Preliminary 2023 Revenue and 2024 Guidance
- 2023 Total Revenue: $430 million to $435 million
- Crysvita Revenue: $325 million to $330 million
- Dojolvi Revenue: $70 million to $71 million
- 2024 Total Revenue Guidance: $500 million to $530 million
- Crysvita Revenue Guidance: $375 million to $400 million
- Dojolvi Revenue Guidance: $75 million to $80 million
Cash Position and Usage
- Year-end 2023 Cash Balance: Approximately $776 million
- 2024 Net Cash Used in Operations Guidance: Less than $400 million
2024 Clinical Milestones
- UX143 (setrusumab) for Osteogenesis Imperfecta (OI): Phase 3 portion of Orbit study expected to be fully enrolled in the first quarter of 2024.
- GTX-102 for Angelman Syndrome: Phase 1/2 fully enrolled; expansion data expected in the first half of 2024.
- UX701 for Wilson Disease: Interim Stage 1 data expected in the first half of 2024.
- UX111 for Sanfilippo syndrome (MPS IIIA): Updated data from pivotal Transfer A study to be presented at WORLDSymposium™ in February 2024.
- DTX401 for Glycogen Storage Disease Type Ia (GSDIa): Phase 3 data readout expected in the first half of 2024.
- DTX301 for Ornithine Transcarbamylase (OTC) Deficiency: Phase 3 study dosing patients; expect enrollment to be completed in the first half of 2024.
Ultragenyx is focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases. The company anticipates 2024 to be a year of significant momentum, with clinical catalysts across multiple value-driving programs and meaningful revenue growth from its commercial products.
