Ultragenyx Pharmaceutical (NASDAQ:RARE) Posts Earnings Results

Ultragenyx Pharmaceutical (NASDAQ:RARE) announced its quarterly earnings results on Thursday. The biopharmaceutical company reported ($1.29) earnings per share for the quarter, missing analysts’ consensus estimates of ($1.20) by ($0.09), Zacks reports. The company had revenue of $207.00 million during the quarter, compared to analyst estimates of $199.60 million. Ultragenyx Pharmaceutical had a negative net margin of 91.95% and a negative return on equity of 414.17%. The firm’s revenue for the quarter was up 25.5% compared to the same quarter last year. During the same quarter last year, the business earned ($1.39) EPS.

Here are the key takeaways from Ultragenyx Pharmaceutical’s conference call:

• Ultragenyx received an incomplete response letter (IRL) for the UX111 BLA requiring additional CMC supportive documentation, which will delay approval timing despite long‑term clinical and biomarker data the company describes as robust.
• The company rolled a BLA for DTX‑401 (GSD Ia) at year‑end and expects a PDUFA action date in Q3 2026, presenting a near‑term approval and commercial opportunity.
• A Phase 3 readout for GTX‑102 (Angelman) is expected in H2 2026; the trial uses Bayley‑IV cognition as primary endpoint with centralized testing and a Multidomain Responder Index to bolster the dataset.
• Management announced a strategic restructuring including a 10% workforce reduction (≈130 roles) and guidance that combined R&D+SG&A will be flat-to-down in 2026 and down ≥15% in 2027, aiming for profitability in 2027.
• 2025 results showed momentum with $673 million revenue (+20% YoY) and $738 million in cash, but a $575 million net loss; upcoming launches and two potential PRVs could materially change the cash outlook.

Ultragenyx Pharmaceutical Price Performance

RARE traded down $2.45 during trading hours on Friday, reaching $21.05. 1,552,944 shares of the stock traded hands, compared to its average volume of 2,537,597. Ultragenyx Pharmaceutical has a fifty-two week low of $18.41 and a fifty-two week high of $46.27. The company has a market cap of $2.03 billion, a price-to-earnings ratio of -3.54 and a beta of 0.16. The firm’s fifty day simple moving average is $27.23 and its two-hundred day simple moving average is $29.78.

Insider Buying and Selling

In other Ultragenyx Pharmaceutical news, CFO Howard Horn sold 3,081 shares of the firm’s stock in a transaction that occurred on Wednesday, December 10th. The stock was sold at an average price of $36.38, for a total transaction of $112,086.78. Following the completion of the sale, the chief financial officer owned 95,146 shares in the company, valued at $3,461,411.48. This trade represents a 3.14% decrease in their position. The sale was disclosed in a document filed with the Securities & Exchange Commission, which is available through the SEC website. In the last quarter, insiders have sold 6,227 shares of company stock valued at $187,126. Insiders own 5.50% of the company’s stock.

Hedge Funds Weigh In On Ultragenyx Pharmaceutical

A number of hedge funds have recently added to or reduced their stakes in RARE. CANADA LIFE ASSURANCE Co boosted its position in shares of Ultragenyx Pharmaceutical by 2.0% during the third quarter. CANADA LIFE ASSURANCE Co now owns 26,932 shares of the biopharmaceutical company’s stock worth $817,000 after buying an additional 534 shares during the period. Van ECK Associates Corp grew its stake in shares of Ultragenyx Pharmaceutical by 26.1% in the third quarter. Van ECK Associates Corp now owns 2,650 shares of the biopharmaceutical company’s stock worth $80,000 after purchasing an additional 549 shares in the last quarter. Tidal Investments LLC grew its stake in shares of Ultragenyx Pharmaceutical by 4.7% in the second quarter. Tidal Investments LLC now owns 12,591 shares of the biopharmaceutical company’s stock worth $458,000 after purchasing an additional 564 shares in the last quarter. E Fund Management Co. Ltd. increased its holdings in shares of Ultragenyx Pharmaceutical by 9.1% during the second quarter. E Fund Management Co. Ltd. now owns 9,052 shares of the biopharmaceutical company’s stock valued at $329,000 after purchasing an additional 752 shares during the period. Finally, Xponance LLC raised its stake in shares of Ultragenyx Pharmaceutical by 6.6% during the fourth quarter. Xponance LLC now owns 14,390 shares of the biopharmaceutical company’s stock valued at $331,000 after purchasing an additional 896 shares in the last quarter. Institutional investors and hedge funds own 97.67% of the company’s stock.

