Barclays has chosen to maintain an Overweight rating on Ultragenyx Pharmaceutical (NASDAQ: RARE), preserving a price target of $44.00 - a figure the research firm said represents nearly 79% upside relative to the then-current share price of $24.61. The reaffirmation forms part of Barclays' continuing coverage of the biopharmaceutical company, which specializes in treatments for rare inherited disorders.
Ultragenyx, with a market capitalization of approximately $2.37 billion, markets approved therapies that include Crysvita - used for X-linked hypophosphatemia - and Mepsevii - for mucopolysaccharidosis VII. The company also maintains a slate of clinical-stage candidates as it focuses on developing interventions for rare genetic diseases that have limited or no existing treatment options.
Barclays' maintained Overweight rating signals ongoing confidence in Ultragenyx's strategic direction and its capacity to grow within the specialized rare disease treatment market. That assessment sits alongside other recent firm-level analyses that display a range of responses to the company's latest clinical and financial developments.
In financial disclosures, Ultragenyx reported preliminary unaudited total revenue for 2025 in the range of $672 million to $674 million. Those figures outpaced the company’s prior guidance and mark a 20% increase from full-year 2024. Crysvita revenue was estimated at $480 million to $482 million, representing a 17% year-over-year gain, while Dojolvi revenue was reported in the range of $95 million to $97 million, consistent with guidance and reflecting 9% year-over-year growth.
Despite the top-line revenue progress, Ultragenyx experienced material clinical setbacks. Pivotal trials of setrusumab for osteogenesis imperfecta failed to meet primary endpoints. The trial outcomes prompted several investment firms to lower their stock price targets even as they retained Buy ratings. H.C. Wainwright set a new price target of $60, Guggenheim reduced its target to $52 and BofA Securities moved its target to $58. In commentary accompanying their adjustments, Guggenheim characterized the trials as "ill-conceived" or "under-powered," while BofA Securities emphasized a lack of statistically significant benefits observed in the studies.
On a separate regulatory front, Ultragenyx completed its Biologics License Application submission to the U.S. Food and Drug Administration for DTX401, a gene therapy candidate for Glycogen Storage Disease Type Ia. The company included the final chemistry, manufacturing, and controls module with the submission, following earlier submissions of non-clinical and clinical modules.
Collectively, the juxtaposition of stronger-than-expected preliminary revenue and pipeline volatility - including both clinical failure and advancement on regulatory filings - frames the near-term outlook for Ultragenyx as a mix of commercial progress and ongoing development risk for investors and market participants.