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.

Ultragenyx Pharmaceutical, Inc. (NASDAQ:RARE ) Q3 2023 Earnings Conference Call November 2, 2023 5:00 PM ET Company Participants Joshua Higa - Vice President of Investor Relations Emil Kakkis - Chief Executive Officer & President Erik Harris - Chief Commercial Officer Howard Horn - Chief Financial Officer Eric Crombez - Chief Medical Officer Conference Call Participants Anupam Rama - JPMorgan Dae Gon Ha - Stifel Michael Riad - Morgan Stanley Tazeen Ahmad - Bank of America Kristen Kluska - Cantor Fitzgerald Maury Raycroft - Jefferies Yaron Werber - TD Cowen Laura Chico - Wedbush. Operator Good afternoon, and welcome to the Ultragenyx Third Quarter 2023 Financial Results Conference Call.

Ultragenyx Pharmaceutical, Inc. (RARE) Q3 2023 Earnings Call Transcript

While the top- and bottom-line numbers for Ultragenyx (RARE) give a sense of how the business performed in the quarter ended September 2023, it could be worth looking at how some of its key metrics compare to Wall Street estimates and year-ago values.

Ultragenyx (RARE) Q3 Earnings: Taking a Look at Key Metrics Versus Estimates

Third quarter 2023 total revenue of $98.1 million, Crysvita® revenue of $74.9 million and Dojolvi® revenue of $16.6 million

Ultragenyx Reports Third Quarter 2023 Financial Results and Corporate Update

NOVATO, Calif., Oct. 26, 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, November 2, 2023, to discuss its financial results and corporate update for the quarter ending September 30, 2023.

Ultragenyx to Host Conference Call for Third 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.

Will Ultragenyx (RARE) Report Negative Q3 Earnings? What You Should Know

NOVATO, Calif., Oct. 23, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today announced the closing of its previously announced underwritten public offering of 9,833,334 shares of its common stock, including the exercise in full by the underwriters of their option to purchase an additional 1,500,000 shares, at a price to the public of $30.00 per share, and, to certain investors in lieu of common stock, pre-funded warrants to purchase 1,666,722 shares of its common stock at a purchase price of $29.999 per pre-funded warrant, which equals the public offering price per share of the common stock less the $0.001 exercise price per share of each pre-funded warrant. The estimated net proceeds to the company, after deducting underwriting discounts and commissions and other offering expenses payable by the company, were approximately $326.1 million.

Ultragenyx Announces Closing of Public Offering of Common Stock and Pre-Funded Warrants and Full Exercise of Underwriters' Option to Purchase Additional Shares

NOVATO, Calif., Oct. 17, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today announced that it has commenced an underwritten public offering of up to $300,000,000 of shares of its common stock. In addition, the company is expected to grant the underwriters of the offering an option for a period of 30 days to purchase up to an additional $45,000,000 of shares of common stock at the public offering price, less the underwriting discount.

Ultragenyx Announces Proposed Public Offering of Common Stock

Ultragenyx (RARE) saw its shares surge in the last session with trading volume being higher than average. The latest trend in earnings estimate revisions may not translate into further price increase in the near term.

Ultragenyx (RARE) Surges 10.3%: Is This an Indication of Further Gains?

Treatment with setrusumab (UX143) for at least 6 months resulted in 67% reduction in annualized fracture rate in patients with OI in Phase 2/3 Orbit study Quantitative data from the Phase 1/2 study of GTX-102 for AS show clinically meaningful improvements in multiple domains as compared to natural history 4 of 5 patients in lowest-dose cohort of Phase 1/2/3 study of UX701 in Wilson disease are tapering off of chelators and/or zinc therapy, including 2 that are now completely off standard therapy Ultragenyx Analyst Day live webcast available today at 8:30 a.m. ET NOVATO, Calif.

Ultragenyx Announces Program and Pipeline Updates at Analyst Day Including Interim Data from Ongoing Studies in Osteogenesis Imperfecta (OI), Angelman Syndrome (AS) and Wilson Disease

Phase 2 data presented at ASBMR 2023 show treatment with setrusumab resulted in 67% reduction in annualized fracture rate associated with continuous and meaningful improvements in bone mineral density (BMD) Ultragenyx hosting Analyst Day on Monday, October 16 at 8:30 a.m. ET NOVATO, Calif.

