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Ultragenyx Pharmaceutical Inc. - RARE STOCK NEWS

Welcome to our dedicated page for Ultragenyx Pharmaceutical news (Ticker: RARE), a resource for investors and traders seeking the latest updates and insights on Ultragenyx Pharmaceutical stock.

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), founded in 2010, is a clinical-stage biotechnology company dedicated to developing and commercializing innovative therapies for the treatment of serious rare and ultra-rare genetic diseases. The company’s core focus is on debilitating metabolic genetic diseases, for which there are high unmet medical needs, and the biology for treatment is well-understood but lacks approved therapies. Ultragenyx's management team brings extensive experience in rare disease therapeutics.

The company’s diverse product portfolio includes approved medicines like Crysvita, Dojolvi, and Mepsevii. Crysvita is designed for the treatment of X-linked hypophosphatemia (XLH), a rare genetic bone disease, and has shown significant growth with revenue reaching $328 million in 2023. Dojolvi targets long-chain fatty acid oxidation disorders and saw a 27% revenue increase in 2023. Mepsevii treats Mucopolysaccharidosis VII, another severe genetic disorder.

Recent Achievements and Financial Performance

In 2023, Ultragenyx reported total revenues of $434 million, a 20% increase from the previous year. The company has a robust pipeline with several candidates in late-stage clinical trials. For instance, the Phase 3 Orbit study for UX143 (setrusumab) in Osteogenesis Imperfecta (OI) completed enrollment and showed promising interim results with a 67% reduction in fracture rates.

Ongoing Projects

  • GTX-102: An antisense oligonucleotide for Angelman syndrome showed significant improvements in clinical trials, with plans for a Phase 3 study in 2024.
  • UX701: A gene therapy for Wilson disease, with data expected in mid-2024.
  • DTX401: A gene therapy for Glycogen Storage Disease Type Ia (GSDIa), achieved its primary endpoint in a Phase 3 study.
  • Setrusumab (UX143): Demonstrated significant fracture rate reduction and bone mineral density improvement in OI patients.

Partnerships and Collaborations

Ultragenyx collaborates with various regulatory bodies and advocacy groups to support patients and ensure high-quality clinical trials. The company’s partnership with Mereo BioPharma on setrusumab exemplifies its collaborative approach to drug development.

Corporate Responsibility

Ultragenyx's 2023 Corporate Responsibility Report highlights its commitment to innovation, patient support, diversity, and environmental sustainability. The company has been recognized with several awards, including the Top Places to Work by the Boston Globe and Healthcare’s Best Companies to Work by U.S. News & World Report.

For more information, visit www.ultragenyx.com.

News

Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)

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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 Reports Fourth Quarter and Full Year 2023 Financial Results and Corporate Update

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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 to Host Conference Call for Fourth Quarter and Full-Year 2023 Financial Results and Corporate Update

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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 Announces Data Demonstrating Treatment with UX111 Results in Significant Reduction in Heparan Sulfate Exposure in Cerebrospinal Fluid Correlated with Improved Long-term Cognitive Function in Patients with Sanfilippo Syndrome Type A (MPS IIIA)

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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 Receives PRIME Designation from European Medicines Agency (EMA) for GTX-102 for the Treatment of Angelman Syndrome

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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 Announces Completion of Dosing Across Stage 1 Cohorts in Pivotal Phase 1/2/3 Cyprus2+ Study Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease

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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 Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)

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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 Reports Preliminary 2023 Revenue; Guidance for 2024 Revenue and Cash Usage; Pipeline Updates and 2024 Milestones

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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 Receives Positive Recommendation from NICE in the U.K. for Evkeeza® ▼ (evinacumab) for Adolescents and Adults Aged 12 Years and Older with Homozygous Familial Hypercholesterolemia (HoFH)

Rhea-AI Summary
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 Announces Completion of Enrollment in Global Phase 1/2 Trial of GTX-102 in Pediatric Patients with Angelman Syndrome

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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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FAQ

What is the current stock price of Ultragenyx Pharmaceutical (RARE)?

The current stock price of Ultragenyx Pharmaceutical (RARE) is $44.22 as of December 20, 2024.

What is the market cap of Ultragenyx Pharmaceutical (RARE)?

The market cap of Ultragenyx Pharmaceutical (RARE) is approximately 4.0B.

What is the primary focus of Ultragenyx Pharmaceutical Inc.?

Ultragenyx focuses on developing therapies for serious rare and ultra-rare genetic diseases, especially debilitating metabolic genetic diseases.

What are some key products of Ultragenyx?

Key products include Crysvita for X-linked hypophosphatemia (XLH), Dojolvi for long-chain fatty acid oxidation disorders, and Mepsevii for Mucopolysaccharidosis VII.

What were the financial highlights for Ultragenyx in 2023?

Ultragenyx reported $434 million in total revenues in 2023, marking a 20% growth compared to the previous year.

What recent achievements has Ultragenyx made in clinical trials?

Recent achievements include the Phase 3 Orbit study for UX143 showing significant reduction in fracture rates in Osteogenesis Imperfecta patients.

Who leads Ultragenyx Pharmaceutical Inc.?

Ultragenyx is led by a management team experienced in rare disease therapeutics, with Emil D. Kakkis, M.D., Ph.D., serving as the CEO and President.

What partnerships does Ultragenyx have?

Ultragenyx collaborates with various regulatory bodies and advocacy groups and has a notable partnership with Mereo BioPharma for the development of setrusumab.

What is Ultragenyx's commitment to corporate responsibility?

Ultragenyx is committed to innovation, patient support, diversity, and environmental sustainability, as highlighted in its 2023 Corporate Responsibility Report.

How does Ultragenyx engage with the rare disease community?

Ultragenyx works closely with advocacy groups to support and engage affected individuals and families in the clinical testing process.

What are the future plans for GTX-102?

GTX-102, an antisense oligonucleotide for Angelman syndrome, showed significant improvements in clinical trials, with plans for a Phase 3 study in 2024.

Where can I find more information about Ultragenyx?

You can find more information on Ultragenyx's official website at www.ultragenyx.com.

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Ultragenyx Pharmaceutical Inc.

Nasdaq:RARE

RARE Rankings

#1422 Ranked by Market Cap
N/A Ranked by Dividends

RARE Stock Data

4.01B
88.07M
3.62%
97.49%
3.62%
Biotechnology
Pharmaceutical Preparations
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United States of America
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