Two failed phase III clinical trials caused the stock price to halve, Jefferies Financial Group Inc. is optimistic about Ultragenyx (RARE.US) rebounding with its candidate drugs by 2026, giving it a "buy" rating.

date
09:15 31/12/2025
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GMT Eight
Due to the failure of two late-stage clinical trials for assets targeting rare bone diseases, Ultragenyx Pharmaceutical (RARE.US) closed down about 42% on Monday.
Due to the failure of two late-stage clinical trials targeting rare bone diseases assets, Ultragenyx Pharmaceutical (RARE.US) closed down by about 42% on Monday, but investment bank Jefferies Financial Group Inc. released a report stating that the company is expected to rebound in 2026 considering other candidate drugs in its pipeline. Jefferies Financial Group Inc. analyst Maury Raycroft has given Ultragenyx a "buy" rating and stated that the company may benefit from the phase three data release of apazunersen (GTX-102) next year, a antisense oligonucleotide drug for the treatment of Angelman syndrome. He has lowered the target price from $114 to $63 (based on Monday's closing price, still around 219% upside). He also mentioned that the company is maintaining its profit guidance for 2027 and has plans to clearly cut expenses in early 2026. Raycroft also pointed out that hope may not be entirely lost for the candidate drug setrusumab, which just failed in the phase three clinical trials for osteogenesis imperfecta. He stated: "The FDA recently listed 'bone mineral density (BMD)' as an alternative endpoint for osteoporosis, which we believe could impact discussions between Ultragenyx, partner Mereo BioPharma (MREO), and the agency." However, Citigroup's sentiment towards Ultragenyx is not as optimistic. The bank has removed the company from its list of upcoming catalysts within the next 90 days.