Welcome to our dedicated page for Ultragenyx Pharmaceutical news (Ticker: RARE), a resource for investors and traders seeking the latest updates and insights on Ultragenyx Pharmaceutical stock.
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) has announced the grant of stock options and restricted stock units to 11 newly hired non-executive officers. The grant includes 11,035 stock options and 17,710 restricted stock units, approved by the compensation committee and awarded under the Ultragenyx Employment Inducement Plan.
The stock options have a ten-year term with an exercise price of $38.20 per share, reflecting the closing price on April 14, 2023. The restricted stock units vest over four years, while the stock options vest at the first anniversary and continue monthly thereafter, contingent on continuous employment.
Ultragenyx focuses on developing novel therapies for rare diseases, aiming to address high unmet medical needs.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that CEO Emil D. Kakkis will present at Guggenheim’s Genomic Medicines and Rare Disease Days on April 3, 2023, at 10:45 a.m. ET. The event will be accessible via a live and archived webcast on the company’s website. Ultragenyx focuses on developing therapies for serious rare and ultrarare genetic diseases, boasting a diverse portfolio of approved therapies. The company aims to provide safe, effective treatments efficiently, addressing high unmet medical needs in the rare disease sector. The webcast replay will be available for one year following the event.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced the grant of stock options and restricted stock units to ten newly hired non-executive officers. A total of 7,130 stock options and 5,885 restricted stock units were awarded under the Ultragenyx Employment Inducement Plan, approved by the compensation committee of the board of directors. The stock options have a ten-year term with an exercise price of $40.22, corresponding to the closing stock price on the grant date of March 16, 2023. Both stock options and restricted units will vest over four years, promoting retention among new employees dedicated to addressing rare diseases.
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