Commercial Growth & Financials - Ultragenyx anticipates Crysvita revenue in Ultragenyx territories to be between $250 million and $260 million for 2022[37] - Dojolvi global revenue is projected to be between $55 million and $65 million in 2022[37] - The company's cash balance as of March 31, 2022, was approximately $814 million[37] Pipeline Development & Milestones - Ultragenyx has 7 clinical programs, with 5 in pivotal studies across various modalities and therapeutic areas[10] - Data from 2 programs are expected in 2022, with execution across 5 pivotal studies[12, 38] - GTX-102 for Angelman Syndrome Phase 1/2 data in 12 Ex-US Patients is expected in mid-2022[38] - Phase 2/3 Orbit study in patients 5-25 years old initiated in April 2022 for UX143 (setrusumab) for Osteogenesis Imperfecta[79] Strategic Collaborations & Product Expansion - Ultragenyx licensed ex-US rights from Regeneron for Evkeeza (evinacumab) in HoFH for $30 million upfront and $63 million in milestones[9, 58] - Evkeeza demonstrated a 49% reduction in LDL-C relative to placebo in all patients in a Phase 3 study[62]
Ultragenyx Pharmaceutical (RARE) Investor Presentation - Slideshow