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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 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.
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.
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