Ultragenyx and Mereo BioPharma Announce Interim Phase 2 Data from Phase 2/3 Orbit Study Demonstrating Setrusumab (UX143) Significantly Reduced Fracture Rates in Patients with Osteogenesis Imperfecta (OI)

Ultragenyx to share data from late-stage program evaluating setrusumab (UX143) in osteogenesis imperfecta at American Society for Bone and Mineral Research

Ultragenyx to Present Setrusumab (UX143) Update at ASBMR 2023 Including New Data from Phase 2/3 Orbit Study in Osteogenesis Imperfecta (OI)

Ultragenyx will host Analyst Day webcast on Monday, October 16, 2023 to provide updates on programs for osteogenesis imperfecta, Angelman syndrome and more

Ultragenyx to Present Pipeline Update at Analyst Day on Monday, October 16, 2023

Evkeeza is a first-in-class medicine approved by Health Canada, the U.S. Food and Drug Administration (FDA) and European Commission (EC) to treat an ultrarare, inherited form of high cholesterol Evkeeza is a first-in-class medicine approved by Health Canada, the U.S. Food and Drug Administration (FDA) and European Commission (EC) to treat an ultrarare, inherited form of high cholesterol

Ultragenyx Launches Evkeeza® (evinacumab for injection) in Canada for the Treatment of Homozygous Familial Hypercholesterolemia (HoFH)

Ultragenyx (RARE) was a big mover last session on higher-than-average trading volume. The latest trend in earnings estimate revisions might not help the stock continue moving higher in the near term.

Ultragenyx (RARE) Soars 6.1%: Is Further Upside Left in the Stock?

Citi BioPharma Conference on September 6 in Boston Morgan Stanley Healthcare Conference on September 12 in New York City NOVATO, Calif., Aug. 31, 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.

Ultragenyx to Participate at Investor Conferences in September

The estate of Henrietta Lacks filed a lawsuit in Maryland federal court on Thursday accusing biopharmaceutical company Ultragenyx Pharmaceutical of unlawfully profiting from cells that were taken from Lacks' body without her consent during a medical procedure in 1951.

Henrietta Lacks' family sues Ultragenyx over use of HeLa cell line

Ultragenyx (RARE) reports mixed second-quarter 2023 results, maintaining its 2023 financial guidance. The company's pipeline progress is also on track.

Ultragenyx (RARE) Q2 Loss Wider Than Expected, Revenues Beat

Ultragenyx Pharmaceutical, Inc. (NASDAQ:RARE ) Q2 2023 Earnings Conference Call August 3, 2023 5:00 PM ET Company Participants Joshua Higa - Director, IR & Corporate Communications Emil Kakkis - Founder, President, CEO & Director Eric Crombez - Chief Medical Officer & EVP Aaron Olsen - SVP, Corporate Strategy & Finance Erik Harris - EVP & Chief Commercial Officer Conference Call Participants Gena Wang - Barclays Bank Liisa Bayko - Evercore ISI Maurice Raycroft - Jefferies Tommie Reerink - Goldman Sachs Group Joon Lee - Truist Securities Joori Park - Leerink Partners Kristen Kluska - Cantor Fitzgerald Dae Gon Ha - Stifel, Nicolaus & Company Yaron Werber - TD Cowen Nicole Gabreski - Piper Sandler & Co. Yigal Nochomovitz - Citigroup Operator Good afternoon, and welcome to the Ultragenyx Second Quarter 2023 Financial Results Conference Call. [Operator Instructions].

Ultragenyx Pharmaceutical, Inc. (RARE) Q2 2023 Earnings Call Transcript

Second quarter 2023 total revenue of $108.3 million, Crysvita® revenue of $83.0 million and Dojolvi® revenue of $16.5 million

Ultragenyx Reports Second Quarter 2023 Financial Results and Corporate Update

NOVATO, Calif., July 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, August 3, 2023, to discuss its financial results and corporate update for the quarter ending June 30, 2023.

Ultragenyx to Host Conference Call for Second Quarter 2023 Financial Results and Corporate Update

NOVATO, Calif., July 12, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today announced that it has appointed Howard Horn as executive vice president and chief financial officer (CFO) effective October 16, 2023. Mr. Horn will be responsible for leading the finance, accounting, corporate strategy and investor relations functions.

Ultragenyx Appoints Howard Horn as Chief Financial Officer and Executive Vice President, Corporate Strategy

Ultragenyx (RARE) announces dosing the first patients in its phase III studies evaluating setrusumab (UX143) in young adult and pediatric patients with osteogenesis imperfecta.

Ultragenyx (RARE) Doses First Patients in Bone Disease Studies

Pivotal Phase 3 portion of Orbit study now enrolling approximately 195 pediatric and young adult patients Newly initiated Phase 3 Cosmic study now enrolling approximately 65 younger pediatric patients NOVATO, Calif., July 06, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the first patients have been dosed in both of its late-stage clinical trials evaluating setrusumab in pediatric and young adult patients with OI sub-types I, III and IV.

Ultragenyx Announces First Patients Dosed in Phase 3 Program Evaluating Setrusumab (UX143) for the Treatment of Osteogenesis Imperfecta (OI)

NOVATO, Calif., June 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 at the upcoming Goldman Sachs 44th Annual Global Healthcare Conference on Monday, June 12, 2023, at 3:20 p.m. PT.

Ultragenyx to Participate in the Goldman Sachs 44th Annual Global Healthcare Conference

Ultragenyx (RARE), along with partner Mereo BioPharma, announces encouraging data on mid-stage study evaluating setrusumab for patients with osteogenesis imperfecta.

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