Document and Entity Information
Document and Entity Information - shares | 6 Months Ended | |
Jun. 30, 2023 | Jul. 31, 2023 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Jun. 30, 2023 | |
Document Fiscal Year Focus | 2023 | |
Document Fiscal Period Focus | Q2 | |
Entity Registrant Name | ORCHARD THERAPEUTICS PLC | |
Current Fiscal Year End Date | --12-31 | |
Entity Central Index Key | 0001748907 | |
Entity Filer Category | Non-accelerated Filer | |
Entity Small Business | true | |
Entity Common Stock, Shares Outstanding | 227,230,079 | |
Entity Shell Company | false | |
Entity Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Entity Incorporation, State or Country Code | X0 | |
Entity Address, Address Line One | 245 Hammersmith Road | |
Entity Address, City or Town | London | |
Entity Address, Postal Zip Code | W6 8PW | |
City Area Code | 44 | |
Local Phone Number | (0) 203 808-8286 | |
Document Quarterly Report | true | |
Document Transition Report | false | |
Entity File Number | 001-38722 | |
Entity Emerging Growth Company | false | |
Entity Tax Identification Number | 00-0000000 | |
Entity Address, Country | GB | |
Title of 12(b) Security | American Depositary Shares, each representing tenordinary shares, nominal value £0.10 per share | |
Trading Symbol | ORTX | |
Security Exchange Name | NASDAQ |
Condensed Consolidated Balance
Condensed Consolidated Balance Sheets (Unaudited) - USD ($) $ in Thousands | Jun. 30, 2023 | Dec. 31, 2022 |
Current assets: | ||
Cash and cash equivalents | $ 38,273 | $ 68,424 |
Marketable securities | 112,468 | 75,326 |
Accounts receivable | 9,547 | 8,467 |
Inventory | 6,937 | 3,400 |
Prepaid expenses and other current assets | 5,540 | 6,586 |
Research and development tax credit receivable | 8,525 | 5,942 |
Total current assets | 181,290 | 168,145 |
Non-current assets: | ||
Operating lease right-of-use-assets | 21,018 | 22,774 |
Property and equipment, net | 7,808 | 8,138 |
Research and development tax credit receivable | 2,101 | |
Restricted cash | 4,215 | 4,215 |
Intangible assets, net | 3,474 | 3,560 |
Other assets | 12,396 | 12,075 |
Total non-current assets | 51,012 | 50,762 |
Total assets | 232,302 | 218,907 |
Current liabilities: | ||
Accounts payable | 6,950 | 9,318 |
Accrued expenses and other current liabilities | 33,786 | 34,437 |
Deferred revenue, current | 752 | 959 |
Operating lease liabilities | 6,600 | 6,424 |
Notes payable, current | 9,429 | 9,429 |
Total current liabilities | 57,517 | 60,567 |
Notes payable, long-term | 18,440 | 22,991 |
Deferred revenue, net of current portion | 10,819 | 10,315 |
Operating lease liabilities, net of current portion | 16,044 | 19,246 |
PIPE Warrant liabilities | 12,266 | |
Other long-term liabilities | 8,169 | 7,524 |
Total liabilities | 123,255 | 120,643 |
Commitments and contingencies (see Note 15) | ||
Shareholders’ equity: | ||
Ordinary shares (voting and non-voting), GBP 0.10 par value; Most recent authority to allot up to a maximum nominal value of GBP 33,556,809 and GBP 13,023,851.50 of shares at June 30, 2023 and December 31, 2022, respectively; Issued and outstanding - 227,215,699 and 126,947,225 shares at June 30, 2022 and December 31, 2022, respectively. | 29,456 | 16,419 |
Additional paid-in capital | 991,642 | 956,711 |
Accumulated other comprehensive income | 18,552 | 26,018 |
Accumulated deficit | (930,603) | (900,884) |
Total shareholders’ equity | 109,047 | 98,264 |
Total liabilities and shareholders’ equity | $ 232,302 | $ 218,907 |
Condensed Consolidated Balanc_2
Condensed Consolidated Balance Sheets (Unaudited) (Parenthetical) - GBP (£) | Jun. 30, 2023 | Jun. 22, 2023 | Mar. 10, 2023 | Dec. 31, 2022 |
Statement of Financial Position [Abstract] | ||||
Ordinary Shares, Par Value | £ 0.10 | £ 0.1 | £ 0.1 | £ 0.10 |
Ordinary Shares, Authorized | £ 33,556,809 | £ 13,023,851.5 | ||
Ordinary Shares, Issued | 227,215,699 | 126,947,225 | ||
Ordinary Shares, Outstanding | 227,215,699 | 126,947,225 |
Condensed Consolidated Statemen
Condensed Consolidated Statements of Operations and Comprehensive Loss (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2023 | Jun. 30, 2022 | Jun. 30, 2023 | Jun. 30, 2022 | |
Total revenue | $ 7,314 | $ 4,368 | $ 8,551 | $ 9,892 |
Costs and operating expenses: | ||||
Cost of product revenue | 2,189 | 1,122 | 2,556 | 2,693 |
Research and development | 16,695 | 21,965 | 32,688 | 50,199 |
Selling, general and administrative | 10,992 | 13,730 | 22,127 | 27,029 |
Total costs and operating expenses | 29,876 | 36,817 | 57,371 | 79,921 |
Loss from operations | (22,562) | (32,449) | (48,820) | (70,029) |
Other income (expense): | ||||
Interest income | 1,391 | 213 | 2,420 | 282 |
Interest expense | (975) | (672) | (1,932) | (1,347) |
Changes in fair value of PIPE Warrant and PIPE Unit liabilities | 8,206 | 12,057 | ||
Other income (expense), net | 1,658 | (18,227) | 6,569 | (24,279) |
Total other income (expense), net | 10,280 | (18,686) | 19,114 | (25,344) |
Net loss before income taxes | (12,282) | (51,135) | (29,706) | (95,373) |
Income tax benefit (expense) | (25) | 219 | (13) | 161 |
Net loss attributable to ordinary shareholders | $ (12,307) | $ (50,916) | $ (29,719) | $ (95,212) |
Net loss per ordinary share attributable to ordinary shareholders, basic | $ (0.07) | $ (0.4) | $ (0.18) | $ (0.75) |
Net loss per ordinary share attributable to ordinary shareholders, diluted | $ (0.07) | $ (0.4) | $ (0.18) | $ (0.75) |
Weighted average ordinary shares outstanding, basic | 189,286,329 | 127,854,596 | 165,482,536 | 127,775,132 |
Weighted average ordinary shares outstanding, diluted | 189,286,329 | 127,854,596 | 165,482,536 | 127,775,132 |
Other comprehensive income: | ||||
Foreign currency translation adjustment | $ (1,770) | $ 17,450 | $ (7,501) | $ 23,045 |
Unrealized gain (loss) on marketable securities | (39) | (1) | 35 | (261) |
Total other comprehensive income (loss) | (1,809) | 17,449 | (7,466) | 22,784 |
Total comprehensive loss | (14,116) | (33,467) | (37,185) | (72,428) |
Product Revenue, Net [Member] | ||||
Total revenue | 6,651 | 3,781 | 7,185 | 8,840 |
Collaboration Revenue [Member] | ||||
Total revenue | $ 663 | $ 587 | $ 1,366 | $ 1,052 |
Condensed Consolidated Statem_2
Condensed Consolidated Statements of Cash Flows (Unaudited) - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2023 | Jun. 30, 2022 | |
Cash flows from operating activities: | ||
Net loss attributable to ordinary shareholders | $ (29,719) | $ (95,212) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Depreciation and amortization | 1,298 | 1,366 |
Share-based compensation | 6,090 | 8,616 |
Non-cash interest expense | 182 | 186 |
Amortization of provision on loss contract | (276) | |
Amortization of premium on marketable securities | (1,553) | 189 |
Deferred income taxes | 448 | |
Change in fair value of warrant and tranche obligation liabilities | (12,057) | |
Unrealized foreign currency and other non-cash adjustments | (9,767) | 25,092 |
Changes in operating assets and liabilities: | ||
Accounts receivable | (693) | (2,997) |
Inventory | (3,316) | (114) |
Research and development tax credit receivable | (4,371) | 13,289 |
Prepaid expenses, other current assets and other assets | 439 | 883 |
Operating leases, right-of-use assets | 2,234 | 2,777 |
Accounts payable, accrued expenses and other liabilities | (2,897) | 4,251 |
Deferred revenue | (193) | (138) |
Operating lease liabilities | (3,466) | (3,887) |
Net cash used in operating activities | (57,341) | (45,975) |
Cash flows from investing activities: | ||
Proceeds from sales and maturities of marketable securities | 79,000 | 97,214 |
Purchases of marketable securities | (114,556) | (50,891) |
Purchases of property and equipment | (483) | (1,572) |
Net cash provided by (used in) investing activities | (36,039) | 44,751 |
Cash flows from financing activities: | ||
Proceeds from employee equity plans, net of taxes withheld | 210 | 137 |
Payment of taxes on restricted stock vesting | (166) | |
Proceeds from the issuance of units in private placement | 68,000 | |
Payment of placement agent fees and offering costs in connection with private placement | (889) | |
Repayment of notes payable | (4,714) | |
Net cash provided by financing activities | 62,441 | 137 |
Effect of exchange rate changes on cash, cash equivalents, and restricted cash | 788 | (1,355) |
Net decrease in cash, cash equivalents and restricted cash | (30,151) | (2,442) |
Cash, cash equivalents, and restricted cash, beginning of period | 72,639 | 60,178 |
Cash, cash equivalents, and restricted cash, end of period | 42,488 | 57,736 |
Supplemental disclosure of noncash activities | ||
Private placement offering costs in accounts payable and accrued expenses | 954 | |
Property and equipment in accounts payable and accrued expenses | 1,046 | |
Supplemental disclosure of cash flow information: | ||
Lease assets obtained in exchange for new operating lease liabilities, net | 4,912 | |
Non-cash adjustments to operating lease right-of-use assets and liabilities | 530 | |
Cash paid for interest | $ 1,593 | $ 1,153 |
Condensed Consolidated Statem_3
Condensed Consolidated Statements of Shareholders' Equity (Unaudited) - USD ($) $ in Thousands | Total | Ordinary Shares | Additional Paid-in Capital | Accumulated Other Comprehensive Income (Loss) | Accumulated Deficit |
Balance at Dec. 31, 2021 | $ 209,950 | $ 16,253 | $ 940,675 | $ 3,246 | $ (750,224) |
Balance, Shares at Dec. 31, 2021 | 125,674,095 | ||||
Share-based compensation expense | 4,660 | 4,660 | |||
Exercise of share options | (1) | $ 28 | (29) | ||
Exercise of share options, Shares | 222,381 | ||||
Vesting of restricted stock units, net of shares withheld for taxes | (3) | $ 1 | (4) | ||
Vesting of restricted stock units, net of shares withheld for taxes, shares | 3,217 | ||||
Ordinary shares issued as part of a consulting agreement | 1 | $ 1 | |||
Ordinary shares issued as part of consulting agreement, Shares | 5,252 | ||||
Foreign currency translation | 5,595 | 5,595 | |||
Unrealized loss on available for sale debt securities | (260) | (260) | |||
Net loss attributable to ordinary shareholders | (44,296) | (44,296) | |||
Balance at Mar. 31, 2022 | 175,646 | $ 16,283 | 945,302 | 8,581 | (794,520) |
Balance, Shares at Mar. 31, 2022 | 125,904,945 | ||||
Balance at Dec. 31, 2021 | 209,950 | $ 16,253 | 940,675 | 3,246 | (750,224) |
Balance, Shares at Dec. 31, 2021 | 125,674,095 | ||||
Foreign currency translation | 23,045 | ||||
Unrealized loss on available for sale debt securities | (261) | ||||
Net loss attributable to ordinary shareholders | (95,212) | ||||
Balance at Jun. 30, 2022 | 146,272 | $ 16,353 | 949,325 | 26,030 | (845,436) |
Balance, Shares at Jun. 30, 2022 | 126,436,213 | ||||
Balance at Mar. 31, 2022 | 175,646 | $ 16,283 | 945,302 | 8,581 | (794,520) |
Balance, Shares at Mar. 31, 2022 | 125,904,945 | ||||
Share-based compensation expense | 3,956 | 3,956 | |||
Exercise of share options | 1 | $ 24 | (23) | ||
Exercise of share options, Shares | 175,153 | ||||
Issuance of ESPP shares | 136 | $ 46 | 90 | ||
Issuance of ESPP shares, Shares | 356,115 | ||||
Foreign currency translation | 17,450 | 17,450 | |||
Unrealized loss on available for sale debt securities | (1) | (1) | |||
Net loss attributable to ordinary shareholders | (50,916) | (50,916) | |||
Balance at Jun. 30, 2022 | 146,272 | $ 16,353 | 949,325 | 26,030 | (845,436) |
Balance, Shares at Jun. 30, 2022 | 126,436,213 | ||||
Balance at Dec. 31, 2022 | 98,264 | $ 16,419 | 956,711 | 26,018 | (900,884) |
Balance, Shares at Dec. 31, 2022 | 126,947,225 | ||||
Share-based compensation expense | 3,512 | 3,512 | |||
Exercise of share options | 41 | $ 43 | (2) | ||
Exercise of share options, Shares | 332,209 | ||||
Vesting of restricted stock units, net of shares withheld for taxes | (166) | $ 40 | (206) | ||
Vesting of restricted stock units, net of shares withheld for taxes, shares | 310,025 | ||||
Sale of voting and non-voting ordinary shares, net of allocated issuance costs | 23,073 | $ 7,369 | 15,704 | ||
Sale of voting and non-voting ordinary shares, net of allocated issuance costs, Shares | 56,666,900 | ||||
Foreign currency translation | (5,731) | (5,731) | |||
Unrealized loss on available for sale debt securities | 74 | 74 | |||
Net loss attributable to ordinary shareholders | (17,412) | (17,412) | |||
Balance at Mar. 31, 2023 | 101,655 | $ 23,871 | 975,719 | 20,361 | (918,296) |
Balance, Shares at Mar. 31, 2023 | 184,256,359 | ||||
Balance at Dec. 31, 2022 | $ 98,264 | $ 16,419 | 956,711 | 26,018 | (900,884) |
Balance, Shares at Dec. 31, 2022 | 126,947,225 | ||||
Exercise of share options, Shares | 332,180 | ||||
Foreign currency translation | $ (7,501) | ||||
Unrealized loss on available for sale debt securities | 35 | ||||
Net loss attributable to ordinary shareholders | (29,719) | ||||
Balance at Jun. 30, 2023 | 109,047 | $ 29,456 | 991,642 | 18,552 | (930,603) |
Balance, Shares at Jun. 30, 2023 | 227,215,699 | ||||
Balance at Mar. 31, 2023 | 101,655 | $ 23,871 | 975,719 | 20,361 | (918,296) |
Balance, Shares at Mar. 31, 2023 | 184,256,359 | ||||
Share-based compensation expense | 2,578 | 2,578 | |||
Issuance of ESPP shares | 169 | $ 59 | 110 | ||
Issuance of ESPP shares, Shares | 455,550 | ||||
Vesting of restricted stock units, net of shares withheld for taxes | $ 1 | (1) | |||
Vesting of restricted stock units, net of shares withheld for taxes, shares | 3,790 | ||||
Sale of voting and non-voting ordinary shares, net of allocated issuance costs | 18,761 | $ 5,525 | 13,236 | ||
Sale of voting and non-voting ordinary shares, net of allocated issuance costs, Shares | 42,500,000 | ||||
Foreign currency translation | (1,770) | (1,770) | |||
Unrealized loss on available for sale debt securities | (39) | (39) | |||
Net loss attributable to ordinary shareholders | (12,307) | (12,307) | |||
Balance at Jun. 30, 2023 | $ 109,047 | $ 29,456 | $ 991,642 | $ 18,552 | $ (930,603) |
Balance, Shares at Jun. 30, 2023 | 227,215,699 |
Condensed Consolidated Statem_4
Condensed Consolidated Statements of Shareholders' Equity (Unaudited) (Parenthetical) - USD ($) $ in Thousands | 3 Months Ended | |
Jun. 30, 2023 | Mar. 31, 2023 | |
Statement of Stockholders' Equity [Abstract] | ||
Sale of voting and non-voting ordinary shares, net of allocated issuance costs | $ 953 | $ 889 |
Nature of the Business and Liqu
Nature of the Business and Liquidity | 6 Months Ended |
Jun. 30, 2023 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Nature of the Business and Liquidity | 1. Nature of the Business and Liquidity Orchard Therapeutics plc (the “Company”) is a global gene therapy company dedicated to transforming the lives of people affected by severe diseases through the development of innovative, potentially curative gene therapies. The Company’s ex vivo autologous hematopoietic stem cell (“HSC”) gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. The Company has a portfolio that includes a commercial-stage product and research and development-stage product candidates. The Company is a public limited company incorporated pursuant to the laws of England and Wales. The Company has American Depositary Shares (“ADSs”) registered with the U.S. Securities and Exchange Commission (the “SEC”). The ADSs were listed on the Nasdaq Global Select Market on October 31, 2018, and were transferred to the Nasdaq Capital Market on September 13, 2022. The Company’s ADSs each represent ten ordinary shares of the Company. Each holder of ordinary shares is entitled to one vote per ordinary share and to receive dividends when and if such dividends are recommended by the board of directors and declared by the shareholders. The Company did no t declare any dividends in 2023 or 2022. Share information presented in these unaudited condensed consolidated financial statements are presented on an ordinary share basis and not on an ADS converted basis unless otherwise indicated. In January 2022, the Company began to generate revenue from product sales of Libmeldy in Europe following the approval of Libmeldy by the European Commission in December 2020 for the treatment of early onset metachromatic leukodystrophy (“MLD”), characterized by biallelic mutations in the arylsulfatase-A ("ARSA") gene leading to a reduction of the ARSA enzymatic activity in children with (i) late infantile or early juvenile forms, without clinical manifestations of the disease, or (ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. On March 6, 2023, the Company entered into a Securities Purchase Agreement (“SPA” or “PIPE”) pursuant to which the Company agreed to sell ordinary shares, non-voting ordinary shares, and warrants to purchase ordinary shares or non-voting ordinary shares in an unregistered offering (the "2023 Private Placement"). The 2023 Private Placement consists of two closings. On March 10, 2023, the Company completed the first closing and issued and sold (i) 56,666,900 ordinary shares and non-voting ordinary shares, nominal value £ 0.10 per share and (ii) warrants to purchase an aggregate of 62,333,590 ordinary shares or non-voting ordinary shares, at a purchase price of $ 6.00 per unit, where each unit consists of ten shares and an accompanying warrant to purchase eleven shares . The initial closing of the 2023 Private Placement resulted in gross proceeds of $ 34.0 million. On June 22, 2023, the