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.

Shares of Ultragenyx Pharmaceutical Inc. RARE, +6.67% and Mereo BioPharma Group PLC MREO, +11.43% gained premarket on Tuesday after the companies on Monday announced results from a study of setrusumab in patients with osteogenesis imperfecta, a group of genetic bone disorders. Patients treated with setrusumab, a monoclonal antibody, had significant increases in a sensitive marker of bone formation and improvement in bone mineral density after three months, the companies said in a release.

Ultragenyx, Mereo shares gain after positive results from study of bone disorder treatment

Pediatric data show substantial induction of bone production in 1 week and a large increase in bone formation within 3 months of initiating monthly setrusumab treatment

Ultragenyx and Mereo BioPharma Announce Positive Data from the Ongoing Phase 2/3 Orbit Study of Setrusumab (UX143) in Osteogenesis Imperfecta (OI)

Ultragenyx's pipeline boasts a diverse array of gene therapy candidates, including UX111, DTX401, DTX301, and UX701, offering transformative potential in the treatment of multiple rare genetic diseases. The FDA recently approved amendments to the Phase 1/2 trial of GTX-102 in young patients with Angelman syndrome, allowing Ultragenyx to synchronize dose scopes and expedite U.S. enrollment. With a strong financial outlook, innovative product portfolio, and a commitment to high-value initiatives, Ultragenyx presents an exceptional long-term investment opportunity in the rare disease biopharmaceutical sector.

Ultragenyx: A Promising Bet On Rare Genetic Disorder Therapies

Ultragenyx has a healthy rare disease product and pipeline. There are some issues with its next emerging catalyst.

Ultragenyx: Despite OI Trial Doubts, This Is A Robust Rare Disease Company

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NOVATO, Calif., May 19, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today announced that Eric Crombez, M.D., the company's chief medical officer, will present at the 2023 Barclays - Gene Editing & Gene Therapy Summit on Wednesday, May 24, 2023, at 8:50 a.m. ET.

Ultragenyx to Present at the 2023 Barclays - Gene Editing & Gene Therapy Summit

Ultragenyx (RARE) stock climbs 5% after the FDA agrees to a protocol amendment to the phase I/II study of GTX-102 in pediatric patients with Angelman syndrome.

Ultragenyx's (RARE) GTX-102 Gets FDA Nod for Uniform Dosing

Amended U.S. protocol increases loading and maintenance doses to similar range as ex-U.S. expansion cohorts Amended U.S. protocol increases loading and maintenance doses to similar range as ex-U.S. expansion cohorts

Ultragenyx Receives FDA Agreement to Expand Ongoing Global Phase 1/2 Trial Evaluating GTX-102 in Patients with Angelman Syndrome in the U.S.

Oral presentations include longer term durability data from DTX301 and DTX401 and showcase the Pinnacle PCL™ (AAV vector producer cell line) platform and technology Oral presentations include longer term durability data from DTX301 and DTX401 and showcase the Pinnacle PCL™ (AAV vector producer cell line) platform and technology

Ultragenyx Announces Upcoming Data Presentations at American Society of Gene & Cell Therapy (ASGCT) 2023 Annual Meeting

NOVATO, Calif., May 05, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's chief executive officer and president, will present at Bank of America's 2023 Health Care Conference on Wednesday, May 10, 2023, at 9:20 a.m. PT.

Ultragenyx to Present at Bank of America's 2023 Health Care Conference

Ultragenyx Pharmaceutical's (RARE) first-quarter 2023 earnings and revenues miss estimates. The company maintains its 2023 guidance.

Ultragenyx (RARE) Q1 Earnings and Revenues Miss Estimates

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE ) Q1 2023 Earnings Conference Call May 4, 2023 5:00 PM ET Company Participants Joshua Higa - Vice President and Head of Investor Relations Emil Kakkis - Founder, President and Chief Executive Officer Erik Harris - Chief Commercial Officer and Executive Vice President Eric Crombez - Chief Medical Officer and Executive Vice President Aaron Olsen - Senior Vice President of Corporate Strategy and Finance Ted Huizenga - Chief Accounting Officer Conference Call Participants Gena Wang - Barclays Maury Raycroft - Jefferies Dae Gon Ha - Stifel Joon Lee - Truist Securities Yaron Werber - TD Cowen Kristen Kluska - Cantor Fitzgerald Michael Riad - Morgan Stanley Joel Beatty - Baird Operator Good afternoon, and welcome to the Ultragenyx First Quarter 2023 Financial Results Conference Call. At this time, all participants are in a listen-only mode.

