Ultragenyx Reports Third Quarter 2026 Financial Results, Announces Substantial Expense Reductions, and Reaffirms Profitability in 2027
The company expects 2027 research and development expenses to fall at least 50% versus 2025 as it shifts toward commercial execution.
Sentiment and the balance of points
Rhea-AI Sentiment reads the wording of the document, how positive or negative its language is on a 1 to 5 scale. The balance of points shown with the takes weighs what the document actually discloses, so the two can disagree, for example when a trial that missed its main goal is described in upbeat language.
Rhea-AI Summary
Ultragenyx Pharmaceutical (NASDAQ: RARE) reported third-quarter revenue of $173 million and reaffirmed its expectation of full-year profitability in 2027. Revenue rose from $160 million a year earlier; net loss narrowed to $118 million, or $1.16 per share, from $180 million, or $1.81. Full-year 2026 revenue guidance remains $730 million to $760 million, excluding new launches.
The FDA approved GENGLYCOS and FAYUVI in the third quarter. Agreements to sell their priority review vouchers total $413 million; one sale has closed. Ultragenyx expects approximately $350 million in combined net proceeds. The company expects 2027 research and development expenses below $350 million and selling, general and administrative expenses below $375 million. Actions include an approximately 20% workforce reduction and ending GTX-102 studies and UX701 development. Fourth-quarter restructuring charges are expected to be approximately $25 million.
How this balance works
Rhea-AI gives every point it takes from this document a weight. Minor counts 1, Moderate 3 and Major 9, so one Major point outweighs several Minor ones. The bar adds up the weights on each side, and when neither side holds more than 65% of the total the balance reads Mixed.
It reads the document as published, with the same rules for every company, and it does not look at what the market expected or at how the stock traded, so a point can be objectively good on a day the stock falls.
Rhea-AI Sentiment measures something else, the tone of the wording.
Hollow bars mark forward-looking points. How the balance works
Positive
- Major pointGENGLYCOS priority review voucher sale closed for $210 million. 13% of market cap
- Major point. Forward-looking: it has not happened yet and may not happen.FAYUVI priority review voucher sale agreement provides for $203 million; closing is expected later in November. 13% of market cap
- Moderate pointThird-quarter revenue rose to $173 million from $160 million a year earlier.
- Moderate pointThird-quarter net loss narrowed to $118 million, or $1.16 per share, from $180 million, or $1.81.
- Moderate point. Forward-looking: it has not happened yet and may not happen.Ultragenyx continues to expect full-year GAAP profitability in 2027 and going forward.
- Moderate pointGENGLYCOS received FDA accelerated approval in August 2026 for GSDIa patients eight years and older.
- Moderate pointFAYUVI received FDA standard full approval in September 2026 for pediatric Sanfilippo syndrome type A.
- Moderate point. Forward-looking: it has not happened yet and may not happen.2027 R&D expenses are expected below $350 million, at least 50% below 2025.
- Moderate point. Forward-looking: it has not happened yet and may not happen.2027 selling, general and administrative expenses are expected below $375 million.
- Moderate pointThird-quarter R&D expenses fell to $154 million from $216 million a year earlier.
- Moderate pointDTX301 Phase 3 results showed improved ammonia control, reduced scavenger-medicine needs and improved global impression scores.
- Moderate pointUX143 Phase 3 analyses showed benefits in bone mineral density, fractures and other endpoints, in the company's assessment.
20 minor points
- Minor point. Forward-looking: it has not happened yet and may not happen.Full-year 2026 revenue guidance remains $730 million to $760 million, excluding revenue from new product launches.
- Minor point. Forward-looking: it has not happened yet and may not happen.Combined voucher-sale net proceeds are expected to be approximately $350 million.
- Minor pointCost reductions include approximately 20% fewer employees, leaving approximately 1,000, and a smaller real estate footprint.
