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 Inc. (NASDAQ: RARE) announced that CEO Emil D. Kakkis will present at two upcoming investor conferences. The events include the Stifel Healthcare Conference on November 15, 2022, at 1:50 PM EST in New York and the Jefferies London Healthcare Conference on November 17, 2022, at 10:25 AM GMT. Live and archived webcasts will be available on the company's website for 90 days. Ultragenyx is dedicated to developing novel therapies for serious rare diseases, focusing on unmet medical needs with its diverse portfolio of approved treatments.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) reported third-quarter 2022 total revenue of $90.7 million, including $64.5 million from Crysvita. The company reaffirms its 2022 revenue guidance for Crysvita between $250 million and $260 million, and Dojolvi between $55 million and $65 million. Operating expenses reached $315.8 million, leading to a net loss of $245.1 million or $3.50 per share. Key clinical milestones include completion of enrollment for several pivotal trials expected by year-end.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced a conference call on November 2, 2022, at 5:00 PM ET to discuss Q3 2022 financial results and corporate updates. This call will provide insights into the company's performance and strategic direction as it focuses on developing therapies for rare genetic diseases. The live webcast will be accessible via the company's website, along with a replay available for a year. Ultragenyx aims to deliver effective therapies promptly, addressing significant unmet medical needs in the rare disease space.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced the grant of non-qualified stock options totaling 13,000 shares and 6,500 restricted stock units to a newly hired non-executive officer. Approved by the compensation committee, these awards are part of the Employment Inducement Plan under Nasdaq Listing Rule 5635(c)(4). The options have a ten-year term with an exercise price of $41.41 per share. The stock options and restricted stock units vest over four years, contingent on continuous employment.
On September 9, 2022, Ultragenyx Pharmaceutical (NASDAQ: RARE) and Mereo BioPharma (NASDAQ: MREO) announced the presentation of new data from Mereo's Phase 2b ASTEROID study and details of the Phase 2/3 Orbit study at the ASBMR Annual Meeting in Austin, Texas, running from September 9-12, 2022. Setrusumab, a monoclonal antibody for Osteogenesis Imperfecta (OI), will be discussed, highlighting its significance for the 60,000 OI patients in the US and Europe. Key presentations include results from the ASTEROID study and insights from the IMPACT Survey.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that Eric Crombez, Chief Medical Officer of Gene Therapy, will join a Rare Disease Panel at Citi's 17th Annual BioPharma Conference on September 7, 2022, at 11:20 AM ET. This panel discussion highlights the company’s commitment to addressing serious rare and ultra-rare genetic diseases. Participants can access the live and archived webcast on Ultragenyx's website, available for 90 days post-event. Ultragenyx focuses on developing innovative therapies for high unmet medical needs in rare diseases.
On August 17, 2022, Ultragenyx Pharmaceutical (NASDAQ: RARE) announced the granting of stock options and restricted stock units to two new non-executive officers. A total of 19,020 stock options and 12,910 restricted stock units were awarded under the Ultragenyx Employment Inducement Plan, following Nasdaq Listing Rule 5635(c)(4). The options have a ten-year term with an exercise price of $49.31, matching the closing price on the grant date. The vesting schedule includes a four-year period with certain conditions based on continued employment.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced the grant of non-qualified stock options for 27,500 shares and 14,500 restricted stock units to two newly hired non-executive officers. This award follows the Ultragenyx Employment Inducement Plan and was approved by the compensation committee on August 1, 2022. The stock options have a ten-year term with an exercise price of $50.63 per share. The restricted stock units vest over four years, promoting employee retention and aligning interests with shareholders.
Ultragenyx Pharmaceutical reported Q2 2022 total revenue of $89.3 million, with Crysvita revenue at $64.0 million. They reaffirmed 2022 revenue guidance for Crysvita between $250 million and $260 million and Dojolvi between $55 million and $65 million. The company completed a $500 million royalty interest sale for Crysvita. Operating expenses reached $230.9 million, leading to a net loss of $158.2 million. The firm continues to develop its extensive pipeline, including late-stage products for various rare diseases.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) will host a conference call on July 28, 2022, at 5:00 PM ET to discuss its financial results and corporate updates for Q2 2022. The call will be available via live webcast on the company's website. Ultragenyx focuses on developing therapies for rare genetic diseases, boasting a portfolio of approved medicines and candidates for unmet medical needs. The company emphasizes efficient drug development to deliver timely therapies to patients.
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