Ultragenyx Pharmaceutical Inc.

Total revenue for the second quarter of 2026 was $214 million. Crysvita contributed $156 million, including $94 million from North America, $54 million from Latin America and Turkey, and $8 million from Europe. DOJOLVI contributed $27 million, Evkeeza contributed $21 million, and MEPSEVII contributed $10 million. Operating expenses were $289 million, including $34 million of cost of sales and $255 million of combined R&D and SG&A, with $34 million of non-cash stock-based compensation. Net loss was $92 million, or $0.90 per share. Cash equivalents and marketable securities were $436 million at June 30, and net cash used in operations was $97 million. Management reaffirmed full-year revenue guidance and combined R&D and SG&A guidance, but did not give new numeric guidance on this call.

Emil Kakkis emphasized execution across both the commercial and development organizations, calling out record quarterly revenue and saying the company is positioned for a major second half of 2026. He framed DTX-401 and UX-111 as potential first-ever treatments for devastating rare diseases, and said the team remains confident in the regulatory work and launch preparedness. His tone was upbeat and urgent, with a clear focus on near-term catalysts and a path to profitability in 2027.

Howard Horn said the quarter’s revenue mix reflected strong underlying demand plus seasonal ordering timing, and noted that operating expenses came in at $289 million with $34 million of stock-based compensation. He highlighted $436 million in cash and marketable securities, and $97 million of operating cash use in Q2, which he said was a significant improvement versus Q1. On the path to profitability, he pointed to three levers: double-digit growth from current products plus launches, continued expense discipline, and potential non-dilutive capital from priority review voucher monetization if DTX-401 and UX-111 are approved.

Analysts focused heavily on GTX-102’s statistical plan, including whether one endpoint alone would be enough for filing, how the Bayley and MDRI endpoints should be interpreted, and whether the study could still support a filing if results were mixed. Management said FDA had agreed to the alpha-sharing approach, that either endpoint can be positive for success, and that the endpoints were chosen to reflect clinically meaningful changes. Questions also centered on setrusumab, where management said FDA and MHRA discussions suggest more clinical data may be needed and that BMD is not yet accepted as a primary basis for approval; on Angelman safety, management said no study-stopping safety issue had emerged and no FDA threshold for lower-extremity weakness was provided.