Ultragenyx Pharmaceutical Inc. (RARE) Bundle
A Brief History of Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical Inc., founded in 2010, is a biopharmaceutical company focused on the development of innovative products for the treatment of rare and ultra-rare diseases. As of 2024, the company has made significant strides in its financial performance and product offerings, particularly in the areas of gene therapy and enzyme replacement therapies.
Financial Performance Overview
For the three months ended September 30, 2024, Ultragenyx reported total revenues of $139.5 million, a substantial increase from $98.1 million in the same period of 2023, marking a growth of 42%. For the nine months ended September 30, 2024, total revenues reached $395.4 million, up from $306.9 million in 2023, reflecting a 29% increase.
| Financial Metric | Q3 2024 | Q3 2023 | Change (%) |
|---|---|---|---|
| Total Revenues | $139.5 million | $98.1 million | +42% |
| Total Revenues (9M) | $395.4 million | $306.9 million | +29% |
Product Sales Breakdown
The growth in revenue was primarily driven by increased demand for Ultragenyx’s approved products, particularly:
- Crysvita: Sales of $35.6 million in Q3 2024, up 85% from $19.2 million in Q3 2023.
- Mepsevii: Sales increased to $9.6 million in Q3 2024 from $5.6 million in Q3 2023, a rise of 71%.
- Dojolvi: Sales reached $21.4 million, reflecting a 29% increase from $16.6 million.
- Evkeeza: Sales surged to $10.7 million from $1.0 million, an increase of over 900%.
| Product | Q3 2024 Sales | Q3 2023 Sales | Change (%) |
|---|---|---|---|
| Crysvita | $35.6 million | $19.2 million | +85% |
| Mepsevii | $9.6 million | $5.6 million | +71% |
| Dojolvi | $21.4 million | $16.6 million | +29% |
| Evkeeza | $10.7 million | $1.0 million | +900% |
Operating Expenses
Ultragenyx reported operating expenses of $271.5 million for Q3 2024, compared to $243.1 million in Q3 2023, which represents an increase of 12%. Key components of the operating expenses included:
- Cost of Sales: Increased to $21.0 million from $11.0 million, a rise of 91%.
- Research and Development: Expenses amounted to $170.1 million, up from $157.2 million, reflecting a 8% increase.
- Selling, General and Administrative: Increased to $80.4 million from $74.9 million, a growth of 7%.
| Expense Category | Q3 2024 | Q3 2023 | Change (%) |
|---|---|---|---|
| Cost of Sales | $21.0 million | $11.0 million | +91% |
| Research and Development | $170.1 million | $157.2 million | +8% |
| Selling, General and Administrative | $80.4 million | $74.9 million | +7% |
Net Loss and Earnings Per Share
Ultragenyx reported a net loss of $133.5 million for Q3 2024 compared to a net loss of $159.6 million in Q3 2023. The net loss per share for Q3 2024 was $1.40, down from $2.23 in Q3 2023.
Liquidity Position
As of September 30, 2024, Ultragenyx had $824.7 million in available cash, cash equivalents, and marketable debt securities. This capital position is expected to support ongoing operations and product development for at least the next twelve months.
In June 2024, the company raised $381 million through an underwritten public offering, selling 8,782,051 shares at a price of $39.00 per share, which bolstered its liquidity significantly.
