Truist says focus now on Apazunersen after Ultragenyx’s Setrusumab miss

Truist analyst Joon Lee notes that Ultragenyx (RARE) disclosed that neither ORBIT study nor COSMIC trial met the primary endpoint of fracture reduction, despite expected increases in bone mineral density. The firm has been skeptical of Setrusumab in osteogenesis imperfecta, despite Phase 2 results and key opinion leaders’ optimistic views. The update puts the pipeline focus squarely back on Apazunersen in Angelman due to report topline from Phase 3 Aspire in the second half of 2026, Truist points out. The firm’s diligence of the program, since its start in 2020, suggests Apazunersen has a much better probability of success in Angelman than Setrusumab had in OI. Truist has a Buy rating on the shares with a price target of $90.