Ultragenyx Reports Fourth Quarter and Full Year 2024 Financial Results and Corporate Update
Rhea-AI Summary
Ultragenyx Pharmaceutical (NASDAQ: RARE) reported strong financial results for Q4 and full year 2024, with total revenue of $560 million, representing 29% growth compared to 2023. Key revenue drivers included Crysvita at $410 million (+25% YoY) and Dojolvi at $88 million (+25% YoY).
The company provided 2025 guidance projecting total revenue between $640-670 million, with Crysvita revenue of $460-480 million and Dojolvi revenue of $90-100 million. Despite revenue growth, Ultragenyx reported a net loss of $569 million ($6.29 per share) for 2024, though improved from $607 million loss in 2023.
The company ended 2024 with $745 million in cash and equivalents, with net cash used in operations of $414 million for the year. Multiple clinical milestones are expected in 2025, including a PDUFA decision for Sanfilippo syndrome gene therapy and Phase 3 results in osteogenesis imperfecta.
Positive
- Total revenue grew 29% YoY to $560 million in 2024
- Crysvita revenue increased 25% to $410 million
- Dojolvi revenue grew 25% to $88 million
- Strong 2025 guidance projecting 14-20% revenue growth
- Cash position remains solid at $745 million
Negative
- Net loss of $569 million in 2024
- Operating expenses increased to $1.096 billion in 2024
- Significant cash burn with $414 million used in operations
News Market Reaction
On the day this news was published, RARE declined 2.37%, reflecting a moderate negative market reaction.
Data tracked by StockTitan Argus on the day of publication.
2024 Total Revenue of
Crysvita® revenue of
2025 Financial Guidance: Total Revenue between
NOVATO, Calif., Feb. 13, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today reported its financial results for the quarter and full year ended December 31, 2024 and shared financial guidance for 2025.
“We have created a next-generation rare disease company on a pathway to profitability with meaningful revenue growth from multiple global products and a series of potential new drug approvals,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “The major regulatory and clinical catalysts ahead of us this year are the pending PDUFA decision for our gene therapy to treat Sanfilippo syndrome, submission of our second gene therapy biologics license application in Glycogen Storage Disease Type Ia, readout of our pivotal Phase 3 results in osteogenesis imperfecta, and completion of enrollment in our Phase 3 trial in Angelman syndrome."
Fourth Quarter and Full Year 2024 Selected Financial Data Tables and Financial Results
| Revenues (dollars in thousands), (unaudited) | |||||||||||||||
| Three Months Ended December 31, | Year Ended December 31, | ||||||||||||||
| 2024 | 2023 | 2024 | 2023 | ||||||||||||
| Crysvita | |||||||||||||||
| Product sales | $ | 22,415 | $ | 18,379 | $ | 134,709 | $ | 75,697 | |||||||
| Revenue in Profit-Share Territory | 85,534 | 70,124 | 248,966 | 231,574 | |||||||||||
| Royalty revenue in European Territory | 7,473 | 5,612 | 25,849 | 20,783 | |||||||||||
| Total Crysvita Revenue | 115,422 | 94,115 | 409,524 | 328,054 | |||||||||||
| Dojolvi | 31,103 | 23,286 | 88,194 | 70,633 | |||||||||||
| Evkeeza | 10,374 | 2,102 | 32,162 | 3,642 | |||||||||||
| Mepsevii | 7,978 | 7,889 | 30,350 | 30,441 | |||||||||||
| Other | — | — | — | 1,479 | |||||||||||
| Total revenues | $ | 164,877 | $ | 127,392 | $ | 560,230 | $ | 434,249 | |||||||
Total Revenues
Ultragenyx reported
Total revenue for the year ended December 31, 2024 was
| Selected Financial Data (dollars in thousands, except per share amounts), (unaudited) | |||||||||||||||
| Three Months Ended December 31, | Year Ended December 31, | ||||||||||||||
| 2024 | 2023 | 2024 | 2023 | ||||||||||||
| Total revenues | $ | 164,877 | $ | 127,392 | $ | 560,230 | $ | 434,249 | |||||||
| Operating expenses: | |||||||||||||||
| Cost of sales | 16,894 | 12,051 | 76,728 | 45,209 | |||||||||||
| Research and development | 187,766 | 160,557 | 697,865 | 648,449 | |||||||||||
| Selling, general and administrative | 82,495 | 76,833 | 321,610 | 309,799 | |||||||||||
| Total operating expenses | 287,155 | 249,441 | 1,096,203 | 1,003,457 | |||||||||||
| Net loss | $ | (133,385 | ) | $ | (123,190 | ) | $ | (569,183 | ) | $ | (606,639 | ) | |||
| Net loss per share, basic and diluted | $ | (1.39 | ) | $ | (1.52 | ) | $ | (6.29 | ) | $ | (8.25 | ) | |||
Operating Expenses
Total operating expenses for the fourth quarter of 2024 were
Net Loss
For the fourth quarter of 2024, Ultragenyx reported net loss of
Cash Balance and Net Cash Used in Operations
Cash, cash equivalents, and marketable debt securities were
2025 Financial Guidance
Revenues are expected to grow approximately 14
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): Phase 3 Orbit study progressing to second interim analysis (IA2) expected in mid-2025
Patients continue dosing in the ongoing Phase 3 Orbit and Cosmic clinical trials, which evaluate setrusumab in pediatric and young adult patients with OI. The randomized, placebo-controlled Phase 3 portion of the Orbit study is progressing towards the second interim analysis in mid-2025 or a final analysis in the fourth quarter 2025. Conduct of the study is going well and patient safety in the Phase 3 is consistent with the Phase 2. Patients in the Cosmic study also are continuing to be treated with either setrusumab or intravenous bisphosphonates (IV-BP) therapy and will be evaluated in parallel with the Orbit interim and final analyses.