Company completed the second closing of the 2023 Private Placement and sold (i) 42,500,00 ordinary shares and non-voting ordinary shares, nominal value £ 0.10 per share and (ii) warrants to purchase an aggregate of 46,750,000 ordinary shares or non-voting ordinary shares, at a purchase price of $ 8.00 per unit, where each unit consists of ten shares and an accompanying warrant to purchase eleven shares . The second closing of the 2023 Private Placement resulted in gross proceeds of $ 34.0 million. Refer to Note 12 for further discussion of the 2023 Private Placement. The Company’s business is subject to risks and uncertainties common to development-stage companies in the biotechnology industry. There can be no assurance that the Company’s research and development will be successfully completed, that adequate protection for the Company’s technology will be obtained, that any products developed will obtain necessary government regulatory approval or that any products, if approved, will be commercially viable. The Company operates in an environment of rapid technological innovation and substantial competition from pharmaceutical and biotechnology companies. In addition, the Company is dependent upon the services of its employees, consultants and service providers. Even if the Company’s product development efforts are successful in gaining regulatory approval, it is uncertain when, if ever, the Company will realize significant revenue from product sales. Through June 30, 2023, the Company funded its operations primarily with proceeds from the sale of equity securities, including ADSs in the Company’s initial public offering (“IPO”) and follow-on offering, ordinary shares in the private placements, and convertible preferred shares. The Company has also financed its operations through proceeds from the Company’s senior term facilities agreement with MidCap Financial (Ireland) Limited, research grants from the California Institute of Regenerative Medicine (“CIRM”), upfront payments from the Company’s collaboration agreement and share purchase agreement with Pharming Group N.V., proceeds from the sales of the Company's Libmeldy product, and reimbursements associated with two UK research and development tax relief programs, the Small and Medium-sized Enterprises research and development tax credit (“SME”) program and the Research and Development Expenditure (“RDEC”) program. The Company has incurred recurring losses since its inception and expects to continue to generate operating losses for the foreseeable future. The Company expects that its cash, cash equivalents, and marketable securities on hand as of June 30, 2023, of $ 150.7 million, together with expected proceeds from sales of Libmeldy, will be sufficient to fund its operations, capital expenditures and debt service payments for at least twelve months from the date of filing of this Form 10-Q. The Company will seek additional funding through private or public equity financings, debt financings, collaborations, strategic alliances and marketing, distribution or licensing arrangements. The Company may not be able to obtain financing on acceptable terms, or at all, and the Company may not be able to enter into collaborations or other arrangements. The terms of any financing may adversely affect the holdings or the rights of the Company's shareholders. The future viability of the Company is dependent on its ability to raise additional capital to finance its operations. If the Company is unable to obtain funding, the Company will be forced to delay, reduce or eliminate some or all of its research and development programs, product portfolio expansion or commercialization efforts, which could adversely affect its business prospects, or the Company may be unable to continue operations. Although management continues to pursue these plans, there is no assurance that the Company will be successful in obtaining sufficient funding on terms acceptable to the Company to fund continuing operations, if at all. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 6 Months Ended |
Jun. 30, 2023 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies The Company's significant accounting policies are disclosed in the audited consolidated financial statements for the year ended December 31, 2022, and notes thereto, which are included in the Company's Annual Report on Form 10-K filed with the SEC on March 14, 2023 (the “Annual Report”). Since the date of those financial statements there have been no material changes to the Company's significant accounting policies other than those discussed below. Basis of presentation These condensed consolidated financial statements of the Company are unaudited and have been prepared in accordance with U.S. generally accepted accounting principles (“GAAP”) for interim financial reporting and in accordance with Regulation S-X, Rule 10-01. Accordingly, they do not include all of the information and footnotes required by GAAP for complete financial statements. Any reference in these notes to applicable guidance is meant to refer to the authoritative GAAP as found in the Accounting Standards Codification (“ASC”), and Accounting Standards Update (“ASU”), of the Financial Accounting Standards Board (“FASB”). These unaudited condensed consolidated financial statements should be read in conjunction with the audited consolidated financial statements and accompanying notes included in the Company’s Annual Report. The condensed consolidated balance sheet as of December 31, 2022, was derived from audited consolidated financial statements included in the Company’s Annual Report but does not include all disclosures required by GAAP. Certain information and footnote disclosures normally included in financial statements prepared in accordance with GAAP have been condensed or omitted from these interim financial statements. However, these interim financial statements include all adjustments, consisting only of normal recurring adjustments, which are, in the opinion of the Company’s management, necessary to fairly state the results of the interim period. The interim results are not necessarily indicative of results to be expected for the full year. Amounts reported are presented in thousands, except percentages, per share amounts or as otherwise noted. As a result, certain totals may not sum due to rounding. Principles of consolidation The accompanying unaudited condensed consolidated financial statements include the accounts of the Company and its wholly owned subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. Use of estimates The preparation of unaudited condensed consolidated financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the unaudited condensed consolidated financial statements and the reported amounts of revenue and expenses during the reporting periods. Significant estimates and assumptions reflected in these unaudited condensed consolidated financial statements include, but are not limited to, the accrual for research and development expenses, the research and development tax credit receivable, share-based compensation, collaboration agreement milestones, variable consideration in revenue recognition, operating lease assets and liabilities, valuation of PIPE Warrants and PIPE Units, and income taxes. Estimates are periodically reviewed in light of changes in circumstances, facts and experience. Actual results could differ from the Company’s estimates. Foreign currency The financial statements of the Company’s subsidiaries with functional currencies other than the U.S. Dollar are translated into U.S. Dollars using period-end exchange rates for assets and liabilities, historical exchange rates for shareholders’ equity and weighted average exchange rates for operating results. Unrealized losses are driven primarily by intercompany balances denominated in currencies other than the functional currency of the entity with the intercompany balance, and typically fluctuates concurrently with fluctuations in the U.S. Dollar, Pounds sterling, and Euro exchange rates. Translation gains and losses are included in accumulated other comprehensive income (loss) in shareholders’ equity. Foreign currency transaction gains and losses are included in other income (expense), net in the results of operations. The Company recorded realized and unrealized foreign currency transactio n losses of $ 7.5 million for the six months ended June 30, 2023 , and realized and unrealized foreign currency transaction losses of $ 24.3 million for the six months ended June 30, 2022 , which is included in other income (expense), net in the condensed consolidated statements of operations and comprehensive loss. Accounts receivable Accounts receivable arise from product revenue and amounts due from the Company's collaboration partners and have payment terms that generally require payment within 30 to 90 days. For some Libmeldy customers, our payment terms can range from 30 days to under one year . The amount from product revenue represents amounts due from distributors in Europe, which are recorded net of reserves for trade discounts and allowances, and other incentives to the extent such amounts are payable to the customer by the Company. The Company monitors economic conditions to identify facts or circumstances that may indicate that its receivables are at risk of collection. The Company provides reserves against accounts receivable for estimated losses, if any, that may result from a customer's inability to pay based on the composition of its accounts receivable, current economic conditions, and historical credit loss activity. Amounts determined to be uncollectible are charged or written-off against the reserve. During the three and six months ended June 30, 2023, the Company did no t record any expected credit losses related to outstanding accounts receivable. Restricted cash Cash and cash equivalents that are restricted as to withdrawal or use under the terms of certain contractual agreements are recorded as restricted cash on the Company’s condensed consolidated balance sheets. The Company has an outstanding letter of credit for $ 3.0 million associated with a lease and is required to hold this amount in a standalone bank account, as of June 30, 2023 , and December 31, 2022. The Company is also contractually required to maintain cash collateral accounts associated with corporate credit cards and other leases in the amount of $ 1.3 million at June 30, 2023 and December 31, 2022. Cash, cash equivalents, and restricted cash were comprised of the following (in thousands): As of June 30, As of December 31, 2023 2022 Cash and cash equivalents $ 38,273 $ 68,424 Restricted cash 4,215 4,215 Total cash, cash equivalents and restricted cash shown in the statement of cash flows $ 42,488 $ 72,639 Recently adopted accounting pronouncements In March 2020, the FASB issued ASU 2020-04 , Reference Rate Reform (Topic 848) : Facilitation of the Effects of Reference Rate Reform on Financial Reporting and issued two subsequent amendments: ASU 2021-01, issued in January 2021, refines the scope of ASU and clarifies some of its guidance as part of the FASB’s monitoring of global reference rate reform activities and ASU 2022-06, issued in December 2022, which extends the effective period of the ASU through December 31, 2023 (collectively, including ASU 2020-04, “ASC 848”). ASC 848 provides temporary optional expedients and exceptions to the GAAP guidance on contract modifications and hedge accounting to ease the financial reporting burdens related to the expected market transition from the London Interbank Offered Rate (“LIBOR”) and other interbank offered rates to alternative reference rates. ASC 848 is effective for all entities as of March 12, 2020, through December 31, 2024, at which time transition is expected to be complete. The Company adopted this standard as of January 1, 2023 . The application of this ASU did not have a material impact on the Company's financial position, results of operations, or cash flows. |
Fair Value Measurements and Mar
Fair Value Measurements and Marketable Securities | 6 Months Ended |
Jun. 30, 2023 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurements and Marketable Securities | 3. Fair value measurements and marketable securities Fair value measurements The following tables present information about the Company’s financial assets and liabilities that have been measured at fair value as of June 30, 2023, and December 31, 2022, and indicate hierarchy of valuation inputs utilized to determine such fair value (in thousands): Fair Value Measurements as of Level 1 Level 2 Level 3 Total Assets Cash equivalents Money market funds $ 3,305 $ — $ — $ 3,305 U.S. treasuries — 1,995 — 1,995 Commercial paper — 6,989 — 6,989 Total cash equivalents $ 3,305 $ 8,984 $ — $ 12,289 Marketable securities U.S. government securities $ — $ 12,326 $ — $ 12,326 U.S. treasuries — 14,277 — 14,277 Corporate bonds — 16,118 — 16,118 Commercial paper — 69,747 — 69,747 Total marketable securities $ — $ 112,468 $ — $ 112,468 Total Assets $ 3,305 $ 121,452 $ — $ 124,757 Liabilities PIPE Warrant liability $ — $ — $ 12,266 $ 12,266 Total Liabilities $ — $ — $ 12,266 $ 12,266 Fair Value Measurements as of Level 1 Level 2 Level 3 Total Assets Cash equivalents Money market funds $ 1,239 $ — $ — $ 1,239 U.S. treasuries — 6,600 — 6,600 U.S. government securities — 5,200 — 5,200 Commercial paper — 14,122 — 14,122 Total cash equivalents $ 1,239 $ 25,922 $ — $ 27,161 Marketable securities U.S. government securities $ — $ 1,984 $ — $ 1,984 Corporate bonds — 25,475 — 25,475 Commercial paper — 47,867 — 47,867 Total marketable securities $ — $ 75,326 $ — $ 75,326 Total Assets $ 1,239 $ 101,248 $ — $ 102,487 The Company classifies its money market funds as Level 1 assets since it measures fair value using quoted prices in active markets for identical assets. The Level 2 assets include commercial paper, U.S. government securities, U.S. treasuries, and corporate bonds and are valued based on quoted prices for similar assets in active markets and inputs other than quoted prices that are derived from observable market data. The Company evaluates transfers between levels at the end of each reporting period. There were no transfers between Level 1 and Level 2 assets during the periods presented. The PIPE Warrants (as defined in Note 12) and PIPE Units (as defined in Note 12) associated with the 2023 Private Placement did not meet the criteria for equity classification, and are therefore accounted for as liabilities at fair value. Refer to Note 12 for the accounting analysis of the PIPE Warrants and PIPE Units. The fair values of the PIPE Warrants and PIPE Units are based on significant unobservable inputs, which represent Level 3 measurements within the fair value hierarchy. The fair value of the PIPE Warrants, inclusive of the warrants to be issued in the Second Closing and the warrants ultimately issued in the Second Closing, was estimated using the Black-Scholes option pricing model, which was then adjusted by the weighted probability of the timing of the anticipated achievement of FDA marketing approval for the sale of Libmeldy in the United States and further adjusted by the probability of the occurrence of achievement of FDA marketing approval for the sale of Libmeldy in the United States. The fair value of the PIPE Units prior to the second closing of the 2023 Private Placement was estimated as the residual of the fair value of the share and warrant units to be sold in the second closing minus the second closing sales price of the share and warrant units to be sold. To calculate the fair value of the shares to be sold in the second closing, the Company utilized the stock price of its ADSs as of the valuation date and discounted it using a discount for lack of marketability ("DLOM"). The second closing was completed in June 2023, upon which, the Company recorded a final fair value adjustment and derecognized the value ascribed to the PIPE Units instrument. The fair value of the PIPE Units at the time of the second closing was calculated by estimating the fair value of the PIPE Warrants and the unregistered shares issued in the second closing. To calculate the fair value of the shares issued in the second closing, the Company utilized the stock price of its ADSs as of the valuation date and discounted it using a DLOM relating to the unregistered status of the shares. The significant unobservable inputs used in the valuation models during the three months ended June 30, 2023, to measure the fair value of the PIPE Warrants and PIPE Units are as follows: PIPE Unit Liability PIPE Warrant Liability Valuation date June 22, 2023 June 30, 2023 Expected warrant exercise date July 30, 2024 April 30, 2025 July 30, 2024 April 30, 2025 Black-Scholes inputs Anticipated common stock price (at exercise date) $ 10.40 $ 10.07 $ 10.39 $ 10.07 Expected term (in years) 1.11 1.86 1.08 1.84 Expected volatility 75.0 % 74.8 % 75.6 % 74.5 % Other significant unobservable inputs Implied probabilities of occurrence of Second Closing and FDA Approval 30.5 % 34.6 % Discount for lack of marketability 5.0 % 0 % The following table provides a rollforward of the aggregate fair values of the Company’s PIPE Warrant liability and PIPE Unit liability, for which fair values are determined using Level 3 inputs (in thousands): PIPE Warrant Liability PIPE Unit Liability Balance at December 31, 2022 $ — $ — Issuances during period 11,098 25 Change in fair value 1,168 ( 9,668 ) Settlement of PIPE Unit liability upon Second Closing — 9,643 Balance at June 30, 2023 $ 12,266 $ - Marketable securities The following tables summarize the amortized cost and fair value of the Company's available-for-sale marketable debt securities (in thousands): June 30, 2023 Amortized Gross Gross Credit Losses Fair Value U.S. government securities $ 12,338 $ 1 $ ( 13 ) $ — $ 12,326 U.S. treasuries 16,280 — ( 8 ) — $ 16,272 Corporate bonds 16,232 — ( 114 ) — 16,118 Commercial paper 76,801 — ( 65 ) — 76,736 Total $ 121,651 $ 1 $ ( 200 ) $ — $ 121,452 December 31, 2022 Amortized Gross Gross Credit Losses Fair Value U.S. government securities $ 7,188 $ 1 $ ( 6 ) $ — $ 7,183 U.S. treasuries 6,599 1 — — $ 6,600 Corporate bonds 25,656 — ( 180 ) — $ 25,476 Commercial paper 62,038 3 ( 52 ) — 61,989 Total $ 101,481 $ 5 $ ( 238 ) $ — $ 101,248 All investments in an unrealized loss position were in this position for less than 12 months. The Company evaluated its securities for potential impairment and considered the decline in market value to be primarily attributable to current economic and market conditions. Additionally, the Company does not intend to sell the securities in an unrealized loss position and does not expect it will be required to sell the securities before recovery of the unamortized cost basis. Given the Company's intent and ability to hold such securities until recovery, and the lack of a significant change in credit risk for these investments, the Company does not consider these investments to be impaired as of June 30, 2023. There were no realized gains or losses recognized on investments for the three or six months ended June 30, 2023. The following table summarizes the Company’s debt securities by contractual maturity, as of June 30, 2023, and December 31, 2022 (in thousands): June 30, December 31, Maturities in one year or less $ 117,460 $ 98,277 Maturities between one and three years 3,992 2,971 Total $ 121,452 $ 101,248 |
