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

Key Points

• Ultragenyx reported EPS of -$1.84, missing estimates by $0.34; Q1 revenue was $136M but heavy spending (operating expenses $305M, net loss $185M, and $197M cash used in operations) left $534M in cash, signaling near‑term cash‑burn pressure.
• The company reaffirmed full‑year 2026 revenue guidance of $730–$760 million and remains on a stated pathway to profitability in 2027.
• Key pipeline and regulatory catalysts: positive long‑term Phase I/II data for GTX‑102 in Angelman syndrome with the Phase III ASPIRE unblind due in H2 2026, and two gene‑therapy BLAs—DTX‑401 PDUFA Aug 23, 2026 and UX‑111 PDUFA Sept 19, 2026—with manufacturing and launch readiness underway.

Ultragenyx Pharmaceutical (NASDAQ:RARE - Get Free Report) announced its quarterly earnings data on Tuesday. The biopharmaceutical company reported ($1.84) EPS for the quarter, missing the consensus estimate 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 in the previous year, the company earned ($1.57) EPS. The business's quarterly revenue was down 2.2% on a year-over-year basis.

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 Up 0.8%

Shares of RARE traded up $0.20 during trading hours on Tuesday, reaching $25.01. The stock had a trading volume of 1,481,031 shares, compared to its average volume of 2,242,071. Ultragenyx Pharmaceutical has a 52 week low of $18.29 and a 52 week high of $42.37. The company has a market cap of $2.46 billion, a P/E ratio of -4.28 and a beta of 0.38. The firm has a 50-day moving average of $22.55 and a 200 day moving average of $26.90.

Insiders Place Their Bets

In other Ultragenyx Pharmaceutical 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 transaction, the executive vice president owned 89,515 shares of the company's stock, 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 filing with the SEC, which is available through the SEC website. Also, EVP Karah Herdman Parschauer sold 8,135 shares of the stock in a transaction on Monday, March 2nd. The stock was sold at an average price of $22.80, for a total transaction of $185,478.00. Following the sale, the executive vice president directly owned 76,346 shares of the company's stock, valued at $1,740,688.80. This represents a 9.63% decrease in their position. Additional details regarding this sale are available in the official SEC disclosure. Insiders sold 111,178 shares of company stock worth $2,534,628 in the last three months. 5.20% of the stock is owned by corporate insiders.

Hedge Funds Weigh In On Ultragenyx Pharmaceutical

Several hedge funds have recently made changes to their positions in the business. Danske Bank A S acquired a new position in Ultragenyx Pharmaceutical during the third quarter worth $39,000. Aster Capital Management DIFC Ltd bought a new position in shares of Ultragenyx Pharmaceutical in the fourth quarter valued at $56,000. Smartleaf Asset Management LLC increased its holdings in Ultragenyx Pharmaceutical by 43.2% during the fourth quarter. Smartleaf Asset Management LLC now owns 2,489 shares of the biopharmaceutical company's stock worth $57,000 after buying an additional 751 shares during the last quarter. Van ECK Associates Corp lifted its stake in Ultragenyx Pharmaceutical by 26.1% in the 3rd quarter. Van ECK Associates Corp now owns 2,650 shares of the biopharmaceutical company's stock valued at $80,000 after purchasing an additional 549 shares during the last quarter. Finally, Empowered Funds LLC bought a new stake in Ultragenyx Pharmaceutical in the fourth quarter valued at approximately $66,000. 97.67% of the stock is owned by institutional investors.

Analysts Set New Price Targets

RARE has been the topic of a number of recent research reports. Wall Street Zen raised Ultragenyx Pharmaceutical from a "sell" rating to a "hold" rating in a research report on Sunday. The Goldman Sachs Group lowered Ultragenyx Pharmaceutical from a "buy" rating to a "neutral" rating and dropped their target price for the stock from $61.00 to $25.00 in a report on Tuesday, March 24th. Canaccord Genuity Group dropped their price objective on shares of Ultragenyx Pharmaceutical from $128.00 to $79.00 and set a "buy" rating on the stock in a research note on Wednesday, February 18th. Wedbush reiterated a "neutral" rating and issued a $25.00 target price on shares of Ultragenyx Pharmaceutical in a research note on Tuesday, February 17th. Finally, Barclays dropped their price target on shares of Ultragenyx Pharmaceutical from $44.00 to $43.00 and set an "overweight" rating on the stock in a research report on Wednesday, April 29th. One equities research analyst has rated the stock with a Strong Buy rating, fourteen have issued a Buy rating, two have issued a Hold rating and one has given a Sell rating to the company. According to data from MarketBeat, the company presently has a consensus rating of "Moderate Buy" and an average price target of $60.76.

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.