Document and Entity Information
Document and Entity Information - USD ($) | 12 Months Ended | ||
Dec. 31, 2023 | Mar. 07, 2024 | Jun. 30, 2023 | |
Document And Entity Information [Abstract] | |||
Document Type | 10-K | ||
Document Annual Report | true | ||
Document Transition Report | false | ||
Document Period End Date | Dec. 31, 2023 | ||
Entity File Number | 001-38520 | ||
Entity Registrant Name | MEIRAGTX HOLDINGS PLC | ||
Entity Incorporation, State or Country Code | E9 | ||
Entity Tax Identification Number | 98-1448305 | ||
Entity Address, Address Line One | 450 East 29th Street | ||
Entity Address, Address Line Two | 14th Floor | ||
Entity Address, City or Town | New York | ||
Entity Address, State or Province | NY | ||
Entity Address, Postal Zip Code | 10016 | ||
City Area Code | 646 | ||
Local Phone Number | 860-7985 | ||
Title of 12(b) Security | Ordinary Shares, $0.00003881 par value per share | ||
Trading Symbol | MGTX | ||
Security Exchange Name | NASDAQ | ||
Entity Well-known Seasoned Issuer | No | ||
Entity Interactive Data Current | Yes | ||
Entity Voluntary Filers | No | ||
Entity Filer Category | Non-accelerated Filer | ||
Entity Current Reporting Status | Yes | ||
Entity Shell Company | false | ||
Entity Small Business | true | ||
Entity Emerging Growth Company | false | ||
ICFR Auditor Attestation Flag | false | ||
Document Financial Statement Error Correction [Flag] | false | ||
Entity Public Float | $ 259,944,229 | ||
Entity Common Stock, Shares Outstanding | 64,217,166 | ||
Auditor Name | Ernst & Young LLP | ||
Auditor Firm ID | 42 | ||
Auditor Location | Jericho, New York | ||
Entity Central Index Key | 0001735438 | ||
Current Fiscal Year End Date | --12-31 | ||
Document Fiscal Year Focus | 2023 | ||
Document Fiscal Period Focus | FY | ||
Amendment Flag | false |
CONSOLIDATED BALANCE SHEETS
CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
CURRENT ASSETS: | ||
Cash and cash equivalents | $ 129,566 | $ 115,516 |
Accounts receivable - related party | 10,138 | 21,334 |
Prepaid expenses | 5,625 | 8,133 |
Tax incentive receivable | 13,277 | 7,689 |
Other current assets | 1,016 | 1,667 |
Total Current Assets | 159,622 | 154,339 |
Property, plant and equipment, net | 115,896 | 109,266 |
Intangible assets, net | 1,118 | 1,335 |
In-process research and development | 742 | |
Restricted cash | 1,083 | |
Other assets | 1,917 | 1,402 |
Equity method and other investments | 6,766 | 6,326 |
Right-of-use assets - operating leases, net | 15,910 | 20,109 |
Right-of-use assets - finance leases, net | 24,432 | 24,718 |
TOTAL ASSETS | 326,744 | 318,237 |
CURRENT LIABILITIES: | ||
Accounts payable | 16,042 | 16,616 |
Accrued expenses | 42,639 | 39,818 |
Lease obligations, current | 4,193 | 3,884 |
Deferred revenue - related party, current | 2,926 | 15,123 |
Other current liabilities | 1,278 | 6,631 |
Total Current Liabilities | 67,078 | 82,072 |
Deferred revenue - related party | 34,017 | 27,436 |
Lease obligations | 12,952 | 17,331 |
Asset retirement obligations | 2,401 | 2,179 |
Deferred income tax liability | 186 | |
Note payable, net | 72,119 | 71,033 |
Other long-term liabilities | 262 | |
TOTAL LIABILITIES | 188,567 | 200,499 |
SHAREHOLDERS' EQUITY: | ||
Ordinary Shares, $0.00003881 par value, 1,288,327,750 authorized, 63,601,015 and 48,477,209 shares issued and outstanding at December 31, 2023 and December 31, 2022, respectively | 2 | 2 |
Capital in excess of par value | 693,841 | 581,893 |
Accumulated other comprehensive income | (1,435) | 6,047 |
Accumulated deficit | (554,231) | (470,204) |
Total Shareholders' Equity | 138,177 | 117,738 |
TOTAL LIABILITIES AND SHAREHOLDERS' EQUITY | $ 326,744 | $ 318,237 |
CONSOLIDATED BALANCE SHEETS (Pa
CONSOLIDATED BALANCE SHEETS (Parenthetical) - $ / shares | Dec. 31, 2023 | Dec. 31, 2022 |
CONSOLIDATED BALANCE SHEETS | ||
Common stock, par value | $ 0.00003881 | |
Common stock, shares authorized | 1,288,327,750 | |
Common stock, shares issued | 63,601,015 | 48,477,209 |
Common stock, shares outstanding | 63,601,015 | 48,477,209 |
CONSOLIDATED STATEMENTS OF OPER
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS | ||
License revenue - related party | $ 14,017 | $ 15,920 |
Operating expenses: | ||
General and administrative | 47,293 | 46,550 |
Research and development | 103,785 | 85,725 |
Total operating expenses | 151,078 | 132,275 |
Loss from operations | (137,061) | (116,355) |
Other non-operating income (expense): | ||
Foreign currency loss | 9,300 | (9,452) |
Interest income | 2,272 | 777 |
Interest expense | (13,245) | (4,946) |
Gain on sale of nonfinancial assets | 54,208 | |
Fair value adjustments | 499 | 361 |
Net income (loss) | (84,027) | (129,615) |
Other comprehensive income (loss): | ||
Foreign currency translation gain | (7,482) | 8,718 |
Comprehensive income (loss) | (91,509) | (120,897) |
Net income (loss) | $ (84,027) | $ (129,615) |
Basic net loss per ordinary share | $ (1.49) | $ (2.87) |
Diluted net loss per ordinary share | $ (1.49) | $ (2.87) |
Weighted-average number of ordinary shares outstanding, basic | 56,486,525 | 45,177,857 |
Weighted-average number of ordinary shares outstanding, diluted | 56,486,525 | 45,177,857 |
CONSOLIDATED STATEMENT OF SHARE
CONSOLIDATED STATEMENT OF SHAREHOLDERS' EQUITY - USD ($) $ in Thousands | Ordinary Shares | Capital in Excess of Par Value | Accumulated Other Comprehensive Loss | Accumulated Deficit | Total |
Beginning Equity Balance at Dec. 31, 2021 | $ 2 | $ 528,659 | $ (2,671) | $ (340,589) | $ 185,401 |
Beginning Equity Balance (shares) at Dec. 31, 2021 | 44,548,925 | ||||
Share based compensation activity | 26,080 | 26,080 | |||
Share based compensation activity (shares) | 185,770 | ||||
Warrants issued in connection with note payable | 2,273 | 2,273 | |||
Issuance of shares in connection with private placement, net of issuance costs | 24,881 | 24,881 | |||
Issuance of shares in connection with private placement, net of issuance costs (shares) | 3,742,514 | ||||
Other comprehensive income | 8,718 | 8,718 | |||
Net income (loss) for the year ended December 31 | (129,615) | (129,615) | |||
Ending Equity Balance at Dec. 31, 2022 | $ 2 | 581,893 | 6,047 | (470,204) | 117,738 |
Ending Equity Balance (shares) at Dec. 31, 2022 | 48,477,209 | ||||
Share based compensation activity | 26,207 | 26,207 | |||
Share based compensation activity (shares) | 309,755 | ||||
Issuance of shares in connection with asset acquisition | 209 | 209 | |||
Issuance of shares in connection with asset acquisitions (shares) | 40,138 | ||||
Issuance of shares in connection with private placement, net of issuance costs | 85,532 | 85,532 | |||
Issuance of shares in connection with private placement, net of issuance costs (shares) | 14,773,913 | ||||
Other comprehensive income | (7,482) | (7,482) | |||
Net income (loss) for the year ended December 31 | (84,027) | (84,027) | |||
Ending Equity Balance at Dec. 31, 2023 | $ 2 | $ 693,841 | $ (1,435) | $ (554,231) | $ 138,177 |
Ending Equity Balance (shares) at Dec. 31, 2023 | 63,601,015 |
CONSOLIDATED STATEMENT OF SHA_2
CONSOLIDATED STATEMENT OF SHAREHOLDERS' EQUITY (Parenthetical) - Private Placement - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Stock issuance costs | $ 6,418 | |
Ordinary Shares | ||
Stock issuance costs | $ 119 |
CONSOLIDATED STATEMENTS OF CASH
CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Cash flows from operating activities: | ||
Net loss | $ (84,027) | $ (129,615) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Share-based compensation expense | 27,716 | 28,623 |
Foreign currency (gain) loss | (9,300) | 9,452 |
Depreciation and amortization | 13,730 | 8,723 |
Net change in right-of-use assets and liabilities | (222) | (153) |
Loss on disposal of equipment, furniture and fixtures | 13 | |
Loss on equity method investment | 6 | |
Amortization of interest on asset retirement obligations | 177 | 168 |
Amortization of debt discount | 1,083 | 444 |
Fair value adjustment | (499) | (361) |
Impairment of acquired in-process research and development and right-of-use assets | 1,149 | |
Gain on sale of nonfinancial assets | (54,208) | |
(Increase) decrease in operating assets: | ||
Accounts receivable - related party | 9,975 | 1,032 |
Prepaid expenses | 2,690 | (329) |
Tax incentive receivable | (5,148) | 4,139 |
Other current assets | 222 | 519 |
Other assets, net | (1,809) | (173) |
Increase (decrease) in operating liabilities: | ||
Accounts payable | 2,330 | 3,737 |
Accrued expenses | (382) | 12,610 |
Other current liabilities | (1,355) | 4,006 |
Deferred revenue - related party | (7,506) | (15,920) |
Net cash used in operating activities | (105,365) | (73,098) |
Cash flows from investing activities: | ||
Purchase of property, plant and equipment | (20,174) | (44,963) |
Proceeds from sale of nonfinancial assets | 54,208 | |
Net cash used in investing activities | 34,034 | (44,963) |
Cash flows from financing activities: | ||
Exercise of share options | 10 | 231 |
Payments of withholdings on shares withheld for income taxes | (1,519) | (2,774) |
Proceeds from issuance of ordinary shares | 91,950 | 25,000 |
Proceeds from issuance of note payable | 75,000 | |
Payment of issuance costs | (6,418) | (2,257) |
Net cash provided by financing activities | 84,023 | 95,200 |
Net decrease in cash, cash equivalents and restricted cash | 12,692 | (22,861) |
Effect of exchange rate changes on cash, cash equivalents and restricted cash | 2,441 | 674 |
Cash, cash equivalents and restricted cash at beginning of the period | 115,516 | 137,703 |
Cash, cash equivalents and restricted cash at end of the period | 130,649 | 115,516 |
Supplemental disclosure of non-cash transactions: | ||
Fixed asset acquisition included in accounts payable and accrued expenses at end of the period | 2,607 | 7,106 |
Right-of-use assets obtained in exchange for lease liabilities | 1,793 | |
Asset retirement obligations incurred in connection with leases | 9 | |
Warrants issued in connection with note payable | 2,273 | |
Issuance of shares in connection with asset acquisition | 209 | |
Supplemental disclosure of cash flow information: | ||
Cash paid for interest | $ 13,054 | $ 329 |
Principal Business Activity
Principal Business Activity | 12 Months Ended |
Dec. 31, 2023 | |
Principal Business Activity | |
Principal Business Activity | 1. Principal Business Activity The Company MeiraGTx Holdings plc and subsidiaries (the “Company” or “Meira Holdings”), an exempted company incorporated under the laws of the Cayman Islands, is a vertically integrated, clinical-stage gene therapy company with a broad pipeline of late stage clinical programs supported by end-to-end manufacturing capabilities. The Company has an internally developed manufacturing platform process, internal plasmid production for GMP, two GMP viral vector production facilities as well as an in-house Quality Control hub for stability and release, all fit for IND through commercial supply. The Company has core capabilities in viral vector design and optimization and a potentially transformative riboswitch gene regulation platform technology that allows for the precise, dose-responsive control of gene expression by oral small molecules. The Company is focusing the riboswitch platform on delivery of metabolic peptides including GLP-1, GIP, glucagon and PYY using oral small molecules, as well as cell therapy for oncology and autoimmune diseases. Although initially focusing on the eye, central nervous system, and salivary gland, the Company has developed the technology to apply genetic medicine to more common diseases, increasing efficacy, addressing novel targets, and expanding access in some of the largest disease areas where the unmet need remains great. The Company also owns and operates a good manufacturing practices, or GMP, multi-product, multi-viral vector manufacturing facility in London, United Kingdom (“UK”), which includes fill and finish capabilities and can supply the Company’s clinical and potential commercial material. Additionally, the Company’s second, large scale viral vector manufacturing facility and its first plasmid and DNA production facility in Shannon, Ireland, both of which are designed to meet GMP requirements, came online in 2022. Asset Purchase and Related Agreements with Janssen Pharmaceuticals, Inc. On January 30, 2019, the Company entered into a collaboration, option and license agreement with Janssen Pharmaceuticals, Inc. (“Janssen”), one of the Janssen Pharmaceuticals Companies of Johnson & Johnson (the “Collaboration Agreement”), for the research, development and commercialization of gene therapies for the treatment of inherited retinal diseases (“IRD”). Under the terms of the Collaboration Agreement, the Company received an upfront payment of $100.0 million in March 2019 and a $30.0 million milestone payment in December 2021. The Company also received funding for certain research, manufacturing, clinical development and commercialization costs, and had the potential to obtain additional milestone payments upon the achievement of such milestones and royalties on future net sales of products. On December 20, 2023, the Company entered into an Asset Purchase Agreement (“Asset Purchase Agreement”) with Janssen pursuant to which the Company sold and assigned to Janssen, and Janssen purchased and assumed, that certain License Agreement, dated February 5, 2019, by and between UCL Business Plc (now UCL Business Ltd.) (“UCLB”), on the one hand, and MeiraGTx UK II Limited and MeiraGTx Limited, on the other hand (the “UCLB RPGR License Agreement”), relating to the research, development, manufacture and exploitation of the RPGR Product, and other related assets as described in the Asset Purchase Agreement. In connection with entering into the Asset Purchase Agreement, the Company entered into a Termination Agreement with Janssen terminating the Collaboration Agreement. The Company and Janssen also entered into a Supply Agreement on December 20, 2023 pursuant to which the Company agreed to manufacture and supply the RPGR Product for Janssen. Under the Asset Purchase Agreement, Janssen paid the Company a non-refundable upfront cash payment of $65.0 million in December 2023. Additionally, pursuant to and subject to the terms and conditions set forth in the Asset Purchase Agreement, Janssen agreed to pay the Company future contingent consideration of up to an aggregate of $350.0 million, as follows: (i) a milestone payment of $50.0 million in connection with the achievement of the initiation of the extension study for the Phase 3 LUMEOS clinical trial for the RPGR Product, which milestone was achieved during the first quarter of 2024; (ii) $10.0 million upon completion of certain specified development services for the drug substance for the RPGR Product; (iii) $5.0 million upon completion of certain specified development services for the drug product for the RPGR Product; (iv) $175.0 million upon the first commercial sale of an RPGR Product in the United States; (v) $75.0 million upon the first commercial sale of an RPGR Product in at least one of the United Kingdom, France, Germany, Spain and Italy; (vi) $25.0 million upon completion of the transfer of certain manufacturing