Inventory
Inventory | 6 Months Ended |
Jun. 30, 2023 | |
Inventory Disclosure [Abstract] | |
Inventory | 4. Inventory Inventory consists of the following (in thousands): June 30, December 31, 2023 2022 Raw materials $ 6,712 $ 3,193 Work in process 225 207 Total inventory $ 6,937 $ 3,400 The Company currently has $ 4.8 million of inventory capitalized related to Libmeldy in Europe that is currently in the process of regulatory review. |
Prepaid Expenses and Other Curr
Prepaid Expenses and Other Current Assets | 6 Months Ended |
Jun. 30, 2023 | |
Prepaid Expense and Other Assets, Current [Abstract] | |
Prepaid Expenses and Other Current Assets | . Prepaid expenses and other current assets Prepaid expenses and other current assets consist of the following (in thousands): June 30, December 31, 2023 2022 Prepaid external research and development expenses $ 378 $ 881 Other prepayments 2,998 1,817 VAT receivable 712 1,077 Non-trade receivables 419 1,851 Rent deposits 1,033 960 Total prepaid expenses and other current assets $ 5,540 $ 6,586 |
Research and Development Tax Cr
Research and Development Tax Credit Receivable | 6 Months Ended |
Jun. 30, 2023 | |
Research and Development Tax Credit Receivable [Abstract] | |
Research and Development Tax Credit Receivable | 6. Research and development tax credit receivable The following table below outlines the changes to the research and development tax credit receivable, including amounts recognized as an offset to research and development expense during the periods (amounts in thousands): Three Months Ended Six Months Ended 2023 2022 2023 2022 Balance at beginning of period $ 6,844 $ 16,649 $ 5,942 $ 30,723 Recognition of credit claims as offset to research and development expense 3,602 1,764 4,371 5,101 Receipt of credit claims — ( 1,916 ) - ( 18,390 ) Foreign currency translation 180 ( 1,321 ) 313 ( 2,258 ) Balance at end of period $ 10,626 $ 15,176 $ 10,626 $ 15,176 The Company's tax incentive receivable from the UK government was approximately $ 10.6 million as of the June 30, 2023. During the three months ended June 30, 2023, the Company recorded $ 2.3 million of additional tax credits related to a change in estimate associated with its U.K. research and development tax credit receivable claim for fiscal year 2022 ($ 8.5 million) and estimate of its 2023 claim through June 30, 2023 ($ 2.1 million). The change in estimate was based on the results of a tax credit analysis associated with the Company’s qualified projects and research and development expenditures completed during the quarter to finalize the 2022 U.K. tax return. This amount was recorded as an offset to research and development expense. |
Intangible Assets, Net
Intangible Assets, Net | 6 Months Ended |
Jun. 30, 2023 | |
Goodwill and Intangible Assets Disclosure [Abstract] | |
Intangible Assets, Net | . Intangible assets, net Intangible assets, net of accumulated amortization, consisted of the following (in thousands): As of June 30, 2023 Cost Accumulated Amortization Net License milestones $ 4,168 $ ( 694 ) $ 3,474 Total $ 4,168 $ ( 694 ) $ 3,474 As of December 31, 2022 Cost Accumulated Amortization Net License milestones $ 4,069 $ ( 509 ) $ 3,560 Total $ 4,069 $ ( 509 ) $ 3,560 License intangibles consist of capitalized milestone payments made upon receiving regulatory approval of Libmeldy in the EU. The license intangibles are being amortized on a straight-line basis over the remaining useful life of the rela ted patents of approximately twelve years . For the three and six months ended June 30, 2023 , amortization of intangible assets totaled $ 0.1 million and $ 0.2 million, respectively. For the three and six months ended June 30, 2022 , amortization of intangible assets totaled $ 0.1 million and $ 0.2 million, respectively. The effect of foreign currency translation on the net carrying value of intangible assets for the three and six months ended June 30, 2023 , was $ 0.1 million. The effect of foreign currency translation on the net carrying value of intangible assets for each of the three and six months ended June 30, 2022 , was $ 0.2 million. T he following table summarizes the estimated future amortization for intangible assets for the next five years and thereafter (in thousands): As of June 30, 2023 (remaining six months) $ 171 2024 342 2025 342 2026 342 2027 342 Thereafter 1,935 Total $ 3,474 |
Accrued Expenses and Other Curr
Accrued Expenses and Other Current Liabilities | 6 Months Ended |
Jun. 30, 2023 | |
Accrued Liabilities and Other Liabilities [Abstract] | |
Accrued Expenses and Other Current Liabilities | 8. Accrued expenses and other current liabilities Accrued expenses and other current liabilities consisted of the following (in thousands): June 30, December 31, 2023 2022 Accrued external research and development expenses $ 10,985 $ 11,230 Accrued payroll and related expenses 9,132 12,312 Accrued professional fees 2,588 2,263 Accrued other 3,459 2,647 Accrued governmental rebates 4,005 2,300 Strimvelis liability - current portion 3,617 3,685 Total accrued expenses and other current liabilities $ 33,786 $ 34,437 |
Notes Payable
Notes Payable | 6 Months Ended |
Jun. 30, 2023 | |
Debt Disclosure [Abstract] | |
Notes Payable | . Notes payable In May 2019 , the Company entered into a senior term facilities agreement, which was amended in April 2020 (the “Original Credit Facility”) with MidCap Financial (Ireland) Limited (“MidCap Financial”), as agent, and additional lenders from time to time (together with MidCap Financial, the “Lenders”), to borrow up to $ 75.0 million in term loans. In May 2021, the Company amended and restated the Original Credit Facility (the “Amended Credit Facility”). Under the Amended Credit Facility, the Lenders agreed to make term loans available to the Company in the aggregate amount of $ 100.0 million, including increasing the principal on the initial term loan to $ 33.0 million, from $ 25.0 million. In January 2023, the Company amended and restated the credit facility to change the reference rate from the London Interbank Offered Rate ("LIBOR") to the Secured Overnight Financing Rate ("SOFR"). As of June 30, 2023, the Company has borrowed $ 33.0 million under the Amended Credit Facility. In March 2023, the Company notified MidCap Financial of its voluntary cancellation of each Lender's remaining commitments under the Amended Credit Facility. As a result of this notice, the Company can no longer draw on the remaining $ 67.0 million that was available on the Amended Credit Facility. Additionally, MidCap agreed to waive certain restrictive covenants of the Amended Credit Facility, specifically related to restrictions on the Company's ability to dispose of intellectual property related to deprioritized products. The Company's borrowings under the Amended Credit Facility bear interest at an annual rate equal to 5.95 % plus SOFR plus a 0.10 % annual increase to the annual rate. The Company was required to make interest only payments on the term loan for 18 months following the date of the Amended Credit Facility. The term loan under the Amended Credit Facility began amortizing on the 18-month anniversary of the Amended Credit Facility (December 2022), with the Company commencing equal monthly payments of principal plus interest to the Lenders to be made in consecutive monthly installments until the loan maturity date in May 2026. In addition, a final payment of 3.5 % is due on the loan maturity date. The Company is accruing the final payment amount of $ 1.2 million associated with the Amended Credit Facility, to outstanding debt by charges to interest expense using the effective-interest method from the date of issuance through the loan maturity date. The Amended Credit Facility includes affirmative and negative covenants. The affirmative covenants include, among others, covenants requiring the Company to maintain their legal existence and governmental approvals, deliver certain financial reports, maintain insurance coverage, maintain property, pay taxes, satisfy certain requirements regarding accounts and comply with laws and regulations. The negative covenants include, among others, restrictions on the Company transferring collateral, incurring additional indebtedness, engaging in mergers or acquisitions, paying dividends or making other distributions, making investments, creating liens, amending material agreements and organizational documents, selling assets, changing the nature of the business and undergoing a change in control, subject to certain exceptions. Notes payable consist of the following (in thousands): June 30, December 31, 2023 2022 Notes payable, net of issuance costs $ 27,297 $ 31,970 Less: current portion ( 9,429 ) ( 9,429 ) Notes payable, net of current portion 17,868 22,541 Accretion related to final payment 572 450 Notes payable, long term $ 18,440 $ 22,991 As of June 30, 2023, the estimated future principal payments due are as follows (in thousands): Aggregate 2023 (remaining six months) $ 4,714 2024 9,429 2025 9,429 2026 5,084 Total 28,656 Less current portion ( 9,429 ) Less unamortized portion of final payment ( 584 ) Less unamortized debt issuance costs ( 203 ) Notes payable, long term $ 18,440 During the three months ended June 30, 2023 and 2022, the Company recognized $ 0.9 million and $ 0.7 million of interest expense related to the term loan, respectively. During the six months ended June 30, 2023 and 2022, the Company recognized $ 1.9 million and $ 1.3 million of interest expense related to the term loan, respectively. The effective annual interest rate as of June 30, 2023, was approximately 12.38 %. |
Shareholders' Equity
Shareholders' Equity | 6 Months Ended |
Jun. 30, 2023 | |
Equity [Abstract] | |
Shareholders' Equity | 10. Shareholders ’ equity The Company ’ s ordinary shares are divided into two classes: (i) Ordinary Shares and (ii) Non-Voting Ordinary Shares. The Non-Voting Ordinary Shares have the same rights and restrictions, shall be subject to the same obligations and liabilities, and shall otherwise rank pari passu in all respects with the Ordinary Shares except as set out below: • a holder of Non-Voting Ordinary Shares shall, in relation to the Non-Voting Ordinary Shares held by such holder, have no right to receive notice of, or to attend or vote at, any general meeting of shareholders (save in relation to a variation of class rights of the Ordinary Shares); and • the Non-Voting Ordinary Shares shall be non-transferable. A holder of the Non-Voting Ordinary Shares may elect to have some or all of such holder’s Non-Voting Ordinary Shares redesignated as Ordinary Shares by providing a written notice in a form reasonably acceptable to the Company, specifying the number of Non-Voting Ordinary Shares it wishes to have redesignated as Ordinary Shares. Upon the redesignation of the Non-Voting Ordinary Shares to Ordinary Shares, such Ordinary Shares shall rank pari passu with the other Ordinary Shares of the Company in all respects. The holders of Non-Voting Shares are subject to certain ownership restrictions as described in the Securities Purchase Agreement, dated March 6, 2023 (the "Purchase Agreement"), including that no purchaser of Non-Voting Ordinary Shares may redesignate such shares if the holder would own in excess of 19.99 % of the number of Ordinary Shares (including Ordinary Shares that may be represented by ADSs) outstanding immediately after giving effect to such redesignation. At the Company's Annual General Meeting of Shareholders in June 2023, the Company received authority to allot (1) up to a maximum nominal value of £ 15,158,359 to any new shares (including both Ordinary Shares and Non-Voting Ordinary Shares) pursuant to the Purchase Agreement (the "PIPE Authority") and (2) up to a maximum nominal value of £ 18,398,450 to any new shares (including both Ordinary Shares and Non-Voting Ordinary Shares) (the "General Authority"). The General Authority was in addition to the previous authority granted to the Board of Directors on June 16, 2021 to grant shares up to a maximum aggregate nominal amount of £ 13,023,851.50 , of which approximately £ 5,526,095.70 remains (the "2021 Authority"). As of June 30, 2023, the Company had 41,613,400 Ordinary Non-Voting shares issued and outstanding and had 185,602,299 Ordinary Shares issued and outstanding. As of June 30, 2023, the Company has a remaining nominal value of £ 10,908,359 it can issue in new shares under the PIPE Authority and £ 23,924,545.70 it can issue in any new shares under the General Authority. Holders of Ordinary Shares may convert such shares to ADSs. The ADSs are listed on The Nasdaq Capital Market, and each ADS represents ten Ordinary Shares. We also have in issue one deferred share with a nominal value of £ 4.89687 . To date, the Company has not declared any dividends. Under English law, a company’s accumulated realized profits must exceed its accumulated realized losses (on a non-consolidated basis) before dividends can be declared and paid. Therefore, the Company must have distributable profits before declaring and paying a dividend. |
Share-based Compensation
Share-based Compensation | 6 Months Ended |
Jun. 30, 2023 | |
Share-Based Payment Arrangement [Abstract] | |
