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Ultragenyx Pharmaceutical (RARE)
NASDAQ:RARE
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Ultragenyx Pharmaceutical
(NASDAQ:RARE)
54Neutral
Positive Factors
Financial Performance
Ultragenyx provided a strong revenue growth guidance with expected 17% year-over-year growth at the midpoint for FY25.
Product Launch
UX111 could be Ultragenyx's first gene therapy launch, setting the stage for several other programs in the pipeline.
Regulatory Milestone
The FDA has accepted the BLA of UX111 for Priority Review under an accelerated approval pathway, indicating a significant regulatory milestone for Ultragenyx.
Negative Factors
Clinical Trial Uncertainty
There is a focus on the second interim analysis for setrusumab in mid-2025, while the pipeline continues to progress nicely.
Commercial Variability
There is a reiterated Buy rating with a price objective of $83, indicating confidence in the stock's future performance.
Investment Risks
There are risks to the investment thesis, including potential clinical trial failures, failure to secure regulatory approvals, and smaller than anticipated commercial opportunities.
Ultragenyx Pharmaceutical (RARE) vs. S&P 500 (SPY)
Market Cap
$3.05B
Dividend YieldN/A
Average Volume (3M)752.84K
Price to Earnings (P/E)―
Beta (1Y)1.21
Revenue Growth29.01%
EPS Growth23.93%
CountryUS
Employees1,294
SectorHealthcare
Sector Strength76
IndustryBiotechnology
Share Statistics
EPS (TTM)-6.34
Shares Outstanding93,899,666
10 Day Avg. Volume750,740
30 Day Avg. Volume752,836
Financial Highlights & Ratios
PEG RatioN/A
Price to Book (P/B)N/A
Price to Sales (P/S)6.47
Price to Cash Flow (P/CF)-11.40
P/FCF Ratio0.00
Enterprise Value/Market CapN/A
Enterprise Value/RevenueN/A
Enterprise Value/Gross ProfitN/A
Enterprise Value/EbitdaN/A
Forecast
1Y Price Target
$91.00Price Target Upside180.60% Upside
Rating ConsensusStrong Buy
Number of Analyst Covering17
Ultragenyx Pharmaceutical Business Overview & Revenue Model
Company DescriptionUltragenyx 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 Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; Bayer Healthcare LLC; GeneTx; Mereo; University of Pennsylvania; Arcturus Therapeutics Holdings Inc., Solid Biosciences Inc.; and Daiichi Sankyo Co., Ltd. Ultragenyx Pharmaceutical Inc. was incorporated in 2010 and is headquartered in Novato, California.
How the Company Makes MoneyUltragenyx Pharmaceutical generates revenue primarily through the sale of its approved therapies for rare genetic diseases. The company's revenue model is centered around its ability to successfully research, develop, and commercialize niche products that cater to specific genetic disorders, which often lack sufficient treatment options. Key revenue streams include product sales from its proprietary drugs like Crysvita, Mepsevii, and Dojolvi. Ultragenyx also engages in strategic partnerships and licensing agreements, which can provide additional revenue through milestone payments and royalties. The company's earnings are significantly influenced by its research and development pipeline, regulatory approvals, and market exclusivity periods granted for orphan drugs.
