NOVATO, Calif., May 14, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) released its 2024 Impact Report today, which covers the company’s approach to corporate responsibility. The report highlights efforts to transform the lives of individuals and families affected by rare diseases, empower its employees, and make a meaningful impact on the communities it operates within and serves. “Since founding Ultragenyx 15 years ago, I’ve sought to build a next-generation rare disease company th ...

Summary of Ultragenyx Pharmaceutical Conference Call Company Overview - **Company**: Ultragenyx Pharmaceutical (RARE) - **Industry**: Rare Disease Biotech - **Profitability Goal**: Targeting full year GAAP profitability by 2027 driven by growth from four commercial programs and three upcoming launches [5][6][79] Key Points and Arguments Financial Projections - **2025 Revenue Forecast**: Expected total revenue between $640 million to $670 million [7] - **Upcoming Data Releases**: Data from the Osteogenesis Imperfecta (OI) program expected this year, with multiple gene therapy programs under review and filing [7][13] Regulatory Environment - **FDA Interactions**: Ongoing positive interactions with the FDA, particularly for the 111 gene therapy program, with inspections on schedule [13][14] - **Impact of Executive Orders**: Uncertainty regarding the impact of new executive orders on the rare disease sector, with market sentiment suggesting minimal impact [8][9] Product Pipeline - **Osteogenesis Imperfecta (OI)**: - Phase three trial ongoing with interim analyses planned [17][18] - Market opportunity estimated at 60,000 patients globally, with a significant portion in the US [29] - Anticipated faster ramp-up compared to previous products due to existing relationships with physicians [30][50] - **Angelman Syndrome**: - Phase three trial enrollment expected to complete this year, with data anticipated next year [58][72] - Market opportunity comparable to OI, with around 60,000 patients globally [76] - **Wilson Disease**: - Currently in phase two, with enrollment of a fourth cohort expected by year-end [83][84] - Market size estimated at 50,000 patients globally [84] - **Sanfilippo Syndrome**: - Market opportunity of 3,000 to 5,000 patients globally, with no approved therapies currently [93] - Potential pricing in the range of $2 to $4 million due to the disease's severity [93] Competitive Landscape - **Market Positioning**: Ultragenyx aims to be a leading therapy in the rare disease space, leveraging existing relationships with healthcare providers [49][50] - **Competition in Angelman**: While the market can support multiple players, success will depend on clinical data outcomes [81][82] Manufacturing and Cost Structure - **Manufacturing Facilities**: Gene therapy manufacturing located in the US, with plans to leverage existing infrastructure for cost efficiency [16][89] - **Cost of Goods Sold (COGS)**: Expected to be lower due to proprietary manufacturing methods [89] Pricing Strategy - **Value Proposition**: Emphasis on responsibly pricing products, particularly in cases where current standard care is inferior but cheaper [90][91] - **Market Acceptance**: Anticipated acceptance of gene therapy pricing due to the urgency and life-threatening nature of certain conditions [91] Additional Important Insights - **Patient-Centric Approach**: The company emphasizes its mission to help rare disease patients globally, which guides its strategic decisions [12] - **Future Investments**: Plans for future R&D investments will depend on the success of current programs and their market performance [80][81] This summary encapsulates the key points discussed during the Ultragenyx Pharmaceutical conference call, highlighting the company's strategic direction, product pipeline, and market positioning within the rare disease biotech industry.

LONDON, England, May 08, 2025 (GLOBE NEWSWIRE) -- Extremely rare 2003 Honda NSX-R stars in latest Hagerty Henry Catchpole videoOne of less than 140 second-generation NSX-R models produced and the first to be available for public auction outside of Japan for many yearsVideo filmed on the historic Gelleråsen Arena circuit in Sweden, a track raced by motorsport legends Jack Brabham and Sir Stirling Moss LONDON, England, May 08, 2025 (GLOBE NEWSWIRE) -- Broad Arrow Auctions, a Hagerty (NYSE: HGTY) company, pres ...