- Minor pointThird-quarter DOJOLVI revenue rose to $30 million from $24 million a year earlier.
- Minor pointThird-quarter EVKEEZA revenue rose to $24 million from $17 million a year earlier.
- Minor pointThird-quarter MEPSEVII revenue rose to $10 million from $7 million a year earlier.
- Minor point. Forward-looking: it has not happened yet and may not happen.Full-year 2026 CRYSVITA revenue guidance remains $500 million to $520 million.
- Minor point. Forward-looking: it has not happened yet and may not happen.Full-year 2026 DOJOLVI revenue guidance remains $100 million to $110 million.
- Minor pointThird-quarter operating loss narrowed to $110 million from $171 million a year earlier.
- Minor pointNine-month revenue rose to $523 million from $466 million a year earlier.
- Minor pointNine-month CRYSVITA revenue rose to $358 million from $336 million a year earlier.
- Minor pointNine-month DOJOLVI revenue rose to $75 million from $64 million a year earlier.
- Minor pointNine-month EVKEEZA revenue rose to $63 million from $42 million a year earlier.
- Minor pointNine-month MEPSEVII revenue rose to $27 million from $24 million a year earlier.
- Minor pointNine-month net loss narrowed to $395 million, or $3.89 per share, from $446 million, or $4.55.
- Minor pointNine-month R&D expenses fell to $508 million from $547 million a year earlier.
- Minor pointNine-month operating loss narrowed to $354 million from $422 million a year earlier.
- Minor pointNon-cash stock compensation fell to $34 million quarterly and $98 million year-to-date, from $37 million and $116 million.
- Minor point. Forward-looking: it has not happened yet and may not happen.DTX301 second primary endpoint data are expected in the first half of 2027.
- Minor point. Forward-looking: it has not happened yet and may not happen.UX016 Phase 1/2 costs are fully externally funded through clinical proof-of-concept; dosing is expected to begin soon.
Negative
- Moderate pointCash, cash equivalents and marketable securities fell to $401 million from $737 million at December 31, 2025.
- Moderate pointThird-quarter operations used $43 million in cash.
- Moderate point. Forward-looking: it has not happened yet and may not happen.UX701 development will be terminated after failing criteria for advancing to pivotal Phase 3 development.
- Moderate pointGTX-102 studies for Angelman syndrome are being wound down.
- Moderate pointUX143's path to a regulatory filing remains undetermined, pending further engagement with regulatory agencies.
- Moderate pointThird-quarter CRYSVITA revenue declined to $109 million from $112 million a year earlier.
- Moderate pointWorking capital declined to $155 million from $567 million at December 31, 2025.
10 minor points
- Minor point. Forward-looking: it has not happened yet and may not happen.Fourth-quarter severance and other non-recurring restructuring charges are expected at approximately $25 million.
- Minor pointFAYUVI voucher sale remains pending closing.
- Minor pointThird-quarter cost of sales rose to $32 million from $28 million a year earlier.
- Minor pointThird-quarter selling, general and administrative expenses rose to $97 million from $87 million a year earlier.
- Minor pointNine-month cost of sales rose to $96 million from $80 million a year earlier.
- Minor pointNine-month selling, general and administrative expenses rose to $273 million from $261 million a year earlier.
- Minor pointNine-month restructuring expense was $30 million, versus none a year earlier.
- Minor pointNon-cash royalty-liability interest expense rose to $20 million quarterly and $63 million year-to-date, from $14 million and $42 million.
- Minor pointTotal assets declined to $1,187 million from $1,532 million at December 31, 2025.
- Minor pointStockholders' deficit widened to $376 million from $80 million at December 31, 2025.
News Explained
The FAYUVI voucher sale remains pending; September 30, 2026 cash, equivalents, and marketable securities were $401 million.
The company has initiated headcount and expense reductions, with the reduction in force expected to be substantially complete by
The GENGLYCOS PRV sale closed last week, while the FAYUVI PRV sale is expected to close later this month, so the second agreed sale remains pending.
