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Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), founded in 2010, is a clinical-stage biotechnology company dedicated to developing and commercializing innovative therapies for the treatment of serious rare and ultra-rare genetic diseases. The company’s core focus is on debilitating metabolic genetic diseases, for which there are high unmet medical needs, and the biology for treatment is well-understood but lacks approved therapies. Ultragenyx's management team brings extensive experience in rare disease therapeutics.
The company’s diverse product portfolio includes approved medicines like Crysvita, Dojolvi, and Mepsevii. Crysvita is designed for the treatment of X-linked hypophosphatemia (XLH), a rare genetic bone disease, and has shown significant growth with revenue reaching $328 million in 2023. Dojolvi targets long-chain fatty acid oxidation disorders and saw a 27% revenue increase in 2023. Mepsevii treats Mucopolysaccharidosis VII, another severe genetic disorder.
Recent Achievements and Financial Performance
In 2023, Ultragenyx reported total revenues of $434 million, a 20% increase from the previous year. The company has a robust pipeline with several candidates in late-stage clinical trials. For instance, the Phase 3 Orbit study for UX143 (setrusumab) in Osteogenesis Imperfecta (OI) completed enrollment and showed promising interim results with a 67% reduction in fracture rates.
Ongoing Projects
- GTX-102: An antisense oligonucleotide for Angelman syndrome showed significant improvements in clinical trials, with plans for a Phase 3 study in 2024.
- UX701: A gene therapy for Wilson disease, with data expected in mid-2024.
- DTX401: A gene therapy for Glycogen Storage Disease Type Ia (GSDIa), achieved its primary endpoint in a Phase 3 study.
- Setrusumab (UX143): Demonstrated significant fracture rate reduction and bone mineral density improvement in OI patients.
Partnerships and Collaborations
Ultragenyx collaborates with various regulatory bodies and advocacy groups to support patients and ensure high-quality clinical trials. The company’s partnership with Mereo BioPharma on setrusumab exemplifies its collaborative approach to drug development.
Corporate Responsibility
Ultragenyx's 2023 Corporate Responsibility Report highlights its commitment to innovation, patient support, diversity, and environmental sustainability. The company has been recognized with several awards, including the Top Places to Work by the Boston Globe and Healthcare’s Best Companies to Work by U.S. News & World Report.
For more information, visit www.ultragenyx.com.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced its participation in the Goldman Sachs 45th Annual Global Healthcare Conference. Dr. Emil Kakkis, the CEO, will engage in a fireside chat on June 11, 2024, at 10:00 a.m. ET. The event will be live-streamed and available for later viewing on Ultragenyx's investor relations website.
Ultragenyx announced positive top-line results from its Phase 3 GlucoGene study evaluating DTX401, a gene therapy for glycogen storage disease type Ia (GSDIa).
The therapy showed a statistically significant reduction in daily cornstarch intake at Week 48 compared to placebo (41.3% vs. 10.3%).
DTX401 also improved glucose control and reduced the number of daily cornstarch doses. Safety profile was consistent with previous studies, showing non-serious, manageable effects.
The results will be discussed with regulatory authorities for a marketing application in 2025.
Ultragenyx Pharmaceutical, a biopharmaceutical company focused on therapies for rare diseases, announced the inducement grant of 17,180 restricted stock units (RSUs) to ten newly hired non-executive officers. The awards, approved by the board's compensation committee under the Ultragenyx Employment Inducement Plan, were granted on May 16, 2024. These RSUs vest over four years, with 25% vesting annually, conditional on continuous employment. This grant aligns with Nasdaq Listing Rule 5635(c)(4), serving as a key employment incentive.
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.
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