Preliminary 2023 Total Revenue of $430 million to $435 million, Crysvita® Revenue of $325 million to $330 million and Dojolvi® revenue of $70 million to $71 million
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
Year-end 2023 cash balance of approximately $776 million and 2024 guidance for net cash used in operations expected to be less than $400 million
NOVATO, Calif., Jan. 07, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, today reported preliminary unaudited 2023 revenue results, cash and investments at year end 2023, and provided 2024 guidance on select key financial metrics including net cash used in operations.
“2024 is poised to be a year of significant momentum for Ultragenyx, with clinical catalysts across multiple value-driving programs, meaningful revenue growth from our commercial products, and continued focus on financial discipline,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “We anticipate several important milestones in the first half of the year, including completing enrollment of our Phase 3 studies in osteogenesis imperfecta, interim data on a substantive portion of patients enrolled in our Phase 1/2 study in Angelman syndrome, Stage 1 data from our pivotal Phase 1/2/3 study in Wilson disease, and topline Phase 3 data from our GSDIa gene therapy program.”
Ultragenyx will present at the 42ᵗʰ annual J.P. Morgan Healthcare Conference on Monday, January 8, 2024 at 3:00 p.m. PT. The live and archived webcast of the presentation will be accessible from the company’s website at https://ir.ultragenyx.com/events-presentations.
Financial Update
2023 Revenue (unaudited) and 2024 Revenue Guidance
| 2023 | 2024 | ||
| ($ in millions) | Preliminary Revenue | Revenue Guidance | |
| Crysvita | $325 - $330 | $375 - $400 | |
| Dojolvi | $70 - $71 | $75 - $80 | |
| Total Revenue | $430 - $435 | $500 - $530 |
2023 Ending Cash Position (unaudited) and 2024 Cash Used in Operations Guidance Cash, cash equivalents, and available-for-sale investments were approximately $776 million as of December 31, 2023. Cash uses in 2023 included the completion of construction of our gene therapy manufacturing facility. With forecasted ~20% increases in revenue and continued focus on key pipeline value drivers, 2024 net cash use is projected to be less than $400 million.
The 2023 revenues and cash position included in this release are preliminary and are therefore subject to adjustment. The preliminary revenue results are based on management’s initial analysis of operations for the year ended December 31, 2023. The Company expects to issue full financial results for the fourth quarter and fiscal year 2023 in February 2024.
Recent Updates and 2024 Clinical Milestones
UX143 (setrusumab) monoclonal antibody for Osteogenesis Imperfecta (OI): Phase 3 portion of Orbit study expected to be fully enrolled in the first quarter of 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 randomized, placebo-controlled Phase 3 portion of the Orbit study is expected to be fully enrolled around the end of the first quarter of 2024. Additional longer-term Phase 2 data from the Orbit study are expected in 2024. 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