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 months ended March 31, 2025, Ultragenyx reported total revenues of $139.3 million, up 28% from the same period in 2024. This increase was largely driven by growing demand for the company’s approved products, including Crysvita, Dojolvi, Evkeeza, and Mepsevii. Crysvita, the company’s largest revenue contributor, saw a 52% increase in product sales compared to the prior year period.

Despite the revenue growth, Ultragenyx continued to incur net losses, reporting a net loss of $151.1 million for the first quarter of 2025, compared to a net loss of $170.7 million in the same period of 2024. The company’s net losses have been primarily driven by its significant research and development (R&D) expenses as it advances its pipeline of product candidates.

Revenue and Profit Trends

Ultragenyx’s total revenues have steadily increased over the past several quarters as the company’s approved products have gained traction in the market. The 28% year-over-year increase in total revenues for the first quarter of 2025 was largely attributable to the following:

Product	Q1 2025 Sales	Q1 2024 Sales	% Change
Crysvita	$55,080,000	$36,241,000	52%
Dojolvi	$17,009,000	$16,362,000	4%
Evkeeza	$11,031,000	$3,275,000	237%
Mepsevii	$8,387,000	$6,611,000	27%

The significant increase in Evkeeza sales was driven by the product’s launch in Japan and several markets in Europe, Middle East, and Africa. Crysvita also saw a substantial increase in demand, particularly in Latin America.

Despite the revenue growth, Ultragenyx continued to report net losses, although the losses have narrowed year-over-year. The company’s net loss of $151.1 million for the first quarter of 2025 was 11.5% lower than the $170.7 million net loss reported in the same period of 2024. This improvement was primarily due to a 7% decrease in R&D expenses, which offset a 12% increase in selling, general, and administrative (SG&A) expenses.

The reduction in R&D expenses was driven by several factors, including:

• A decrease in manufacturing costs for the company’s gene therapy programs, particularly UX701 and DTX301
• A reduction in translational research expenses as the company prioritized its clinical-stage programs
• Lower costs for approved products as the company’s medical affairs and regulatory activities stabilized

The increase in SG&A expenses was mainly attributable to higher employee compensation costs and the recognition of reimbursement for a cost-sharing arrangement that impacted the prior-year period.

Strengths and Weaknesses

Strengths:

1. Diverse and growing portfolio of approved products: Ultragenyx has successfully commercialized four rare disease therapies, providing a stable revenue stream to support its R&D efforts.
2. Robust pipeline of clinical-stage product candidates: The company has a deep pipeline of product candidates across multiple therapeutic modalities, including several programs in late-stage clinical development.
3. Experienced management team: Ultragenyx is led by a seasoned team with expertise in rare disease drug development and commercialization.
4. Strong financial position: The company has a solid cash position of $563.0 million as of March 31, 2025, providing it with the resources to advance its pipeline and support ongoing operations.

Weaknesses:

1. Continued net losses: Despite revenue growth, Ultragenyx has yet to achieve profitability, as its R&D expenses have outpaced its commercial revenues.
2. Reliance on a few key products: While the company has diversified its portfolio, Crysvita remains its primary revenue driver, accounting for a significant portion of total revenues.
3. Competitive landscape: The rare disease market is becoming increasingly crowded, with potential competition for some of Ultragenyx’s product candidates.
4. Manufacturing and supply chain risks: The company’s gene therapy manufacturing facility and reliance on contract manufacturing organizations expose it to potential operational and supply chain disruptions.

Outlook and Future Prospects

Ultragenyx’s future prospects appear promising, as the company continues to advance its diverse pipeline of product candidates and expand the commercial reach of its approved therapies.

In the near term, the company expects to achieve several key milestones:

• Completion of the Phase 3 Orbit study for the OI treatment UX143 (setrusumab) in the fourth quarter of 2025
• Completion of enrollment for the Phase 3 Aspire study of GTX-102 for Angelman syndrome in the second half of 2025
• Submission of a Biologics License Application (BLA) for UX111 for the treatment of MPS IIIA, with a PDUFA action date of August 18, 2025
• Submission of a BLA for DTX401 for the treatment of GSDIa, based on positive data from the Phase 3 GlucoGene study and its Crossover Period

These upcoming milestones, if successful, could further strengthen Ultragenyx’s pipeline and provide additional revenue-generating products to complement its existing commercial offerings.

However, the company will need to continue managing its R&D expenses to improve its path to profitability. The planned launches of new products and the potential for increased competition in the rare disease space may also present challenges that Ultragenyx will need to navigate effectively.

Overall, Ultragenyx’s diverse portfolio, robust pipeline, and experienced management team position the company well to continue its growth trajectory and deliver innovative treatments to patients with rare and ultra-rare genetic diseases.