uniQure N.V.
Business
uniQure N.V. is a leader in gene therapy developing one‑time administered genomic medicines to treat rare and other serious diseases. It advances a focused pipeline of AAV‑based gene therapy product candidates including AMT‑130 for Huntington’s disease, AMT‑162 for SOD1‑ALS, AMT‑260 for refractory mesial temporal lobe epilepsy, AMT‑191 for Fabry disease, and the commercial product HEMGENIX® through a collaboration. Its activities encompass research and development, clinical trials, next‑generation capsid and cargo technology development, intellectual property management, and manufacturing partnerships. It conducts clinical programs and commercial supply through collaborations and partner distribution across the U.S., Europe and other jurisdictions.
Summary from filing dated 2025-02-27
Financials
Consolidated Statement of Income
| Concept | Trend | FY 2025 2025-12-31 | FY 2024 2024-12-31 | FY 2023 2023-12-31 | FY 2022 2022-12-31 | FY 2021 2021-12-31 |
|---|---|---|---|---|---|---|
| Total revenues | 16 | 27 | 16 | 106 | 524 | |
| Cost of license revenues | −2 | −1 | −65,000 | −1 | −25 | |
| Cost of contract manufacturing revenues | — | −17 | −14 | −2 | — | |
| Research and development expenses | −141 | −144 | −215 | −198 | −144 | |
| Selling, general and administrative expenses | −65 | −53 | −75 | −55 | −56 | |
| Total operating expenses | −208 | −215 | −303 | −256 | −225 | |
| Other income | 14 | 8 | 6 | 7 | 12 | |
| Other expense | −8 | −5 | −2 | −800,000 | −876,000 | |
| Loss from operations | −185 | −184 | −283 | −143 | 311 | |
| Interest income | 17 | 21 | 20 | 609,000 | 162,000 | |
| Interest expense | −62 | −64 | −42 | −12 | — | |
| Foreign currency gains / (losses), net | 26 | −11 | −2 | 23 | 30 | |
| Other non-operating gains, net | 11 | — | — | 3 | −160,000 | |
| Loss before income tax expense | −193 | −237 | −307 | −128 | 333 | |
| Income tax expense | −6 | −2 | −2 | 1 | −3 | |
| Net loss | −199 | −240 | −308 | — | — | |
| Foreign currency translation (losses) / gains, net | −6 | 426,000 | 7 | −29 | −39 | |
| Defined benefit pension gain / (loss), net of taxes | 771,000 | 327,000 | −2 | — | — | |
| Total comprehensive loss | −204 | −239 | −304 | −156 | 291 | |
| Basic net loss per ordinary share | −3.46 | −4.92 | −6.47 | −2.71 | 7.17 | |
| Diluted net loss per ordinary share | −3.46 | −4.92 | −6.47 | −2.71 | 7.04 | |
| Weighted average shares - basic | 58 | 49 | 48 | 47 | 46 | |
| Weighted average shares - diluted | 58 | 49 | 48 | 47 | 47 |
Consolidated Balance Sheet
| Concept | Trend | FY 2025 2025-12-31 | FY 2024 2024-12-31 | FY 2023 2023-12-31 | FY 2022 2022-12-31 | FY 2021 2021-12-31 |
|---|---|---|---|---|---|---|
| Cash and cash equivalents | 80 | 159 | 241 | 228 | 556 | |
| Current investment securities | 542 | 209 | 377 | 125 | — | |
| Accounts receivable | 6 | 6 | 4 | 102 | 59 | |
| Prepaid expenses | 21 | 9 | 15 | 12 | 11 | |
| Other current assets and receivables | 7 | 8 | 3 | 3 | 3 | |
| Total current assets | 656 | 390 | 652 | 477 | 628 | |
| Property, plant and equipment, net | 14 | 20 | 47 | 51 | 44 | |
| Other investments | 30 | 27 | — | — | — | |
| Operating lease right-of-use assets | 13 | 14 | 29 | 33 | 26 | |
| Intangible assets, net | 73 | 71 | 60 | — | — | |
| Goodwill | 25 | 22 | 26 | 26 | 28 | |
| Deferred tax assets, net | 9 | 10 | 12 | 15 | 16 | |
| Other non-current assets | 6 | 1 | 3 | 6 | 6 | |
| Total non-current assets | 169 | 166 | 180 | 228 | 181 | |
| Total assets | 825 | 557 | 832 | 705 | 809 | |
| Accounts payable | 5 | 7 | 7 | 11 | 3 | |
| Accrued expenses and other current liabilities | 41 | 29 | 31 | 31 | 28 | |
| Liability related to pre-funded warrants | 13 | — | — | — | — | |
| Current portion of operating lease liabilities | 4 | 4 | 8 | 8 | 6 | |
| Total current liabilities | 63 | 40 | 74 | 76 | 37 | |
| Long-term debt | 50 | 51 | 102 | 103 | 101 | |
| Liability from royalty financing agreement | 473 | 435 | 394 | — | — | |