technology for drug substance and drug product from the Company to Janssen; and (vii) $10.0 million upon regulatory approval of a Janssen-selected manufacturing facility in each of the United States and European Union for commercial manufacture of the RPGR Product. Janssen is also responsible for any royalty or milestone amounts that become payable on the RPGR Product under the UCLB RPGR License Agreement. Basis of Presentation The accompanying consolidated financial statements have been prepared in conformity with accounting principles generally accepted in the United States of America (“GAAP”). Any reference in these notes to applicable guidance is meant to refer to the authoritative United States generally accepted accounting principles as found in the Accounting Standards Codification (“ASC”) and Accounting Standards Update (“ASU”) of the Financial Accounting Standards Board (“FASB”). Liquidity The Company has not yet achieved profitable operations. There is no assurance that profitable operations, if ever achieved, could be sustained on a continuing basis. In addition, development activities, clinical and preclinical testing, and commercialization of the Company’s product candidates will require significant additional financing. The Company’s accumulated deficit at December 31, 2023 totaled $554.2 As of December 31, 2023, the Company had cash, cash equivalents and restricted cash in the amount of $130.6 million, which consisted of depository and money market accounts held at large international banks. The Company estimates that its cash and cash equivalents on hand and accounts receivable – related party at December 31, 2023 will be sufficient to cover its expenses for at least the next twelve months from the date of issuance of these consolidated financial statements. Risks and Uncertainties The Company operates in an industry that is subject to intense competition, government regulation and rapid technological change. The Company’s operations are subject to significant risk and uncertainties including financial, operational, technological, regulatory and other risks, including the potential risk of business failure. The Company’s capital resources and operations to date have been funded primarily with the proceeds from the Collaboration Agreement, Asset Purchase Agreement and private and public equity offerings, as well as the proceeds from the debt financing described in Note 13. In the future, the Company may seek to raise additional capital through equity offerings, debt financings, marketing and distribution arrangements and other collaborations, strategic alliances and licensing arrangements or other sources to enable it to complete the development and potential commercialization of its product candidates. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 12 Months Ended |
Dec. 31, 2023 | |
Summary of Significant Accounting Policies | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies Consolidation The accompanying consolidated financial statements include the accounts of Meira Holdings and its wholly owned subsidiaries: MeiraGTx Limited, a limited company incorporated under the laws of England and Wales; MeiraGTx, LLC, a Delaware limited liability company (“Meira LLC”); MeiraGTx UK II Limited, a limited company incorporated under the laws of England and Wales (“Meira UK II”); MeiraGTx Ireland DAC, a designated activity company incorporated under the laws of Ireland (“Meira Ireland”); MeiraGTx Netherlands B.V., a private company with limited liability incorporated under the laws of the Netherlands (“Meira Netherlands”); MeiraGTx Belgium, a private company with limited liability incorporated under the laws of Belgium (“Meira Belgium”); BRI-Alzan, Inc., a Delaware corporation (“BRI-Alzan”); MeiraGTx Bio Inc., a Delaware corporation (“Meira Bio”); MeiraGTx B.V., a private company with limited liability incorporated under the laws of the Netherlands (“Meira B.V.”); MeiraGTx Neurosciences, Inc., a Delaware corporation (“Meira Neuro”); MeiraGTx Therapeutics, Inc., a Delaware corporation (“Meira Therapeutics”); and MeiraGTx UK Limited, a limited company incorporated under the laws of England and Wales (“Meira UK”). All intercompany balances and transactions between the consolidated companies have been eliminated in consolidation. Use of Estimates Management considers many factors in selecting appropriate financial accounting policies and controls, and in developing the estimates and assumptions that are used in the preparation of these consolidated financial statements. Management must apply significant judgment in this process. In addition, other factors may affect estimates, including expected business and operational changes, sensitivity and volatility associated with the assumptions used in developing estimates, and whether historical trends are expected to be representative of future trends. The estimation process often may yield a range of potentially reasonable estimates of the ultimate future outcomes and management must select an amount that falls within that range of reasonable estimates. This process may result in actual results differing materially from those estimated amounts used in the preparation of the financial statements if these results differ from historical experience, or other assumptions do not turn out to be substantially accurate, even if such assumptions are reasonable when made. In preparing these consolidated financial statements, management used significant estimates in the following areas, among others: collaboration revenue, fair value of nonfinancial assets, stand-alone selling price and material rights in connection with the Asset Purchase and Commercial Supply Agreements, the accounting for research and development costs, share-based compensation, leases, asset retirement obligations, fair value of financial instruments and tax incentive receivable. Cash and Cash Equivalents The Company considers all highly liquid instruments with an original maturity of 90 days or less at the time of purchase to be cash equivalents. Cash and cash equivalents consist of checking and money market accounts held at large international banks that are readily convertible into cash. Restricted Cash Restricted cash represents a guarantee put in place as required by the terms of the research and innovation grant from IDA Ireland which offers financial assistance in establishing the Company’s operations in Shannon, Ireland. The following table provides a reconciliation of the components of cash and cash equivalents and restricted cash reported in the Company’s consolidated balance sheets to the total of the amount presented in the consolidated statements of cash flows (in thousands): December 31, December 31, 2023 2022 Cash and cash equivalents $ 129,566 $ 115,516 Restricted cash 1,083 — Total cash, cash equivalents and restricted cash in the condensed consolidated statement of cash flows $ 130,649 $ 115,516 Financial Instruments The carrying value of accounts receivable-related party, tax incentive receivable, other current assets, and accounts payable reported in the consolidated balance sheets equal or approximate fair value due to their short maturities. Tax Incentive Receivable Meira UK II is eligible to participate in a UK research and development tax incentive programs under which it is eligible to receive a cash refund from His Majesty’s Revenue & Customs (“HMRC”) for a percentage of the qualified research and development costs expended by Meira UK II under the small and medium sized enterprises (“SME”) program and the research and development expenditures credit (“RDEC”) program. The SME cash refund is available to companies with less than 500 employees and annual aggregate revenue of less than 100.0 million euros or total aggregate assets less than 86.0 million euros during the reimbursable period. The Company’s estimate of the amount of cash refund it expects to receive related to the SME and RDEC programs is included in tax incentive receivable in the accompanying consolidated balance sheets and such amounts are recorded as a reduction of research and development expense in the statements of operations. During the years ended December 31, 2023 and 2022, the Company recorded reductions to research and development expenses of $2.9 million and $6.8 million, respectively. In addition, the Company incurs Value Added Tax (“VAT”) on services provided by UK and EU vendors, which it is entitled to reclaim. The Company’s estimate of the amount of cash refund it expects to receive related to VAT was $0.6 million and $1.1 million as of December 31, 2023 and 2022, respectively, which is included in other current assets in the accompanying consolidated balance sheets. Fair Value Measurements Fair value is defined as the price that would be received upon sale of an asset or paid upon transfer of a liability in an orderly transaction between market participants at the measurement date and in the principal or most advantageous market for that asset or liability. The fair value should be calculated based on assumptions that market participants would use in pricing the asset or liability, not on assumptions specific to the entity. In addition, the fair value of liabilities should include consideration of non-performance risk including the Company’s own credit risk. The Company follows ASC Topic 820, Fair Value Measurements and Disclosures three levels based on the extent to which inputs used in measuring fair value are observable in the market. Each fair value measurement is reported in one of the three levels which are determined by the lowest level input that is significant to the fair value measurement in its entirety. These levels are: ● Level 1: Observable inputs such as quoted prices in active markets for identical assets the reporting entity has the ability to access as of the measurement date; ● Level 2: Inputs, other than the quoted prices in active markets, that are observable either directly or indirectly; and ● Level 3: Unobservable inputs in which there is little or no market data, which require the reporting entity to develop its own assumptions. The table below represents the values of the Company’s financial assets and liabilities that are required to be measured at fair value on a recurring basis (in thousands): Fair Value Measurement Using: Significant Significant Other Significant December 31, Observable Inputs Observable Inputs Unobservable Description 2023 (Level 1) (Level 2) (Level 3) Cash equivalents $ 46,868 $ 46,868 $ — $ — Restricted cash $ 1,083 $ 1,083 $ — $ — Fair Value Measurement Using: Significant Significant Other Significant December 31, Observable Inputs Observable Inputs Unobservable Description 2022 (Level 1) (Level 2) (Level 3) Cash equivalents $ 57,336 $ 57,336 $ — $ — Other long-term liabilities $ 262 $ 262 $ — $ — Equity Method and Other Investments The Company accounts for equity investments under the equity method of accounting when the requirements for consolidation are not met, and the Company has significant influence over the operations of the investee. Equity method investments are initially recorded at cost and subsequently adjusted for the Company’s share of net income or loss and cash contributions and distributions and are included in equity method and other investments in the accompanying consolidated balance sheets. Equity investments that do not result in consolidation and are not accounted for under the equity method are measured at fair value, with any changes in fair value recognized in net income (loss). For any such investments that do not have readily determinable fair values, the Company elects the measurement alternative to measure the investments at cost minus impairment, if any, plus or minus changes resulting from observable price changes in orderly transactions for the identical or a similar investment of the same issuer. Equity method investments are reviewed for impairment whenever events or changes in circumstances indicate that the carrying amount may not be recoverable. If it is determined that a loss in value of the equity method investment is other than temporary, an impairment loss is measured based on the excess of the carrying amount of an investment over its estimated fair value. Impairment analyses are based on current plans, intended holding periods, and available information at the time the analysis is prepared. Concentrations of Credit Risk The Company maintains its cash and cash equivalents primarily in depository and money market accounts within two large financial institutions in the United States and one large financial institution in the United Kingdom and Ireland. Cash balances deposited at these major financial banking institutions exceed the insured limit. The Company has not experienced any losses on its bank deposits and believes these deposits do not expose the Company to any significant credit risk. Intangible Assets Intangible assets consist of purchased rights to licensed technology as it relates to the Company’s manufacturing processes and has future alternative use in the Company’s operations. The licensed technology is being amortized on a straight-line basis over Property, Plant and Equipment, Net Property, plant and equipment are stated at cost, net of accumulated depreciation. Depreciation is calculated using the straight-line method over the estimated useful lives of the respective assets. Leasehold improvements are depreciated over the lesser of their useful lives or the term of the lease (see Note 5). The estimated useful lives of the asset categories are as follows: Asset Category Useful Lives Computer and office equipment 3 years Laboratory equipment 5 years Manufacturing equipment 7 years Furniture and fixtures 5 years Leasehold improvements lesser of useful life or remaining term of lease Expenditures for leasehold improvements are capitalized, and expenditures for maintenance and repairs are expensed to operations as incurred. ASC Topic 360, Property, Plant and Equipment Leases The Company accounts for leases in accordance with ASC 842. The Company determines if an arrangement is a lease at contract inception. A lease exists when a contract conveys the right to control the use of identified property, plant, or equipment for a period of time in exchange for consideration. The definition of a lease embodies two conditions: (1) there is an identified asset in the contract that is land or a depreciable asset (i.e., property, plant, and equipment), and (2) the Company has the right to control the use of the identified asset. The Company accounts for the lease and non-lease components as a single lease component. From time to time the Company enters into direct financing lease arrangements that include a lessee obligation to purchase the leased asset at the end of the lease term, a bargain purchase option, or provides for minimum lease payments with a present value of 90% or more of the fair value of the leased asset at the date of lease inception. Operating leases where the Company is the lessee are included in right-of-use (“ROU”) assets – operating leases and lease obligations are included on the Company’s consolidated balance sheets. The lease