2023 Total Revenue of $434 million, Crysvita® revenue of $328 million and Dojolvi® revenue of $71 million
2024 Financial Guidance: Total Revenue between $500 million and $530 million, Crysvita revenue of $375 million to $400 million, and Dojolvi revenue of $75 million to $80 million
Year-end 2023 cash balance of $777 million and 2024 guidance for Net Cash Used in Operations expected to be less than $400 million
NOVATO, Calif., Feb. 15, 2024 (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, 2023 and reaffirmed its financial guidance for 2024.
“Last year, we made significant progress across our pipeline and look forward to multiple catalysts in 2024 with substantive data in Angelman syndrome that could enable a Phase 3 study, pivotal Phase 3 data from our GSDIa gene therapy program, dose-finding data from our seamless study in Wilson disease, and longer-term Phase 2 data in Osteogenesis Imperfecta,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “In 2023, we saw increased demand for both Crysvita and Dojolvi, and achieved multiple regulatory and reimbursement milestones for our commercial products to support continued revenue growth of approximately 20% in 2024.”
Fourth Quarter and Full Year 2023 Selected Financial Data Tables and Financial Results
| Revenues (dollars in thousands), (unaudited) | |||||||||||
| Three Months Ended December 31, | Year Ended December 31, | ||||||||||
| 2023 | 2022 | 2023 | 2022 | ||||||||
| Crysvita | |||||||||||
| Product sales | $ | 18,379 | $ | 7,698 | $ | 75,697 | $ | 42,678 | |||
| Revenue in Profit-Share Territory | 70,124 | 66,903 | 231,574 | 215,024 | |||||||
| Royalty revenue in European Territory | 5,612 | 6,058 | 20,783 | 21,692 | |||||||
| Total Crysvita Revenue | 94,115 | 80,659 | 328,054 | 279,394 | |||||||
| Dojolvi | 23,286 | 16,412 | 70,633 | 55,612 | |||||||
| Mepsevii | 7,889 | 4,798 | 30,441 | 20,637 | |||||||
| Evkeeza | 2,102 | — | 3,642 | — | |||||||
| Daiichi Sankyo | — | 1,479 | 1,479 | 7,686 | |||||||
| Total revenues | $ | 127,392 | $ | 103,348 | $ | 434,249 | $ | 363,329 | |||
Total RevenuesUltragenyx reported $127 million in total revenue for the fourth quarter 2023, which represents 23% growth compared to the same period in 2022. Fourth quarter 2023 Crysvita revenue was $94 million, which represents 17% growth compared to the same period in 2022. This includes product sales of $18 million from Latin America and Turkey, which represents 139% growth compared to the same period in 2022. Dojolvi revenue in the fourth quarter 2023 was $23 million, which represents 42% growth compared to the same period in 2022.
Total revenue for the year ended December 31, 2023 was $434 million, which represents 20% growth compared to the prior year. Full year 2023 Crysvita revenue was $328 million, which represents 17% growth compared to the same period in 2022. This includes product sales of $76 million from Latin America and Turkey, which represents 77% growth compared to the same period in 2022. Dojolvi revenue in 2023 was $71 million, which represents 27% growth compared to the same period in 2022.
| Selected Financial Data (dollars in thousands, except per share amounts), (unaudited) | |||||||||||||||
| Three Months Ended December 31, | Year Ended December 31, | ||||||||||||||
| 2023 | 2022 | 2023 | 2022 | ||||||||||||
| Total revenues | $ | 127,392 | $ | 103,348 | $ | 434,249 | $ | 363,329 | |||||||
| Operating expenses: | |||||||||||||||
| Cost of sales | 12,051 | 5,319 | 45,209 | 28,320 | |||||||||||
| Research and development | 160,557 | 170,808 | 648,449 | 705,789 | |||||||||||
| Selling, general and administrative | 76,833 | 72,849 | 309,799 | 278,139 | |||||||||||
| Total operating expenses | 249,441 | 248,976 | 1,003,457 | 1,012,248 | |||||||||||
| Net loss | $ | (123,190 | ) | $ | (151,833 | ) | $ | (606,639 | ) | $ | (707,421 | ) | |||
| Net loss per share, basic and diluted | $ | (1.52 | ) | $ | (2.16 | ) | $ | (8.25 | ) | $ | (10.12 | ) | |||
Operating Expenses Total operating expenses for the fourth quarter of 2023 were $249 million, including non-cash stock-based compensation of $34 million. Total operating expenses for the year ended December 31, 2023 were $1,003 million, including $135 million of non-cash stock-based compensation. In 2024, annual operating expenses are expected to be stable or to decrease as the company continues to manage its costs, focus its investment on advancing multiple Phase 3 programs, and execute on commercial product launches.
Net Loss For the fourth quarter of 2023, Ultragenyx reported net loss of $123 million, or $1.52 per share basic and diluted, compared with a net loss for the fourth quarter of 2022 of $152 million, or $2.16 per share, basic and diluted. For the year ended December 31, 2023, Ultragenyx reported net loss of $607 million, or $8.25 per share basic and diluted, compared with a net loss the prior year of $707 million, or $10.12 per share, basic and diluted.
Net Cash Used in Operations and Cash BalanceNet cash used in operations for the year ended December 31, 2023 was $475 million. Cash, cash equivalents, and marketable debt securities were $777 million as of December 31, 2023.
2024 Financial Guidance For the full year 2024, the company reaffirms:
- Total revenue in the range of $500 million to $530 million
- Crysvita revenue in the range of $375 million to $400 million. This includes all regions where Ultragenyx will recognize revenue: product sales in Latin America and Turkey, royalties in Europe, which have been ongoing, and royalties in North America, which began in April 2023.
- Dojolvi revenue in the range of $75 million to $80 million
- Net Cash Used in Operations to be less than $400 million
Recent Updates and Clinical Milestones
UX143 (setrusumab) monoclonal antibody for Osteogenesis Imperfecta (OI): Phase 3 portion of Orbit study expected to be fully enrolled around the end of the first quarter of 2024Patients are being dosed in the late-stage clinical trials, Orbit and Cosmic, which are evaluating setrusumab in pediatric and young adult patients with OI. The randomized, placebo-controlled Phase 3 portion of the Orbit study is expected to enroll approximately 150 patients and be fully enrolled around the end of the first quarter of 2024. Additional longer-term Phase 2 safety and efficacy data from the Orbit study are expected in the second half of 2024.
The Phase 3 Cosmic study is an active-controlled study evaluating the effect of setrusumab compared to intravenous bisphosphonate (IV-BP) therapy on annualized total fracture rate in patients age 2 to