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.
GeneTx Biotherapeutics and Ultragenyx Pharmaceutical (NASDAQ: RARE) announced the FDA's removal of the clinical hold on GTX-102, an experimental treatment for Angelman syndrome. This allows GeneTx to commence dosing naïve pediatric patients in a Phase 1/2 study. The trial aims to evaluate the drug's safety and effectiveness, enrolling eight patients aged 4 to <8 years in the U.S. The therapy targets the UBE3A gene, which is crucial for brain function and is absent in individuals with Angelman syndrome. There are currently no approved treatments for this condition.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that CEO Emil D. Kakkis will present at the Morgan Stanley 19th Annual Healthcare Conference on September 14, 2021, at 4:15 PM ET. The presentation will be available via a live and archived webcast on the company’s website, with the replay accessible for 90 days. Ultragenyx focuses on developing therapies for serious rare and ultra-rare genetic diseases, emphasizing novel products with high unmet medical needs. The company aims for efficient drug development to deliver urgent solutions to patients.
On September 1, 2021, Ultragenyx Pharmaceutical (NASDAQ: RARE) announced that CEO Emil D. Kakkis will speak at Citi's 16th Annual BioPharma Healthcare Conference on September 9, 2021, at 4:10 PM ET. The panel, titled Finding a Needle in a Haystack - Drug Development Strategies for Rare Disease, focuses on innovative approaches to developing therapies for rare genetic conditions. A live and archived webcast of the presentation will be available on the company's website for 90 days.
Ultragenyx is committed to addressing unmet medical needs in rare diseases through efficient drug development.
Dojolvi has been approved by Brazil's National Health Surveillance Agency (ANVISA) for treatment of long-chain fatty acid oxidation disorders (LC-FAOD), marking an essential therapeutic advance for patients. This decision is crucial for managing LC-FAOD, which affects 8,000 to 14,000 individuals in developed countries, leading to severe health complications. Ultragenyx Pharmaceutical Inc. aims to secure reimbursement to enhance patient access. The drug is already FDA-approved and available through early access programs outside the U.S.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced the grant of stock options totaling 14,445 shares and 4,815 restricted stock units to two newly hired employees. This grant, which took place on August 16, 2021, aligns with the company's Employment Inducement Plan and Nasdaq Listing Rule 5635(c)(4). The stock options have a ten-year term and an exercise price of $88.00 per share. Vested shares will be distributed over four years, contingent on ongoing employment. Ultragenyx continues to focus on developing therapies for rare diseases.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that CEO Emil D. Kakkis will participate in a panel discussion titled Miss Con-GENE-iality - Updates in Gene Tx at the Wedbush PacGrow Healthcare Conference on August 11, 2021, at 12:00 PM ET. The event will be accessible via live and archived webcasts on the company’s website for 90 days. Ultragenyx focuses on developing therapies for serious rare and ultra-rare genetic diseases, with a commitment to efficient drug development and addressing unmet medical needs.
Ultragenyx Pharmaceutical reported Q2 2021 total revenue of $87.0 million, reflecting a 38% increase from Q2 2020, driven by strong Crysvita sales of $44.7 million. The guidance for Crysvita remains at $180-$190 million for 2021. Dojolvi continues to gain traction with 220 patients on therapy. Operating expenses rose 36% to $169.8 million. The company expects to initiate four pivotal clinical trials in the next six months, enhancing its late-stage pipeline. As of June 30, cash and equivalents stood at $973.8 million.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) will host a conference call on August 2, 2021, at 5 PM ET to discuss its financial results for Q2 2021, ending June 30. The company is known for developing therapies for serious rare genetic diseases and has a diverse portfolio aimed at unmet medical needs. Interested participants can access the live webcast through their website or dial in by phone. This update underscores the company's commitment to efficient drug development and patient care in the rare disease sector.
Ultragenyx Pharmaceutical announced that the FDA and the European Commission have granted Orphan Drug Designation for UX053, aimed at treating Glycogen Storage Disease Type III (GSDIII), affecting over 10,000 patients in developed nations. Enrollment for a Phase 1/2 clinical trial is anticipated in late 2021, evaluating the drug's safety, tolerability, and efficacy. The Orphan Drug Designation provides significant market exclusivity benefits, including seven years in the U.S. and ten years in the EU.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced the appointment of Corsee Sanders, Ph.D., to its Board of Directors, effective June 29, 2021. Dr. Sanders brings extensive experience in global clinical development and new technologies from her previous roles at Celgene and Juno Therapeutics. CEO Emil D. Kakkis highlighted her expertise as critical for advancing ongoing clinical trials and expanding Ultragenyx's rare disease pipeline. Dr. Sanders holds degrees in statistics and has served on multiple biotechnology boards, further enhancing the company’s leadership in developing therapies for rare genetic diseases.
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