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) and Abeona Therapeutics (NASDAQ: ABEO) have entered into an exclusive license agreement for the gene therapy ABO-102 (now UX111) for treating Sanfilippo syndrome type A (MPS IIIA). Ultragenyx will oversee the program, while Abeona is eligible for tiered royalties up to 10% on net sales post-approval. The ongoing Transpher A trial shows promising interim results, indicating preserved neurocognitive development and no serious adverse events. The therapy has received multiple designations from the FDA and EMA, highlighting its potential in a field with high unmet medical needs.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced that they will present significant data from their investigational gene therapy programs at the ASGCT 25th Annual Meeting, scheduled for May 16-19, 2022. Key presentations include safety and efficacy results from DTX301 and DTX401 clinical trials. Notable sessions include oral presentations on Ornithine Transcarbamylase Deficiency and Glycogen Storage Disease Type Ia. Additionally, the company will showcase advancements in their manufacturing platform, Pinnacle PCL™. CEO Emil D. Kakkis will speak on accelerated approval for gene therapies.
Ultragenyx Pharmaceutical Inc. reported first-quarter 2022 revenue of $79.9 million, with Crysvita revenue at $54.6 million in its territories. The company reaffirmed its 2022 guidance for Crysvita revenue between $250 million and $260 million and Dojolvi revenue of $55 million to $65 million. The company's cash balance stood at approximately $814 million as of March 31, 2022. Despite revenue growth, net loss for the quarter reached $152.3 million, or $2.19 per share, reflecting increased operating expenses.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced the grant of non-qualified stock options for 20,480 shares and 11,620 restricted stock units to two new non-executive officers. Approved by the compensation committee, these awards aim to attract talent under the Ultragenyx Employment Inducement Plan. The options have a ten-year term with an exercise price of $70.69 per share, equivalent to the closing price on April 29, 2022. The restricted stock units vest over four years, while the options vest gradually over the same period, contingent on ongoing employment.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) will present at the BofA Securities 2022 Healthcare Conference on May 10, 2022, at 5:40 PM ET. Key executives, including Chief Medical Officer Camille Bedrosian and Chief Financial Officer Mardi Dier, will lead the presentation. Interested parties can access the live and archived webcast on the company's website for 90 days following the event. Ultragenyx is dedicated to developing innovative therapies for rare and ultra-rare genetic diseases, with a focus on addressing high unmet medical needs.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced its upcoming conference call scheduled for May 5, 2022, at 5:00 PM ET to discuss financial results for Q1 2022. The call will provide updates on the company's performance and strategy in treating rare genetic diseases. Investors can participate via phone or access a live and recorded webcast on the company’s website. Ultragenyx is focused on developing novel therapies for serious rare and ultra-rare genetic disorders, emphasizing time-efficient drug development to meet patient needs.
Ultragenyx Pharmaceutical (NASDAQ: RARE) has appointed Amrit Ray, M.D., M.B.A., to its Board of Directors, specifically on the Research and Development Committee. Dr. Ray brings extensive experience as a physician researcher and executive, previously serving as Chief Patient Officer at Biohaven Pharmaceuticals and holding senior roles at Pfizer and Johnson & Johnson. His expertise is expected to enhance Ultragenyx's commitment to developing innovative treatments for rare diseases. The company focuses on delivering novel therapeutic products to address significant unmet medical needs.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced the initiation of the global Orbit study, a pivotal Phase 2/3 trial for UX143 to treat Osteogenesis Imperfecta (OI) in patients aged 5 to <26. This study follows the promising results of the Phase 2b ASTEROID study, which showed significant improvements in bone density. The Orbit study aims to assess fracture risk reduction in younger populations, with plans for additional studies in other age groups. The collaboration with Mereo BioPharma continues to support the development of UX143 for OI.
Ultragenyx Pharmaceutical (NASDAQ: RARE) will have its CEO, Emil D. Kakkis, participate in the Orphan CNS Panel at the Cowen 42nd Annual Health Care Conference on March 8, 2022, at 2:10 PM ET. The event will be accessible via a live webcast on the company’s website, with a replay available for 90 days. Ultragenyx focuses on developing therapies for rare and ultra-rare genetic diseases, boasting a diverse portfolio aimed at addressing high unmet medical needs. The management team is dedicated to efficient drug development to deliver safe and effective therapies rapidly.
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