Ultragenyx Pharmaceutical (NASDAQ:RARE) Posts Earnings Results, Misses Estimates By $0.09 EPS

Ultragenyx Pharmaceutical (NASDAQ:RARE) announced its earnings results on Thursday. The biopharmaceutical company reported ($1.29) earnings per share (EPS) for the quarter, missing the consensus estimate of ($1.20) by ($0.09), Zacks reports. Ultragenyx Pharmaceutical had a negative net margin of 85.54% and a negative return on equity of 566.58%. The business had revenue of $207.00 million for the quarter, compared to the consensus estimate of $199.60 million. During the same quarter last year, the firm posted ($1.39) earnings per share. The business’s quarterly revenue was up 25.5% compared to the same quarter last year.

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

Shares of RARE opened at $20.52 on Friday. The firm has a market cap of $1.98 billion, a P/E ratio of -3.51 and a beta of 0.16. Ultragenyx Pharmaceutical has a 1 year low of $18.41 and a 1 year high of $46.27. The company has a 50 day simple moving average of $26.91 and a two-hundred day simple moving average of $29.73.

Trending Headlines about Ultragenyx Pharmaceutical

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

• Positive Sentiment: Q4 revenue beat and strong year-over-year growth — Ultragenyx reported $207M in Q4 revenue (up ~25% Y/Y) and 2025 total revenue of $673M, driven by Crysvita and Dojolvi sales, showing commercial momentum that supports the franchise. Ultragenyx Q4 Loss Wider Than Expected, Revenues Increase Y/Y
• Positive Sentiment: Pipeline upside remains a near-term catalyst — management highlighted upcoming potential product launches and an important gene‑therapy data readout that could materially accelerate commercial revenue if successful. Why Is Ultragenyx Stock Sinking Friday?
• Neutral Sentiment: Some analysts still rate RARE favorably — the stock carries an average analyst rating around “Moderate Buy,” which could temper further downside if pipeline readouts stay on track. Ultragenyx Receives Average Rating of “Moderate Buy”
• Negative Sentiment: EPS miss and weaker guidance — Q4 EPS missed estimates (loss of $1.29 vs. -$1.20 expected) and FY2026 revenue guidance ($730M–$760M) came in below Street forecasts, reducing near-term visibility on profitability. Ultragenyx (RARE) Reports Q4 Loss, Beats Revenue Estimates
• Negative Sentiment: Cost cuts and layoffs announced — management disclosed a strategic restructuring and ~10% workforce reduction as it pushes to reach profitability by 2027, signaling tougher near-term execution and one-time charges. Ultragenyx lays off 10% of workforce as FDA continues to stall gene therapy approval plans
• Negative Sentiment: Multiple securities-class-action notices filed — several law firms have announced class actions and investor alerts covering purchases from Aug 3, 2023 to Dec 26, 2025, creating legal overhang and potential liability/management distraction. RARE Investors Have Opportunity to Lead Ultragenyx Securities Fraud Lawsuit
• Negative Sentiment: Street price-target cuts and sell-side concern — reporters note multiple analyst target reductions and growing clinical/pipeline uncertainty, which pressurizes the stock until clearer clinical or commercial milestones are achieved. RARE stock slumps on multiple Wall Street price target slashes, clinical pipeline uncertainty

Analysts Set New Price Targets

Several equities research analysts have recently commented on RARE shares. Bank of America reduced their price objective on Ultragenyx Pharmaceutical from $72.00 to $58.00 and set a “buy” rating for the company in a research note on Friday, January 2nd. Weiss Ratings reissued a “sell (e+)” rating on shares of Ultragenyx Pharmaceutical in a report on Wednesday, January 21st. Barclays decreased their price objective on Ultragenyx Pharmaceutical from $81.00 to $50.00 and set an “overweight” rating on the stock in a research note on Monday, November 24th. Truist Financial lowered their price objective on Ultragenyx Pharmaceutical from $100.00 to $90.00 and set a “buy” rating on the stock in a research report on Wednesday, November 5th. Finally, Wells Fargo & Company reduced their target price on shares of Ultragenyx Pharmaceutical from $65.00 to $45.00 and set an “overweight” rating for the company in a report on Tuesday, December 30th. Sixteen research analysts have rated the stock with a Buy rating, one has assigned a Hold rating and one has given a Sell rating to the company’s stock. Based on data from MarketBeat, Ultragenyx Pharmaceutical currently has a consensus rating of “Moderate Buy” and a consensus target price of $72.29.

Insider Buying and Selling at Ultragenyx Pharmaceutical

In related news, CFO Howard Horn sold 3,081 shares of the company’s stock in a transaction on Wednesday, December 10th. The shares were sold at an average price of $36.38, for a total transaction of $112,086.78. Following the completion of the transaction, the chief financial officer owned 95,146 shares in the company, valued at approximately $3,461,411.48. This represents a 3.14% decrease in their ownership of the stock. The sale was disclosed in a legal filing with the Securities & Exchange Commission, which can be accessed through this hyperlink. Insiders have sold a total of 6,227 shares of company stock worth $187,126 in the last three months. 5.50% of the stock is currently owned by corporate insiders.

Institutional Trading of Ultragenyx Pharmaceutical

Several hedge funds have recently bought and sold shares of the stock. Jacobs Levy Equity Management Inc. grew its position in shares of Ultragenyx Pharmaceutical by 614.1% during the third quarter. Jacobs Levy Equity Management Inc. now owns 2,167,729 shares of the biopharmaceutical company’s stock valued at $65,205,000 after purchasing an additional 1,864,164 shares in the last quarter. Vestal Point Capital LP bought a new position in Ultragenyx Pharmaceutical during the third quarter valued at approximately $22,560,000. State Street Corp grew its holdings in Ultragenyx Pharmaceutical by 16.5% during the 4th quarter. State Street Corp now owns 4,303,046 shares of the biopharmaceutical company’s stock valued at $98,970,000 after buying an additional 610,559 shares in the last quarter. Schonfeld Strategic Advisors LLC grew its holdings in Ultragenyx Pharmaceutical by 101.8% during the 3rd quarter. Schonfeld Strategic Advisors LLC now owns 1,144,589 shares of the biopharmaceutical company’s stock valued at $34,429,000 after buying an additional 577,414 shares in the last quarter. Finally, HRT Financial LP bought a new stake in Ultragenyx Pharmaceutical in the 4th quarter worth approximately $11,538,000. Hedge funds and other institutional investors own 97.67% of the company’s stock.

Ultragenyx Pharmaceutical Company Profile

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.