| Operating lease liabilities, net of current portion | 10 | 11 | 28 | 32 | 29 | |
| Contingent consideration | 19 | 11 | 15 | 9 | 30 | |
| Deferred tax liability, net | 8 | 7 | 8 | 8 | 13 | |
| Other non-current liabilities, net of current portion | 4 | 8 | 4 | 935,000 | 4 | |
| Total non-current liabilities | 563 | 523 | 550 | 153 | 177 | |
| Total liabilities | 626 | 563 | 624 | 229 | 213 | |
| Commitments and contingencies | — | — | — | — | — | |
| Ordinary shares, 0.05 par value: 80,000,000 shares authorized as of December 31, 2025 and December 31, 2024 and 62,336,717 and 48,988,087 ordinary shares issued and outstanding as of December 31, 2025 and December 31, 2024, respectively | 4 | 3 | 3 | 3 | 3 | |
| Additional paid-in-capital | 1,582 | 1,173 | 1,149 | 1,113 | 1,077 | |
| Accumulated other comprehensive loss | −58 | −53 | −54 | −58 | −29 | |
| Accumulated deficit | −1,329 | −1,130 | −890 | −582 | −455 | |
| Total shareholders' equity / (deficit) | 199 | −7 | 208 | 476 | 596 | |
| Total liabilities and shareholders' equity / (deficit) | 825 | 557 | 832 | 705 | 809 |
Consolidated Statement of Cash Flows
| Concept | Trend | FY 2025 2025-12-31 | FY 2024 2024-12-31 | FY 2023 2023-12-31 | FY 2022 2022-12-31 | FY 2021 2021-12-31 |
|---|---|---|---|---|---|---|
| Net loss | −199 | −240 | −308 | −127 | 330 | |
| Depreciation and amortization | 15 | 13 | 12 | 9 | 7 | |
| Amortization of discount on investment securities | −10 | −11 | −11 | — | — | |
| Share-based compensation expense | 18 | 22 | 35 | 34 | 26 | |
| Royalty financing agreement interest expense, net of interest paid | 39 | 44 | 26 | — | — | |
| Deferred tax expense | 2 | 2 | 2 | −1 | 3 | |
| Changes in fair value of contingent consideration | 6 | −2 | 16 | — | — | |
| Changes in fair value of liability related to pre-funded warrants | −12 | — | — | — | — | |
| Unrealized foreign exchange (gains) / losses, net | −23 | 15 | −2 | −22 | −31 | |
| Other items, net | −5 | −4 | 5 | 2 | −3 | |
| Accounts receivable, prepaid expenses, and other current assets and receivables | −9 | −2 | −1 | — | — | |
| Accounts payable | −3 | 2 | −4 | 9 | −727,000 | |
| Accrued expenses, other liabilities, and operating leases | 4 | −6 | −5 | 3 | 9 | |
| Contingent consideration milestone payments | — | −20 | −2 | — | — | |
| Inventories | — | 2 | −7 | −7 | — | |
| Contract asset related to CSL Behring milestone payments | — | — | 100 | −45 | −55 | |
| Net cash used in operating activities | −178 | −183 | −146 | −145 | 288 | |
| Proceeds on maturity of debt securities | 337 | 534 | 168 | — | — | |
| Investment in debt securities | −658 | −360 | −366 | −163 | 0 | |
| Divestment of commercial manufacturing facility | — | −8 | — | — | — | |
| Purchases of property, plant, and equipment | −439,000 | −3 | −7 | −18 | −17 | |
| Net cash (used in) / generated from investing activities | −322 | 163 | −206 | −183 | −67 | |
| Proceeds from follow-on public offerings of ordinary shares, net of issuance costs | 381 | — | — | — | — | |
| Proceeds from issuance of pre-funded warrants, net of issuance costs | 23 | — | — | — | — | |
| Proceeds from issuance of ordinary shares related to employee stock options and purchase plans | 11 | 2 | 308,000 | 1 | 3 | |
| Repayment of long-term debt | — | −53 | — | — | — | |
| Contingent consideration milestone payments | — | −9 | −8 | — | — | |
| Proceeds from royalty financing agreement, net of issuance costs | — | — | 370 | — | — | |
| Net cash generated from / (used in) financing activities | 415 | −59 | 363 | 1 | 95 | |
| Currency effect on cash, cash equivalents and restricted cash | 6 | −5 | 2 | −2 | −4 | |
| Net (decrease) / increase in cash, cash equivalents and restricted cash | −79 | −84 | 13 | −328 | 312 | |
| Cash paid for interest | −25 | −21 | −17 | −9 | −7 | |
| Non-cash decrease in accounts payables and accrued expenses and other current liabilities related to purchases of property, plant, and equipment | −99,000 | −672,000 | −513,000 | −964,000 | 1 |