Ultragenyx Pharmaceutical NASDAQ: RARE executives said the company is positioned for what Chief Executive Officer Emil Kakkis described as a “transformative” year, pointing to expectations for growing revenue, potential approvals for two gene therapies, and a key late-stage readout for its Angelman syndrome program.
Speaking on the company’s first-quarter 2026 earnings call, Kakkis said Ultragenyx is “on track to well exceed $700 million in revenue from our global commercial business,” supported by “a consistent track record of double-digit annual revenue growth.” He also highlighted ongoing manufacturing of gene therapy products at the company’s facility in Bedford, Massachusetts.
First-quarter revenue totals $136 million
Chief Financial Officer Howard Horn reported total revenue of $136 million for the first quarter of 2026.
- CRYSVITA revenue was $93 million, including $39 million from North America, $46 million from Latin America and Turkey, and $8 million from Europe.
- DOJOLVI revenue was $18 million.
- EVKEEZA revenue was $18 million, which Horn said represented 64% growth versus the first quarter of 2025 as demand built in Ultragenyx territories outside the U.S.
- MEPSEVII revenue was $7 million.
Total operating expenses were $305 million, including $30 million in cost of sales and $275 million in combined R&D and SG&A. Horn said operating expenses included $30 million of non-cash stock-based compensation and $30 million related to a restructuring announced in the prior quarter.
Ultragenyx posted a net loss of $185 million, or $1.84 per share. The company ended the quarter with $534 million in cash, equivalents, and marketable securities. Net cash used in operations was $197 million, which Horn attributed in part to typical first-quarter items such as annual bonus payments, along with $38 million in UX143 manufacturing payments and $5 million tied to severance and other reduction-in-force costs.
Ultragenyx reaffirms 2026 guidance
Horn said the company is reaffirming its 2026 revenue outlook. Ultragenyx expects total 2026 revenue of $730 million to $760 million, representing 8% to 13% growth over 2025 and excluding any potential revenue from new product launches.
By product, the company reiterated:
- CRYSVITA revenue of $500 million to $520 million, reflecting “growing underlying global demand,” partially offset by ordering patterns in Brazil that Horn said the company anticipates will normalize in 2027.
- DOJOLVI revenue of $100 million to $110 million.
Ultragenyx also reaffirmed expense expectations, with Horn saying 2026 combined R&D and SG&A expenses are expected to be “flat to down low single digits” versus 2025, and that 2027 combined R&D and SG&A expenses are expected to decrease at least 15% versus 2025.
Commercial update highlights CRYSVITA demand and DOJOLVI momentum
Chief Commercial Officer Erik Harris said demand for Ultragenyx’s marketed products continues to grow and that the company remains “fully confident” in its 2026 revenue guidance.
On CRYSVITA, Harris said first-quarter revenue across North America, Latin America, and Turkey was consistent with expected ordering patterns and historical trends. He cited continued strength in underlying demand and noted quarter-to-quarter variability in markets such as Brazil due to bulk orders by the Ministry of Health. Harris also said approximately 30 patients began commercial therapy in Latin America during the quarter, bringing the region to more than 950 patients on CRYSVITA.
On DOJOLVI, Harris described continued steady growth “six years post-approval.” He said the North American team generated more than 30 start forms in the first quarter, “far exceeding” the team’s target. Harris said there are now more than 675 North American patients on reimbursed therapy, and in Europe approximately 300 are treated through named patient or early access programs. He also said the “next stage of growth is likely to come from Japan,” where DOJOLVI received conditional approval last year and the company expects a full approval and launch “this quarter.”
For EVKEEZA, Harris said the product is seeing “exceptional growth” outside the U.S. as the company works through country-by-country reimbursement and early access requests. He said approximately 370 patients across 18 countries are receiving EVKEEZA.
