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 Inc. (NASDAQ: RARE) reported preliminary 2021 revenue for Crysvita of $191-$193 million, exceeding guidance, and Dojolvi revenue of $38-$40 million. For 2022, Crysvita is projected at $250-$260 million, a 33% increase, while Dojolvi is expected between $55-$65 million, reflecting 60% growth. The year-end 2021 cash position stands at approximately $1 billion. The company is focused on commercializing Evkeeza in Europe and advancing clinical programs for rare diseases.
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Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has announced that CEO Emil D. Kakkis will present at the H.C. Wainwright BioConnect Conference on January 13, 2022, at 7:00 AM ET. The presentation will be available via live and archived webcast on the company's website for 90 days. Ultragenyx focuses on developing therapies for rare and ultra-rare genetic diseases, with a portfolio aimed at addressing conditions with high unmet medical needs. The company emphasizes efficient drug development to deliver urgent therapies to patients.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) and GeneTx Biotherapeutics announced that the first four patients in their Phase 1/2 study of GTX-102, a treatment for Angelman syndrome, have received multiple doses. No serious adverse events were reported, and preliminary assessments indicate early signs of clinical activity. The Data Safety Monitoring Board recommended dose escalation for Cohort 4. Initial improvements in clinical assessments are noted, with further data expected by mid-2022. GTX-102 aims to reactivate the paternal UBE3A allele, addressing symptoms of Angelman syndrome.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that CEO Emil D. Kakkis will present at the 40th Annual JP Morgan Healthcare Conference on January 10, 2022, at 3:45 PM ET. The presentation will be accessible via a live webcast on the company's website, with a replay available for 90 days afterward. Ultragenyx focuses on developing therapies for serious rare and ultra-rare genetic diseases, boasting a diverse portfolio aimed at high unmet medical needs. The company emphasizes efficient drug development to deliver safe and effective treatments urgently.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) and GeneTx Biotherapeutics presented new data on GTX-102, an investigational treatment for Angelman syndrome, at the 2021 FAST Global Summit. The Phase 1/2 study included findings from initial patients, focusing on adaptive behavior scales and EEG readings, indicating potential effectiveness. Enrollment for treatment-naive patients has begun. GTX-102 targets the UBE3A gene, with no approved therapies available for the disorder, which affects 1 in 12,000 to 20,000 people globally. A further update is anticipated by year-end 2021.
Ultragenyx Pharmaceutical (NASDAQ: RARE) has announced the first patient dosing in its Phase 1/2 clinical trial for UX053, an mRNA therapy aimed at treating Glycogen Storage Disease Type III (GSDIII). The trial aims to evaluate safety, tolerability, and efficacy in adults aged 18 and older, with the potential to be the first approved medicine for GSDIII. UX053 targets the deficiency in glycogen debranching enzyme, a condition affecting over 10,000 patients worldwide. The FDA and European Commission have granted Orphan Drug Designation for UX053.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that CEO Emil D. Kakkis will present at the Evercore ISI 4th Annual HealthconX Conference on December 1, 2021, at 12:10 PM ET. The presentation will be available via live and archived webcast on the company's website for 90 days. Ultragenyx is dedicated to developing therapies for serious rare and ultra-rare genetic diseases, with a focus on unmet medical needs. The management team emphasizes efficient drug development to deliver safe and effective treatments rapidly.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced impressive long-term data from Phase 1/2 trials at the ICIEM. For DTX401 in Glycogen Storage Disease Type Ia, mean daily cornstarch intake reduction reached 69.9%, with significant glucose control improvements observed over three years. DTX301 for Ornithine Transcarbamylase deficiency demonstrated durable metabolic stability, with responders stable up to four years post-treatment. Notably, no major adverse events were reported, signaling potential for enhanced patient quality of life.
Ultragenyx Pharmaceutical reported Q3 2021 revenues of $81.6 million, driven by $50.3 million from Crysvita, marking a 35% year-over-year increase. The company anticipates total Crysvita revenue for the year at the higher end of $180 million to $190 million. Dojolvi revenue surged by 176% to $10.7 million. However, operating expenses rose to $171.5 million, a 30% increase due to pipeline advancements, leading to a net loss of $73 million. Ultragenyx is poised for multiple clinical study initiations, aiming to expand its innovative therapies for rare diseases.
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