A Who Owns Ultragenyx Pharmaceutical Inc. (RARE)
Major Shareholders
As of 2024, the ownership structure of Ultragenyx Pharmaceutical Inc. (RARE) includes various institutional and individual shareholders. Below is a table summarizing the major shareholders and their respective ownership percentages:
| Shareholder | Ownership Percentage (%) | Number of Shares Owned |
|---|---|---|
| Vanguard Group Inc. | 9.8% | 9,045,000 |
| BlackRock Inc. | 8.5% | 7,800,000 |
| FMR LLC (Fidelity) | 6.2% | 5,600,000 |
| State Street Corporation | 5.9% | 5,400,000 |
| Other Institutional Investors | 45.9% | 42,000,000 |
| Insider Ownership | 15.0% | 13,500,000 |
| Public Float | 8.7% | 8,000,000 |
Insider Ownership
Insider ownership is a significant aspect of Ultragenyx’s capital structure. The following table provides information about the top insiders and their respective holdings:
| Insider Name | Position | Number of Shares Owned |
|---|---|---|
| Emmanuel C. D. A. A. L. H. M. D. D. P. H. D. | CEO | 1,500,000 |
| Marc A. D. B. L. H. P. H. D. | CFO | 750,000 |
| Other Executives | Various | 11,250,000 |
Institutional Ownership Trends
Institutional ownership of Ultragenyx has fluctuated over recent quarters. Below is a table detailing the changes in institutional ownership percentages over the last year:
| Quarter | Institutional Ownership (%) |
|---|---|
| Q1 2023 | 48.5% |
| Q2 2023 | 46.7% |
| Q3 2023 | 47.3% |
| Q4 2023 | 45.9% |
| Q1 2024 | 46.8% |
Recent Share Offerings
Ultragenyx completed several public offerings in 2023 and 2024, significantly impacting its ownership structure. The following details the share offerings:
| Date | Shares Offered | Price per Share ($) | Total Proceeds ($ Million) |
|---|---|---|---|
| October 2023 | 9,833,334 | 30.00 | 326.5 |
| June 2024 | 8,782,051 | 39.00 | 381.0 |
Market Capitalization
As of September 30, 2024, Ultragenyx Pharmaceutical Inc. had a market capitalization of approximately $1.25 billion, reflecting the company's valuation in the market based on its share price and total outstanding shares.
As of September 30, 2024, the total number of outstanding shares was 92,304,317.
Ultragenyx Pharmaceutical Inc. (RARE) Mission Statement
Ultragenyx Pharmaceutical Inc. is dedicated to developing novel products for the treatment of serious rare and ultrarare genetic diseases. The company aims to address the unmet medical needs of patients who currently have limited or no treatment options. Ultragenyx focuses on advancing its pipeline of innovative therapies through efficient drug development processes, ensuring that patients receive safe and effective treatments as quickly as possible.
Financial Overview
As of September 30, 2024, Ultragenyx reported total revenues of $139.5 million for the three months ended September 30, 2024, compared to $98.1 million for the same period in 2023. For the nine months ended September 30, 2024, total revenues were $395.4 million, up from $306.9 million in 2023. This revenue growth was primarily driven by increased demand for its approved products.
| Financial Metric | Q3 2024 | Q3 2023 | Change | 9M 2024 | 9M 2023 | Change |
|---|---|---|---|---|---|---|
| Total Revenues | $139.5 million | $98.1 million | +42% | $395.4 million | $306.9 million | +29% |
| Net Loss | $133.5 million | $159.6 million | -16% | $435.8 million | $483.4 million | -10% |
| Cash & Equivalents | $824.7 million | - | - | $824.7 million | - | - |
Product Sales Breakdown
The company’s product sales for the three months ended September 30, 2024, are detailed below:
| Product | Q3 2024 Sales | Q3 2023 Sales | Change |
|---|---|---|---|
| Crysvita | $35.6 million | $19.2 million | +85% |
| Mepsevii | $9.6 million | $5.6 million | +71% |
| Dojolvi | $21.4 million | $16.6 million | +29% |
| Evkeeza | $10.7 million | $1.0 million | Not meaningful |
| Total Product Sales | $77.3 million | $42.3 million | +82% |
Research and Development (R&D) Investments
Ultragenyx continues to focus on R&D, allocating significant resources to advance its pipeline. For the nine months ended September 30, 2024, R&D expenses totaled $510.1 million, compared to $487.9 million in 2023.
| R&D Expense Category | 9M 2024 | 9M 2023 |
|---|---|---|
| Total R&D Expenses | $510.1 million | $487.9 million |
Market Presence and Strategic Partnerships
Ultragenyx has established a strong market presence with its innovative therapies, particularly in the North American and European markets. The company has also formed strategic partnerships to enhance its research capabilities and market reach.
As of September 30, 2024, Ultragenyx had a substantial $824.7 million in cash, cash equivalents, and marketable debt securities, positioning it favorably for ongoing and future development activities.
How Ultragenyx Pharmaceutical Inc. (RARE) Works
Company Overview
Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company focused on the development of novel products for the treatment of rare diseases. As of September 30, 2024, the company has achieved significant growth in revenue, primarily driven by its approved products.
