Canaccord Genuity trimmed its price target on Ultragenyx Pharmaceuticals (NASDAQ:RARE) to $79 from $128 on Tuesday, while maintaining a Buy rating. The move comes as the stock has weakened substantially, falling 15.49% over the past week and 53.74% over the past year, with shares quoted at $20.18, according to InvestingPro data.
Reasons cited by the analyst
Canaccord attributed the adjustment primarily to the company’s fourth-quarter 2025 results and a subsequent restructuring plan announced after unfavorable news relating to osteogenesis imperfecta. Management provided guidance showing that combined research and development (R&D) and selling, general and administrative (SG&A) expenses for 2026 are expected to be flat to down by single-digit percentages versus 2025. Additionally, Ultragenyx signaled a marked reduction in R&D spending over the longer term, with R&D costs projected to fall by approximately 38% in 2027 compared with 2025.
The firm also highlighted a regulatory development: Ultragenyx received an information request letter from the U.S. Food and Drug Administration after resubmitting the biologics license application for UX111 in January 2026. Canaccord noted this FDA interaction as a material concern and said it is at odds with the agency’s stated support for rare disease and gene therapy development. That point is notable because much of Ultragenyx’s strategic approach depends on collaborative engagement with the FDA, including flexibility around rare disease endpoints.
Valuation and pipeline assumptions
In its updated modeling, Canaccord projects peak annual sales for UX111 at about $280 million. The firm said that current market prices for Ultragenyx shares essentially reflect the underlying base business at a discount and ascribe a zero value to the remainder of the pipeline in their present valuation.
Despite the setback around UX111’s regulatory pathway, Canaccord still expects positive Phase 3 data from GTX-102 for Angelman syndrome in the second half of 2026, with primary completion estimated in July of that year. InvestingPro analysis cited alongside the note indicates that some market participants view Ultragenyx as undervalued at recent prices, and that a consensus of analysts continues to recommend a "Strong Buy," noting revenue growth of 20.13% over the last twelve months.
Quarterly financials
Ultragenyx reported Q4 2025 revenue of $207 million, above the $192.15 million forecast, representing a 7.73% upside to expectations. The company, however, missed on earnings per share, reporting an EPS of -$1.29 versus the anticipated -$1.15, a shortfall representing a 12.17% negative surprise. These mixed results corresponded with a modest decline in Ultragenyx’s shares during after-hours trading following the release.
The quarter’s numbers highlight a dual picture: top-line performance that outpaced forecasts alongside earnings that fell short of projections. Investors and analysts will likely weigh both elements as they assess Ultragenyx’s near-term prospects alongside the implications of the FDA information request and the company’s announced cost reductions.
What this means going forward
Canaccord’s decision to lower the price target while keeping its Buy rating reflects a recalibration of expected upside tied to regulatory uncertainty and planned cost-cutting. The firm’s modeling choices, including valuing the pipeline at zero outside the base business, underscore the immediate emphasis on confirmed commercial performance and near-term clinical readouts. Expectations for GTX-102’s Phase 3 readout in H2 2026 remain a key event cited by the analyst.
For market participants, the combination of regulatory questions, a reshaped expense profile, and mixed quarterly results creates a complex risk-reward profile that will inform trading and coverage decisions over the coming quarters.