Ultragenyx Reports Second Quarter 2025 Financial Results and Corporate Update
Rhea-AI Summary
Ultragenyx (NASDAQ: RARE) reported Q2 2025 financial results with total revenue of $166 million, representing 13% growth year-over-year. Key revenue drivers include Crysvita at $120 million and Dojolvi at $23 million.
The company reaffirmed its 2025 guidance, expecting total revenue between $640-670 million. Despite a net loss of $115 million ($1.17 per share), Ultragenyx maintains its path to profitability target for 2027. Cash position stands at $539 million as of June 30, 2025.
Notable pipeline updates include: UX143 Phase 3 data for osteogenesis imperfecta expected by year-end, GTX-102 receiving FDA Breakthrough Therapy Designation for Angelman syndrome with Phase 3 fully enrolled, and planned BLA submission for DTX401 in Q4 2025.
Positive
- Revenue growth of 13% year-over-year to $166 million in Q2 2025
- Net loss improved to $115 million from $132 million in Q2 2024
- GTX-102 received FDA Breakthrough Therapy Designation for Angelman syndrome
- Phase 3 Aspire study for GTX-102 completed enrollment ahead of schedule
- UX143 demonstrated acceptable safety profile in Phase 3 interim analysis
Negative
- Operating expenses increased to $274 million from $263 million year-over-year
- Net cash used in operations increased to $275 million for H1 2025
- FDA issued Complete Response Letter for UX111 BLA requiring additional CMC information
- Net loss continues at $115 million despite revenue growth
- Increased cash burn expected compared to 2024 due to program delays
News Market Reaction
On the day this news was published, RARE declined 3.01%, reflecting a moderate negative market reaction. Our momentum scanner triggered 4 alerts that day, indicating moderate trading interest and price volatility. This price movement removed approximately $78M from the company's valuation, bringing the market cap to $2.52B at that time.
Data tracked by StockTitan Argus on the day of publication.
Second quarter total revenue of
Crysvita® revenue of
Reaffirm 2025 Revenue Guidance: Total revenue between
UX143 for osteogenesis imperfecta Phase 3 data from Orbit and Cosmic studies expected around the end of the year
GTX-102 for Angelman syndrome received Breakthrough Therapy Designation from FDA;
Phase 3 Aspire study fully enrolled
NOVATO, Calif., Aug. 05, 2025 (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 June 30, 2025.
“In the first half of the year, we delivered
Second Quarter 2025 Selected Financial Data Tables and Financial Results
| Revenues (dollars in thousands), (unaudited) | ||||||||||||||||
| Three Months Ended June 30, | Six Months Ended June 30, | |||||||||||||||
| 2025 | 2024 | 2025 | 2024 | |||||||||||||
| Crysvita | ||||||||||||||||
| Product sales – Latin America and Türkiye | $ | 34,727 | $ | 40,449 | $ | 89,807 | $ | 76,690 | ||||||||
| Royalty revenue – U.S. and Canada | 79,083 | 67,045 | 119,936 | 107,447 | ||||||||||||
| Royalty revenue – Europe | 6,596 | 6,176 | 13,528 | 12,118 | ||||||||||||
| Total Crysvita Revenue | 120,406 | 113,670 | 223,271 | 196,255 | ||||||||||||
| Dojolvi | 23,207 | 19,355 | 40,216 | 35,717 | ||||||||||||
| Evkeeza | 14,573 | 7,856 | 25,604 | 11,131 | ||||||||||||
| Mepsevii | 8,310 | 6,145 | 16,697 | 12,756 | ||||||||||||
| Total revenues | $ | 166,496 | $ | 147,026 | $ | 305,788 | $ | 255,859 | ||||||||
Total Revenues
Ultragenyx reported
| Selected Financial Data (dollars in thousands, except per share amounts), (unaudited) | ||||||||||||||||
| Three Months Ended June 30, | Six Months Ended June 30, | |||||||||||||||
| 2025 | 2024 | 2025 | 2024 | |||||||||||||
| Total revenues | $ | 166,496 | $ | 147,026 | $ | 305,788 | $ | 255,859 | ||||||||
| Operating expenses: | ||||||||||||||||
| Cost of sales | 23,002 | 21,280 | 51,664 | 38,813 | ||||||||||||
| Research and development | 164,736 | 161,503 | 330,508 | 339,990 | ||||||||||||
| Selling, general and administrative | 86,646 | 80,604 | 174,443 | 158,764 | ||||||||||||
| Total operating expenses | 274,384 | 263,387 | 556,615 | 537,567 | ||||||||||||
| Net loss | $ | (114,951 | ) | $ | (131,598 | ) | $ | (266,031 | ) | $ | (302,282 | ) | ||||
| Net loss per share, basic and diluted | $ | (1.17 | ) | $ | (1.52 | ) | $ | (2.73 | ) | $ | (3.54 | ) | ||||
Operating Expenses
Total operating expenses for the second quarter of 2025 were
Net Loss
For the second quarter of 2025, Ultragenyx reported net loss of
Cash Balance and Net Cash Used in Operations
Cash, cash equivalents, and marketable debt securities were
