Ultragenyx Pharmaceutical(RARE) - 2021 Q1 - Earnings Call Presentation

Commercial Performance & Financial Position - Ultragenyx reported total revenue of $99.4 million in Q1 2021[21] - Crysvita in Ultragenyx territories contributed $42.1 million in revenue[21] - Dojolvi generated $7.0 million in revenue[21] - The company's cash balance as of Q1 2021 was approximately $1.0 billion[21] - Ultragenyx anticipates Crysvita revenue in Ultragenyx territories to be between $180 million and $190 million for 2021[21] Clinical Development & Pipeline - Ultragenyx plans to initiate four pivotal clinical studies, including three gene therapy programs[6, 22] - A Phase 3 study for DTX401 in GSDIa is expected to begin in early 2H21[22] - A Phase 1/2/3 study for UX701 in Wilson Disease is expected to begin in early 2H21[22] - A Phase 2/3 study for Setrusumab in pediatric Osteogenesis Imperfecta is planned for late 2021[22, 28] Therapeutic Focus & Market Potential - Ultragenyx is targeting three therapeutic areas with the potential to generate $1 billion in product revenue within the next five years[11] - Setrusumab for Osteogenesis Imperfecta targets a worldwide prevalence of approximately 60,000 patients[19, 25] - GTX-102 for Angelman Syndrome targets a worldwide prevalence of approximately 60,000 patients[19, 31]