Ultragenyx Analyst Day Presentation

Diverse commercial and clinical pipeline. Candidate Kyowa Kirin CRYSVITA Mepsevii Description Pre-Clinical IND Phase 1 Anti-FGF23 Monoclonal Antibody X-Linked Hypophosphatemia (XLH) & Tumor-Induced Osteomalacia (TIO) Enzyme Replacement Mucopolysaccharidosis Type VII (MPS VII) Regeneron Anti-ANGPTL3 2 Homozygous Familial Hypercholesterolemia (HoFH) Evkeeza® Monoclonal Antibody² Mereo Biopharma Osteogenesis Imperfecta (OI) Anti-Sclerostin Monoclonal Antibody Substrate Replacement UX143 (setrusumab) DOJOLVI UX111 (ABO-102) AAV9 Gene Therapy DTX401 AAV8-G6Pase Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Sanfilippo Syndrome (MPS IIIA) Glycogen Storage Disease Type la (GSDIa) DTX301 Gene Therapy AAV8-OTC Gene Therapy Ornithine Transcarbamylase (OTC) Deficiency UX701 AAV9-ATP7B Gene Therapy Wilson Disease (WD) AAV9 UX055 CDKL5 Deficiency Disorder Gene Therapy Microdystrophin UX810 Duchenne Muscular Dystrophy Gene Therapy GTX-102 Antisense Oligonucleotide Angelman Syndrome (AS) 1: Prevalence in commercially accessible geographies 2: Ultragenyx licensed ex-US rights to Evkeeza from Regeneron 3: Excludes the US, where Regeneron has rights 9 Confidential and Proprietary Phase 2 Phase 3 Approved Prevalence¹ Key Protein Biologic Small Molecule Gene Therapy Nucleic Acid ~50,000 ~200 ~3,000 5,000³ ~60,000 ~8,000 14,000 ~3,000 - 5,000 ~6,000 ~10,000 ~50,000 ~20,000 30,000 ~40,000 ~60,000 ultragenyx

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