In conjunction with the 2025 J.P. Morgan Healthcare Conference, the analyst hosted an investor lunch with Ultragenyx Pharmaceutical Inc RARE.The analyst keeps an Overweight rating on Ultragenyx with a price target of $102 and says the company is uniquely positioned in the long term with an expansive and diverse pipeline of orphan disease assets and highly regarded management. JP Morgan sees multiple value-creating catalysts over the next six to 18 months.Ultragenyx’s recent meeting focused on setrusumab and ...

2024 Highlights Include Proposed Strategic Business Combination and Progress at Flagship Peak ProjectKey Takeaways Tactical Resources’ proposed business combination with Plum Acquisition Corp. III sets the stage for a Nasdaq listing and the opportunity to raise significant growth capital.The Peak Project’s strategic readiness positions Tactical Resources to support U.S. critical mineral needs amid China’s export restrictions.Advancements in Phase 1 technical work and corporate alignment with U.S. defense ob ...

财务业绩与报告 - Ultragenyx Pharmaceutical Inc. 预计将在2025年2月报告2024财年全年业绩[6] - 公司公布了2024财年Crysvita®和Dojolvi®的初步未经审计收入数据[6] - 2024财年末的现金、现金等价物及可供出售投资数据已公布[6] - 公司提供了2025财年的财务指引[6] 新闻稿与文件 - 2025年1月12日发布的新闻稿作为Exhibit 99.1附在8-K表格中[7]

Preliminary 2024 total revenue of $555 million to $560 million, exceeding top end of guidance, including Crysvita® revenue of $405 million to $410 million, and Dojolvi® revenue of $87 million to $89 million 2025 expected total revenue guidance of $640 million to $670 millionUX143 (setrusumab) Phase 3 Orbit study for osteogenesis imperfecta progressing to second interim analysis in mid-2025 GTX-102 Phase 3 Aspire study for Angelman syndrome expected to complete enrollment in second half of 2025 NOVATO, Cali ...

Ultragenyx Pharmaceutical (RARE) announced that the European Commission (EC) has expanded the eligible patient population for one of its marketed products, Evkeeza (evinacumab), an ANGPTL3 inhibitor. Per the latest approval, Evkeeza is now indicated as an adjunct to diet and other lipid-lowering therapies to treat children aged six months and older with homozygous familial hypercholesterolemia (HoFH). The EC’s approval makes Evkeeza the first HoFH medicine indicated for this pediatric population in the EU.H ...

NOVATO, Calif., Jan. 06, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's chief executive officer and president, will present at the 43rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025, at 10:30 AM PT. The live and archived webcast of the presentation will b ...

First and only medicine approved in the EU for paediatric patients aged 6-months to 5 years old with HoFH, an ultrarare, inherited form of high cholesterolBASEL, Switzerland, Jan. 06, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialisation of novel therapies for rare and ultrarare genetic diseases, today announced that the European Commission (EC) has extended the approval of Evkeeza® (evinacumab) as an adjunct to d ...

Ultragenyx Pharmaceutical (RARE) announced that it has submitted a biologics license application (BLA) to the FDA for its UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA) under the accelerated approval pathway.Earlier this year, the FDA aligned with Ultragenyx, after a thorough evaluation, on the use of cerebral spinal fluid (CSF) heparan sulfate (HS) as a well-characterized biomarker to support an accelerated approval pathway for mucopolysaccharidoses ...

LONDON, Dec. 20, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialisation of novel therapies for rare and ultrarare genetic diseases, today announced that NHS England has implemented the commissioning of Evkeeza (evinacumab) following the National Institute for Health and Care Excellence (NICE) final guidance in September. The use of Evkeeza in eligible people aged 12 years and older will be routinely commissioned by ...

Company on track to initiate the Aurora study to evaluate GTX-102 in other Angelman syndrome genotypes and in other age groups in 2025NOVATO, Calif., Dec. 19, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), today announced that the first patient has been dosed in the pivotal Phase 3 Aspire study (NCT06617429) evaluating the efficacy and safety of GTX-102, its investigational antisense oligonucleotide (ASO) for Angelman syndrome. "Initiation of patient dosing in our Phase 3 Aspire stu ...