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 Pharmaceutical (NASDAQ: RARE) announced the commercial availability of Dojolvi™ (triheptanoin) for treating long-chain fatty acid oxidation disorders (LC-FAOD) following FDA approval on June 30, 2020. This marks the first FDA-approved therapy for LC-FAOD, a serious condition affecting approximately 2,000 to 3,500 patients in the U.S. Dojolvi aims to provide essential calories and fatty acids, with support available through Ultragenyx's UltraCare program for patient access. Caution is advised due to potential gastrointestinal side effects and contraindications for certain patients.
Ultragenyx Pharmaceutical has received FDA approval for Dojolvi™ (triheptanoin), a treatment for long-chain fatty acid oxidation disorders (LC-FAOD), marking its fourth FDA approval in three years. Dojolvi offers a new source of calories and fatty acids for patients, many of whom face serious health challenges despite existing treatments. The company estimates LC-FAOD affects 2,000 to 3,500 individuals in the U.S. and plans to make Dojolvi available within 30 days through its UltraCare® program for patient support and insurance navigation.
Ultragenyx Pharmaceutical (NASDAQ: RARE) and Kyowa Kirin announced FDA approval for Crysvita (burosumab) as the first therapy for tumor-induced osteomalacia (TIO) that cannot be surgically removed. This decision represents the second FDA-approved indication for Crysvita, previously authorized for X-linked hypophosphatemia (XLH). TIO affects approximately 500-1,000 patients in the U.S., and half are inoperable. Crysvita acts by blocking FGF23, alleviating symptoms like severe hypophosphatemia. The approval follows successful Phase 2 trials, showcasing significant improvements in phosphate levels and bone health.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has announced virtual presentations at investor conferences. CFO Shalini Sharp will present at the Jefferies Healthcare Conference on June 3, 2020, at 2 PM ET, followed by CEO Emil D. Kakkis at the Goldman Sachs 41st Annual Global Healthcare Conference on June 9, 2020, at 3:50 PM ET. Live and archived webcasts will be available on the company's website for 90 days post-event. Ultragenyx is focused on developing novel treatments for rare genetic diseases, aiming for efficient drug development to meet high unmet medical needs.
Ultragenyx Pharmaceutical (Nasdaq: RARE) has exercised an option to purchase 600,000 shares of Arcturus Therapeutics (Nasdaq: ARCT) at $16.00 each, increasing its ownership to 3,000,000 shares, or 14.6% of Arcturus. This investment supports the advancement of Arcturus's mRNA platform, including a COVID-19 vaccine candidate, and Ultragenyx's preclinical mRNA candidate for Glycogen Storage Disease Type III. The strategic collaboration between both companies focuses on developing nucleic acid therapies for rare diseases, with an IND application for UX053 anticipated in 2021.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced positive long-term data from its Phase 1/2 study of DTX301, a gene therapy for ornithine transcarbamylase (OTC) deficiency. All three patients in Cohort 3 responded, maintaining clinical stability after stopping ammonia scavengers. Six of nine patients showed a response. Key results include significant improvements in ureagenesis and ammonia levels. No serious adverse events were reported. The company aims to start a Phase 3 trial in early 2021, with ammonia levels as a primary endpoint. A recap call is scheduled for May 15, 2020.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that CFO Shalini Sharp will present at the BofA Securities Global Health Care Conference on May 14, 2020, at 10:20 AM ET. The event will be available via live and archived webcast on Ultragenyx's website for 90 days post-event. The company focuses on developing therapies for serious rare and ultra-rare genetic diseases, aiming for efficient drug development and urgency in delivering effective treatments to patients with high unmet medical needs.
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