The stock price fell due to two phase III clinical setbacks. Jefferies is optimistic that Ultragenyx (RARE.US) will rebound with drug candidates in 2026 and give it a “buy” rating

The Zhitong Finance App learned that Ultragenyx Pharmaceutical (RARE.US) closed down about 42% on Monday due to the failure of two late-stage clinical trials targeting rare bone disease assets, but investment bank Jefferies released a report saying the company is expected to rebound in 2026 due to other drug candidates in its pipeline.

Jefferies analyst Maury Raycroft gave Ultragenyx a “buy” rating and said that the company may benefit from the release of Apazunersen (GTX-102) phase III data next year, an antisense oligonucleic acid drug used to treat Angelman syndrome (Angelman syndrome). He lowered his price target from $114 to $63 (there is still room for about 219% increase based on Monday's closing price).

He added that the company maintains its 2027 profit guidelines and will clarify plans to cut expenses in early 2026.

Raycroft also pointed out that hopes may not have been completely dashed for the drug candidate setrusumab, which has just failed in the phase III clinical trial of osteogenesis imperfecta. He said, “The US Food and Drug Administration (FDA) recently listed 'bone density (BMD) 'as an alternative indicator for osteoporosis, and we believe this may influence subsequent discussions between Ultragenyx and partner Mereo BioPharma (MREO) with the agency.”

However, Citi's attitude towards Ultragenyx is less optimistic. The bank has removed the company from its 90-day upward catalyst watch list.