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.

Established UltraCare® Program Provides Ongoing Patient Support Services Established UltraCare® Program Provides Ongoing Patient Support Services

Ultragenyx Announces U.S. Commercial Launch of Dojolvi™ (triheptanoin), the First FDA-Approved Therapy for the Treatment of Long-chain Fatty Acid Oxidation Disorders

Ultragenyx (RARE) saw a big move last session, as its shares jumped more than 8% on the day, amid huge volumes.

Ultragenyx (RARE) Looks Good: Stock Adds 8.2% in Session

Ultragenyx (RARE) gets FDA approval forDojolvi for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders.

Ultragenyx's Dojolvi Gets FDA Nod to Treat Rare Disease

Shares of Ultragenyx Pharmaceutical Inc. RARE, +6.19% gained 1% in premarket trading on Tuesday, the day after the Food and Drug Administration approved Dojolvi in the treatment of a rare set of genetic metabolic disorders.

Ultragenyx gets second FdA approval in June

Fourth U.S. FDA Approval for Ultragenyx in the Past Three Years – All for Rare Diseases which Previously Had No Approved Therapies

Ultragenyx Announces U.S. FDA Approval of Dojolvi™ (UX007/triheptanoin), the First FDA-Approved Therapy for the Treatment of Long-chain Fatty Acid Oxidation Disorders

Ultragenyx (RARE) gets FDA approval for the label expansion of Crysvita into tumor-induced osteomalacia.

Ultragenyx Gets FDA Nod for Label Expansion of Crysvita

The Food and Drug Administration on Thursday approved Ultragenyx Pharmaceutical Inc.

FDA grants a second approval to Ultragenyx drug for rare disease

First Approved Therapy in the United States for Patients with TIO Who Cannot Undergo Surgical Removal of Tumors

Ultragenyx and Kyowa Kirin Announce U.S. FDA Approval of Crysvita® (burosumab) for the Treatment of Tumor-Induced Osteomalacia (TIO)

Ultragenyx's (RARE) shares rise more than 60% year to date on various positive pipeline developments.

Utragenyx Shares Soar Above 60% YTD on Pipeline Developments

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

Why Is Ultragenyx (RARE) Up 10.7% Since Last Earnings Report?

Arcturus Therapeutics

NOVATO, Calif. and SAN DIEGO, May  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 and Arcturus Therapeutics Holdings Inc. (Nasdaq: ARCT), a leading messenger RNA medicines company, today announced that Ultragenyx has exercised its option to purchase 600,000 shares of Arcturus common stock at $16.00 per share.

Ultragenyx Announces Exercise of Option to Purchase Additional Stock of Arcturus Therapeutics

All three patients in Cohort 3 now confirmed responders

Ultragenyx Announces Positive Longer-term Results from First Three Cohorts of Phase 1/2 Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency

Ultragenyx (RARE) reports wider-than-expected first-quarter 2020 loss.

Ultragenyx (RARE) Q1 Earnings & Sales Fall Short of Estimates

Ultragenyx Pharmaceutical Inc. (RARE) CEO Emil Kakkis on Q1 2020 Results - Earnings Call Transcript

As of late, it has definitely been a great time to be an investor Ultragenyx Pharmaceutical

Will Ultragenyx Pharmaceutical Continue to Surge Higher?

SARASOTA, Fla. and NOVATO, Calif., May  04, 2020  (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to GTX-102 for the treatment of Angelman syndrome (AS). GTX-102 is an investigational antisense oligonucleotide currently being evaluated in a Phase 1/2 study for the treatment of AS.

GeneTx and Ultragenyx Announce GTX-102 Granted Fast Track Designation by U.S. FDA for Treatment of Angelman Syndrome

Ultragenyx (RARE) is seeing favorable earnings estimate revision activity and has a positive Zacks Earnings ESP heading into earnings season.

Is a Surprise Coming for Ultragenyx (RARE) This Earnings Season?

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: Should You Buy?

Daiichi Sankyo granted non-exclusive license to Ultragenyx HeLa manufacturing platform

Ultragenyx Enters into Strategic Partnership with Daiichi Sankyo for Gene Therapy Manufacturing Technology

- REGENXBIO grants Ultragenyx exclusive, worldwide rights to NAV AAV8 and AAV9 Vectors for the development and commercialization of gene therapy treatments for a rare metabolic disorder  - Agreement for use of NAV Vectors expands ongoing relationship between Ultragenyx and REGENXBIO

REGENXBIO and Ultragenyx Announce New License Agreement for Use of NAV® Technology Platform for the Treatment of Rare Metabolic Disorder

SARASOTA, Fla. and NOVATO, Calif., March  16, 2020  (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare diseases, today announced that it has dosed the first patient in its KIK-AS (Knockdown of UBE3A-antisense in Kids with Angelman Syndrome) study of GTX-102, an experimental antisense oligonucleotide being evaluated for the treatment of Angelman syndrome (AS).

GeneTx and Ultragenyx Announce First Patient Dosed in Phase 1/2 Clinical Trial of GTX-102 in Patients with Angelman Syndrome

Ultragenyx (RARE) Down 31.1% Since Last Earnings Report: Can It Rebound?

CFO Shalini Sharp to continue in her role until September 2020 or earlier completion of transition CFO Shalini Sharp to continue in her role until September 2020 or earlier completion of transition

Ultragenyx Announces Planned Transition of Chief Financial Officer

NOVATO, Calif. and TOKYO, Feb.  27, 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 diseases, and Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151), a global specialty pharmaceutical company creating innovative medical solutions using the latest biotechnology, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the supplemental Biologics License Application (sBLA) for Crysvita® (burosumab) for the treatment of FGF23-related hypophosphatemia associated with phosphaturic mesenchymal tumors (tumor-induced osteomalacia; TIO) that cannot be curatively resected or localized. The FDA has assigned priority review designation with a Prescription Drug User Fee Act (PDUFA) target date of June 18, 2020.

Ultragenyx and Kyowa Kirin Announce FDA Acceptance and Priority Review Designation of Supplemental Biologics License Application for Crysvita® (burosumab) for Tumor-Induced Osteomalacia (TIO)

BioXcel Therapeutics

Viking Therapeutics

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