Ultragenyx Reports Preliminary 2024 Revenue, Financial Guidance for 2025, Pipeline Updates, and 2025 Milestones

Financial Performance and Guidance - Total revenue for 2024 is estimated to be $555 million to$560 million, exceeding the updated guidance range and representing approximately 29% growth versus 2023 [7] - Crysvita revenue for 2024 is estimated to be $405 million to$410 million, representing approximately 24% growth versus 2023 [7] - Dojolvi revenue for 2024 is estimated to be $87 million to$89 million, representing approximately 25% growth versus 2023 [7] - 2025 total revenue is expected to be between $640 million and$670 million, with a growth of approximately 14-20% compared to 2024 [1][6] - Cash, cash equivalents, and available-for-sale investments were approximately $745 million as of December 31, 2024 [1] Clinical Trials and Pipeline Updates - Phase 3 Orbit and Cosmic clinical trials for setrusumab in pediatric and young adult patients with osteogenesis imperfecta (OI) are ongoing, with the second interim analysis expected in mid-2025 and a potential final analysis in Q4 2025 [2][8] - UX111 AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA) has a Biologics License Application (BLA) submitted, with a PDUFA decision and launch expected in the second half of 2025 [3] - UX701 AAV gene therapy for Wilson Disease is in Phase 1/2/3 study, with Cohort 4 enrollment expected to complete in the second half of 2025 [5][15] - GTX-102 Phase 3 Aspire study for Angelman syndrome is enrolling, with completion expected in the second half of 2025 [13] - DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa) is expected to file a BLA in mid-2025 [14] - DTX301 AAV gene therapy for Ornithine Transcarbamylase (OTC) Deficiency is in Phase 3 study, with enrollment completion expected in early 2025 [16] Strategic Outlook and Milestones - The company anticipates important pivotal Phase 3 results in osteogenesis imperfecta and completion of enrollment in the Phase 3 trial for Angelman syndrome in 2025 [11] - The company is preparing for the potential launch of its first gene therapy for Sanfilippo syndrome and filing a BLA for its second gene therapy for Glycogen Storage Disease Type Ia in 2025 [11] - The company expects 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 [11] Corporate Overview - Ultragenyx is a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases [17] - The company has a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need [17] - The company is led by a management team experienced in the development and commercialization of rare disease therapeutics [18]