obligations are initially and subsequently measured at the present value of the unpaid lease payments at the lease commencement date and subsequent reporting periods. Finance leases where the Company is the lessee are included in ROU assets – finance leases, net and lease obligations on the Company’s consolidated balance sheets. The lease obligations are initially measured in the same manner as for operating leases and are subsequently measured at amortized cost using the effective interest method. Key estimates and judgments include how the Company determined (1) the discount rate used to discount the unpaid lease payments to present value, (2) lease term and (3) lease payments. ASC 842 requires a lessee to discount its unpaid lease payments using the interest rate implicit in the lease or, if that rate cannot be readily determined, its incremental borrowing rate. As most of the Company’s leases where it is the lessee do not provide an implicit rate, the Company uses its incremental borrowing rate based on the information available at commencement date in determining the present value of lease payments. The Company’s incremental borrowing rate for a lease is the rate of interest it would have to pay on a collateralized basis to borrow an amount equal to the lease payments under similar terms. The Company uses the implicit rate when readily determinable. The lease term for all of the Company’s leases includes the non-cancellable period of the lease plus any additional periods covered by either a lessee option to extend (or not to terminate) the lease that is reasonably certain to be exercised, or an option to extend (or not to terminate) the lease controlled by the lessor. The ROU asset is initially measured at cost, which comprises the initial amount of the lease liability adjusted for lease payments made at or before the lease commencement date less any lease incentives received. For operating leases, the ROU asset is subsequently measured throughout the lease term at the carrying amount of the lease liability, minus any accrued lease payments, less the unamortized balance of lease incentives received. Lease expense for lease payments is recognized on a straight-line basis over the lease term. For finance leases, the ROU asset is subsequently amortized using the straight-line method from the lease commencement date to the earlier of the end of its useful life or the end of the lease term unless the lease transfers ownership of the underlying asset, or the Company is reasonably certain to exercise an option to purchase the underlying asset. In those cases, the ROU asset is amortized over the useful life of the underlying asset. Amortization of the ROU asset is recognized and presented separately from interest expense on the lease liability. The Company has elected not to recognize ROU assets and lease liabilities for all short-term leases that have a lease term of 12 months or less at lease commencement. Lease payments associated with short-term leases are recognized as an expense on a straight-line basis over the lease term. Asset Retirement Obligations Accounting for asset retirement obligations requires legal obligations associated with the retirement of long-lived assets to be recognized at fair value when incurred and capitalized as part of the related long-lived asset. In the absence of quoted market prices, the Company estimates the fair value of its asset retirement obligations using Level 3 present value techniques, in which estimates of future cash flows associated with retirement activities are discounted using a credit-adjusted risk-free rate. Asset retirement obligations currently reported on the Company’s consolidated balance sheets were measured during a period of historically low interest rates. The impact on measurements of new asset retirement obligations using different rates in the future may be significant. The Company uses estimates to determine the asset retirement obligations at the end of the lease term and discounts such asset retirement obligations using an estimated discount rate. Interest on the discounted asset retirement obligation is amortized over the term of the lease using the effective interest method and is recorded as interest expense in the consolidated statements of operations and comprehensive loss. The change in asset retirement obligations is as follows (in thousands): For the Years Ended December 31, 2023 2022 Balance at beginning of period $ 2,179 $ 2,081 Additional asset retirement obligations incurred during the period — 9 Amortization of interest 177 168 Effects of exchange rate changes 45 (79) Balance at end of period $ 2,401 $ 2,179 IDA Ireland Grant In August 2021, Meira Ireland entered into an agreement pursuant to which it received a grant from IDA Ireland for financial assistance in establishing its operations in Shannon, Ireland. Under the terms of the grant, Meira Ireland is eligible to receive the lesser of €1.0 million or €10,000 for each job created (the “employment grant”) and the lesser of €1.2 million or 4% of the actual expenditure on the provision of machinery and equipment (the “capital grant”). Meira Ireland may apply for a drawdown of the employment grant once a job has been created and the position has been held for a period of at least one month, and may apply for a drawdown of the capital grant once an eligible asset has been purchased and installed, conditioned on the creation of a cumulative number of jobs by the end of the immediately preceding year. An aggregate of 100 jobs must be created to receive the maximum benefit under the capital grant. An application for a drawdown must be accompanied by an audit certification for compliance with the terms of the grant. The Company has a guarantee in place with a bank in favor of IDA Ireland, pursuant to which it restricts cash in the amount of claims made under the grant such that the Company maintains the funds to cover any portion of the grant income that may become repayable in the future. This amount is presented as restricted cash in the accompanying consolidated balance sheet. All expenditures must be completed by December 31, 2024, and the agreement terminates on the later of five years from the date of the last payment from the grant or five years from completion of the capital investment, which is expenditure of at least €30.0 million on eligible machinery and equipment. The Company recognizes grant income when there is reasonable assurance that the Company will comply with the conditions attached to the grant and that it will receive the grant. Grant income from the employment grant is recognized as a deduction from the amount of the related expense, and grant income from the capital grant is deducted from the carrying amount of the related asset and recognized in income over the asset’s useful life in the form of a reduced depreciation charge. The Company received its first drawdown under the grant in 2023, which was comprised of During the six Share-Based Compensation Expense Options The Company grants share options to employees, non-employee members of the Company’s board of directors and non-employee consultants as compensation for services performed. Employee and non-employee members of the board of directors’ awards of share-based compensation are accounted for in accordance with ASC 718, Compensation Stock Compensation, . Using this model, fair value is calculated based on assumptions with respect to (i) the fair value of the Company’s ordinary shares on the grant date; (ii) expected volatility of the Company’s ordinary share price, (iii) the periods of time over which the optionees are expected to hold their options prior to exercise (expected term), (iv) expected dividend yield on the Company’s ordinary shares, and (v) risk-free interest rates. The assumptions underlying these valuations represented management’s best estimate, which involved inherent uncertainties and the application of management’s judgment. As a result, if the Company had used different assumptions or estimates, the fair value of its ordinary shares and its share-based compensation expense could have been materially different. The expected term of share options granted to the optionees is determined using the average of the vesting period and contractual life of the option, an accepted method for the Company’s option grants under the Securities and Exchange Commission’s (“SEC”) Staff Accounting Bulletin No. 107 and No. 110, Share-Based Payment. The Company believes that its future volatility could differ materially during the expected term from the volatility that would be calculated from its historical share prices to date. Consequently, expected volatility is based on an analysis of guideline companies and the Company’s own volatility in accordance with ASC 718. The expected dividend yield is zero as the Company has never paid dividends and does not currently anticipate paying any in the foreseeable future. Risk-free interest rates are based on quoted U.S. Treasury rates for securities with maturities approximating the option’s expected term. Restricted Share Units The Company grants restricted share units (“RSUs”) to employees, non-employee members of the Company’s board of directors and non-employee consultants as compensation for services performed. Awards of RSUs are accounted for in accordance with ASC 718, Compensation Stock Compensation, . Collaboration Arrangements The Company evaluates its collaborative arrangements pursuant to ASC 808, Collaborative Arrangements Revenue from Contracts with Customers with respect to the arrangement, the Company accounts for the arrangement as a collaboration under ASC 808. To date, the Company has entered into two separate collaboration agreements, both of which are with Janssen, which were determined to be within the scope of ASC 808. ASC 808 does not address recognition or measurement matters related to collaborative arrangements. Payments between participants pursuant to a collaborative arrangement that are within the scope of other authoritative accounting literature on income statement classification are accounted for using the relevant provisions of that literature. If the Company concludes that some or all aspects of the arrangement are within the scope of ASC 808 and do not represent a transaction with a customer, the Company recognizes its allocation of the shared costs incurred with respect to the jointly conducted activities pursuant to ASC 730, Research and Development Refer to the discussion in Note 11 for further information related to the accounting for the Collaboration Agreement. Revenue Recognition The Company evaluates the promised goods or services to determine which promises, or group of promises, represent performance obligations in contracts with the Company’s. In contemplation of whether a promised good or service meets the criteria required of a performance obligation, the Company considers the stage of development of the underlying intellectual property, the capabilities and expertise of the customer relative to the underlying intellectual property, and whether the promised goods or services are integral to or dependent on other promises in the contract. When accounting for an arrangement that contains multiple performance obligations, the Company must develop judgmental assumptions, which may include market conditions, reimbursement rates for personnel costs, development timelines and probabilities of regulatory success to determine the stand-alone selling price for each performance obligation identified in the contract. When the Company concludes that a contract should be accounted for as a combined performance obligation and recognized over time, the Company must then determine the period over which revenue should be recognized and the method by which to measure revenue. The Company generally recognizes revenue using a cost-based input method. At inception, the Company determines whether contracts are within the scope of ASC 606 or other topics. For contracts that are determined to be within the scope of ASC 606, the Company recognizes revenue when its customer or collaborator obtains control of promised goods or services, in an amount that reflects the consideration which the Company expects to receive in exchange for those goods or services. To determine revenue recognition the Company performs the following five steps: i. identify the contract(s) with a customer; ii. identify the performance obligations in the contract; iii. determine the transaction price; iv. allocate the transaction price to the performance obligations within the contract; and v. recognize revenue when (or as) the entity satisfies a performance obligation. The Company only applies the five-step model to contracts when it determines that it is probable it will collect the consideration it is entitled to in exchange for the goods or services it transfers to the customer. At contract inception, the Company assesses the goods or services promised within the contract to determine whether each promised good or service is a performance obligation. The promised goods or services in the Company’s arrangements typically consist of a license to the Company’s intellectual property and research, development and manufacturing services. The Company may provide options to additional items in such arrangements, which are accounted for as separate contracts when the customer elects to exercise such options, unless the option provides a material right to the customer. Performance obligations are promises in a contract to transfer a distinct good or service to the customer that (i) the customer can benefit from on its own or together with other readily available resources, and (ii) is separately identifiable from other promises in the contract. Goods or services that are not individually distinct performance obligations are combined with other promised goods or services until such combined group of promises meet the requirements of a performance obligation. The Company determines transaction price based on the amount of consideration the Company expects to receive for transferring the promised goods or services in the contract. Consideration may be fixed, variable, or a combination of both. At contract inception for arrangements that include variable consideration, the Company estimates the probability and extent of consideration it expects to receive under the contract utilizing either the most likely amount method or expected amount method, whichever best estimates the amount expected to be received. The Company then considers any constraints on the variable consideration and includes in the transaction price variable consideration to the extent it is deemed probable that a significant reversal in the amount of cumulative revenue recognized will not occur when the uncertainty associated with the variable consideration is subsequently resolved. The Company then allocates the transaction price to each performance obligation based on the relative standalone selling price and recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) control is transferred to the customer and the performance obligation is satisfied. For performance obligations