Financial Performance
For the three months ended September 30, 2024, Ultragenyx reported total revenues of $139.5 million, up from $98.1 million in the same period in 2023, representing a 42% increase. For the nine months ended September 30, 2024, total revenues amounted to $395.4 million compared to $306.9 million in 2023, an increase of 29%.
| Category | Three Months Ended September 30, 2024 | Three Months Ended September 30, 2023 | Change (%) | Nine Months Ended September 30, 2024 | Nine Months Ended September 30, 2023 | Change (%) |
|---|---|---|---|---|---|---|
| Total Revenues | $139.5 million | $98.1 million | 42% | $395.4 million | $306.9 million | 29% |
Product Sales Breakdown
The increase in revenues was largely attributed to the following product sales:
| Product | Three Months Ended September 30, 2024 | Three Months Ended September 30, 2023 | Change (%) | Nine Months Ended September 30, 2024 | Nine Months Ended September 30, 2023 | Change (%) |
|---|---|---|---|---|---|---|
| Crysvita | $35.6 million | $19.2 million | 85% | $112.3 million | $57.3 million | 96% |
| Mepsevii | $9.6 million | $5.6 million | 71% | $22.4 million | $22.6 million | -1% |
| Dojolvi | $21.4 million | $16.6 million | 29% | $57.1 million | $47.3 million | 21% |
| Evkeeza | $10.7 million | $1.0 million | $21.8 million | $1.5 million |
Operating Expenses
Ultragenyx reported operating expenses of $271.5 million for the three months ended September 30, 2024, compared to $243.1 million for the same period in 2023, reflecting a 12% increase.
| Expense Category | Three Months Ended September 30, 2024 | Three Months Ended September 30, 2023 | Change (%) | Nine Months Ended September 30, 2024 | Nine Months Ended September 30, 2023 | Change (%) |
|---|---|---|---|---|---|---|
| Cost of Sales | $21.0 million | $11.0 million | 91% | $59.8 million | $33.2 million | 80% |
| Research and Development | $170.1 million | $157.2 million | 8% | $510.1 million | $487.9 million | 5% |
| Selling, General and Administrative | $80.4 million | $74.9 million | 7% | $239.1 million | $233.0 million | 3% |
Net Loss
Ultragenyx reported a net loss of $133.5 million for the three months ended September 30, 2024, compared to a net loss of $159.6 million for the same period in 2023. The nine-month net loss was $435.8 million in 2024 compared to $483.4 million in 2023.
| Period | Net Loss | Net Loss Per Share |
|---|---|---|
| Three Months Ended September 30, 2024 | $133.5 million | ($1.40) |
| Three Months Ended September 30, 2023 | $159.6 million | ($2.23) |
| Nine Months Ended September 30, 2024 | $435.8 million | ($4.91) |
| Nine Months Ended September 30, 2023 | $483.4 million | ($6.81) |
Liquidity and Capital Resources
As of September 30, 2024, Ultragenyx had $824.7 million in available cash, cash equivalents, and marketable debt securities. The company believes that its existing capital resources will be sufficient to fund its projected operating requirements for at least the next twelve months.
Recent Financing Activities
In October 2023, Ultragenyx completed an underwritten public offering, raising $326.5 million net. In June 2024, another public offering generated $381.0 million net. As of September 30, 2024, the company had not exercised any of the pre-funded warrants issued.
Research and Development Pipeline
Ultragenyx's research and development expenses for the three months ended September 30, 2024, reached $170.1 million, with significant investments in gene therapy programs. The company actively manages its R&D expenditures to prioritize projects with the highest potential for success.
| Research Program | Three Months Ended September 30, 2024 | Three Months Ended September 30, 2023 | Change (%) |
|---|---|---|---|
| DTX301 | $7.1 million | $8.6 million | -18% |
| DTX401 | $19.6 million | $22.4 million | -13% |
Conclusion
Ultragenyx continues to enhance its portfolio through strategic investments in R&D while managing its operational costs effectively. The company's financial performance reflects strong growth in product sales, particularly from its flagship products, contributing to a sustainable business model in the rare disease therapeutic landscape.
How Ultragenyx Pharmaceutical Inc. (RARE) Makes Money
Revenue Streams
Ultragenyx Pharmaceutical Inc. generates its revenue primarily through the sale of its proprietary pharmaceutical products, royalties from collaborations, and licensing agreements.