GTX-102 an antisense oligonucleotide for Angelman syndrome: Phase 3 study enrolling; expect enrollment completion in second half of 2025
Enrollment in the global Phase 3 Aspire study began in December 2024 and is expected to enroll approximately 120 children ages four to 17 with Angelman syndrome with a genetically confirmed diagnosis of full maternal UBE3A gene deletion. Participants will be 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 will be 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 2025.
UX111 AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA): Biologics license application (BLA) submitted; expect Prescription Drug User Fee Act (PDUFA) decision on the application and potential launch in second half of 2025
In December 2024, Ultragenyx submitted a BLA to the U.S. Food and Drug Administration for UX111 supported by the available data, including from the ongoing pivotal Transpher A study. New clinical data were recently presented at WORLDSymposium™ 2025, that demonstrated treatment with UX111 led to a statistically significant improvement in the Bayley-III raw scores for the subdomains of cognition, receptive communication and expressive communication in patients with MPS IIIA compared to natural history data from untreated patients. These clinical endpoints were correlated with substantial and sustained reduction in levels of heparan sulfate in cerebrospinal fluid. A PDUFA decision and potential launch are expected in the second half of 2025.
DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa): BLA filing expected in mid-2025
Ultragenyx previously announced positive topline results from the Phase 3 GlucoGene study for the treatment of patients aged eight years and older. The study achieved its primary endpoint, demonstrating that treatment with DTX401 resulted in a statistically significant and clinically meaningful reduction in daily cornstarch intake compared with placebo at Week 48.
After the 48-week primary efficacy analysis period, crossover patients (previously treated with placebo) were eligible to receive DTX401. These patients were able to titrate cornstarch much more rapidly once they were confirmed to have been treated and had timely direct access to their glucose levels. Patients from the original DTX401 treatment arm who have reached 78 weeks also continued to reduce their daily cornstarch intake, while maintaining glycemic control. DTX401 has demonstrated a consistent and acceptable safety profile with no new safety concerns identified as of the data cut-off.
These results have been discussed with regulatory authorities in a pre-BLA meeting and will be included as part of a BLA submission in mid-2025.
DTX301 AAV gene therapy for Ornithine Transcarbamylase (OTC) Deficiency: Enrollment in Phase 3 study complete
Enrollment in the Phase 3 Enh3ance study has been completed with a total of 37 patients. The pivotal study enrolled participants 12 years of age and older, randomized 1:1 to DTX301 or placebo. The primary endpoints are response as measured by change in 24-hour ammonia levels and removal of ammonia-scavenger medications and protein-restricted diet. Based on the recently amended protocol, the change in 24-hour ammonia levels will be measured through Week 36 after which the study would unblind and patients will be followed for a total of up to 64 weeks to determine the complete responders able to remove safely both ammonia-scavenger medications and protein-restricted diet control.
UX701 AAV gene therapy for Wilson Disease: Phase 1/2/3 study ongoing; expect Cohort 4 enrollment completion in second half of 2025
In Stage 1 of the Phase 1/2/3 Cyprus2+ study, 15 patients across three sequential dose cohorts were enrolled and demonstrated clinical activity as well as improvements in copper metabolism. Multiple responders completely tapered off their standard-of-care treatment with responses seen in all three dose cohorts.