More Ultragenyx Pharmaceutical News

Here are the key news stories impacting Ultragenyx Pharmaceutical this week:

• Positive Sentiment: Strong revenue and product sales: Ultragenyx reported FY2025 total revenue of $673M with Crysvita® at $481M and Dojolvi® at $96M; Q4 revenue topped consensus, showing continued commercial traction. Ultragenyx Reports Fourth Quarter and Full Year 2025 Financial Results and Corporate Update
• Positive Sentiment: Analyst stance remains constructive overall — coverage shows an average rating of “Moderate Buy,” which can underpin investor demand despite near-term headwinds. Ultragenyx Receives Average Rating of “Moderate Buy” from Analysts
• Neutral Sentiment: Mixed quarter: EPS of ($1.29) missed estimates (~$1.20) even as revenue grew ~25% YoY — a mixed earnings picture that partially offsets the revenue beat. Ultragenyx (RARE) Reports Q4 Loss, Beats Revenue Estimates
• Negative Sentiment: FY2026 revenue guidance of $730M–$760M missed Street expectations (~$785M), prompting investor concern about near-term growth and driving downward pressure on the stock. Ultragenyx shares fall as 2026 revenue guidance misses expectations
• Negative Sentiment: Program setback and workforce reduction: Ultragenyx laid off ~10% of staff after a Phase 3 brittle-bone (setrusumab) failure and amid FDA delays on gene therapy approvals — a material clinical and execution risk. Ultragenyx lays off 10% of workforce as FDA continues to stall gene therapy approval plans
• Negative Sentiment: Rising litigation risk: Multiple law firms have filed or solicited investors for securities class actions covering Aug 3, 2023–Dec 26, 2025; potential legal exposure and distraction are additional headwinds. ROSEN Encourages Ultragenyx Investors to Secure Counsel

Wall Street Analyst Weigh In

A number of equities analysts have recently commented on RARE shares. Wells Fargo & Company cut their price objective on Ultragenyx Pharmaceutical from $65.00 to $45.00 and set an “overweight” rating on the stock in a report on Tuesday, December 30th. TD Cowen cut their price target on shares of Ultragenyx Pharmaceutical from $86.00 to $75.00 and set a “buy” rating on the stock in a report on Wednesday, November 5th. Leerink Partners reduced their price target on shares of Ultragenyx Pharmaceutical from $80.00 to $70.00 and set an “outperform” rating on the stock in a research report on Tuesday, December 30th. Jefferies Financial Group reduced their target price on shares of Ultragenyx Pharmaceutical from $114.00 to $63.00 and set a “buy” rating on the stock in a report on Tuesday, December 30th. Finally, Bank of America lowered their price target on Ultragenyx Pharmaceutical from $72.00 to $58.00 and set a “buy” rating for the company in a report on Friday, January 2nd. Sixteen analysts have rated the stock with a Buy rating, one has assigned a Hold rating and one has assigned a Sell rating to the stock. Based on data from MarketBeat, the stock currently has a consensus rating of “Moderate Buy” and an average target price of $72.29.

About Ultragenyx Pharmaceutical

Ultragenyx Pharmaceutical Inc is a biopharmaceutical company focused on developing and commercializing therapies for rare and ultra-rare genetic disorders. Since its founding in 2010 and headquarters in Novato, California, the company has built expertise in protein replacement therapies, small molecules and gene therapy approaches to address high-unmet medical needs. Ultragenyx applies a precision medicine model, leveraging both in-house research and strategic collaborations to advance its product pipeline from discovery through regulatory approval.

The company’s commercial portfolio includes Crysvita (burosumab-tmyl) for X-linked hypophosphatemia, Mepsevii (vestronidase alfa-vjbk) for mucopolysaccharidosis VII and Dojolvi (triheptanoin) for long-chain fatty acid oxidation disorders.