At
Details
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Key Figures
- Q3 total revenue
- $173 million
- Third quarter 2026
- 2026 total revenue guidance
- $730 million to $760 million
- Full year 2026; excludes revenue from new product launches
- Net loss
- $118 million
- Third quarter 2026
- Cash, cash equivalents, and marketable securities
- $401 million
- As of September 30, 2026
- PRV sale agreements
- $413 million
- Combined sales price for two vouchers
- 2027 R&D expenses
- Less than $350 million
- Company expectation following expense reductions
- 2027 SG&A expenses
- Less than $375 million
- Company expectation following expense reductions
- Net cash used in operations
- $43 million
- Three months ended September 30, 2026
Previous Earnings Reports
-
Reported $214 million revenue and reaffirmed 2026 revenue guidance.
-
Reported $136 million revenue and reaffirmed the 2026 guidance range.
-
Reported year-over-year revenue growth and reaffirmed full-year revenue guidance.
24h Move is the share-price change in the day after each event; other market factors may also have contributed.
Key Terms
priority review voucher regulatory
aav8 technical
aav9 technical
AI-generated analysis. How Rhea-AI works. Not financial advice.
Third quarter total revenue of
GENGLYCOS™ and FAYUVI™ BLAs approved by FDA in the third quarter
Two Priority Review Vouchers (PRVs) under agreement for combined sales price of
Substantial reduction of expenses and headcount underway, expected to drive at least a
NOVATO, Calif., Nov. 03, 2026 (GLOBE NEWSWIRE) -- 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 September 30, 2026, announced substantial expense reductions, and reaffirmed its guidance for 2026 revenue and 2027 profitability.
“We received two BLA approvals from the FDA within one month for our first gene therapy treatments and the launches are progressing well with growing demand for these products,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “With substantial reductions in headcount and expenses, combined with two launches and continued growth in our base business, we expect meaningful profitability in 2027 and going forward.”
Third Quarter 2026 Revenue Highlights and 2026 Revenue Guidance
- Total revenue in the third quarter 2026 was
$173 million . The company reaffirms its full year 2026 total revenue guidance of$730 million to$760 million , which excludes revenue from new product launches. - CRYSVITA revenue in the third quarter 2026 was
$109 million , consistent with expected ordering patterns. The company reaffirms its full year 2026 Crysvita revenue guidance of$500 million to$520 million . - DOJOLVI revenue in the third quarter 2026 was
$30 million . The company reaffirms its full year 2026 Dojolvi revenue guidance of$100 million to$110 million . - EVKEEZA® revenue in the third quarter 2026 was
$24 million , driven by increased demand from new country launches and early access. - MEPSEVII® revenue in the third quarter 2026 was
$10 million .
