Ultragenyx Analyst Day Presentation

UX701 for Wilson Disease: Four of five in Cohort 1 tapering SOC, including two completely off chelators and/or zinc therapy 1 Cohort 1 Weeks on Study Reduction of SOC [Chelator and/or zinc therapy] Copper Trafficking Patient 1 82 0% Patient 2 70 100% Patient 3 44 100% Patient 4 20 33% Patient 5 16 50% 1: Data presented at the company's Analyst Day on October 16, 2023 14 Confidential and Proprietary Indeterminate Reduced urinary copper and improved trafficking by copper oxidase assay Reduced urinary copper and improved trafficking by copper oxidase assay Reduced urinary copper; pending trafficking assessment Reduced urinary copper; pending trafficking assessment ultragenyx

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