Ultragenyx Pharmaceutical

Ultragenyx Pharmaceutical, Inc. is a biopharmaceutical company, engaged in the identification, acquisition, development and commercialization of novel products for the treatment of serious rare and ultra-rare genetic diseases. Its products include Mepsevii and Crysvita. Mepsevii is an intravenous enzyme replacement therapy, which is used for the treatment of Mucopolysaccharidosis VII. Crysvita is an antibody administered via subcutaneous injection used for the treatment of XLH. The company was founded by Emil D. Kakkis on April 22, 2010, and is headquartered in Novato, CA.

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Ultragenyx Pharmaceutical reports strong revenue growth and clinical advancements

Ultragenyx Pharmaceutical's strong commercial performance in 2022, strategic investments, and positive clinical results position the company for future growth.
Challenges such as high operating expenses, regulatory uncertainties, and seasonal revenue effects require careful management and strategic planning.
Opportunities lie in clinical advancements, FDA discussions, and upcoming product launches, offering avenues for market expansion and revenue growth.
Overall, Ultragenyx Pharmaceutical's focus on profitability, product development, and market resilience sets a positive trajectory for future success.

Ultragenyx reports robust Q1 revenue, advances in rare disease treatments

Ultragenyx's Q1 2023 performance showed strong revenue figures and ongoing progress in key clinical programs.
The company faces challenges in bone deformation measurement and program prioritization but has opportunities in gene therapy studies and treatment response predictions.
Emphasizing efficient commercialization and safety improvements in dosing, Ultragenyx aims to address rare diseases effectively.
Revenue guidance for Crysvita and Dojolvi indicates a positive outlook for 2023, with a focus on accelerating approval pathways for ultra-rare genetic diseases.

Ultragenyx reports revenue growth amidst challenges and opportunities

Ultragenyx's Q2 2023 performance showed revenue growth but with high expenses
Strengths include positive clinical data and progress in key programs
Challenges exist in patient responsiveness and revenue clarification
Opportunities lie in accelerated approvals and manufacturing flexibility

Ultragenyx reports strong Q3 revenue, positive clinical data outlook

Ultragenyx Pharmaceutical showcased strong commercial performance and positive clinical data across key programs, positioning the company for future growth.
While facing financial challenges and regulatory hurdles, Ultragenyx's patient-centric approach and strategic focus on upcoming catalysts offer opportunities for success.
The company's emphasis on chronic dosing, patient support, and market access strategies underscore a commitment to long-term therapeutic impact and revenue growth.
Overall, Ultragenyx's Q3 earnings call highlights a balance of achievements, challenges, and opportunities in its pursuit of advancing innovative therapies.

Ultragenyx Pharmaceutical reports robust 2023 performance and optimistic 2024 outlook

Ultragenyx Pharmaceutical's strong financial performance and positive clinical updates position the company well for growth in 2024.
Challenges around trial designs, regulatory pathways, and endpoint selection require careful navigation for successful program advancement.
Opportunities lie in upcoming clinical data readouts, potential accelerated approvals, and advancements in patient outcomes and quality of life.

Ultragenyx Pharmaceutical reports strong Q1 performance and growth opportunities

Ultragenyx Pharmaceutical's Q1 2024 earnings call highlighted positive clinical program data, strong revenue from key products, and a focus on upcoming milestones. Despite a net loss, the company maintains a solid financial position and is poised for growth with ongoing studies and potential FDA approvals.

Ultragenyx reports strong Q2 revenue, raises guidance amid clinical advancements

Ultragenyx Pharmaceutical (RARE) demonstrated strong revenue performance and promising clinical advancements in Q2 2024.
Despite challenges in market positioning and financial performance, the company's raised revenue guidance and robust cash position signal growth potential.
Opportunities lie in expanding market reach, advancing clinical programs, and navigating regulatory processes to drive future success.

