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 unaudited 2020 revenues for Crysvita between $137 million and $139 million, aligned with prior guidance despite COVID-19 challenges. The company projects 2021 revenues for Crysvita between $180 million and $190 million. As of December 31, 2020, their cash position was approximately $1.2 billion. The PR highlights advancements in clinical trials for several therapies, including DTX401 and DTX301, with expectations for Phase 3 studies commencing in 2021.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced favorable long-term safety and efficacy data from Phase 1/2 studies of DTX401 for Glycogen Storage Disease Type Ia (GSDIa) and DTX301 for ornithine transcarbamylase (OTC) deficiency. All patients in these studies showed improved metabolic control, with significant cornstarch dependency reduction. Phase 3 trials for both therapies are set to initiate in 2021. Additionally, Ultragenyx submitted an IND for UX701 targeting Wilson Disease, with clinical entry expected in the first half of 2021, leveraging their HeLa PCL platform for manufacturing.
Ultragenyx Pharmaceutical, a biopharmaceutical firm focused on rare genetic diseases, announced that CEO Emil D. Kakkis will present at the 39th Annual J.P. Morgan Virtual Healthcare Conference. The presentation is scheduled for January 12, 2021, at 2:50 PM EST. The live webcast will be accessible from their website, with a replay available for 30 days. Ultragenyx aims to develop novel therapies to address high unmet medical needs in rare diseases, guided by an experienced management team dedicated to efficient drug development.
Ultragenyx Pharmaceutical (Nasdaq: RARE) has entered a collaboration with Mereo BioPharma for setrusumab, a monoclonal antibody targeting osteogenesis imperfecta (OI). Ultragenyx will lead development, paying an upfront fee of $50 million and up to $254 million in milestones. OI affects around 60,000 patients in developed countries, and there are currently no approved therapies. Setrusumab shows promise in increasing bone density and strength, with plans for pediatric studies set to begin in 2021.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced that the FDA has granted Orphan Drug Designation to UX701 for treating Wilson disease, a rare metabolic disorder. This designation marks an important milestone in UX701's development, addressing a significant unmet need as current treatments often lead to severe side effects. UX701 is an investigational gene therapy aimed at normalizing copper metabolism in the liver. The company plans to file an IND application by the end of the year.
The press release discusses the presentation of data from a Phase 1/2 study of GTX-102, an experimental treatment for Angelman syndrome, at the FAST Global Summit. Key findings indicate initial clinical activity, as reflected in positive EEG results and improvements in communication scores among patients. Importantly, there were no new serious adverse events reported. The companies aim to resume the trial with a modified design to minimize risks observed at higher doses. GTX-102 has received multiple FDA designations, emphasizing its potential in treating this rare disease.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced that CEO Emil D. Kakkis will present at two upcoming investor conferences. The Piper Sandler's 32nd Annual Healthcare Conference will be held on December 1, 2020, with a pre-recorded presentation available on the company's website. Additionally, Kakkis will present at the Evercore ISI HealthCONx Conference on December 2, 2020, at 1:00 PM ET. A live webcast will be accessible via the company's site, with replays available for 90 days.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has announced plans to construct a 100,000 square foot gene therapy manufacturing facility in Bedford, Massachusetts. This state-of-the-art facility will allow in-house production of their AAV-based gene therapies, including DTX301, DTX401, and UX701. The facility is projected to create 100 to 150 jobs over five years. Construction is underway, with completion expected in 2023. The facility will improve production processes and scalability to meet demand for Ultragenyx's clinical and approved products.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that CEO Emil D. Kakkis will present at two upcoming investor conferences: the Credit Suisse 29th Annual Virtual Healthcare Conference on November 10, 2020, at 2:00 PM ET, and the Barclays Gene Editing & Gene Therapy Summit on November 16, 2020, at 3:00 PM ET. Live and archived webcasts of the presentations will be available on the company's website for 90 days. Ultragenyx focuses on developing therapies for serious rare and ultra-rare genetic diseases, with an experienced management team committed to urgent and efficient drug development.
Ultragenyx Pharmaceutical (NASDAQ:RARE) has successfully completed an underwritten public offering of 5,111,110 shares of common stock at $90.00 per share, raising approximately $435.4 million in net proceeds after underwriting costs. The offering included the full exercise of an option for an additional 666,666 shares by underwriters. J.P. Morgan, Goldman Sachs, BofA Securities, and Cowen served as joint book-running managers for this offering. Ultragenyx focuses on developing novel therapies for rare genetic diseases, reinforcing its commitment to urgent drug development.
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