Ultragenyx (NASDAQ: RARE) Q2 revenue hits $214 million as 2026 guidance reaffirmed
Rhea-AI Filing Summary
Ultragenyx Pharmaceutical Inc. reported second quarter 2026 results, with total revenue of $214 million, up from $167 million a year earlier and the highest quarterly revenue in its history. Key contributors were Crysvita at $156 million, Dojolvi at $27 million, Evkeeza at $21 million and Mepsevii at $10 million.
The company recorded a net loss of $92 million, or $0.90 per share, compared with a net loss of $115 million, or $1.17 per share, in second quarter 2025. Cash, cash equivalents and marketable securities were $436 million as of June 30, 2026, with $97 million of net cash used in operations in the quarter.
Ultragenyx reaffirmed 2026 guidance for total revenue of $730 million to $760 million, Crysvita revenue of $500 million to $520 million, and Dojolvi revenue of $100 million to $110 million, and continues to target profitability in 2027. Upcoming catalysts include FDA PDUFA action dates for DTX401 on August 23, 2026 and UX111 on September 19, 2026, plus a pivotal Phase 3 GTX-102 data readout in Angelman syndrome.
Positive
- Q2 revenue reached $214 million, up from $167 million in Q2 2025.
- Net loss declined to $92 million ($0.90 per share) from $115 million ($1.17 per share).
- Reaffirmed 2026 total revenue guidance of $730 million to $760 million and maintained a 2027 profitability target.
Negative
- Cash, cash equivalents and marketable securities were $436 million at June 30, 2026, down from $737 million at December 31, 2025, with $97 million of net cash used in operations in Q2.
Filing Explained
At June 30, Ultragenyx reported $436 million in cash and securities.
This Form 8-K furnishes Ultragenyx’s reported results for the quarter ended
For existing common holders, the material structural update is the balance-sheet position at June 30:
The filing identifies UX701 pivotal-study data as expected in the fourth quarter of 2026 and DTX301 second-primary-endpoint data as expected in the first half of 2027; neither outcome is reported here.
8-K Event Classification
Key Figures
Key Terms
Biologics License Application (BLA) regulatory
Prescription Drug User Fee Act (PDUFA) action date regulatory
Conditional Approval System for Pharmaceuticals regulatory
Investigational New Drug (IND) application regulatory
non-cash stock-based compensation financial
Earnings Snapshot
Reaffirmed 2026 total revenue guidance of $730 million to $760 million, Crysvita revenue of $500 million to $520 million, Dojolvi revenue of $100 million to $110 million, and expects combined R&D and SG&A expenses flat to down low-single digits vs 2025 in 2026 and at least 15% lower in 2027, targeting profitability in 2027.
AI-generated analysis. How Rhea-AI works. Not financial advice.
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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934
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Securities registered pursuant to Section 12(b) of the Act:
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Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).
Emerging growth company
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
Item 2.02 |
Results of Operations and Financial Condition. |
On August 4, 2026, Ultragenyx Pharmaceutical Inc. (the “Company”) issued a press release announcing its financial results for the three months ended June 30, 2026 (the “Press Release”). A copy of the Press Release is furnished herewith as Exhibit 99.1
The information set forth under Item 2.02 and in Exhibit 99.1 shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, except as shall be expressly set forth by specific reference in such filing.
Item 9.01 |
Financial Statements and Exhibits. |
(d) Exhibits
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Description |
99.1 |
Press Release, dated August 4, 2026. |
104 |
The cover page from the Company’s Current Report on Form 8-K dated August 4, 2026 formatted in Inline XBRL. |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
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Ultragenyx Pharmaceutical Inc. |
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August 4, 2026 |
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/s/Howard Horn |
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Howard Horn |
Exhibit 99.1
Contacts Ultragenyx Pharmaceutical Inc.
Investors
Joshua Higa
ir@ultragenyx.com
Media
Jess Rowlands
media@ultragenyx.com
Ultragenyx Reports Second Quarter 2026 Financial Results and Corporate Update
Second quarter total revenue of $214 million,
Crysvita® revenue of $156 million and Dojolvi® revenue of $27 million
Reaffirm 2026 financial guidance, including total revenue of $730 million to $760 million and combined R&D and SG&A expenses to be flat to slightly down versus 2025; remain on path to profitability in 2027
Catalysts in second half of 2026 include two PDUFA dates and pivotal data readout from
GTX-102 Phase 3 Aspire study for Angelman syndrome
NOVATO, Calif. – August 4, 2026 – Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today reported its financial results for the quarter ended June 30, 2026 and reaffirmed its financial guidance for 2026.
“In the second quarter we generated the highest quarterly revenue in the history of the company, supporting our full-year revenue guidance, and keeping us on track toward profitability in 2027,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “As we look to the second half of the year, we are entering a transformative period with multiple important catalysts. We are ready to launch two gene therapy products and are preparing for a pivotal Phase 3 GTX-102 data readout in Angelman syndrome. This puts us in position to broaden our patient impact with sustained growth in the years to come.”
