From GlobeNewswire:

Ultragenyx Pharmaceutical Inc. reported 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. For 2024, the company expects a 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. They also reported a year-end 2023 cash balance of approximately $776 million and 2024 guidance for net cash used in operations to be less than $400 million.

The company’s CEO, Emil D. Kakkis, M.D., Ph.D., expressed optimism for Ultragenyx in 2024 due to significant momentum and meaningful revenue growth from commercial products. Ultragenyx also anticipates several important milestones in the first half of the year, including substantial data releases from various clinical studies. They will present at the 42ᵗʰ annual J.P. Morgan Healthcare Conference on Monday, January 8, 2024. Preliminary 2023 revenue results are subject to adjustment, and full financial results will be issued in February 2024. Ultragenyx expects to complete enrollment of the Phase 3 studies in osteogenesis imperfecta and release interim data on a substantial portion of patients for their Angelman syndrome study. They also anticipate the release of Stage 1 data from their pivotal Phase 1/2/3 study in Wilson disease, and topline Phase 3 data from their GSDIa gene therapy program.

Moreover, Ultragenyx has reached a cash balance of approximately $776 million in 2023, partly due to the completion of the construction of their gene therapy manufacturing facility. The company forecasts a net cash use of less than $400 million in 2024, partly attributed to a projected ~20% increase in revenue and continued focus on key pipeline value drivers. Additionally, 2024 will see the full enrollment of the Phase 3 portion of the Orbit study for their OI treatment and additional longer-term Phase 2 data from that study. Patients are already being dosed in the late-stage clinical trials, Orbit and Cosmic, for their treatment for osteogenesis imperfecta.



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