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

Ultragenyx Pharmaceutical (NASDAQ:RARE) announced its earnings results on Thursday. The biopharmaceutical company reported ($1.29) EPS for the quarter, missing analysts’ consensus estimates of ($1.20) by ($0.09), Zacks reports. The business 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 business’s revenue was up 25.5% compared to the same quarter last year. During the same period in the previous year, the firm earned ($1.39) earnings per share.

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 Stock Performance

Ultragenyx Pharmaceutical stock opened at $23.50 on Friday. Ultragenyx Pharmaceutical has a one year low of $18.41 and a one year high of $46.27. The business has a fifty day moving average of $27.23 and a two-hundred day moving average of $29.78. The company has a market cap of $2.27 billion, a P/E ratio of -3.96 and a beta of 0.16.

Insider Buying and Selling at Ultragenyx Pharmaceutical

In related news, CFO Howard Horn sold 3,081 shares of the business’s stock in a transaction dated Wednesday, December 10th. The shares were sold at an average price of $36.38, for a total transaction of $112,086.78. Following the sale, the chief financial officer owned 95,146 shares of the company’s stock, valued at approximately $3,461,411.48. The trade was a 3.14% decrease in their ownership of the stock. The transaction was disclosed in a filing with the Securities & Exchange Commission, which is available at this link. Over the last 90 days, insiders have sold 6,227 shares of company stock worth $187,126. Corporate insiders own 5.50% of the company’s stock.

Institutional Investors Weigh In On Ultragenyx Pharmaceutical

Institutional investors have recently modified their holdings of the company. American Century Companies Inc. acquired a new position in Ultragenyx Pharmaceutical during the second quarter worth about $366,000. Cerity Partners LLC purchased a new position in shares of Ultragenyx Pharmaceutical during the 4th quarter worth approximately $361,000. Mercer Global Advisors Inc. ADV acquired a new position in shares of Ultragenyx Pharmaceutical during the 3rd quarter worth approximately $359,000. Xponance LLC lifted its stake in Ultragenyx Pharmaceutical by 6.6% in the 4th quarter. Xponance LLC now owns 14,390 shares of the biopharmaceutical company’s stock valued at $331,000 after buying an additional 896 shares in the last quarter. Finally, E Fund Management Co. Ltd. grew its holdings in Ultragenyx Pharmaceutical by 9.1% during the 2nd quarter. E Fund Management Co. Ltd. now owns 9,052 shares of the biopharmaceutical company’s stock valued at $329,000 after buying an additional 752 shares during the last quarter. Hedge funds and other institutional investors own 97.67% of the company’s stock.

Wall Street Analysts Forecast Growth

Several equities research analysts have recently weighed in on the stock. HC Wainwright reiterated a “buy” rating and set a $60.00 target price on shares of Ultragenyx Pharmaceutical in a research note on Tuesday, February 3rd. Truist Financial decreased their price objective on Ultragenyx Pharmaceutical from $100.00 to $90.00 and set a “buy” rating for the company in a research report on Wednesday, November 5th. Guggenheim dropped their target price on Ultragenyx Pharmaceutical from $64.00 to $52.00 and set a “buy” rating on the stock in a report on Monday, January 5th. Wells Fargo & Company decreased their price target on Ultragenyx Pharmaceutical from $65.00 to $45.00 and set an “overweight” rating for the company in a report on Tuesday, December 30th. Finally, Bank of America dropped their price objective on shares of Ultragenyx Pharmaceutical from $72.00 to $58.00 and set a “buy” rating on the stock in a research note on Friday, January 2nd. Sixteen equities 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 stock. Based on data from MarketBeat.com, the company presently has a consensus rating of “Moderate Buy” and an average target price of $72.29.

Key Headlines Impacting Ultragenyx Pharmaceutical

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

• Positive Sentiment: Q4 results beat on revenue and roughly in line on EPS — revenue $207M vs $199.6M est; EPS loss of $1.29 vs -1.30 est; revenue up ~25.5% YoY, signaling underlying product sales strength. Ultragenyx Q4 Earnings Call Highlights
• Positive Sentiment: 2025 product performance: total revenue $673M with Crysvita® $481M and Dojolvi® $96M — recurring product revenue provides a stable base while pipeline assets mature. Ultragenyx Q4 & Full-Year 2025 Results
• Positive Sentiment: Investors cite potential upside from gene‑therapy programs that could offset recent drug setbacks over the medium term — pipeline optimism remains a driver for longer‑term upside. Investing: Gene therapy hopes offset drug flop
• Positive Sentiment: Analyst consensus is constructive — recent coverage shows an average “Moderate Buy” rating, which supports share upside if guidance concerns abate. Analyst Ratings Summary
• Neutral Sentiment: Analyst write‑ups and metric reviews highlight the beat but remind investors Ultragenyx remains loss‑making with negative margins and ROE; watch cadence of product sales and pipeline catalysts. Zacks: Q4 metrics analysis
• Negative Sentiment: FY2026 revenue guidance missed Street expectations — management guided $730–760M vs consensus ~$785M; the guidance shortfall is the primary near‑term driver of the share decline. Investing: Guidance misses expectations
• Negative Sentiment: Multiple securities class‑action filings and investor alerts from several law firms allege misstatements related to company disclosures (lead plaintiff deadline April 6, 2026). The proliferation of lawsuits increases legal risk and could pressure the stock or distract management. PR Newswire: Investor Alert on Class Action
• Negative Sentiment: Several additional law firms (Rosen, Pomerantz, Levi & Korsinsky, Kessler Topaz, etc.) are soliciting lead plaintiffs and filing notices — amplifies headline risk and potential future litigation costs. Newsfile: Rosen shareholder alert

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.