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. develops and commercializes therapies for serious rare and ultra-rare genetic diseases. Company news commonly covers revenue from approved medicines such as Crysvita, Dojolvi, and Mepsevii, financial guidance, and updates to a portfolio of approved therapies and product candidates for diseases with high unmet medical need and clear biology.
Recurring developments include FDA interactions and clinical data for investigational programs such as UX111 for Sanfilippo syndrome type A, GTX-102 for Angelman syndrome, UX016 for GNE myopathy, and AAV gene therapy candidates for rare metabolic disorders. Updates also include healthcare conference participation, employment inducement equity grants under Nasdaq rules, and corporate restructuring or expense-management actions when disclosed.
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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