Ultragenyx Pharmaceutical (NASDAQ:RARE) Posts Earnings Results, Beats Estimates By $0.32 EPS

Key Points

• Ultragenyx beat earnings expectations: The company reported a quarterly loss of $0.90 per share, $0.32 better than the consensus estimate of a $1.22 loss. Revenue rose 28.5% year over year to a record $214 million, led by Crysvita, Dojolvi, Evkeeza and Mepsevii.
• Several major pipeline catalysts are approaching: The FDA is expected to decide on gene therapies DTX-401 and UX-111 in August and September, while Phase III GTX-102 results for Angelman syndrome are anticipated in September or October.
• Risks remain around Setrusumab: The therapy’s Phase III trials missed their primary fracture-reduction endpoints, leaving its regulatory path uncertain and potentially requiring additional clinical data or a new randomized study.

Ultragenyx Pharmaceutical (NASDAQ:RARE - Get Free Report) released its quarterly earnings results on Tuesday. The biopharmaceutical company reported ($0.90) earnings per share for the quarter, topping the consensus estimate of ($1.22) by $0.32, Zacks reports. Ultragenyx Pharmaceutical had a negative return on equity of 1,024.42% and a negative net margin of 91.03%.The business's revenue was up 28.5% on a year-over-year basis. During the same quarter in the previous year, the company posted ($1.17) earnings per share.

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

• Record quarterly revenue reached $214 million, up from contributions across Crysvita ($156 million), DOJOLVI ($27 million), Evkeeza ($21 million, up 50% year over year), and MEPSEVII ($10 million). Management reaffirmed full-year revenue and operating-expense guidance.
• Ultragenyx is preparing for potential FDA decisions on its first two gene therapies: DTX-401 for GSDIa on August 23 and UX-111 for Sanfilippo syndrome type A on September 19. The company said payer engagement, Qualified Treatment Center contracting, and launch preparations are progressing as planned.
• The Phase III ASPIRE trial of GTX-102 for Angelman syndrome remains on track for top-line results in September or October. Management said either the Bayley-4 cognition endpoint or the clinically meaningful Multi-Domain Responder Index could independently establish statistical success, although the results remain uncertain.
• Management reiterated a path to profitability in 2027, supported by double-digit growth from existing products, potential gene-therapy launches, expense discipline, and possible non-dilutive proceeds from monetizing priority review vouchers. The company ended the quarter with $436 million in cash and marketable securities.
• Setrusumab’s regulatory path remains uncertain after Phase III studies failed to meet their primary fracture-reduction endpoints. The FDA is considering alternative fracture analyses, but additional clinical data may be required, and the U.K. regulator indicated that a new randomized study could be necessary.

Ultragenyx Pharmaceutical Trading Up 3.2%

NASDAQ RARE traded up $0.79 during midday trading on Tuesday, hitting $25.81. The company had a trading volume of 2,103,532 shares, compared to its average volume of 2,228,654. The firm has a 50-day moving average price of $27.71 and a 200-day moving average price of $24.74. The company has a market capitalization of $2.54 billion, a PE ratio of -4.22 and a beta of 0.35. Ultragenyx Pharmaceutical has a twelve month low of $18.29 and a twelve month high of $39.89.

Wall Street Analyst Weigh In

RARE has been the topic of a number of research reports. Wells Fargo & Company increased their price objective on Ultragenyx Pharmaceutical from $45.00 to $47.00 and gave the stock an "overweight" rating in a research report on Wednesday, May 6th. Wedbush reduced their target price on shares of Ultragenyx Pharmaceutical from $27.00 to $26.00 and set a "neutral" rating for the company in a research report on Wednesday, May 6th. Wall Street Zen upgraded shares of Ultragenyx Pharmaceutical from a "strong sell" rating to a "sell" rating in a research note on Saturday, June 6th. Morgan Stanley raised their price target on shares of Ultragenyx Pharmaceutical from $50.00 to $67.00 and gave the stock an "overweight" rating in a research report on Thursday, April 16th. Finally, Barclays dropped their price objective on shares of Ultragenyx Pharmaceutical from $44.00 to $43.00 and set an "overweight" rating for the company in a research note on Wednesday, April 29th. One investment analyst has rated the stock with a Strong Buy rating, fourteen have given a Buy rating, two have issued a Hold rating and one has assigned a Sell rating to the stock. According to MarketBeat, Ultragenyx Pharmaceutical presently has an average rating of "Moderate Buy" and a consensus target price of $57.94.

Insider Buying and Selling

In related news, EVP Karah Herdman Parschauer sold 1,899 shares of the business's stock in a transaction dated Monday, June 15th. The stock was sold at an average price of $24.62, for a total value of $46,753.38. Following the completion of the transaction, the executive vice president owned 94,462 shares of the company's stock, valued at $2,325,654.44. The trade was a 1.97% decrease in their ownership of the stock. The transaction was disclosed in a document filed with the Securities & Exchange Commission, which is available through this link. Also, Director Corazon (Corsee) D. Sanders sold 2,000 shares of the company's stock in a transaction dated Monday, June 15th. The stock was sold at an average price of $25.05, for a total transaction of $50,100.00. Following the sale, the director directly owned 21,095 shares of the company's stock, valued at $528,429.75. This represents a 8.66% decrease in their position. The SEC filing for this sale provides additional information. In the last 90 days, insiders sold 14,292 shares of company stock valued at $351,644. Company insiders own 5.20% of the company's stock.

Institutional Trading of Ultragenyx Pharmaceutical

Large investors have recently made changes to their positions in the company. CANADA LIFE ASSURANCE Co lifted its position in shares of Ultragenyx Pharmaceutical by 2.0% during the 3rd quarter. CANADA LIFE ASSURANCE Co now owns 26,932 shares of the biopharmaceutical company's stock worth $817,000 after buying an additional 534 shares during the last quarter. Van ECK Associates Corp grew its position in Ultragenyx Pharmaceutical by 26.1% in the third quarter. Van ECK Associates Corp now owns 2,650 shares of the biopharmaceutical company's stock valued at $80,000 after acquiring an additional 549 shares during the last quarter. Tidal Investments LLC grew its position in Ultragenyx Pharmaceutical by 4.7% in the second quarter. Tidal Investments LLC now owns 12,591 shares of the biopharmaceutical company's stock valued at $458,000 after acquiring an additional 564 shares during the last quarter. Rafferty Asset Management LLC raised its stake in Ultragenyx Pharmaceutical by 0.4% during the fourth quarter. Rafferty Asset Management LLC now owns 157,061 shares of the biopharmaceutical company's stock worth $3,612,000 after acquiring an additional 577 shares in the last quarter. Finally, Smartleaf Asset Management LLC raised its stake 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 acquiring an additional 751 shares in the last quarter. Institutional investors and hedge funds 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.

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