Summary of Third Quarter 2026 Financial Results
| Selected Financial Data (dollars in millions, except per share amounts), (unaudited) | |||||||||||||||
| Three Months Ended September 30, | Nine Months Ended September 30, | ||||||||||||||
| 2026 | 2025 | 2026 | 2025 | ||||||||||||
| Total revenues | $ | 173 | $ | 160 | $ | 523 | $ | 466 | |||||||
| Operating expenses: | |||||||||||||||
| Cost of sales | 32 | 28 | 96 | 80 | |||||||||||
| Research and development | 154 | 216 | 508 | 547 | |||||||||||
| Selling, general and administrative | 97 | 87 | 273 | 261 | |||||||||||
| Total operating expenses | 283 | 331 | 877 | 888 | |||||||||||
| Net loss | $ | (118 | ) | $ | (180 | ) | $ | (395 | ) | $ | (446 | ) | |||
| Net loss per share, basic and diluted | $ | (1.16 | ) | $ | (1.81 | ) | $ | (3.89 | ) | $ | (4.55 | ) | |||
Operating Expenses
Total operating expenses for the third quarter 2026 were
Net Loss
Net loss for the third quarter 2026 was
Cash Balance and Net Cash Used in Operations
Cash, cash equivalents, and marketable securities were
In October 2026, the Company entered into two definitive agreements to sell the PRVs received upon the approval of GENGLYCOS and FAYUVI for a combined price of
Initiation of substantial expense reductions and reaffirming profitability in 2027
With the recent completion of late-stage development programs, including first-ever FDA approvals in glycogen storage disease type Ia (GSDIa) and Sanfilippo syndrome type A, Ultragenyx is entering a new stage focused on commercial execution and a smaller, more targeted R&D footprint. To support this transition, the company has initiated substantial headcount and expense reductions and expects to reduce 2027 R&D expenses to less than
- Reducing headcount by approximately
20% , resulting in a revised workforce of approximately 1,000 employees - Completing wind-down of all ongoing studies for GTX-102 for Angelman syndrome
- Terminating UX701 for Wilson disease based on not achieving study goals
- Reducing expenses across the company, including reduction of real estate footprint
In addition to executing these critical expense reductions, the company is focused on driving successful launches for GENGLYCOS and FAYUVI and continuing to grow current product revenues. R&D investments will be focused on delivering primary data from the responder endpoint of the ongoing Phase 3 study for DTX301 for ornithine transcarbamylase deficiency, determining if there is a viable path forward for UX143 (setrusumab) for osteogenesis imperfecta, and measured spending to progress early-stage programs for future INDs.
Fourth quarter 2026 financial results are expected to include approximately
Recent Corporate Updates and Upcoming Milestones
- GENGLYCOS (pariglasgene brecaparvovec) AAV8 gene therapy for the treatment of glycogen storage disease type Ia (GSDIa): In August 2026, the U.S. Food and Drug Administration (FDA) granted accelerated approval for GENGLYCOS as a treatment for adult and pediatric patients eight years and older with GSDIa. The company received a PRV upon this approval and sold it for
$210 million . - FAYUVI (rebisufligene etisparvovec) AAV9 gene therapy for the treatment of Sanfilippo syndrome type A (MPS IIIA): In September 2026, the FDA granted standard full approval for FAYUVI as a treatment of pediatric patients with Sanfilippo syndrome Type A. The company received a PRV upon this approval and, in October 2026, entered into a definitive agreement to sell the PRV for
$203 million . - UX701 (rivunatpagene miziparvovec) AAV9 gene therapy for the treatment of Wilson disease: Additional data in the dose-finding stage of the Cyprus2+ study of UX701 for the treatment of Wilson disease, including data from the fourth dosing cohort, demonstrated clinical activity in some patients, but did not meet the criteria that the majority of patients achieve biochemical response and elimination of standard of care, to advance the program to the pivotal Phase 3, stage of development. UX701 demonstrated a favorable safety profile across all cohorts dosed. Data from the completed study is expected to be presented at an upcoming medical or scientific congress.
The company will terminate further development of this program and is working with clinical sites and patient advocacy groups to support the patient community through this transition.
- UX016 novel prodrug for sialic acid used as a substrate replacement therapy for the treatment of GNE myopathy: The Phase 1/2 study is evaluating safety and dose of UX016 and its impact on muscle strength in adults with GNEM. The UX016 program is fully funded through clinical proof-of-concept by an external venture philanthropy agreement with a patient group, which supports all costs for the Phase 1/2 study and dosing will be initiated soon.
- DTX301 (avalotcagene ontaparvovec) AAV8 gene therapy for the treatment of Ornithine Transcarbamylase, or OTC, deficiency: The Phase 3 Enh3ance placebo-controlled study showed substantial improvement in ammonia control and reduction in the need for scavenger medicines along with profound improvement in global impression scale. The study continues with patients in both treatment and cross-over groups progressing through 64 weeks of follow-up to evaluate the second primary endpoint of the reduction in the use of ammonia scavengers and dietary management. Data are expected in the first half of 2027.