Ultragenyx Pharmaceutical Financial Statement Overview
Summary
Ultragenyx Pharmaceutical demonstrates strong revenue growth and a robust gross profit margin. However, the company faces substantial profitability and cash flow challenges, with ongoing net losses and negative free cash flow, despite a low debt-to-equity ratio.Income Statement
Ultragenyx Pharmaceutical shows a consistent increase in revenue over the years, with a significant revenue growth rate of 29% from 2022 to 2023. However, the company continues to face substantial losses, as indicated by negative EBIT and net income margins. The gross profit margin remains strong, but high operating expenses contribute to persistent net losses.40
Negative
Balance Sheet
The company maintains a relatively low debt-to-equity ratio, indicating prudent use of leverage. However, the declining stockholders' equity and a lower equity ratio suggest potential risks in financial stability. The return on equity remains negative due to ongoing net losses, which could affect long-term shareholder value.55
Neutral
Cash Flow
Ultragenyx has a negative free cash flow, although there is an improvement in free cash flow from 2023 to 2024. The operating cash flow to net income ratio indicates operational cash flow challenges. Despite positive financing cash flows, sustained negative operating cash flows highlight the need for enhanced operational efficiencies.50
Neutral
| Breakdown | ||||
| Dec 2024 | Dec 2023 | Dec 2022 | Dec 2021 | Dec 2020 |
|---|---|---|---|---|
| Income Statement | Total Revenue | |||
| 560.23M | 434.25M | 363.33M | 351.41M | 271.03M | Gross Profit |
| 483.50M | 389.04M | 335.01M | 335.40M | 264.90M | EBIT |
| -535.97M | -569.21M | -648.92M | -381.74M | -330.12M | EBITDA |
| -469.00M | -516.45M | -640.51M | -410.32M | -139.81M | Net Income Common Stockholders |
| -569.18M | -606.64M | -707.42M | -454.02M | -186.57M |
| Balance Sheet | Cash, Cash Equivalents and Short-Term Investments | |||
| 610.02M | 577.21M | 896.73M | 999.13M | 1.21B | Total Assets |
| 1.50B | 1.49B | 1.55B | 1.52B | 1.76B | Total Debt |
| 10.30M | 43.17M | 31.59M | 41.97M | 48.23M | Net Debt |
| -163.43M | -170.41M | -101.35M | -265.61M | -665.30M | Total Liabilities |
| 1.24B | 1.22B | 1.19B | 599.84M | 605.18M | Stockholders Equity |
| 255.30M | 275.41M | 352.49M | 922.56M | 1.15B |
| Cash Flow | Free Cash Flow | |||
| -421.68M | -521.57M | -526.59M | -411.79M | -176.13M | Operating Cash Flow |
| -414.19M | -474.81M | -380.46M | -338.69M | -132.22M | Investing Cash Flow |
| -17.77M | 168.00M | -291.65M | -195.37M | -179.12M | Financing Cash Flow |
| 399.24M | 388.14M | 501.21M | 118.55M | 600.27M |
Ultragenyx Pharmaceutical Technical Analysis
Negative
32.43
Price Trends
40.87
Negative
43.00
Negative
46.93
Negative
Market Momentum
-1.40
Positive
27.75
Positive
16.19
Positive
Evaluating momentum and price trends is crucial in stock analysis to make informed investment decisions. For RARE, the sentiment is Negative. The current price of 32.43 is below the 20-day moving average (MA) of 37.83, below the 50-day MA of 40.87, and below the 200-day MA of 46.93, indicating a bearish trend. The MACD of -1.40 indicates Positive momentum. The RSI at 27.75 is Positive, neither overbought nor oversold. The STOCH value of 16.19 is Positive, not indicating any strong overbought or oversold conditions. Overall, these indicators collectively point to a Negative sentiment for RARE.
Ultragenyx Pharmaceutical Risk Analysis
Ultragenyx Pharmaceutical disclosed 62 risk factors in its most recent earnings report. Ultragenyx Pharmaceutical reported the most risks in the “Tech & Innovation” category.
Finance & Corporate - Financial and accounting risks. Risks related to the execution of corporate activity and strategy
Latest Risks Added 0 New Risks
Ultragenyx Pharmaceutical Peers Comparison
Overall Rating
UnderperformOutperform
Sector (48)
Financial Indicators
Name | Overall Rating | Market Cap | P/E Ratio | ROE | Dividend Yield | Revenue Growth | EPS Growth |
|---|---|---|---|---|---|---|---|
78 Outperform | $11.50B | 27.40 | 8.05% | ― | 17.35% | 151.40% | |
68 Neutral | $5.28B | 22.84 | 19.71% | ― | 52.97% | ― | |
64 Neutral | $122.02B | ― | -3.15% | ― | 11.64% | -114.72% | |
61 Neutral | $2.30B | ― | -31.68% | ― | 32.29% | 64.39% | |
54 Neutral | $3.05B | ― | -214.50% | ― | 29.01% | 23.93% | |
48 Neutral | $6.36B | 1.09 | -49.92% | 2.63% | 17.17% | 0.95% | |
46 Neutral | $2.81B | ― | -19.20% | ― | -89.95% | -123.71% |
* Healthcare Sector Average
Performance Comparison
RARE
Ultragenyx Pharmaceutical
32.43
-17.80
-35.44%
FOLD
Amicus
7.50
-4.10
-35.34%
BMRN
BioMarin Pharmaceutical
60.26
-27.02
-30.96%
VRTX
Vertex Pharmaceuticals
474.62
70.40
17.42%
SRPT
Sarepta Therapeutics
54.43
-71.96
-56.93%
CRSP
Crispr Therapeutics AG
32.65
-31.44
-49.06%
Ultragenyx Pharmaceutical Earnings Call Summary
Earnings Call Date: Feb 13, 2025 | % Change Since: -25.48% | Next Earnings Date: May 1, 2025
Earnings Call Sentiment Positive
The earnings call reflected strong revenue growth and successful international expansion, alongside notable advancements in clinical programs. However, significant net losses and high operating expenses present financial challenges. The positive highlights slightly outweigh the concerns, indicating a cautiously optimistic outlook.Highlights
Exceeding Revenue Expectations
Ultragenyx reported a total revenue of $560 million for 2024, representing a 29% growth over 2023. They exceeded their increased guidance for total revenue, surpassing the upper end of the range.
