Two Phase 3 Trial Failures Halve Stock Price; Jefferies Sees Ultragenyx (RARE.US) Rebounding in 2026 with Pipeline Candidates, Initiates "Buy" Rating

Stock News2025-12-31

Ultragenyx Pharmaceutical (RARE.US) saw its stock plummet approximately 42% at Monday's close following the failure of two late-stage clinical trials for its rare bone disease assets. However, investment bank Jefferies issued a report suggesting the company is positioned for a rebound by 2026, thanks to other candidates in its pipeline. Jefferies analyst Maury Raycroft initiated coverage on Ultragenyx with a "Buy" rating, stating the company could benefit next year from Phase 3 data for apazunersen (GTX-102), an antisense oligonucleotide drug for treating Angelman syndrome. He reduced his price target from $114 to $63, which still implies a potential upside of about 219% from Monday's closing price. Raycroft added that the company has maintained its 2027 profitability guidance and plans to outline a cost-cutting strategy early in 2026. He also noted that hope may not be entirely lost for setrusumab, the candidate that just failed its Phase 3 trial in osteogenesis imperfecta. He stated, "The U.S. Food and Drug Administration (FDA) recently designated 'bone mineral density (BMD)' as a surrogate endpoint for osteoporosis, which we believe could influence Ultragenyx and its partner Mereo BioPharma's (MREO) subsequent discussions with the agency." Conversely, Citigroup expressed a less optimistic stance on Ultragenyx, removing the company from its 90-day positive catalyst watch list.

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