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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) 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.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) plans to host a conference call on November 2, 2021, at 5 PM ET, to discuss its financial results and corporate update for Q3 2021. The live call can be accessed by phone or via webcast through the company's website. Ultragenyx focuses on developing therapies for rare genetic diseases and has established a diverse portfolio of approved medicines. The company emphasizes efficient drug development to meet urgent patient needs.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced that CEO Emil D. Kakkis will present at the Jefferies Gene Therapy/Editing Summit on October 28, 2021, at 3:30 PM ET. The presentation will focus on the company's commitment to developing therapies for rare and ultra-rare genetic diseases. A live and archived webcast of the event will be available on Ultragenyx's website, with replays accessible for 90 days. The company prioritizes efficient drug development to meet the urgent needs of patients lacking effective treatment options.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced the successful screening and enrollment of patients for its pivotal Phase 1/2/3 study of UX701, an innovative gene therapy for treating Wilson disease. This seamless study design will evaluate the therapy's safety and efficacy in patients currently receiving standard care. Following a structured 52-week period, primary efficacy endpoints include changes in urinary copper concentration and reductions in standard care medication. UX701 aims to address significant unmet needs in a condition affecting over 50,000 individuals in developed countries.
The first patient has been dosed in a Phase 1/2 study for GTX-102, targeting Angelman syndrome, by GeneTx and Ultragenyx (NASDAQ: RARE). The study involves multiple-dose, dose-escalating administration to evaluate safety and efficacy across various patient domains. Preliminary updates will be available by year-end, with complete data expected in mid-2022. GTX-102 aims to reactivate the paternal UBE3A allele, potentially improving neurological symptoms. The study design includes specific dosing for different age cohorts, based on prior successful trials.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced the grant of non-qualified stock options for 8,620 shares and 2,880 restricted stock units to a newly hired non-executive officer. This award, approved by the company's compensation committee, is part of the Ultragenyx Employment Inducement Plan, effective October 1, 2021. The stock options have a ten-year term with an exercise price of $87.80, equal to the stock's closing price on the grant date. The restricted stock units vest over four years, promoting long-term employment alignment.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced that new data on Crysvita® for X-linked hypophosphatemia (XLH) and UX143 (setrusumab) for osteogenesis imperfecta (OI) will be presented at the ASBMR 2021 Annual Meeting from October 1-4 in San Diego. Key presentations include oral sessions on neurological manifestations of XLH and the Phase 2b ASTEROID study results for setrusumab. The conference will also feature real-time virtual access. Crysvita is indicated for treating XLH in patients aged six months and older, showcasing significant advancements in rare disease therapies.
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