Core Insights - The Phase 3 Orbit study for UX143 (setrusumab) in pediatric and young adult patients with osteogenesis imperfecta (OI) is on track for final analysis by the end of the year [1][3] - The Data Monitoring Committee has confirmed an acceptable safety profile for UX143, allowing the study to proceed [2] - Ultragenyx and Mereo BioPharma are collaborating on the development of setrusumab, targeting OI sub-types I, III, and IV [5][12] Study Details - The Phase 3 Orbit study has enrolled 159 patients across 45 sites in 11 countries, with a primary efficacy endpoint focused on annualized clinical fracture rate [7] - The Cosmic study, which is also in Phase 3, has enrolled 69 patients aged 2 to <7 years, comparing setrusumab to intravenous bisphosphonates [8] - Both studies will conduct final analyses after patients have been on therapy for at least 18 months, with specific statistical thresholds set for each study [4] Background on Osteogenesis Imperfecta - OI is a genetic disorder affecting bone metabolism, primarily caused by mutations in the COL1A1 or COL1A2 genes, leading to increased bone brittleness and a high rate of fractures [9] - Approximately 60,000 individuals are affected by OI in commercially accessible regions, with no globally approved treatments available [9] Mechanism of Setrusumab - Setrusumab is a fully human monoclonal antibody that inhibits sclerostin, which negatively regulates bone formation, potentially increasing bone mass and strength [10] - Previous studies have shown that anti-sclerostin antibodies can significantly improve bone formation and density in OI models [11] Company Profiles - Ultragenyx is focused on developing therapies for rare genetic diseases, with a portfolio aimed at addressing high unmet medical needs [13] - Mereo BioPharma is also dedicated to innovative therapeutics for rare diseases, with setrusumab as one of its key candidates [15]

Pipeline and Approvals - Ultragenyx expects Phase 3 data readout for UX143 for Osteogenesis Imperfecta (OI) in 2025[21, 32] - Ultragenyx anticipates completing Phase 3 enrollment for GTX-102 for Angelman Syndrome (AS) in the second half of 2025[46, 49] - The company has a PDUFA date of August 18, 2025, for UX111 for Sanfilippo syndrome (MPS IIIA), with a potential commercial launch in the second half of 2025[63, 67] - Ultragenyx expects to submit a BLA for DTX401 for Glycogen Storage Disease Type Ia (GSDIa) in mid-2025, with a potential launch in 2026[78, 80] Financial Performance and Projections - Ultragenyx projects total revenue to grow by 14-20% in 2025, reaching $640-670 million[93, 95] - The company anticipates Crysvita revenue to be $460-480 million in 2025, representing a 12-17% increase[95] - Dojolvi revenue is projected to be $90-100 million in 2025, a 2-13% increase[95] - Ultragenyx aims to achieve full-year GAAP profitability in 2027, driven by revenue growth, new product launches, and expense management[2, 96, 99] Clinical Trial Results - Phase 2 data for UX143 in OI showed a 67% reduction in annualized fracture rate (AFR) [33] - Phase 3 data for DTX401 in GSDIa demonstrated a statistically significant 41% reduction in daily cornstarch intake at Week 48 (p < 0.0001)[83] - UX111 for MPS IIIA showed >80% of participants reduced CSF HS by 50% in efficacy set[71]

