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.

Ultragenyx (RARE) reports a wider-than-expected loss and beats sales estimates in the fourth quarter of 2019.

Ultragenyx (RARE) Reports Wider-Than-Expected Loss in Q4

Ultragenyx Pharmaceutical Inc. (RARE) CEO Emil Kakkis on Q4 2019 Results - Earnings Call Transcript

Ultragenyx Pharmaceutical: Gearing Up For FDA Approvals And Data Readouts

SARASOTA, Fla. and NOVATO, Calif., Jan.  15, 2020  (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, today announced that GeneTx’s Investigational New Drug (IND) Application for GTX-102, an experimental antisense oligonucleotide being evaluated for the treatment of Angelman syndrome (AS), filed with the U.S. Food and Drug Administration (FDA) is now active. Enrollment in the Phase 1/2 study is expected to begin in the first half of 2020.

GeneTx and Ultragenyx Announce Investigational New Drug (IND) Application Active for GTX-102 in Patients with Angelman Syndrome

Ultragenyx (RARE) provides preliminary 2019 revenue numbers and 2020 outlook for Crysvita, along with other pipeline updates.

Ultragenyx Provides Preliminary Crysvita 2019 Revenues

Shares of Ultragenyx (RARE) rise 23.4% after releasing positive top-line data from the phase I/II study of its gene therapy candidate DTX301 for the treatment of OTC deficiency.

Ultragenyx's Gene Therapy Positive in Genetic Disorder Study

Preliminary 2019 total revenue is approximately $102 million to $104 million

Ultragenyx Reports Preliminary 2019 Revenue and Provides 2020 Crysvita Revenue Guidance

NOVATO, Calif. and TOKYO, Jan.  13, 2020  (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, and Kyowa Kirin Co., Ltd., (Kyowa Kirin, TYO: 4151) today announced that they submitted a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) on December 18, 2019, for Crysvita® (burosumab) for the treatment of FGF23-related hypophosphatemia associated with phosphaturic mesenchymal tumors (tumor-induced osteomalacia; TIO) that cannot be curatively resected or localized. The companies expect to hear back from FDA on submission acceptance and review designation in February 2020.

Ultragenyx and Kyowa Kirin Announce Submission of Supplemental Biologics License Application to U.S. FDA for Crysvita® (burosumab) for Tumor-Induced Osteomalacia (TIO)

A promising clinical trial update has investors increasingly optimistic about the company's pipeline.

Why Ultragenyx Pharmaceutical Stock Surged 23.4% Today

More uniform response in Cohort 3 with two confirmed responders and one potential responder

Ultragenyx Announces Positive Topline Cohort 3 Results and Improved Longer-Term Cohort 2 Results from Phase 1/2 Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency

NOVATO, Calif. and NEW YORK, Dec.  18, 2019  (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, and Royalty Pharma today announced that Ultragenyx has sold to Royalty Pharma for $320 million a royalty right due to Ultragenyx from Kyowa Kirin Co., Ltd for the net sales of Crysvita® (burosumab) in the European Union (EU), the United Kingdom (UK), and Switzerland. Crysvita has received conditional marketing authorization in Europe for the treatment of X-linked hypophosphatemia (XLH) with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons, and an application for the expanded use in adults with XLH is currently under review by the European Medicines Agency.  

Ultragenyx Announces Sale of Future European Royalties on Crysvita® (burosumab) for $320 Million to Royalty Pharma

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

Ultragenyx (RARE) Up 13.6% Since Last Earnings Report: Can It Continue?

NOVATO, Calif., Nov.  26, 2019  (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Shalini Sharp, the company’s Chief Financial Officer, will present at the Piper Jaffray 31st Annual Healthcare Conference on Wednesday, December 4, 2019 at 2:00 PM ET in New York, NY.

Ultragenyx to Present at Piper Jaffray 31st Annual Healthcare Conference

Ultragenyx Pharmaceuticals (NASDAQ: RARE), a biopharmaceutical company focused on serious rare and ultra-rare genetic diseases, has seen its stock price decline by close to 40% over the last 3 months. The decline could be due to the fact that the company posted wider than expected losses.

Why Ultragenyx Pharmaceutical Stock Has Dropped 40% In The Last Quarter

Ultragenyx (RARE) reports wider-than-expected loss and misses sales estimates in the third quarter of 2019.

Ultragenyx (RARE) Reports Wider-Than-Expected Loss in Q3

Ultragenyx Pharmaceutical Inc. (RARE) CEO Emil Kakkis on Q3 2019 Results - Earnings Call Transcript

Ultragenyx (RARE) delivered earnings and revenue surprises of -14.62% and -9.80%, respectively, for the quarter ended September 2019. Do the numbers hold clues to what lies ahead for the stock?

Ultragenyx (RARE) Reports Q3 Loss, Lags Revenue Estimates

Ultragenyx (RARE) announces FDA's acceptance of the NDA for UX007 for the treatment of LC-FAOD.

Ultragenyx's (RARE) NDA for LC-FAOD Drug Accepted by FDA

NOVATO, Calif., Oct.  14, 2019  (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the company’s New Drug Application (NDA) for UX007 (triheptanoin) for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD), a group of genetic disorders in which the body is unable to convert long-chain fatty acids into energy. The FDA has assigned a standard review designation with a Prescription Drug User Fee Act (PDUFA) target date of July 31, 2020.

Ultragenyx Announces FDA Accepts New Drug Application for UX007 (triheptanoin) for Treatment of Long-chain Fatty Acid Oxidation Disorders

Ultragenyx (RARE) and partner Kyowa Kirin get FDA approval for the label expansion of Crysvita in pediatric patients with X-linked hypophosphatemia.

Ultragenyx Gets FDA Approval for Label Expansion of Crysvita

Expanded Label Includes Additional Clinical Data Highlighting Superiority Over Conventional Therapy for Pediatric Patients and Extends Current U.S. Indication to Patients Aged Six Months and Older

Ultragenyx and Kyowa Kirin Announce U.S. FDA Approves Label Update for Crysvita® (burosumab) for the Treatment of X-Linked Hypophosphatemia (XLH)

Patients diagnosed with rare, ultra-rare genetic or non-genetic diseases are an underserved therapeutics patient population.

Ultragenyx Pharmaceutical And Ultra-Rare Diseases: A Voice For The Clinically Underserved

Ultragenyx Pharmaceuticals And Ultra-Rare Diseases: A Voice For The Clinically Underserved

Ultragenyx (RARE) and partner Kyowa Kirin plan to submit an sBLA to the FDA for the label expansion of Crysvita.

News for Ultragenyx Pharmaceutical Stock (RARE)