Share-based Compensation | 11 . Share-based compensation The Company maintains four equity compensation plans: the Orchard Therapeutics Limited Employee Share Option Plan with Non-Employee Sub-Plan and U.S. Sub-Plan (the “2016 Plan”), the Orchard Therapeutics plc 2018 Share Option and Incentive Plan (the “2018 Plan”), the 2018 Employee Share Purchase Plan (the “ESPP”), and the 2020 Inducemen t Equity Plan (the “Inducement Plan”). The board of directors has determined it will not make any further awards under the 2016 plan. As of June 30, 2023, there were 12,421,580 ordinary shares available for grant under the 2018 Plan, 730,500 ordinary shares available for grant under the Inducement Plan, and 1,460,590 ordinary shares available for grant under the ESPP. The number of options and restricted stock units, the weighted average grant date fair value per stock option and per share, and the weighted average exercise price in the tables below are all shown below on a per ordinary share basis. The Company’s ADSs that are listed on the NASDAQ Capital Market each represent ten ordinary shares. Share option activity The following table summarizes option activity for ordinary shares under the plans for the six months ended June 30, 2023: Shares Weighted Outstanding at December 31, 2022 16,424,167 $ 1.56 Granted 5,085,330 0.49 Exercised ( 332,180 ) 0.12 Forfeited ( 795,167 ) 4.27 Outstanding at June 30, 2023 20,382,150 $ 1.21 Vested and expected to vest at June 30, 2023 20,382,150 $ 1.21 Exercisable at June 30, 2023 10,452,810 $ 1.82 The total intrinsic value of options exercised was $ 0.1 million for the three and six months ended June 30, 2023. T he weighted-average grant date fair value of ordinary share options granted during the six months ended June 30, 2023 and 2022, was $ 2.81 and $ 0.32 per ordinary share, respectively . As of June 30, 2023, total unrecognized compensation cost related to options was $ 10.4 million. This amount is expected to be recognized over a weighted average period of 2.59 years. Restricted share units Performance-based restricted share units In April 2020, the Company granted 195,000 performance-based restricted share units (“RSUs”) with a total grant date fair value of $ 1.4 million to its Chief Executive Officer, Bobby Gaspar, M.D., Ph.D. Dr. Gaspar earns one-third of the award for each of the first three to occur of four milestones ( 65,000 RSUs are earned for each achieved milestone). The milestones relate to specific clinical and regulatory goals which have to be achieved before December 31, 2023 in order for the underl ying RSUs to be eligible to vest. Vesting for any earned shares occurs on January 2, 2024, so long as Dr. Gaspar remains continuously employed with the Company through that date. The Company determined that two of the milestones were probable of being achieved and recognized stock-compensation expense of $ 0.1 million and $ 0.8 million for the three and six months ended June 30, 2023. Time-based restricted share units Time-based restricted share units generally vest in equal annual installments over a three-year period. Restricted share unit activity The following table summarizes award activity for the six months ended June 30, 2023: Performance-based RSUs Time-based RSUs Total RSUs Weighted Average Grant Date Fair Value per Share Unvested at December 31, 2022 195,000 1,868,876 2,063,876 $ 0.55 Granted — 1,700,200 1,700,200 0.46 Vested — ( 609,170 ) ( 609,170 ) 0.52 Forfeited — ( 152,806 ) ( 152,806 ) 0.46 Unvested at June 30, 2023 195,000 2,807,100 3,002,100 $ 0.51 As of June 30, 2023, total unrecognized compensation cost related to time-based RSUs was $ 1.1 million. This amount is expected to be recognized over a weighted average period of 2.11 years. As of June 30, 2023, the total unrecognized compensation cost related to performance-based RSUs for which vesting was deemed probable was $ 0.1 million, which is expected to be recognized over a period of 0.5 years. As of June 30, 2023, the total unrecognized compensation cost related to performance-based RSUs for which vesting was deemed not probable was $ 0.5 million, the timing of recognition will be dependent upon achievement of remaining milestones. Share-based compensation expense Share-based compensation expense related to share options, restricted share unit awards, and the employee stock purchase plan was classified in the unaudited condensed consolidated statements of operations and comprehensive loss as follows (in thousands): Three Months Ended Six Months Ended 2023 2022 2023 2022 Research and development $ 1,025 $ 1,744 $ 2,261 $ 3,706 Selling, general and administrative 1,553 2,212 3,829 4,910 Total $ 2,578 $ 3,956 $ 6,090 $ 8,616 Total share-based compensation by award type was as follows (in thousands): Three Months Ended Six Months Ended 2023 2022 2023 2022 Share options $ 2,253 $ 3,774 $ 4,808 $ 8,282 Restricted share units 325 182 1,282 334 Total $ 2,578 $ 3,956 $ 6,090 $ 8,616 |
Private Placement Transaction
Private Placement Transaction | 6 Months Ended |
Jun. 30, 2023 | |
Equity [Abstract] | |
Private placement transaction | 12. Private placement transaction On March 6, 2023, the Company entered into the SPA with several investors, pursuant to which the Company agreed to sell, in an unregistered offering (the “2023 Private Placement”), up to an aggregate of (i) 99,166,900 ordinary shares and non-voting ordinary shares, nominal value £ 0.10 per share (collectively, the “Shares”) and (ii) warrants to purchase an aggregate of 109,083,590 Shares. The 2023 Private Placement consisted of two closings, referred to herein as the "First Closing" and "Second Closing". At each closing, the Shares were sold in fixed combinations with the warrants as units, with each unit consisting of 10 Shares and 1 accompanying warrant to purchase 11 shares . The warrants are contingently exercisable for 30 days following (i) the Company’s public announcement of its receipt of marketing approval of its biologics license application (“BLA”) submitted to the FDA with respect to OTL-200 (the “Vesting Event”) and (ii) receipt of shareholder approval to increase the number of authorized shares. Each warrant will have an exercise price equal to $ 1.10 per share in the event the Vesting Event occurs on or prior to December 31, 2024, and $ 0.95 per share in the event the Vesting Event occurs after December 31, 2024. The warrants will expire at the conclusion of the 30 -day exercise period or on March 10, 2026, whichever is earlier. Each warrant holder has the option, in their sole discretion, to exercise the warrants for either ordinary shares or non-voting ordinary shares. On March 10, 2023, the Company completed the First Closing and issued and sold (i) 40,053,500 ordinary shares, (ii) 16,613,400 non-voting ordinary shares, and (iii) warrants to purchase an aggregate of 62,333,590 ordinary shares or non-voting ordinary shares, at a purchase price of $ 6.00 per unit, generating gross proceeds of $ 34.0 million, before deducting offering expenses of $ 0.9 million payable by the Company. On June 22, 2023, the Company completed the Second Closing and issued and sold (i) 13,408,074 ordinary shares, (ii) 29,091,926 non-voting ordinary shares, and (iii) warrants to purchase an aggregate of 46,750,000 ordinary shares or non-voting ordinary shares, at a purchase price of $ 8.00 per unit (collectively, the “PIPE Units”). The Second Closing was contingent upon (x) the Company’s announcement of its intention to file a BLA submission following receipt of the minutes from the U.S. Food and Drug Administration (“FDA”) in connection with the Company’s pre-BLA meeting for OTL-200, provided such minutes do not expressly advise the Company not to proceed with a BLA submission, and (y) shareholder approval to increase the number of authorized shares. The Company generated gross proceeds of $ 34 million from the Second Closing before deducting offering expenses of $ 1.2 million payable by the Company. The warrants issued in the First closing and the Second Closing of the 2023 Private Placement are referred to collectively herein as the “PIPE Warrants”. Accounting Analysis Upon execution of the SPA on March 6, 2023, the Company determined that each of the instruments to be issued in the First Closing and Second Closing did not meet the criteria for equity classification as they were not considered indexed to the Company’s stock. The Company recorded these instruments at a fair value of $ 34.0 million, which was comprised of $ 27.5 million, $ 6.4 million, and $ 0.02 million allocated to the Shares, PIPE Warrants, and PIPE Units, respectively. The Company incurred issuance costs of $ 0.5 million in connection with executing the SPA, which were expensed upon execution within other income (expense) on the unaudited condensed consolidated statements of operations and comprehensive loss. Upon the First Closing on March 10, 2023, the Company determined that the ordinary shares met the criteria for equity classification. The PIPE Warrants issued in the First Closing and the PIPE Units that were issued in the Second Closing did not meet the criteria for equity classification due to the lack of sufficient authorized and unissued shares and the price adjustment feature depending on the timing of the occurrence of a Vesting Event. The Company reclassified the ordinary shares to equity and recorded a mark-to-market adjustment of a $ 3.6 million gain, which represents the change in fair value of the ordinary shares between March 6, 2023, and March 10, 2023. As a result of the First Closing, the Company recorded $ 24.0 million to equity. The Company incurred issuance costs of $ 1.1 million which were contingent on the closing of the SPA. These issuance costs were allocated between the ordinary shares, PIPE Warrants, and PIPE Units, on a relative fair value basis. Issuance costs of $ 0.9 million allocated to the ordinary shares were recognized as a discount to the ordinary shares recorded in permanent equity. Issuance costs of $ 0.2 million allocated to the PIPE Warrants and PIPE Units were expensed within other income (expense) on the unaudited condensed consolidated statement of operations and comprehensive loss. Upon the Second Closing on June 22, 2023, the PIPE Units were settled. The Company recognized a final fair value adjustment to the PIPE Units immediately prior to the Second Closing. The fair value adjustment was a $ 9.7 million gain. Upon issuance of the shares and warrants under the Second Closing, the Company recorded $ 19.7 million to equity, recorded $ 4.6 million to the PIPE Warrants liability, and derecognized the PIPE Units asset of $ 9.6 million. The Company incurred issuance costs of $ 1.2 million. These issuance costs were allocated between the ordinary shares and the PIPE Warrants on a relative fair value basis. Issuance costs of $ 1.0 million allocated to the ordinary shares were recognized as a discount to the ordinary shares recorded in permanent equity. Issuance costs of $ 0.2 million allocated to the PIPE Warrants were expensed within other income (expense) on the unaudited condensed consolidated statement of operations and comprehensive loss. The PIPE Warrants are classified within non-current liabilities on the unaudited condensed consolidated balance sheets as of June 30, 2023, and will be adjusted to fair value at each subsequent balance sheet date until the warrants are reclassified to equity or settled. Changes in the fair value of the PIPE Warrants are recognized as a component of other income (expense) in the unaudited condensed consolidated statement of operations and comprehensive loss. For the three and six months ended June 30, 2023, the Company recognized changes in fair value resulting in a loss of $ 1.4 million and $ 1.2 million, respectively, related to the PIPE Warrants. As of June 30, 2023, warrants to purchase an aggregate of 109,083,590 ordinary shares or non-voting ordinary shares were issued and outstanding, and no ne were exercisable. The PIPE Units are no longer outstanding as of June 30, 2023. Changes in the fair value of the PIPE Units were recognized as a component of other income (expense), net i n the unaudited condensed consolidated statement of operations and comprehensive loss. For the three and six months ended June 30, 2023, the Company recognized changes in fair value resulting in a gain of $ 9.7 million and $ 9.6 million , respectively, related to the PIPE Units. |
Restructuring Charges
Restructuring Charges | 6 Months Ended |
Jun. 30, 2023 | |
Restructuring and Related Activities [Abstract] | |
Restructuring Charges | . Restructuring charges On March 30, 2022, the Company announced its commitment to focus on severe neurometabolic diseases and early research programs, and to discontinue its investment in and seek strategic alternatives for the Company’s programs in rare primary immune deficiencies, including OTL-103 for treatment of Wiskott Aldrich syndrome (“WAS”), OTL-102 for treatment of X-linked chronic granulomatous disease (“X-CGD”), and Strimvelis for adenosine deaminase severe combined immunodeficiency (“ADA-SCID”). The Company recognized a one-time charge in the first six months of 2022 of approximately $ 1.9 million, which relates to employee-related termination costs. For the six months ended June 30, 2022, approximately $ 1.6 million and $ 0.4 million is recognized in research and development expenses and selling, general, and administrative expenses, respectively, on the Company's unaudited condensed consolidated statements of operations and comprehensive loss. The restructuring charge was included in accrued expenses and other current liabilities in the Company’s unaudited condensed consolidated balance sheets. Activity for the six months ended June 30, 2022, is summarized as follows (in thousands): Three Months Ended June 30, 2022 Six Months Ended June 30, 2022 Balance at beginning of period $ 2,481 $ 6 Charged to expense - 2,481 Non-cash adjustments and foreign currency translation ( 554 ) ( 554 ) Payments made ( 1,089 ) ( 1,095 ) Balance at end of period $ 838 $ 838 There was no restructuring activity in the six months ended June 30, 2023. |
Product Revenue, Net
Product Revenue, Net | 6 Months Ended |
Jun. 30, 2023 | |
Revenue from Contract with Customer [Abstract] | |
Product Revenue, Net | 14. Product revenue, net The following table presents the Company's net revenue by product (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2023 2022 2023 2022 Libmeldy $ 6,651 $ 3,145 $ 7,185 $ 8,204 Strimvelis — 636 — 636 Total $ 6,651 $ 3,781 $ 7,185 $ 8,840 Activity in each of the product revenue allowance and reserve categories for Libmeldy is summarized as follows (in thousands): Trade discounts and allowances Government rebates Total Balance as of December 31, 2022 $ 4,390 $ 2,300 $ 6,690 Provision Related to sales in the current year 3,950 1,655 5,605 Credits and payments made during the period ( 7,344 ) — ( 7,344 ) Foreign currency translation — 50 50 Balance as of June 30, 2023 $ 996 $ 4,005 $ 5,001 The total reserves described above are summarized as components of the Company's condensed consolidated balance sheets as follows (in thousands): As of As of Reduction of accounts receivable, net $ 996 $ 4,390 Component of accrued expenses and other current liabilities 4,005 2,300 Total revenue-related reserves $ 5,001 $ 6,690 |
Collaboration Revenue
Collaboration Revenue | 6 Months Ended |
Jun. 30, 2023 | |
Collaborative Arrangement [Abstract] | |
Collaboration Revenue | 15. Collaboration revenue On July 1, 2021, the Company entered into a strategic collaboration with Pharming Group N.V. (“Pharming”) to research, develop, manufacture, and commercialize OTL-105, an investigational ex vivo autologous HSC gene therapy for the treatment of hereditary angioedema (HAE), a life-threatening rare disorder that causes recurring swelling attacks in the face, throat, extremities and abdomen (the “Collaboration Agreement”). Under the terms of the Collaboration Agreement, Pharming was granted worldwide rights to OTL-105 and will be responsible for clinical development, regulatory filings and commercialization of the investigational gene therapy, including associated costs. The Company will lead the completion of IND-enabling activities and oversee manufacturing of OTL-105 during pre-clinical and clinical development, which will be funded by Pharming. In addition, both the Company and Pharming will explore the application of non-toxic conditioning regimen for use with OTL-105 administration. The Company received an upfront payment of $ 10.0 million in cash from Pharming and will receive cost reimbursements for ongoing research, development, and manufacturing services to be provided under the Collaboration Agreement. The Company is also eligible to receive up to $ 189.5 million in development, regulatory and sales milestones as well as mid-single to low double-digit percentage royalty payments on future worldwide sales. The Company also entered into a Share Purchase Agreement with Pharming on July 1, 2021 (the “Pharming SPA”), pursuant to which the Company issued 1,227,738 ordinary shares to Pharming for total consideration of $ 7.5 million. The consideration is payment for the fair value of ordinary shares with a fair value of $ 4.1 million plus a $ 3.4 million premium on the fair value of the Company’s ordinary shares. The “Collaboration Agreement” and the “Pharming SPA” are referred to together as the “Pharming Agreements.” Accounting Analysis At the commencement of the arrangement, two units of accounting were identified, which include: 1) the issuance of 1,227,738 of the Company’s ordinary shares as part of the Pharming SPA and 2) the license and collaboration agreement, which conveys the license and provides for the Company to provide research, development, manufacturing services for OTL-105. The Pharming Agreements were entered into concurrently as part of a single commercial objective and the Company considers them a single arrangement for accounting purposes. The total upfront payments of $ 17.5 million are comprised of $ 4.1 million attributed to the equity sold to Pharming and $ 13.4 million attributed to the Collaboration Agreement. The Company accounts for the Collaboration Agreement component within the scope of ASC Topic 606, Revenue from Contracts with Customers . The Company has concluded that the conveyance of the license for the HAE program and the provision of research, development, and manufacturing services for the HAE program represent a series of distinct services that are accounted for as a single performance obligation within the Collaboration Agreement. The Company determined that the transaction price includes: the $ 13.4 million attributed to the Collaboration Agreement and the variable consideration for estimated reimbursement payments at agreed upon contractual rates to be received from Pharming for the Company’s on-going research, development, and manufacturing services. There is uncertainty as to whether the future milestone payments will ultimately be achieved. Accordingly, the Company has fully constrained the variable consideration associated with the milestones. The royalty payments were also excluded from the transaction price as the Company is not required to estimate variable consideration for the royalty payments. The Company re-evaluates the transaction price as of the end of each reporting period. The Company recognizes revenue associated with the performance obligation as the research, development, and manufacturing services are provided using an input method, based on the cumulative costs incurred compared to the total estimated costs expected to be incurred to satisfy the performance obligation. The transfer of control to the customer occurs over the time period that the research, development and manufacturing services are provided by the Company. For the three and six months ended June 30, 2023 , the Company recognized collaboration revenue under the Collaboration Agreement of $ 0.7 million and $ 1.4 million, respectively. For the three and six months ended June 30, 2022 , the Company recognized collaboration revenue under the Collaboration Agreement of $ 0.6 million and $ 1.1 million, respectively. As of June 30, 2023 there is $ 0.8 million and $ 10.8 million of current and long-term deferred revenue, respectively, related to the Collaboration Agreement. |
