Welcome to our dedicated page for Ultragenyx Pharm news (Ticker: RARE), a resource for investors and traders seeking the latest updates and insights on Ultragenyx Pharm stock.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) is a biopharmaceutical company focused on novel therapies for serious rare and ultra-rare genetic diseases. The RARE news feed highlights company announcements on commercial performance, clinical development, and key corporate decisions that shape its rare disease portfolio.
Investors and followers of Ultragenyx can find updates on revenue trends from its approved products Crysvita, Dojolvi, Mepsevii, and Evkeeza in select territories, as well as guidance ranges and commentary on operating expenses and cash position. Earnings-related press releases and Form 8-K summaries detail product sales, royalty revenue, and royalty financing transactions, including agreements involving future Crysvita royalties with OMERS.
The news stream also covers Ultragenyx’s late-stage pipeline and regulatory milestones. Readers can track progress of AAV gene therapy programs such as DTX401 for glycogen storage disease type Ia, UX111 for Sanfilippo syndrome type A, and UX701 for Wilson disease, along with monoclonal antibody UX143 for osteogenesis imperfecta and antisense oligonucleotide GTX-102 for Angelman syndrome. Company releases describe Phase 3 study results, longer-term follow-up data, regulatory designations, rolling BLA submissions, and responses to FDA communications such as Complete Response Letters.
In addition, the RARE news page features items on conference presentations, investor events, and equity inducement grants under Nasdaq Listing Rule 5635(c)(4). By reviewing these updates, users can follow how Ultragenyx manages its commercial portfolio, advances its clinical pipeline, and executes financing and collaboration strategies in the rare and ultra-rare disease space.
Ultragenyx and Mereo BioPharma announced positive 14-month results from their ongoing Phase 2/3 Orbit study of setrusumab (UX143) for Osteogenesis Imperfecta (OI). The treatment showed a substantial 67% reduction in annualized fracture rate and a median annualized fracture rate of 0.00 (p=0.0014). Bone mineral density (BMD) improved by 22% on average (p<0.0001), with a mean Z-score increase of +1.25 (p<0.0001).
The study, which included 24 patients treated for at least 14 months, demonstrated sustained reductions in fractures. No treatment-related serious adverse events were reported, and adverse events were consistent with previous studies.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced its participation in the Goldman Sachs 45th Annual Global Healthcare Conference. Dr. Emil Kakkis, the CEO, will engage in a fireside chat on June 11, 2024, at 10:00 a.m. ET. The event will be live-streamed and available for later viewing on Ultragenyx's investor relations website.
Ultragenyx announced positive top-line results from its Phase 3 GlucoGene study evaluating DTX401, a gene therapy for glycogen storage disease type Ia (GSDIa).
The therapy showed a statistically significant reduction in daily cornstarch intake at Week 48 compared to placebo (41.3% vs. 10.3%).
DTX401 also improved glucose control and reduced the number of daily cornstarch doses. Safety profile was consistent with previous studies, showing non-serious, manageable effects.
The results will be discussed with regulatory authorities for a marketing application in 2025.
Ultragenyx Pharmaceutical, a biopharmaceutical company focused on therapies for rare diseases, announced the inducement grant of 17,180 restricted stock units (RSUs) to ten newly hired non-executive officers. The awards, approved by the board's compensation committee under the Ultragenyx Employment Inducement Plan, were granted on May 16, 2024. These RSUs vest over four years, with 25% vesting annually, conditional on continuous employment. This grant aligns with Nasdaq Listing Rule 5635(c)(4), serving as a key employment incentive.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) will have its chief medical officer, Eric Crombez, participate in a fireside at Bank of America’s 2024 Healthcare Conference on May 14, 2024. The company focuses on developing therapies for rare genetic diseases.
Ultragenyx Pharmaceutical Inc. reported first quarter total revenue of $109 million with Crysvita® revenue at $83 million and Dojolvi® revenue at $16 million. The company reaffirmed its 2024 total revenue guidance of $500 million to $530 million, with Crysvita revenue between $375 million to $400 million and Dojolvi revenue at $75 million to $80 million. Positive Phase 1/2 data from the GTX-102 Angelman syndrome study was presented, showing significant improvement. The company continues to see revenue growth and has completed enrollment for its Phase 3 program in osteogenesis imperfecta.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) released its 2023 Corporate Responsibility Report, emphasizing its commitment to improving equity and access to innovation in rare diseases. The report showcases efforts in drug development, patient support, employee diversity, community impact, environmental sustainability, and governance. Ultragenyx received multiple awards for its corporate responsibility initiatives, including high rankings in the biotech industry and recognition as a top workplace. The company's dedication to rare disease medicine and community welfare is evident in its comprehensive report.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) completed patient enrollment in Phase 3 studies for setrusumab (UX143) in treating osteogenesis imperfecta (OI). The Orbit study included 158 patients aged 5-25, while the Cosmic study enrolled 66 patients aged 2-7. Positive Phase 2 results showcased a 67% reduction in fracture rates and significant improvements in bone mineral density. Setrusumab demonstrated good tolerability with no serious adverse events reported. The company aims to provide a new treatment option for OI patients, enhancing their quality of life by strengthening bones.
Summary not available.
Summary not available.