Ultragenyx Reports Strong Growth Amid Challenges

Ultragenyx Pharmaceutical, Inc. ((RARE)) has held its Q1 earnings call. Read on for the main highlights of the call.

Ultragenyx Pharmaceutical, Inc. showcased a mixed sentiment during its latest earnings call, highlighting strong revenue growth and significant progress in its clinical pipeline. Despite these positive strides, the company also reported a substantial operating loss and high cash usage, with some variability in revenue. Nevertheless, the forward-looking statements reflect confidence in achieving full-year GAAP profitability by 2027.

Strong Revenue Growth

Ultragenyx reported impressive revenue growth in Q1 2025, reaching $139 million, which marks a 28% increase compared to the same period in 2024. The company’s flagship product, Crysvita, contributed $103 million to this total, demonstrating a 25% growth over the previous year. Notably, Crysvita saw a 52% growth in Latin America and Turkey, underscoring the company’s successful expansion in these regions.

Progress in Clinical Development

The company reported significant advancements in its late-stage clinical programs, particularly with UX143 for osteogenesis imperfecta and GTX-102 for Angelman syndrome. Phase 3 trials are progressing well, with key data readouts anticipated in 2026, positioning Ultragenyx for potential future successes in these areas.

Global Commercial Expansion

Ultragenyx has expanded its global commercial presence, particularly with Crysvita in Latin America and Evkeeza in Europe and Japan. Successful reimbursement negotiations in Brazil and Mexico have been pivotal in driving growth, reflecting the company’s strategic focus on international markets.

Pipeline Advancements

The company is advancing its pipeline with two Biologics License Applications (BLAs) in progress. UX111 for Sanfilippo syndrome is under FDA review, and DTX401 for GSDIa is on track for a mid-2025 submission, highlighting Ultragenyx’s commitment to addressing unmet medical needs.

Operating Loss

Despite the revenue growth, Ultragenyx reported a net loss of $151 million for Q1 2025, with total operating expenses reaching $282 million. This includes a substantial R&D investment of $166 million, reflecting the company’s focus on advancing its clinical pipeline.

Cash Usage

The company reported net cash usage of $166 million in operations for Q1 2025, which includes a $30 million milestone payment for GTX-102. This high cash usage is typical for the first quarter, reflecting upfront costs associated with ongoing projects.

Variability in Revenue

Ultragenyx noted some variability in its quarter-to-quarter revenue, primarily due to uneven ordering patterns for Crysvita in Latin America. This highlights the challenges of managing revenue streams in diverse international markets.

Forward-Looking Guidance

Ultragenyx provided optimistic guidance for 2025, projecting total revenue between $640 million and $670 million, representing a 14% to 20% growth over 2024. Crysvita is expected to generate between $460 million and $480 million, while Dojolvi is projected to contribute $90 million to $100 million. The company remains on track to achieve GAAP profitability by 2027, with key clinical milestones anticipated, including the UX143 Phase 3 trial and BLA submissions for DTX401.

In conclusion, Ultragenyx Pharmaceutical, Inc.’s latest earnings call reflects a company poised for growth, with strong revenue figures and promising clinical advancements. Despite challenges such as operating losses and cash usage, the company’s strategic global expansion and robust pipeline provide a positive outlook for future profitability.