Ultragenyx Pharmaceutical (NASDAQ:RARE) Releases Quarterly Earnings Results, Misses Estimates By $0.34 EPS

Ultragenyx Pharmaceutical (NASDAQ:RARE) announced its quarterly earnings results on Tuesday. The biopharmaceutical company reported ($1.84) earnings per share (EPS) for the quarter, missing the consensus estimate of ($1.50) by ($0.34), Zacks reports. Ultragenyx Pharmaceutical had a negative net margin of 91.03% and a negative return on equity of 2,272.48%. The firm had revenue of $136.00 million for the quarter, compared to analyst estimates of $158.38 million. During the same period last year, the firm posted ($1.57) EPS. The business’s revenue 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 Stock Performance

NASDAQ RARE traded up $0.13 on Thursday, reaching $26.06. The company’s stock had a trading volume of 1,185,704 shares, compared to its average volume of 2,241,768. Ultragenyx Pharmaceutical has a twelve month low of $18.29 and a twelve month high of $42.37. The stock has a market cap of $2.56 billion, a PE ratio of -4.26 and a beta of 0.39. The firm’s 50 day moving average is $22.61 and its two-hundred day moving average is $26.84.

Insider Buying and Selling

In related news, EVP Eric Crombez sold 7,029 shares of the business’s 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 $160,261.20. Following the sale, the executive vice president directly owned 76,301 shares in the company, valued at $1,739,662.80. The trade was a 8.44% decrease in their position. The transaction was disclosed in a document filed with the Securities & Exchange Commission, which is available through this link. Also, CFO Howard Horn sold 4,683 shares of the business’s stock in a transaction that occurred on Friday, May 1st. The stock was sold at an average price of $24.90, for a total transaction of $116,606.70. Following the completion of the sale, the chief financial officer owned 110,342 shares in the company, valued at $2,747,515.80. The trade was a 4.07% decrease in their ownership of the stock. Additional details regarding this sale are available in the official SEC disclosure. Insiders sold 111,522 shares of company stock worth $2,543,214 in the last ninety days. Insiders own 5.20% of the company’s stock.

Institutional Trading of Ultragenyx Pharmaceutical

A number of hedge funds and other institutional investors have recently made changes to their positions in the business. American Century Companies Inc. acquired a new position in shares of Ultragenyx Pharmaceutical in the 2nd quarter valued at $366,000. Mercer Global Advisors Inc. ADV acquired a new position in shares of Ultragenyx Pharmaceutical in the 3rd quarter valued at $359,000. Xponance LLC boosted its position in shares of 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 purchasing an additional 896 shares during the period. ADAR1 Capital Management LLC acquired a new position in shares of Ultragenyx Pharmaceutical in the 3rd quarter valued at $271,000. Finally, Mariner LLC acquired a new position in shares of Ultragenyx Pharmaceutical in the 4th quarter valued at $236,000. 97.67% of the stock is currently owned by institutional investors.

Wall Street Analysts Forecast Growth

A number of research analysts have issued reports on the company. Wells Fargo & Company boosted their price target on Ultragenyx Pharmaceutical from $45.00 to $47.00 and gave the stock an “overweight” rating in a research report on Wednesday. Wall Street Zen upgraded Ultragenyx Pharmaceutical from a “sell” rating to a “hold” rating in a research report on Sunday. Morgan Stanley boosted their price target on Ultragenyx Pharmaceutical from $50.00 to $67.00 and gave the stock an “overweight” rating in a research report on Thursday, April 16th. The Goldman Sachs Group lowered Ultragenyx Pharmaceutical from a “buy” rating to a “neutral” rating and reduced their price target for the stock from $61.00 to $25.00 in a research report on Tuesday, March 24th. Finally, Barclays reduced their price target on Ultragenyx Pharmaceutical from $44.00 to $43.00 and set an “overweight” rating for the company in a research report on Wednesday, April 29th. 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 assigned a Sell rating to the company’s stock. According to MarketBeat, the company presently has an average rating of “Moderate Buy” and an average target price of $60.94.

More Ultragenyx Pharmaceutical News

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

• Positive Sentiment: Wells Fargo raised its price target to $47 and kept an overweight rating, signaling strong analyst conviction on Ultragenyx’s longer-term upside. Ultragenyx Given New $47.00 Price Target at Wells Fargo
• Positive Sentiment: Ultragenyx will present at Bank of America’s 2026 Healthcare Conference (May 12), giving management an opportunity to discuss commercial performance, outlook and pipeline details directly with investors. Ultragenyx to Participate at Bank of America’s 2026 Healthcare Conference
• Positive Sentiment: Coverage highlighting Ultragenyx’s Rare Bootcamp shows strong patient/advocate engagement and positions the company favorably in the rare-disease community, supporting long-term franchise value. Rare disease families find roadmap to drug development at bootcamps
• Neutral Sentiment: An insider sold 344 shares to cover tax withholding on vested awards; the sale is small relative to total insider holdings and was disclosed as routine. SEC filing: Insider Sale
• Negative Sentiment: Q1 results missed expectations: GAAP EPS of ($1.84) vs. consensus (~$1.50 loss) and revenue $136M vs. ~$158M expected; Crysvita sales showed seasonal weakness while Evkeeza and Dojolvi partially offset declines. This earnings miss and revenue shortfall are the primary near-term negative catalyst. Ultragenyx Reports First-Quarter 2026 Financial Results and Corporate Update
• Negative Sentiment: Wedbush trimmed its price target to $26 and set a neutral rating, reflecting more cautious near-term views and providing downside pressure relative to bullish calls. Ultragenyx Price Target Lowered to $26.00 at Wedbush

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.