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 (NASDAQ:RARE) moved higher in after-market trading after the company reported Q2 results. Quarterly Results Earnings per share were down 61.61% year over year to ($1.81), which missed the estimate of ($1.36).

Ultragenyx Pharmaceutical: Q2 Earnings Insights

Second quarter 2021 total revenue of $87.0 million including Crysvita 1 revenue to Ultragenyx of $44.7 million

Ultragenyx Reports Second Quarter 2021 Financial Results and Corporate Update

NOVATO, Calif., July 27, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel therapies for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call on Monday, August 2, 2021 at 5pm ET to discuss its financial results and corporate update for the quarter ended June 30, 2021.

Ultragenyx to Host Conference Call for Second Quarter 2021 Financial Results and Corporate Update

Ultragenyx targets rare diseases with high unmet need.

Ultragenyx: Prices Are Attractively Low

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) Q2 Earnings Expected to Decline

TOKYO--(BUSINESS WIRE)--An additional option is now available in the treatment of XLH, a rare metabolic bone disease

Kyowa Kirin Announces EU Approval for the Self-administration of CRYSVITA® ▼ (burosumab) to Treat X-Linked Hypophosphataemia (XLH)

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MADRID, Spain, July 13, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Mepsevii® (vestronidase alfa) has been approved for reimbursement by the Sistema Nacional de Salud (SNS) for the treatment of pediatric and adult patients with Mucopolysaccharidosis VII (MPS VII; Sly syndrome) for patients of all ages.

MEDIA ADVISORY: Ultragenyx Announces Mepsevii® (vestronidase alfa) Receives Reimbursement Approval for Treatment of Mucopolysaccharidosis VII in Spain

NOVATO, Calif., June 30, 2021 (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 the appointment of Corsee Sanders, Ph.D., to the Board of Directors, effective June 29, 2021. Dr. Sanders, an experienced veteran of the biopharma industry, will serve as an independent director.

Ultragenyx Appoints Corsee Sanders, Ph.D., to Board of Directors

A BofA Securities analyst is turning bullish on shares of Ultragenyx Pharmaceuticals (NASDAQ:RARE). Here's why.

Ultragenyx Pharmaceuticals Analyst Turns Bullish: What Investors Need To Know

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Patient enrollment to begin in early second half of 2021 in U.K. and Canada

GeneTx and Ultragenyx Receive Approval from U.K. Regulatory Agency to Begin Clinical Study of GTX-102 for the Treatment of Angelman Syndrome

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NOVATO, Calif., June 03, 2021 (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 Goldman Sachs 42nd Annual Healthcare Conference on Thursday, June 10, 2021 at 3:00 PM ET.

Ultragenyx to Present at Goldman Sachs 42nd Annual Healthcare Conference

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

Ultragenyx (RARE) Down 7.8% Since Last Earnings Report: Can It Rebound?

NOVATO, Calif., May 26, 2021 (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 Jefferies Virtual Healthcare Conference on Tuesday, June 1, 2021 at 4:30 PM ET.

Ultragenyx to Present at Jefferies Virtual Healthcare Conference

GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc (NASDAQ: RARE) have received clearance from Health Canada to begin enrolling in the Phase 1/2 study of GTX-102 pediatric patients with Angelman syndrome in Canada. Angelman syndrome is a rare neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene.

GeneTx Bio-Ultragenyx's Angelman Syndrome Study To Start Enrollment In Canada

SARASOTA, Fla. and NOVATO, Calif., May 19, 2021 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), companies partnered in the development of intrathecally administered GTX-102, an investigational treatment for Angelman syndrome, today announced that they have received clearance from Health Canada to begin enrolling the Phase 1/2 study of GTX-102 in pediatric patients with Angelman syndrome in Canada. The first patient in Canada is expected to be enrolled in the early second half of 2021, with clinical data from some patients in the study expected before the end of 2021.

GeneTx and Ultragenyx Receive Clearance from Health Canada to Begin Clinical Study of GTX-102 in Canada for the Treatment of Angelman Syndrome

Ultragenyx Pharmaceutical Inc (NASDAQ: RARE) has announced longer-term data from the Glycogen Storage Disease Type Ia (GSDIa) and Ornithine Transcarbamylase (OTC) Deficiency Phase 1/2 studies. Data were presented at the American Society of Gene & Cell Therapy (ASGCT) 24th Annual Meeting.

Ultragenyx's Gene Therapies For Genetic Metabolic Disorders Show Longer-Term Durable Responses

Durable and Clinically Meaningful Responses Maintained at More than 2.5 and 3 Years Post-Treatment in Phase 1/2 Studies of DTX401 for GSDIa and DTX301 for OTC

Ultragenyx Announces Positive Multi-Year Durability Data from Ongoing Phase 1/2 Gene Therapy Studies and Data on HeLa 3.0 Manufacturing Platform at American Society of Gene & Cell Therapy 2021 Annual Meeting

NOVATO, Calif., May 10, 2021 (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 Mardi Dier, the company's Chief Financial Officer and Camille Bedrosian, M.D., the company's Chief Medical Officer, will present at the BofA Securities Healthcare Conference on Wednesday, May 12, 2021 at 3:30 PM ET.

Ultragenyx to Present at Bank of America Securities Healthcare Conference

TOKYO--(BUSINESS WIRE)--Self-administration of CRYSVITA could help healthcare professionals meet the needs of XLH patients during and beyond the COVID-19 pandemic

Kyowa Kirin Receives Positive CHMP Opinion for the Self-administration of CRYSVITA® ▼ (burosumab) to Treat X-Linked Hypophosphataemia (XLH), a Rare Metabolic Bone Disorder

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Ultragenyx Pharmaceutical (RARE) Q1 sales increase year over year while loss narrows

Ultragenyx (RARE) Q1 Loss Narrows Y/Y, Revenues Beat Estimates

Ultragenyx Pharmaceutical Inc.'s (RARE) CEO Emil Kakkis on Q1 2021 Results - Earnings Call Transcript

News for Ultragenyx Pharmaceutical Stock (RARE)