Ultragenyx Pharmaceutical (RARE) Investor Presentation - Slideshow

Commercial Performance & Financials - Ultragenyx reported total revenue of $87 million in 2Q21[21] - Crysvita in Ultragenyx territories contributed $44.7 million to the 2Q21 revenue[21] - Dojolvi generated $10 million in revenue during 2Q21[21] - Mepsevii brought in $5.4 million in revenue for 2Q21[21] - The company's cash balance as of 2Q21 was approximately $973.8 million[21] - Ultragenyx anticipates Crysvita revenue in Ultragenyx territories to be between $180 million and $190 million for 2021[21] Clinical Development Programs & Milestones - Ultragenyx is initiating four pivotal clinical studies, including three gene therapy programs[6, 22] - A Phase 2/3 study of Setrusumab for Osteogenesis Imperfecta is planned, aiming to initiate in late 2021[13, 28] - GTX-102 for Angelman Syndrome showed positive interim Phase 1/2 efficacy data, with a mean global score of +2.4 across patients on the CGI-I-AS scale[29, 33] - DTX401, a gene therapy for GSDIa, is progressing to a Phase 3 trial with a planned 1:1 randomization of DTX401 to placebo in 50 patients[15, 48] - DTX301, a gene therapy for OTC Deficiency, is also advancing to a Phase 3 trial with a planned 1:1 randomization of DTX301 to placebo in 50 patients[15, 54]