- UX143 (setrusumab) monoclonal antibody for the treatment of osteogenesis imperfecta: Phase 3 data with pre- and post-hoc analyses were presented at the American Society for Bone and Mineral Research 2026 Annual Meeting showing a meaningful benefit on bone mineral density, fractures, and other endpoints in the two Phase 3 studies. The company will continue to engage regulatory agencies to determine the necessary data to support a regulatory filing and if there is a potential path forward for UX143.
Conference Call and Webcast Information
Ultragenyx will host a conference call today, Tuesday, November 3, 2026, at 2 p.m. PT/5 p.m. ET to discuss the third 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.
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.
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 2027, the expected timing of closing and net proceeds from the sales of its PRVs, the anticipated scope, timing, costs, benefits and savings of its headcount and expense reductions, including expected severance and other restructuring charges and the expected timing of completion of the reduction in force, and the timing and sustainability of profitability, including expected full-year GAAP profitability in 2027 and going forward; the timing, progress, results and plans for its clinical programs and studies, including the wind-down of the GTX-102 studies, the termination of the UX701 program, the anticipated timing of data from the Phase 3 Enh3ance study of DTX301, engagement with regulatory agencies regarding UX143 and whether there is a potential path forward for UX143, and initiation of dosing in the UX016 study; Ultragenyx’s ability to successfully launch and commercialize GENGLYCOS and FAYUVI, demand for these products, and Ultragenyx’s ability to satisfy post-marketing requirements, including confirmatory study requirements associated with the accelerated approval of GENGLYCOS; 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 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, the risk that the FDA may withdraw the accelerated approval of GENGLYCOS if confirmatory studies do not verify clinical benefit, risks related to serious or undesirable side effects of our products and 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 launching and commercializing gene therapy products, 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, the risk that the PRV sales may not close on the expected timeline or at all, delays or unexpected costs and other adverse effects related to the headcount and expense reductions, including disruption to our operations and our ability to retain key employees, 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 marketable securities 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 product candidates. 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 August 5, 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-/).
| Ultragenyx Pharmaceutical Inc. Selected Revenue Data (in millions) (unaudited) | |||||||||||
| Three Months Ended September 30, | Nine Months Ended September 30, | ||||||||||
| 2026 | 2025 | 2026 | 2025 | ||||||||
| CRYSVITA | |||||||||||
| Product sales - Latin America and Türkiye | $ | 41 | $ | 47 | $ | 141 | $ | 137 | |||
| Royalty revenue - U.S. and Canada | 61 | 57 | 194 | 177 | |||||||
| Royalty revenue - Europe | 7 | 8 | 23 | 22 | |||||||
| Total CRYSVITA Revenue | 109 | 112 | 358 | 336 | |||||||
| DOJOLVI | 30 | 24 | 75 | 64 | |||||||
| EVKEEZA | 24 | 17 | 63 | 42 | |||||||
| MEPSEVII | 10 | 7 | 27 | 24 | |||||||
| Total revenues | $ | 173 | $ | 160 | $ | 523 | $ | 466 | |||
| Ultragenyx Pharmaceutical Inc. | |||||||||||||||
| Selected Statement of Operations Financial Data | |||||||||||||||
| (in millions, except per share amounts) | |||||||||||||||
| (unaudited) | |||||||||||||||
