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.

Company will host investor call on April 17 at 8:00 a.m. ET to discuss new data from GTX-102 Phase 1/2 clinical study in patients with Angelman syndrome Company will host investor call on April 17 at 8:00 a.m. ET to discuss new data from GTX-102 Phase 1/2 clinical study in patients with Angelman syndrome

Ultragenyx Announces Upcoming Data Presentations at American Academy of Neurology (AAN) 2024 Annual Meeting

44 th Annual Cowen Healthcare Conference on March 4 Leerink Partners Global Biopharma Conference on March 12 Barclays 26 th Annual Global Healthcare Conference on March 13 NOVATO, Calif., Feb. 27, 2024 (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 management will be participating in three upcoming investor conferences.

Ultragenyx to Participate at Investor Conferences in March

Ultragenyx (RARE) reports better-than-expected fourth-quarter 2023 results, beating both earnings and revenue estimates, driven by strong sales trends of Crysvita and Dojolvi.

Ultragenyx (RARE) Q4 Loss Narrower Than Expected, Revenues Beat

Ultragenyx Pharmaceutical, Inc. (RARE) Q4 2023 Earnings Call Transcript

Although the revenue and EPS for Ultragenyx (RARE) give a sense of how its business performed in the quarter ended December 2023, it might be worth considering how some key metrics compare with Wall Street estimates and the year-ago numbers.

Compared to Estimates, Ultragenyx (RARE) Q4 Earnings: A Look at Key Metrics

Ultragenyx (RARE) came out with a quarterly loss of $1.52 per share versus the Zacks Consensus Estimate of a loss of $1.65. This compares to loss of $2.16 per share a year ago.

Ultragenyx (RARE) Reports Q4 Loss, Tops Revenue Estimates

2023 Total Revenue of $434 million, Crysvita® revenue of $328 million and Dojolvi® revenue of $71 million 2024 Financial Guidance: Total Revenue between $500 million and $530 million, Crysvita revenue of $375 million to $400 million, and Dojolvi revenue of $75 million to $80 million Year-end 2023 cash balance of $777 million and 2024 guidance for Net Cash Used in Operations expected to be less than $400 million NOVATO, Calif., Feb. 15, 2024 (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 reported its financial results for the quarter and full year ended December 31, 2023 and reaffirmed its financial guidance for 2024.

Ultragenyx Reports Fourth Quarter and Full Year 2023 Financial Results and Corporate Update

Looking beyond Wall Street's top -and-bottom-line estimate forecasts for Ultragenyx (RARE), delve into some of its key metrics to gain a deeper insight into the company's potential performance for the quarter ended December 2023.

What Analyst Projections for Key Metrics Reveal About Ultragenyx (RARE) Q4 Earnings

NOVATO, Calif., Feb. 08, 2024 (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, February 15, 2024, to discuss its financial results and corporate update for the fourth quarter and the year ending December 31, 2023.

Ultragenyx to Host Conference Call for Fourth Quarter and Full-Year 2023 Financial Results and Corporate Update

Ultragenyx (RARE) possesses 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) Expected to Beat Earnings Estimates: Can the Stock Move Higher?

The latest data from the pivotal Transpher A and long-term follow-up studies will be presented at WORLDSymposium™ 2024 The latest data from the pivotal Transpher A and long-term follow-up studies will be presented at WORLDSymposium™ 2024

Ultragenyx Announces Data Demonstrating Treatment with UX111 Results in Significant Reduction in Heparan Sulfate Exposure in Cerebrospinal Fluid Correlated with Improved Long-term Cognitive Function in Patients with Sanfilippo Syndrome Type A (MPS IIIA)

GTX-102 is the first Angelman syndrome therapeutic candidate to receive PRIME designation Phase 1/2 study fully enrolled; expansion data expected in first half of 2024 NOVATO, Calif., Feb. 05, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the European Medicines Agency (EMA) has granted Priority Medicine (PRIME) designation to GTX-102 for the treatment of Angelman syndrome (AS).

Ultragenyx Receives PRIME Designation from European Medicines Agency (EMA) for GTX-102 for the Treatment of Angelman Syndrome

Safety and initial efficacy data from Stage 1 expected in the first half of 2024 Dose selection and initiation of Stage 2 to follow in the second half of 2024 NOVATO, Calif., Jan. 25, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that all patients have been dosed with UX701 across the three dose-escalation cohorts in Stage 1 of its pivotal Phase 1/2/3 Cyprus 2+ study.

