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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) announced it will present data at the American Society of Gene & Cell Therapy (ASGCT) 24th Annual Meeting, held virtually from May 11-14, 2021. The presentations will cover their investigational gene therapy programs, including advancements in their HeLa producer cell line for AAV production, and long-term results from Phase 1/2 studies on Ornithine Transcarbamylase (OTC) Deficiency and Glycogen Storage Disease Type Ia (GSDIa). Key presentations are scheduled for May 11, 13, and 14.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced the completion of an End-of-Phase 2 meeting with the FDA for its DTX301 gene therapy aimed at treating ornithine transcarbamylase (OTC) deficiency. The finalized Phase 3 study design includes a 64-week efficacy analysis with approximately 50 patients. Co-primary endpoints will assess plasma ammonia levels and patient response. The trial is set to begin in the second half of 2021, with all initial placebo patients eligible for treatment afterward. DTX301 has shown promising results in earlier trials and has Orphan Drug Designation in both the U.S. and Europe.
The n-Lorem Foundation has partnered with Ultragenyx Pharmaceutical (NASDAQ:RARE) to provide free, lifetime supplies of individualized RNA-targeted medicines for patients with ultra-rare diseases. This collaboration aims to enhance n-Lorem's mission of delivering potentially life-saving treatments for conditions that affect 1 to 30 patients globally. Both organizations aim to expedite access to treatments using advanced genomic diagnostics and antisense oligonucleotides (ASOs). The partnership promises to bring hope and rapid treatment for patients suffering from rare genetic disorders.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced FDA clearance for the Investigational New Drug (IND) application of UX053, an mRNA therapy for Glycogen Storage Disease Type III (GSDIII). This paves the way for clinical trials, with enrollment expected in H2 2021. UX053 could become the first pharmacologic option for GSDIII patients, who currently have no approved treatments. The Phase 1/2 trial will assess safety, tolerability, and efficacy among 28 adults. GSDIII affects over 10,000 patients in developed countries, highlighting the need for effective therapies.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that CEO Emil D. Kakkis will present at the Barclays Global Healthcare Conference on March 10, 2021, at 4:45 PM ET. The presentation will be available via live and archived webcast on the company’s website for 90 days. Ultragenyx focuses on developing treatments for rare and ultra-rare genetic diseases, with a commitment to delivering effective therapies efficiently. The company emphasizes the urgency of meeting high unmet medical needs through innovative drug development strategies.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has announced that CEO Emil D. Kakkis will present at two upcoming investor conferences. The first is the SVB Leerink 10th Annual Global Healthcare Conference on February 24, 2021, at 2:20 PM ET, followed by the Cowen 41st Annual Health Care Conference on March 1, 2021, at 2:00 PM ET. Live and archived webcasts of the presentations will be available on the company’s website for 90 days. Ultragenyx focuses on therapies for rare genetic diseases, emphasizing urgent drug development for unmet medical needs.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has received Health Canada's approval for Dojolvi™ (triheptanoin), the first treatment for Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) in adults and pediatric patients. This synthetic triglyceride serves as a vital energy source for individuals affected by this life-threatening genetic condition. The product is expected to be available in Canada by April 2021, with Ultragenyx working on insurance coverage through its UltraCare® program to assist patients.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) reported its 2020 financial results, with total revenue reaching $271.0 million and Crysvita revenue of $138.9 million. The 2021 guidance for Crysvita revenue is reaffirmed at $180 million to $190 million. The company launched Dojolvi in July 2020, serving approximately 130 patients in the U.S. with a significant quarterly growth. Operating expenses were $601.1 million, leading to a net loss of $186.6 million. Key clinical trials are set to advance, including three pivotal gene therapy studies in 2021.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has announced a conference call on February 11, 2021, at 5 pm ET, to discuss its fourth-quarter and full-year financial results for 2020. The call will provide a corporate update and insights into the company’s progress in developing novel treatments for serious rare and ultra-rare genetic diseases. The information will be accessible through their website, with a dedicated phone line for participants.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has received FDA clearance for the IND application of UX701, a gene therapy targeting Wilson Disease. The company plans to initiate a seamless Phase 1/2/3 clinical trial in the first half of 2021, evaluating safety and efficacy across multiple dosing levels. UX701 aims to improve copper regulation by delivering a modified ATP7B copper transporter. This therapy could potentially alleviate unmet medical needs for patients suffering from Wilson Disease, which affects over 50,000 individuals in developed regions.
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