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.

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The recent acquisition of rare dug heavyweight Alexion Pharmaceuticals signals a willingness of big pharma to enter the field. Typically, big pharma was interested in larger patient populations however the lack of commercial ready products has broaden their focus.

Ultragenyx: Upside But Not Without Risk

Ultragenyx (RARE) and Mereo BioPharma ink an agreement for setrusumab, a monoclonal antibody in clinical development for osteogenesis imperfecta.

Ultragenyx (RARE), Mereo Ink Deal for Bone Disease Candidate

Clinical-stage monoclonal antibody in development for rare genetic bone disease that builds on Ultragenyx's existing bone franchise

Ultragenyx and Mereo BioPharma Announce Collaboration and License Agreement for Setrusumab in Osteogenesis Imperfecta

Ultragenyx (RARE) gets Orphan Drug Designation from the FDA for UX701 for the treatment of Wilson disease.

Ultragenyx (RARE) Gets Orphan Drug Tag for Wilson Disease Drug

NOVATO, Calif., Dec. 09, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare genetic diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to UX701 for the treatment of Wilson disease, a larger rare metabolic disease that affects more than 50,000 people in the developed world.

Ultragenyx Announces Orphan Drug Designation for UX701 for the Treatment of Wilson Disease

Results presented at Foundation for Angelman Syndrome Therapeutics (FAST) Global Summit

GeneTx and Ultragenyx Announce Presentation of Phase 1/2 Data on Investigational GTX-102 in Patients with Angelman Syndrome

State-of-the-art facility to be located in Bedford, Massachusetts

Ultragenyx Announces Plans to Build Large-scale Gene Therapy Manufacturing Facility to Support Pipeline of Therapies for Rare Diseases

NOVATO, Calif., Nov. 02, 2020 (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 the closing of its previously announced underwritten public offering of 5,111,110 shares of its common stock, including the exercise in full by the underwriters of their option to purchase an additional 666,666 shares, at a price to the public of $90.00 per share. The estimated net proceeds to the company, after deducting underwriting discounts and commissions and other offering expenses payable by the company, were approximately $435.4 million.

Ultragenyx Announces Closing of Public Offering of Common Stock and Full Exercise of Underwriters' Option to Purchase Additional Shares

NOVATO, Calif., Oct. 28, 2020 (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 the pricing of its underwritten public offering of 4,444,444 shares of its common stock at a price to the public of $90.00 per share resulting in gross proceeds of $400 million, before underwriting discounts. In addition, the company has granted the underwriters of the offering an option for a period of 30 days to purchase up to an additional 666,666 shares of the company's common stock at the public offering price, less the underwriting discount.

Ultragenyx Announces Pricing of Public Offering of Common Stock

Ultragenyx (RARE) reports a narrower loss year over year and sales beat estimates in the third quarter of 2020.

Ultragenyx (RARE) Loss Narrows Y/Y, Revenues Beat Estimates

Ultragenyx Pharmaceutical Inc. (RARE) CEO Emil Kakkis on Q3 2020 Results - Earnings Call Transcript

Ultragenyx Pharmaceutical posted third-quarter results late Tuesday that beat views on the top and bottom lines after RARE stock hit a nearly five-year high, revenue more than triples. The post Ultragenyx Pharmaceutical Sales More Than Triple, Pares Losses appeared first on Investor's Business Daily.

Ultragenyx Pharmaceutical Sales More Than Triple, Pares Losses

Shares of Ultragenyx Pharmaceutical (NASDAQ:RARE) were flat in after-market trading after the company reported Q3 results. Quarterly Results Earnings per share rose 42.35% over the past year to ($1.13), which beat the estimate of ($1.27).

Recap: Ultragenyx Pharmaceutical Q3 Earnings

Third quarter 2020 total revenue of $ 81.5 million and Crysvita 1 revenue of $37.3 million

Ultragenyx Reports Third Quarter 2020 Financial Results and Corporate Update

Efficacy in multiple functional measures observed in all five patients treated

GeneTx and Ultragenyx Announce Positive Interim Phase 1/2 Data on Investigational GTX-102 Demonstrating Improvement in Patients with Angelman Syndrome

- Collaboration combines Solid's differentiated microdystrophin construct and Ultragenyx's HeL a PCL manufacturing platform for use with AAV8 and variants -

Ultragenyx and Solid Biosciences Announce Strategic Collaboration to Develop and Commercialize New Gene Therapies for Duchenne Muscular Dystrophy

NOVATO, Calif. , Oct. 21, 2020 (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 it will host a conference call on Tuesday, October 27, 2020 at 5pm ET to discuss third quarter 2020 financial results and provide a corporate update.

Ultragenyx to Host Conference Call for Third Quarter 2020 Financial Results and Corporate Update

Ms. Dier will start in new role in November 2020 Ms. Dier will start in new role in November 2020

Ultragenyx Appoints Mardi C. Dier as Chief Financial Officer

NOVATO, Calif., Sept.  01, 2020  (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 it has appointed Ernie Meyer as Executive Vice President and Chief Human Resources Officer, effective September 1, 2020. In this newly created role, Mr. Meyer will lead all aspects of the Global Human Resources and Facilities operations. He will report to Emil D. Kakkis, M.D., Ph.D., Ultragenyx’s Chief Executive Officer, and will serve on the Executive Leadership Team.  

Ultragenyx Expands Leadership Team and Appoints Ernie Meyer as Chief Human Resources Officer

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

Ultragenyx (RARE) Up 10.4% Since Last Earnings Report: Can It Continue?

Ultragenyx (RARE) reports a narrower-than-expected loss in the second quarter of 2020 and beats sales estimates.

Ultragenyx (RARE) Loss Narrows Y/Y & Revenues Beat Estimates

Ultragenyx Pharmaceutical Inc.'s (RARE) CEO Emil Kakkis on Q2 2020 Results - Earnings Call Transcript

Two U.S. FDA approvals in second quarter 2020

Ultragenyx Reports Second Quarter 2020 Financial Results and Corporate Update

NOVATO, Calif., July  24, 2020  (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 it will host a conference call on Thursday, July 30, 2020 at 5pm ET to discuss second quarter 2020 financial results and provide a corporate update.

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