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.

NOVATO, Calif., Jan. 04, 2023 (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 41st Annual J.P. Morgan Healthcare Conference on Tuesday, January 10, 2023, at 12:45 PM ET.

Ultragenyx to Present at the 41st Annual J.P. Morgan Healthcare Conference

Ultragenyx's (RARE) marketed drugs, which are approved for various indications, are driving the top line. The company also has a strong pipeline.

Ultragenyx's (RARE) Portfolio of Marketed Drugs Aids Growth

NOVATO, Calif., Nov. 22, 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 Piper Sandler Annual Healthcare Conference on Tuesday, November 29, 2022, at 10:00 AM ET in New York.

Ultragenyx to Present at Piper Sandler Healthcare Conference

Ultragenyx Pharmaceutical Inc. has a number of approved products in a variety of rare diseases, and a solid pipeline. They are making over a quarter billion dollars every year (2021-22 figures).

Ultragenyx: Great Medicine, Good Execution, Stagnant Stock

NOVATO, Calif., Nov. 10, 2022 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that Mardi Dier, Chief Financial Officer (CFO) and Executive Vice President, is leaving the company, effective November 15, 2022, to assume the dual role of CFO and Chief Business Development Officer at privately held ACELYRIN, INC. Ted Huizenga, Chief Accounting Officer, and Aaron Olsen, Senior Vice President of Corporate Strategy and Finance, will lead ongoing finance activities during the search for a successor.

Ultragenyx Announces Departure of Chief Financial Officer

NOVATO, Calif., Nov. 09, 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 following upcoming investor conferences:

Ultragenyx to Present at Upcoming Healthcare Conferences

Ultragenyx (RARE) reports a wider-than-expected loss in the third quarter. Revenues, though up year over year, miss estimates.

Ultragenyx's (RARE) Q3 Earnings Miss Estimates, Revenues Up Y/Y

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE ) Q3 2022 Earnings Conference Call November 2, 2022 5:00 PM ET Company Participants Joshua Higa - Head of Investor Relations Emil Kakkis - Founder, President and Chief Executive Officer Erik Harris - Executive Vice President and Chief Commercial Officer Mardi Dier - Executive Vice President and Chief Financial Officer Camille Bedrosian - Executive Vice President and Chief Medical Officer Conference Call Participants Gena Wang - Barclays Joori Park - SVB Securities LLC Christopher Raymond - Piper Sandler Tazeen Ahmad - Bank of America Merrill Lynch Dae Gon Ha - Stifel Maurice Raycroft - Jefferies LLC Yigal Nochomovitz - Citigroup Inc. Joon Lee - Truist Securities Yaron Werber - Cowen and Company, LLC Elizabeth Webster - Goldman Sachs Group, Inc. Joel Beatty - Robert W. Baird & Company, Inc. Liisa Bayko - Evercore ISI Operator Thank you for standing by.

Ultragenyx Pharmaceutical Inc. (RARE) Q3 2022 Earnings Call Transcript

Ultragenyx (RARE) delivered earnings and revenue surprises of -6.11% and 8.16%, respectively, for the quarter ended September 2022. Do the numbers hold clues to what lies ahead for the stock?

Ultragenyx (RARE) Reports Q3 Loss, Misses Revenue Estimates

NOVATO, Calif., Oct. 27, 2022 (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 Wednesday, November 2, 2022, at 5:00pm ET to discuss its financial results and corporate update for the quarter ended September 30, 2022.

Ultragenyx to Host Conference Call for Third Quarter 2022 Financial Results and Corporate Update

Ultragenyx (RARE) doesn't possess the right combination of the two key ingredients for a likely earnings beat in its upcoming report. Get prepared with the key expectations.

Earnings Preview: Ultragenyx (RARE) Q3 Earnings Expected to Decline

NOVATO, Calif. and MOUNTAIN VIEW, Calif. and LONDON, Sept. 09, 2022 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), (“Ultragenyx”), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases and Mereo BioPharma Group plc (NASDAQ: MREO), (“Mereo”), a clinical-stage biopharmaceutical company focused on rare diseases and oncology, today announced that additional data from Mereo's Phase 2b ASTEROID study related to setrusumab (UX143) and information on the Phase 2/3 Orbit study will be presented at the American Society for Bone and Mineral Research 2022 Annual Meeting (ASBMR) being held September 9-12, 2022, in Austin, Texas. Setrusumab is a monoclonal antibody in development for the treatment of Osteogenesis Imperfecta (OI), a rare disease affecting approximately 60,000 individuals in the US and Europe.