Second Quarter 2026 Revenue Highlights and 2026 Revenue Guidance
Milestones and Upcoming Catalysts
Enrollment in the open-label Phase 2/3 Aurora study, evaluating GTX-102 in other genotypes and ages, began in October 2025 and is expected to complete in the second half of 2026.
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Summary of Second Quarter 2026 Financial Results
Selected Financial Data (dollars in millions, except per share amounts), (unaudited)
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Three Months Ended June 30, |
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Six Months Ended June 30, |
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2026 |
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2025 |
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2026 |
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2025 |
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Total revenues |
$ |
214 |
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$ |
167 |
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$ |
350 |
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$ |
306 |
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Operating expenses: |
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|
|
|
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Cost of sales |
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34 |
|
|
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23 |
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|
64 |
|
|
|
52 |
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Research and development |
|
167 |
|
|
|
165 |
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|
354 |
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|
|
331 |
|
Selling, general and administrative |
|
88 |
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|
|
87 |
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|
176 |
|
|
|
174 |
|
Total operating expenses |
|
289 |
|
|
|
275 |
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|
594 |
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|
|
557 |
|
Net loss |
$ |
(92 |
) |
|
$ |
(115 |
) |
$ |
(277 |
) |
|
$ |
(266 |
) |
Net loss per share, basic and diluted |
$ |
(0.90 |
) |
|
$ |
(1.17 |
) |
$ |
(2.73 |
) |
|
$ |
(2.73 |
) |
Operating Expenses
Total operating expenses for the second quarter 2026 were $289 million, including $34 million of non-cash stock-based compensation. The company reaffirms its full year 2026 and 2027 guidance for combined R&D and SG&A operating expenses: compared to 2025, combined R&D and SG&A expenses in 2026 are expected to be flat to down low‑single digits, and combined R&D and SG&A expenses in 2027 are expected to decrease by at least 15%.
Net Loss
Net loss for the second quarter 2026 was $92 million, or $0.90 per share basic and diluted, compared with a net loss for the second quarter 2025 of $115 million, or $1.17 per share basic and diluted.
Cash Balance and Net Cash Used in Operations
Cash, cash equivalents, and marketable securities were $436 million as of June 30, 2026. For the three months ended June 30, 2026, net cash used in operations was $97 million.
Conference Call and Webcast Information
Ultragenyx will host a conference call today, Tuesday, August 4, 2026, at 2 p.m. PT/5 p.m. ET to discuss the second quarter financial results and provide a corporate update. The live and
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replayed webcast of the call will be available through the company’s website at https://ir.ultragenyx.com/events-presentations. The replay of the call will be available for three months.
About Ultragenyx
Ultragenyx is a biopharmaceutical company committed to bringing novel therapies to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved medicines and treatment candidates aimed at addressing diseases with high unmet medical need and clear biology, for which there are typically no approved therapies treating the underlying disease.
The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.
For more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com.
Forward-Looking Statements and Use of Digital Media
Except for the historical information contained herein, the matters set forth in this press release, including statements regarding Ultragenyx’s expectations and projections concerning its future operating results and financial performance, including its 2026 revenue guidance for total revenue, Crysvita and Dojolvi, its anticipated R&D and SG&A expenses in 2026 and 2027, anticipated benefits and savings from its strategic restructuring plan, and the timing and sustainability of profitability; the timing, progress, results and plans for its clinical programs and studies, including the anticipated timing and outcome of the Phase 3 Aspire study of GTX-102, enrollment in the Aurora study, data from the UX701 and DTX301 studies, and initiation of the UX016 study; the FDA’s review of the BLAs for DTX401 and UX111, including the anticipated PDUFA action dates, the potential approval of either product candidate, whether the FDA may require additional information, studies or manufacturing changes, and the timing and outcome of regulatory inspections; Ultragenyx’s manufacturing and commercial readiness and the timing and success of any potential launches of DTX401 and UX111, if approved; and the potential patient impact, commercial opportunity and growth associated with Ultragenyx’s products and product candidates, are forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause the company’s clinical development programs, commercial success of its products and product candidates, continued collaboration with third parties, future results, performance or achievements to differ
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significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainty of clinical drug development and unpredictability and lengthy process for obtaining regulatory approvals, risks related to serious or undesirable side effects of our product candidates, the company’s ability to achieve its projected development goals in its expected timeframes, risks related to reliance on third party partners to conduct certain activities on the company’s behalf, our limited experience in generating revenue from product sales, risks related to product liability lawsuits, our dependence on Kyowa Kirin for the commercialization of Crysvita in certain major markets, including the U.S. and Canada, and for our commercial supply of Crysvita in those markets, fluctuations in buying or distribution patterns from distributors and specialty pharmacies, smaller than anticipated market opportunities for the company’s products and product candidates, manufacturing risks, our ability to successfully manage the expansion of our company, delays or unexpected costs and other adverse effects related to the strategic restructuring plan, competition from other therapies or products, regulatory scrutiny of the company’s products and product candidates, the company’s limited experience as a company in operating its own manufacturing facility, market acceptance of our products, uncertainty related to insurance coverage and reimbursement, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company’s future operating results and financial performance, the timing of clinical trial activities and reporting results from same, and the availability or commercial potential of Ultragenyx’s products and drug candidate. Ultragenyx undertakes no obligation to update or revise any forward-looking statements.
