Form 10-Q for the Quarter Ended June 30, 2026

Overview of Ultragenyx’s Financial Performance

Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company focused on developing and commercializing treatments for rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates, including biologics, small molecules, gene therapies, and nucleic acid products.

For the three and six months ended June 30, 2026, Ultragenyx reported total revenues of $214 million and $350 million, respectively, representing increases of 28% and 14% compared to the same periods in 2025. This revenue growth was largely driven by increased demand for the company’s approved products, such as Crysvita, Dojolvi, Evkeeza, and Mepsevii, as well as the timing of product shipments.

However, Ultragenyx continued to incur net losses during this period, reporting $92 million and $277 million in net losses for the three and six months ended June 30, 2026, respectively. These losses were primarily due to the company’s ongoing investments in research and development (R&D) activities, as well as selling, general, and administrative (SG&A) expenses associated with its operations.

Revenue and Profit Trends

Ultragenyx’s product sales increased by 38% and 17% for the three and six months ended June 30, 2026, respectively, compared to the same periods in 2025. This growth was driven by the following key factors:

• Crysvita sales increased by 54% and 11% for the three and six months, respectively, primarily due to an increase in the number of patients in Latin American territories and the timing of shipments.
• Dojolvi sales increased by 17% and 13% for the three and six months, respectively, as the product continued to gain traction.
• Evkeeza sales increased by 50% and 56% for the three and six months, respectively, due to the continued expansion of the product in territories outside the United States.
• Mepsevii sales increased by 11% for the three months but remained flat for the six months.

In addition to product sales, Ultragenyx also generates revenue from Crysvita royalties, which increased by 19% and 11% for the three and six months ended June 30, 2026, respectively, compared to the same periods in 2025. This increase was primarily due to an increase in the number of patients on Crysvita therapy and the timing of orders.

Despite the revenue growth, Ultragenyx continued to report net losses during the period, as the company’s R&D and SG&A expenses remained high. R&D expenses increased by 1% and 7% for the three and six months ended June 30, 2026, respectively, compared to the same periods in 2025. This increase was primarily driven by higher costs associated with the company’s gene therapy programs, such as DTX401 and UX111, as it prepared for potential commercial launches. SG&A expenses remained relatively flat, increasing by only 1% for both the three and six months ended June 30, 2026, compared to the same periods in 2025.

Strengths and Weaknesses

Ultragenyx’s key strengths include its diverse portfolio of approved therapies and product candidates, its focus on rare and ultra-rare genetic diseases with high unmet medical need, and its experienced management team. The company’s approved products, such as Crysvita, Dojolvi, Evkeeza, and Mepsevii, have demonstrated revenue growth, indicating strong commercial execution and market acceptance.

Additionally, Ultragenyx’s pipeline of clinical-stage product candidates, including gene therapies, biologics, and small molecules, provides a robust platform for future growth. The company has received various regulatory designations, such as Rare Pediatric Disease, RMAT, Fast Track, and Orphan Drug, for several of its product candidates, which may expedite the development and approval process.

However, Ultragenyx’s key weakness is its continued net losses, which have persisted since the company’s inception. The company’s significant investments in R&D, particularly for its clinical-stage product candidates, have resulted in substantial operating expenses that have outpaced its revenue growth. This has led to a need for ongoing capital raises to fund the company’s operations and development activities.

Another potential weakness is the company’s reliance on strategic collaborations and partnerships, such as its agreement with Mereo for the development of UX143 (setrusumab) for the treatment of Osteogenesis Imperfecta. While these collaborations can provide additional resources and expertise, they also introduce complexities and potential risks related to the successful execution and commercialization of the partnered programs.

Outlook and Future Prospects

Ultragenyx’s outlook appears cautiously optimistic, as the company continues to make progress with its pipeline of product candidates and expects several key milestones in the near future:

• Dojolvi was launched in Japan in May 2026 after receiving manufacturing and marketing approval under the Conditional Approval System for Pharmaceuticals.
• The company’s Biologics License Application (BLA) for DTX401 for the treatment of Glycogen Storage Disease Type Ia (GSDIa) was accepted for review by the FDA, with a PDUFA action date of August 23, 2026.
• The BLA for UX111 for the treatment of Sanfilippo syndrome Type A (MPS IIIA) was also accepted for review by the FDA, with a PDUFA action date of September 19, 2026.
• Enrollment in the pivotal Phase 3 “Aspire” study for GTX-102 in Angelman syndrome is complete, with data expected in the September or October 2026 timeframe.
• Enrollment in the Phase ²⁄₃ “Aurora” study for GTX-102 in other Angelman syndrome genotypes and ages is expected to be completed in the second half of 2026.
• Data from Stage 1 of the pivotal “Cyprus2+” study for UX701 in Wilson disease are expected in the fourth quarter of 2026.
• The Phase ¹⁄₂ study for UX016 in GNE myopathy is expected to begin enrolling patients in the second half of 2026.

However, the company also faced a setback with its UX143 (setrusumab) program for the treatment of Osteogenesis Imperfecta (OI), as the Phase 3 “Orbit” and “Cosmic” studies did not achieve their primary endpoint of reduction in annualized clinical fracture rate. Ultragenyx will continue to engage with regulatory agencies to determine the necessary data to support a potential regulatory filing for UX143 and explore a possible path forward for the program.

To address its ongoing net losses and focus resources on its highest value drivers, Ultragenyx implemented a strategic restructuring plan in February 2026, which included a 10% reduction in its workforce and the curtailment of certain operating activities, including UX143 manufacturing. The company expects this restructuring to result in a decrease in R&D expenses in the near term, as several of its Phase 3 clinical programs are expected to be completed.

Overall, Ultragenyx’s financial performance reflects the challenges and opportunities inherent in the rare disease drug development and commercialization landscape. While the company has made progress in growing its revenue and advancing its pipeline, it continues to face the need to balance its investments in R&D with the goal of achieving profitability. The company’s ability to successfully navigate this balance and execute on its upcoming milestones will be crucial in determining its long-term success.