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.
GeneTx and Ultragenyx have received approval from the U.K. MHRA for a Phase 1/2 clinical trial of GTX-102, an investigational treatment for Angelman syndrome. Patient enrollment is set to begin in the U.K. and Canada in the second half of 2021, with clinical data expected by year-end. Following a productive FDA meeting, GeneTx plans to submit a revised protocol to resume studies in the U.S. GTX-102 has shown clinical activity in initial patients, and the new trial design aims to improve safety through repeated lower dosing. This therapy addresses a significant unmet medical need.
Ultragenyx Pharmaceutical (NASDAQ: RARE) will have CEO Emil D. Kakkis present at the Goldman Sachs 42nd Annual Healthcare Conference on June 10, 2021, at 3:00 PM ET. The presentation will be available via a live and archived webcast on the company’s website, accessible for 90 days following the event.
Ultragenyx is focused on developing therapies for serious rare genetic diseases and has a diverse portfolio aimed at addressing high unmet medical needs.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced that CEO Emil D. Kakkis will present at the Jefferies Virtual Healthcare Conference on June 1, 2021, at 4:30 PM ET. The event will be accessible via live and archived webcast on the company’s website, with a replay available for 90 days. Ultragenyx specializes in developing therapies for serious rare and ultra-rare genetic diseases and aims to provide safe and effective treatments efficiently.
GeneTx Biotherapeutics and Ultragenyx Pharmaceutical have received Health Canada clearance to enroll patients in a Phase 1/2 study of GTX-102, an investigational treatment for Angelman syndrome. Expected to begin in early 2021, the study will evaluate the safety and tolerability of GTX-102 in pediatric patients. The trial includes two cohorts, enrolling approximately 12 patients aged 4 to 17 years. The first patient in Canada is set to be enrolled in the second half of 2021, with preliminary clinical data anticipated before year-end.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced the grant of stock options and restricted stock units to a new non-executive officer. The compensation committee approved 4,677 stock options and 7,100 restricted stock units under the Ultragenyx Employment Inducement Plan, effective May 16, 2021. The restricted stock units vest over four years, while stock options vest 25% after the first year and monthly thereafter. The exercise price of the stock options is set at $110.88, reflecting the closing price on May 14, 2021. Ultragenyx focuses on novel therapies for rare diseases.
Ultragenyx Pharmaceutical (NASDAQ: RARE) reported positive long-term results from Phase 1/2 trials for its gene therapies DTX401 (for GSDIa) and DTX301 (for OTC deficiency). DTX401 showed durable responses lasting over 2.5 years with a 79% reduction in cornstarch intake, while DTX301 demonstrated stability after more than three years. No serious adverse events were reported in either study. The company is set to initiate Phase 3 studies for both therapies later in 2021, enhancing its proprietary HeLa cell line manufacturing platform, which has improved product yield significantly.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that Mardi Dier, Chief Financial Officer, and Camille Bedrosian, M.D., Chief Medical Officer, will present at the BofA Securities Healthcare Conference on May 12, 2021, at 3:30 PM ET. The live webcast can be accessed from the company's website, with a replay available for 90 days. Ultragenyx focuses on developing therapies for rare genetic diseases and has a diverse portfolio of approved medicines aimed at addressing high unmet medical needs.
Ultragenyx Pharmaceutical reported first quarter 2021 revenue of $99.4 million, with Crysvita revenue in its territories at $42.1 million, marking a 46% increase year-over-year. The company reaffirmed its 2021 Crysvita revenue guidance of $180 million to $190 million. The Dojolvi launch shows strong momentum, currently serving around 180 patients. Operating expenses rose by 31% to $206 million, primarily due to an upfront payment related to a collaboration. As of March 31, 2021, Ultragenyx holds $1.0 billion in cash and equivalents.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced that Emil Kakkis, M.D., Ph.D., will present at the 7th Annual Truist Securities Life Sciences Summit on May 5, 2021, at 1:00 PM ET. The live and archived webcast of the presentation can be accessed through the company's website. Ultragenyx is dedicated to developing therapies for rare genetic diseases, focusing on addressing significant unmet medical needs. The company employs a management team experienced in creating effective treatments efficiently and urgently. For more details, visit www.ultragenyx.com.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced a conference call scheduled for May 4, 2021, at 5 PM ET, to discuss its Q1 financial results for the period ending March 31, 2021. The call will be accessible via the company website and phone. Ultragenyx focuses on developing therapies for serious rare and ultra-rare genetic diseases and has a diverse portfolio of approved treatments. The management team emphasizes efficient drug development to meet urgent patient needs.
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