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 and Solid Biosciences announced a strategic collaboration to develop gene therapies for Duchenne muscular dystrophy. The agreement includes a $40 million upfront investment from Ultragenyx, with potential milestone payments totaling up to $255 million and tiered royalties. Solid retains exclusive rights to its microdystrophin technology for other uses. The collaboration aims to harness Solid's microdystrophin construct and Ultragenyx’s HeLa PCL platform to enhance treatment efficacy. The partnership is positioned to leverage both companies' strengths in advancing therapies for rare diseases.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has announced a conference call scheduled for October 27, 2020, at 5 PM ET to discuss its third quarter 2020 financial results and provide a corporate update. The call will be accessible via the company's website, and participants can join by phone. Ultragenyx focuses on developing and commercializing novel therapies for serious rare and ultra-rare genetic diseases, aiming for efficient drug development to address high unmet medical needs.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announces CEO Emil D. Kakkis will present virtually at the Jefferies Virtual Gene Therapy/Editing Summit on October 2, 2020, at 1:00 PM ET. The presentation will be accessible live and as an archive on their website. Ultragenyx focuses on developing treatments for serious rare and ultra-rare genetic diseases, with a commitment to efficient drug development. They aim to provide safe and effective therapies to patients with high unmet medical needs. Visit their website for more details.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that CEO Emil D. Kakkis will present at three upcoming investor conferences. The events include the Citi’s 15th Annual BioPharma Virtual Conference on September 9, 2020, the Morgan Stanley Virtual 18th Annual Global Healthcare Conference on September 15, 2020, and the BofA Securities Virtual Global Healthcare Conference on September 16, 2020. Live and archived webcasts of these presentations will be available on the company’s website for 90 days.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has appointed Mardi C. Dier as Executive Vice President and Chief Financial Officer, effective November 2, 2020. Dier will oversee corporate finance, strategy, investor relations, corporate communications, and information technology. She joins from Portola Pharmaceuticals, where she served as CFO, enhancing her experience in financial leadership. Ultragenyx aims to grow its commercial opportunities and pipeline for rare diseases, while thanking outgoing CFO Shalini Sharp for her contributions.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced the appointment of Ernie Meyer as the new Executive Vice President and Chief Human Resources Officer, effective September 1, 2020. In this role, Meyer will oversee Global Human Resources and Facilities operations, reporting directly to CEO Emil D. Kakkis. Meyer brings extensive experience from previous roles at Portola Pharmaceuticals and Celgene, where he contributed to significant organizational growth. His leadership is expected to enhance Ultragenyx's commitment to addressing rare genetic diseases as it enters a new growth phase.
Ultragenyx Pharmaceutical reported $61.7 million in total revenue for Q2 2020, with $32.4 million from Crysvita. The company achieved two FDA approvals: Dojolvi for LC-FAOD and Crysvita for TIO. Net income was $25.3 million ($0.42/share), a significant recovery from a $99.2 million loss in Q2 2019. Operating expenses totaled $124.8 million, influenced by increased costs in commercial and development activities. Ultragenyx maintains its full-year guidance for Crysvita revenue between $125 million and $140 million, despite COVID-19 impacts.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced a conference call set for July 30, 2020, at 5pm ET to discuss its second-quarter 2020 financial results and provide corporate updates. The call will be accessible via phone and through a live webcast on the company’s website. Ultragenyx is dedicated to developing treatments for rare genetic diseases, featuring a diverse portfolio of approved therapies and product candidates aimed at high unmet medical needs. The management team emphasizes efficient drug development to deliver therapies urgently.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced the commercial availability of Dojolvi™ (triheptanoin) for treating long-chain fatty acid oxidation disorders (LC-FAOD) following FDA approval on June 30, 2020. This marks the first FDA-approved therapy for LC-FAOD, a serious condition affecting approximately 2,000 to 3,500 patients in the U.S. Dojolvi aims to provide essential calories and fatty acids, with support available through Ultragenyx's UltraCare program for patient access. Caution is advised due to potential gastrointestinal side effects and contraindications for certain patients.
Ultragenyx Pharmaceutical has received FDA approval for Dojolvi™ (triheptanoin), a treatment for long-chain fatty acid oxidation disorders (LC-FAOD), marking its fourth FDA approval in three years. Dojolvi offers a new source of calories and fatty acids for patients, many of whom face serious health challenges despite existing treatments. The company estimates LC-FAOD affects 2,000 to 3,500 individuals in the U.S. and plans to make Dojolvi available within 30 days through its UltraCare® program for patient support and insurance navigation.
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