Ultragenyx Pharmaceutical Inc. (RARE) Q1 2023 Earnings Call Transcript

First quarter 2023 total revenue of $100.5 million, Crysvita® revenue of $76.0 million and Dojolvi® revenue of $14.3 million

Ultragenyx Reports First Quarter 2023 Financial Results and Corporate Update

Huang brings decades of expertise to MassBio Board to aid in organizational mission Huang brings decades of expertise to MassBio Board to aid in organizational mission

Dennis Huang of Ultragenyx Named to MassBio's Board of Directors

NOVATO, Calif., April 27, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, today announced that it will host a conference call at 5:00 p.m. ET on Thursday, May 4, 2023, to discuss its financial results and corporate update for the quarter ending March 31, 2023.

Ultragenyx to Host Conference Call for First Quarter 2023 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.

Ultragenyx (RARE) May Report Negative Earnings: Know the Trend Ahead of Q1 Release

Ultragenyx (RARE) witnessed a jump in share price last session on above-average trading volume. The latest trend in earnings estimate revisions for the stock doesn't suggest further strength down the road.

Strength Seen in Ultragenyx (RARE): Can Its 6.7% Jump Turn into More Strength?

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NOVATO, Calif., March 28, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's chief executive officer and president, will present at Guggenheim's Genomic Medicines and Rare Disease Days on Monday, April 3, 2023, at 10:45 a.m. ET.

Ultragenyx to Present at Guggenheim's Genomic Medicines and Rare Disease Days

Dr. Camille Bedrosian to continue employment as strategic development advisor with the company Dr. Camille Bedrosian to continue employment as strategic development advisor with the company

Ultragenyx Appoints Dr. Eric Crombez Chief Medical Officer and Executive Vice President

NOVATO, Calif., Feb. 28, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's chief executive officer and president, will participate in an Orphan Neuro Panel at Cowen's 43rd Annual Health Care Conference on Tuesday, March 7, 2023, at 10:30 a.m. ET.

Ultragenyx to Participate in Orphan Neuro Panel at Cowen Healthcare Conference

Ultragenyx (RARE) reports a wider loss in the fourth quarter while revenues increase on growth in demand for its drugs.

Ultragenyx (RARE) Q4 Earnings Miss, Revenues Increase Y/Y

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE ) Q4 2022 Earnings Conference Call February 16, 2023 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 Camille Bedrosian - Executive Vice President and Chief Medical Officer Aaron Olson - SVP, Corporate Strategy and Finance Ted Hazinga - Chief Accounting Officer Conference Call Participants Gena Wang - Barclays Chris Raymond - Piper Sandler Joon Lee - Truist Securities Yigal Nochomovitz - Citi Research Tazeen Ahmad - Bank of America Jeff Hung - Morgan Stanley Maury Raycroft - Jefferies LLC Dae Gon Ha - Stifel Liisa Bayko - Evercore Anupam Rama - JPMorgan Joel Beatty - R W. Baird Operator Good afternoon, and welcome to the Ultragenyx Fourth Quarter and Full Year 2022 Financial Results Conference Call.

Ultragenyx Pharmaceutical Inc. (RARE) Q4 2022 Earnings Call Transcript

2022 Total Collaboration and Product Revenue of $355.6 million, Crysvita® revenue of $ 279.4 million and Dojolvi® revenue of $55.6 million

Ultragenyx Reports Fourth Quarter and Full Year 2022 Financial Results and Corporate Update

Ultragenyx has been punished on the chart over the past 2 years to date. Investors haven't rewarded the company's top-line growth, nor recognized the potential in its investigational pathways.

News for Ultragenyx Pharmaceutical Stock (RARE)