Ultragenyx Reports Second Quarter 2026 Financial Results and Corporate Update
Ultragenyx Pharmaceutical Inc. (RARE) filed an SEC Form 8-K — Results of Operations and Financial Condition. 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 $
How this was made
The 30-second read
Why it matters
Traders can update expectations for 2026 revenue trajectory and model probability-weighted outcomes for multiple late-2026 FDA and Phase 3 catalysts.
Market read
The filing combines quantified financial guidance with a dated catalyst schedule, which can reprice near-term risk and event-driven trading setups.
What to watch
Cash burn remains substantial (net cash used in operations $97M in Q2), so any delay or adverse regulatory/clinical outcome could outweigh near-term guidance confidence.
Second quarter total revenue of $214 million, Crysvita® revenue of $156 million and Dojolvi® revenue of $27 million
Second-quarter revenue reached the highest quarterly level in the company’s history, total revenue increased versus the prior-year quarter, net loss narrowed, and the company reaffirmed 2026 revenue and expense guidance. The period remains loss-making, with $97 million of net cash used in operations and substantial upcoming regulatory and clinical catalysts.
Key metrics
shortened, hover for the filing’s print| Metric | Value | q/q | y/y |
|---|---|---|---|
| Total revenuesGAAP | $214M | – | – |
| Cost of salesGAAP | $34M | – | – |
| Research and developmentGAAP | $167M | – | – |
| Selling, general and administrativeGAAP | $88M | – | – |
| Total operating expensesGAAP | $289M | – | – |
| Non-cash stock-based compensationother | $34M | – | – |
| Net lossGAAP | $92M | – | – |
| Net loss per share, basic and dilutedGAAP | $0.90 per share basic and diluted | – | – |
| Six Months Ended June 30 Total revenuesGAAP | $350M | – | – |
| Six Months Ended June 30 Cost of salesGAAP | $64M | – | – |
| Six Months Ended June 30 Research and developmentGAAP | $354M | – | – |
| Six Months Ended June 30 Selling, general and administrativeGAAP | $176M | – | – |
| Six Months Ended June 30 Total operating expensesGAAP | $594M | – | – |
| Six Months Ended June 30 Net lossGAAP | $277M | – | – |
| Six Months Ended June 30 Net loss per share, basic and dilutedGAAP | $2.73 per share basic and diluted | – | – |
Segments
| Segment | Revenue | q/q | y/y |
|---|---|---|---|
| Crysvita®Consistent with expected seasonality in the U.S. and Canada and ordering patterns in Latin America. | $156M | – | – |
| Dojolvi®Dojolvi was listed on the National Health Insurance drug price list and launched in Japan in May 2026. | $27M | – | – |
| Evkeeza®Driven by increased demand from new country launches and early access. | $21M | – | – |
| Mepsevii® | $10M | – | – |
full year 2026 outlook
- Revenue$730 million to $760 million
- Operating expensescombined R&D and SG&A expenses in 2026 are expected to be flat to down low-single digits compared to 2025
- NoteCrysvita revenue guidance of $500 million to $520 million
- NoteDojolvi revenue guidance of $100 million to $110 million
- NoteTotal revenue guidance excludes revenue from potential new product launches.
- NoteCombined R&D and SG&A expenses in 2027 are expected to decrease by at least 15% compared to 2025.
- NoteRemain on path to profitability in 2027.
What drove it
- Total revenue in the second quarter of 2026 was $214 million.
- Crysvita revenue was consistent with expected seasonality in the U.S. and Canada and ordering patterns in Latin America.
- Evkeeza revenue was driven by increased demand from new country launches and early access.
- Dojolvi launched in Japan in May 2026 following National Health Insurance drug price-list inclusion.
- The company has PDUFA action dates for DTX401 on August 23, 2026 and UX111 on September 19, 2026.
- Data from the GTX-102 Phase 3 Aspire study are expected in the September or October timeframe.
Concerns
- The company reported a net loss of $92 million and net cash used in operations of $97 million for the three months ended June 30, 2026.
- Total operating expenses were $289 million, including research and development expense of $167 million.
- Crysvita revenue is subject to expected seasonality in the U.S. and Canada and ordering patterns in Latin America.
- The company identifies dependence on Kyowa Kirin for commercialization and commercial supply of Crysvita in certain major markets, including the U.S. and Canada.
- Potential approvals, launches, and clinical readouts remain subject to regulatory, clinical, manufacturing, reimbursement, and market-acceptance risks.
What to watch
- DTX401 PDUFA action date of August 23, 2026.
- UX111 PDUFA action date of September 19, 2026.
- GTX-102 Phase 3 Aspire study data expected in the September or October timeframe.
- Completion of enrollment in the GTX-102 Phase 2/3 Aurora study expected in the second half of 2026.
- UX701 pivotal Cyprus2+ dose-finding-stage data expected in the fourth quarter of 2026.
- UX016 externally funded Phase 1/2 study expected to begin in the second half of 2026.
