Phase 3 portion of Orbit Study and Phase 3 Cosmic Study of setrusumab for treatment of Osteogenesis Imperfecta (OI) conducted by partner Ultragenyx expected to complete enrollment around the end of 1Q 2024 and 1H 2024, respectively; Additional Phase 2 data expected in 2024
Alignment on design for single, global Phase 3 study of alvelestat in Alpha-1 Antitrypsin Deficiency-associated Lung Disease (AATD-LD) following additional interactions with U.S. FDA in 2H 2023
Signs global licensing deal for development and commercialization of leflutrozole
Reiterates previous cash runway guidance; current cash expected to fund operations into 2026
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