Income Taxes
Income Taxes | 6 Months Ended |
Jun. 30, 2023 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | 16. Income taxes The Company recorded an income tax expense of $ 13 thousand and an income tax benefit of $ 161 thousand for the six months ended June 30, 2023 and 2022, respectively. The Company recorded income tax expense of $ 25 thousand and a benefit of $ 219 thousand for the three months ended June 30, 2023 and 2022, respectively. The Company records no income tax benefits for the net operating losses incurred in each period in the U.K. due to the uncertainty regarding the realizability of the deferred tax asset . The Company's income tax relates to its subsidiaries in Europe and the U.S. The Company's income tax computed at its effective income tax rate for the three and six months ended June 30, 2023 differed from income taxes computed at the U.K. statutory tax rate primarily due to the U.S. deduction for foreign derived intangible income, U.S. tax credits, and share-based compensation. The Company's income tax computed at its effective income tax rate for the three and six months ended June 30, 2022 differed from income taxes computed at the U.K. statutory tax rate primarily due to the U.S. deduction for foreign derived intangible income, U.S. tax credits, share-based compensation, and provision to return adjustments. |
Commitments and Contingencies
Commitments and Contingencies | 6 Months Ended |
Jun. 30, 2023 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 17. Commitments and contingencies Legal proceedings The Company is not a party to any material litigation and does not have contingency reserves established for any litigation liabilities. Manufacturing and technology development master agreement with AGC Biologics The Company is party to an Agreement with AGC Biologic S.p.A (“AGC”) pursuant to which the Company is obligated to pay AGC for a minimum product manufacturing commitment, dedicated manufacturing and development resources, and for a lease component associated with the right of use of exclusive manufacturing suites within AGC’s existing facilities. The following table outlines the current commitments associated with the agreement, as of June 30, 2023 (amounts in thousands): Due in: Product manufacturing commitments Dedicated manufacturing and development resources Exclusive transduction suites Total AGC Commitment 2023 (remaining six months) $ 976 $ 3,236 $ — $ 4,212 2024 1,952 6,472 2,169 10,593 2025 976 3,236 1,085 5,297 Total manufacturing commitments $ 3,904 $ 12,944 $ 3,254 $ 20,102 Tabular disclosure above has been translated to U.S. Dollar, from Euro, using the period end exchange rate of €1.00 to $ 1.08 . Indemnification agreements In the ordinary course of business, the Company may provide indemnification of varying scope and terms to vendors, lessors, business partners, and other parties with respect to certain matters including, but not limited to, losses arising out of breach of such agreements or from intellectual property infringement claims made by third parties. In addition, the Company has entered into indemnification agreements with members of its board of directors and senior management that will require the Company, among other things, to indemnify them against certain liabilities that may arise by reason of their status or service as directors or officers. The maximum potential amount of future payments the Company could be required to make under these indemnification agreements is, in many cases, unlimited. To date, the Company has not incurred any material costs as a result of such indemnifications. The Company is not aware of any claims under indemnification arrangements, and it has not accrued any liabilities related to such obligations in its unaudited condensed consolidated financial statements as of June 30, 2023. |
Net loss per ordinary share
Net loss per ordinary share | 6 Months Ended |
Jun. 30, 2023 | |
Earnings Per Share [Abstract] | |
Net Loss per Ordinary Share | 18 . Net loss per ordinary share The Company is in a loss position for all periods presented. As such, the basic net loss per ordinary share is the same as the diluted net loss per ordinary share as the inclusion of all potential ordinary share equivalents outstanding would have been anti-dilutive. The following securities, presented based on amounts outstanding at each period end, are considered to be ordinary share equivalents, but were excluded from the computation of diluted net loss per ordinary share because to do so would have been anti-dilutive: June 30, 2023 2022 Share options 17,034,942 13,894,428 Unvested restricted share units 3,191,423 500,989 Ordinary shares to be issued upon exercise of warrants (1) 109,083,590 — 129,309,955 14,395,417 (1) The warrants included in the table are exercisable subject to future contingencies (See Note 12) |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 6 Months Ended |
Jun. 30, 2023 | |
Accounting Policies [Abstract] | |
Basis of presentation | Basis of presentation These condensed consolidated financial statements of the Company are unaudited and have been prepared in accordance with U.S. generally accepted accounting principles (“GAAP”) for interim financial reporting and in accordance with Regulation S-X, Rule 10-01. Accordingly, they do not include all of the information and footnotes required by GAAP for complete financial statements. Any reference in these notes to applicable guidance is meant to refer to the authoritative GAAP as found in the Accounting Standards Codification (“ASC”), and Accounting Standards Update (“ASU”), of the Financial Accounting Standards Board (“FASB”). These unaudited condensed consolidated financial statements should be read in conjunction with the audited consolidated financial statements and accompanying notes included in the Company’s Annual Report. The condensed consolidated balance sheet as of December 31, 2022, was derived from audited consolidated financial statements included in the Company’s Annual Report but does not include all disclosures required by GAAP. Certain information and footnote disclosures normally included in financial statements prepared in accordance with GAAP have been condensed or omitted from these interim financial statements. However, these interim financial statements include all adjustments, consisting only of normal recurring adjustments, which are, in the opinion of the Company’s management, necessary to fairly state the results of the interim period. The interim results are not necessarily indicative of results to be expected for the full year. Amounts reported are presented in thousands, except percentages, per share amounts or as otherwise noted. As a result, certain totals may not sum due to rounding. |
Principles of consolidation | Principles of consolidation The accompanying unaudited condensed consolidated financial statements include the accounts of the Company and its wholly owned subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. |
Use of estimates | Use of estimates The preparation of unaudited condensed consolidated financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the unaudited condensed consolidated financial statements and the reported amounts of revenue and expenses during the reporting periods. Significant estimates and assumptions reflected in these unaudited condensed consolidated financial statements include, but are not limited to, the accrual for research and development expenses, the research and development tax credit receivable, share-based compensation, collaboration agreement milestones, variable consideration in revenue recognition, operating lease assets and liabilities, valuation of PIPE Warrants and PIPE Units, and income taxes. Estimates are periodically reviewed in light of changes in circumstances, facts and experience. Actual results could differ from the Company’s estimates. |
Foreign currency | Foreign currency The financial statements of the Company’s subsidiaries with functional currencies other than the U.S. Dollar are translated into U.S. Dollars using period-end exchange rates for assets and liabilities, historical exchange rates for shareholders’ equity and weighted average exchange rates for operating results. Unrealized losses are driven primarily by intercompany balances denominated in currencies other than the functional currency of the entity with the intercompany balance, and typically fluctuates concurrently with fluctuations in the U.S. Dollar, Pounds sterling, and Euro exchange rates. Translation gains and losses are included in accumulated other comprehensive income (loss) in shareholders’ equity. Foreign currency transaction gains and losses are included in other income (expense), net in the results of operations. The Company recorded realized and unrealized foreign currency transactio n losses of $ 7.5 million for the six months ended June 30, 2023 , and realized and unrealized foreign currency transaction losses of $ 24.3 million for the six months ended June 30, 2022 , which is included in other income (expense), net in the condensed consolidated statements of operations and comprehensive loss. |
Accounts receivable | Accounts receivable Accounts receivable arise from product revenue and amounts due from the Company's collaboration partners and have payment terms that generally require payment within 30 to 90 days. For some Libmeldy customers, our payment terms can range from 30 days to under one year . The amount from product revenue represents amounts due from distributors in Europe, which are recorded net of reserves for trade discounts and allowances, and other incentives to the extent such amounts are payable to the customer by the Company. The Company monitors economic conditions to identify facts or circumstances that may indicate that its receivables are at risk of collection. The Company provides reserves against accounts receivable for estimated losses, if any, that may result from a customer's inability to pay based on the composition of its accounts receivable, current economic conditions, and historical credit loss activity. Amounts determined to be uncollectible are charged or written-off against the reserve. During the three and six months ended June 30, 2023, the Company did no t record any expected credit losses related to outstanding accounts receivable. |
Restricted cash | Restricted cash Cash and cash equivalents that are restricted as to withdrawal or use under the terms of certain contractual agreements are recorded as restricted cash on the Company’s condensed consolidated balance sheets. The Company has an outstanding letter of credit for $ 3.0 million associated with a lease and is required to hold this amount in a standalone bank account, as of June 30, 2023 , and December 31, 2022. The Company is also contractually required to maintain cash collateral accounts associated with corporate credit cards and other leases in the amount of $ 1.3 million at June 30, 2023 and December 31, 2022. Cash, cash equivalents, and restricted cash were comprised of the following (in thousands): As of June 30, As of December 31, 2023 2022 Cash and cash equivalents $ 38,273 $ 68,424 Restricted cash 4,215 4,215 Total cash, cash equivalents and restricted cash shown in the statement of cash flows $ 42,488 $ 72,639 |
Recently adopted accounting pronouncements | Recently adopted accounting pronouncements In March 2020, the FASB issued ASU 2020-04 , Reference Rate Reform (Topic 848) : Facilitation of the Effects of Reference Rate Reform on Financial Reporting and issued two subsequent amendments: ASU 2021-01, issued in January 2021, refines the scope of ASU and clarifies some of its guidance as part of the FASB’s monitoring of global reference rate reform activities and ASU 2022-06, issued in December 2022, which extends the effective period of the ASU through December 31, 2023 (collectively, including ASU 2020-04, “ASC 848”). ASC 848 provides temporary optional expedients and exceptions to the GAAP guidance on contract modifications and hedge accounting to ease the financial reporting burdens related to the expected market transition from the London Interbank Offered Rate (“LIBOR”) and other interbank offered rates to alternative reference rates. ASC 848 is effective for all entities as of March 12, 2020, through December 31, 2024, at which time transition is expected to be complete. The Company adopted this standard as of January 1, 2023 . The application of this ASU did not have a material impact on the Company's financial position, results of operations, or cash flows. |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 6 Months Ended |
Jun. 30, 2023 | |
Accounting Policies [Abstract] | |
Schedule of Cash, Cash Equivalents and Restricted Cash | Cash, cash equivalents, and restricted cash were comprised of the following (in thousands): As of June 30, As of December 31, 2023 2022 Cash and cash equivalents $ 38,273 $ 68,424 Restricted cash 4,215 4,215 Total cash, cash equivalents and restricted cash shown in the statement of cash flows $ 42,488 $ 72,639 |
Fair Value Measurements and M_2
Fair Value Measurements and Marketable Securities (Tables) | 6 Months Ended |
Jun. 30, 2023 | |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Abstract] | |
Schedule of Cash Equivalents and Marketable Securities | The following tables present information about the Company’s financial assets and liabilities that have been measured at fair value as of June 30, 2023, and December 31, 2022, and indicate hierarchy of valuation inputs utilized to determine such fair value (in thousands): Fair Value Measurements as of Level 1 Level 2 Level 3 Total Assets Cash equivalents Money market funds $ 3,305 $ — $ — $ 3,305 U.S. treasuries — 1,995 — 1,995 Commercial paper — 6,989 — 6,989 Total cash equivalents $ 3,305 $ 8,984 $ — $ 12,289 Marketable securities U.S. government securities $ — $ 12,326 $ — $ 12,326 U.S. treasuries — 14,277 — 14,277 Corporate bonds — 16,118 — 16,118 Commercial paper — 69,747 — 69,747 Total marketable securities $ — $ 112,468 $ — $ 112,468 Total Assets $ 3,305 $ 121,452 $ — $ 124,757 Liabilities PIPE Warrant liability $ — $ — $ 12,266 $ 12,266 Total Liabilities $ — $ — $ 12,266 $ 12,266 Fair Value Measurements as of Level 1 Level 2 Level 3 Total Assets Cash equivalents Money market funds $ 1,239 $ — $ — $ 1,239 U.S. treasuries — 6,600 — 6,600 U.S. government securities — 5,200 — 5,200 Commercial paper — 14,122 — 14,122 Total cash equivalents $ 1,239 $ 25,922 $ — $ 27,161 Marketable securities U.S. government securities $ — $ 1,984 $ — $ 1,984 Corporate bonds — 25,475 — 25,475 Commercial paper — 47,867 — 47,867 Total marketable securities $ — $ 75,326 $ — $ 75,326 Total Assets $ 1,239 $ 101,248 $ — $ 102,487 |
Schedule of Significant Unobservable Inputs used in Valuation Model to Measure PIPE Warrants and PIPE Units Liabilities | The significant unobservable inputs used in the valuation models during the three months ended June 30, 2023, to measure the fair value of the PIPE Warrants and PIPE Units are as follows: PIPE Unit Liability PIPE Warrant Liability Valuation date June 22, 2023 June 30, 2023 Expected warrant exercise date July 30, 2024 April 30, 2025 July 30, 2024 April 30, 2025 Black-Scholes inputs Anticipated common stock price (at exercise date) $ 10.40 $ 10.07 $ 10.39 $ 10.07 Expected term (in years) 1.11 1.86 1.08 1.84 Expected volatility 75.0 % 74.8 % 75.6 % 74.5 % Other significant unobservable inputs Implied probabilities of occurrence of Second Closing and FDA Approval 30.5 % 34.6 % Discount for lack of marketability 5.0 % 0 % |
Schedule of Aggregate Fair Values of PIPE Warrant Liability and PIPE Unit Liability | The following table provides a rollforward of the aggregate fair values of the Company’s PIPE Warrant liability and PIPE Unit liability, for which fair values are determined using Level 3 inputs (in thousands): PIPE Warrant Liability PIPE Unit Liability Balance at December 31, 2022 $ — $ — Issuances during period 11,098 25 Change in fair value 1,168 ( 9,668 ) Settlement of PIPE Unit liability upon Second Closing — 9,643 Balance at June 30, 2023 $ 12,266 $ - |
