Ultragenyx Pharmaceutical (NASDAQ:RARE) issues quarterly earnings results, misses estimates by $0.06 EPS. Ultragenyx Pharmaceutical (NASDAQ:RARE - Get Free Report) issued its quarterly earnings data on Tuesday. The biopharmaceutical company reported ($1.16) EPS for the quarter, missing the consensus estimate of ($1.10) by ($0.06), FiscalAI reports. The company had revenue of $173.00 million for the quarter, compared to analyst estimates of $182.49 million. During the same period last year, the firm posted ($1.81) earnings per share. Ultragenyx Pharmaceutical's revenue was up 8.2% compared to the same quarter last year. Here are the key takeaways from Ultragenyx Pharmaceutical's conference call: * GENGLYCOS and FAYUVI received FDA approval and have begun launching, with strong early demand, 35 qualified treatment centers active, and the company on track to exceed 40 by year-end. * Ultragenyx reaffirmed its 2026 revenue guidance of $730 million-$760 million and expects to achieve full-year GAAP profitability and positive operating cash flow in 2027, supported by revenue growth and substantial cost reductions. * The company expects 2027 R&D expense below $350 million and SG&A below $375 million after reducing headcount by approximately 20%; it also secured roughly $350 million in net, non-dilutive proceeds from selling two priority review vouchers. * Disappointing phase III results led Ultragenyx to discontinue GTX-102 for Angelman syndrome, while UX701 for Wilson disease was terminated after failing to produce a sufficient biochemical response in most patients, contributing to significant workforce reductions. * Management remains encouraged by setrusumab activity in osteogenesis imperfecta and is discussing potential regulatory paths with the FDA, but has not yet determined whether another prospective trial will be required. Ultragenyx Pharmaceutical price performance. NASDAQ RARE traded down $0.12 on Wednesday, reaching $16.51. The company had a trading volume of 787,012 shares, compared to its average volume of 2,840,884. The firm has a market cap of $1.63 billion, a price-to-earnings ratio of -2.83 and a beta of 0.37. Ultragenyx Pharmaceutical has a fifty-two week low of $12.73 and a fifty-two week high of $39.89. The business has a 50 day moving average of $16.64 and a two-hundred day moving average of $22.81. Key headlines impacting Ultragenyx Pharmaceutical. Here are the key news stories impacting Ultragenyx Pharmaceutical this week: * Ultragenyx reaffirmed its 2026 revenue and 2027 profitability guidance. It expects expense and workforce reductions to cut 2027 R&D spending by at least 50% compared with 2025. * The company said it has agreements to sell two Priority Review Vouchers for a combined $413 million, providing a substantial source of cash. FDA approvals of GENGLYCOS and FAYUVI were also announced during the quarter. * Revenue rose 8.2% year over year to $173 million, including $109 million from CRYSVITA and $30 million from DOJOLVI. The quarterly loss narrowed to $1.16 per share from $1.81 a year earlier. * Despite year-over-year improvement, the $1.16 per-share loss was slightly wider than analysts' roughly $1.10-$1.15 expected loss, and revenue of $173 million fell short of the $182.5 million consensus estimate. Analysts set new price targets. Several brokerages have recently issued reports on RARE. Weiss Ratings reiterated a "sell (d-)" rating on shares of Ultragenyx Pharmaceutical in a report on Tuesday, October 6th. Cantor Fitzgerald boosted their price objective on Ultragenyx Pharmaceutical from $33.00 to $39.00 and gave the stock an "overweight" rating in a research report on Friday, September 18th. Wells Fargo & Company cut Ultragenyx Pharmaceutical from an "overweight" rating to an "equal weight" rating and lowered their target price for the stock from $50.00 to $18.00 in a report on Thursday, September 3rd. Bank of America cut Ultragenyx Pharmaceutical from a "buy" rating to a "neutral" rating in a report on Thursday, September 3rd. Finally, Jefferies Financial Group dropped their target price on Ultragenyx Pharmaceutical from $77.00 to $28.00 and set a "buy" rating for the company in a research report on Thursday, September 3rd. One research analyst has rated the stock with a Strong Buy rating, nine have given a Buy rating, eleven have given a Hold rating and one has assigned a Sell rating to the stock. According to MarketBeat, Ultragenyx Pharmaceutical has a consensus rating of "Hold" and a consensus price target of $33.60. Insider transactions at Ultragenyx Pharmaceutical. In other Ultragenyx Pharmaceutical news, CFO Howard Horn sold 7,066 shares of Ultragenyx Pharmaceutical stock in a transaction dated Thursday, October 15th. The stock was sold at an average price of $15.27, for a total transaction of $107,897.82. Following the transaction, the chief financial officer directly owned 89,229 shares of the company's stock, valued at approximately $1,362,526.83. The trade was a 7.34% decrease in their position. The sale was disclosed in a filing with the Securities & Exchange Commission, which can be accessed through this link. The sale was made to cover tax withholding obligations related to the vesting of equity awards. Over the last three months, insiders sold 8,510 shares of company stock worth $127,561. 