Earnings Date
H: $-1.11
A: $-1.33
L: $-1.41
H: 170.30M
A: 161.29M
L: 148.00M
Profile
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.
Seasonals
Price Target
87.21 USD
The 39 analysts offering 1 year price forecasts for RARE have a max estimate of 136.00 and a min estimate of 34.00.
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Related Companies
Powered by LiveAIWeight: 95
Direct competitor in the biotechnology space, particularly focused on therapies for serious and life-threatening rare and ultra-rare genetic diseases. Considered Ultragenyx's biggest rival. [2, 12, 15]
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Direct competitor in the biotechnology space, focused on RNA-based therapeutics for rare and infectious diseases. [2, 3, 12]
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Direct competitor in the pharmaceutical products industry, developing medicines for genetic diseases. [1]