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 (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.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced it will present data at the American Society of Gene & Cell Therapy (ASGCT) 24th Annual Meeting, held virtually from May 11-14, 2021. The presentations will cover their investigational gene therapy programs, including advancements in their HeLa producer cell line for AAV production, and long-term results from Phase 1/2 studies on Ornithine Transcarbamylase (OTC) Deficiency and Glycogen Storage Disease Type Ia (GSDIa). Key presentations are scheduled for May 11, 13, and 14.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced the completion of an End-of-Phase 2 meeting with the FDA for its DTX301 gene therapy aimed at treating ornithine transcarbamylase (OTC) deficiency. The finalized Phase 3 study design includes a 64-week efficacy analysis with approximately 50 patients. Co-primary endpoints will assess plasma ammonia levels and patient response. The trial is set to begin in the second half of 2021, with all initial placebo patients eligible for treatment afterward. DTX301 has shown promising results in earlier trials and has Orphan Drug Designation in both the U.S. and Europe.
The n-Lorem Foundation has partnered with Ultragenyx Pharmaceutical (NASDAQ:RARE) to provide free, lifetime supplies of individualized RNA-targeted medicines for patients with ultra-rare diseases. This collaboration aims to enhance n-Lorem's mission of delivering potentially life-saving treatments for conditions that affect 1 to 30 patients globally. Both organizations aim to expedite access to treatments using advanced genomic diagnostics and antisense oligonucleotides (ASOs). The partnership promises to bring hope and rapid treatment for patients suffering from rare genetic disorders.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced FDA clearance for the Investigational New Drug (IND) application of UX053, an mRNA therapy for Glycogen Storage Disease Type III (GSDIII). This paves the way for clinical trials, with enrollment expected in H2 2021. UX053 could become the first pharmacologic option for GSDIII patients, who currently have no approved treatments. The Phase 1/2 trial will assess safety, tolerability, and efficacy among 28 adults. GSDIII affects over 10,000 patients in developed countries, highlighting the need for effective therapies.
FAQ
What is the current stock price of Ultragenyx Pharmaceutical (RARE)?
What is the market cap of Ultragenyx Pharmaceutical (RARE)?
What is the primary focus of Ultragenyx Pharmaceutical Inc.?
What are some key products of Ultragenyx?
What were the financial highlights for Ultragenyx in 2023?
What recent achievements has Ultragenyx made in clinical trials?
Who leads Ultragenyx Pharmaceutical Inc.?
What partnerships does Ultragenyx have?
What is Ultragenyx's commitment to corporate responsibility?
How does Ultragenyx engage with the rare disease community?
What are the future plans for GTX-102?
Where can I find more information about Ultragenyx?
