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Ultragenyx Pharmaceutical Inc.

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Ultragenyx Pharmaceutical Inc. • NASDAQ
$34.75 ▲ 2.93% (+0.99)
Market Cap $3.35 B
52w High $50.00
52w Low $25.81
Dividend Yield 0%
P/E -5.86
Volume 785.18K
Outstanding Shares 96.30M

Income Statement

Period Revenue Operating Expense Net Income Net Profit Margin Earnings Per Share EBITDA
Q3-2025 $159.933M ▼ $302.832M ▲ $-180.413M ▼ -112.805% ▼ $-1.808 ▼ $-156.786M ▼
Q2-2025 $166.496M ▲ $251.382M ▼ $-114.951M ▲ -69.041% ▲ $-1.17 ▲ $-91.344M ▲
Q1-2025 $139.292M ▼ $253.569M ▼ $-151.08M ▼ -108.463% ▼ $-1.57 ▼ $-126.438M ▼
Q4-2024 $164.877M ▲ $270.261M ▲ $-133.385M ▲ -80.9% ▲ $-1.39 ▲ $-108.803M ▼
Q3-2024 $139.494M $250.46M $-133.516M -95.715% $-1.4 $-108.744M

Balance Statement

Period Cash & Short-term Total Assets Total Liabilities Total Equity
Q3-2025 $425.248M ▼ $1.19B ▼ $1.174B ▲ $9.159M ▼
Q2-2025 $477.385M ▼ $1.306B ▼ $1.148B ▼ $151.286M ▲
Q1-2025 $494.44M ▼ $1.312B ▼ $1.161B ▼ $144.246M ▼
Q4-2024 $610.025M ▼ $1.503B ▼ $1.241B ▲ $255.297M ▼
Q3-2024 $621.608M $1.538B $1.185B $346.827M

Cash Flow Statement

Period Net Income Cash From Operations Cash From Investing Cash From Financing Net Change Free Cash Flow
Q3-2025 $-180.413M ▼ $-91.423M ▲ $117.182M ▲ $2K ▼ $26.055M ▼ $-92.701M ▲
Q2-2025 $-114.951M ▲ $-108.273M ▲ $71.175M ▼ $83.649M ▲ $49.465M ▲ $-95.733M ▲
Q1-2025 $-151.08M ▼ $-166.475M ▼ $119.89M ▲ $157K ▼ $-44.98M ▼ $-182.799M ▼
Q4-2024 $-133.385M ▲ $-79.503M ▼ $102.735M ▲ $3.186M ▼ $23.945M ▲ $-80.157M ▼
Q3-2024 $-133.516M $-66.995M $-275.502M $11.174M $-330.048M $-68.612M

Revenue by Products

Product Q4-2024Q1-2025Q2-2025Q3-2025
Product
Product
 $70.00M ▲$90.00M ▲$80.00M ▼$90.00M ▲
Royalty
Royalty
 $90.00M ▲$50.00M ▼$90.00M ▲$60.00M ▼

Five-Year Company Overview

Income Statement

Income Statement Ultragenyx has been growing its revenue at a healthy pace over the past several years, showing that its marketed rare-disease drugs are gaining commercial traction. Gross profit is strong relative to sales, which indicates good pricing power in its niche markets. However, the company remains meaningfully unprofitable: operating losses and net losses have been sizable every year, driven mainly by high research and development and commercialization expenses. Losses widened as the pipeline and commercial footprint expanded, and only recently show early signs of stabilizing rather than accelerating. Overall, the income statement reflects a company still firmly in an investment and build-out phase, not yet in a steady-profit phase.

Balance Sheet

Balance Sheet The balance sheet shows a solid base of total assets that has stayed fairly stable over time, but the mix has changed in ways that matter. Cash has come down sharply from earlier levels, suggesting prior cash reserves have been used to fund operations and capital projects. At the same time, debt has jumped recently from very low levels to a much more meaningful amount, indicating a shift toward borrowing to support the business. Shareholders’ equity has been shrinking, reflecting cumulative losses eating into the capital base. In simple terms, Ultragenyx still has a respectable asset base, but it is now more leveraged and has less balance sheet cushion than a few years ago.

Cash Flow

Cash Flow Cash flow from operations has been consistently negative, which means the company is using cash rather than generating it from its current product portfolio. Free cash flow has also been deeply negative, as operating outflows are layered on top of ongoing investment needs, including manufacturing and infrastructure build-out. Capital spending itself is not huge but is meaningful for a company of this size and reinforces the picture of a business still investing heavily in future capacity. Overall, Ultragenyx relies on external funding (equity, debt, or partnerships) rather than internal cash generation to support its strategy, and the cash burn remains a central financial risk to watch.

Competitive Edge

Competitive Edge Ultragenyx occupies a focused and differentiated position in rare and ultra-rare diseases, where competition is thinner and unmet medical need is high. It has multiple approved therapies addressing serious, often underserved conditions, which provides brand recognition with specialists and deep relationships in small patient communities. Its proprietary gene therapy manufacturing platform and in-house facility add a technical and operational barrier to entry that many smaller peers lack. Strategic collaborations with larger partners extend its reach and validate its scientific approach. The flip side is exposure to concentrated revenue sources, dependence on favorable reimbursement for high-cost therapies, and the risk that larger pharmaceutical companies or emerging gene therapy players intensify competition over time.

Innovation and R&D

Innovation and R&D Innovation is clearly the core of Ultragenyx’s identity and explains much of its financial profile. The company is investing heavily in a broad pipeline across several technology types—gene therapies, biologics, small molecules, and antisense drugs—targeting diverse rare genetic conditions. Its Pinnacle PCL gene therapy platform and dedicated manufacturing plant are long-term bets on scalability and quality in a complex field. Several late-stage programs, including treatments for disorders like Angelman syndrome and osteogenesis imperfecta, could materially change the business if they succeed, but each carries the usual clinical, regulatory, and commercial uncertainties. The recent regulatory setback on one gene therapy, tied to manufacturing rather than clinical performance, highlights both the promise and the complexity of this strategy: the science may be compelling, yet execution and compliance remain crucial.

Summary

Ultragenyx is a science-driven rare-disease company that has converted its innovation into steadily rising revenue but not yet into profitability. Financially, it is still a cash-consuming enterprise with persistent losses, a thinner equity cushion, and more reliance on debt than in the past. Strategically, it holds a strong niche position supported by specialized products, deep rare-disease expertise, and proprietary gene therapy manufacturing capabilities. The large, diverse pipeline offers meaningful upside potential but also concentrates risk in successful execution of complex clinical, regulatory, and manufacturing plans. In essence, Ultragenyx looks like a high-innovation, high-investment biotech story where the long-term outcome will depend on how effectively it can turn its pipeline and platform into durable, cash-generating franchises before balance sheet and cash-flow pressures become more constraining.

NEWS

November 4, 2025 · 4:01 PM UTC

Ultragenyx Announces Sale of a Portion of Future North American Royalties on Crysvita® (burosumab) for $400 Million to OMERS Life Sciences

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October 30, 2025 · 9:00 AM UTC

Kiora Pharmaceuticals Joins RARE-X Vision Consortium Promoting Collaboration and Development of New Treatments for Rare Ocular Disorders

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October 28, 2025 · 4:30 PM UTC

Ultragenyx to Host Conference Call for Third Quarter 2025 Financial Results and Corporate Update

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October 24, 2025 · 4:30 PM UTC

Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)

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October 3, 2025 · 4:30 PM UTC

Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)

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