Second quarter 2023 total revenue of $108.3 million, Crysvita® revenue of $83.0 million and Dojolvi® revenue of $16.5 million
Total revenue grew 21% and total Crysvita revenue grew 20% versus the second 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., Aug. 03, 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 June 30, 2023 and provided its financial guidance for the year.
“In the second quarter, our global commercial efforts have resulted in continued meaningful growth of Crysvita, Dojolvi and Mepsevii revenue, including in our key territories outside of the U.S.,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “At the same time, we have continued to advance key clinical programs for osteogenesis imperfecta (OI), Angelman syndrome and Wilson disease, which are expected to generate a number of data catalysts over the next few quarters.”
Second Quarter 2023 Selected Financial Data Tables and Financial Results
| Revenues (dollars in thousands), (unaudited) | |||||||||||||||
| Three Months Ended June 30, | Six Months Ended June 30, | ||||||||||||||
| 2023 | 2022 | 2023 | 2022 | ||||||||||||
| Crysvita | |||||||||||||||
| Collaboration revenue in profit-share territory | $ | 19,799 | $ | 51,609 | $ | 69,705 | $ | 96,773 | |||||||
| Royalty revenue | 29,061 | — | 29,061 | — | |||||||||||
| Non-cash royalty revenue | 17,270 | 5,423 | 22,152 | 10,261 | |||||||||||
| Product sales | 16,884 | 12,402 | 38,118 | 21,796 | |||||||||||
| Total Crysvita revenue | 83,014 | 69,434 | 159,036 | 128,830 | |||||||||||
| Dojolvi | 16,491 | 13,497 | 30,794 | 25,926 | |||||||||||
| Mepsevii | 8,439 | 4,933 | 16,919 | 9,794 | |||||||||||
| Evkeeza | 365 | — | 577 | — | |||||||||||
| Daiichi Sankyo | — | 1,479 | 1,479 | 4,728 | |||||||||||
| Total revenues | $ | 108,309 | $ | 89,343 | $ | 208,805 | $ | 169,278 | |||||||
Total RevenuesUltragenyx reported $108.3 million in total revenue for the second quarter of 2023, which represents 21% growth compared to the second quarter 2022. This includes second quarter 2023 Crysvita revenue in North America of $61.3 million, which represents 19% growth versus the same time period in 2022. The growth in the region continued on its current trajectory while commercialization responsibilities for Crysvita in North America were transitioned from Ultragenyx to its collaboration partner Kyowa Kirin in April 2023.
| Selected Financial Data (dollars in thousands, except per share amounts), (unaudited) | |||||||||||||||
| Three Months Ended June 30, | Six Months Ended June 30, | ||||||||||||||
| 2023 | 2022 | 2023 | 2022 | ||||||||||||
| Total revenues | $ | 108,309 | $ | 89,343 | $ | 208,805 | $ | 169,278 | |||||||
| Operating expenses: | |||||||||||||||
| Cost of sales | 9,914 | 8,270 | 22,171 | 14,370 | |||||||||||
| Research and development | 164,949 | 154,529 | 330,647 | 297,684 | |||||||||||
| Selling, general and administrative | 81,403 | 68,137 | 158,049 | 135,449 | |||||||||||
| Total operating expense | 256,266 | 230,936 | 510,867 | 447,503 | |||||||||||
| Net loss | $ | (159,828 | ) | $ | (158,162 | ) | $ | (323,800 | ) | $ | (310,482 | ) | |||
| Net loss per share, basic and diluted | $ | (2.25 | ) | $ | (2.26 | ) | $ | (4.58 | ) | $ | (4.45 | ) | |||
Operating Expenses Total operating expenses for the second quarter of 2023 were $256.3 million, including non-cash stock-based compensation of $34.7 million and a $9.0 million milestone expense upon initiation of the UX143 Phase 3 Orbit study. 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 second quarter of 2023, Ultragenyx reported net loss of $159.8 million, or $2.25 per share basic and diluted, compared with a net loss for the second quarter of 2022 of $158.2 million, or $2.26 per share, basic and diluted.
Cash, Cash Equivalents and Marketable Debt SecuritiesCash, cash equivalents, and marketable debt securities were approximately $618.4 million as of June 30, 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 $400 million
Recent Updates and Clinical Milestones
UX143 (setrusumab) monoclonal antibody for Osteogenesis Imperfecta (OI): Two Phase 3 studies enrolling, additional Phase 2 data expected in mid-October
Positive data from the dose-selection Phase 2 portion of the Phase 2/3 Orbit study were announced in June 2023 and showed that setrusumab rapidly induced bone production in patients with OI. Setrusumab treatment induced statistically significant increases in levels of serum P1NP, a sensitive marker of bone formation, and a substantial and significant improvement in bone mineral density (BMD) by three months. Serum P1NP levels through at least 1 month of treatment were available from all 24 patients enrolled in Orbit and demonstrated that treatment with setrusumab significantly increased serum P1NP, peaking at one to two weeks and again, as expected, after the 2-month dosing timepoint. The Phase 2 Orbit data demonstrated meaningful response in serum P1NP and BMD across both cohorts, with the majority of the effect observed at 20 mg/kg when compared to the 40 mg/kg cohort.
The large increase in BMD observed in the Orbit patient population over the first 3 months was consistent with the rapid increase in serum P1NP levels and was similar to results that took 1 year to achieve in the ASTEROID study in adult OI patients. Treatment with setrusumab for 3 months resulted in an increase in lumbar spine BMD from baseline of 9.4% at 20 mg/kg (n=10), which represents a substantial mean change in Z-score of +0.65 from -2.12 (n=11) at baseline. Treatment with 40 mg/kg (n=7) resulted in a 9.8% BMD increase. Patients on placebo at the 3-month timepoint (n=2) showed no significant change in BMD or change in lumbar spine Z-score.
As of the data cut-off, there had been no treatment-related serious adverse events observed in the study. Reported adverse events were generally consistent with those observed in the ASTEROID study and included infusion associated events, headache and sinusitis. There were no reported hypersensitivity reactions related to setrusumab. There were no safety-related differences observed between dosing groups or age groups.
In July we announced, the first patients were dosed in both of the late-stage clinical trials, Orbit and Cosmic, which evaluate setrusumab in pediatric and young adult patients with OI. Orbit is expected to enroll approximately 195 patients at more than 40 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