For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on May 6, 2026, and its subsequent periodic reports filed with the SEC.
In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyx’s Investor Relations website (https://ir.ultragenyx.com/) and LinkedIn website (https://www.linkedin.com/company/ultragenyx-pharmaceutical-inc-/).
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Ultragenyx Pharmaceutical Inc.
Selected Revenue Data
(in millions)
(unaudited)
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Three Months Ended June 30, |
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Six Months Ended June 30, |
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2026 |
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2025 |
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2026 |
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2025 |
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Crysvita |
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Product sales - Latin America and Türkiye |
$ |
54 |
|
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$ |
35 |
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$ |
100 |
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|
$ |
90 |
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Royalty revenue - U.S. and Canada |
|
94 |
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|
79 |
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|
133 |
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120 |
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Royalty revenue - Europe |
|
8 |
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7 |
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16 |
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14 |
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Total Crysvita Revenue |
|
156 |
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121 |
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249 |
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224 |
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Dojolvi |
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27 |
|
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23 |
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45 |
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40 |
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Evkeeza |
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21 |
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14 |
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39 |
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25 |
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Mepsevii |
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10 |
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9 |
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17 |
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17 |
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Total revenues |
$ |
214 |
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$ |
167 |
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$ |
350 |
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$ |
306 |
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6
Ultragenyx Pharmaceutical Inc.
Selected Statement of Operations Financial Data
(in millions, except per share amounts)
(unaudited)
|
Three Months Ended June 30, |
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Six Months Ended June 30, |
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2026 |
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2025 |
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2026 |
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2025 |
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Statement of Operations Data: |
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Revenues: |
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Product sales |
$ |
112 |
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$ |
81 |
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$ |
201 |
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$ |
172 |
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Royalty revenue |
|
102 |
|
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|
86 |
|
|
149 |
|
|
|
134 |
|
Total revenues |
|
214 |
|
|
|
167 |
|
|
350 |
|
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|
306 |
|
Operating expenses: |
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|
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|
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||||
Cost of sales |
|
34 |
|
|
|
23 |
|
|
64 |
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|
|
52 |
|
Research and development |
|
167 |
|
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|
165 |
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|
354 |
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|
331 |
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Selling, general and administrative |
|
88 |
|
|
|
87 |
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|
176 |
|
|
|
174 |
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Total operating expenses |
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289 |
|
|
|
275 |
|
|
594 |
|
|
|
557 |
|
Loss from operations |
|
(75 |
) |
|
|
(108 |
) |
|
(244 |
) |
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(251 |
) |
Non-cash interest expense on liabilities for sales of future royalties |
|
(22 |
) |
|
|
(14 |
) |
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(43 |
) |
|
|
(28 |
) |
Other income, net |
|
6 |
|
|
|
8 |
|
|
12 |
|
|
|
15 |
|
Loss before income taxes |
|
(91 |
) |
|
|
(114 |
) |
|
(275 |
) |
|
|
(264 |
) |
Provision for income taxes |
|
(1 |
) |
|
|
(1 |
) |
|
(2 |
) |
|
|
(2 |
) |
Net loss |
$ |
(92 |
) |
|
$ |
(115 |
) |
$ |
(277 |
) |
|
$ |
(266 |
) |
Net loss per share, basic and diluted |
$ |
(0.90 |
) |
|
$ |
(1.17 |
) |
$ |
(2.73 |
) |
|
$ |
(2.73 |
) |
Shares used in computing net loss per share, basic and diluted |
|
101.9 |
|
|
|
98.5 |
|
|
101.3 |
|
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|
97.4 |
|
7
Ultragenyx Pharmaceutical Inc.
Selected Activity included in Operating Expenses
(in millions)
(unaudited)
|
Three Months Ended June 30, |
|
Six Months Ended June 30, |
|
||||||||||
|
2026 |
|
|
2025 |
|
2026 |
|
|
2025 |
|
||||
Non-cash stock-based compensation |
$ |
34 |
|
|
$ |
39 |
|
$ |
64 |
|
|
$ |
79 |
|
Restructuring expense |
|
— |
|
|
|
— |
|
|
30 |
|
|
|
— |
|
Ultragenyx Pharmaceutical Inc.
Selected Balance Sheet Financial Data
(in millions)
(unaudited)
|
June 30, |
|
|
December 31, |
|
||
|
2026 |
|
|
2025 |
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||
Balance Sheet Data: |
|
|
|
|
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||
Cash, cash equivalents, and marketable securities |
$ |
436 |
|
|
$ |
737 |
|
Working capital |
|
255 |
|
|
|
567 |
|
Total assets |
|
1,264 |
|
|
|
1,532 |
|
Total stockholders' deficit |
|
(291 |
) |
|
|
(80 |
) |
8