Schedule of Amortized Cost and Fair Value of Available-for-Sale Marketable Debt Securities | The following tables summarize the amortized cost and fair value of the Company's available-for-sale marketable debt securities (in thousands): June 30, 2023 Amortized Gross Gross Credit Losses Fair Value U.S. government securities $ 12,338 $ 1 $ ( 13 ) $ — $ 12,326 U.S. treasuries 16,280 — ( 8 ) — $ 16,272 Corporate bonds 16,232 — ( 114 ) — 16,118 Commercial paper 76,801 — ( 65 ) — 76,736 Total $ 121,651 $ 1 $ ( 200 ) $ — $ 121,452 December 31, 2022 Amortized Gross Gross Credit Losses Fair Value U.S. government securities $ 7,188 $ 1 $ ( 6 ) $ — $ 7,183 U.S. treasuries 6,599 1 — — $ 6,600 Corporate bonds 25,656 — ( 180 ) — $ 25,476 Commercial paper 62,038 3 ( 52 ) — 61,989 Total $ 101,481 $ 5 $ ( 238 ) $ — $ 101,248 |
Schedule of Debt Securities by Contractual Maturity | The following table summarizes the Company’s debt securities by contractual maturity, as of June 30, 2023, and December 31, 2022 (in thousands): June 30, December 31, Maturities in one year or less $ 117,460 $ 98,277 Maturities between one and three years 3,992 2,971 Total $ 121,452 $ 101,248 |
Inventory (Tables)
Inventory (Tables) | 6 Months Ended |
Jun. 30, 2023 | |
Inventory Disclosure [Abstract] | |
Schedule of Inventory | Inventory consists of the following (in thousands): June 30, December 31, 2023 2022 Raw materials $ 6,712 $ 3,193 Work in process 225 207 Total inventory $ 6,937 $ 3,400 |
Prepaid Expenses and Other Cu_2
Prepaid Expenses and Other Current Assets (Tables) | 6 Months Ended |
Jun. 30, 2023 | |
Prepaid Expense and Other Assets, Current [Abstract] | |
Schedule of Prepaid Expenses and Other Current Assets | Prepaid expenses and other current assets consist of the following (in thousands): June 30, December 31, 2023 2022 Prepaid external research and development expenses $ 378 $ 881 Other prepayments 2,998 1,817 VAT receivable 712 1,077 Non-trade receivables 419 1,851 Rent deposits 1,033 960 Total prepaid expenses and other current assets $ 5,540 $ 6,586 |
Research and Development Tax _2
Research and Development Tax Credit Receivable (Tables) | 6 Months Ended |
Jun. 30, 2023 | |
Research and Development Tax Credit Receivable [Abstract] | |
Summary of Amounts Recognized to Offset Research and Development Expense | The following table below outlines the changes to the research and development tax credit receivable, including amounts recognized as an offset to research and development expense during the periods (amounts in thousands): Three Months Ended Six Months Ended 2023 2022 2023 2022 Balance at beginning of period $ 6,844 $ 16,649 $ 5,942 $ 30,723 Recognition of credit claims as offset to research and development expense 3,602 1,764 4,371 5,101 Receipt of credit claims — ( 1,916 ) - ( 18,390 ) Foreign currency translation 180 ( 1,321 ) 313 ( 2,258 ) Balance at end of period $ 10,626 $ 15,176 $ 10,626 $ 15,176 |
Intangible Assets, Net (Tables)
Intangible Assets, Net (Tables) | 6 Months Ended |
Jun. 30, 2023 | |
Goodwill and Intangible Assets Disclosure [Abstract] | |
Schedule of Intangible Assets Net of Accumulated Amortization | Intangible assets, net of accumulated amortization, consisted of the following (in thousands): As of June 30, 2023 Cost Accumulated Amortization Net License milestones $ 4,168 $ ( 694 ) $ 3,474 Total $ 4,168 $ ( 694 ) $ 3,474 As of December 31, 2022 Cost Accumulated Amortization Net License milestones $ 4,069 $ ( 509 ) $ 3,560 Total $ 4,069 $ ( 509 ) $ 3,560 |
Schedule of Estimated Future Amortization For Intangible Assets | T he following table summarizes the estimated future amortization for intangible assets for the next five years and thereafter (in thousands): As of June 30, 2023 (remaining six months) $ 171 2024 342 2025 342 2026 342 2027 342 Thereafter 1,935 Total $ 3,474 |
Accrued Expenses and Other Cu_2
Accrued Expenses and Other Current Liabilities (Tables) | 6 Months Ended |
Jun. 30, 2023 | |
Accrued Liabilities and Other Liabilities [Abstract] | |
Schedule of Accrued Expenses and Other Current Liabilities | Accrued expenses and other current liabilities consisted of the following (in thousands): June 30, December 31, 2023 2022 Accrued external research and development expenses $ 10,985 $ 11,230 Accrued payroll and related expenses 9,132 12,312 Accrued professional fees 2,588 2,263 Accrued other 3,459 2,647 Accrued governmental rebates 4,005 2,300 Strimvelis liability - current portion 3,617 3,685 Total accrued expenses and other current liabilities $ 33,786 $ 34,437 |
Notes Payable (Tables)
Notes Payable (Tables) | 6 Months Ended |
Jun. 30, 2023 | |
Debt Disclosure [Abstract] | |
Summary of Notes Payable | Notes payable consist of the following (in thousands): June 30, December 31, 2023 2022 Notes payable, net of issuance costs $ 27,297 $ 31,970 Less: current portion ( 9,429 ) ( 9,429 ) Notes payable, net of current portion 17,868 22,541 Accretion related to final payment 572 450 Notes payable, long term $ 18,440 $ 22,991 |
Summary of Estimated Future Principal Payments Due | As of June 30, 2023, the estimated future principal payments due are as follows (in thousands): Aggregate 2023 (remaining six months) $ 4,714 2024 9,429 2025 9,429 2026 5,084 Total 28,656 Less current portion ( 9,429 ) Less unamortized portion of final payment ( 584 ) Less unamortized debt issuance costs ( 203 ) Notes payable, long term $ 18,440 |
Share-based Compensation (Table
Share-based Compensation (Tables) | 6 Months Ended |
Jun. 30, 2023 | |
Share-Based Payment Arrangement [Abstract] | |
Summary of Option Activity | The following table summarizes option activity for ordinary shares under the plans for the six months ended June 30, 2023: Shares Weighted Outstanding at December 31, 2022 16,424,167 $ 1.56 Granted 5,085,330 0.49 Exercised ( 332,180 ) 0.12 Forfeited ( 795,167 ) 4.27 Outstanding at June 30, 2023 20,382,150 $ 1.21 Vested and expected to vest at June 30, 2023 20,382,150 $ 1.21 Exercisable at June 30, 2023 10,452,810 $ 1.82 |
Summary of Award Activity | The following table summarizes award activity for the six months ended June 30, 2023: Performance-based RSUs Time-based RSUs Total RSUs Weighted Average Grant Date Fair Value per Share Unvested at December 31, 2022 195,000 1,868,876 2,063,876 $ 0.55 Granted — 1,700,200 1,700,200 0.46 Vested — ( 609,170 ) ( 609,170 ) 0.52 Forfeited — ( 152,806 ) ( 152,806 ) 0.46 Unvested at June 30, 2023 195,000 2,807,100 3,002,100 $ 0.51 As of June 30, 2023, total unrecognized compensation cost related to time-based RSUs was $ 1.1 million. This amount is expected to be recognized over a weighted average period of 2.11 years. As of June 30, 2023, the total unrecognized compensation cost related to performance-based RSUs for which vesting was deemed probable was $ 0.1 million, which is expected to be recognized over a period of 0.5 years. As of June 30, 2023, the total unrecognized compensation cost related to performance-based RSUs for which vesting was deemed not probable was $ 0.5 million, the timing of recognition will be dependent upon achievement of remaining milestones. |
Share-based Compensation Expense | Share-based compensation expense related to share options, restricted share unit awards, and the employee stock purchase plan was classified in the unaudited condensed consolidated statements of operations and comprehensive loss as follows (in thousands): Three Months Ended Six Months Ended 2023 2022 2023 2022 Research and development $ 1,025 $ 1,744 $ 2,261 $ 3,706 Selling, general and administrative 1,553 2,212 3,829 4,910 Total $ 2,578 $ 3,956 $ 6,090 $ 8,616 Total share-based compensation by award type was as follows (in thousands): Three Months Ended Six Months Ended 2023 2022 2023 2022 Share options $ 2,253 $ 3,774 $ 4,808 $ 8,282 Restricted share units 325 182 1,282 334 Total $ 2,578 $ 3,956 $ 6,090 $ 8,616 |
Restructuring Charges (Tables)
Restructuring Charges (Tables) | 6 Months Ended |
Jun. 30, 2023 | |
Restructuring and Related Activities [Abstract] | |
Summary of Restructuring Charges | Activity for the six months ended June 30, 2022, is summarized as follows (in thousands): Three Months Ended June 30, 2022 Six Months Ended June 30, 2022 Balance at beginning of period $ 2,481 $ 6 Charged to expense - 2,481 Non-cash adjustments and foreign currency translation ( 554 ) ( 554 ) Payments made ( 1,089 ) ( 1,095 ) Balance at end of period $ 838 $ 838 |
Product Revenue, Net (Tables)
Product Revenue, Net (Tables) | 6 Months Ended |
Jun. 30, 2023 | |
Revenue from Contract with Customer [Abstract] | |
Disaggregated Product Revenue | The following table presents the Company's net revenue by product (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2023 2022 2023 2022 Libmeldy $ 6,651 $ 3,145 $ 7,185 $ 8,204 Strimvelis — 636 — 636 Total $ 6,651 $ 3,781 $ 7,185 $ 8,840 |
Summary of Activity of Product Revenue Allowance and Reserve Categories | Activity in each of the product revenue allowance and reserve categories for Libmeldy is summarized as follows (in thousands): Trade discounts and allowances Government rebates Total Balance as of December 31, 2022 $ 4,390 $ 2,300 $ 6,690 Provision Related to sales in the current year 3,950 1,655 5,605 Credits and payments made during the period ( 7,344 ) — ( 7,344 ) Foreign currency translation — 50 50 Balance as of June 30, 2023 $ 996 $ 4,005 $ 5,001 |
Summary as Components of Balance Sheets | The total reserves described above are summarized as components of the Company's condensed consolidated balance sheets as follows (in thousands): As of As of Reduction of accounts receivable, net $ 996 $ 4,390 Component of accrued expenses and other current liabilities 4,005 2,300 Total revenue-related reserves $ 5,001 $ 6,690 |
Commitments and Contingencies (
Commitments and Contingencies (Tables) | 6 Months Ended |
Jun. 30, 2023 | |
Commitments and Contingencies Disclosure [Abstract] | |
Schedule of Annual Commitments Associated with the Contract | The following table outlines the current commitments associated with the agreement, as of June 30, 2023 (amounts in thousands): Due in: Product manufacturing commitments Dedicated manufacturing and development resources Exclusive transduction suites Total AGC Commitment 2023 (remaining six months) $ 976 $ 3,236 $ — $ 4,212 2024 1,952 6,472 2,169 10,593 2025 976 3,236 1,085 5,297 Total manufacturing commitments $ 3,904 $ 12,944 $ 3,254 $ 20,102 Tabular disclosure above has been translated to U.S. Dollar, from Euro, using the period end exchange rate of €1.00 to $ 1.08 . |
Net Loss per Ordinary Share (Ta
Net Loss per Ordinary Share (Tables) | 6 Months Ended |
Jun. 30, 2023 | |
Earnings Per Share [Abstract] | |
Securities Excluded in the Computation of Diluted Net Loss Per Ordinary Share | The following securities, presented based on amounts outstanding at each period end, are considered to be ordinary share equivalents, but were excluded from the computation of diluted net loss per ordinary share because to do so would have been anti-dilutive: June 30, 2023 2022 Share options 17,034,942 13,894,428 Unvested restricted share units 3,191,423 500,989 Ordinary shares to be issued upon exercise of warrants (1) 109,083,590 — 129,309,955 14,395,417 (1) The warrants included in the table are exercisable subject to future contingencies (See Note 12) |
Nature of the Business and Li_2
Nature of the Business and Liquidity - Additional Information (Details) | 6 Months Ended | |||||||||
Jun. 22, 2023 USD ($) shares | Mar. 10, 2023 USD ($) shares | Jun. 30, 2023 USD ($) | Jun. 30, 2022 USD ($) | Jun. 30, 2023 £ / shares | Jun. 30, 2023 USD ($) | Jun. 22, 2023 £ / shares $ / shares shares | Mar. 10, 2023 £ / shares shares | Mar. 06, 2023 $ / shares | Dec. 31, 2022 £ / shares | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | ||||||||||
Ordinary shares voting rights description | Each holder of ordinary shares is entitled to one vote per ordinary share | |||||||||
Ordinary shares dividends declared | $ 0 | $ 0 | ||||||||
Number of ordinary shares issued and sold | shares | 4,250,000 | 56,666,900 | ||||||||
Nominal value of shares issued and sold | £ / shares | £ 0.10 | £ 0.1 | £ 0.1 | £ 0.10 | ||||||
Warrants to purchase ordinary shares | shares | 46,750,000 | 62,333,590 | ||||||||
Warrants to purchase price per unit | $ / shares | 8 | 6 | ||||||||
Gross proceeds of offering | $ 34,000,000 | $ 34,000,000 | ||||||||
Cash, cash equivalents and marketable securities | $ 150,700,000 |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies - Additional Information (Details) - USD ($) | 6 Months Ended | ||
Jun. 30, 2023 | Jun. 30, 2022 | Dec. 31, 2022 | |
Summary Of Significant Accounting Policies [Line Items] | |||
Foreign currency transaction gain (loss) | $ (7,500,000) | $ (24,300,000) | |
Cash collateral associated with corporate credit cards and other leases | 1,300,000 | $ 1,300,000 | |
Credit losses related to outstanding accounts receivable | 0 | ||
Outstanding letter of credit associated with a lease | $ 3,000,000 | $ 3,000,000 | |
Change in accounting principle, accounting standards update, adopted | true | ||
Change in accounting principle, accounting standards update, adoption date | Jan. 01, 2023 | ||
Change in accounting principle, accounting standards update, immaterial effect | true | ||
New accounting pronouncement or change in accounting principle, description | ASU 2020-04 | ||
Minimum | |||
Summary Of Significant Accounting Policies [Line Items] | |||
General payment terms | 30 days | ||
Maximum | |||
Summary Of Significant Accounting Policies [Line Items] | |||
General payment terms | 90 days | ||
Libmeldy | Minimum | |||
Summary Of Significant Accounting Policies [Line Items] | |||
General payment terms | 30 days | ||
Libmeldy | Maximum | |||
Summary Of Significant Accounting Policies [Line Items] | |||
General payment terms | 1 year |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies - Schedule of Cash, Cash Equivalents and Restricted Cash (Details) - USD ($) $ in Thousands | Jun. 30, 2023 | Dec. 31, 2022 | Jun. 30, 2022 | Dec. 31, 2021 |
Summary Of Significant Accounting Policies [Abstract] | ||||
Cash and cash equivalents | $ 38,273 | $ 68,424 | ||
Restricted cash | 4,215 | 4,215 | ||
Total cash, cash equivalents and restricted cash shown in the statement of cash flows | $ 42,488 | $ 72,639 | $ 57,736 | $ 60,178 |
Fair Value Measurements and M_3
Fair Value Measurements and Marketable Securities - Schedule of Cash Equivalents and Marketable Securities (Details) - USD ($) $ in Thousands | Jun. 30, 2023 | Dec. 31, 2022 |
Cash equivalents | ||
Total cash equivalents | $ 12,289 | $ 27,161 |
Marketable securities | ||
Total marketable securities | 112,468 | 75,326 |
Total | 124,757 | 102,487 |
Liabilities | ||
Total Liabilities | 12,266 | |
Level 1 | ||
Cash equivalents | ||
Total cash equivalents | 3,305 | 1,239 |
Marketable securities | ||
Total | 3,305 | 1,239 |
Level 2 | ||
Cash equivalents | ||
Total cash equivalents | 8,984 | 25,922 |
Marketable securities | ||
Total marketable securities | 112,468 | 75,326 |
Total | 121,452 | 101,248 |
Level 3 | ||
Liabilities | ||
Total Liabilities | 12,266 | |
PIPE Warrant Liability | ||
Liabilities | ||
Total Liabilities | 12,266 | |
PIPE Warrant Liability | Level 3 | ||
Liabilities | ||
Total Liabilities | 12,266 | |
Money Market Funds | ||
Cash equivalents | ||
Total cash equivalents | 3,305 | 1,239 |
Money Market Funds | Level 1 | ||
Cash equivalents | ||
Total cash equivalents | 3,305 | 1,239 |
U.S. Treasuries | ||
Cash equivalents | ||
Total cash equivalents | 1,995 | 6,600 |
Marketable securities | ||
Total marketable securities | 14,277 | |
U.S. Treasuries | Level 2 | ||
Cash equivalents | ||
Total cash equivalents | 1,995 | 6,600 |
Marketable securities | ||
Total marketable securities | 14,277 | |
U.S. Government Securities | ||
Cash equivalents | ||
Total cash equivalents | 5,200 | |
Marketable securities | ||
Total marketable securities | 12,326 | 1,984 |
U.S. Government Securities | Level 2 | ||
Cash equivalents | ||
Total cash equivalents | 5,200 | |
Marketable securities | ||
Total marketable securities | 12,326 | 1,984 |
Corporate Bonds | ||
Marketable securities | ||
Total marketable securities | 16,118 | 25,475 |
Corporate Bonds | Level 2 | ||
Marketable securities | ||
Total marketable securities | 16,118 | 25,475 |
Commercial Paper | ||
Cash equivalents | ||
Total cash equivalents | 6,989 | 14,122 |
Marketable securities | ||
Total marketable securities | 69,747 | 47,867 |
Commercial Paper | Level 2 | ||
Cash equivalents | ||
Total cash equivalents | 6,989 | 14,122 |
Marketable securities | ||
Total marketable securities | $ 69,747 | $ 47,867 |
Fair Value Measurements and M_4
Fair Value Measurements and Marketable Securities - Additional Information (Details) - USD ($) | 3 Months Ended | 6 Months Ended | ||||
Jun. 30, 2023 | Jun. 30, 2023 | Jun. 22, 2023 | Mar. 10, 2023 | Mar. 06, 2023 | Dec. 31, 2022 | |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||||
Fair value of assets transfers between level 1 and level 2 | $ 0 | $ 0 | $ 0 | |||
Warrants to purchase ordinary shares | 46,750,000 | 62,333,590 | ||||
Realized gains or losses recognized on investments | $ 0 | $ 0 | ||||
Securities Purchase Agreement | 2023 Private Placement | ||||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||||
Warrants to purchase ordinary shares | 109,083,590 | |||||
