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.
The n-Lorem Foundation has partnered with Ultragenyx Pharmaceutical (NASDAQ:RARE) to provide free, lifetime supplies of individualized RNA-targeted medicines for patients with ultra-rare diseases. This collaboration aims to enhance n-Lorem's mission of delivering potentially life-saving treatments for conditions that affect 1 to 30 patients globally. Both organizations aim to expedite access to treatments using advanced genomic diagnostics and antisense oligonucleotides (ASOs). The partnership promises to bring hope and rapid treatment for patients suffering from rare genetic disorders.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced FDA clearance for the Investigational New Drug (IND) application of UX053, an mRNA therapy for Glycogen Storage Disease Type III (GSDIII). This paves the way for clinical trials, with enrollment expected in H2 2021. UX053 could become the first pharmacologic option for GSDIII patients, who currently have no approved treatments. The Phase 1/2 trial will assess safety, tolerability, and efficacy among 28 adults. GSDIII affects over 10,000 patients in developed countries, highlighting the need for effective therapies.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that CEO Emil D. Kakkis will present at the Barclays Global Healthcare Conference on March 10, 2021, at 4:45 PM ET. The presentation will be available via live and archived webcast on the company’s website for 90 days. Ultragenyx focuses on developing treatments for rare and ultra-rare genetic diseases, with a commitment to delivering effective therapies efficiently. The company emphasizes the urgency of meeting high unmet medical needs through innovative drug development strategies.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has announced that CEO Emil D. Kakkis will present at two upcoming investor conferences. The first is the SVB Leerink 10th Annual Global Healthcare Conference on February 24, 2021, at 2:20 PM ET, followed by the Cowen 41st Annual Health Care Conference on March 1, 2021, at 2:00 PM ET. Live and archived webcasts of the presentations will be available on the company’s website for 90 days. Ultragenyx focuses on therapies for rare genetic diseases, emphasizing urgent drug development for unmet medical needs.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has received Health Canada's approval for Dojolvi™ (triheptanoin), the first treatment for Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) in adults and pediatric patients. This synthetic triglyceride serves as a vital energy source for individuals affected by this life-threatening genetic condition. The product is expected to be available in Canada by April 2021, with Ultragenyx working on insurance coverage through its UltraCare® program to assist patients.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) reported its 2020 financial results, with total revenue reaching $271.0 million and Crysvita revenue of $138.9 million. The 2021 guidance for Crysvita revenue is reaffirmed at $180 million to $190 million. The company launched Dojolvi in July 2020, serving approximately 130 patients in the U.S. with a significant quarterly growth. Operating expenses were $601.1 million, leading to a net loss of $186.6 million. Key clinical trials are set to advance, including three pivotal gene therapy studies in 2021.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has announced a conference call on February 11, 2021, at 5 pm ET, to discuss its fourth-quarter and full-year financial results for 2020. The call will provide a corporate update and insights into the company’s progress in developing novel treatments for serious rare and ultra-rare genetic diseases. The information will be accessible through their website, with a dedicated phone line for participants.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has received FDA clearance for the IND application of UX701, a gene therapy targeting Wilson Disease. The company plans to initiate a seamless Phase 1/2/3 clinical trial in the first half of 2021, evaluating safety and efficacy across multiple dosing levels. UX701 aims to improve copper regulation by delivering a modified ATP7B copper transporter. This therapy could potentially alleviate unmet medical needs for patients suffering from Wilson Disease, which affects over 50,000 individuals in developed regions.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) reported preliminary unaudited 2020 revenues for Crysvita between $137 million and $139 million, aligned with prior guidance despite COVID-19 challenges. The company projects 2021 revenues for Crysvita between $180 million and $190 million. As of December 31, 2020, their cash position was approximately $1.2 billion. The PR highlights advancements in clinical trials for several therapies, including DTX401 and DTX301, with expectations for Phase 3 studies commencing in 2021.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced favorable long-term safety and efficacy data from Phase 1/2 studies of DTX401 for Glycogen Storage Disease Type Ia (GSDIa) and DTX301 for ornithine transcarbamylase (OTC) deficiency. All patients in these studies showed improved metabolic control, with significant cornstarch dependency reduction. Phase 3 trials for both therapies are set to initiate in 2021. Additionally, Ultragenyx submitted an IND for UX701 targeting Wilson Disease, with clinical entry expected in the first half of 2021, leveraging their HeLa PCL platform for manufacturing.