The company expects to enroll a fourth cohort in Stage 1 at a moderately increased dose and with an optimized immunomodulation regimen to enhance the efficiency and efficacy of the gene therapy, with the objective of having the majority of patients come off standard-of-care treatment before selecting a dose for the randomized placebo-controlled stage of the study. 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, Thursday, February 13, 2025, at 2 p.m. PT/5 p.m. ET to discuss the fourth quarter and full year 2024 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 ultrarare 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, and the components and timing of regulatory submissions 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 commercial supply of Crysvita, fluctuations in buying or distribution patterns from distributors and specialty pharmacies, the transition back to Kyowa Kirin of our exclusive rights to promote Crysvita in the United States and Canada and unexpected costs, delays, difficulties or adverse impact to revenue related to such transition, smaller than anticipated market opportunities for the company’s products and product candidates, manufacturing risks, competition from other therapies or products, 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 November 6, 2024, 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 December 31, | Year Ended December 31, | ||||||||||||||||
| 2024 | 2023 | 2024 | 2023 | ||||||||||||||
| Statement of Operations Data: | |||||||||||||||||
| Revenues: | |||||||||||||||||
| Product sales | $ | 71,870 | $ | 51,656 | $ | 285,415 | $ | 180,413 | |||||||||
| Royalty revenue | 93,007 | 75,736 | 274,815 | 182,652 | |||||||||||||
| Collaboration and license | — | — | — | 71,184 | |||||||||||||
| Total revenues | 164,877 | 127,392 | 560,230 | 434,249 | |||||||||||||
| Operating expenses: | |||||||||||||||||
| Cost of sales | 16,894 | 12,051 | 76,728 | 45,209 | |||||||||||||
| Research and development | 187,766 | 160,557 | 697,865 | 648,449 | |||||||||||||
| Selling, general and administrative | 82,495 | 76,833 | 321,610 | 309,799 | |||||||||||||
| Total operating expenses | 287,155 | 249,441 | 1,096,203 | 1,003,457 | |||||||||||||
| Loss from operations | (122,278 | ) | (122,049 | ) | (535,973 | ) | (569,208 | ) | |||||||||
| Change in fair value of equity investments | (1,548 | ) | 1,889 | (1,115 | ) | 397 | |||||||||||
| Non-cash interest expense on liabilities for sales of future royalties | (15,522 | ) | (17,328 | ) | (63,041 | ) | (66,004 | ) | |||||||||
| Other income, net | 5,944 | 10,596 | 32,543 | 26,351 | |||||||||||||
| Loss before income taxes | (133,404 | ) | (126,892 | ) | (567,586 | ) | (608,464 | ) | |||||||||
| Benefit from (provision for) income taxes | 19 | 3,702 | (1,597 | ) | 1,825 | ||||||||||||
| Net loss | $ | (133,385 | ) | $ | (123,190 | ) | $ | (569,183 | ) | $ | (606,639 | ) | |||||
| Net loss per share, basic and diluted | $ | (1.39 | ) | $ | (1.52 | ) | $ | (6.29 | ) | $ | (8.25 | ) | |||||
| Shares used in computing net loss per share, basic and diluted | 95,681,451 | 81,118,873 | 90,538,118 | 73,543,862 | |||||||||||||
| Ultragenyx Pharmaceutical Inc. Selected Activity included in Operating Expense (in thousands) (unaudited) | |||||||||||||||
| Three Months Ended December 31, | Year Ended December 31, | ||||||||||||||
| 2024 | 2023 | 2024 | 2023 | ||||||||||||
| Non-cash stock based compensation | $ | 40,190 | $ | 33,744 | $ | 158,056 | $ | 135,213 | |||||||
| GTX-102 clinical milestone | $ | 30,000 | — | $ | 30,000 | — | |||||||||
| UX143 clinical milestone | — | — | — | $ | 9,000 | ||||||||||
| Ultragenyx Pharmaceutical Inc. | ||||||||
| Selected Balance Sheet Financial Data | ||||||||
| (in thousands) | ||||||||
| (unaudited) | ||||||||
| December 31, | December 31, | |||||||
| 2024 | 2023 | |||||||
| Balance Sheet Data: | ||||||||
| Cash, cash equivalents, and marketable debt securities | $ | 745,029 | $ | 777,110 | ||||
| Working capital | 472,970 | 451,747 | ||||||
| Total assets | 1,503,456 | 1,491,013 | ||||||
| Total stockholders' equity | 255,297 | 275,414 | ||||||
Contacts Ultragenyx Pharmaceutical Inc.
Investors
Joshua Higa
ir@ultragenyx.com
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