Securities Purchase Agreement | 2023 Private Placement | Initial Closing | ||||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||||
Warrants to purchase ordinary shares | 62,333,590 |
Fair Value Measurements and M_5
Fair Value Measurements and Marketable Securities - Schedule of Significant Unobservable Inputs used in Valuation Model to Measure PIPE Warrants and PIPE Units Liabilities (Details) | Jun. 30, 2023 $ / shares | Jun. 22, 2023 $ / shares |
PIPE Units Liability | Implied Probabilities of Occurrence of Second Closing and FDA Approval | ||
Fair Value Measurement Inputs and Valuation Techniques [Line Items] | ||
Units measurement input | 0.305 | |
PIPE Units Liability | Discount for Lack of Marketability | ||
Fair Value Measurement Inputs and Valuation Techniques [Line Items] | ||
Units measurement input | 0.05 | |
PIPE Units Liability | Expected Warrant Exercise Date July 30, 2024 | Anticipated Common Stock Price (at Exercise Date) | ||
Fair Value Measurement Inputs and Valuation Techniques [Line Items] | ||
Units measurement input, stock price | $ 10.4 | |
PIPE Units Liability | Expected Warrant Exercise Date July 30, 2024 | Expected Term (in years) | ||
Fair Value Measurement Inputs and Valuation Techniques [Line Items] | ||
Units measurement input, term | 1 year 1 month 9 days | |
PIPE Units Liability | Expected Warrant Exercise Date July 30, 2024 | Expected Volatility | ||
Fair Value Measurement Inputs and Valuation Techniques [Line Items] | ||
Units measurement input | 0.75 | |
PIPE Units Liability | Expected Warrant Exercise Date April 30, 2025 | Anticipated Common Stock Price (at Exercise Date) | ||
Fair Value Measurement Inputs and Valuation Techniques [Line Items] | ||
Units measurement input, stock price | $ 10.07 | |
PIPE Units Liability | Expected Warrant Exercise Date April 30, 2025 | Expected Term (in years) | ||
Fair Value Measurement Inputs and Valuation Techniques [Line Items] | ||
Units measurement input, term | 1 year 10 months 9 days | |
PIPE Units Liability | Expected Warrant Exercise Date April 30, 2025 | Expected Volatility | ||
Fair Value Measurement Inputs and Valuation Techniques [Line Items] | ||
Units measurement input | 0.748 | |
PIPE Warrant Liability | Implied Probabilities of Occurrence of Second Closing and FDA Approval | ||
Fair Value Measurement Inputs and Valuation Techniques [Line Items] | ||
Units measurement input | 0.346 | |
PIPE Warrant Liability | Discount for Lack of Marketability | ||
Fair Value Measurement Inputs and Valuation Techniques [Line Items] | ||
Units measurement input | 0 | |
PIPE Warrant Liability | Expected Warrant Exercise Date July 30, 2024 | Anticipated Common Stock Price (at Exercise Date) | ||
Fair Value Measurement Inputs and Valuation Techniques [Line Items] | ||
Warrants measurement input, stock price | $ 10.39 | |
PIPE Warrant Liability | Expected Warrant Exercise Date July 30, 2024 | Expected Term (in years) | ||
Fair Value Measurement Inputs and Valuation Techniques [Line Items] | ||
Warrants measurement input, term | 1 year 29 days | |
PIPE Warrant Liability | Expected Warrant Exercise Date July 30, 2024 | Expected Volatility | ||
Fair Value Measurement Inputs and Valuation Techniques [Line Items] | ||
Warrants measurement input | 0.756 | |
PIPE Warrant Liability | Expected Warrant Exercise Date April 30, 2025 | Anticipated Common Stock Price (at Exercise Date) | ||
Fair Value Measurement Inputs and Valuation Techniques [Line Items] | ||
Warrants measurement input, stock price | $ 10.07 | |
PIPE Warrant Liability | Expected Warrant Exercise Date April 30, 2025 | Expected Term (in years) | ||
Fair Value Measurement Inputs and Valuation Techniques [Line Items] | ||
Warrants measurement input, term | 1 year 10 months 2 days | |
PIPE Warrant Liability | Expected Warrant Exercise Date April 30, 2025 | Expected Volatility | ||
Fair Value Measurement Inputs and Valuation Techniques [Line Items] | ||
Warrants measurement input | 0.745 |
Fair Value Measurements and M_6
Fair Value Measurements and Marketable Securities - Schedule of Aggregate Fair Values of PIPE Warrant Liability and PIPE Unit Liability (Details) $ in Thousands | 6 Months Ended |
Jun. 30, 2023 USD ($) | |
PIPE Warrant Liability | |
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items] | |
Balance at December 31, 2022 | $ 0 |
Issuances during period | 11,098 |
Change in fair value | 1,168 |
Balance at March 31, 2023 | 12,266 |
PIPE Units Liability | |
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items] | |
Balance at December 31, 2022 | 0 |
Issuances during period | 25 |
Change in fair value | (9,668) |
Settlement of PIPE Unit liability upon Second Closing | 9,643 |
Balance at March 31, 2023 | $ 0 |
Fair Value Measurements and M_7
Fair Value Measurements and Marketable Securities - Schedule of Amortized Cost and Fair Value of Available-for-Sale Marketable Debt Securities (Details) - USD ($) $ in Thousands | Jun. 30, 2023 | Dec. 31, 2022 |
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Amortized Cost | $ 121,651 | $ 101,481 |
Gross Unrealized Gains | 1 | 5 |
Gross Unrealized Losses | (200) | (238) |
Fair Value | 121,452 | 101,248 |
U.S. Government Securities | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Amortized Cost | 12,338 | 7,188 |
Gross Unrealized Gains | 1 | 1 |
Gross Unrealized Losses | (13) | (6) |
Fair Value | 12,326 | 7,183 |
U.S. Treasuries | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Amortized Cost | 16,280 | 6,599 |
Gross Unrealized Gains | 1 | |
Gross Unrealized Losses | (8) | |
Fair Value | 16,272 | 6,600 |
Corporate Bonds | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Amortized Cost | 16,232 | 25,656 |
Gross Unrealized Losses | (114) | (180) |
Fair Value | 16,118 | 25,476 |
Commercial Paper | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Amortized Cost | 76,801 | 62,038 |
Gross Unrealized Gains | 3 | |
Gross Unrealized Losses | (65) | (52) |
Fair Value | $ 76,736 | $ 61,989 |
Fair Value Measurements and M_8
Fair Value Measurements and Marketable Securities - Schedule of Debt Securities by Contractual Maturity (Details) - USD ($) $ in Thousands | Jun. 30, 2023 | Dec. 31, 2022 |
Fair Value Disclosures [Abstract] | ||
Maturities in one year or less | $ 117,460 | $ 98,277 |
Maturities between one and three years | 3,992 | 2,971 |
Total | $ 121,452 | $ 101,248 |
Inventory - Schedule of Invento
Inventory - Schedule of Inventory (Details) - USD ($) $ in Thousands | Jun. 30, 2023 | Dec. 31, 2022 |
Inventory Disclosure [Abstract] | ||
Raw materials | $ 6,712 | $ 3,193 |
Work in process | 225 | 207 |
Total inventory | $ 6,937 | $ 3,400 |
Inventory - Additional Informat
Inventory - Additional Information (Details) $ in Millions | Jun. 30, 2023 USD ($) |
Inventory Disclosure [Abstract] | |
Inventory capitalized | $ 4.8 |
Prepaid Expenses and Other Cu_3
Prepaid Expenses and Other Current Assets - Schedule of Prepaid Expenses and Other Current Assets (Details) - USD ($) $ in Thousands | Jun. 30, 2023 | Dec. 31, 2022 |
Prepaid Expense and Other Assets, Current [Abstract] | ||
Prepaid external research and development expenses | $ 378 | $ 881 |
Other prepayments | 2,998 | 1,817 |
VAT receivable | 712 | 1,077 |
Non-trade receivables | 419 | 1,851 |
Rent deposits | 1,033 | 960 |
Total prepaid expenses and other current assets | $ 5,540 | $ 6,586 |
Research and Development Tax _3
Research and Development Tax Credit Receivable - Summary of Amounts Recognized to Offset Research and Development Expense (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2023 | Jun. 30, 2022 | Jun. 30, 2023 | Jun. 30, 2022 | |
Tax Credit Carryforward [Line Items] | ||||
Foreign currency translation | $ (7,500) | $ (24,300) | ||
United Kingdom | Research and Development Expense | ||||
Tax Credit Carryforward [Line Items] | ||||
Balance at beginning of period | $ 6,844 | $ 16,649 | 5,942 | 30,723 |
Recognition of credit claims as offset to research and development expense | 3,602 | 1,764 | 4,371 | 5,101 |
Receipt of credit claims | (1,916) | (18,390) | ||
Foreign currency translation | 180 | (1,321) | 313 | (2,258) |
Balance at end of period | $ 10,626 | $ 15,176 | $ 10,626 | $ 15,176 |
Research and Development Tax _4
Research and Development Tax Credit Receivable - Additional Information (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Jun. 30, 2023 | Dec. 31, 2022 | |
Tax Credit Carryforward [Line Items] | ||
Tax incentive receivable, current | $ 8,525 | $ 5,942 |
United Kingdom | ||
Tax Credit Carryforward [Line Items] | ||
Tax incentive receivable, current | 10,600 | |
United Kingdom | Research and Development Expense | ||
Tax Credit Carryforward [Line Items] | ||
Tax incentive receivable, current | 2,100 | $ 8,500 |
Additional income tax credits | $ 2,300 |
Intangible Assets, Net - Schedu
Intangible Assets, Net - Schedule of Intangible Assets Net of Accumulated Amortization (Details) - USD ($) $ in Thousands | Jun. 30, 2023 | Dec. 31, 2022 |
Finite Lived Intangible Assets [Line Items] | ||
Intangible Assets, Cost | $ 4,168 | $ 4,069 |
Intangible Assets, Accumulated Amortized | (694) | (509) |
Intangible Assets, Net | 3,474 | 3,560 |
License Milestones | ||
Finite Lived Intangible Assets [Line Items] | ||
Intangible Assets, Cost | 4,168 | 4,069 |
Intangible Assets, Accumulated Amortized | (694) | (509) |
Intangible Assets, Net | $ 3,474 | $ 3,560 |
Intangible Assets, Net - Additi
Intangible Assets, Net - Additional Information (Details) - USD ($) $ in Millions | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2023 | Jun. 30, 2022 | Jun. 30, 2023 | Jun. 30, 2022 | |
Finite Lived Intangible Assets [Line Items] | ||||
Intangible asset, useful life | 12 years | 12 years | ||
Amortization of intangible assets | $ 0.1 | $ 0.1 | $ 0.2 | $ 0.2 |
Effect of foreign currency translation on the net carrying value of intangible assets | $ 0.1 | $ 0.2 | $ 0.1 | $ 0.2 |
Intangible Assets, Net - Sche_2
Intangible Assets, Net - Schedule of Estimated Future Amortization For Intangible Assets (Details) - USD ($) $ in Thousands | Jun. 30, 2023 | Dec. 31, 2022 |
Goodwill and Intangible Assets Disclosure [Abstract] | ||
2023 (remaining six months) | $ 171 | |
2024 | 342 | |
2025 | 342 | |
2026 | 342 | |
2027 | 342 | |
Thereafter | 1,935 | |
Intangible Assets, Net | $ 3,474 | $ 3,560 |
Accrued Expenses and Other Cu_3
Accrued Expenses and Other Current Liabilities - Schedule of Accrued Expenses and Other Current Liabilities (Details) - USD ($) $ in Thousands | Jun. 30, 2023 | Dec. 31, 2022 |
Accrued Liabilities and Other Liabilities [Abstract] | ||
Accrued external research and development expenses | $ 10,985 | $ 11,230 |
Accrued payroll and related expenses | 9,132 | 12,312 |
Accrued professional fees | 2,588 | 2,263 |
Accrued other | 3,459 | 2,647 |
Accrued governmental rebates | 4,005 | 2,300 |
Strimvelis liability - current portion | 3,617 | 3,685 |
Total accrued expenses and other current liabilities | $ 33,786 | $ 34,437 |
Notes Payable - Additional Info
Notes Payable - Additional Information (Details) - USD ($) | 1 Months Ended | 3 Months Ended | 6 Months Ended | |||
May 31, 2021 | May 31, 2019 | Jun. 30, 2023 | Jun. 30, 2022 | Jun. 30, 2023 | Jun. 30, 2022 | |
Debt Instrument [Line Items] | ||||||
Line of credit facility agreement date | May 31, 2019 | |||||
Credit facility maximum borrowings | $ 100,000,000 | $ 75,000,000 | ||||
Credit facility, remaining borrowings | $ 67,000,000 | $ 67,000,000 | ||||
Line of credit facility amortization term | 18 months | |||||
Line of credit facility interest payments term | 18 months | |||||
Line of credit facility, frequency of payments | monthly payments | |||||
Line of credit facility, percentage of final payment | 3.50% | |||||
Final payment amount | $ 1,200,000 | |||||
Term Loan | ||||||
Debt Instrument [Line Items] | ||||||
Debt instrument, effective annual interest rate | 12.38% | |||||
Debt instrument, interest expense | $ 900,000 | $ 700,000 | $ 1,900,000 | $ 1,300,000 | ||
LIBOR | ||||||
Debt Instrument [Line Items] | ||||||
Debt instrument, annual interest rate | 5.95% | |||||
Initial Term Loan | ||||||
Debt Instrument [Line Items] | ||||||
Credit facility maximum borrowings | $ 33,000,000 | $ 25,000,000 | ||||
Amended Credit Facility | ||||||
Debt Instrument [Line Items] | ||||||
Proceeds from lines of credit | $ 33,000,000 | |||||
Amended Credit Facility | SOFR | ||||||
Debt Instrument [Line Items] | ||||||
Debt instrument, effective annual interest rate | 0.10% |
Notes Payable - Summary of Note
Notes Payable - Summary of Notes Payable (Details) - USD ($) $ in Thousands | Jun. 30, 2023 | Dec. 31, 2022 |
Notes and Loans, Noncurrent [Abstract] | ||
Notes payable, net of issuance costs | $ 27,297 | $ 31,970 |
Less: current portion | 9,429 | (9,429) |
Notes payable, net of current portion | 17,868 | 22,541 |
Accretion related to final payment | 572 | 450 |
Notes payable, long term | $ 18,440 | $ 22,991 |
Notes Payable - Summary of Esti
Notes Payable - Summary of Estimated Future Principal Payments Due (Details) - USD ($) $ in Thousands | Jun. 30, 2023 | Dec. 31, 2022 |
Long-Term Debt, Fiscal Year Maturity [Abstract] | ||
2023 (remaining six months) | $ 4,714 | |
2024 | 9,429 | |
2025 | 9,429 | |
2026 | 5,084 | |
Total | 28,656 | |
Less: current portion | (9,429) | |
Less: unamortized portion of final payment | 584 | |
Less: unamortized debt issuance costs | (203) | |
Notes payable, long term | $ 18,440 | $ 22,991 |
Shareholders' Equity - Addition
Shareholders' Equity - Additional Information (Details) - GBP (£) | 6 Months Ended | ||
Jun. 30, 2023 | Dec. 31, 2022 | Jun. 16, 2021 | |
Class of Stock [Line Items] | |||
Percentage rate of excess ordinary shares | 19.99% | ||
Ordinary shares, authorized | £ 33,556,809 | £ 13,023,851.5 | |
Ordinary shares, issued | 227,215,699 | 126,947,225 | |
Ordinary shares, outstanding | 227,215,699 | 126,947,225 | |
PIPE Authority | |||
Class of Stock [Line Items] | |||
Ordinary shares, authorized | £ 15,158,359 | ||
Remaining ordinary shares | 10,908,359 | ||
General Authority | |||
Class of Stock [Line Items] | |||
Ordinary shares, authorized | 18,398,450 | ||
Remaining ordinary shares | £ 23,924,545.7 | ||
2021 Authority | |||
Class of Stock [Line Items] | |||
Ordinary shares, authorized | £ 13,023,851.5 | ||
Remaining ordinary shares | £ 5,526,095.7 | ||
Ordinary Shares | |||
Class of Stock [Line Items] | |||
Ordinary shares, issued | 185,602,299 | ||
Ordinary shares, outstanding | 185,602,299 | ||
Number of deferred share | 1 | ||
Nominal value of deferred share | £ 4.89687 | ||
Non-voting Ordinary Shares | |||
Class of Stock [Line Items] | |||
Ordinary shares, issued | 41,613,400 | ||
Ordinary shares, outstanding | 41,613,400 |
Share-based Compensation - Addi
Share-based Compensation - Additional Information (Details) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | 3 Months Ended | 6 Months Ended | ||
Apr. 30, 2020 | Jun. 30, 2023 | Jun. 30, 2022 | Jun. 30, 2023 | Jun. 30, 2022 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Weighted-average grant date fair value of share options granted | $ 2.81 | $ 0.32 | |||
Total intrinsic value of share options exercised | $ 100 | $ 100 | |||
Number of shares granted | 1,700,200 | ||||
Stock compensation expense | 2,578 | $ 3,956 | $ 6,090 | $ 8,616 | |
RSUs vested | 609,170 | ||||
Total unrecognized compensation cost of stock option and time-based RSUs | 10,400 | $ 10,400 | |||
Compensation cost expected to be recognized weighted average period | 2 years 7 months 2 days | ||||
Total unrecognized compensation cost of performance-based RSUs | $ 500 | $ 500 | |||
2018 Plan | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Remaining shares available for issuance | 12,421,580 | 12,421,580 | |||
Inducement Plan | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Remaining shares available for issuance | 730,500 | 730,500 | |||
2018 ESPP | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Remaining shares available for issuance | 1,460,590 | 1,460,590 | |||
Performance-based Restricted Share Units ("RSUs") | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Compensation cost expected to be recognized weighted average period | 6 months | ||||
Total unrecognized compensation cost of performance-based RSUs | $ 100 | $ 100 | |||
Time-based Restricted Share Units | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Number of shares granted | 1,700,200 | ||||
RSUs vested | 609,170 | ||||
Award vesting period | 3 years | ||||
Total unrecognized compensation cost of stock option and time-based RSUs | 1,100 | $ 1,100 | |||
Compensation cost expected to be recognized weighted average period | 2 years 1 month 9 days | ||||
Chief Executive Officer | Performance-based Restricted Share Units ("RSUs") | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Number of shares granted | 195,000 | ||||
Total grant date fair value | $ 1,400 | ||||
Vesting rights | In April 2020, the Company granted 195,000 performance-based restricted share units (“RSUs”) with a total grant date fair value of $1.4 million to its Chief Executive Officer, Bobby Gaspar, M.D., Ph.D. Dr. Gaspar earns one-third of the award for each of the first three to occur of four milestones (65,000 RSUs are earned for each achieved milestone). The milestones relate to specific clinical and regulatory goals which have to be achieved before December 31, 2023 in order for the underlying RSUs to be eligible to vest. | ||||