Harris said the company’s global commercial infrastructure is a key advantage as it prepares for potential launches of two gene therapies: DTX401 (PDUFA date Aug. 23, 2026) and UX111 (PDUFA date Sept. 19, 2026).
GTX-102: long-term Phase I/II data and Phase III Aspire readout expected in 2H 2026
Ultragenyx spent much of the call discussing GTX-102, an antisense oligonucleotide for Angelman syndrome. Kakkis said the first patients in the Phase III Aspire study have reached the day 338 visit and transitioned to the open-label extension, with remaining patients expected to complete the blinded portion “in the next few months.” He said Aspire topline data remains on track for the second half of 2026, though he declined to narrow the timing further, citing the work involved in closing out an international sham-controlled study.
Chief Medical Officer Eric Crombez reviewed longer-term data from the company’s Phase I/II open-label, single-arm program, while cautioning that these results are “not necessarily predictive” of Phase III outcomes due to differences in study design.
Crombez said 74 patients have been treated across the Phase I/II program, with 66 patients currently in long-term extension. Patients are generally receiving a 14-milligram quarterly intrathecal maintenance dose, and have been on therapy for an average of three years, with some approaching five years. He said patients continue to show improvement across multiple developmental domains, and that there have been “no new cases of transient lower extremity weakness.”
On efficacy measures, Crombez said that among 53 patients with Bayley-4 cognitive raw score data at month 12, the mean change from baseline was approaching 10 points, exceeding what he called a meaningful difference of six points. He also discussed the Multi-Domain Responder Index (MDRI), which evaluates response across cognition, communication, behavior, sleep, and gross motor using clinically meaningful score differences consistent with FDA guidance. Crombez said that for the Phase I/II dataset, MDRI comparisons to baseline at months 12, 24, and 36 yielded a P value less than 0.0001 across those time points.
In Aspire, Crombez said the primary statistical alpha is split between Bayley-4 cognitive raw score and MDRI and will be tested in parallel. He said the study would be statistically successful if Bayley cognition is less than 0.04 or MDRI is less than 0.01. Kakkis told analysts the company views a positive MDRI result without Bayley-4 success as still supporting efficacy, reflecting a design the company said it negotiated with FDA.
During Q&A, Crombez said randomization in Aspire is stratified by age and cognitive raw score. He also said Aspire includes patients with full deletions to reduce variability, while the separate Aurora study is intended to expand treatment to other ages and genotypes and is still enrolling.
Executives also addressed assessment methods, noting that Phase III Bayley-4 cognition analyses will exclude caregiver input at FDA request. Crombez said Phase I/II used caregiver input, but the company believes removing those items does not meaningfully affect cognitive raw scores.
Upcoming PDUFAs, manufacturing plans, and PRV assumptions
Kakkis said Ultragenyx continues to work with FDA on DTX401 for glycogen storage disease type 1a and reiterated the Aug. 23 PDUFA date, adding that FDA has indicated an advisory committee is not planned. For UX111 in Sanfilippo syndrome, he said a resubmitted BLA is under review with a Sept. 19 PDUFA date and characterized post-resubmission interactions as “routine.”
On launch readiness and manufacturing, Kakkis said the company has been manufacturing inventory since last year. He said DTX401 drug substance and drug product are made at the Bedford facility, while UX111 uses a contract manufacturer in Ohio for drug substance with fill-finish at Bedford.
Horn discussed priority review vouchers, saying Ultragenyx plans to monetize two PRVs—one each for UX111 and DTX401—and has “baked” proceeds into internal modeling at “a little over $100 million each,” with any amount above that considered upside. He added that a PRV associated with GTX-102 would also represent upside.
Horn reiterated the company’s stated pathway to profitability in 2027 and said Ultragenyx does not expect to “hang out at the razor’s edge,” adding that how much the company reinvests versus drops to the bottom line will depend on launch performance. He also told analysts that reaching profitability does not require all upcoming launches to be successful, pointing to multiple levers the company can pull.
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.
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