Core Insights - Ultragenyx Pharmaceuticals (RARE) has received FDA Breakthrough Therapy designation for its investigational therapy GTX-102 (apazunersen) aimed at treating Angelman syndrome (AS) [1][5]. FDA Breakthrough Therapy Designation - The Breakthrough Therapy designation accelerates the development and review of drugs for serious conditions, granted when early clinical evidence indicates significant improvement over existing treatments [2]. - Drugs with this designation benefit from enhanced guidance and support from senior FDA officials [2]. Clinical Study Results - GTX-102 demonstrated rapid and lasting improvements in AS patients during a phase I/II study lasting up to three years, involving 74 patients aged 4 to 17 with complete maternal UBE3A gene deletion [5][6]. - Participants showed consistent developmental progress and ongoing improvements across multiple symptom areas during treatment [6]. Current and Future Studies - Ultragenyx is currently enrolling patients for a phase III Aspire study and plans to initiate a phase II/III Aurora study to evaluate GTX-102 for a broader range of AS genotypes [8]. - The Aurora study is expected to start in the second half of 2025 [8]. Market Context - AS is a rare neurogenetic disorder affecting approximately 60,000 individuals in accessible markets, leading to severe developmental challenges with no approved therapies currently available [7]. Other Clinical Programs - Ultragenyx is developing other gene therapy candidates, including UX143 for osteogenesis imperfecta, which received Breakthrough Therapy designation in October 2024 [9]. - The company is also evaluating UX701 for Wilson disease and has achieved significant results in the phase III GlucoGene study for glycogen storage disease type Ia [10]. - A regulatory application for UX111, an AAV gene therapy for Sanfilippo syndrome type A, is under Priority Review, with a decision expected on August 18, 2025 [11].

Core Insights - Ultragenyx Pharmaceutical Inc. has received Breakthrough Therapy Designation from the FDA for GTX-102 as a treatment for Angelman syndrome [1][2] - The designation highlights the urgent need for effective treatments and the promising clinical results observed with GTX-102 [2] - The Phase 3 Aspire study is currently enrolling participants, with plans for an additional Aurora study to evaluate GTX-102 in other genotypes and ages expected to start later in 2025 [3] Company Overview - Ultragenyx is a biopharmaceutical company focused on developing therapies for serious rare and ultra-rare genetic diseases [7] - The company has a diverse portfolio of approved medicines and treatment candidates aimed at addressing high unmet medical needs [7] - Ultragenyx's management team has extensive experience in the development and commercialization of rare disease therapeutics [8] Product Information - GTX-102 (apazunersen) is an investigational antisense oligonucleotide therapy designed to reactivate expression of the UBE3A gene [4] - The therapy has received multiple designations from the FDA, including Orphan Drug Designation and Fast Track Designation [4] - The Phase 1/2 study demonstrated consistent developmental gains in 74 patients aged 4-17 with a full maternal UBE3A gene deletion [2] Angelman Syndrome Insights - Angelman syndrome is a rare neurogenetic disorder affecting approximately 60,000 people in commercially accessible geographies [5] - The disorder is characterized by cognitive impairment, motor impairment, and seizures, requiring continuous care for affected individuals [6] - There are currently no approved therapies for Angelman syndrome, but animal models suggest potential for symptom improvement at any age [6]

Summary of Ultragenyx Pharmaceutical (RARE) FY Conference Call Company Overview - Ultragenyx Pharmaceutical is at a significant inflection point, achieving revenues between $640 million to $670 million from four products: Crysvita, Nepsevi, Zolcivi, and Evkesa [2][4] - The company is experiencing a global commercial growth rate of approximately 20% annually [2] - Six late-stage programs are in development, with three expected to be filed within the year [4] Pipeline and Product Development - Key late-stage programs include: - Gene therapy for MPS IIIA, currently under review [2] - Gene therapy for GSD I, expected to be filed soon [2] - Osteogenesis Imperfecta (OI) program with high confidence in its transformative potential [5][6] - Angelman syndrome program (ASPIRE study) is on track to complete enrollment by the end of the year [49] - The company aims to become profitable by 2027, leveraging its existing products and new launches [4][68] Clinical Trials and Expectations - The OI program (cetrusumab) shows promising data from phase two trials, with a 67% reduction in fractures [6][19] - A significant threshold for commercial success is expected to be a fracture reduction of over 40%, with hopes for results above 50% [20][21] - The COSMIC trial aims to demonstrate the superiority of cetrusumab over bisphosphonates, which is crucial for market positioning [29][33] Market Strategy and Commercialization - The company plans to focus on key opinion leaders and centers with a high patient volume for initial launches [46] - A field force of 40 to 50 personnel is expected to support the launch, with an emphasis on home infusion models [42][48] - The commercial launch is anticipated to be easier due to a concentrated patient population in clinics [25] Gene Therapy Opportunities - The MPS IIIA program targets a severe disease with a small patient population but high urgency for treatment [59][60] - GSD I has a larger patient population, estimated at 1,500 to 8,000 in the US, with a significant need for treatment [63] - The company believes that urgency and the severity of conditions will drive adoption of gene therapies [66] Financial Outlook - The company reiterated its 2025 revenue guidance, projecting a 14% to 20% year-over-year growth, primarily driven by existing products [67][68] - Approximately 85% of revenue is expected to come from current products, with new launches contributing a smaller portion initially [68][69] Additional Insights - The company emphasizes the importance of patient quality of life and how treatments improve their daily activities, which will be a key driver for adoption [22][24] - Secondary endpoints in clinical trials are considered important for understanding the broader impact of treatments on patients [26][28] This summary encapsulates the key points discussed during the Ultragenyx Pharmaceutical FY Conference Call, highlighting the company's current status, pipeline developments, market strategies, and financial outlook.