Achievement of at Least Three of Four Milestones | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
RSUs vested | 65,000 | ||||
Achievement of Two Milestones | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Stock compensation expense | $ 100 | $ 800 |
Share-based Compensation - Summ
Share-based Compensation - Summary of Option Activity (Details) | 6 Months Ended |
Jun. 30, 2023 $ / shares shares | |
Share-Based Payment Arrangement [Abstract] | |
Number of Options, Outstanding at December 31, 2022 | shares | 16,424,167 |
Number of Options, Granted | shares | 5,085,330 |
Number of Options, Exercised | shares | (332,180) |
Number of Options, Forfeited | shares | (795,167) |
Number of Options, Outstanding at June 30, 2023 | shares | 20,382,150 |
Number of Options, Vested and expected to vest at June 30, 2023 | shares | 20,382,150 |
Number of Options, Exercisable, June 30, 2023 | shares | 10,452,810 |
Weighted Average Exercise Price, Outstanding at December 31, 2022 | $ / shares | $ 1.56 |
Weighted Average Exercise Price, Granted | $ / shares | 0.49 |
Weighted Average Exercise Price, Exercised | $ / shares | 0.12 |
Weighted Average Exercise Price, Forfeited | $ / shares | 4.27 |
Weighted Average Exercise Price, Outstanding at June 30, 2023 | $ / shares | 1.21 |
Weighted Average Exercise Price, Vested and expected to vest at June 30, 2023 | $ / shares | 1.21 |
Weighted Average Exercise Price, Exercisable at June 30, 2023 | $ / shares | $ 1.82 |
Share-based Compensation - Su_2
Share-based Compensation - Summary of Award Activity (Details) | 6 Months Ended |
Jun. 30, 2023 $ / shares shares | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
RSUs, Unvested at December 31, 2022 | 2,063,876 |
RSUs, Granted | 1,700,200 |
RSUs, Vested | (609,170) |
RSUs, Forfeited | (152,806) |
RSUs, Unvested at June 30, 2023 | 3,002,100 |
Weighted Average Grant Date Fair Value per Share, Unvested at December 31, 2022 | $ / shares | $ 0.55 |
Weighted Average Grant Date Fair Value per Share, Granted | $ / shares | 0.46 |
Weighted Average Grant Date Fair Value per Share, Vested | $ / shares | 0.52 |
Weighted Average Grant Date Fair Value per Share, Forfeited | $ / shares | 0.46 |
Weighted Average Grant Date Fair Value per Share, Unvested at June 30, 2023 | $ / shares | $ 0.51 |
Performance-based RSUs | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
RSUs, Unvested at December 31, 2022 | 195,000 |
RSUs, Unvested at June 30, 2023 | 195,000 |
Time-based RSUs | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
RSUs, Unvested at December 31, 2022 | 1,868,876 |
RSUs, Granted | 1,700,200 |
RSUs, Vested | (609,170) |
RSUs, Forfeited | (152,806) |
RSUs, Unvested at June 30, 2023 | 2,807,100 |
Share-based Compensation - Shar
Share-based Compensation - Share-based Compensation Expense (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2023 | Jun. 30, 2022 | Jun. 30, 2023 | Jun. 30, 2022 | |
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||||
Total share-based compensation | $ 2,578 | $ 3,956 | $ 6,090 | $ 8,616 |
Research and Development | ||||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||||
Total share-based compensation | 1,025 | 1,744 | 2,261 | 3,706 |
Selling, General and Administrative | ||||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||||
Total share-based compensation | 1,553 | 2,212 | 3,829 | 4,910 |
Share Options | ||||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||||
Total share-based compensation | 2,253 | 3,774 | 4,808 | 8,282 |
Restricted Share Units | ||||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||||
Total share-based compensation | $ 325 | $ 182 | $ 1,282 | $ 334 |
Private Placement Transaction S
Private Placement Transaction Share - Additional Information (Details) $ / shares in Units, $ in Thousands | 3 Months Ended | 6 Months Ended | |||||||||||||||||||
Jun. 22, 2023 USD ($) shares | Mar. 10, 2023 USD ($) shares | Mar. 06, 2023 USD ($) shares | Jun. 30, 2023 USD ($) | Mar. 31, 2023 USD ($) | Jun. 30, 2023 USD ($) | Jan. 01, 2025 $ / shares | Dec. 31, 2024 $ / shares | Jun. 30, 2023 £ / shares | Jun. 30, 2023 USD ($) shares | Jun. 22, 2023 £ / shares | Jun. 22, 2023 USD ($) $ / shares shares | Mar. 10, 2023 £ / shares | Mar. 10, 2023 USD ($) $ / shares shares | Mar. 06, 2023 £ / shares | Mar. 06, 2023 USD ($) shares | Dec. 31, 2022 £ / shares | Dec. 31, 2022 USD ($) | Jun. 30, 2022 USD ($) | Mar. 31, 2022 USD ($) | Dec. 31, 2021 USD ($) | |
Class of Stock [Line Items] | |||||||||||||||||||||
Number of ordinary shares issued and sold | shares | 4,250,000 | 56,666,900 | |||||||||||||||||||
Ordinary shares, nominal value | £ / shares | £ 0.10 | £ 0.1 | £ 0.1 | £ 0.10 | |||||||||||||||||
Warrants to purchase ordinary shares | shares | 46,750,000 | 62,333,590 | |||||||||||||||||||
Gross proceeds of offering | $ 34,000 | $ 34,000 | |||||||||||||||||||
Issuance costs | $ 889 | ||||||||||||||||||||
Equity | $ 101,655 | $ 109,047 | $ 98,264 | $ 146,272 | $ 175,646 | $ 209,950 | |||||||||||||||
Changes in fair value | $ (12,057) | ||||||||||||||||||||
Securities Purchase Agreement | 2023 Private Placement | |||||||||||||||||||||
Class of Stock [Line Items] | |||||||||||||||||||||
Number of ordinary shares issued and sold | shares | 99,166,900 | ||||||||||||||||||||
Ordinary shares, nominal value | £ / shares | £ 0.1 | ||||||||||||||||||||
Warrants to purchase ordinary shares | shares | 109,083,590 | ||||||||||||||||||||
Warrants expiration, description | The warrants will expire at the conclusion of the 30-day exercise period or on March 10, 2026, whichever is earlier. | ||||||||||||||||||||
Warrants exercisable term | 30 days | ||||||||||||||||||||
Shares sold in fixed combinaton of warrants as units | The 2023 Private Placement consisted of two closings, referred to herein as the "First Closing" and "Second Closing". At each closing, the Shares were sold in fixed combinations with the warrants as units, with each unit consisting of 10 Shares and 1 accompanying warrant to purchase 11 shares. The warrants are contingently exercisable for 30 days following (i) the Company’s public announcement of its receipt of marketing approval of its biologics license application (“BLA”) submitted to the FDA with respect to OTL-200 (the “Vesting Event”) and (ii) receipt of shareholder approval to increase the number of authorized shares. | ||||||||||||||||||||
Fair value of financial instruments | $ 34,000 | ||||||||||||||||||||
Issuance costs | 1,200 | 1,100 | $ 500 | ||||||||||||||||||
Gain on mark-to-market adjustment | 3,600 | ||||||||||||||||||||
Equity | $ 19,700 | $ 24,000 | |||||||||||||||||||
Changes in fair value | 9,700 | ||||||||||||||||||||
Warants outstanding | shares | 109,083,590 | ||||||||||||||||||||
Warrant exercisable | shares | 0 | ||||||||||||||||||||
Securities Purchase Agreement | 2023 Private Placement | Scenario Forecast | |||||||||||||||||||||
Class of Stock [Line Items] | |||||||||||||||||||||
Warrants to purchase price per share | $ / shares | $ 0.95 | $ 1.1 | |||||||||||||||||||
Securities Purchase Agreement | 2023 Private Placement | Initial Closing | |||||||||||||||||||||
Class of Stock [Line Items] | |||||||||||||||||||||
Warrants to purchase ordinary shares | shares | 62,333,590 | ||||||||||||||||||||
Warrants to purchase price per share | $ / shares | $ 6 | ||||||||||||||||||||
Gross proceeds of offering | 34,000 | ||||||||||||||||||||
Deducting offering expenses | 900 | ||||||||||||||||||||
Securities Purchase Agreement | 2023 Private Placement | Second Closing | |||||||||||||||||||||
Class of Stock [Line Items] | |||||||||||||||||||||
Warrants to purchase ordinary shares | shares | 46,750,000 | ||||||||||||||||||||
Warrants to purchase price per share | $ / shares | $ 8 | ||||||||||||||||||||
Gross proceeds of offering | 34,000 | ||||||||||||||||||||
Deducting offering expenses | 1,200 | ||||||||||||||||||||
Securities Purchase Agreement | 2023 Private Placement | PIPE Warrants | |||||||||||||||||||||
Class of Stock [Line Items] | |||||||||||||||||||||
Fair value of financial instruments | 6,400 | ||||||||||||||||||||
Issuance costs | 200 | 200 | |||||||||||||||||||
Warrant liability | $ 4,600 | ||||||||||||||||||||
Changes in fair value | $ 1,400 | $ 1,200 | |||||||||||||||||||
Securities Purchase Agreement | 2023 Private Placement | PIPE Units | |||||||||||||||||||||
Class of Stock [Line Items] | |||||||||||||||||||||
Fair value of financial instruments | 20 | ||||||||||||||||||||
Issuance costs | 200 | ||||||||||||||||||||
Derecognized assets | $ 9,600 | ||||||||||||||||||||
Changes in fair value | $ 9,700 | $ 9,600 | |||||||||||||||||||
Securities Purchase Agreement | 2023 Private Placement | Ordinary Shares | |||||||||||||||||||||
Class of Stock [Line Items] | |||||||||||||||||||||
Fair value of financial instruments | $ 27,500 | ||||||||||||||||||||
Issuance costs | $ 1,000 | $ 900 | |||||||||||||||||||
Securities Purchase Agreement | 2023 Private Placement | Ordinary Shares | Initial Closing | |||||||||||||||||||||
Class of Stock [Line Items] | |||||||||||||||||||||
Number of ordinary shares issued and sold | shares | 40,053,500 | ||||||||||||||||||||
Securities Purchase Agreement | 2023 Private Placement | Ordinary Shares | Second Closing | |||||||||||||||||||||
Class of Stock [Line Items] | |||||||||||||||||||||
Number of ordinary shares issued and sold | shares | 13,408,074 | ||||||||||||||||||||
Securities Purchase Agreement | 2023 Private Placement | Non-voting Ordinary Shares | Initial Closing | |||||||||||||||||||||
Class of Stock [Line Items] | |||||||||||||||||||||
Number of ordinary shares issued and sold | shares | 16,613,400 | ||||||||||||||||||||
Securities Purchase Agreement | 2023 Private Placement | Non-voting Ordinary Shares | Second Closing | |||||||||||||||||||||
Class of Stock [Line Items] | |||||||||||||||||||||
Number of ordinary shares issued and sold | shares | 29,091,926 |
Restructuring Charges - Additio
Restructuring Charges - Additional Information (Details) - USD ($) | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2023 | Jun. 30, 2022 | Jun. 30, 2023 | Jun. 30, 2022 | |
Restructuring Cost And Reserve [Line Items] | ||||
Research and development | $ 16,695,000 | $ 21,965,000 | $ 32,688,000 | $ 50,199,000 |
Selling, general and administrative | $ 10,992,000 | $ 13,730,000 | 22,127,000 | 27,029,000 |
Restructuring charges | $ 0 | 2,481,000 | ||
Neurometabolic Diseases and Early Research Programs | ||||
Restructuring Cost And Reserve [Line Items] | ||||
Research and development | 1,600,000 | |||
Selling, general and administrative | 400,000 | |||
Neurometabolic Diseases and Early Research Programs | Employee-Related Termination Costs | ||||
Restructuring Cost And Reserve [Line Items] | ||||
One-time charge | $ 1,900,000 |
Restructuring Charges - Summary
Restructuring Charges - Summary of Restructuring Charges (Details) - USD ($) | 3 Months Ended | 6 Months Ended | |
Jun. 30, 2022 | Jun. 30, 2023 | Jun. 30, 2022 | |
Restructuring and Related Activities [Abstract] | |||
Balance at beginning of period | $ 2,481,000 | $ 6,000 | |
Charged to expense | $ 0 | 2,481,000 | |
Non-cash adjustments and foreign currency translation | (554,000) | (554,000) | |
Payments made | (1,089,000) | (1,095,000) | |
Balance at end of period | $ 838,000 | $ 838,000 |
Product Revenue, Net - Schedule
Product Revenue, Net - Schedule of Net Revenue by Product (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2023 | Jun. 30, 2022 | Jun. 30, 2023 | Jun. 30, 2022 | |
Disaggregation of Revenue [Line Items] | ||||
Net revenue | $ 7,314 | $ 4,368 | $ 8,551 | $ 9,892 |
Libmeldy | ||||
Disaggregation of Revenue [Line Items] | ||||
Net revenue | 6,651 | 3,145 | 7,185 | 8,204 |
Strimvelis | ||||
Disaggregation of Revenue [Line Items] | ||||
Net revenue | 636 | 636 | ||
Product | ||||
Disaggregation of Revenue [Line Items] | ||||
Net revenue | $ 6,651 | $ 3,781 | $ 7,185 | $ 8,840 |
Product Revenue, Net - Summary
Product Revenue, Net - Summary of Activity of Product Revenue Allowance and Reserve Categories (Details) $ in Thousands | 6 Months Ended |
Jun. 30, 2023 USD ($) | |
SEC Schedule, 12-09, Valuation and Qualifying Accounts Disclosure [Line Items] | |
Balance as of December 31, 2022 | $ 6,690 |
Provision Related to sales in the current year | 5,605 |
Credits and payments made during the period | (7,344) |
Foreign currency translation | 50 |
Balance as of June 30, 2023 | 5,001 |
Trade Discounts and Allowances | |
SEC Schedule, 12-09, Valuation and Qualifying Accounts Disclosure [Line Items] | |
Balance as of December 31, 2022 | 4,390 |
Provision Related to sales in the current year | 3,950 |
Credits and payments made during the period | (7,344) |
Balance as of June 30, 2023 | 996 |
Government Rebates | |
SEC Schedule, 12-09, Valuation and Qualifying Accounts Disclosure [Line Items] | |
Balance as of December 31, 2022 | 2,300 |
Provision Related to sales in the current year | 1,655 |
Foreign currency translation | 50 |
Balance as of June 30, 2023 | $ 4,005 |
Product Revenue, Net - Summar_2
Product Revenue, Net - Summary as Components of Balance Sheets (Details) - USD ($) $ in Thousands | 6 Months Ended | 12 Months Ended |
Jun. 30, 2023 | Dec. 31, 2022 | |
SEC Schedule, 12-09, Valuation and Qualifying Accounts [Abstract] | ||
Reduction of accounts receivable, net | $ 996 | $ 4,390 |
Component of accrued expenses and other current liabilities | 4,005 | 2,300 |
Total revenue-related reserves | $ 5,001 | $ 6,690 |
Collaboration Revenue - Additio
Collaboration Revenue - Additional Information (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||||||
Jun. 22, 2023 | Mar. 10, 2023 | Jul. 01, 2021 | Jun. 30, 2023 | Mar. 31, 2023 | Jun. 30, 2022 | Jun. 30, 2023 | Jun. 30, 2022 | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Issuance of ordinary shares | 4,250,000 | 56,666,900 | ||||||
Total consideration | $ 18,761 | $ 23,073 | ||||||
Net revenue | 7,314 | $ 4,368 | $ 8,551 | $ 9,892 | ||||
Strategic Collaboration Agreement with Pharming | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Cash upfront payment received | $ 10,000 | |||||||
Strategic Collaboration Agreement with Pharming | Maximum | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Milestone payments receivable | $ 189,500 | |||||||
Share Purchase Agreement with Pharming | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Issuance of ordinary shares | 1,227,738 | |||||||
Total consideration | $ 7,500 | |||||||
Common stock consideration | 4,100 | |||||||
Premium consideration | 3,400 | |||||||
Pharming Agreements | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Total upfront payments | 17,500 | |||||||
Upfront payments attributed to equity sold | 4,100 | |||||||
Upfront payments attributed to research activities | 13,400 | |||||||
Estimated reimbursement payments | $ 13,400 | |||||||
Collaborative Arrangement | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Net revenue | 700 | $ 600 | 1,400 | $ 1,100 | ||||
Contract liabilities, current | 800 | 800 | ||||||
Contract liabilities, long-term | $ 10,800 | $ 10,800 |
Income Taxes - Additional Infor
Income Taxes - Additional Information (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2023 | Jun. 30, 2022 | Jun. 30, 2023 | Jun. 30, 2022 | |
Income Tax Disclosure [Abstract] | ||||
Income tax expense (benefit) | $ 25 | $ (219) | $ 13 | $ (161) |
Commitments and Contingencies -
Commitments and Contingencies - Schedule of Annual Commitments Associated with the Contract (Details) $ in Thousands | Jun. 30, 2023 USD ($) |
Other Commitments [Line Items] | |
2023 (remaining six months) | $ 4,212 |
2024 | 10,593 |
2025 | 5,297 |
Total manufacturing commitments | 20,102 |
Product Manufacturing Commitments | |
Other Commitments [Line Items] | |
2023 (remaining six months) | 976 |
2024 | 1,952 |
2025 | 976 |
Total manufacturing commitments | 3,904 |
Dedicated Manufacturing And Development Resources | |
Other Commitments [Line Items] | |
2023 (remaining six months) | 3,236 |
2024 | 6,472 |
2025 | 3,236 |
Total manufacturing commitments | 12,944 |
Exclusive Transduction Suites | |
Other Commitments [Line Items] | |
2024 | 2,169 |
2025 | 1,085 |
Total manufacturing commitments | $ 3,254 |
Commitments and Contingencies_2
Commitments and Contingencies - Schedule of Annual Commitments Associated with the Contract (Parenthetical) (Details) | Jun. 30, 2023 |
Commitments and Contingencies Disclosure [Abstract] | |
Exchange rate, Euro to U.S. Dollar | 1.08 |
Net Loss per Ordinary Share - S
Net Loss per Ordinary Share - Securities Excluded in the Computation of Diluted Net Loss Per Ordinary Share (Details) - shares | 6 Months Ended | |
Jun. 30, 2023 | Jun. 30, 2022 | |
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Common shares attributable to anti-dilutive shares | 129,309,955 | 14,395,417 |
Share Options | ||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Common shares attributable to anti-dilutive shares | 17,034,942 | 13,894,428 |
Unvested Restricted Shares Units | ||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Common shares attributable to anti-dilutive shares | 3,191,423 | 500,989 |
Ordinary Shares | ||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Common shares attributable to anti-dilutive shares | 109,083,590 |