which consist of licenses and other promises, the Company utilizes judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time and, if over time, the appropriate method of measuring progress. The Company evaluates the measure of progress each reporting period and, if necessary, adjusts the measure of performance and related revenue recognition. If there are multiple performance obligations, the Company allocates the transaction price to each performance obligation based on their estimated standalone selling prices (“SSP”). The Company estimates the SSP for each performance obligation by considering information such as market conditions, entity-specific factors, and information about its customer that is reasonably available. The Company considers estimation approaches that allow it to maximize the use of observable inputs. These estimation approaches may include the adjusted market assessment approach, the expected cost plus a margin approach or the residual approach. The Company also considers whether to use a different estimation approach or a combination of approaches to estimate the SSP for each performance obligation. Developing certain assumptions (e.g., treatable patient population, expected market share, probability of success and product profitability, and discount rate based on weighted-average cost of capital) to estimate the SSP of a performance obligation requires significant judgment. The Company records amounts as accounts receivable when the right to consideration is deemed unconditional. When consideration is received, or such consideration is unconditionally due, from a customer prior to transferring goods or services to the customer under the terms of a contract, a contract liability is recorded as deferred revenue. Amounts received prior to satisfying the revenue recognition criteria are recognized as deferred revenue in the Company’s consolidated balance sheet. Amounts expected to be recognized as revenue within the 12 months following the balance sheet date are classified as deferred revenue – related party, current. Amounts not expected to be recognized as revenue within the 12 months following the balance sheet date are classified as deferred revenue – related party. The Company’s collaboration and revenue arrangements include the following: Up-front License Fees: If a license is determined to be distinct from the other performance obligations identified in the arrangement, the Company recognizes revenues from nonrefundable, up-front fees allocated to the license when the license is transferred to the licensee and the licensee is able to use and benefit from the license. For licenses that are bundled with other promises, the Company utilizes judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time and, if over time, the appropriate method of measuring progress for purposes of recognizing revenue from non-refundable, up-front fees. The Company evaluates the measure of progress each reporting period and, if necessary, adjusts the measure of performance and related revenue recognition. Milestone Payments: At the inception of an agreement that includes research and development milestone payments, the Company evaluates each milestone to determine when and how much of the milestone to include in the transaction price. The Company first estimates the amount of the milestone payment that the Company could receive using either the expected value or the most likely amount approach. The Company primarily uses the most likely amount approach as that approach is generally most predictive for milestone payments with a binary outcome. Then, the Company considers whether any portion of that estimated amount is subject to the variable consideration constraint (that is, whether it is probable that a significant reversal of cumulative revenue would not occur upon resolution of the uncertainty.) The Company updates the estimate of variable consideration included in the transaction price at each reporting date which includes updating the assessment of the likely amount of consideration and the application of the constraint to reflect current facts and circumstances. Royalties: For arrangements that include sales-based royalties, including milestone payments based on a level of sales, and the license is deemed to be the predominant item to which the royalties relate, the Company will recognize revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied). To date, the Company has not recognized a |
Equity Method and Other Investm
Equity Method and Other Investments | 12 Months Ended |
Dec. 31, 2023 | |
Equity Method and Other Investments | |
Equity Method and Other Investments | 3. Equity Method and Other Investments The Company’s investments consist of the following (in thousands): December 31, 2023 Investee Investment Type Ownership Percentage Carrying Value Cost Basis Visiogene LLC Equity Method Investment 25 % $ 5,150 $ 5,165 Other Equity Investment 1.6 % 1,616 1,500 Total equity method and other investments $ 6,766 $ 6,665 Visiogene LLC On January 4, 2021, the Company and Visiogene LLC (“Visiogene”) entered into a License and Investment Agreement (“Visiogene License Agreement”) for an exclusive, worldwide license to certain of Visiogene’s intellectual property relating to ocular gene therapy. Concurrently, the Company and Visiogene entered into a Preferred Unit Purchase Agreement (“Visiogene Unit Agreement”) pursuant to which the Company purchased 3,000,000 Visiogene preferred units. In connection with the two Visiogene agreements, the Company paid $5.0 million in cash and issued to Visiogene 75,000 ordinary shares of the Company with a fair market value of $1.2 million based on the closing price of the Company’s ordinary shares on the date of closing. The Company accounted for the payments under the Visiogene License Agreement and Visiogene Unit Agreement as a basket transaction and allocated $1.0 million to the Visiogene License Agreement and the remaining $5.2 million was allocated to the Visiogene preferred units. The $1.0 million allocated to the Visiogene License Agreement was expensed as acquired in-process research and development as the Company determined there was no alternative future use. The Company accounts for this investment using the equity method of accounting. During the years ended December 31, 2023 and 2022, the Company recorded de minimis research and development expenses related to the Company’s share of Visiogene’s losses. Other Equity Investment During the years ended December 31, 2023 and 2022, the Company recognized a $0.5 million increase in carrying value and $0.3 million impairment, respectively due to a change in the value of the Company’s investment. The change in carrying value was recorded as |
Prepaid Expenses
Prepaid Expenses | 12 Months Ended |
Dec. 31, 2023 | |
Prepaid Expenses | |
Prepaid Expenses | 4. Prepaid Expenses Prepaid expenses at December 31, 2023 and 2022 consist of the following (in thousands): December 31, December 31, 2023 2022 Clinical trial costs $ 1,530 $ 3,411 Dues and license fees 1,439 909 Insurance 861 1,485 Research and development 685 1,220 Facilities costs 630 539 Consulting 243 — Manufacturing costs 38 347 Other 199 222 $ 5,625 $ 8,133 |
Property, Plant and Equipment,
Property, Plant and Equipment, net | 12 Months Ended |
Dec. 31, 2023 | |
Property, Plant and Equipment, net | |
Property, Plant and Equipment, net | 5. Property, Plant and Equipment, net Property, plant and equipment, net at December 31, 2023 and 2022 consist of the following (in thousands): December 31, December 31, 2023 2022 Leasehold improvements $ 103,082 $ 91,053 Manufacturing equipment 22,646 17,373 Laboratory equipment 15,389 13,804 Computer and office equipment 7,370 6,787 Furniture and fixtures 734 642 149,221 129,659 Less: Accumulated depreciation and amortization (33,325) (20,393) $ 115,896 $ 109,266 In connection with certain operating leases, the Company has determined that it has asset retirement obligations in the aggregate amount of $3.9 million at the end of those leases. The Company discounted the asset retirement obligations using an 8% discount rate and recorded an asset retirement obligation in the aggregate amount of $1.8 million, which is included in leasehold improvements and is being amortized over the term of the respective leases. Depreciation and amortization expense related to property, plant and equipment was $12.3 million and $7.3 million for the years ended December 31, 2023 and 2022, respectively. |
Intangible Assets
Intangible Assets | 12 Months Ended |
Dec. 31, 2023 | |
Intangible Assets | |
Intangible Assets | 6. Intangible Assets In November 2020, the Company entered into a non-exclusive, royalty-free technology license agreement that required the Company to pay an upfront payment to the licensor of $2.1 million. The Company accounted for the transaction as an asset acquisition and recorded an intangible asset as it was determined to have alternative future uses in connection with the Company’s manufacturing capabilities. The following table presents the details of the Company’s intangible assets as of December 31, 2023 and 2022 (in thousands): December 31, December 31, 2023 2022 Licensed Technology $ 2,000 $ 1,900 Less: Accumulated amortization (882) (565) $ 1,118 $ 1,335 The intangible asset is being amortized over a period of seven years. Amortization expense of $0.3 million and $0.2 million was recorded as a component of research and development expenses for the years ended December 31, 2023 and 2022, respectively. As of December 31, 2023, the expected amortization expense for the next four years is as follows (in thousands): Amortization Expense 2024 $ 285 2025 285 2026 285 2027 263 Total amortization $ 1,118 |
Accrued Expenses
Accrued Expenses | 12 Months Ended |
Dec. 31, 2023 | |
Accrued Expenses | |
Accrued Expenses | 7. Accrued Expenses Accrued expenses at December 31, 2023 and 2022 were comprised of the following (in thousands): December 31, December 31, 2023 2022 Compensation and benefits $ 12,129 $ 9,600 Clinical trial costs 8,713 13,041 Professional fees 6,499 732 Research and development 5,834 7,400 Interest on Tranche 1 Notes 2,936 — Manufacturing costs 2,634 4,326 Consulting 2,104 694 Fixed assets 1,472 3,093 Rent and facilities costs 142 — Other 176 932 $ 42,639 $ 39,818 |
Share-Based Compensation
Share-Based Compensation | 12 Months Ended |
Dec. 31, 2023 | |
Share-Based Compensation | |
Share-Based Compensation | 8. Share-Based Compensation Equity Incentive Plans The Company’s 2018 Incentive Award Plan and 2016 Equity Incentive Plan (collectively, the “Plans”), were adopted by the Company’s board of directors and shareholders. Under the Plans, the Company has granted share options and restricted share units (“RSUs”) to selected officers, employees, non-employee members of the Company’s board of directors and non-employee consultants. The Company’s board of directors or a committee thereof administers the Plans. Upon the adoption of the 2018 Incentive Award Plan, the Company ceased issuing awards under the 2016 Equity Incentive Plan. The number of shares available for issuance under the 2018 Incentive Award Plan are increased on January 1 of each calendar year beginning in 2019 and ending in and including 2028, by an amount equal to the lesser of (A) 4% of the ordinary shares outstanding on the final day of the immediately preceding calendar year and (B) a smaller number of shares determined by the Company's board of directors. Under the 2018 Incentive Award Plan the Company initially reserved up to 3,054,996 shares for issuance, which has been increased to 11,110,748 as of December 31, 2023. As of December 31, 2023, 310,488 Options A summary of the Company’s share option activity related to employees, non-employee members of the board of directors and non-employee consultants as of and for the years ended December 31, 2023 and 2022 is as follows (in thousands, except share and per share amounts): Weighted- Weighted- Average Average Remaining Number of Exercise Contractual Options Price Term (years) Outstanding at December 31, 2022 6,858,409 $ 14.03 6.86 years Granted 1,546,840 $ 8.49 Exercised (3,864) $ 2.64 Forfeited (174,678) $ 14.95 Outstanding at December 31, 2023 8,226,707 $ 12.96 6.35 years Options exercisable at December 31, 2023 5,709,190 $ 13.30 5.40 years Options vested and expected to vest at December 31, 2023 8,226,707 $ 12.96 6.35 years Aggregate intrinsic value of options outstanding as of December 31, 2023 $ 2,262 Aggregate intrinsic value of options exercisable as of December 31, 2023 $ 2,228 Options granted under the Plans have a maximum contractual term of ten years. Options granted generally vest 25% on the first anniversary of the date of grant and the balance ratably over the next 36 months. Options granted to directors when they join the board generally vest in 36 equal monthly installments following the date of grant, and annual options granted to directors generally vest on the earlier of the first anniversary of the date of grant or the day before the Company’s next annual meeting of shareholders after the date of grant. The company recorded the following share-based compensation expense in connection with the options for the years ended December 31, 2023 and 2022 (in thousands): Years Ended December 31, 2023 2022 Research and development $ 8,577 $ 10,431 General and administrative 5,054 5,654 Total share-based compensation $ 13,631 $ 16,085 The total fair value of options vested during the years ended December 31, 2023 and 2022 was $15.3 million and $16.2 million, respectively. The weighted average grant date fair value of options granted during the years ended December 31, 2023 and 2022 was $5.67 and $12.81, respectively. The grant date fair values of the share options granted were estimated using the Black-Scholes option valuation model with the following ranges of assumptions (see Note 2): 2023 2022 Risk-free interest rate 3.86 - 4.48% 1.56 - 4.23% Expected volatility 72% 80% Expected dividend yield 0% 0% Expected term (in years) 3.6 - 6.1 5.5 - 6.1 As of December 31, 2023, the total compensation expense relating to unvested options granted that had not yet been recognized was $18.3 million, which is expected to be recognized over a period of 3.7 years. The Company will issue shares upon exercise of options from ordinary shares reserved under the Plans. Restricted Share Units A summary of the Company’s RSU activity related to employees, non-employee members of the board of directors and non-employee consultants for the years ended December 31, 2023 and 2022 is as follows: Weighted- Number of Average Restricted Grant Date Share Units Fair Value Outstanding at December 31, 2022 2,182,500 $ 18.59 Granted 1,097,500 $ 8.52 Vested (618,750) $ 15.17 Outstanding at December 31, 2023 2,661,250 $ 15.24 RSUs granted generally vest 50% on the second anniversary of the date of grant and 25% on the third and fourth The company recorded the following share-based compensation expense in connection with the RSUs for the years ended December 31, 2023 and 2022 (in thousands): Years Ended December 31, 2023 2022 Research and development $ 4,203 $ 3,734 General and administrative 