2022 Total Collaboration and Product Revenue of $355.6 million, Crysvita® revenue of $279.4 million and Dojolvi® revenue of $55.6 million
2023 expected Total Revenue guidance between $425 million to $450 million, Crysvita revenue of $325 million to $340 million, and Dojolvi revenue of $65 million to $75 million
Year-end 2022 cash balance of $896.7 million and 2023 guidance for Net Cash used in Operations expected to be less than $400 million
NOVATO, Calif., Feb. 16, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, today reported its financial results for the quarter and full year ended December 31, 2022 and reaffirmed its financial guidance for 2023.
“In 2022, we continued to reach more patients around the world as we successfully commercialized multiple products to treat rare diseases,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “2023 will be a year of meaningful development progress for us as we focus on data generation from two of our key programs, UX143 in osteogenesis imperfecta and GTX-102 in Angelman syndrome, and the continued advancement of our late-stage gene therapy programs.”
Fourth Quarter and Full Year 2022 Selected Financial Data Tables and Financial Results
| Revenues (dollars in thousands) (unaudited) | |||||||||||||||
| Three Months Ended December 31, | Year Ended December 31, | ||||||||||||||
| 2022 | 2021 | 2022 | 2021 | ||||||||||||
| Crysvita Collaboration and Product Revenues: | |||||||||||||||
| North America Collaboration | $ | 66,903 | $ | 50,211 | $ | 215,024 | $ | 171,198 | |||||||
| ROW Product Sales | 7,698 | 5,272 | 42,678 | 21,422 | |||||||||||
| Crysvita in Ultragenyx Territories1 | 74,601 | 55,483 | 257,702 | 192,620 | |||||||||||
| EU Royalty Revenue | 6,058 | 4,741 | 21,692 | 18,195 | |||||||||||
| Total Crysvita Revenue | 80,659 | 60,224 | 279,394 | 210,815 | |||||||||||
| Dojolvi | 16,412 | 11,825 | 55,612 | 39,560 | |||||||||||
| Mepsevii | 4,798 | 3,111 | 20,637 | 16,035 | |||||||||||
| Total Collaboration and Product Revenues | 101,869 | 75,160 | 355,643 | 266,410 | |||||||||||
| Daiichi Sankyo | 1,479 | 8,229 | 7,686 | 84,996 | |||||||||||
| Total Revenue | $ | 103,348 | $ | 83,389 | $ | 363,329 | $ | 351,406 | |||||||
Net RevenuesFor the fourth quarter of 2022, Ultragenyx reported $103.3 million in total revenue. Ultragenyx recognized $74.6 million in Crysvita (burosumab) revenue in the Ultragenyx territories1, which includes $66.9 million in collaboration revenue in the North American profit-share territory and net product sales in other regions of $7.7 million. Total royalty revenue related to European Crysvita sales was $6.1 million. Dojolvi (triheptanoin) revenue in the fourth quarter of 2022 was $16.4 million. Mepsevii (vestronidase alfa) revenue for the fourth quarter of 2022 was $4.8 million. Total revenue for the fourth quarter also includes $1.5 million of revenue related to the collaboration and license agreement with Daiichi Sankyo that was executed in March 2020.
Revenue for the year ended December 31, 2022 was $363.3 million, including $257.7 million in Crysvita revenue in the Ultragenyx territories1. Crysvita collaboration revenue in the North American profit-share territory was $215.0 million and net Crysvita product sales in other regions were $42.7 million. Total royalty revenue related to European Crysvita royalties was $21.7 million. Dojolvi product revenue for the year ended December 31, 2022 was $55.6 million. Mepsevii product revenue was $20.6 million. For the year ended December 31, 2022, revenue related to our strategic manufacturing partnership with Daiichi Sankyo was $7.7 million.
| Selected Financial Data (dollars in thousands, except per share amounts) (unaudited) | |||||||||||||||
| Three Months Ended December 31, | Year Ended December 31, | ||||||||||||||
| 2022 | 2021 | 2022 | 2021 | ||||||||||||
| Total revenue | $ | 103,348 | $ | 83,389 | $ | 363,329 | $ | 351,406 | |||||||
| Operating expense: | |||||||||||||||
| Cost of sales | 5,319 | 3,509 | 28,320 | 16,008 | |||||||||||
| Research and development | 170,808 | 123,013 | 705,789 | 497,153 | |||||||||||
| Selling, general and administrative | 72,849 | 59,431 | 278,139 | 219,982 | |||||||||||
| Total operating expense | 248,976 | 185,953 | 1,012,248 | 733,143 | |||||||||||
| Net loss | $ | (151,833 | ) | $ | (122,458 | ) | $ | (707,421 | ) | $ | (454,025 | ) | |||
| Net loss per share, basic and diluted | $ | (2.16 | ) | $ | (1.79 | ) | $ | (10.12 | ) | $ | (6.70 | ) | |||
Operating Expenses Total operating expenses for the fourth quarter of 2022 were $249.0 million, including non-cash stock-based compensation of $29.4 million. Total operating expenses for the year ended December 31, 2022 were $1,012.2 million, which includes $130.4 million of non-cash stock-based compensation and $75.0 million of in-process research and development expense associated with the acquisition of GeneTx in July 2022. In 2023, annual operating expenses are expected to decrease as the company manages headcount and increases operational leverage while executing on high value programs.
Net Loss For the fourth quarter of 2022, Ultragenyx reported net loss of $151.8 million, or $2.16 per share basic and diluted, compared with a net loss for the fourth quarter of 2021 of $122.5 million, or $1.79 per share, basic and diluted. For the year ended December 31, 2022, net loss was $707.4 million, or $10.12 per share, basic and diluted, compared with a net loss for the same period in 2021 of $454.0 million, or $6.70 per share, basic and diluted.
Cash, Cash Equivalents and Marketable Debt SecuritiesCash, cash equivalents, and marketable debt securities were approximately $896.7 million as of December 31, 2022.
2023 Financial Guidance For the full year 2023, the company reaffirms:
- Total revenue in the range of $425 million to $450 million
- Crysvita revenue in the range of $325 million to $340 million. This includes all regions where Ultragenyx will recognize revenue, including the royalties in Europe, which have been ongoing, and the royalties in North America, which will begin in April 2023.
- Dojolvi revenue in the range of $65 million to $75 million
- Net Cash Used in Operations is expected to be less than $400 million
Recent Updates and 2023 Clinical Milestones
UX143 (setrusumab) monoclonal antibody for Osteogenesis Imperfecta (OI): Phase 2/3 study dosing patients; Phase 2 enrollment complete, data expected in mid-2023Ultragenyx is currently dosing patients in the Phase 2/3 Orbit study of UX143 in pediatric and adult patients with OI aged five to