Ultragenyx Pharmaceutical (NASDAQ:RARE) reported second-quarter 2026 revenue of $214 million, led by growth across its commercial rare-disease portfolio, while the company prepared for potential regulatory decisions on two gene therapies and a late-stage data readout in Angelman syndrome.
Chief Executive Officer and President Emil Kakkis said the quarter marked the company’s highest quarterly revenue to date and supported its reaffirmed full-year revenue guidance. He also said Ultragenyx sees a path to profitability in 2027 through continued product growth, potential contributions from upcoming launches, expense discipline and possible monetization of priority review vouchers.
Second-Quarter Financial Results
Total operating expenses were $289 million, including $34 million in cost of sales and $255 million in combined research and development and selling, general and administrative expenses. Operating expenses included $34 million of non-cash stock-based compensation.
Ultragenyx posted a net loss of $92 million, or $0.90 per share, for the quarter. As of June 30, the company had $436 million in cash equivalents and marketable securities. Net cash used in operations was $97 million, which Horn said was significantly lower than in the first quarter and in line with prior expectations.
The company reaffirmed its revenue guidance and its guidance for combined R&D and SG&A expenses. Horn said 2026 combined R&D and SG&A spending is expected to be flat to down low single digits from 2025, while 2027 spending is expected to decline by at least 15%, including planned investments for potential launches.
Commercial Portfolio Expands Patient Reach
Chief Commercial Officer Erik Harris said underlying demand remained strong despite seasonal factors that can affect quarterly ordering patterns. In Latin America, approximately 50 patients began commercial Crysvita therapy during the quarter, bringing the regional total to 1,000 patients. More than 100 patients in Turkey are receiving Crysvita through a named-patient program.
For DOJOLVI, Ultragenyx generated about 30 start forms in North America and had approximately 675 patients on reimbursed treatment. About 300 patients in Europe were receiving treatment through named-patient or early-access programs. The company also began treating patients in Japan after the therapy was listed on the country’s National Health Insurance Drug Price List.
Evkeeza was being provided to more than 500 patients in 25 countries outside the U.S., Harris said. He added that Ultragenyx’s commercial infrastructure is designed to support patient identification, reimbursement navigation and field execution across individual markets.
Gene Therapy Decisions Approach
Ultragenyx is awaiting FDA action on DTX-401, an investigational gene therapy for glycogen storage disease type Ia, with a Prescription Drug User Fee Act date of Aug. 23. The company’s second anticipated decision is for UX-111, a gene therapy for Sanfilippo syndrome type A, with a PDUFA date of Sept. 19.
Kakkis said DTX-401 clinical studies showed that treated patients could significantly reduce the amount and frequency of cornstarch dosing to a prespecified clinically meaningful degree. He said the therapy is designed to provide expression of the missing G6Pase enzyme and improve glucose regulation during fasting or metabolic stress.
For UX-111, Kakkis said clinical data showed reductions in heparan sulfate and stabilization or retention of cognitive function relative to natural history. He said earlier treatment appeared to produce better results, although patients across the age range studied showed benefits after treatment.
Harris said launch preparations for the two potential gene therapies were progressing, including work with qualified treatment centers and more than 200 payer engagements. He said the company already has commercial experience in relevant treatment settings, with approximately 75% overlap between institutions and healthcare providers involved with the potential gene therapies and those serving DOJOLVI and MEPSEVII patients.
During the question-and-answer session, Kakkis said the FDA review of DTX-401 had proceeded normally, with the company responding to information requests involving clinical and chemistry, manufacturing and controls matters. He said Ultragenyx could not predict the agency’s ultimate action.
Angelman Readout and Setrusumab Regulatory Path
Ultragenyx expects to report top-line Phase III ASPIRE data for GTX-102, also known as apazunersen, in Angelman syndrome during September or October. The randomized, double-blind, sham-controlled study enrolled patients with a full maternal UBE3A gene deletion.
Chief Medical Officer Eric Crombez said the study’s statistical alpha is split between the Bayley-4 cognitive raw score, which receives 80%, and the Multi-Domain Responder Index, or MDRI, which receives 20%. The endpoints are tested in parallel rather than hierarchically. A statistically successful study would require a Bayley endpoint p-value of 0.04 or less, or an MDRI p-value of 0.01 or less.
Kakkis said a five- to six-point change on the Bayley assessment would be clinically meaningful and that the study was powered to detect an average change in that range. He also said the company’s Phase I/II program has shown a consistent safety profile, and the Phase III study had not been stopped because of a safety issue.
Separately, Ultragenyx continues discussions with regulators regarding UX143, or setrusumab, for osteogenesis imperfecta. Crombez said neither of the Phase III ORBIT and COSMIC studies met their primary endpoints for annualized fracture reduction, though the studies showed improvements in bone mineral density and patient-reported outcomes, along with fracture reductions in certain bones and patient groups.
The U.K.’s Medicines and Healthcare products Regulatory Agency indicated that a new randomized study may be required before it would consider an application, according to Crombez. The FDA has been open to discussing alternative fracture analyses, but Ultragenyx said further discussions are needed to determine the additional clinical data required for a potential biologics license application.
About Ultragenyx Pharmaceutical (NASDAQ:RARE)
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.