9,882 8,804 Total share-based compensation $ 14,085 $ 12,538 As of December 31, 2023, the total compensation expense relating to unvested RSUs granted that had not yet been recognized was $25.3 million, which is expected to be recognized over a period of 3.4 years To satisfy employee minimum statutory tax withholding requirements for restricted share units that vest, the Company withholds a portion of the vesting ordinary shares. During the years ended December 31, 2023 and 2022, the Company withheld 237,859 and 128,812 ordinary shares with a total value of approximately $1.5 million and $2.8 million, respectively. These amounts are presented as a cash outflow from financing activities in the accompanying consolidated statement of cash flows. During the years ended December 31, 2023 and 2022 the Company recognized total share-based compensation expense in the accompanying consolidated statements of operations and comprehensive loss as follows (in thousands): Years Ended December 31, 2023 2022 Research and development $ 12,780 $ 14,165 General and administrative 14,936 14,458 Total share-based compensation $ 27,716 $ 28,623 During the year ended December 31, 2023, the Company modified certain awards for six participants and recognized $0.3 million related to the modifications, $0.1 million of which was recognized in research and development expense and $0.2 million was recognized in general and administrative expense. During the year ended December 31, 2022, the Company modified certain awards for three participants and recognized $0.3 million related to the modifications, $0.2 million of which was recognized in research and development expense and $0.1 million was recognized in general and administrative expense. The terms of such modifications included, on an award-by-award basis, acceleration of the vesting period and/or extensions of the post-employment period to exercise. The Company does not expect to realize any tax benefits from its share option activity or the recognition of share-based compensation expense because the Company currently has net operating losses and has a full valuation allowance against its deferred tax assets. Accordingly, no amounts related to excess tax benefits have been reported in cash flows from operations or cash flows from financing |
Ordinary Shares
Ordinary Shares | 12 Months Ended |
Dec. 31, 2023 | |
Ordinary Shares | |
Ordinary Shares | 9. Ordinary Shares 2023 May 2023 Private Placement On May 3, 2023, the Company entered into a securities purchase agreement with certain accredited investors, pursuant to which the Company, in a private placement, agreed to issue and sell an aggregate of 10,773,913 ordinary shares at a purchase price of $5.75 per share, for gross proceeds of approximately $62.0 million and incurred issuance costs of approximately $4.1 million. The closing occurred on May 5, 2023. Sanofi Private Placement On October 30 , 2023, the Company entered into a n Investment Agreement with Sanofi Foreign Participations B.V., a wholly-owned subsidiary of Sanofi, and solely for the limited purposes set forth therein, Sanofi, pursuant to which, the Company, in a private placement, issued an aggregate of 4,000,000 ordinary shares, at a purchase price of $7.50 per share for gross proceeds of $30.0 million and incurred issuance costs of approximately $2.3 million. 2022 Private Placement On November 9, 2022, the Company entered into a securities purchase agreement with Johnson & Johnson Innovation – JJDC, Inc. (“JJDC”), the investment arm of Johnson & Johnson, pursuant to which the Company, in a private placement, agreed to issue and sell to JJDC an aggregate of 3,742,514 ordinary shares at a purchase price of $6.68 per share, for gross proceeds of approximately $25.0 million. |
Income Taxes
Income Taxes | 12 Months Ended |
Dec. 31, 2023 | |
Income Taxes | |
Income Taxes | 10. Income Taxes For the years ended December 31, 2023 and 2022, the Company recognized a tax benefit As of December 31, 2023, the Company had U.S. federal and state net operating losses (“NOLs”) and foreign carryforward tax losses which are available to reduce future taxable income of (in thousands): Federal State/City United Kingdom $ 169,599 $ — United States $ 65,549 $ 14,741 Ireland $ 47,681 $ — Other $ 29,399 $ — All of the Company’s carryforward tax losses will be indefinitely carried forward. Also, as of December 31, 2023, the Company had orphan drug and research and development credits in the U.S. in the amount of million in the UK which can be carried forward indefinitely. Due to changes in apportionment and legislative changes, state NOLs decreased by change of ownership occurred in April 2016 and again in June 2018, but there was not a limit for utilizing these losses to offset the 2022 income. The Company’s pre-tax income (loss) is as follows (in thousands): December 31, 2023 December 31, 2022 United Kingdom $ 12,881 $ (54,636) United States (63,017) (54,513) Ireland (29,965) (16,661) Other (3,926) (3,805) $ (84,027) $ (129,615) The Company is subject to the corporate tax rate in the UK as a limited UK corporation. The following table summarizes a reconciliation of income tax benefit compared with the amounts at the UK statutory income tax rate (in thousands): December 31, 2023 December 31, 2022 Statutory rate (19,746) 23.50 % (24,627) 19.00 % Permanent differences - other 2,391 (2.85) % 2,323 (1.79) % RTP and other adjustment (1,165) 1.39 % 1,244 (0.96) % State and local rate, net of federal tax (698) 0.83 % (5,660) 4.37 % U.K. tax credit (4,028) 4.79 % 2,296 (1.77) % U.S. tax credit (9,830) 11.70 % (2,436) 1.88 % Foreign tax rate differential 4,558 (5.42) % 195 (0.15) % UK rate change (25% & 19%, respectively) 239 (0.28) % (1,973) 1.52 % US state rate change 15,394 (18.32) % (3) 0.00 % Section 162(m) deferred adjustment 2,728 (3.25) % 1,386 (1.07) % Disallowed interest 2,812 (3.35) % — 0.00 % Change in valuation allowance 7,345 (8.74) % 27,255 (21.03) % Actual income tax benefit effective tax rate — 0.00 % — 0.00 % The Expense/(Benefit) for income taxes from continuing operations consists of the following (in thousands): December 31, 2023 December 31, 2022 Current Tax Expense/(Benefit) United Kingdom — — United States — — Other — — Total Current — — Deferred Tax Expense/(Benefit) United Kingdom 2,393 (8,708) United States (6,251) (17,466) Ireland (3,963) (1,498) Netherlands (1,840) 1,717 Total Deferred (9,661) (25,955) Change in Valuation Allowance 9,661 25,955 Total Income Tax Expense/(Benefit) — — Deferred Tax Assets/(Liabilities) (in thousands): December 31, 2023 December 31, 2022 Deferred Tax Assets: Net operating loss carryforwards $ 70,650 $ 74,350 Capitalized research and development 19,606 16,288 Share-based compensation 14,290 13,684 R&D credit 21,687 10,837 Lease liability 4,070 6,461 Other 1,121 3,745 Deferred tax assets 131,424 125,365 Deferred Tax Liabilities: Indefinite-lived intangibles and fixed assets — (186) Depreciation (1,763) (2,935) Right of use assets (3,777) (6,207) Less: valuation allowance (125,884) (116,223) Net deferred tax liability $ — $ (186) ASC 740 requires a valuation allowance to reduce the deferred tax assets reported if, based on the weight of available evidence, it is more likely than not that some portion or all of the deferred tax assets will not be realized. After consideration of all the evidence, both positive and negative, the Company has recorded a full valuation allowance, after consideration of the reversal of the deferred tax liabilities for the ROU assets and fixed assets, against its deferred tax assets at December 31, 2023 and 2022 because the Company's management has determined that is it more likely than not that these assets will not be fully realized. Changes to the UK corporation tax rates have been announced which will impact future accounting periods. Finance Act 2021 increases the UK corporation tax rate from 19% to 25% effective April 1, 2023 for companies with profits in excess of GBP 250,000. As the Company does not expect to be able to utilize its carryforward tax losses in the UK until after April 2023, the deferred tax has been calculated using a tax rate of 25%. As of December 31, 2023 and 2022, the Company recorded unrecognized tax positions of $2.0 million and $0.9 million, respectively. The unrecognized tax positions are netted with deferred tax assets above with full valuation allowance. The changes to unrecognized tax positions for 2023 and 2022 were as follows (in thousands): December 31, 2023 December 31, 2022 Unrecognized tax benefits as of January 1 $ 937 $ 666 Gross increases/(decreases) related to current year 756 279 Gross increases/(decreases) related to prior years 336 (8) Foreign currency translation — — Unrecognized tax positions as of December 31 $ 2,029 $ 937 The Company will recognize interest and penalties related to uncertain tax positions in income tax expense. As of December 31, 2023 and 2022, the Company had no accrued The Company files income tax returns in the United States, UK, various foreign jurisdictions and various U.S. state jurisdictions. In the U.S., all years remain subject to examination. The earliest year subject to examination in the UK is 2020. MeiraGTx Holdings plc is a UK tax resident with no earnings in its foreign subsidiaries and the Company does not expect any temporary basis difference in its investment in these subsidiaries to reverse in the foreseeable future. Therefore, the Company has not recorded deferred taxes on the outside basis difference in its foreign subsidiaries. It is not probable to compute the amounts, if any. |
Related Party Transactions
Related Party Transactions | 12 Months Ended |
Dec. 31, 2023 | |
Related Party Transactions | |
Related Party Transactions | 11. Related Party Transactions Relationship with Janssen Pharmaceuticals, Inc. Collaboration Agreement On January 30, 2019, the Company entered into a Collaboration Agreement with Janssen for the research, development and commercialization of gene therapies for the treatment of IRD. Under the agreement, Janssen paid the Company a non-refundable upfront fee of $100.0 million. Janssen and the Company agreed to collaborate to develop the Company’s clinical programs in retinitis pigmentosa and two genetic forms of achromatopsia and Janssen had the exclusive right to commercialize these three product candidates (“Clinical IRD Product Candidates”) globally. Pursuant to the Collaboration Agreement, the Company and Janssen also agreed on a research collaboration to develop a pipeline of preclinical inherited retinal disease gene therapy candidates (“Research IRD Product Candidates”). The parties agreed to select and prioritize the Research IRD Product Candidates and Janssen had the right to opt-in for a fee for each of the specified targets (each an “Option Target”) to obtain certain development, manufacturing and commercialization rights for the Research IRD Product Candidates. Unless terminated earlier under certain termination clauses, the Collaboration Agreement was to continue in effect, on a product-by-product and country-by-country basis, until such time as the royalty terms expired in such country. The Company had determined enforceable rights existed in the Collaboration Agreement as the termination clauses were substantive termination penalties by way of the non-refundable upfront fee and the reversion of any licensed intellectual property granted to Janssen upon the termination of the agreement. Under the Collaboration Agreement, the Company and Janssen were jointly developing Clinical IRD Product Candidates to permit Janssen to commercialize such Clinical IRD Product Candidates under an exclusive license from the Company. In general, the Company had the primary responsibility to develop each Clinical IRD Product Candidate in accordance with the development plan for each Clinical IRD Product Candidate, including where applicable, conducting any necessary research in order to submit the applicable regulatory filings to regulatory authorities. The Company agreed to manufacture these products in its GMP manufacturing facilities for both clinical and commercial supply. Janssen agreed to pay 100% of the clinical and commercialization costs of the products and the Company was eligible to receive untiered 20% royalties on net sales of products and additional development and commercialization milestones up to $340.0 million. The Company received a milestone payment of Asset Purchase and Related Agreements On December 20, 2023, the Company entered into the Asset Purchase Agreement with Janssen pursuant to which the Company sold and assigned to Janssen, and Janssen purchased and assumed, the UCLB RPGR License Agreement relating to the research, development, manufacture and exploitation of the RPGR Product, and other related assets as described in the Asset Purchase Agreement. Simultaneously, the Company and Janssen also entered into a Supply Agreement pursuant to which the Company agreed to manufacture and supply the RPGR Product for Janssen. Under the Supply Agreement, MeiraGTx UK II, together with its affiliates, will manufacture commercial supply of the RPGR Product for Janssen for an initial term of four years, with Janssen having an option to extend the Supply Agreement for a fifth year upon written notification. Janssen may terminate the Supply Agreement for convenience upon 90 days’ written notice with payment of a termination fee. Under the Asset Purchase Agreement, Janssen paid the Company a non-refundable upfront fee of $65.0 million in December 2023 and the Company is eligible to receive fees from commercial supply of the RPGR Product and in addition, milestones of up to $350.0 million, as follows: (i) a milestone payment of $50.0 million in connection with the achievement of the initiation of the extension study for the Phase 3 LUMEOS clinical trial for the RPGR Product, which milestone was achieved during the first quarter of 2024; (ii) $10.0 million upon completion of certain specified development services for the drug substance for the RPGR Product; (iii) $5.0 million upon completion of certain specified development services for the drug product for the RPGR Product; (iv) $175.0 million upon the first commercial sale of an RPGR Product in the United States; (v) $75.0 million upon the first commercial sale of an RPGR Product in at least one of the United Kingdom, France, Germany, Spain and Italy; (vi) $25.0 million upon completion of the transfer of certain manufacturing technology for drug substance and drug product from the Company to Janssen; and (vii) $10.0 million upon regulatory approval of a Janssen-selected manufacturing facility in each of the United States and European Union for commercial manufacture of the RPGR Product. Janssen is also responsible for any royalty or milestone amounts that become payable on the RPGR Product under the UCLB RPGR License Agreement. Revenue Recognition under the Janssen Agreements Collaboration Agreement The Company evaluated the potential performance obligations in the Collaboration Agreement pursuant to ASC 606, which included the exclusive license to Clinical IRD Product Candidates, the research, development and