| Three Months Ended September 30, | Nine Months Ended September 30, | ||||||||||||||
| 2026 | 2025 | 2026 | 2025 | ||||||||||||
| Statement of Operations Data: | |||||||||||||||
| Revenues: | |||||||||||||||
| Product sales | $ | 105 | $ | 95 | $ | 306 | $ | 267 | |||||||
| Royalty revenue | 68 | 65 | 217 | 199 | |||||||||||
| Total revenues | 173 | 160 | 523 | 466 | |||||||||||
| Operating expenses: | |||||||||||||||
| Cost of sales | 32 | 28 | 96 | 80 | |||||||||||
| Research and development | 154 | 216 | 508 | 547 | |||||||||||
| Selling, general and administrative | 97 | 87 | 273 | 261 | |||||||||||
| Total operating expenses | 283 | 331 | 877 | 888 | |||||||||||
| Loss from operations | (110 | ) | (171 | ) | (354 | ) | (422 | ) | |||||||
| Non-cash interest expense on liabilities for sales of future royalties | (20 | ) | (14 | ) | (63 | ) | (42 | ) | |||||||
| Other income, net | 3 | 6 | 15 | 21 | |||||||||||
| Loss before income taxes | (127 | ) | (179 | ) | (402 | ) | (443 | ) | |||||||
| Benefit from (provision for) income taxes | 9 | (1 | ) | 7 | (3 | ) | |||||||||
| Net loss | $ | (118 | ) | $ | (180 | ) | $ | (395 | ) | $ | (446 | ) | |||
| Net loss per share, basic and diluted | $ | (1.16 | ) | $ | (1.81 | ) | $ | (3.89 | ) | $ | (4.55 | ) | |||
| Shares used in computing net loss per share, basic and diluted | 102.1 | 99.8 | 101.6 | 98.2 | |||||||||||
| Ultragenyx Pharmaceutical Inc. Selected Activity included in Operating Expenses (in millions) (unaudited) | |||||||||||
| Three Months Ended September 30, | Nine Months Ended September 30, | ||||||||||
| 2026 | 2025 | 2026 | 2025 | ||||||||
| Non-cash stock-based compensation | $ | 34 | $ | 37 | $ | 98 | $ | 116 | |||
| Restructuring expense | — | — | 30 | — | |||||||
| Ultragenyx Pharmaceutical Inc. | ||||||||
| Selected Balance Sheet Financial Data | ||||||||
| (in millions) | ||||||||
| (unaudited) | ||||||||
| September 30, | December 31, | |||||||
| 2026 | 2025 | |||||||
| Balance Sheet Data: | ||||||||
| Cash, cash equivalents, and marketable securities | $ | 401 | $ | 737 | ||||
| Working capital | 155 | 567 | ||||||
| Total assets | 1,187 | 1,532 | ||||||
| Total stockholders' deficit | (376 | ) | (80 | ) | ||||
Contacts Ultragenyx Pharmaceutical Inc.
Investors
Joshua Higa
ir@ultragenyx.com
Media
Jess Rowlands
media@ultragenyx.com
FAQ
AI-generated questions and answers. How Rhea-AI works. Not financial advice.
What were Ultragenyx's third-quarter 2026 revenue and earnings results?
Ultragenyx reported $173 million in revenue and a net loss of $118 million, or $1.16 per share, for the quarter ended September 30, 2026. A year earlier, revenue was $160 million and net loss was $180 million, or $1.81 per share.
Why is Ultragenyx terminating UX701 development for Wilson disease?
UX701 did not meet the advancement criteria requiring a majority of patients to achieve biochemical response and elimination of standard-of-care treatment. Additional dose-finding data showed clinical activity in some patients, and the company described safety across dosed cohorts as favorable. Ultragenyx is working with clinical sites and patient advocacy groups during the transition.
When does Ultragenyx expect the next DTX301 Phase 3 data?
Ultragenyx expects data on DTX301's second primary endpoint in the first half of 2027. Patients in the treatment and crossover groups are progressing through 64 weeks of follow-up to assess reductions in ammonia-scavenger medicines and dietary management for ornithine transcarbamylase deficiency.
When will Ultragenyx complete its 2026 workforce reduction?
The workforce reduction is expected to be substantially completed by the end of 2026. Ultragenyx plans to reduce headcount by approximately 20%, resulting in a revised workforce of approximately 1,000 employees.