Financial Data and Key Metrics Changes - Total revenue for Q2 2025 was reported at $166 million, representing a 13% increase over Q2 2024 and a 20% growth year-to-date [19] - Crysvita contributed $120 million in revenue, with $79 million from North America, $35 million from Latin America and Turkey, and $7 million from Europe [19] - The net loss for the quarter was $115 million, or $1.17 per share, with total operating expenses of $274 million [20] Business Line Data and Key Metrics Changes - Crysvita continues to grow, with approximately 50 new start forms leading to around 50 new patients on reimbursed therapy in Latin America [12] - DERJOVY generated $23 million in revenue, with approximately 600 patients on reimbursed therapy since its launch in 2020 [14] - Efkesa has seen growth with nearly 285 patients across 15 countries in the EMEA region [16] Market Data and Key Metrics Changes - The company expects Crysvita revenue in the U.S. and Canada to continue growing as new pediatric and adult patients are identified [13] - The EMEA region shows strong demand for DERJOVY, particularly in France, Kuwait, Saudi Arabia, and Greece, despite not actively marketing the therapy [15] Company Strategy and Development Direction - The company aims to achieve GAAP profitability by 2027 while focusing on revenue growth and managing expenses [21][30] - The clinical pipeline is being refilled with five Phase III clinical programs fully enrolled or at the BLA submission stage [4] - The company is prioritizing the resolution of CMC issues related to UX-111 before submitting the BLA for GSD1a [65] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing clinical studies and the potential of their pipeline, particularly UX-143 and GTX-102 [6][28] - The company is optimistic about the upcoming Phase III data readouts and believes they will support the efficacy of their treatments [28] - Management acknowledged the challenges posed by recent FDA leadership changes but remains confident in their interactions with the agency [33][92] Other Important Information - The company has $539 million in cash, cash equivalents, and marketable securities as of June 30, 2025 [20] - The company is reaffirming its revenue guidance for 2025, expecting total revenue between $640 million and $670 million [21] Q&A Session Summary Question: Recent FDA interactions and changes in leadership - Management reported productive interactions with the FDA since the CRL and is working towards a Type A meeting package [33] Question: Differences in dosing regimens for GTX-102 - The LNA chemistry used in GTX-102 is more potent, allowing for lower dosing regimens compared to other drugs [36][37] Question: COSMIC trial rationale and assumptions - The COSMIC trial aims to evaluate young patients on bisphosphonates, with the goal of demonstrating a 50% improvement in fracture rates [40][41] Question: Expectations for the Orbit study - Management believes that even if the fracture data is slightly under expectations, other supportive data will still be compelling for FDA approval [60][61] Question: Additional clinical data for UX-111 resubmission - The FDA requested updated clinical endpoint data and biomarker data for the resubmission of UX-111 [55] Question: Financial strategy and cost management - The company is prioritizing cost management and delaying certain expenses to maintain cash flow while working towards profitability [84][86]

Group 1: Methode Electronics - Methode Electronics reported a fourth-quarter adjusted EPS loss of 77 cents per share, missing market estimates of earnings of 4 cents per share [1] - The company's sales were $257.10 million, exceeding estimates of $232.87 million [1] - Shares of Methode Electronics fell sharply in pre-market trading following the earnings report [1] Group 2: Other Stocks - Mereo BioPharma Group plc shares tumbled 36.6% to $1.87 in pre-market trading [4] - Ultragenyx Pharmaceutical Inc. shares dipped 21.3% to $32.50 in pre-market trading [4] - Wolfspeed, Inc. shares fell 11% to $1.79 in pre-market trading after a 20% drop on Wednesday [4] - Scage Future shares fell 10.5% to $5.89 in pre-market trading after a significant gain of over 56% on Wednesday [4] - Altimmune, Inc. shares dipped 8.4% to $4.34 in pre-market trading following the initiation of a Phase 2 trial [4] - Bitmine Immersion Technologies, Inc. shares fell 7.7% to $61.60 in pre-market trading after a 40% drop on Wednesday due to a mixed shelf filing [4] - Hotel101 Global Holdings Corp. shares dipped 5.3% to $3.42 in after-hours trading [4] - Metsera, Inc. shares fell 4.3% to $33.80 in pre-market trading after a 17% gain on Wednesday [4]

Financial Data and Key Metrics Changes - In Q1 2025, Ultragenyx reported revenue of $139 million, representing a 28% increase compared to Q1 2024 [18] - The net loss for the quarter was $151 million, or $1.57 per share, with total operating expenses of $282 million [20] - Cash, cash equivalents, and marketable securities stood at $563 million as of March 31, 2025 [20] Business Line Data and Key Metrics Changes - Crysvita generated $103 million in revenue, with contributions of $41 million from North America, $55 million from Latin America and Turkey, and $7 million from Europe, marking a 25% growth over 2024 [18] - DeJolvi contributed $17 million, consistent with its expected growth trajectory, while EVKYSA generated $11 million as demand builds [19] - Mepsevii contributed $8 million as the company continues to treat patients in this ultra-rare indication [19] Market Data and Key Metrics Changes - In Latin America and Turkey, Crysvita revenue grew by 52% over 2024, driven by successful reimbursement negotiations [18] - The U.S. market for Crysvita is expected to continue growing as the company identifies new pediatric and adult patients [11] - The EMEA region saw over 260 patients treated with DERJOVY under named patient sales, with strong demand noted particularly in France [13] Company Strategy and Development Direction - The company is focused on expanding its commercial operations globally while preparing for new product launches in the U.S. and worldwide [5] - Ultragenyx is advancing multiple late-stage programs, including UX143 for osteogenesis imperfecta and GTX-102 for Angelman syndrome, with data expected in 2026 [7][26] - The company is on track to submit two Biologics License Applications (BLAs) in 2025, with one currently under review and another expected mid-year [8][9] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence that 2025 will be a transformational year, with expectations of significant revenue growth and multiple clinical data readouts [32] - The company is optimistic about the demand for its products, particularly in Latin America and the pediatric and adult XLH markets in the U.S. [21] - Management acknowledged the evolving tariff landscape but does not expect material exposure for any products, including Crysvita [23] Other Important Information - The company is actively monitoring regulatory interactions and has scheduled inspections of manufacturing facilities and clinical sites [9] - The company expects net cash used in operations to decrease in the remaining quarters of 2025, aiming for full-year GAAP profitability by 2027 [21] Q&A Session Summary Question: Insights on setrusumab and the second interim analysis - Management discussed the variability in fracture rates among patients and the statistical methods used to analyze data, emphasizing confidence in achieving significant results [35][39] Question: Likelihood of success if the study moves to a third interim read - Management expressed confidence in hitting the p-value threshold based on observed data trends and the size of the study [46][49] Question: Impact of recent CBER nomination on the rare disease space - Management noted concerns about the nomination but emphasized the importance of maintaining accelerated approval pathways for rare diseases [51][54] Question: Enrollment status for the Angelman program - Management confirmed that all global sites for the Angelman program are active and screening patients [66][68] Question: Clarification on the timing of interim data release - Management indicated that data cleaning and analysis for the interim results would take approximately eight weeks, with a commitment to timely communication of results [90][92]