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First quarter total revenue of $109 million, Crysvita® revenue of $83 million and Dojolvi® revenue of $16 million
Reaffirmed 2024 expected total revenue guidance between $500 million to $530 million, Crysvita revenue of $375 million to $400 million, and Dojolvi revenue of $75 million to $80 million
Presented positive interim Phase 1/2 data from GTX-102 Angelman syndrome study demonstrating that Expansion Cohorts showed rapid, clinically meaningful improvement across multiple domains consistent with or exceeding Dose-escalation Cohort data at Day 170
NOVATO, Calif., May 02, 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 March 31, 2024 and reaffirmed its financial guidance for 2024.
“In the first quarter we continued to see rapid revenue growth in the international markets with steady progress globally across our four commercial products. We also just completed enrollment of our Phase 3 program in osteogenesis imperfecta supported by strong enthusiasm from treating physicians and the patient community,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “Last month we shared new data from the GTX-102 program for Angelman syndrome and have received an overwhelming response of excitement and support from the clinical experts and advocates in the Angelman community. We are on track to hold regulatory discussions in mid-2024 with the goal of initiating a Phase 3 trial before the end of the year.”
First Quarter 2024 Selected Financial Data Tables and Financial Results
| Revenues (dollars in thousands), (unaudited) | |||||
| Three Months Ended March 31, | |||||
| 2024 | 2023 | ||||
| Crysvita | |||||
| Product sales | $ | 36,241 | $ | 21,234 | |
| Revenue in Profit-Share Territory | 40,402 | 49,906 | |||
| Royalty revenue in European Territory | 5,942 | 4,882 | |||
| Total Crysvita Revenue | 82,585 | 76,022 | |||
| Dojolvi | 16,362 | 14,303 | |||
| Mepsevii | 6,611 | 8,480 | |||
| Evkeeza | 3,275 | 212 | |||
| Daiichi Sankyo | — | 1,479 | |||
| Total revenues | $ | 108,833 | $ | 100,496 | |
Total RevenuesUltragenyx reported $109 million in total revenue for the first quarter of 2024, which represents 8% growth compared to the same period in 2023. First quarter 2024 Crysvita revenue was $83 million, which represents 9% growth compared to the same period in 2023. This includes product sales of $36 million from Latin America and Turkey, which represents 71% growth compared to the same period in 2023. In April 2023, commercialization responsibilities in the Profit-share Territory transitioned to Kyowa Kirin Co and commensurately, revenue in that region transitioned from a profit-share to a royalty ranging from the mid-20’s to 30% calculated based on annual revenue tiers. Dojolvi revenue in the first quarter 2024 was $16 million, which represents 14% growth compared to the same period in 2023.
| Selected Financial Data (dollars in thousands, except per share amounts), (unaudited) | |||||||
| Three Months Ended March 31, | |||||||
| 2024 | 2023 | ||||||
| Total revenues | $ | 108,833 | $ | 100,496 | |||
| Operating expenses: | |||||||
| Cost of sales | 17,533 | 12,257 | |||||
| Research and development | 178,487 | 165,698 | |||||
| Selling, general and administrative | 78,160 | 76,646 | |||||
| Total operating expenses | 274,180 | 254,601 | |||||
| Net loss | $ | (170,684 | ) | $ | (163,972 | ) | |
| Net loss per share, basic and diluted | $ | (2.03 | ) | $ | (2.33 | ) | |
Operating Expenses Total operating expenses for the first quarter of 2024 were $274 million, including non-cash stock-based compensation of $37 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 first quarter of 2024, Ultragenyx reported net loss of $171 million, or $2.03 per share basic and diluted, compared with a net loss for the first quarter of 2023 of $164 million, or $2.33 per share, basic and diluted.
Net Cash Used in Operations and Cash BalanceNet cash used in operations for the quarter ended March 31, 2024 was $191 million. Cash, cash equivalents, and marketable debt securities were $569 million as of March 31, 2024.
2024 Financial Guidance For the full year 2024, the company reaffirms:
Recent Updates and Clinical Milestones
UX143 (setrusumab) monoclonal antibody for Osteogenesis Imperfecta (OI): Enrollment complete for Phase 3 Orbit and Cosmic studies; expect additional Phase 2 data update in second half of 2024 The Phase 3 portion of the Orbit study has completed enrollment with 158 patients. Orbit is a randomized placebo-controlled study evaluating the effect of setrusumab compared to placebo on the rate of annualized clinical fractures in patients aged 5 to