- DTX301 Phase 3 Enh3ance second-primary-endpoint data expected in the first half of 2027.
- Delivery against full-year 2026 total revenue guidance of $730 million to $760 million and combined R&D and SG&A expense guidance.
Balance sheet and cash flow
- 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.
Analysis
Ultragenyx reported second-quarter total revenue of $214 million, compared with $167 million in the prior-year quarter. The company described this as its highest quarterly revenue in its history and reaffirmed full-year 2026 total revenue guidance of $730 million to $760 million, excluding potential new-product-launch revenue. Crysvita contributed $156 million, while Dojolvi, Evkeeza, and Mepsevii contributed $27 million, $21 million, and $10 million, respectively.
Crysvita remains the largest reported product-revenue contributor. Management attributed its quarterly performance to expected seasonality in the U.S. and Canada and ordering patterns in Latin America. Dojolvi had been launched in Japan during May 2026 after National Health Insurance drug price-list inclusion. Evkeeza revenue was driven by increased demand from new country launches and early access. The company reaffirmed full-year Crysvita revenue guidance of $500 million to $520 million and Dojolvi guidance of $100 million to $110 million.
The company remained loss-making despite higher revenue. Total operating expenses were $289 million, compared with $275 million in the prior-year quarter, including $167 million of research and development expense and $88 million of selling, general and administrative expense. Net loss improved to $92 million from $115 million, while net loss per share improved to $0.90 from $1.17. The company reaffirmed that combined R&D and SG&A expenses in 2026 are expected to be flat to down low-single digits compared to 2025, and that such expenses in 2027 are expected to decrease by at least 15% compared to 2025.
Liquidity and cash consumption remain important. Cash, cash equivalents, and marketable securities were $436 million as of June 30, 2026, and net cash used in operations was $97 million for the quarter. No capital-return activity or debt balance was reported in the supplied release.
The second half of 2026 includes material regulatory and clinical events. DTX401 has an August 23, 2026 PDUFA action date, and UX111 has a September 19, 2026 PDUFA action date. GTX-102 Phase 3 Aspire data are expected in the September or October timeframe, UX701 data are expected in the fourth quarter of 2026, and DTX301 second-primary-endpoint data are expected in the first half of 2027. These events, together with the company’s stated path to profitability in 2027, are central to the outlook.
Management, verbatim
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,
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.
Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx
Not in the filing
stated, not guessed- Gross profit and gross margin
- Operating income or loss
- Non-GAAP revenue, earnings, EPS, or expense metrics
- Prior-quarter comparisons for reported financial metrics
- Reported year-over-year or quarter-over-quarter percentage changes for financial metrics and product revenues
- Product revenue comparisons for Crysvita, Dojolvi, Evkeeza, and Mepsevii
- Free cash flow
- Debt balance
- Capital returns, including share repurchases and dividends
- Tax rate
- Gross-margin guidance
- Tax-rate guidance
- Prior outlook section for comparison with reported results
AlphAI analysis generated from the company’s SEC earnings filing (Form 8-K Item 2.02, or Form 6-K for a foreign private issuer). Every figure was cross-checked against the filing text; consensus estimates, price targets and share-price reactions are not shown because they are not in the filing. AI-generated research, not investment advice.
Background
This is an SEC Form 8-K (Item 2.02) with Exhibit 99.1 covering Q2 2026 financial results and a corporate update for Ultragenyx.
Ticker impact
Ultragenyx reported Q2 2026 results and reaffirmed 2026 revenue guidance, while flagging upcoming PDUFA dates and GTX-102 Phase 3 data timing.
Likely positive bias with event-driven swings around Aug 23 and Sep 19 PDUFAs and the Sep-Oct GTX-102 data window.
The filing provides concrete financial guidance ranges, cash balance, and specific regulatory/clinical milestones with expected timing, which are direct inputs to risk/reward for the stock.
Market effects
Reinforces investor focus on gene therapy and rare-disease biotech catalyst calendars tied to PDUFA and pivotal readouts.
Limited direct regional spillover; Japan launch and NHI listing may support incremental demand narrative.
Moderate global relevance via FDA review timelines and multinational commercialization updates.
Counterpoint
Reaffirmed guidance may be viewed as maintenance rather than upside, and the largest value inflection (GTX-102 Phase 3) is still pending.
Key entities
- issuerUltragenyx Pharmaceutical Inc.
Reported Q2 2026 financial results, reaffirmed 2026 guidance, and outlined upcoming regulatory and clinical milestones.
- productCrysvita
Reported Q2 2026 revenue of $156M and reaffirmed 2026 guidance of $500M to $520M.
- productDojolvi
Reported Q2 2026 revenue of $27M and reaffirmed 2026 guidance of $100M to $110M, with Japan launch and NHI listing noted.
- clinical_programDTX401
FDA accepted BLA for review with PDUFA action date of Aug 23, 2026.
- clinical_programUX111
FDA accepted resubmitted BLA for accelerated approval with PDUFA action date of Sep 19, 2026.
