2021 total revenue of $351.4 million and 2021 Crysvita® revenue in Ultragenyx territories1 of $192.6 million
2022 Crysvita revenue in Ultragenyx territories guidance of $250 million to $260 million and Dojolvi revenue of $55 million to $65 million
Strong Dojolvi® launch continues with approximately 270 patients on reimbursed commercial therapy in the United States as of the end of 2021
Year-end 2021 cash balance of approximately $1.0 billion
NOVATO, Calif., Feb. 10, 2022 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today reported its financial results for the quarter and full year ended December 31, 2021 and reaffirmed its financial guidance for 2022.
“Over the last quarter we made significant progress across our early and late-stage clinical pipeline, including resuming the Phase 1/2 study for Angelman syndrome and initiating two pivotal gene therapy studies in GSDIa and Wilson disease. Our recently announced collaboration with Regeneron for Evkeeza® gives us a fourth approved product adding to the ongoing successful launches of Crysvita, Mepsevii, and Dojolvi,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “In 2022, we will leverage our strong capital position to continue building on this momentum, enrolling patients in our six clinical programs, launching Evkeeza in Europe, expanding our global commercial efforts to Japan, and substantially complete the construction of our gene therapy manufacturing facility.”
Fourth Quarter and Full Year 2021 Financial Results
Net RevenuesFor the fourth quarter of 2021, Ultragenyx reported $83.4 million in total revenue. Ultragenyx recognized $55.5 million in Crysvita (burosumab) revenue in the Ultragenyx territories, which includes $50.2 million in collaboration revenue in the North American profit share territory and net product sales in other regions of $5.3 million. Total royalty revenue related to European Crysvita sales were $4.7 million. Dojolvi (triheptanoin) product sales in the fourth quarter of 2021 were $11.8 million. Mepsevii (vestronidase alfa) product sales for the fourth quarter of 2021 were $3.1 million. Total revenue for the fourth quarter also includes $8.2 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, 2021 was $351.4 million, including $192.6 million in Crysvita revenue in the Ultragenyx territories. Crysvita collaboration revenue in the North American profit share territory was $171.2 million and net Crysvita product sales in other regions were $21.4 million. Total royalty revenue related to European Crysvita royalties was $18.2 million. Dojolvi product revenue for the year ended December 31, 2021 was $39.6 million. Mepsevii product revenue was $16.0 million. For the year ended December 31, 2021, revenue related to our strategic manufacturing partnership with Daiichi Sankyo was $85.0 million.
Operating Expenses Total operating expenses for the fourth quarter of 2021 were $186.0 million, including non-cash stock-based compensation of $26.5 million. Total operating expenses for the year ended December 31, 2021 were $733.1 million, which includes a $50.0 million expense for the upfront payment of the license and collaboration agreement with Mereo Biopharma in January 2021 and non-cash stock-based compensation of $105.0 million.
Net Loss For the fourth quarter of 2021, Ultragenyx reported net loss of $122.5 million, or $1.79 per share basic and diluted, compared with a net loss for the fourth quarter of 2020 of $24.0 million, or $0.37 per share, basic and diluted. For the year ended December 31, 2021, net loss was $454.0 million, or $6.70 per share, basic and diluted, compared with a net loss for the same period in 2020 of $186.6 million, or $3.07 per share, basic and diluted. Net cash used in operations for the year ended December 31, 2021 was $338.7 million.
Cash, Cash Equivalents and Marketable Debt SecuritiesCash, cash equivalents, and marketable debt securities were approximately $1.0 billion as of December 31, 2021.
2022 Financial Guidance
The company expects 2022 revenue for Crysvita in Ultragenyx territories to be between $250 million and $260 million and Dojolvi revenue to be between $55 million and $65 million.
Fourth Quarter and Full Year 2021 Revenue and Selected Financial Data Tables
| Revenues (dollars in thousands) | |||||||||||||||
| Three Months Ended December 31, | Year Ended December 31, | ||||||||||||||
| 2021 | 2020 | 2021 | 2020 | ||||||||||||
| Crysvita Collaboration and Product Revenues: | |||||||||||||||
| North America Collaboration | $ | 50,211 | $ | 37,518 | $ | 171,198 | $ | 128,597 | |||||||
| ROW Product Sales | 5,272 | 2,914 | 21,422 | 10,350 | |||||||||||
| Crysvita in Ultragenyx Territories | 55,483 | 40,432 | 192,620 | 138,947 | |||||||||||
| EU Royalty Revenue | 4,741 | 3,567 | 18,195 | 14,493 | |||||||||||
| Total Crysvita Revenue | 60,224 | 43,999 | 210,815 | 153,440 | |||||||||||
| Dojolvi | 11,825 | 6,390 | 39,560 | 13,028 | |||||||||||
| Mepsevii | 3,111 | 3,656 | 16,035 | 15,342 | |||||||||||
| Daiichi Sankyo | 8,229 | 37,497 | 84,996 | 89,220 | |||||||||||
| Total Revenue | $ | 83,389 | $ | 91,542 | $ | 351,406 | $ | 271,030 | |||||||
| Selected Financial Data (dollars in thousands, except per share amounts) | |||||||||||||||
| Three Months Ended December 31, | Year Ended December 31, | ||||||||||||||
| 2021 | 2020 | 2021 | 2020 | ||||||||||||
| Total revenue | $ | 83,389 | $ | 91,542 | $ | 351,406 | $ | 271,030 | |||||||
| Operating expense: | |||||||||||||||
| Cost of sales | 3,509 | 5,481 | 16,008 | 6,129 | |||||||||||
| Research and development | 123,013 | 131,100 | 497,153 | 412,084 | |||||||||||
| Selling, general and administrative | 59,431 | 51,042 | 219,982 | 182,933 | |||||||||||
| Total operating expense | 185,953 | 187,623 | 733,143 | 601,146 | |||||||||||
| Net loss | $ | (122,458 | ) | $ | (24,011 | ) | $ | (454,025 | ) | $ | (186,566 | ) | |||
| Net loss per share, basic and diluted | $ | (1.79 | ) | $ | (0.37 | ) | $ | (6.70 | ) | $ | (3.07 | ) | |||
Recent Updates and 2022 Milestones
Evkeeza (evinacumab) for Homozygous Familial Hypercholesterolemia (HoFH): Submission of reimbursement dossiers expected in European countries in 2022Ultragenyx and Regeneron announced a license and collaboration agreement for Ultragenyx to commercialize and distribute Evkeeza in countries outside of the U.S. This includes the European Economic Area where Evkeeza was approved in June 2021 as a first-in-class monoclonal antibody targeting ANGPTL3 for use together with diet and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies to treat adults and adolescents aged 12 years and older with HoFH.
Ultragenyx plans to submit reimbursement dossiers with national health authorities in Europe in 2022.
UX143 (setrusumab) for Osteogenesis Imperfecta (OI): Dosing in the Pivotal Phase 2/3 Orbit study is expected to begin in the first half of 2022; Phase 2 study in children under age 5 planned for second half of 2022Ultragenyx expects to begin dosing in the seamless Phase 2/3 Orbit study of UX143 in pediatric and adult patients ages 5 to