Ultragenyx Pharmaceutical Inc. (RARE)

Key figures

The last twelve months reported, with the latest price and balance sheet.

Revenue, last twelve months

$717.2M

Revenue growth

17.5%

Net income, last twelve months

-$586.0M

Earnings per share

-$5.84

Operating margin

-73.6%

Net margin

-81.7%

Market value

$1.7B

Cash

$125.0M

Debt

$0

Shares outstanding

98.6M

Financial statements

Revenue, profit and cash flow by fiscal year, each year linked to its annual report.

By quarter

What the company said with its results

From the second quarter of 2026 results, filed 4 August 2026.

• Record quarterly revenue reported. Total revenue was $214 million, up from $167 million in the second quarter of 2025. Management described it as the highest quarterly revenue in company history.
• Net loss narrowed year on year. Net loss was $92 million, or $0.90 per share, against $115 million, or $1.17 per share, in the second quarter of 2025.
• Six-month revenue higher. Six-month total revenue was $350 million, against $306 million a year earlier. Six-month net loss was $277 million, the same per-share loss as the prior year period.
• Crysvita revenue split across lines. Crysvita revenue includes Latin America and Türkiye product sales, U.S. and Canada royalties, and Europe royalties. Management tied the quarter to seasonality and ordering patterns.
• Evkeeza growth from new countries. Evkeeza revenue rose on demand from new country launches and early access, according to management.
• Mepsevii revenue up slightly. Mepsevii revenue rose from the second quarter of 2025, and six-month revenue was level with the prior year.
• Operating expenses and stock compensation. Total operating expenses were $289 million, against $275 million a year earlier. Non-cash stock-based compensation was $34 million, against $39 million.
• Research and development close to prior year. Research and development expense was close to the level of the second quarter of 2025, and SG&A was nearly unchanged.
• Restructuring expense not repeated. The company recorded $30 million of restructuring expense in the first quarter of 2026 and none in the second quarter.
• Royalty financing interest expense rose. Non-cash interest expense on liabilities for sales of future royalties was $22 million in the quarter, against $14 million a year earlier.
• Cash balance declined this year. Cash, cash equivalents and marketable securities were $436 million at June 30, 2026, down from $534 million at March 31 and $737 million at December 31, 2025.
• Operating cash use lower than first quarter. Net cash used in operations was $97 million in the quarter. The first quarter included annual bonus payments and $38 million tied to UX143 manufacturing.
• Stockholders' deficit and working capital. Working capital was $255 million at June 30, 2026, and the total stockholders' deficit was $291 million, against $80 million at December 31, 2025.
• Weighted share count increased. Shares used in computing net loss per share were 101.9 million in the quarter, against 98.5 million a year earlier.

Revenue by segment

As the company reports it in the segment note of its annual report.

By product, FY2025

By region, FY2025

Company events

The newest 8-K filings of 209 held.

Every filing

The newest filings with the SEC of 354 held.

Insider trades

Form 4 trades by officers, directors and 10% owners, last 12 months.

Planned insider sales

Form 144 notices, filed when the sell order goes to the broker.

Large holders

Schedule 13D and 13G filings by holders of more than 5% of the voting shares.

Trades by members of the House

From each member's periodic transaction report.

Short interest

Shares sold short and not yet bought back, on each FINRA settlement date.

Funds that hold it

From each fund's latest Form 13F.

354 funds held it at 30 Jun 2026: 169 added and 98 trimmed in the quarter. The largest:

Peers

Companies in the same industry.

• Eli Lilly & Co. (LLY): revenue $79.7B, market value $1.21T
• Johnson & Johnson (JNJ): revenue $99.3B, market value $652.9B
• AbbVie Inc. (ABBV): revenue $64.4B, market value $506.1B
• Merck & Co., Inc. (MRK): revenue $66.6B, market value $348.1B
• Novartis AG (NVS): revenue $54.5B, market value $297.8B
• AstraZeneca PLC (AZN): revenue $58.7B, market value $263.1B
• Abbott Laboratories (ABT): revenue $46.6B, market value $177.7B
• Novo Nordisk A/S (NVO): revenue $46.8B, market value $171.0B
• Pfizer Inc. (PFE): revenue $63.7B, market value $168.0B
• Vertex Pharmaceuticals Inc. (VRTX): revenue $12.6B, market value $134.2B

What the company does

Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company that develops treatments for serious rare and ultra-rare genetic diseases. Emil Kakkis, M.D., Ph.D., founded it in April 2010 and is its President and Chief Executive Officer. It has four commercially approved products: Crysvita, Mepsevii, Dojolvi and Evkeeza. Its clinical pipeline spans four product categories: biologics, small molecules, AAV gene therapy and nucleic acids. As of 31 December 2025 it had 1,371 employees.

What it sells

Crysvita is a fully human monoclonal antibody that targets fibroblast growth factor 23. It treats X-Linked Hypophosphatemia, or XLH, and Tumor-Induced Osteomalacia, or TIO. The filing estimates approximately 48,000 XLH patients in the developed world.

