Andrzej Rostek/iStock via Getty Images Goldman Sachs has upgraded Ultragenyx Pharmaceutical ( NASDAQ: RARE ) to buy from neutral as it has increasing confidence in setrusumab, its candidate for the bone disorder osteogenesis imperfecta, as well as promise in its pipeline. The firm has a $67 price target (~62% upside). Analyst Salveen Richter said that based on discussions with key opinion leaders, phase 2 data on setrusumab is viewed positively, especially regarding fracture and bone mineral density data as well as "the potential for setrusumab to improve bone formation and strength in addition to reducing fractures via its sclerostin inhibition mechanism, a key differentiator vs.
standard-of-care bisphosphonates." Elsewhere in the pipeline, he is keeping an eye on phase 3 data for GTX-102 in Angelman syndrome and proof-of-concept data for Wilson’s gene therapy UX701. He also anticipates a potential 2026 approval or launch for gene therapy DTX401 in glycogen storage disease type 1.
Richter also sees Ultragenyx ( RARE ) achieving GAAP profitability in 2027. More on Ultragenyx Pharmaceutical Ultragenyx Pharmaceutical Earnings: No Visible Path To Financial Success Yet Ultragenyx Phase 3 study for DTX401 meets primary endpoint Ultragenyx Pharmaceutical GAAP EPS of -$2.03 misses by $0.
28, revenue of $108.8M misses by $7.64M Ultragenyx Pharmaceutical, Inc.
(RARE) Q1 2024 Earnings Call Transcript Seeking Alpha's Quant Rating on Ultragenyx Pharmaceutical.