2025 Financial Guidance
Ultragenyx reaffirmed its revenue guidance for 2025. Total revenues are expected to grow approximately 14
Reaffirm for the full year 2025:
- Total revenue to be in the range of
$640 million to$670 million - Crysvita revenue to be in the range of
$460 million to$480 million - Dojolvi revenue to be in the range of
$90 million to$100 million
Recent Updates and Clinical Milestones
UX143 (setrusumab) monoclonal antibody for osteogenesis imperfecta (OI): Final analysis for Phase 3 Orbit and Cosmic studies around the end of 2025
The Phase 3 Orbit and Cosmic studies, which evaluate setrusumab in pediatric and young adult patients with OI, are progressing towards their final analyses around the end of 2025. The randomized, placebo-controlled Phase 3 portion of the Orbit study was evaluated by the Data Monitoring Committee at an interim analysis in July 2025 and they informed the company that UX143 demonstrated an acceptable safety profile and that the study should continue to the final analysis. Conduct of the study is going well and patient safety in the Phase 3 is consistent with the Phase 2.
Data from the Cosmic study were not analyzed at the interim timepoint, consistent with the statistical analysis plan. Study conduct is going well and safety in this younger patient population is consistent with the safety profile in the other studies.
Patients will continue dosing in the ongoing Phase 3 Orbit and Cosmic clinical studies with the final analyses to be conducted after patients have been on therapy for at least 18-months. The threshold for the Phase 3 Orbit final analysis is p<0.04 and for the Phase 3 Cosmic final analysis is p<0.05.
GTX-102 an antisense oligonucleotide for Angelman syndrome: Phase 3 study fully enrolled; Phase 3 data expected in the second half of 2026
In June 2025, Breakthrough Therapy Designation (BTD) was granted by the FDA for GTX-102 as a treatment for Angelman syndrome. The FDA’s decision was based on preliminary clinical evidence including positive data from the Phase 1/2 study in 74 patients (4-17 years of age) with a full maternal UBE3A gene deletion, that showed participants have made consistent developmental gains with rapid, sustained and continuing improvements across multiple symptom domains when treated for up to 3 years. BTD aims to expedite the development and review of drugs that are intended to treat serious or life-threatening diseases and whose preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on one or more clinically significant endpoints over existing therapies.
In July 2025, enrollment of the global Phase 3 Aspire study was completed, ahead of plan due to patient and investigator interest, with 129 patients screened and randomized across 28 global sites. Participants are randomized 1:1 to receive GTX-102 by intrathecal injection via lumbar puncture or to the sham comparator group during the 48-week primary efficacy analysis period. The primary endpoint is improvement in cognition assessed by Bayley-4 cognitive raw score, and the key secondary endpoint (with a
The Phase 2/3 Aurora study, which will evaluate GTX-102 in other Angelman syndrome genotypes and ages, is expected to initiate in the second half of 2025.
UX111 AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA): Working with FDA to resolve observations from Complete Response Letter (CRL)
In July 2025, the FDA issued a CRL for the Biologics License Application (BLA) for UX111 requesting additional information and improvements related to specific aspects of chemistry, manufacturing and controls (CMC) procedures and validation as well as observations from the recently completed manufacturing facility inspections. The company believes the observations are readily addressable and many have been addressed. The company will work with the FDA through a Type A meeting to agree on the planned resolution of the observations. Once agreement on the contents of a filing have been reached, the company expects to resubmit the BLA and anticipates up to a 6-month review period to follow the resubmission.
Clinical review had been ongoing and the FDA has acknowledged that the neurodevelopmental outcome data provided to date are robust and the biomarker data provide additional supportive evidence. The CRL did not note any review issues related to the clinical data package nor clinical inspections and specified that updated clinical data for particular endpoints from the current patients be included in the resubmission.
DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa): BLA submission expected in the fourth quarter of 2025
A BLA for DTX401 for the treatment of GSDIa is planned to be submitted in the fourth quarter of 2025. The BLA will include data from the randomized, placebo controlled Phase 3 study and the previously disclosed 96-week data that demonstrated patients had even greater reductions in total daily cornstarch at their last visit compared to baseline in both the ongoing DTX401 group (-
UX701 AAV gene therapy for Wilson Disease: Phase 1/2/3 study ongoing; Cohort 4 enrollment ongoing, completion expected in second half of 2025
Enrollment is ongoing in the fourth cohort evaluating a 4.0e13 GC/kg dose in the ongoing, dose-finding, stage of the pivotal Cyprus2+ study of UX701 for the treatment of Wilson disease. The company is on track to enroll five patients in Cohort 4 who will receive immunomodulation therapy with rituximab and tacrolimus, in addition to the prophylactic oral corticosteroid regimen patients in Cohorts 1 through 3 received, prior to being dosed with UX701. Enrollment in Cohort 4 is expected to complete in the second half of 2025.
Conference Call and Webcast Information
Ultragenyx will host a conference call today, Tuesday, August 5, 2025, at 2 p.m. PT/5 p.m. ET to discuss the second quarter 2025 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.
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, anticipated cost or expense reductions, 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 to the satisfaction of the FDA, the timing of resubmission of the BLA and the timing of FDA review of any such resubmission, the timing and outcome of any FDA inspections related to UX111, the timing of future regulatory interactions related to UX111 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 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, 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 7, 2025, 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 Statement of Operations Financial Data | ||||||||||||||||
| (in thousands, except share and per share amounts) | ||||||||||||||||
| (unaudited) | ||||||||||||||||
| Three Months Ended June 30, | Six Months Ended June 30, | |||||||||||||||
| 2025 | 2024 | 2025 | 2024 | |||||||||||||
| Statement of Operations Data: | ||||||||||||||||
| Revenues: | ||||||||||||||||
| Product sales | $ | 80,817 | $ | 73,805 | $ | 172,324 | $ | 136,294 | ||||||||
| Royalty revenue | 85,679 | 73,221 | 133,464 | 119,565 | ||||||||||||
| Total revenues | 166,496 | 147,026 | 305,788 | 255,859 | ||||||||||||
| Operating expenses: | ||||||||||||||||
| Cost of sales | 23,002 | 21,280 | 51,664 | 38,813 | ||||||||||||
| Research and development | 164,736 | 161,503 | 330,508 | 339,990 | ||||||||||||
| Selling, general and administrative | 86,646 | 80,604 | 174,443 | 158,764 | ||||||||||||
| Total operating expenses | 274,384 | 263,387 | 556,615 | 537,567 | ||||||||||||
| Loss from operations | (107,888 | ) | (116,361 | ) | (250,827 | ) | (281,708 | ) | ||||||||
| Change in fair value of equity investments | (9 | ) | (3,991 | ) | (166 | ) | (245 | ) | ||||||||
| Non-cash interest expense on liabilities for sales of future royalties | (14,041 | ) | (15,960 | ) | (28,383 | ) | (31,807 | ) | ||||||||
| Other income, net | 7,934 | 5,572 | 15,602 | 12,791 | ||||||||||||
| Loss before income taxes | (114,004 | ) | (130,740 | ) | (263,774 | ) | (300,969 | ) | ||||||||
| Provision for income taxes | (947 | ) | (858 | ) | (2,257 | ) | (1,313 | ) | ||||||||
| Net loss | $ | (114,951 | ) | $ | (131,598 | ) | $ | (266,031 | ) | $ | (302,282 | ) | ||||
| Net loss per share, basic and diluted | $ | (1.17 | ) | $ | (1.52 | ) | $ | (2.73 | ) | $ | (3.54 | ) | ||||
| Shares used in computing net loss per share, basic and diluted | 98,460,445 | 86,580,516 | 97,381,745 | 85,433,443 | ||||||||||||
| Ultragenyx Pharmaceutical Inc. Selected Activity included in Operating Expenses (in thousands) (unaudited) | ||||||||||||||||
| Three Months Ended June 30, | Six Months Ended June 30, | |||||||||||||||
| 2025 | 2024 | 2025 | 2024 | |||||||||||||
| Non-cash stock-based compensation | $ | 38,615 | $ | 39,363 | $ | 78,525 | $ | 76,297 | ||||||||
| Ultragenyx Pharmaceutical Inc. | ||||||||
| Selected Balance Sheet Financial Data | ||||||||
| (in thousands) | ||||||||
| (unaudited) | ||||||||
| June 30, | December 31, | |||||||
| 2025 | 2024 | |||||||
| Balance Sheet Data: | ||||||||
| Cash, cash equivalents, and marketable debt securities | $ | 539,039 | $ | 745,029 | ||||
| Working capital | 426,544 | 472,970 | ||||||
| Total assets | 1,306,265 | 1,503,456 | ||||||
| Total stockholders' equity | 151,286 | 255,297 | ||||||
Contacts Ultragenyx Pharmaceutical Inc.
Investors
Joshua Higa
ir@ultragenyx.com
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