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Ultragenyx Pharmaceutical Inc. (RARE) granted 10,995 restricted stock units to nine new non-executive officers under the Employment Inducement Plan. The awards vest over four years, with 25% vesting annually, subject to continuous employment.
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Ultragenyx Pharmaceutical Inc. reported a total revenue of $434 million in 2023, with Crysvita revenue of $328 million and Dojolvi revenue of $71 million. The company provided financial guidance for 2024, expecting total revenue between $500 million and $530 million, Crysvita revenue between $375 million and $400 million, and Dojolvi revenue between $75 million and $80 million. Ultragenyx also disclosed a year-end 2023 cash balance of $777 million and projected net cash used in operations to be less than $400 million for 2024.
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Ultragenyx Pharmaceutical Inc. will host a conference call to discuss its financial results and corporate update for the fourth quarter and the year ending December 31, 2023. The live and replayed webcast of the call will be available through the company’s website. Participants can register for the live call and access the replay for 3 months.
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Ultragenyx Pharmaceutical Inc. (RARE) announced positive data from the pivotal Transpher A and long-term follow-up studies for UX111 (ABO-102) AAV gene therapy for Sanfilippo syndrome type A. The data demonstrated rapid and sustained decreased levels of heparan sulfate (HS) in cerebrospinal fluid (CSF), correlated with improved long-term cognitive development. Treatment resulted in a 51% reduction in CSF-HS levels and a 63% reduction in CSF HS exposure over time. Cognitive function showed stability or gains in 16 of 17 patients, with a significant correlation between CSF HS exposure and cognitive function. The most frequently reported treatment-related adverse events were mild or moderate. The study enrolled 28 patients across 3 dose Cohorts at 5 sites in 3 countries, with the high dose Cohort 3 (3x1013 vg/kg) showing promising results.
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Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that the European Medicines Agency (EMA) has granted Priority Medicine (PRIME) designation to GTX-102 for the treatment of Angelman syndrome. This designation was granted based on compelling early clinical data from the extension cohorts in the Phase 1/2 study of GTX-102, showing improvements in neurodevelopmental domains. The EMA's recognition of the potential for GTX-102 to address the critical need for new treatments for Angelman syndrome in the EU is a significant development for Ultragenyx Pharmaceutical Inc.
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Ultragenyx Pharmaceutical Inc. (RARE) announced the completion of Stage 1 enrollment in the Cyprus2+ program, moving one step closer to beginning Stage 2, the pivotal, randomized placebo-controlled stage of the study. Safety and initial efficacy data from Stage 1 are expected in the first half of 2024, with dose selection and initiation of Stage 2 to follow in the second half of 2024. The investigational AAV9 gene therapy, UX701, aims to deliver stable expression of the ATP7B copper transporter to normalize copper metabolism in patients with Wilson disease. Data from the first dose cohort showed well-tolerated treatment with no unexpected adverse events, and four out of five patients started tapering standard-of-care treatment. The study design includes three stages, with Stage 1 evaluating up to three dose levels of UX701 over 52 weeks, and Stage 2 involving a new cohort of patients randomized to receive the selected dose or placebo.
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Ultragenyx Pharmaceutical Inc. grants 12,130 restricted stock units to new non-executive officers under the Employment Inducement Plan. The awards were approved by the compensation committee and vest over four years. The grant is an inducement material to new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4).
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Ultragenyx Pharmaceutical Inc. (RARE) reported preliminary 2023 total revenue of $430-435 million, with Crysvita revenue of $325-330 million and Dojolvi revenue of $70-71 million. The 2024 expected revenue guidance is between $500-530 million, with Crysvita revenue of $375-400 million and Dojolvi revenue of $75-80 million. The year-end 2023 cash balance was approximately $776 million, and the 2024 guidance for net cash used in operations is expected to be less than $400 million. Ultragenyx expects significant momentum in 2024, with clinical catalysts across multiple value-driving programs, meaningful revenue growth from commercial products, and financial discipline. The company also provided updates on clinical milestones for various gene therapy programs.
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Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that the National Institute for Health and Care Excellence (NICE) has recommended Evkeeza® (evinacumab) to NHS England. The drug is the first angiopoietin-like 3 (ANGPTL3) inhibitor treatment indicated for adults and adolescent patients aged 12 years and older with homozygous familial hypercholesterolemia (HoFH). NICE acknowledged the clinical and economic benefits of Evkeeza, highlighting its commitment to making the drug broadly available to the HoFH community in the U.S., England and Wales, Canada, Italy, and Germany.
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Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) completes patient enrollment in Phase 1/2 clinical trial of GTX-102 for the treatment of pediatric patients with Angelman syndrome. The company remains on track to report results in the first half of 2024 from at least 20 expansion cohort patients on therapy for a minimum of 6 months.
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Ultragenyx Pharmaceutical Inc.
Nasdaq:RARE
RARE Rankings
RARE Stock Data
3.59B
72.17M
4.11%
101.15%
4.76%
Pharmaceutical Preparation Manufacturing
Manufacturing
United States of America
NOVATO
About RARE
ultragenyx is a clinical-stage biotechnology company committed to bringing to market novel products for the treatment of rare and ultra-rare diseases, with an initial focus on serious, debilitating metabolic genetic diseases. founded in 2010, the company has rapidly built a diverse portfolio of product candidates with the potential to address diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are no approved therapies. our company is led by an experienced management team in rare disease therapeutics. recognizing that our primary responsibility is to our patients, we are working with advocacy groups to provide support and outreach to individuals and families affected by these disorders and engage them in the clinical testing process. we are also working with regulatory agencies to design and conduct high quality clinical studies that meet the requirements for approval. we are creating an improved model for successful rare disease d