Ultragenyx Pharmaceutical reports robust Q3 growth and prepares for key launches

Ultragenyx Pharmaceutical's Q3 2024 performance reflects strong revenue growth and progress in late-stage programs, positioning the company well for future success.
While facing financial challenges and regulatory hurdles, the company's leadership team and innovative pipeline demonstrate resilience and potential.
Analysts' inquiries and upcoming milestones present opportunities for growth and improvement in operational efficiency and market competitiveness.
Overall, Ultragenyx Pharmaceutical's strategic focus on commercialization, clinical advancements, and upcoming launches sets a positive trajectory for the company.

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Ultragenyx Pharmaceutical, Inc. engages in the identification, acquisition, development, and commercialization of novel products for the treatment of genetic diseases. Its products include Crysvita, Mepsevii, Dojolvi, and Evkeeza. The company was founded by Emil D. Kakkis in April 2010, and is headquartered in Novato, CA.

Preliminary 2021 Crysvita revenue in Ultragenyx territories of $191 million to $193 million and Dojolvi revenue of $38 million to $40 million

Ultragenyx Reports Preliminary 2021 Revenue and 2022 Revenue Guidance for Crysvita® in Ultragenyx Territories* And Dojolvi® Globally; Provides Pipeline Updates and 2022 Milestones

NOVATO, Calif., Jan. 06, 2022 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will present at the H.C. Wainwright BioConnect Conference on Thursday, January 13, 2022 at 7:00 AM ET.

Ultragenyx to Present at H.C. Wainwright BioConnect Healthcare Conference

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NOVATO, Calif., Jan. 03, 2022 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will present at the 40th Annual JP Morgan Healthcare Conference on Monday, January 10, 2022 at 3:45 PM ET.

Ultragenyx to Present at 40th Annual JP Morgan Healthcare Conference

Ultragenyx (RARE) reported earnings 30 days ago. What's next for the stock?

Why Is Ultragenyx (RARE) Down 11.4% Since Last Earnings Report?

Ultragenyx (RARE) doses the first patient in a phase I/II study evaluating its investigational mRNA candidate, UX053, for treating glycogen storage disease type III.

Ultragenyx (RARE) Begins Dosing in Phase I/II Study of UX053

Overall GTX-102 program update on track for the end of 2021 Overall GTX-102 program update on track for the end of 2021

GeneTx and Ultragenyx Announce Presentations at Upcoming 2021 FAST Global Summit & Gala

NOVATO, Calif., Dec. 01, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that the first patient has been dosed in its Phase 1/2 study of UX053, an investigational messenger RNA (mRNA) therapy in development for the treatment of Glycogen Storage Disease Type III (GSDIII).

Ultragenyx Announces First Patient Dosed in Phase 1/2 Clinical Study of UX053, an mRNA Therapy for the Treatment of Glycogen Storage Disease Type III

NOVATO, Calif., Nov. 29, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will present at the Evercore ISI 4th Annual HealthconX Conference on December 1, 2021 at 12:10 PM ET.

Ultragenyx to Present at Evercore 4th Annual HealthconX Conference

Ultragenyx (RARE) reports narrower-than-expected loss in the third quarter of 2021 while revenues surpass estimates.

Ultragenyx's (RARE) Q3 Earnings & Revenues Beat Estimates

Ultragenyx Pharmaceutical Inc. (RARE) CEO Emil Kakkis on Q3 2021 Results - Earnings Call Transcript

Third quarter 2021 total revenue of $81.6 million including Crysvita 1 revenue to Ultragenyx of $50.3 million

Ultragenyx Reports Third Quarter 2021 Financial Results and Corporate Update

NOVATO, Calif., Oct. 26, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel therapies for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call on Tuesday, November 2, 2021 at 5pm ET to discuss its financial results and corporate update for the quarter ended September 30, 2021.

Ultragenyx to Host Conference Call for Third Quarter 2021 Financial Results and Corporate Update

NOVATO, Calif., Oct. 25, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will present at the Jefferies Gene Therapy/Editing Summit on October 28, 2021 at 3:30 PM ET.

