Gene therapy reviews and Q1 2026 results at Ultragenyx (RARE)
Rhea-AI Filing Summary
Ultragenyx Pharmaceutical reported first quarter 2026 revenue of $136 million, slightly below $139 million a year earlier, with Crysvita contributing $93 million, Dojolvi $18 million, Evkeeza $18 million and Mepsevii $7 million. Product sales were $89 million and royalty revenue $47 million.
The company posted a net loss of $185 million, or $1.84 per share, compared with a $151 million loss, or $1.57 per share, in 2025 as total operating expenses rose to $305 million. Cash, cash equivalents and marketable securities were $534 million as of March 31, 2026, down from $737 million at year-end, with $197 million of net cash used in operations.
Ultragenyx reaffirmed its 2026 guidance, targeting total revenue of $730–$760 million and combined R&D and SG&A expenses flat to slightly down versus 2025, and continues to aim for profitability in 2027. The company highlighted two gene therapy BLAs under FDA Priority Review for DTX401 and UX111, with PDUFA action dates on August 23, 2026 and September 19, 2026, respectively, and positive longer-term data from the GTX-102 Angelman syndrome program ahead of a Phase 3 readout in the second half of 2026.
Positive
- Two late-stage gene therapy BLAs under FDA Priority Review, DTX401 with an August 23, 2026 PDUFA date and UX111 with a September 19, 2026 PDUFA date, provide near-term regulatory catalysts that could meaningfully expand Ultragenyx’s commercial portfolio if approved.
- Reaffirmed 2026 financial guidance, including total revenue of $730–$760 million and expectations for combined R&D and SG&A expenses to be flat to slightly down versus 2025, while maintaining a stated goal of reaching profitability in 2027.
Negative
- Widening net loss and high cash burn, with Q1 2026 net loss increasing to $185 million from $151 million and net cash used in operations of $197 million, contributed to cash and marketable securities declining from $737 million to $534 million over the quarter.
- Balance sheet moved deeper into deficit, as total stockholders’ equity shifted from a deficit of $80 million at December 31, 2025 to a deficit of $236 million at March 31, 2026, reflecting cumulative losses and financial leverage.
Insights
Ultragenyx balances widening losses with solid revenue base and late-stage gene therapy catalysts.
Ultragenyx generated $136 million in Q1 2026 revenue, with Crysvita at $93 million and additional contributions from Dojolvi, Evkeeza and Mepsevii. Guidance for 2026 revenue of $730–$760 million was reaffirmed, signaling confidence in the commercial portfolio.
Losses remain substantial: net loss widened to $185 million versus $151 million a year earlier, and operating expenses reached $305 million, including $30 million of restructuring costs. Cash and marketable securities fell to $534 million, and operating cash outflow was $197 million, underscoring the importance of future revenue growth and cost control.
Pipeline news is significant. The FDA granted Priority Review for DTX401 with an August 2026 PDUFA date and accepted the UX111 resubmitted BLA with a September 2026 PDUFA date. Longer-term GTX-102 data and an upcoming Phase 3 readout in the second half of 2026 add further potential catalysts. Actual impact will depend on regulatory outcomes and subsequent launches.
8-K Event Classification
Key Figures
Key Terms
Priority Review regulatory
Biologics License Application (BLA) regulatory
PDUFA action date regulatory
antisense oligonucleotide (ASO) technical
Phase 1/2 medical
stock-based compensation financial
Earnings Snapshot
2026 total revenue guidance of $730–$760 million; combined R&D and SG&A expenses expected flat to down low-single digits versus 2025; profitability targeted in 2027.
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 May 5, 2026, Ultragenyx Pharmaceutical Inc. (the “Company”) issued a press release announcing its financial results for the three months ended March 31, 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
Exhibit No. |
Description |
99.1 |
Press Release, dated May 5, 2026. |
104 |
The cover page from the Company’s Current Report on Form 8-K dated May 5, 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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May 5, 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 First Quarter 2026 Financial Results and Corporate Update
First quarter total revenue of $136 million,
Crysvita® revenue of $93 million and Dojolvi® revenue of $18 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
GTX-102 for Angelman syndrome: New longer-term data from Phase 1/2 clinical study support durable and improving effects across multiple domains;
Phase 3 data expected in the second half of 2026
NOVATO, Calif. – May 05, 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 March 31, 2026 and reaffirmed its financial guidance for 2026.
“This is an important year for Ultragenyx with two BLAs under review and our Angelman Phase 3 readout coming soon. We have the opportunity to meaningfully accelerate our consistent commercial revenue growth over the last few years as we prepare for two potential gene therapy approvals and launches in two urgent diseases without any approved therapies,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “The latest long-term GTX-102 Phase 1/2 data further support the potential of the program, as it heads toward Phase 3 results later this year.”
First Quarter 2026 Revenue Highlights and 2026 Revenue Guidance
Recent Clinical Milestones and 2026 Catalysts
The Phase 3 Aspire study, in patients with a full maternal UBE3A gene deletion, enrolled 129 patients, randomized 1:1 to GTX-102 or sham. Data from this study are expected in the second half of 2026.
