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 has announced the promotion of Dr. Eric Crombez to chief medical officer and executive vice president, effective May 1, 2023. He succeeds Dr. Camille Bedrosian, who will transition to a strategic advisory role. Dr. Crombez, who has been pivotal in developing clinical programs for rare genetic disorders, previously served as CMO for gene therapy and inborn errors of metabolism. Under his leadership, the company aims to enhance its pipeline focused on treatments for rare diseases. Ultragenyx is committed to delivering therapies with high unmet needs efficiently and effectively.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced that CEO Emil D. Kakkis will speak at the Orphan Neuro Panel during Cowen's 43rd Annual Health Care Conference on March 7, 2023, at 10:30 a.m. ET. This event highlights the company's commitment to developing therapies for rare genetic diseases. The live and archived webcast will be available on the company's website for 30 days following the event. Ultragenyx focuses on creating innovative products to meet urgent medical needs with no existing treatments available, led by a team skilled in rare disease therapeutics.
Ultragenyx Pharmaceutical reported a total revenue of $363.3 million for 2022, marking a growth from $351.4 million in 2021. Key drivers included Crysvita, contributing $279.4 million, and Dojolvi with $55.6 million. For 2023, the company projects total revenue between $425 million and $450 million, with Crysvita revenue expected to reach $325 million to $340 million. Despite strong revenue growth, Ultragenyx reported a net loss of $707.4 million for 2022, an increase from $454 million in 2021. The company also holds a healthy cash balance of $896.7 million as of December 31, 2022.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced a conference call on February 16, 2023, at 5:00 p.m. ET, to discuss its financial results for Q4 and the full year ending December 31, 2022. This call will provide insights into the company's performance in developing therapies for rare genetic diseases. Interested participants can access the live call via the company's website, and a replay will be available for one year. Ultragenyx focuses on novel therapies to meet the high unmet medical needs of patients with rare diseases.
Ultragenyx Pharmaceutical reported preliminary total product revenue for 2022 between $352 million to $356 million, with Crysvita revenue estimated at $257 million to $258 million and Dojolvi revenue at $55 million to $56 million. For 2023, the company guides total revenue of $425 million to $450 million, driven by Crysvita and Dojolvi. The cash balance at year-end 2022 was approximately $900 million. Expectations for net cash used in operations in 2023 are under $400 million. The company emphasizes advancements in its pipeline and a focus on reducing cash spending.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that CEO Emil D. Kakkis will present at the 41st Annual J.P. Morgan Healthcare Conference on January 10, 2023, at 12:45 PM ET. The presentation will focus on the company's commitment to developing therapies for rare genetic diseases. A live webcast will be available on their website, with a replay accessible for 30 days afterward. Ultragenyx aims for efficient drug development to provide effective treatments for patients with significant unmet medical needs.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced the grant of stock options and restricted stock units to three newly hired non-executive officers, totaling 29,950 shares and 17,505 units, respectively. The awards, approved by the compensation committee, are part of the Ultragenyx Employment Inducement Plan with a grant date of December 16, 2022. Stock options vest over four years with a ten-year term at an exercise price of $45.63 per share. This move reflects Ultragenyx's commitment to attracting talent essential for developing therapies for rare diseases.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that CEO Emil D. Kakkis will present at the Piper Sandler Annual Healthcare Conference on November 29, 2022, at 10:00 AM ET in New York.
The live presentation and archived webcast will be available on the company’s website for 120 days. Ultragenyx is dedicated to developing therapies for serious rare and ultra-rare genetic diseases, focusing on unmet medical needs with a diverse portfolio of treatments.
Ultragenyx Pharmaceutical (NASDAQ: RARE) has approved stock options and restricted stock units for a newly hired non-executive officer. The grant, dated November 16, 2022, includes 5,380 stock options and 5,000 restricted stock units. The stock options have a ten-year term with an exercise price of $35.22. Both the stock options and restricted stock units vest over four years, contingent on continuous employment. This decision aligns with Nasdaq Listing Rule 5635(c)(4) and reflects the company’s commitment to attracting talent in the biopharmaceutical sector focused on rare diseases.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced that CFO Mardi Dier will leave the company on November 15, 2022, for a new role at ACELYRIN, INC. Ted Huizenga and Aaron Olsen will manage finance activities during the search for Dier's successor. CEO Emil Kakkis praised Dier's contributions over her two-plus years at Ultragenyx, highlighting the company's fiscal responsibility heading into 2023. Ultragenyx focuses on developing novel therapies for rare genetic diseases, aiming to provide safe and effective treatments.
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