Ultragenyx to Present at Jefferies Gene Therapy/Editing Summit

Ultragenyx (RARE) and GeneTx Biotherapeutics dose the first patient in a phase I/II study on GTX-102 for treating patients with Angelman syndrome in Canada.

Ultragenyx (RARE) Begins Angelman Syndrome Study in Canada

Ultragenyx (RARE) and GeneTx Biotherapeutics to start dosing in the phase I/II study on GTX-102 for treating pediatric patients with Angelman syndrome after the FDA removes its clinical hold on the same.

Ultragenyx (RARE) Reports FDA Removal of Clinical Hold on Study

NOVATO, Calif., Sept. 27, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that new data highlighting Crysvita® (burosumab-twza) for the treatment of X-linked hypophosphatemia (XLH) and UX143 (setrusumab) for the treatment of osteogenesis imperfecta (OI) will be presented at the American Society for Bone and Mineral Research (ASBMR) 2021 Annual Meeting. The meeting will take place October 1-4 in-person in San Diego with programming also virtually accessible in real-time.

Ultragenyx Announces Crysvita® (burosumab) and UX143 (setrusumab) Data Presentations at Upcoming American Society for Bone and Mineral Research (ASBMR) 2021 Annual Meeting

The FDA has removed the clinical hold on GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc's (NASDAQ: RARE) GTX-102 trial for Angelman syndrome. The companies can begin dosing naïve patients in the Phase 1/2 study of GTX-102 in pediatric patients.

Ultragenyx Resumes Dosing Antisense Trial In Rare Neurogenetic Disorder

TOKYO--(BUSINESS WIRE)--New data highlighting the sustained benefits of treatment with CRYSVITA® (burosumab) in adults with XLH, a rare genetic metabolic bone disease

Long-Term CRYSVITA® ▼ (burosumab) Treatment Reduces the Burden of Disease in Adults With X-Linked Hypophosphataemia (XLH), a Rare Genetic Metabolic Bone Disease

NOVATO, Calif., Sept. 09, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Mepsevii® (vestronidase alfa) has been approved for reimbursement by the Agenzia Italiana del Farmaco (Italian Medicines Agency – AIFA) for the treatment of pediatric and adult patients with Mucopolysaccharidosis VII (MPS VII; Sly syndrome).

MEDIA ADVISORY: Ultragenyx Announces Mepsevii® (vestronidase alfa) Receives Reimbursement Approval for Treatment of Mucopolysaccharidosis VII in Italy

NOVATO, Calif., Sept. 07, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President will present at the Morgan Stanley 19th Annual Healthcare Conference on September 14, 2021 at 4:15 PM ET.

Ultragenyx to Present at Morgan Stanley Healthcare Conference

OTTAWA, Sept. 07, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced that Crysvita™ (Burosumab Injection) has been approved by Health Canada for the treatment of Tumour Induced Osteomalacia (TIO) in adults. Crysvita is also approved for the treatment of X-Linked Hypophosphatemia.

MEDIA ADVISORY: Ultragenyx Canada Announces Health Canada Approval of a Second Indication for Crysvita™ (Burosumab Injection) for the Treatment of Tumour Induced Osteomalacia in Adults

NOVATO, Calif., Sept. 01, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President will participate in a panel titled Finding a Needle in a Haystack - Drug Development Strategies for Rare Disease on Thursday, September 9, 2021 at Citi's 16th Annual BioPharma Healthcare Conference at 4:10 PM ET.

Ultragenyx to Participate in Panel at Citi's 16th Annual BioPharma Healthcare Conference

Ultragenyx (RARE) Up 15% Since Last Earnings Report: Can It Continue?

Dojolvi is the only approved therapy for this rare, life-threatening metabolic disorder Dojolvi is the only approved therapy for this rare, life-threatening metabolic disorder

Ultragenyx Announces Approval of Dojolvi® (triheptanoin) in Brazil for the Treatment of Long-chain Fatty Acid Oxidation Disorders in Adults and Children

News for Ultragenyx Pharmaceutical Stock (RARE)