Enrollment in the open-label Phase 2/3 Aurora study, evaluating GTX-102 in other genotypes and ages, began enrollment in October 2025 and is expected to complete enrollment in the second half of 2026.
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Per the protocol, the study is continuing to its second primary endpoint, which evaluates reduction in treatment burden, including use of ammonia scavengers and dietary management, across both the treatment and placebo-crossover groups following treatment with DTX301 through 64 weeks of follow-up. Data are expected in the first half of 2027.
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Summary of First Quarter 2026 Financial Results
Selected Financial Data (dollars in millions, except per share amounts), (unaudited)
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Three Months Ended March 31, |
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2026 |
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2025 |
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Total revenues |
$ |
136 |
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$ |
139 |
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Operating expenses: |
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Cost of sales |
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30 |
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29 |
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Research and development |
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187 |
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166 |
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Selling, general and administrative |
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88 |
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87 |
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Total operating expenses |
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305 |
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282 |
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Net loss |
$ |
(185 |
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$ |
(151 |
) |
Net loss per share, basic and diluted |
$ |
(1.84 |
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$ |
(1.57 |
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Operating Expenses
Total operating expenses for the first quarter 2026 were $305 million, including $30 million of non-cash stock-based compensation and $30 million of expense related to the restructuring announced last quarter. 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 first quarter 2026 was $185 million, or $1.84 per share basic and diluted, compared with a net loss for the first quarter 2025 of $151 million, or $1.57 per share basic and diluted.
Cash Balance and Net Cash Used in Operations
Cash, cash equivalents, and marketable securities were $534 million as of March 31, 2026. For the three months ended March 31, 2026, net cash used in operations was $197 million and includes the payment of annual bonuses and $38 million of payments related to UX143 manufacturing activities.
Conference Call and Webcast Information
Ultragenyx will host a conference call today, Tuesday, May 5, 2026, at 2 p.m. PT/5 p.m. ET to discuss the first quarter financial results and provide a corporate update. The live and 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.
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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 related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, including the company’s expectations for profitability in 2027, anticipated cost or expense reductions, including the company’s expectations related to benefits and savings from the strategic restructuring plan, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, the components and timing of regulatory submissions, the company’s ability to provide the requested documentation and address the comments in the CRL for UX111 to the satisfaction of the FDA, the timing of FDA review of the company’s BLA submissions, the timing and outcome of any FDA inspections related to UX111 or other clinical product candidates, the timing of future regulatory interactions related to the company’s clinical 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 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
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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 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) on February 18, 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 March 31, |
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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 |
$ |
46 |
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$ |
55 |
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Royalty revenue - U.S. and Canada |
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39 |
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41 |
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Royalty revenue - Europe |
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8 |
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7 |
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Total Crysvita Revenue |
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93 |
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103 |
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Dojolvi |
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18 |
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17 |
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Evkeeza |
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18 |
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11 |
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Mepsevii |
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7 |
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8 |
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Total revenues |
$ |
136 |
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$ |
139 |
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Ultragenyx Pharmaceutical Inc.
Selected Statement of Operations Financial Data
(in millions, except per share amounts)
(unaudited)
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Three Months Ended March 31, |
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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 |
$ |
89 |
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$ |
91 |
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Royalty revenue |
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47 |
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48 |
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Total revenues |
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136 |
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139 |
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Operating expenses: |
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Cost of sales |
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30 |
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29 |
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Research and development |
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187 |
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166 |
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Selling, general and administrative |
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88 |
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87 |
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Total operating expenses |
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305 |
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282 |
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Loss from operations |
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(169 |
) |
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(143 |
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Non-cash interest expense on liabilities for sales of future royalties |
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(21 |
) |
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(14 |
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Other income, net |
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6 |
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7 |
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Loss before income taxes |
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(184 |
) |
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(150 |
) |
Provision for income taxes |
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(1 |
) |
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(1 |
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Net loss |
$ |
(185 |
) |
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$ |
(151 |
) |
Net loss per share, basic and diluted |
$ |
(1.84 |
) |
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$ |
(1.57 |
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Shares used in computing net loss per share, basic and diluted |
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100.6 |
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96.3 |
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Ultragenyx Pharmaceutical Inc.
Selected Activity included in Operating Expenses
(in millions)
(unaudited)
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Three Months Ended March 31, |
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2026 |
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2025 |
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Non-cash stock-based compensation |
$ |
30 |
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$ |
40 |
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Restructuring expense |
$ |
30 |
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— |
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Ultragenyx Pharmaceutical Inc.
Selected Balance Sheet Financial Data
(in millions)
(unaudited)
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March 31, |
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December 31, |
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2026 |
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2025 |
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Balance Sheet Data: |
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Cash, cash equivalents, and marketable securities |
$ |
534 |
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$ |
737 |
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Working capital |
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332 |
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567 |
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Total assets |
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1,296 |
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1,532 |
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Total stockholders' equity (deficit) |
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(236 |
) |
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(80 |
) |
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