A clinical-stage biopharma firm specializing in innovative treatments for rare genetic diseases with a diversified revenue portfolio growing from $363M in 2022 to $673M in 2025.
Investors may find value in RARE due to its growing revenue base exceeding $673M and a differentiated portfolio of orphan drugs with long-term pricing power. The company's strategic asset acquisitions and strong cash position of $421M provide runway to advance its high-value pipeline while generating royalty income.
The stock faces substantial downside risk given the retained earnings deficit of -$4.53B and a net loss that remains elevated at -$575M, raising concerns about sustainability without major commercial breakthroughs. High R&D spending of $750M continues to outpace revenue growth, suggesting significant dilution pressure from equity financing needs.
Ultragenyx Pharmaceutical Inc. (RARE) is a biopharmaceutical company focused on developing and commercializing therapies for rare diseases and orphan indications across the United States and Europe. The company leverages its proprietary platform to address high-need unmet medical needs in genetic disorders, leveraging a diverse pipeline of small molecules and gene therapies.
Despite a net loss widening slightly to -$575M in 2025, the company successfully expanded its revenue by $113M year-over-year to reach $673M, driven by strong commercial performance of existing assets. However, the massive operating expenses and negative cash flow from operations of -$466M highlight the capital-intensive nature of scaling rare disease therapies.
The rare disease sector is characterized by high barriers to entry, substantial government incentives like orphan drug exclusivity, and a patient population that is small but highly vulnerable. Companies in this space often face long development cycles with binary clinical outcomes but offer the potential for blockbuster per-patient revenues once approved.
| Metric | 2022 | 2023 | 2024 | 2025 |
|---|---|---|---|---|
| Revenue | $363.3M | $434.2M | $560.2M | $673.0M |
| Cost of Revenue | $0 | $0 | $0 | $0 |
| Gross Profit | $363.3M | $434.2M | $560.2M | $673.0M |
| Operating Expenses | $1.01B | $1.00B | $1.10B | $1.21B |
| Operating Income | $-648.9M | $-569.2M | $-536.0M | $-535.0M |
| R&D | $705.8M | $648.4M | $697.9M | $750.0M |
| G&A | $278.1M | $309.8M | $321.6M | $349.0M |
| Stock-Based Comp | $130.4M | $135.2M | $158.1M | $153.0M |
| D&A | $18.2M | $26.0M | $35.5M | $35.0M |
| Other OpEx | $28.3M | $45.2M | $76.7M | $109.0M |
| Non-Operating Income | $-52.8M | $-39.3M | $-31.6M | $-36.0M |
| Interest Income | $11.1M | $26.7M | $36.5M | $25.0M |
| Pre-Tax Income | $-701.7M | $-608.5M | $-567.6M | $-571.0M |
| Income Tax | $5.7M | $-1.8M | $1.6M | $4.0M |
| Continuing Ops | $-707.4M | $-606.6M | $-569.2M | $-575.0M |
| Discontinued Ops | $0 | $0 | $0 | $0 |
| Net Income | $-707.4M | $-606.6M | $-569.2M | $-575.0M |
| Minority Interest | $0 | $0 | $0 | $0 |
| Net to Common | $-707.4M | $-606.6M | $-569.2M | $-575.0M |
| Metric | 2022 | 2023 | 2024 | 2025 |
|---|---|---|---|---|
| Current Assets | $883.9M | $732.2M | $817.1M | $951.0M |
| Cash & Equivalents | $132.9M | $213.6M | $173.7M | $421.0M |
| Accounts Receivable | $40.4M | $73.4M | $121.8M | $158.0M |
| Inventory | $26.8M | $34.0M | $45.0M | $52.0M |
| Prepaid Expenses | $68.9M | — | $40.3M | $61.0M |
| Other Current Assets | $614.8M | $411.2M | $436.3M | $259.0M |
| Long-Term Assets | $661.5M | $758.8M | $686.3M | $581.0M |
| PP&E (Net) | $259.7M | $290.6M | $265.9M | $244.0M |
| Intangibles | $160.1M | $166.3M | $178.3M | $176.0M |
| Goodwill | $44.4M | $44.4M | $44.4M | $44.0M |
| Operating Leases | $26.0M | $23.9M | $25.5M | $23.0M |
| Other LT Assets | $171.3M | $233.7M | $172.2M | $94.0M |
| Total Assets | $1.55B | $1.49B | $1.50B | $1.53B |
| Current Liabilities | $261.2M | $280.4M | $344.2M | $384.0M |
| Accounts Payable | $43.3M | $42.1M | $38.8M | $31.0M |
| Current Leases | $11.6M | $12.6M | $10.3M | $12.0M |
| Other Current Liab | $1.7M | $29.2M | $54.1M | $76.0M |
| Long-Term Liabilities | $931.7M | $935.2M | $897.0M | $1.22B |
| LT Operating Leases | $19.8M | $30.6M | $30.0M | $24.0M |
| Other LT Liab | $911.9M | $904.6M | $867.0M | $1.20B |
| Total Liabilities | $1.19B | $1.22B | $1.24B | $1.60B |
| Shareholders' Equity | $352.5M | $275.4M | $262.3M | $-73.0M |
| Paid-In Capital | $3.14B | $3.66B | $4.21B | $4.45B |
| Retained Earnings | $-2.78B | $-3.39B | $-3.96B | $-4.53B |
| AOCI | $-6.6M | $647.0K | $-643.0K | $1.0M |
| Treasury Stock | — | $432.0K | $3.6M | $8.0M |
| Total L&E | $1.55B | $1.49B | $1.50B | $1.53B |
| Metric | 2022 | 2023 | 2024 | 2025 |
|---|---|---|---|---|
| Net Income (CFO) | $-707.4M | $-606.6M | $-569.2M | $-575.0M |
| Non-Cash Adjustments | $310.6M | $120.7M | $121.9M | $62.0M |
| Working Capital Changes | $16.3M | $11.2M | $33.1M | $47.0M |
| Cash from Operations | $-380.5M | $-474.8M | $-414.2M | $-466.0M |
| Capital Expenditures | $116.1M | $44.3M | $7.5M | $6.0M |
| Cash from Investing | $-291.7M | $168.0M | $-17.8M | $236.0M |
| Cash from Financing | $501.2M | $388.1M | $399.2M | $478.0M |
| FX Effect | $-1.1M | $462.0K | $-2.5M | $4.0M |
| Net Change in Cash | $-172.0M | $81.8M | $-35.2M | $252.0M |
| Beginning Cash | $309.6M | $137.6M | $219.4M | $184.0M |
| Ending Cash | $137.6M | $219.4M | $184.2M | $436.0M |
The company operates primarily through its small molecule drug portfolio, which generated the majority of recent gross profit of $673M, alongside an emerging gene therapy segment and strategic licensing partnerships that contribute to recurring royalty streams and milestone revenue.
Significant cash burn from R&D expenses totaling $750M in 2025 creates liquidity pressure, reliance on specific regulatory approvals for orphan drugs, potential patent cliffs on key revenue drivers, and the inherent risk of clinical trial failures in complex genetic indications.
Ultragenyx possesses a deep proprietary knowledge base in rare disease mechanisms, a robust commercial infrastructure for orphan drug markets, and a unique portfolio of high-value assets with limited generic competition.
Not financial advice. All content is for informational purposes only. Data may contain errors. Consult a qualified financial advisor before making investment decisions.