Ultragenyx Pharmaceutical (RARE) Investor Presentation - Slideshow

Financial Performance & Guidance - Ultragenyx reported total revenue of $271 million in 2020[23] - Crysvita in Ultragenyx territories generated $138.9 million in revenue in 2020[23] - The company anticipates Crysvita revenue in Ultragenyx territories to be between $180 million and $190 million in 2021[23] - The company's cash balance as of 4Q20 was approximately $1.2 billion[23] - The company expects three therapeutic areas will approach $1 billion of product revenue in the next five years[11] Pipeline Development & Clinical Trials - Ultragenyx plans to initiate three gene therapy pivotal studies[6] - A Phase 3 study for DTX401 (GSDIa) is expected to enroll its first patient in 1H21[24,54] - A Phase 3 study for DTX301 (OTC Deficiency) is expected to enroll its first patient in 2H21[24,60] - A Phase 1/2/3 study for UX701 (Wilson Disease) is planned to initiate in 1H21[24,61] - A pediatric Phase 2/3 study for Setrusumab (Osteogenesis Imperfecta) is planned to initiate in late-2021[24,31] Key Products & Programs - Dojolvi generated $13 million in revenue in 2020[23] - Mepsevii generated $15.3 million in revenue in 2020[23]