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$162 Million


Follow-on Offering

Bookrunner, February 2015

Ultragenyx is a biotechnology company focused on the identification, acquisition, development, and commercialization of novel products for the treatment of serious rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. Ultragenyx has a robust pipeline of orphan disease products, including two approved assets, Mepsevii in MPS VII and Crysvita in X-linked hypophosphatemia (XLH). The Company has an additional five indications in clinical development, Crysvita in tumor-induced osteomalacia (TIO), UX007 in long-chain fatty acid oxidation disorders (LC-FAOD), DTX301 in ornithine transcarbamylase (OTC) deficiency, DTX401 in Glycogen Storage Disease Type Ia (GSDIa) and DTX701 in Wilson Disease. The Company anticipates continued commercial opportunity in 2019 with an NDA filing for UX007 in LC-FAOD anticipated mid-2019 and finalized registrational plans to expand Crysvita into TIO. Ultragenyx recently reported topline Phase I/II data for DTX301 in OTC with a clean safety profile and initial signs of efficacy with Cohort 3 data anticipated mid-2019.