Ultragenyx Pharmaceutical(RARE) - 2025 Q2 - Earnings Call Transcript

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]