manufacturing services (“the services”), and the participation in various joint committees and determined that none of the performance obligations by themselves were distinct. Goods and services that are not distinct are bundled with other goods or services in the contract until a bundle of goods or services that is distinct is created. The services, when combined with the licenses, represent a bundle and should be accounted for as a single performance obligation due to the relevance of the services to the value of the early-stage license and the potential for the intellectual property to be significantly modified during the services period. The Company also evaluated whether or not the right to purchase exclusive option rights for specified Research IRD Product Candidates represents future performance obligations and concluded that these represent a separate buyer decision at market rates, rather than a material right performance obligation. As such, these options have been excluded from the initial allocation of transaction price and the Company will account for these options as separate contracts when and if Janssen elects to exercise the options. Under ASC 606, the Company recognized collaboration revenue using the cost-to-cost input method, which it believes best depicts the transfer of control to the customer. Under the cost-to-cost input method, the extent of progress towards completion is measured based on the ratio of actual costs incurred to the total estimated costs expected upon satisfying the combined performance obligation by the potential product candidate. Under this method, revenue is being recorded as a percentage of the estimated transaction price based on the extent of progress towards completion. Under ASC 606, the estimated transaction price includes variable consideration subject to constraints. The Company does not include variable consideration to the extent that it is probable that a significant reversal in the amount of cumulative revenue recognized will occur when any uncertainty associated with the variable consideration is resolved. The estimate of the Company’s measure of progress and estimate of variable consideration to be included in the transaction price will be updated at each reporting date as a change in estimate. The amount related to the unsatisfied portion will be recognized as that portion is satisfied over time. Under ASC 606 the Company accounts for (i) the licenses it conveyed with respect to the Clinical IRD Product Candidates and (ii) its obligations to perform services as a single performance obligation under the Collaboration Agreement with Janssen on a product candidate basis. Janssen’s right to purchase exclusive options to obtain certain development, manufacturing and commercialization rights are accounted for separately as they do not represent material rights, based on the criteria of ASC 606. Upon the exercise of any purchased option by Janssen, the contract promises associated with an Option Target would use a separate cost-to-cost model for purposes of revenue recognition under ASC 606. In 2019, the Company received a $100.0 million non-refundable upfront fee from Janssen and during the year ended December 31, 2021, the Company received a $30.0 million milestone payment. The Company allocated these amounts plus other variable consideration not subject to constraint to each identified performance obligation using a combination of methods allowable under ASC 606. The Company applies the practical expedient in Topic 606 and does not include disclosures regarding amounts for variable consideration allocated to wholly-unsatisfied performance obligations or wholly-unsatisfied distinct goods that form part of a single performance obligation, if any. This variable consideration includes expected reimbursement of research and development costs. Asset Purchase and Related Agreements The agreements entered into in December 2023 were executed at the same time and were negotiated with a single commercial objective; therefore, the contracts were combined and accounted for as a single contract. These agreements were accounted for as a termination of the existing Collaboration Agreement and the creation of a new contract where the transaction price includes the remaining deferred revenue – related party from the terminated agreement of $30.6 million, the fixed upfront payment of $65.0 million under the Asset Purchase Agreement, and an aggregate of $1.8 million estimated variable consideration for transition services, offset by a credit of $5.1 million for pre-funded inventory, totaling $92.3 million. The transaction price was allocated to four performance obligations on a relative SSP basis, subject to certain exceptions for discounts and variable consideration. As the SSPs are not directly observable for any of the distinct goods and services, the SSPs were estimated based on a valuation. The total transaction price of $92.3 million was allocated to the performance obligations with respect to SSPs as follows: process performance qualification (“PPQ”) services in the amount of $2.9 million, material rights representing the commercial supply of RPGR Product and an in-substance contract renewal option in the amount of $6.9 million, manufacturing technology transfer in the amount of $28.7 million, and the sale of nonfinancial assets representing the sale and transfer of all the Company’s right, title, and interest in the intellectual property related to the RPGR Product and the assignment of the UCLB RPGR License Agreement to Janssen in the amount of $53.8 million. The transaction price allocated to PPQ services will be recognized over time using an inputs method measure of progress. The transaction price allocated to the material right for the commercial supply of RPGR Product will be recorded as deferred revenue until Janssen exercises its option to purchase supply and the Company transfers control of such supply to Janssen. The transaction price allocated to the in-substance renewal option (material right) will be recorded as deferred revenue until Janssen exercises the option and the Company transfers control of the underlying goods or services to Janssen. The Company will account for the exercise of the in-substance renewal option (material right) as a continuation of the existing contract (i.e., a change in the transaction price). The transaction price allocated to the technology transfer will be recognized over time using an inputs method measure of progress. The Company will recognize a gain for the difference between the carrying amount of the nonfinancial assets and the consideration allocated to that unit of account when control of the nonfinancial assets transfers in accordance with ASC 610-20, Other Income - Gains and Losses from the Derecognition of Nonfinancial Assets During the year ended December 31, 2023, the Company recognized a gain of $54.2 million related to the sale of nonfinancial assets which is included in other income in the consolidated statement of operations and comprehensive loss. As of December 31, 2023, the aggregate transaction price allocated to unsatisfied performance obligations was $36.9 million which the Company expects to recognize over an estimated period of approximately 4.0 years. A summary of the deferred revenue recognition is as follows (in thousands): Deferred revenue at December 31, 2021 $ 64,866 Deferred revenue recognized as license revenue during the year ended December 31, 2022 (15,920) Effects of exchange rate changes (6,387) Deferred revenue at December 31, 2022 $ 42,559 Deferred revenue recognized as license revenue during the year ended December 31, 2023 (14,017) Effects of exchange rate changes 2,033 Remaining deferred revenue under the Collaboration Agreement 30,575 Standalone selling price allocated to performance obligations 7,469 Effects of exchange rate changes (1,101) Deferred revenue at December 31, 2023 $ 36,943 During the years ended December 31, 2023 and 2022, the Company recognized $14.0 million and $15.9 million, respectively, of deferred revenue – related party in connection with the Collaboration Agreement as license revenue. The remaining deferred revenue – related party from the terminated Collaboration Agreement of $30.6 million was included in the overall transaction price of the agreements entered into in December 2023. The Company also recognized $70.4 million and $73.3 million during the years ended December 31, 2023 and 2022, respectively, related to the reimbursement of research and development expenses under the Collaboration Agreement. Private Placement On February 27, 2019, in connection with a private placement, the Company issued 2,898,550 ordinary shares to JJDC, the investment arm of Johnson & Johnson and owner of Janssen, on the same terms and conditions as the other investors in the offering. After the offering, JJDC became a related party. On November 9, 2022, the Company entered into a securities purchase agreement with JJDC, pursuant to which the Company, in a private placement, agreed to issue and sell to JJDC an aggregate of 3,742,514 ordinary shares at a purchase price of $6.68 per share, for gross proceeds of approximately $25.0 million. Debt Financing On August 2, 2022 the Company, as borrower, and Meira UK II and Meira Ireland, as guarantors (the “Subsidiary Guarantors”), entered into a senior secured financing arrangement (the “Financing Agreement”) by and among the Company, the Subsidiary Guarantors, the lenders and other parties from time to time party thereto and Perceptive Credit Holdings III, LP, as administrative agent and lender (“Perceptive”). On December 19, 2022, the Financing Agreement was converted to a notes purchase agreement and guaranty (the “Notes Purchase Agreement”) between the same parties and under substantially the same terms and conditions as the Financing Agreement, subject to certain customary note constitution terms. Perceptive Advisors, LLC, an affiliate of Perceptive, is a 18.0% holder of the ordinary shares of the Company. Additionally, Ellen Hukkelhoven, Ph.D., a director of the Company, is an employee of Perceptive Advisors, LLC. Refer to the discussion in Note 13 for further information related to the accounting for the debt financing. May 2023 Private Placement On May 3, 2023, the Company issued 10,773,913 ordinary shares in a private placement for gross proceeds of $62.0 million, excluding offering costs of approximately $4.1 million. Perceptive Advisors, LLC and Adage Capital Partners, L.P. a greater than 5% holder of the ordinary shares of the Company, purchased 4,347,826 and 1,565,217 of the ordinary shares, respectively, issued on the same terms and conditions as the other investors in the offering. |
Leases
Leases | 12 Months Ended |
Dec. 31, 2023 | |
Leases | |
Leases | 12. Leases The Company has commitments under operating leases for laboratory, warehouse, clinical trial sites and office space. The Company also has finance leases for manufacturing space and office equipment. The Company’s leases have initial lease terms ranging from 3 years to 191 years. Certain lease agreements contain provisions for future rent increases. Payments due under the lease contracts include fixed payments. Total rent expense recorded under these leases was $5.5 million and $5.3 million for the years ended December 31, 2023 and 2022, respectively. On August 4, 2020, Meira Ireland entered into two agreements with Shannon Commercial Enterprises DAC trading as Shannon Commercial Properties, to acquire two properties in the Shannon Free Zone in Shannon, Ireland for an aggregate price of €18 million, or approximately $21.2 million. These properties were acquired to serve as the Company’s second, large scale GMP viral vector manufacturing facility and its first GMP plasmid and DNA production facility. The closing for the first building occurred in August 2020 and the closing for the second building occurred in January 2021. The total cost of the first and second buildings, including taxes and legal fees, was €11.9 million and €7.5 million, or approximately $13.8 million and $8.9 million, respectively, and have been recorded as right of use assets in the consolidated balance sheets. There is no corresponding lease liability as the Company paid the full cost on the date of the closings. At the closings, Meira Ireland entered into a lease for each property providing for a long leasehold interest of approximately 191 years. The leases also include customary terms and conditions, with a nominal annual lease cost and annual maintenance fees of approximately €0.3 million, or approximately $0.4 million, in the aggregate, which amount is subject to change depending on the annual maintenance costs within the Shannon Free Zone development. During the year ended December 31, 2022, the Company recognized three operating leases for locations in connection with its clinical trials for its IRD product candidates and office and warehouse space, with initial lease terms between 3 years and 9 years. Payments due under the lease contracts include fixed payments. In conjunction with these operating leases, the Company recognized initial operating lease right-of-use assets in the amount of $1.8 million and corresponding lease liabilities in the amount of $1.8 million which are included in the right-of-use assets and lease obligations in the consolidated balance sheets as of December 31, 2022. The components of lease cost for the years ended December 31, 2023 and 2022 are as follows (in thousands): Years Ended December 31, 2023 2022 Finance lease cost Amortization of right-of-use assets $ 1,124 $ 1,103 Interest on lease liabilities — 1 Total finance lease cost 1,124 1,104 Operating lease cost 5,473 5,307 Short-term lease cost 159 154 Total lease cost $ 6,756 $ 6,565 Amounts reported in the consolidated balance sheets for leases where the Company is the lessee as of December 31, 2023 and 2022 were as follows (in thousands): December 31, December 31, 2023 2022 Operating leases Right-of-use asset $ 15,910 $ 20,109 Capitalized lease obligations $ 17,145 $ 21,215 Finance leases Right-of-use asset $ 24,432 $ 24,718 Capitalized lease obligations $ — $ — Weighted-average remaining lease term Operating leases 4.3 years 5.6 years Finance leases 174.8 years 175.8 years Weighted-average discount rate Operating leases 8.8 % 8.8 % Finance leases 8.0 % 8.0 % Other information related to leases as of the years ended December 31, 2023 and 2022 are as follows (in thousands): Years Ended December 31, 2023 2022 Cash paid for amounts included in the measurement of lease liabilities Operating cash flows from finance leases $ — $ 52 Operating cash flows from operating leases $ 5,662 $ 5,384 Financing cash flows from finance leases $ — $ — Right-of-use assets obtained in exchange for lease liabilities Operating leases $ — $ 1,793 Future minimum lease payments under non-cancellable leases as of December 31, 2023 are as follows (in thousands): Operating Leases 2024 $ 5,565 2025 5,524 2026 5,623 2027 1,626 2028 1,335 Thereafter 706 Total undiscounted lease payments $ 20,379 Less: Imputed interest (3,234) Total lease liabilities $ 17,145 |
Debt Financing
