Ultragenyx Sets Sights on a Strong Year of Value Creation in 2025
The biotechnology company, Ultragenyx, has reported a successful fourth quarter and full year 2024 financial results conference call, highlighting its progress in advancing late-stage programs for serious genetic conditions. The company's CEO and President, Emil Kakkis, emphasized that 2024 was a pivotal year for the company, with significant advancements in its pipeline and commercial products.
In 2024, Ultragenyx advanced six late-stage programs, most without any approved therapies, while also expanding access and growing revenue from its four commercial products worldwide. The company increased its guidance for total revenue and exceeded the upper end of that range for 2024, confirming a strong year of financial performance.
The company's international growth was particularly impressive in 2024, with successful launches of Evkeeza in Europe, Canada, and Japan, as well as broadening access to other commercial therapies in Latin America, Canada, and Turkey. Ultragenyx is looking forward to another year of strong global revenue growth in 2025, supported by multiple products in launch mode globally.
One of the key highlights from the quarter was the progress made on the UX111 program for Sanfilippo syndrome, which has the potential to be the company's next approved product and its first commercial gene therapy program. Last week, Ultragenyx presented important new clinical data at the World Symposium in San Diego that were also included in its BLA submission last December.
The new data show a sustained reduction in CSF HS exposure, which is directly responsible for disease pathology and progression, and are statistically associated with significant continued growth in cognitive abilities when compared to natural history data. The company also saw older children with more advanced disease at the time of treatment retain clinically meaningful functional abilities, including communication, ambulation, and self-feeding following treatment with UX111.
These results give confidence that UX111 will be a successful product once approved, with the potential to make a meaningful difference for patients with Sanfilippo syndrome Type A and their families. Ultragenyx's progress in Sanfilippo and other companies' progress in the MPS field are made possible by FDA's willingness to accept CSF HS as a primary biomarker endpoint.
The company is set for a strong year of value creation greater than any year in its history, with expected Phase III data on UX143 in osteogenesis imperfecta and full enrollment of its Phase III for GTX-102 for Angelman syndrome. With six commercial products on the market if both BLAs are approved, Ultragenyx is firmly on a path toward full-year GAAP profitability in 2027.