International Expansion Success
Ultragenyx successfully launched EVKEEZA in Europe, Canada, and Japan, and expanded access in Latin America, Canada, and Turkey. This contributed to a 78% growth in CRYSVITA sales in Latin America and Turkey over 2023.
Clinical Program Advancements
Ultragenyx advanced 6 late-stage programs, including the filing of their first BLA for Sanfilippo syndrome and expected Phase 3 data on UX143 for osteogenesis imperfecta.
Strong Product Performance
CRYSVITA contributed $410 million in revenue, with DOJOLVI contributing $88 million, each showing significant growth over the previous year.
Lowlights
Significant Net Loss
Ultragenyx reported a net loss of $569 million for 2024, which translates to a loss of $6.29 per share.
High Operating Expenses
Total operating expenses for the year were $1.1 billion, including R&D expenses of $698 million and SG&A expenses of $322 million.
Cash Flow Concerns
Net cash used in operations was $414 million in 2024, with expectations of reduced net cash usage in 2025, but still indicating a high cash burn rate.
Company Guidance
During the Ultragenyx Fourth Quarter and Full Year 2024 Financial Results Conference Call, the company reported impressive metrics and provided guidance for 2025. Ultragenyx announced a 2024 total revenue of $560 million, marking a 29% growth over the previous year. CRYSVITA contributed $410 million, DOJOLVI $88 million, EVKEEZA $32 million, and MEPSEVII $30 million to the total revenue. Operating expenses for 2024 reached $1.1 billion, resulting in a net loss of $569 million. Looking ahead to 2025, Ultragenyx anticipates total revenue between $640 million and $670 million, representing a growth range of 14% to 20% over 2024. Specifically, CRYSVITA revenue is expected to reach between $460 million and $480 million, and DOJOLVI revenue is projected between $90 million and $100 million. The company aims for full-year GAAP profitability by 2027 and continues to prioritize expense management while advancing its clinical pipeline and preparing for potential product launches.Glossary
OutperformA stock rated as "Outperform" is expected to perform better than the overall market or a specific benchmark over the near-to-medium term. This rating suggests that the stock is likely to deliver higher returns compared to the average returns of other stocks in the same sector or market index. Investors might consider this stock a good buying opportunity.
NeutralA stock rated as "Neutral" is expected to perform in line with the overall market or a specific benchmark. This rating indicates that the stock is neither particularly attractive nor unattractive for investment. Investors may consider holding onto the stock, as it is not expected to either significantly outperform or underperform the market.
UnderperformA stock rated as "Underperform" is expected to perform worse than the overall market or a specific benchmark over the near-to-medium term. This rating suggests that the stock may deliver lower returns compared to the average returns of other stocks in the same sector or market index. Investors might consider selling the stock or avoiding it as an investment.
Disclaimer
This AI Analyst Stock Report is automatically generated by our AI systems using advanced algorithms and publicly available financial, technical, and market data. While the information provided aims to be accurate and insightful, it is intended for informational purposes only and should not be considered financial advice. Any content created by an AI (Artificial Intelligence) system may contain inaccuracies and/or contain errors. Investing in stocks carries inherent risks, and past performance is not indicative of future results. This report does not account for your personal financial circumstances, objectives, or risk tolerance. Always conduct your own research or consult with a qualified financial advisor before making investment decisions. The analysis and recommendations provided are based on historical and current data and may not fully reflect future market conditions or unexpected developments. Neither the creators of this report nor its affiliated entities guarantee the accuracy, completeness, or reliability of the information presented. Use this report at your own discretion and risk.