Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has announced preliminary unaudited 2024 revenue results, showcasing significant growth and providing financial guidance for 2025. The company, focused on developing and commercializing novel products for serious rare and ultrarare genetic diseases, also highlighted key program updates, including advancements in its late-stage pipeline. Preliminary total revenue for 2024 is estimated between $555 million and $560 million, exceeding the high end of the company's guidance.
Financial Performance and 2025 Outlook
Ultragenyx's 2024 revenue represents approximately 29% growth compared to 2023. Crysvita revenue is estimated between $405 million and $410 million, a 24% increase year-over-year, while Dojolvi revenue is projected between $87 million and $89 million, reflecting a 25% rise. The company's cash, cash equivalents, and available-for-sale investments totaled approximately $745 million as of December 31, 2024.
For 2025, Ultragenyx anticipates total revenue between $640 million and $670 million. The company plans to provide specific guidance on Crysvita and Dojolvi revenue as part of its fourth-quarter and fiscal year 2024 financial disclosures in February 2025. Ultragenyx also expects a decline in 2025 net cash used in operations compared to 2024, driven by revenue growth of approximately 14-20% and continued expense management.
Pipeline Advancements and Clinical Milestones
Ultragenyx is making strides in its clinical programs targeting rare genetic diseases. Key updates include:
- Setrusumab (UX143) for Osteogenesis Imperfecta (OI): The Phase 3 Orbit study is progressing towards its second interim analysis (IA2), expected in mid-2025. Patients are currently being dosed in the ongoing Phase 3 Orbit and Cosmic clinical trials.
- GTX-102 for Angelman Syndrome: Enrollment in the global Phase 3 Aspire study began in December 2024, with completion expected in the second half of 2025. The study aims to enroll approximately 120 children aged four to 17 with Angelman syndrome, characterized by a genetically confirmed full maternal UBE3A gene deletion. The primary endpoint is improvement in cognition, assessed by Bayley-4 cognitive raw score.
- UX111 for Sanfilippo Syndrome Type A (MPS IIIA): A Biologics License Application (BLA) has been submitted to the U.S. Food and Drug Administration (FDA). A Prescription Drug User Fee Act (PDUFA) decision and potential launch are anticipated in the second half of 2025. Data from the ongoing pivotal Transpher A study demonstrated that UX111 treatment resulted in rapid and sustained decreases in heparan sulfate levels in cerebral spinal fluid (CSF), correlating with improved long-term cognitive development.
- DTX401 for Glycogen Storage Disease Type Ia (GSDIa): A BLA filing is expected in mid-2025, supported by positive topline results from the Phase 3 GlucoGene study. The study demonstrated a statistically significant and clinically meaningful reduction in daily cornstarch intake compared to placebo at Week 48.
- UX701 for Wilson Disease: The Phase 1/2/3 Cyprus2+ study is ongoing, with Cohort 4 enrollment expected to complete in the second half of 2025. Initial results have shown clinical activity and improvements in copper metabolism.
- DTX301 for Ornithine Transcarbamylase (OTC) Deficiency: Patient dosing is underway in the Phase 3 study, with enrollment expected to complete in early 2025. The study will evaluate the change in 24-hour ammonia levels and the removal of ammonia-scavenger medications and protein-restricted diet.
Management Commentary
"In 2024 we grew our business with four products in five indications globally, exceeding the updated revenue guidance we provided in August, and continuing our path toward profitability," said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. "In 2025, we will continue to expand our commercial base of business while we also prepare for the potential launch of our first gene therapy, in Sanfilippo syndrome, and to file a BLA for our second gene therapy, in Glycogen Storage Disease Type Ia. We are also executing on one of the most valuable late-stage pipelines in rare disease as we anticipate important pivotal Phase 3 results in osteogenesis imperfecta and completion of enrollment in our Phase 3 trial in Angelman syndrome. This progress puts us in the unique position to potentially launch three to four new therapies over the next couple of years, accumulating a total of eight to nine approved products over a 10-year period."
