Third quarter 2023 total revenue of $98.1 million, Crysvita® revenue of $74.9 million and Dojolvi® revenue of $16.6 million
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
Presented positive updates on three lead clinical programs at Analyst Day in October
NOVATO, Calif., Nov. 02, 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 September 30, 2023 and provided its financial guidance for the year.
"We're in a strong financial position due to growing demand for our commercial products, completion of our recent offering, and our disciplined expense and portfolio management," said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. "We've also made significant progress on our key clinical programs and highlighted new, interim data from UX143 for osteogenesis imperfecta, GTX-102 for Angelman syndrome, and UX701 for Wilson Disease at our Analyst Day in October demonstrating promising therapeutic potential in these larger indications with further updates to come in 2024."
Third Quarter 2023 Selected Financial Data Tables and Financial Results
| Revenues (dollars in thousands), (unaudited) | |||||||||||||||
| Three Months Ended September 30, | Nine Months Ended September 30, | ||||||||||||||
| 2023 | 2022 | 2023 | 2022 | ||||||||||||
| Crysvita | |||||||||||||||
| Product sales | $ | 19,200 | $ | 13,184 | $ | 57,318 | $ | 34,980 | |||||||
| Royalty revenue | 35,160 | — | 64,221 | — | |||||||||||
| Non-cash royalty revenue | 20,543 | 5,373 | 42,695 | 15,634 | |||||||||||
| Revenue in profit-share territory | — | 51,348 | 69,705 | 148,121 | |||||||||||
| Total Crysvita revenue | 74,903 | 69,905 | 233,939 | 198,735 | |||||||||||
| Dojolvi | 16,553 | 13,274 | 47,347 | 39,200 | |||||||||||
| Mepsevii | 5,633 | 6,045 | 22,552 | 15,839 | |||||||||||
| Evkeeza | 963 | — | 1,540 | — | |||||||||||
| Daiichi Sankyo | — | 1,479 | 1,479 | 6,207 | |||||||||||
| Total revenues | $ | 98,052 | $ | 90,703 | $ | 306,857 | $ | 259,981 | |||||||
Total RevenuesUltragenyx reported $98.1 million in total revenue for the third quarter 2023, which represents 8% growth compared to the third quarter 2022. Third quarter 2023 Crysvita product sales, primarily in Latin America, were $19.2 million, which represents 46% growth compared to the same period in 2022 and 14% growth over the second quarter 2023. Third quarter 2023 also includes Crysvita royalty and non-cash royalty revenue in North America of $50.2 million, which was impacted by a decrease in channel inventory related to Kyowa Kirin Co., Ltd.’s (KKC) change from Ultragenyx labeled product to KKC’s labeled product as part of the transition of North America commercialization responsibilities for Crysvita from Ultragenyx to KKC. This one-time change occurred in the third quarter 2023, and the Company expects Crysvita channel inventories to increase to more normal levels at the end of the year. Third quarter 2023 non-cash royalty revenue in Europe was $5.5 million.
| Selected Financial Data (dollars in thousands, except per share amounts), (unaudited) | |||||||||||||||
| Three Months Ended September 30, | Nine Months Ended September 30, | ||||||||||||||
| 2023 | 2022 | 2023 | 2022 | ||||||||||||
| Total revenues | $ | 98,052 | $ | 90,703 | $ | 306,857 | $ | 259,981 | |||||||
| Operating expense: | |||||||||||||||
| Cost of sales | 10,987 | 8,631 | 33,158 | 23,001 | |||||||||||
| Research and development | 157,245 | 237,297 | 487,892 | 534,981 | |||||||||||
| Selling, general and administrative | 74,917 | 69,841 | 232,966 | 205,290 | |||||||||||
| Total operating expense | 243,149 | 315,769 | 754,016 | 763,272 | |||||||||||
| Net loss | $ | (159,649 | ) | $ | (245,106 | ) | $ | (483,449 | ) | $ | (555,588 | ) | |||
| Net loss per share, basic and diluted | $ | (2.23 | ) | $ | (3.50 | ) | $ | (6.81 | ) | $ | (7.96 | ) | |||
Operating Expenses Total operating expenses for the third quarter of 2023 were $243.1 million, including non-cash stock-based compensation of $34.9 million. In 2023, annual operating expenses are expected to decrease compared to 2022, as the company manages headcount and increases operational leverage while executing on its high-value programs.
Net Loss For the third quarter of 2023, Ultragenyx reported net loss of $159.6 million, or $2.23 per share basic and diluted, compared with a net loss for the third quarter of 2022 of $245.1 million, or $3.50 per share, basic and diluted.
Cash, Cash Equivalents and Marketable Debt SecuritiesCash, cash equivalents, and marketable debt securities were $524.2 million as of September 30, 2023. This excludes net proceeds of $326.5 million from an underwritten public offering of common stock and pre-funded warrants that closed in October 2023.
2023 Financial Guidance For the full year 2023, the company expects:
- 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 to be around $425 million
Recent Updates and Clinical Milestones
UX143 (setrusumab) monoclonal antibody for Osteogenesis Imperfecta (OI): Phase 2 demonstrated 67% reduction in annualized fracture rate and continuous improvements in bone mineral densityAt the American Society of Bone and Mineral Research 2023 Annual Meeting (ASBMR), interim data from the Phase 2 portion of the Phase 2/3 Orbit study were presented that demonstrated treatment with setrusumab significantly reduced incidence of fractures in patients with OI with at least 6 months of follow-up and continued to demonstrate ongoing and meaningful improvements in lumbar spine bone mineral density (BMD). As of the cut-off date on August 4, 2023 and following at least six months of treatment with setrusumab, the annualized fracture rate across all 24 patients in the Phase 2 portion of the study was reduced by 67%. The median annualized fracture rate of 0.72 in the two years prior to treatment was reduced to 0.00 (n=24, p=0.042) during the mean treatment duration period of nine months. As of the data cut-off, there were no treatment-related serious adverse events (SAEs) observed in the study. Additional longer-term Phase 2 data are expected in 2024.
Patients are being dosed in the late-stage clinical trials, Orbit and Cosmic, which evaluate setrusumab in pediatric and young adult patients with OI. The Phase 3 portion of the Orbit study is targeting to enroll up to 195 patients at more than 50 sites across 12 countries. 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 aged 2 to