Company Overview - Ultragenyx has seen its shares increase by approximately 9.6% over the past month, outperforming the S&P 500 [1] - The stock currently holds a Zacks Rank of 3 (Hold), indicating an expectation of an in-line return in the coming months [4] Earnings Estimates - Fresh estimates for Ultragenyx have trended upward in the past month, although the magnitude of these revisions suggests a downward shift [2][4] - The company has a subpar Growth Score of D, a strong Momentum Score of A, and a poor Value Score of F, placing it in the bottom 20% for value investment strategy [3] Industry Performance - Ultragenyx is part of the Zacks Medical - Biomedical and Genetics industry, where Agios Pharmaceuticals has gained 20.6% over the past month [5] - Agios Pharmaceuticals reported revenues of $8.73 million for the last quarter, reflecting a year-over-year increase of 6.6% [6] - The expected loss per share for Agios Pharmaceuticals in the current quarter is projected at $1.74, which is a 3% decrease year-over-year [6]

Company Overview - Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company focused on developing and commercializing novel therapies for serious rare and ultra-rare genetic diseases [3] - The company has established a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical needs [3] Management and Strategy - The management team of Ultragenyx is experienced in the development and commercialization of therapeutics for rare diseases [4] - The company's strategy emphasizes time- and cost-efficient drug development to deliver safe and effective therapies to patients urgently [4] Upcoming Events - Emil Kakkis, M.D., Ph.D., the CEO of Ultragenyx, will participate in a fireside chat at the Goldman Sachs 46th Annual Global Healthcare Conference on June 10, 2025, at 10:00 a.m. ET [1] - A live and archived webcast of the panel will be available on the company's website [2]

Core Viewpoint - Ultragenyx Pharmaceutical Inc. released its 2024 Impact Report, emphasizing its commitment to corporate responsibility and its mission to transform the lives of individuals affected by rare diseases [1][2] Group 1: Corporate Responsibility Approach - The company's corporate responsibility is structured around six key pillars: Innovation, Patients, People, Communities, Planet, and Governance, aligning with its vision to lead in rare disease medicine [2] - The 2024 Impact Report includes updates for all six pillars and references Sustainability Accounting Standards Board (SASB) and Global Reporting Initiative (GRI) indices [2] Group 2: Innovation - Ultragenyx is dedicated to developing transformative treatments for rare diseases where no options currently exist [3] Group 3: Patients - The company supports the rare disease community by developing novel therapies, sharing scientific expertise, ensuring access to screening and treatment, and collaborating with policymakers for impactful changes [4] Group 4: People - Ultragenyx focuses on maintaining an inclusive, safe, and healthy work environment, along with fair and equitable compensation practices [5] Group 5: Communities - The company is committed to initiatives that positively impact the rare disease community, including public health access, STEAM education, and support for at-risk local communities [6] Group 6: Achievements and Recognition - In 2024, Ultragenyx successfully transferred the manufacturing process of its DTX401 gene therapy and advanced several key clinical programs for rare diseases [7] - The company supported over 80 clinical and non-clinical investigator-sponsored trials globally and assisted more than 650 patients in 50 countries through various access programs since 2013 [7] - Ultragenyx received multiple accolades in 2024, including being named one of the 100 Most Sustainable Companies by Barron's and recognized as one of the Top Places to Work in the USA for the second consecutive year [11]

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.

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