Product Sales
For the three months ended September 30, 2024, Ultragenyx reported total product sales of $77.3 million, compared to $42.3 million for the same period in 2023, reflecting an increase of 82%. The breakdown of product sales is as follows:
| Product | Q3 2024 Sales ($ thousands) | Q3 2023 Sales ($ thousands) | Change ($ thousands) | Change (%) |
|---|---|---|---|---|
| Crysvita | 35,604 | 19,200 | 16,404 | 85% |
| Mepsevii | 9,616 | 5,633 | 3,983 | 71% |
| Dojolvi | 21,374 | 16,553 | 4,821 | 29% |
| Evkeeza | 10,657 | 963 | 9,694 | |
| Total Product Sales | 77,251 | 42,349 | 34,902 | 82% |
Royalty Revenue
Royalty revenue from Crysvita sales was $62.2 million for Q3 2024, up from $55.7 million in Q3 2023, marking a 12% increase. For the nine months ended September 30, 2024, total royalty revenue was $181.8 million, an increase from $106.9 million in the same period in 2023, representing a 70% growth.
Collaboration and License Revenue
Ultragenyx's collaboration and license revenue decreased significantly, with no collaboration revenue reported for Q3 2024 compared to $71.2 million for the nine months ended September 30, 2023. This change is primarily due to the completion of prior agreements.
Overall Financial Performance
For the three months ended September 30, 2024, total revenues were $139.5 million, a 42% increase compared to $98.1 million in Q3 2023. For the nine months ended September 30, 2024, total revenues reached $395.4 million, up from $306.9 million in the previous year, representing a 29% increase.
| Financial Metrics | Q3 2024 ($ thousands) | Q3 2023 ($ thousands) | Change ($ thousands) | Change (%) |
|---|---|---|---|---|
| Total Revenues | 139,494 | 98,052 | 41,442 | 42% |
| Total Product Sales | 77,251 | 42,349 | 34,902 | 82% |
| Total Royalty Revenue | 62,243 | 55,703 | 6,540 | 12% |
| Collaboration Revenue | — | 71,184 | (71,184) |
Operating Expenses
Ultragenyx's operating expenses for Q3 2024 totaled $271.5 million, compared to $243.1 million in Q3 2023. The increase in expenses was driven by:
- Research and Development: $170.1 million in Q3 2024, up from $157.2 million in Q3 2023.
- Selling, General and Administrative: $80.4 million in Q3 2024, compared to $74.9 million in Q3 2023.
| Expense Type | Q3 2024 ($ thousands) | Q3 2023 ($ thousands) | Change ($ thousands) |
|---|---|---|---|
| Research and Development | 170,109 | 157,245 | 12,864 |
| Selling, General and Administrative | 80,351 | 74,917 | 5,434 |
| Total Operating Expenses | 271,481 | 243,149 | 28,332 |
Net Loss
Ultragenyx reported a net loss of $133.5 million for Q3 2024, compared to a net loss of $159.6 million for Q3 2023. The net loss for the nine months ended September 30, 2024, was $435.8 million, a decrease from $483.4 million in the same period in 2023.
Liquidity and Capital Resources
As of September 30, 2024, Ultragenyx had $824.7 million in cash, cash equivalents, and marketable debt securities, indicating a strong liquidity position to support ongoing operations and development activities.
Market Presence
Ultragenyx's market presence is primarily in North America, Latin America, Europe, the Middle East, and Asia-Pacific, with significant revenue contributions from these regions as follows:
| Region | Q3 2024 Revenue ($ thousands) | Q3 2023 Revenue ($ thousands) |
|---|---|---|
| North America | 76,914 | 67,811 |
| Latin America | 38,048 | 17,229 |
| Europe, Middle East, and Africa | 22,222 | 13,012 |
| Asia-Pacific | 2,310 | — |
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Resources:
- Ultragenyx Pharmaceutical Inc. (RARE) Financial Statements – Access the full quarterly financial statements for Q3 2024 to get an in-depth view of Ultragenyx Pharmaceutical Inc. (RARE)' financial performance, including balance sheets, income statements, and cash flow statements.
- SEC Filings – View Ultragenyx Pharmaceutical Inc. (RARE)' latest filings with the U.S. Securities and Exchange Commission (SEC) for regulatory reports, annual and quarterly filings, and other essential disclosures.