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Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) will have its chief medical officer, Eric Crombez, participate in a fireside at Bank of America’s 2024 Healthcare Conference on May 14, 2024. The company focuses on developing therapies for rare genetic diseases.
Ultragenyx Pharmaceutical Inc. reported first quarter total revenue of $109 million with Crysvita® revenue at $83 million and Dojolvi® revenue at $16 million. The company reaffirmed its 2024 total revenue guidance of $500 million to $530 million, with Crysvita revenue between $375 million to $400 million and Dojolvi revenue at $75 million to $80 million. Positive Phase 1/2 data from the GTX-102 Angelman syndrome study was presented, showing significant improvement. The company continues to see revenue growth and has completed enrollment for its Phase 3 program in osteogenesis imperfecta.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) released its 2023 Corporate Responsibility Report, emphasizing its commitment to improving equity and access to innovation in rare diseases. The report showcases efforts in drug development, patient support, employee diversity, community impact, environmental sustainability, and governance. Ultragenyx received multiple awards for its corporate responsibility initiatives, including high rankings in the biotech industry and recognition as a top workplace. The company's dedication to rare disease medicine and community welfare is evident in its comprehensive report.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) completed patient enrollment in Phase 3 studies for setrusumab (UX143) in treating osteogenesis imperfecta (OI). The Orbit study included 158 patients aged 5-25, while the Cosmic study enrolled 66 patients aged 2-7. Positive Phase 2 results showcased a 67% reduction in fracture rates and significant improvements in bone mineral density. Setrusumab demonstrated good tolerability with no serious adverse events reported. The company aims to provide a new treatment option for OI patients, enhancing their quality of life by strengthening bones.
