Second quarter total revenue of $147 million, Crysvita® revenue of $114 million and Dojolvi® revenue of $19 million
Increased 2024 expected total revenue guidance to $530 million to $550 million
NOVATO, Calif., Aug. 01, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today reported its financial results for the quarter ended June 30, 2024.
“Our strong financial performance in the second quarter was driven by growing revenue across our commercial therapies from increasing global demand, leading us to raise our total revenue guidance for this year,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “In the quarter, we also reported positive data from our Phase 1/2 study in Angelman syndrome, our Phase 2/3 study in osteogenesis imperfecta, and our Phase 3 study in GSDIa. We are in an excellent position to achieve additional key milestones in the second half of the year including initiating our Phase 3 Angelman study and filing for accelerated approval for UX111 in Sanfilippo syndrome type A.”
Second Quarter 2024 Selected Financial Data Tables and Financial Results
| Revenues (dollars in thousands), (unaudited) | |||||||||||||||
| Three Months Ended June 30, | Six Months Ended June 30, | ||||||||||||||
| 2024 | 2023 | 2024 | 2023 | ||||||||||||
| Crysvita | |||||||||||||||
| Product sales | $ | 40,449 | $ | 16,884 | $ | 76,690 | $ | 38,118 | |||||||
| Revenue in Profit-Share Territory | 67,045 | 61,314 | 107,447 | 111,220 | |||||||||||
| Royalty revenue in European Territory | 6,176 | 4,816 | 12,118 | 9,698 | |||||||||||
| Total Crysvita Revenue | 113,670 | 83,014 | 196,255 | 159,036 | |||||||||||
| Dojolvi | 19,355 | 16,491 | 35,717 | 30,794 | |||||||||||
| Mepsevii | 6,145 | 8,439 | 12,756 | 16,919 | |||||||||||
| Evkeeza | 7,856 | 365 | 11,131 | 577 | |||||||||||
| Daiichi Sankyo | — | — | — | 1,479 | |||||||||||
| Total revenues | $ | 147,026 | $ | 108,309 | $ | 255,859 | $ | 208,805 | |||||||
Total RevenuesUltragenyx reported $147 million in total revenue for the second quarter of 2024, which represents 36% growth compared to the same period in 2023. Second quarter 2024 Crysvita revenue was $114 million, which represents 37% growth compared to the same period in 2023. This includes product sales of $40 million from Latin America and Turkey, which represents 140% growth compared to the same period in 2023. Dojolvi revenue in the second quarter 2024 was $19 million, which represents 17% growth compared to the same period in 2023. Evkeeza revenue in the second quarter 2024 was $8 million, as demand continues to build in the company’s territories outside of the United States.
| Selected Financial Data (dollars in thousands, except per share amounts), (unaudited) | |||||||||||||||
| Three Months Ended June 30, | Six Months Ended June 30, | ||||||||||||||
| 2024 | 2023 | 2024 | 2023 | ||||||||||||
| Total revenues | $ | 147,026 | $ | 108,309 | $ | 255,859 | $ | 208,805 | |||||||
| Operating expenses: | |||||||||||||||
| Cost of sales | 21,280 | 9,914 | 38,813 | 22,171 | |||||||||||
| Research and development | 161,503 | 164,949 | 339,990 | 330,647 | |||||||||||
| Selling, general and administrative | 80,604 | 81,403 | 158,764 | 158,049 | |||||||||||
| Total operating expenses | 263,387 | 256,266 | 537,567 | 510,867 | |||||||||||
| Net loss | $ | (131,598 | ) | $ | (159,828 | ) | $ | (302,282 | ) | $ | (323,800 | ) | |||
| Net loss per share, basic and diluted | $ | (1.52 | ) | $ | (2.25 | ) | $ | (3.54 | ) | $ | (4.58 | ) | |||
Operating Expenses Total operating expenses for the second quarter of 2024 were $263 million, including non-cash stock-based compensation of $39 million. In 2024, annual operating expenses are expected to be stable or to decrease as the company continues to manage its costs and focus its investment on advancing multiple Phase 3 programs and executing on commercial product launches.
Net Loss For the second quarter of 2024, Ultragenyx reported net loss of $132 million, or $1.52 per share basic and diluted, compared with a net loss for the second quarter of 2023 of $160 million, or $2.25 per share basic and diluted.
Net Cash Used in Operations and Cash BalanceFor the three months ended June 30, 2024, net cash used in operations was $77 million and for the six months ended June 30, 2024 it was $268 million. Cash, cash equivalents, and marketable debt securities were $874 million as of June 30, 2024, which includes $381 million of net proceeds from issuance of common stock and pre-funded warrants in connection with an underwritten public offering in June 2024.
2024 Full Year Financial Guidance
- Total revenue guidance increased to be in the range of $530 million to $550 million (previously $500 million to $530 million)
- Crysvita revenue expected to be towards the upper end of the range of $375 million to $400 million. This includes all regions where Ultragenyx will recognize revenue: product sales in Latin America and Turkey, royalties in Europe, which have been ongoing, and royalties in North America, which began in April 2023.
- Dojolvi revenue in the range of $75 million to $80 million
- Net Cash Used in Operations less than $400 million
Recent Updates and Clinical Milestones
UX143 (setrusumab) monoclonal antibody for Osteogenesis Imperfecta (OI): 14-month data resulted in a large, sustained 67% reduction in annualized fracture rate and persistent median annualized fracture rate of 0.00 (p=0.0014)Positive 14-month results from the Phase 2 portion of the ongoing Phase 2/3 Orbit study demonstrated that, as of the May 24, 2024 data cut-off date, treatment with setrusumab continued to significantly reduce incidence of fractures in patients with OI. Treatment with setrusumab also resulted in ongoing and meaningful improvements in lumbar spine bone mineral density (BMD) at month 12 without evidence of plateau.
The median annualized rate of radiologically confirmed fractures across all 24 patients in the 2 years prior to treatment was 0.72. Following a mean treatment duration period of 16 months, the median annualized fracture rate was reduced 67% to 0.00 (p=0.0014; n=24). The reduction in annualized fracture rates was associated with continued, clinically meaningful increases in BMD. Treatment with setrusumab at 12-month demonstrated a mean increase in lumbar spine BMD from baseline of 22% (p