5.20% of the stock is owned by company insiders. Ultragenyx Pharmaceutical company profile. Ultragenyx Pharmaceutical Inc is a biopharmaceutical company focused on developing and commercializing treatments for rare and ultra-rare diseases. The company concentrates on conditions with significant unmet medical needs, including genetic, metabolic and endocrine disorders, and uses biologics, small molecules and gene therapy approaches in its research and development programs. Ultragenyx's commercial portfolio includes Crysvita (burosumab), a treatment for X-linked hypophosphatemia and related disorders; Mepsevii (vestronidase alfa), an enzyme replacement therapy for mucopolysaccharidosis VII; and Dojolvi (triheptanoin), a therapy for long-chain fatty acid oxidation disorders. Receive News & Ratings for Ultragenyx Pharmaceutical Daily - Enter your email address below to receive a concise daily summary of the latest news and analysts' ratings for Ultragenyx Pharmaceutical and related companies with MarketBeat.com's FREE daily email newsletter.

Ultragenyx Pharmaceutical won two US gene therapy approvals in the third quarter but scrapped two development programmes and is cutting about 20% of its workforce, to roughly 1,000 employees. The Food and Drug Administration approved GENGLYCOS for glycogen storage disease type Ia in August and FAYUVI for Sanfilippo syndrome type A in September. They are the company's first commercial gene therapies and bring its approved medicines to six. Ultragenyx ended GTX-102 for Angelman syndrome after a failed phase III study, and terminated UX701 for Wilson disease. It reaffirmed 2026 revenue guidance of $730 million to $760 million, excluding the new launches. It expects GAAP profitability in 2027 and says it has enough cash, including about $350 million from selling two priority review vouchers, to get there without raising funds.

Ultragenyx gets first Euro nod for rare disease drug Dojolvi. Several years after it was approved in the US, Ultragenyx's therapy for long-chain fatty acid oxidation disorders (LC-FAOD), Dojolvi, has been greenlit in the UK. The Medicines and Healthcare products Regulatory Agency (MHRA) has granted a conditional marketing authorisation to Dojolvi, an oral liquid formulation of triheptanoin, to treat adult and paediatric patients with LC-FAOD. The UK approval is believed to be the first for the drug in Europe. LC-FAOD is a group of rare, inherited genetic metabolic conditions where the body cannot convert long-chain fatty acids into energy. That means that during periods of fasting, when the body switches to running on fat reserves, rather than carbohydrates, organs that need a lot of energy, like the brain, heart, and muscles, stop working properly. Moreover, unprocessed fats and their byproducts can accumulate in tissues and organs, causing toxicity. Management of the disorders currently relies on careful management of dietary fat intake, with frequent meals, avoiding prolonged fasting, low-fat, or controlled diets, and supplementation with medium-chain triglyceride (MCT) oil, which bypasses the defective LC-FA breakdown pathway and provides a safe, usable source of energy for the body. Dojolvi is a pharmaceutical-grade form of MCT, with important differences in composition that improve its efficacy, and is taken daily as part of a carefully controlled diet. MCT oils can become less effective over time and sometimes fail to prevent severe metabolic crises like cardiomyopathy. "Enabling patient access to high-quality, safe, and effective medical products is important to us," said MHRA's executive director of operations, Julian Beach. "Patients living with rare diseases often face significant barriers in accessing effective treatments, and we're assured that the appropriate regulatory standards for the approval of this medicine have been met," he added. "As with all products, we will keep the safety of triheptanoin under close review." Patients with LC-FAOD hoping for access to Dojolvi will still have to wait for the conclusion of an appraisal by reimbursement authority NICE, unless they are able to pay for a private prescription. An update on the NICE process, posted to the agency's website in August, said the appraisal is "anticipated to begin in October 2027." Dojolvi was approved in the US in 2020 and was also launched in Japan earlier this year. Ultragenyx recorded sales of $45 million in the first half of this year, and has said it expects full-year revenues from the drug to reach $100 million to $110 million. 2 November, 2026