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 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.
Ultragenyx Pharmaceutical (NASDAQ: RARE) and Kyowa Kirin announced FDA approval for Crysvita (burosumab) as the first therapy for tumor-induced osteomalacia (TIO) that cannot be surgically removed. This decision represents the second FDA-approved indication for Crysvita, previously authorized for X-linked hypophosphatemia (XLH). TIO affects approximately 500-1,000 patients in the U.S., and half are inoperable. Crysvita acts by blocking FGF23, alleviating symptoms like severe hypophosphatemia. The approval follows successful Phase 2 trials, showcasing significant improvements in phosphate levels and bone health.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has announced virtual presentations at investor conferences. CFO Shalini Sharp will present at the Jefferies Healthcare Conference on June 3, 2020, at 2 PM ET, followed by CEO Emil D. Kakkis at the Goldman Sachs 41st Annual Global Healthcare Conference on June 9, 2020, at 3:50 PM ET. Live and archived webcasts will be available on the company's website for 90 days post-event. Ultragenyx is focused on developing novel treatments for rare genetic diseases, aiming for efficient drug development to meet high unmet medical needs.
Ultragenyx Pharmaceutical (Nasdaq: RARE) has exercised an option to purchase 600,000 shares of Arcturus Therapeutics (Nasdaq: ARCT) at $16.00 each, increasing its ownership to 3,000,000 shares, or 14.6% of Arcturus. This investment supports the advancement of Arcturus's mRNA platform, including a COVID-19 vaccine candidate, and Ultragenyx's preclinical mRNA candidate for Glycogen Storage Disease Type III. The strategic collaboration between both companies focuses on developing nucleic acid therapies for rare diseases, with an IND application for UX053 anticipated in 2021.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced positive long-term data from its Phase 1/2 study of DTX301, a gene therapy for ornithine transcarbamylase (OTC) deficiency. All three patients in Cohort 3 responded, maintaining clinical stability after stopping ammonia scavengers. Six of nine patients showed a response. Key results include significant improvements in ureagenesis and ammonia levels. No serious adverse events were reported. The company aims to start a Phase 3 trial in early 2021, with ammonia levels as a primary endpoint. A recap call is scheduled for May 15, 2020.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that CFO Shalini Sharp will present at the BofA Securities Global Health Care Conference on May 14, 2020, at 10:20 AM ET. The event will be available via live and archived webcast on Ultragenyx's website for 90 days post-event. The company focuses on developing therapies for serious rare and ultra-rare genetic diseases, aiming for efficient drug development and urgency in delivering effective treatments to patients with high unmet medical needs.
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