MARKET COMPOSITE
RARE - Ultragenyx Pharmaceutical Inc.8:00:00 PM 5/1/2024
Price
$43.30
+ 0.76 (1.79%)
Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in the United States. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia; and Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII. The company is also developing small-molecule pipeline comprising UX007, a synthetic triglyceride for the treatment of long chain fatty-acid oxidation disorders, which is a set of rare metabolic diseases that prevents the conversion of fat into energy; and gene therapy pipeline consisting of DTX301, an adeno-associated virus 8 gene therapy product candidate for the treatment of patients with ornithine transcarbamylase, as well as DTX401, an AAV8 gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia. In addition, the company is developing UX068, which is in preclinical development for the treatment of creatine transporter deficiency (CTD); and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Arcturus Therapeutics Holdings Inc. to develop additional nucleic acid therapies for rare diseases. Ultragenyx Pharmaceutical Inc. was founded in 2010 and is headquartered in Novato, California.
Financials
Quarterly financials
(USD)Dec 2023Q/Q
Revenue127.4MM+30%
Operating Income-122MM-16%
Operating Expenses249.4MM-
Net Income-123.2MM-23%
R&D160.6MM+2%
G&A76.8MM+3%
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Stock news

    NOVATO, Calif., April 30, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today released its 2023 Corporate Responsibility Report, which showcases the company’s efforts to transform the lives of individuals and families impacted by rare and ultrarare diseases, empower its employees, and make a meaningful impact on the communities it partners with. “In rare disease, corporate responsibility means pushing for progress and change to support drug development and access for the

    NOVATO, Calif., April 30, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that all patients have been enrolled across the Phase 3 Orbit and Cosmic studies evaluating setrusumab (UX143) in pediatric and young adult patients with osteogenesis imperfecta (OI). The pivotal Phase 3 portion of the Orbit study has randomized 158 patients ages 5 to 25 years, and the Cosmic study has completed enrollment of 66 patients ages 2 to <7 years. “We would like to thank the

    NOVATO, Calif., April 25, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, today announced that it will host a conference call at 5:00 p.m. ET on Thursday, May 2, 2024, to discuss its financial results and corporate update for the quarter ending March 31, 2024. The live and replayed webcast of the call will be available through the

    NOVATO, Calif., April 19, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today reported the grant of 44,965 restricted stock units of the company’s common stock to 14 newly hired non-executive officers of the company. The awards were approved by the compensation committee of the company’s board of directors and granted under the Ultragenyx Empl

    Ultragenyx (RARE) reports positive interim data from the phase I/II study of GTX-102 for Angelman syndrome. However, the stock declines due to safety concerns.

    Treatment appeared to result in functional and cognitive gains in people with the neurological disorder. Three participants experienced lower extremity weakness, however.

    Expansion Cohorts showed rapid, clinically meaningful improvement across multiple domains; improvements consistent or exceeding Dose-escalation Cohorts data at Day 170 Additional long-term data in Dose-escalation Cohorts showed increasing and sustained clinical benefit through Day 758 Company will host investor call at 8:00 a.m. ET NOVATO, Calif., April 15, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced new data from the Phase 1/2 study of GTX-102 for the

    Conference call now scheduled for Monday, April 15 at 8:00 a.m. ET Updated registration link below NOVATO, Calif., April 12, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the conference call to discuss new clinical efficacy and safety data from the Phase 1/2 study of GTX-102 for the treatment of Angelman syndrome that will be presented at the American Academy of Neurology Meeting (AAN), will now be taking place on Monday, April 15, 2024 at 8 a.m. Eas

    Company will host investor call on April 17 at 8:00 a.m. ET to discuss new data from GTX-102 Phase 1/2 clinical study in patients with Angelman syndromeNOVATO, Calif., April 12, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that preclinical and clinical data, including new clinical efficacy and safety data from the expansion cohorts in the Phase 1/2 study of GTX-102 for the treatment of Angelman syndrome, will be presented at the 76th Annual American Acad

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