Debt Financing | 12 Months Ended |
Dec. 31, 2023 | |
Debt Financing | |
Debt Financing | 13. Debt Financing On August 2, 2022 the Company, and the Subsidiary Guarantors, entered into the Financing Agreement with Perceptive. On December 19, 2022, the Financing Agreement was converted to a Notes Purchase Agreement between the same parties and under substantially the same terms and conditions as the Financing Agreement, subject to certain customary note constitution terms. The Company and the Subsidiary Guarantors entered into a Consent and Amendment with Perceptive on August 10, 2023 (the “First Consent and Amendment), and the Company and the Subsidiary Guarantors entered into a second Consent and Amendment with Perceptive on December 20, 2023. The Notes Purchase Agreement provides for an initial $75.0 million notes issuance (the “Tranche 1 Notes”). Pursuant to the First Consent and Amendment, the Company may request in its sole discretion, and Perceptive has agreed to subscribe to purchase upon such request, an additional $25.0 million notes issuance (the “Tranche 2 Notes”) at any time before August 2, 2024 subject to the terms of the Notes Purchase Agreement. Previously, the Company’s request for issuance of the Tranche 2 Notes was to be determined at Perceptive’s sole discretion. The Notes Purchase Agreement matures on August 2, 2026 and is interest-only during the term. The Company has the option to redeem outstanding principal notes at any time along with an applicable early redemption fee. Under each of the First Consent and Amendment and the Second Consent and Amendment, the Notes Purchase Agreement was amended to increase the applicable early redemption fee. Outstanding amounts under the Notes Purchase Agreement bear interest at a fluctuating rate per annum equal to 10.00% plus the secured overnight financing rate administered by the Federal Reserve Bank of New York for a one-month tenor, subject to a 1.00% floor. The annual interest rate was 15.32% at December 31, 2023. As of December 31, 2023, the outstanding balance of the Tranche 1 Notes was $75.0 million plus accrued interest of $3.0 million. During the years ended December 31, 2023 and 2022, the Company recorded interest expense of $11.3 million and $4.0 million, respectively. The Company’s obligations under the Notes Purchase Agreement are secured by the Company’s London, UK and Shannon, Ireland manufacturing facilities, $3.0 million of the Company’s cash and the bank accounts of the Subsidiary Guarantors, and the issued and outstanding equity interests of the Subsidiary Guarantors. The Notes Purchase Agreement imposes certain covenants and restrictions on the Company and the Subsidiary Guarantors, including restrictions pertaining to: (i) the incurrence of additional indebtedness, (ii) limitations on liens, (iii) limitations on certain investments, (iv) making distributions, dividends and other payments, (v) mergers, consolidations and acquisitions, (vi) dispositions of assets, (vii) the Company’s maintenance of at least $3.0 million in a U.S. bank account, (viii) transactions with affiliates, (ix) changes to governing documents, (x) changes to certain agreements and leases and (xi) changes in control; however, certain of these restrictions contain exceptions which allow the Company to license, sell and monetize assets in its AAV-hAQP1 program in development to treat radiation-induced xerostomia, its AAV-GAD program in development to treat Parkinson’s disease and its gene regulation platform technologies. In connection with entering into the Financing Agreement, the Company granted warrants to Perceptive to purchase up to (i) 400,000 ordinary shares of the Company at an exercise price of $15.00 per share and (ii) 300,000 ordinary shares of the Company at an exercise price of $20.00 per share. The warrants are exercisable immediately and expire on August 2, 2027. The Company recorded a debt discount of $2.3 million for the allocated fair value of the warrants. The Company also capitalized certain lender and legal costs associated with the Notes Purchase Agreement totaling $2.1 million, which were recorded as a discount to the loan. The aggregate discount of $4.4 million is being amortized to interest expense over the term of the Notes Purchase Agreement. The Company amortized $1.1 million and $0.4 million of the discount to interest expense during the years ended December 31, 2023 and 2022, respectively. At December 31, 2023, the remaining unamortized discount was $2.9 million. |
Commitments and Contingencies
Commitments and Contingencies | 12 Months Ended |
Dec. 31, 2023 | |
Commitments and Contingencies | |
Commitments and Contingencies | 14. Commitments and Contingencies There were no new material commitments or contingencies entered into during the year ended December 31, 2023. |
Employee Benefit Plans
Employee Benefit Plans | 12 Months Ended |
Dec. 31, 2023 | |
Employee Benefit Plans | |
Employee Benefit Plans | 15. Employee Benefit Plans United States On January 1, 2017, Meira LLC adopted a defined contribution retirement plan that complies with Section 401(k) of the Internal Revenue Code. All Meira LLC employees over the age of 21 are eligible to participate in the plan after three consecutive months of service. Employees are able to defer a portion of their pay into the plan on the first day of the month or after the day all age and service requirements have been met. The plan provides for a Company matching contribution. All eligible employees receive an employer matching contribution equal to the lesser of the amount the employee contributes to the plan or 6% of their salary up to the annual IRS limit. United Kingdom On August 1, 2016, Meira UK II adopted a defined contribution group personal pension plan that complies with HMRC for tax relief. All Meira UK II employees are eligible to participate in the plan upon joining the company and providing the required services. All eligible employees, if they elect to join the pension scheme, receive an employer pension contribution equal to 7.5% to 10.0% of their pensionable earnings. Currently, employees are required to contribute 0.5%, to meet minimum legal pension funding levels of 8%, but may make optional contributions up to the annual allowance HMRC limits. Netherlands Meira Netherlands operates a defined contribution pension. All of its employees participate in the plan. All eligible employees receive an employer pension contribution and are also required to contribute. Ireland On November 20, 2020, MeiraGTx Ireland adopted a defined contribution pension plan. All MeiraGTx Ireland employees are eligible to participate in the plan upon joining the Company. Belgium Meira Belgium operates a defined contribution pension plan. All eligible employees receive an employer pension contribution of 8% of their annual salary. Employees do not make contributions to the plan. During the years ended December 31, 2023 and 2022, employer contributions to all plans were $2.5 million and $2.0 million, respectively. |
Subsequent Event
Subsequent Event | 12 Months Ended |
Dec. 31, 2023 | |
Subsequent Event | |
Subsequent Event | 16. Subsequent Event In the first quarter of 2024, the Company received a milestone payment of $50.0 million in connection with the Asset Purchase Agreement with Janssen for the achievement of the initiation of the extension study for the Phase 3 LUMEOS clinical trial for the RPGR Product. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 12 Months Ended |
Dec. 31, 2023 | |
Summary of Significant Accounting Policies | |
Consolidation | Consolidation The accompanying consolidated financial statements include the accounts of Meira Holdings and its wholly owned subsidiaries: MeiraGTx Limited, a limited company incorporated under the laws of England and Wales; MeiraGTx, LLC, a Delaware limited liability company (“Meira LLC”); MeiraGTx UK II Limited, a limited company incorporated under the laws of England and Wales (“Meira UK II”); MeiraGTx Ireland DAC, a designated activity company incorporated under the laws of Ireland (“Meira Ireland”); MeiraGTx Netherlands B.V., a private company with limited liability incorporated under the laws of the Netherlands (“Meira Netherlands”); MeiraGTx Belgium, a private company with limited liability incorporated under the laws of Belgium (“Meira Belgium”); BRI-Alzan, Inc., a Delaware corporation (“BRI-Alzan”); MeiraGTx Bio Inc., a Delaware corporation (“Meira Bio”); MeiraGTx B.V., a private company with limited liability incorporated under the laws of the Netherlands (“Meira B.V.”); MeiraGTx Neurosciences, Inc., a Delaware corporation (“Meira Neuro”); MeiraGTx Therapeutics, Inc., a Delaware corporation (“Meira Therapeutics”); and MeiraGTx UK Limited, a limited company incorporated under the laws of England and Wales (“Meira UK”). All intercompany balances and transactions between the consolidated companies have been eliminated in consolidation. |
Use of Estimates | Use of Estimates Management considers many factors in selecting appropriate financial accounting policies and controls, and in developing the estimates and assumptions that are used in the preparation of these consolidated financial statements. Management must apply significant judgment in this process. In addition, other factors may affect estimates, including expected business and operational changes, sensitivity and volatility associated with the assumptions used in developing estimates, and whether historical trends are expected to be representative of future trends. The estimation process often may yield a range of potentially reasonable estimates of the ultimate future outcomes and management must select an amount that falls within that range of reasonable estimates. This process may result in actual results differing materially from those estimated amounts used in the preparation of the financial statements if these results differ from historical experience, or other assumptions do not turn out to be substantially accurate, even if such assumptions are reasonable when made. In preparing these consolidated financial statements, management used significant estimates in the following areas, among others: collaboration revenue, fair value of nonfinancial assets, stand-alone selling price and material rights in connection with the Asset Purchase and Commercial Supply Agreements, the accounting for research and development costs, share-based compensation, leases, asset retirement obligations, fair value of financial instruments and tax incentive receivable. |
Cash and Cash Equivalents | Cash and Cash Equivalents The Company considers all highly liquid instruments with an original maturity of 90 days or less at the time of purchase to be cash equivalents. Cash and cash equivalents consist of checking and money market accounts held at large international banks that are readily convertible into cash. |
Restricted Cash | Restricted Cash Restricted cash represents a guarantee put in place as required by the terms of the research and innovation grant from IDA Ireland which offers financial assistance in establishing the Company’s operations in Shannon, Ireland. The following table provides a reconciliation of the components of cash and cash equivalents and restricted cash reported in the Company’s consolidated balance sheets to the total of the amount presented in the consolidated statements of cash flows (in thousands): December 31, December 31, 2023 2022 Cash and cash equivalents $ 129,566 $ 115,516 Restricted cash 1,083 — Total cash, cash equivalents and restricted cash in the condensed consolidated statement of cash flows $ 130,649 $ 115,516 |
Financial Instruments | Financial Instruments The carrying value of accounts receivable-related party, tax incentive receivable, other current assets, and accounts payable reported in the consolidated balance sheets equal or approximate fair value due to their short maturities. |
Tax Incentive Receivable | Tax Incentive Receivable Meira UK II is eligible to participate in a UK research and development tax incentive programs under which it is eligible to receive a cash refund from His Majesty’s Revenue & Customs (“HMRC”) for a percentage of the qualified research and development costs expended by Meira UK II under the small and medium sized enterprises (“SME”) program and the research and development expenditures credit (“RDEC”) program. The SME cash refund is available to companies with less than 500 employees and annual aggregate revenue of less than 100.0 million euros or total aggregate assets less than 86.0 million euros during the reimbursable period. The Company’s estimate of the amount of cash refund it expects to receive related to the SME and RDEC programs is included in tax incentive receivable in the accompanying consolidated balance sheets and such amounts are recorded as a reduction of research and development expense in the statements of operations. During the years ended December 31, 2023 and 2022, the Company recorded reductions to research and development expenses of $2.9 million and $6.8 million, respectively. In addition, the Company incurs Value Added Tax (“VAT”) on services provided by UK and EU vendors, which it is entitled to reclaim. The Company’s estimate of the amount of cash refund it expects to receive related to VAT was $0.6 million and $1.1 million as of December 31, 2023 and 2022, respectively, which is included in other current assets in the accompanying consolidated balance sheets. |
Fair Value Measurements | Fair Value Measurements Fair value is defined as the price that would be received upon sale of an asset or paid upon transfer of a liability in an orderly transaction between market participants at the measurement date and in the principal or most advantageous market for that asset or liability. The fair value should be calculated based on assumptions that market participants would use in pricing the asset or liability, not on assumptions specific to the entity. In addition, the fair value of liabilities should include consideration of non-performance risk including the Company’s own credit risk. The Company follows ASC Topic 820, Fair Value Measurements and Disclosures three levels based on the extent to which inputs used in measuring fair value are observable in the market. Each fair value measurement is reported in one of the three levels which are determined by the lowest level input that is significant to the fair value measurement in its entirety. These levels are: ● Level 1: Observable inputs such as quoted prices in active markets for identical assets the reporting entity has the ability to access as of the measurement date; ● Level 2: Inputs, other than the quoted prices in active markets, that are observable either directly or indirectly; and ● Level 3: Unobservable inputs in which there is little or no market data, which require the reporting entity to develop its own assumptions. The table below represents the values of the Company’s financial assets and liabilities that are required to be measured at fair value on a recurring basis (in thousands): Fair Value Measurement Using: Significant Significant Other Significant December 31, Observable Inputs Observable Inputs Unobservable Description 2023 (Level 1) (Level 2) (Level 3) Cash equivalents $ 46,868 $ 46,868 $ — $ — Restricted cash $ 1,083 $ 1,083 $ — $ — Fair Value Measurement Using: Significant Significant Other Significant December 31, Observable Inputs Observable Inputs Unobservable Description 2022 (Level 1) (Level 2) (Level 3) Cash equivalents $ 57,336 $ 57,336 $ — $ — Other long-term liabilities $ 262 $ 262 $ — $ — |
