First quarter 2023 total revenue of $100.5 million, Crysvita® revenue of $76.0 million and Dojolvi® revenue of $14.3 million
Total revenue grew 26% and Crysvita revenue grew 28% versus the first quarter 2022
Reaffirmed 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
NOVATO, Calif., May 04, 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 ended March 31, 2023 and reaffirmed its financial guidance for 2023.
“In the first quarter we continued the strong growth in product revenue and advanced our clinical programs including completion of the Phase 2 portion of our pivotal study of UX143 in osteogenesis imperfecta. We are analyzing the data and look forward to sharing the results soon. Once completed, we will initiate enrollment of the Phase 3 portion in mid-2023,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “We are also making rapid progress in our Phase 1/2 study of GTX-102 in Angelman syndrome, dosing in the expansion cohorts and activating sites in new countries.”
First Quarter 2023 Selected Financial Data Tables and Financial Results
| Revenues (dollars in thousands), (unaudited) | |||||
| Three Months Ended March 31, | |||||
| 2023 | 2022 | ||||
| Crysvita | |||||
| Revenue in profit-share territory | $ | 49,906 | $ | 45,164 | |
| Product revenue | 21,234 | 9,394 | |||
| Non-cash royalty revenue in European territory | 4,882 | 4,838 | |||
| Total Crysvita revenue | 76,022 | 59,396 | |||
| Dojolvi | 14,303 | 12,429 | |||
| Mepsevii | 8,480 | 4,861 | |||
| Evkeeza | 212 | — | |||
| Daiichi Sankyo | 1,479 | 3,249 | |||
| Total revenues | $ | 100,496 | $ | 79,935 | |
Net RevenuesUltragenyx reported $100.5 million in total revenue for the first quarter of 2023, which represents 26% growth compared to the first quarter 2022. The growth is primarily driven by increased demand for Crysvita in Latin America and steady growth from the other regions and products. The first quarter 2023 also included $1.5 million of revenue from the technology transfer which was completed during the quarter as part of the collaboration and license agreement with Daiichi Sankyo.
| Selected Financial Data (dollars in thousands, except per share amounts), (unaudited) | |||||||
| Three Months Ended March 31, | |||||||
| 2023 | 2022 | ||||||
| Total revenues | $ | 100,496 | $ | 79,935 | |||
| Operating expense: | |||||||
| Cost of sales | 12,257 | 6,100 | |||||
| Research and development | 165,698 | 143,155 | |||||
| Selling, general and administrative | 76,646 | 67,312 | |||||
| Total operating expense | 254,601 | 216,567 | |||||
| Net loss | $ | (163,972 | ) | $ | (152,320 | ) | |
| Net loss per share, basic and diluted | $ | (2.33 | ) | $ | (2.19 | ) | |
Operating Expenses Total operating expenses for the first quarter of 2023 were $254.6 million, including non-cash stock-based compensation of $31.9 million. 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 first quarter of 2023, Ultragenyx reported net loss of $164.0 million, or $2.33 per share basic and diluted, compared with a net loss for the first quarter of 2022 of $152.3 million, or $2.19 per share, basic and diluted.
Cash, Cash Equivalents and Marketable Debt SecuritiesCash, cash equivalents, and marketable debt securities were approximately $714.6 million as of March 31, 2023.
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 began 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 Clinical Milestones
UX143 (setrusumab) monoclonal antibody for Osteogenesis Imperfecta (OI): Phase 2 enrollment complete; Phase 2 data and Phase 3 initiation expected in mid-2023Ultragenyx is continuing to dose patients in the Phase 2/3 Orbit study of UX143 in pediatric and adult patients with OI aged five to