Mepsevii is an enzyme replacement therapy given intravenously for Mucopolysaccharidosis VII, a condition affecting an estimated 200 patients in the developed world.

Dojolvi is an oral synthetic fatty acid triglyceride for Long-chain Fatty Acid Oxidation Disorders, a group with approximately 8,000 to 14,000 patients in the developed world.

Evkeeza is a fully human monoclonal antibody given by IV for Homozygous Familial Hypercholesterolemia. It is approved in the United States, where Regeneron Pharmaceuticals markets it. It is also approved in the European Economic Area, Brazil, Mexico and Japan, and Ultragenyx holds the rights outside the United States.

How the products reach revenue

Customers include collaboration partners, drug wholesalers and retail pharmacy distributors. Kyowa Kirin Co., Ltd., or KKC, generated 45% of total revenues in 2025.

The Crysvita arrangement with KKC differs by region. In the United States and Canada, KKC has had commercial responsibility since April 2023, and Ultragenyx is entitled to a revenue share intended to approximate the profit-share. In Europe, KKC books the sales and pays Ultragenyx a royalty, which Ultragenyx sold to RPI Finance Trust, an affiliate of Royalty Pharma, in December 2019. In Latin America and Türkiye, Ultragenyx commercializes Crysvita itself. In Latin America it books the sales, and KKC supplies the product for a transfer price and a royalty. Ultragenyx also sold a percentage of future United States and Canada Crysvita royalties to an investment vehicle for the Ontario Municipal Employees Retirement System in July 2022 and November 2025.

The pipeline

- UX111 is an AAV9 gene therapy for Sanfilippo syndrome type A. The FDA issued a Complete Response Letter in July 2025. A resubmitted application in January 2026 drew an Incomplete Response Letter in February 2026, and the company says it plans to resubmit. - DTX401 is an AAV8 gene therapy for Glycogen Storage Disease Type Ia. A rolling BLA submission was completed in December 2025, and the company says it expects an FDA action date in the third quarter of 2026. - GTX-102 is an antisense oligonucleotide for Angelman syndrome. The Phase 3 Aspire study enrolled 129 patients aged four to 17, and the Phase 2/3 Aurora study began enrolling in October 2025. - DTX301 is an AAV8 gene therapy for ornithine transcarbamylase deficiency. Its Phase 3 study enrolled 37 patients. - UX701 is an AAV9 gene therapy for Wilson disease. The pivotal Cyprus2+ study is in its dose-finding stage. - UX143 (setrusumab) is an antibody for Osteogenesis Imperfecta. In December 2025 the Phase 3 Orbit and Cosmic studies did not reach their primary endpoint, and further analyses are ongoing.

Partners and licences

Ultragenyx says its products and candidates were generally in-licensed from academic institutions or came from partnerships with other pharmaceutical companies. It says it tries to keep global commercialization rights.

- KKC (Crysvita) - Regeneron (Evkeeza outside the United States, supplied by Regeneron at a purchase price) - Saint Louis University (Mepsevii) - Baylor Research Institute (Dojolvi) - REGENXBIO Inc. (DTX301 and DTX401) - Abeona Therapeutics (UX111) - Mereo (setrusumab) - University of Pennsylvania (gene therapy research)

Ultragenyx exercised its option to acquire GeneTx Biotherapeutics LLC, the developer of GTX-102, in July 2022.

Making and selling

Manufacturing combines internal capability with contract manufacturers. Ultragenyx runs its own gene therapy manufacturing facility, and its contract manufacturers for product candidates are in Western Europe or North America. KKC makes Crysvita in Japan, and Rentschler Biopharma SE makes the Mepsevii drug substance. IOI Oleo GmbH in Germany supplies the Dojolvi drug substance. Regeneron makes the Evkeeza drug substance in Rensselaer, New York, and Baxter Pharmaceutical Solutions, LLC makes the drug product in Bloomington, Indiana.

The company has built its own commercial organizations in North America, Europe, Latin America and Japan, and uses a network of third-party distributors in smaller markets. It describes rare disease commercialization as relying on a targeted specialty field organization, and it says finding eligible patients is a particular challenge.

Patents and its people

As of 31 December 2025, Ultragenyx owned, jointly owned or held exclusive rights to more than 300 issued and in-force patents, including more than 50 from the USPTO. It also held more than 300 pending applications. It uses orphan drug status and marketing or data exclusivity periods alongside patents.

Of its 1,371 employees, 916 work in research and development and 455 in sales, general and administrative roles. In all, 1,144 are based in the United States, including at sites in Novato and Brisbane in California and in Somerville, Bedford and Woburn in Massachusetts. The other 227 are at international locations. In February 2026 the company started a restructuring plan that includes a 10% workforce reduction of approximately 130 employees.

*From Ultragenyx's annual report on Form 10-K for the year ended 31 December 2025. [The filing](https://www.sec.gov/Archives/edgar/data/1515673/000119312526057329/rare-20251231.htm)*

From the company's 10-K.