Financial Data and Key Metrics Changes - In Q1 2025, the company reported revenue of $139 million, representing a 28% increase compared to Q1 2024 [15] - The net loss for the quarter was $151 million, or $1.57 per share, with total operating expenses of $282 million [18] - Cash, cash equivalents, and marketable securities stood at $563 million as of March 31, 2025 [18] - The company reaffirmed its 2025 revenue guidance of $640 million to $670 million, indicating a growth of 14% to 20% over 2024 [19] Business Line Data and Key Metrics Changes - Crysvita generated $103 million in revenue, with significant contributions from North America ($41 million), Latin America and Turkey ($55 million), and Europe ($7 million), marking a 25% growth over 2024 [16] - DeJolvi contributed $17 million, while EVKYSA and Mepsevii contributed $11 million and $8 million, respectively [17] - In Latin America and Turkey, Crysvita saw a remarkable 52% growth over 2024 [16] Market Data and Key Metrics Changes - The commercial team in Latin America reported approximately 775 patients on reimbursed therapy for Crysvita, with expectations for continued growth following successful reimbursement negotiations [8] - In the U.S., the adult patient population for Crysvita has exceeded initial expectations, contributing to revenue growth [9] - The EMEA region showed strong demand for DERJOVY, with over 260 patients treated under named patient sales [11] Company Strategy and Development Direction - The company is focused on expanding its commercial operations outside the U.S. and preparing for new product launches [3] - The development pipeline includes multiple late-stage programs, with significant progress in Phase III studies for various treatments [5] - The company is working on two separate Biologics License Applications (BLAs), with one under review and another expected to be submitted mid-2025 [6] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in 2025 being a transformational year, with expectations for substantial revenue growth and successful clinical data readouts [29] - The company is actively monitoring regulatory changes and does not anticipate material exposure from tariffs affecting its products [21] - Management highlighted the importance of continued demand for products in Latin America and the U.S. pediatric and adult markets [19] Other Important Information - The company is on track for a PDUFA action date of August 18 for the UX-111 BLA under review by the FDA [7] - The company expects net cash used in operations to decrease in the remaining quarters of 2025, aiming for full-year GAAP profitability by 2027 [19] Q&A Session Summary Question: Discussion on setrusumab and fracture reduction - Management explained the concept of variation in fracture rates among patients and its impact on study outcomes, emphasizing confidence in achieving significant results [34][36] Question: Likelihood of success in the third interim read for OI - Management expressed optimism about meeting the p-value threshold based on the study's design and patient data [44][46] Question: Impact of recent FDA appointments on the rare disease space - Management indicated that while there are concerns, they remain confident in their clinical data supporting their programs [50][52] Question: Growth drivers for Crysvita in Latin America - Management noted that patient experiences and physician engagement are key factors driving growth in the region [116]