NOVATO, Calif., May 07, 2025 (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 Howard Horn, the company's chief financial officer and executive vice president, will participate in a fireside at Bank of America’s 2025 Healthcare Conference on Tuesday, May 13, 2025, at 8:00 a.m. PT. The live and archived webcast of the panel wil ...

Ultragenyx Pharmaceutical (RARE) reported first-quarter 2025 loss of $1.57 per share, wider than the Zacks Consensus Estimate of a loss of $1.54. The company had incurred a loss of $2.03 per share in the year-ago quarter.Ultragenyx’s total revenues amounted to $139.3 million in the reported quarter, up 28% year over year, on the back of higher product sales. The top line, however, missed the Zacks Consensus Estimate of $142 million. (See the Zacks Earnings Calendar to stay ahead of market-making news.)The c ...

For the quarter ended March 2025, Ultragenyx (RARE) reported revenue of $139.29 million, up 28% over the same period last year. EPS came in at -$1.57, compared to -$2.03 in the year-ago quarter. As these metrics influence top- and bottom-line performance, comparing them to the year-ago numbers and what analysts estimated helps investors project a stock's price performance more accurately. Here is how Ultragenyx performed in the just reported quarter in terms of the metrics most widely monitored and projecte ...

Company Performance - Ultragenyx reported a quarterly loss of $1.57 per share, slightly worse than the Zacks Consensus Estimate of a loss of $1.54, but an improvement from a loss of $2.03 per share a year ago, indicating a year-over-year improvement in loss figures [1] - The company posted revenues of $139.29 million for the quarter ended March 2025, missing the Zacks Consensus Estimate by 1.90%, but showing an increase from $108.83 million in the same quarter last year [2] - Over the last four quarters, Ultragenyx has surpassed consensus EPS estimates two times and topped consensus revenue estimates three times [2] Stock Outlook - Ultragenyx shares have declined approximately 7.6% since the beginning of the year, compared to a decline of 3.9% for the S&P 500 [3] - The current consensus EPS estimate for the upcoming quarter is -$1.27 on revenues of $160.05 million, and for the current fiscal year, it is -$5.14 on revenues of $653.73 million [7] - The estimate revisions trend for Ultragenyx is mixed, resulting in a Zacks Rank 3 (Hold), suggesting the stock is expected to perform in line with the market in the near future [6] Industry Context - The Medical - Biomedical and Genetics industry, to which Ultragenyx belongs, is currently ranked in the top 31% of over 250 Zacks industries, indicating a favorable industry outlook [8] - Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can impact Ultragenyx's stock performance [5]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934. For the quarterly period ended March 31, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934. For the transition period from to . Commission File No. 001-36276 ULTRAGENYX PHARMACEUTICAL INC. (Exact name of registrant as specified in its charter) Delaware 27-2546083 (State or other j ...

Ultragenyx Pharmaceutical (RARE) Q1 2025 Earnings Call May 06, 2025 05:00 PM ET Speaker0 Good afternoon, and welcome to the Ultragenyx First Quarter twenty twenty five Financial Results Conference Call. At this time, all participants are in a listen only mode. At the end of the prepared remarks, you will have an opportunity to ask questions during the Q and A portion of the call. It is now my pleasure to turn the call over to Joshua Higa, Vice President of Investor Relations. Speaker1 Thank you. We have iss ...

Ultragenyx Pharmaceutical (RARE) Q1 2025 Earnings Call May 06, 2025 05:00 PM ET Company Participants Joshua Higa - VP - Investor RelationsEmil Kakkis - Founder, President, CEO & DirectorErik Harris - Executive VP & Chief Commercial OfficerHoward Horn - Executive VP of Corporate Strategy & CFOEric Crombez - Chief Medical Officer & Executive VPTazeen Ahmad - MD - US Equity ResearchGena Wang - MD - Biotech Equity ResearchYigal Nochomovitz - DirectorWill Soghikian - Vice President - Equity ResearchLiisa Bayko - ...