Ultragenyx Pharmaceutical (NASDAQ:RARE) Announces Earnings Results

05 May 2026, 22:02 by Doug Wharley · The Cerbat Gem

Ultragenyx Pharmaceutical (NASDAQ:RARE – Get Free Report) announced its quarterly earnings results on Tuesday. The biopharmaceutical company reported ($1.84) earnings per share for the quarter, missing analysts’ consensus estimates of ($1.50) by ($0.34), Zacks reports. Ultragenyx Pharmaceutical had a negative net margin of 85.54% and a negative return on equity of 1,024.42%. During the same quarter last year, the firm earned ($1.57) EPS. The firm’s revenue for the quarter was down 2.2% compared to the same quarter last year.

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

Ultragenyx reaffirmed full-year 2026 revenue guidance of $730–$760 million, expects to exceed $700 million from its global commercial business and remains on a stated pathway to profitability in 2027.
The company highlighted long‑term phase I/II data for GTX‑102 in Angelman syndrome (66 patients on therapy, average ~3 years) showing a ~10‑point mean improvement on Bayley‑4 cognition at 12 months and an MDRI p‑value 0.0001, with the phase III ASPIRE unblind expected in the second half of 2026.
Two gene‑therapy BLAs are on regulatory timelines—DTX‑401 PDUFA Aug 23, 2026 and UX‑111 PDUFA Sept 19, 2026—and the company says manufacturing and launch readiness (including runs and inventory in its Bedford, MA facility) are underway.
Q1 results show $136 million revenue but heavy spending: total operating expenses of $305 million (including $30M SBC and $30M restructuring), a net loss of $185 million, and $197 million net cash used in operations, leaving $534 million in cash and securities—near‑term cash burn and one‑time costs could pressure the balance sheet until cash use declines.
Commercial momentum across existing rare‑disease products remains solid—CRYSVITA $93 million in Q1 (NA, LATAM/Turkey, Europe split), DOJOLVI $18 million with >675 patients in NA, and EVKEEZA $18 million (64% growth YoY), with sales in 35+ countries forming the base the company expects to scale with upcoming launches.

Ultragenyx Pharmaceutical Price Performance

Shares of RARE stock traded up $0.20 during trading on Tuesday, reaching $25.01. The company’s stock had a trading volume of 1,481,031 shares, compared to its average volume of 1,660,688. The company has a market capitalization of $2.46 billion, a price-to-earnings ratio of -4.28 and a beta of 0.39. The stock’s 50-day moving average price is $22.55 and its 200 day moving average price is $26.90. Ultragenyx Pharmaceutical has a 1-year low of $18.29 and a 1-year high of $42.37.

Insider Transactions at Ultragenyx Pharmaceutical

In related news, EVP Erik Harris sold 10,539 shares of Ultragenyx Pharmaceutical stock in a transaction that occurred on Monday, March 2nd. The shares were sold at an average price of $22.80, for a total value of $240,289.20. Following the completion of the transaction, the executive vice president owned 89,515 shares in the company, valued at approximately $2,040,942. The trade was a 10.53% decrease in their ownership of the stock. The sale was disclosed in a document filed with the SEC, which is accessible through this hyperlink. Also, CFO Howard Horn sold 8,409 shares of Ultragenyx Pharmaceutical stock in a transaction that occurred on Monday, March 2nd. The shares were sold at an average price of $22.70, for a total transaction of $190,884.30. Following the completion of the transaction, the chief financial officer owned 86,669 shares of the company’s stock, valued at $1,967,386.30. This trade represents a 8.84% decrease in their position. The disclosure for this sale is available in the SEC filing. In the last ninety days, insiders have sold 111,178 shares of company stock valued at $2,534,628. 5.20% of the stock is owned by corporate insiders.

Institutional Inflows and Outflows

Institutional investors and hedge funds have recently added to or reduced their stakes in the company. Mercer Global Advisors Inc. ADV acquired a new position in Ultragenyx Pharmaceutical during the 3rd quarter worth approximately $359,000. 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 worth $329,000 after acquiring an additional 752 shares in the last quarter. Brevan Howard Capital Management LP acquired a new position in Ultragenyx Pharmaceutical during the 2nd quarter worth approximately $297,000. ADAR1 Capital Management LLC acquired a new position in Ultragenyx Pharmaceutical during the 3rd quarter worth approximately $271,000. Finally, Mariner LLC acquired a new position in Ultragenyx Pharmaceutical during the 4th quarter worth approximately $236,000. Institutional investors and hedge funds own 97.67% of the company’s stock.

Wall Street Analyst Weigh In

A number of equities research analysts have weighed in on RARE shares. Morgan Stanley lifted their price target on shares of Ultragenyx Pharmaceutical from $50.00 to $67.00 and gave the company an “overweight” rating in a research report on Thursday, April 16th. Wall Street Zen upgraded shares of Ultragenyx Pharmaceutical from a “sell” rating to a “hold” rating in a research report on Sunday. HC Wainwright dropped their price target on shares of Ultragenyx Pharmaceutical from $60.00 to $50.00 and set a “buy” rating for the company in a research report on Tuesday, February 17th. JPMorgan Chase & Co. dropped their price target on shares of Ultragenyx Pharmaceutical from $120.00 to $74.00 and set an “overweight” rating for the company in a research report on Thursday, March 12th. Finally, Wedbush reiterated a “neutral” rating and set a $25.00 price target on shares of Ultragenyx Pharmaceutical in a research report on Tuesday, February 17th. One investment analyst has rated the stock with a Strong Buy rating, fourteen have given a Buy rating, two have given a Hold rating and one has given a Sell rating to the company. According to MarketBeat, Ultragenyx Pharmaceutical currently has a consensus rating of “Moderate Buy” and a consensus target price of $60.76.

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About Ultragenyx Pharmaceutical

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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.