Ultragenyx Analyst Day Presentation
UX143 (setrusumab) for osteogenesis imperfecta:
Phase 2 data showed 67% reduction in AFR post-treatment
Radiographically Confirmed Fractures¹
2
Phase 3 Endpoint
(excluding Fingers, Toes,
Face, and Skull)
P=0.042
SUPER MAHO
Median Annualized Fracture Rate (AFR)
0.5
1.5
1
0.72
0.0
0
Pre-
Treatment²
After Setrusumab
Initiation
1: Data presented at the company's Analyst Day on October 16, 2023
2: Pre-Treatment period includes fractures in the two years before screening based on
medical record review and patient report, and fractures between screening and first dose
11
Confidential and Proprietary
Data presented at the company's Analyst Day on October 16, 2023
6 y/o male patient with Type IV OI,
increased mobility after 17 months on study
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