Ultragenyx and Mereo BioPharma to Present Setrusumab Data Update at ASBMR

NOVATO, Calif., Sept. 01, 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 Eric Crombez, Chief Medical Officer, Gene Therapy, will participate in a Rare Disease Panel at Citi's 17th Annual BioPharma Conference on Wednesday, September 7, 2022, at 11:20 AM ET.

Ultragenyx to Participate at Citi BioPharma Conference

TOKYO, Japan--(BUSINESS WIRE)--Kyowa Kirin Co., Ltd. (TSE: 4151, Kyowa Kirin) today announced that the European Commission (EC) approved CRYSVITA® (burosumab) for the treatment of FGF23-related hypophosphataemia in Tumour-Induced Osteomalacia (TIO) associated with phosphaturic mesenchymal tumours (PMTs) that cannot be curatively resected or localised in children and adolescents aged 1 to 17 years and in adults.1 CRYSVITA is also already licensed in the EU for use in the rare disease X-Linked Hy

Kyowa Kirin Receives European Commission Approval for Use of CRYSVITA®▼(burosumab) for the Treatment of Tumour-Induced Osteomalacia (TIO)

Ultragenyx (RARE) reports a wider-than-expected loss in the second quarter. Nevertheless, revenues marginally beat estimates.

Ultragenyx's (RARE) Q2 Earnings Miss Estimates, Revenues Up

Second quarter 2022 total revenue of $89.3 million and Crysvita® revenue in Ultragenyx territories 1 of $64.0 million

Ultragenyx Reports Second Quarter 2022 Financial Results and Corporate Update

NOVATO, Calif., July 25, 2022 (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 28, 2022, at 5:00pm ET to discuss its financial results and corporate update for the quarter ended June 30, 2022.

Ultragenyx to Host Conference Call for Second Quarter 2022 Financial Results and Corporate Update

Earlier today, Ultragenyx Pharmaceutical Inc (NASDAQ: RARE) released updated interim GTX-102 data, including Day 128 assessments from 9 patients.  The Overall CGI-C-AS score improved by 1 point in 5/6 patients (average 0.8) in Cohort 4 and 1 point in 2/3 patients (average 0.7).

Ultragenyx Shares Tumble On Initial Efficacy Missing Expectations, This Analyst Sees 'Viable Path Forward"

Shareholders aren't happy with the biotech's decision to acquire an early-stage asset for Angelman syndrome.

Why Ultragenyx Pharmaceutical Stock Is Cratering Today

Ultragenyx (RARE) and GeneTx Biotherapeutics provide an update and positive interim data from the phase I/II study on GTX-102 for treating patients with Angelman syndrome.

Ultragenyx (RARE) Provides Update From Angelman Syndrome Study

Doses up to 10 mg show good tolerability and meaningful clinical activity in multiple domains

Ultragenyx and GeneTx Provide Program Update on GTX-102 for Angelman Syndrome Including Promising Interim Data from Phase 1/2 Study

Ultragenyx (RARE) sells 30% of the royalty interest receivable on future North America sales of Crysvita for $500 million. The total payments are capped at 1.45 times the purchase price.

Ultragenyx (RARE) Sells Portion of Royalty Interest for $500M

Strengthens balance sheet with non-dilutive capital at an attractive cost

Ultragenyx Announces Sale of a Portion of Future North American Royalties on Crysvita® (burosumab) for $500 Million to OMERS Capital Markets

TOKYO--(BUSINESS WIRE)--Kyowa Kirin Co., Ltd. (TSE: 4151, Kyowa Kirin) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended that CRYSVITA® (burosumab) be approved for the treatment of FGF23-related hypophosphataemia in Tumour-Induced Osteomalacia (TIO) associated with phosphaturic mesenchymal tumours (PMTs) that cannot be curatively resected or localised in children and adolescents aged 1 to 17 years and in adults.

Kyowa Kirin Receives Positive CHMP Opinion for Use of CRYSVITA® ▼(burosumab) for the Treatment of Tumour-Induced Osteomalacia (TIO)

Ultragenyx is a rare disease drug developer. Its execution, so far, has been near perfect.

News for Ultragenyx Pharmaceutical Stock (RARE)