Ultragenyx Announces Completion of Dosing Across Stage 1 Cohorts in Pivotal Phase 1/2/3 Cyprus2+ Study Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease

Ultragenyx Pharmaceutical Inc. (RARE) reached a significant support level, and could be a good pick for investors from a technical perspective. Recently, RARE's 50-day simple moving average broke out above its 200-day moving average; this is known as a "golden cross.

After Golden Cross, Ultragenyx (RARE)'s Technical Outlook is Bright

Ultragenyx (RARE) reports preliminary full-year 2023 revenues, along with business updates, and shares 2024 revenue guidance.

Ultragenyx's (RARE) Preliminary 2023 Revenues Disappoint

Preliminary 2023 Total Revenue of $430 million to $435 million,  Crysvita® Revenue of $325 million to $330 million and Dojolvi® revenue of $70 million to $71 million

Ultragenyx Reports Preliminary 2023 Revenue; Guidance for 2024 Revenue and Cash Usage; Pipeline Updates and 2024 Milestones

Ultragenyx (RARE) completes enrollment in the phase I/II study of GTX-102 for the treatment of pediatric patients with AS. Top-line data from the study is expected in the first half of 2024.

Ultragenyx (RARE) Posts Update From AS Study of GTX-102

NOVATO, Calif., Jan. 04, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the National Institute for Health and Care Excellence (NICE) has issued a final draft guidance recommending Evkeeza® (evinacumab) to NHS England. Evkeeza is recommended within its marketing authorization as an adjunct to diet and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies for the treatment of adults and adolescent patients aged 12 years and older with homozygous familial hypercholesterolemia (HoFH). Evkeeza is the first angiopoietin-like 3 (ANGPTL3) inhibitor treatment indicated for this rare and debilitating condition.

Ultragenyx Receives Positive Recommendation from NICE in the U.K. for Evkeeza® ▼ (evinacumab) for Adolescents and Adults Aged 12 Years and Older with Homozygous Familial Hypercholesterolemia (HoFH)

Data from at least 20 patients enrolled in dose expansion cohorts anticipated in the first half of 2024 Data from at least 20 patients enrolled in dose expansion cohorts anticipated in the first half of 2024

Ultragenyx Announces Completion of Enrollment in Global Phase 1/2 Trial of GTX-102 in Pediatric Patients with Angelman Syndrome

NOVATO, Calif., Jan. 02, 2024 (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 the 42nd Annual J.P. Morgan Healthcare Conference on Monday, January 8, 2024, at 3:00 PM PT.

Ultragenyx to Present at the 42nd Annual J.P. Morgan Healthcare Conference

The mean of analysts' price targets for Ultragenyx (RARE) points to an 87.4% upside in the stock. While this highly sought-after metric has not proven reasonably effective, strong agreement among analysts in raising earnings estimates does indicate an upside in the stock.

Does Ultragenyx (RARE) Have the Potential to Rally 87.43% as Wall Street Analysts Expect?

For the first time, paediatric patients aged 5-11 years with HoFH in the EU have access to a first-in-class medicine to treat an ultrarare, inherited form of high cholesterol For the first time, paediatric patients aged 5-11 years with HoFH in the EU have access to a first-in-class medicine to treat an ultrarare, inherited form of high cholesterol

Ultragenyx Receives European Commission Decision for Evkeeza® (evinacumab) Expanded Indication in Children Aged 5 Years and Older with Homozygous Familial Hypercholesterolemia (HoFH)

6 th Annual Evercore ISI HealthCONx on November 28 in Miami Piper Sandler 35th Annual Healthcare Conference on November 29 in New York City NOVATO, Calif., Nov. 20, 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 management, will participating in two upcoming investor conferences.

Ultragenyx to Participate at Investor Conferences in November

NOVATO, Calif., Nov. 07, 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 a fireside chat at the upcoming Jefferies London Healthcare Conference on Tuesday, November 14, 2023, at 12:30 p.m. GMT.

Ultragenyx to Participate in the Jefferies London Healthcare Conference

Ultragenyx (RARE) reports weaker-than-expected third-quarter 2023 results, missing earnings and sales estimates. Management reiterates the 2023 financial outlook.

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