Equity Method and Other Investments | Equity Method and Other Investments The Company accounts for equity investments under the equity method of accounting when the requirements for consolidation are not met, and the Company has significant influence over the operations of the investee. Equity method investments are initially recorded at cost and subsequently adjusted for the Company’s share of net income or loss and cash contributions and distributions and are included in equity method and other investments in the accompanying consolidated balance sheets. Equity investments that do not result in consolidation and are not accounted for under the equity method are measured at fair value, with any changes in fair value recognized in net income (loss). For any such investments that do not have readily determinable fair values, the Company elects the measurement alternative to measure the investments at cost minus impairment, if any, plus or minus changes resulting from observable price changes in orderly transactions for the identical or a similar investment of the same issuer. Equity method investments are reviewed for impairment whenever events or changes in circumstances indicate that the carrying amount may not be recoverable. If it is determined that a loss in value of the equity method investment is other than temporary, an impairment loss is measured based on the excess of the carrying amount of an investment over its estimated fair value. Impairment analyses are based on current plans, intended holding periods, and available information at the time the analysis is prepared. |
Concentrations of Credit Risk | Concentrations of Credit Risk The Company maintains its cash and cash equivalents primarily in depository and money market accounts within two large financial institutions in the United States and one large financial institution in the United Kingdom and Ireland. Cash balances deposited at these major financial banking institutions exceed the insured limit. The Company has not experienced any losses on its bank deposits and believes these deposits do not expose the Company to any significant credit risk. |
Intangible Assets | Intangible Assets Intangible assets consist of purchased rights to licensed technology as it relates to the Company’s manufacturing processes and has future alternative use in the Company’s operations. The licensed technology is being amortized on a straight-line basis over |
Property, Plant and Equipment, Net | Property, Plant and Equipment, Net Property, plant and equipment are stated at cost, net of accumulated depreciation. Depreciation is calculated using the straight-line method over the estimated useful lives of the respective assets. Leasehold improvements are depreciated over the lesser of their useful lives or the term of the lease (see Note 5). The estimated useful lives of the asset categories are as follows: Asset Category Useful Lives Computer and office equipment 3 years Laboratory equipment 5 years Manufacturing equipment 7 years Furniture and fixtures 5 years Leasehold improvements lesser of useful life or remaining term of lease Expenditures for leasehold improvements are capitalized, and expenditures for maintenance and repairs are expensed to operations as incurred. ASC Topic 360, Property, Plant and Equipment |
Leases | Leases The Company accounts for leases in accordance with ASC 842. The Company determines if an arrangement is a lease at contract inception. A lease exists when a contract conveys the right to control the use of identified property, plant, or equipment for a period of time in exchange for consideration. The definition of a lease embodies two conditions: (1) there is an identified asset in the contract that is land or a depreciable asset (i.e., property, plant, and equipment), and (2) the Company has the right to control the use of the identified asset. The Company accounts for the lease and non-lease components as a single lease component. From time to time the Company enters into direct financing lease arrangements that include a lessee obligation to purchase the leased asset at the end of the lease term, a bargain purchase option, or provides for minimum lease payments with a present value of 90% or more of the fair value of the leased asset at the date of lease inception. Operating leases where the Company is the lessee are included in right-of-use (“ROU”) assets – operating leases and lease obligations are included on the Company’s consolidated balance sheets. The lease obligations are initially and subsequently measured at the present value of the unpaid lease payments at the lease commencement date and subsequent reporting periods. Finance leases where the Company is the lessee are included in ROU assets – finance leases, net and lease obligations on the Company’s consolidated balance sheets. The lease obligations are initially measured in the same manner as for operating leases and are subsequently measured at amortized cost using the effective interest method. Key estimates and judgments include how the Company determined (1) the discount rate used to discount the unpaid lease payments to present value, (2) lease term and (3) lease payments. ASC 842 requires a lessee to discount its unpaid lease payments using the interest rate implicit in the lease or, if that rate cannot be readily determined, its incremental borrowing rate. As most of the Company’s leases where it is the lessee do not provide an implicit rate, the Company uses its incremental borrowing rate based on the information available at commencement date in determining the present value of lease payments. The Company’s incremental borrowing rate for a lease is the rate of interest it would have to pay on a collateralized basis to borrow an amount equal to the lease payments under similar terms. The Company uses the implicit rate when readily determinable. The lease term for all of the Company’s leases includes the non-cancellable period of the lease plus any additional periods covered by either a lessee option to extend (or not to terminate) the lease that is reasonably certain to be exercised, or an option to extend (or not to terminate) the lease controlled by the lessor. The ROU asset is initially measured at cost, which comprises the initial amount of the lease liability adjusted for lease payments made at or before the lease commencement date less any lease incentives received. For operating leases, the ROU asset is subsequently measured throughout the lease term at the carrying amount of the lease liability, minus any accrued lease payments, less the unamortized balance of lease incentives received. Lease expense for lease payments is recognized on a straight-line basis over the lease term. For finance leases, the ROU asset is subsequently amortized using the straight-line method from the lease commencement date to the earlier of the end of its useful life or the end of the lease term unless the lease transfers ownership of the underlying asset, or the Company is reasonably certain to exercise an option to purchase the underlying asset. In those cases, the ROU asset is amortized over the useful life of the underlying asset. Amortization of the ROU asset is recognized and presented separately from interest expense on the lease liability. The Company has elected not to recognize ROU assets and lease liabilities for all short-term leases that have a lease term of 12 months or less at lease commencement. Lease payments associated with short-term leases are recognized as an expense on a straight-line basis over the lease term. |
Asset Retirement Obligations | Asset Retirement Obligations Accounting for asset retirement obligations requires legal obligations associated with the retirement of long-lived assets to be recognized at fair value when incurred and capitalized as part of the related long-lived asset. In the absence of quoted market prices, the Company estimates the fair value of its asset retirement obligations using Level 3 present value techniques, in which estimates of future cash flows associated with retirement activities are discounted using a credit-adjusted risk-free rate. Asset retirement obligations currently reported on the Company’s consolidated balance sheets were measured during a period of historically low interest rates. The impact on measurements of new asset retirement obligations using different rates in the future may be significant. The Company uses estimates to determine the asset retirement obligations at the end of the lease term and discounts such asset retirement obligations using an estimated discount rate. Interest on the discounted asset retirement obligation is amortized over the term of the lease using the effective interest method and is recorded as interest expense in the consolidated statements of operations and comprehensive loss. The change in asset retirement obligations is as follows (in thousands): For the Years Ended December 31, 2023 2022 Balance at beginning of period $ 2,179 $ 2,081 Additional asset retirement obligations incurred during the period — 9 Amortization of interest 177 168 Effects of exchange rate changes 45 (79) Balance at end of period $ 2,401 $ 2,179 |
IDA Ireland Grant | IDA Ireland Grant In August 2021, Meira Ireland entered into an agreement pursuant to which it received a grant from IDA Ireland for financial assistance in establishing its operations in Shannon, Ireland. Under the terms of the grant, Meira Ireland is eligible to receive the lesser of €1.0 million or €10,000 for each job created (the “employment grant”) and the lesser of €1.2 million or 4% of the actual expenditure on the provision of machinery and equipment (the “capital grant”). Meira Ireland may apply for a drawdown of the employment grant once a job has been created and the position has been held for a period of at least one month, and may apply for a drawdown of the capital grant once an eligible asset has been purchased and installed, conditioned on the creation of a cumulative number of jobs by the end of the immediately preceding year. An aggregate of 100 jobs must be created to receive the maximum benefit under the capital grant. An application for a drawdown must be accompanied by an audit certification for compliance with the terms of the grant. The Company has a guarantee in place with a bank in favor of IDA Ireland, pursuant to which it restricts cash in the amount of claims made under the grant such that the Company maintains the funds to cover any portion of the grant income that may become repayable in the future. This amount is presented as restricted cash in the accompanying consolidated balance sheet. All expenditures must be completed by December 31, 2024, and the agreement terminates on the later of five years from the date of the last payment from the grant or five years from completion of the capital investment, which is expenditure of at least €30.0 million on eligible machinery and equipment. The Company recognizes grant income when there is reasonable assurance that the Company will comply with the conditions attached to the grant and that it will receive the grant. Grant income from the employment grant is recognized as a deduction from the amount of the related expense, and grant income from the capital grant is deducted from the carrying amount of the related asset and recognized in income over the asset’s useful life in the form of a reduced depreciation charge. The Company received its first drawdown under the grant in 2023, which was comprised of During the six |
Share-Based Compensation Expense | Share-Based Compensation Expense Options The Company grants share options to employees, non-employee members of the Company’s board of directors and non-employee consultants as compensation for services performed. Employee and non-employee members of the board of directors’ awards of share-based compensation are accounted for in accordance with ASC 718, Compensation Stock Compensation, . Using this model, fair value is calculated based on assumptions with respect to (i) the fair value of the Company’s ordinary shares on the grant date; (ii) expected volatility of the Company’s ordinary share price, (iii) the periods of time over which the optionees are expected to hold their options prior to exercise (expected term), (iv) expected dividend yield on the Company’s ordinary shares, and (v) risk-free interest rates. The assumptions underlying these valuations represented management’s best estimate, which involved inherent uncertainties and the application of management’s judgment. As a result, if the Company had used different assumptions or estimates, the fair value of its ordinary shares and its share-based compensation expense could have been materially different. The expected term of share options granted to the optionees is determined using the average of the vesting period and contractual life of the option, an accepted method for the Company’s option grants under the Securities and Exchange Commission’s (“SEC”) Staff Accounting Bulletin No. 107 and No. 110, Share-Based Payment. The Company believes that its future volatility could differ materially during the expected term from the volatility that would be calculated from its historical share prices to date. Consequently, expected volatility is based on an analysis of guideline companies and the Company’s own volatility in accordance with ASC 718. The expected dividend yield is zero as the Company has never paid dividends and does not currently anticipate paying any in the foreseeable future. Risk-free interest rates are based on quoted U.S. Treasury rates for securities with maturities approximating the option’s expected term. |
Restricted Share Units | Restricted Share Units The Company grants restricted share units (“RSUs”) to employees, non-employee members of the Company’s board of directors and non-employee consultants as compensation for services performed. Awards of RSUs are accounted for in accordance with ASC 718, Compensation Stock Compensation, . |
Collaboration Arrangements | Collaboration Arrangements The Company evaluates its collaborative arrangements pursuant to ASC 808, Collaborative Arrangements Revenue from Contracts with Customers with respect to the arrangement, the Company accounts for the arrangement as a collaboration under ASC 808. To date, the Company has entered into two separate collaboration agreements, both of which are with Janssen, which were determined to be within the scope of ASC 808. ASC 808 does not address recognition or measurement matters related to collaborative arrangements. Payments between participants pursuant to a collaborative arrangement that are within the scope of other authoritative accounting literature on income statement classification are accounted for using the relevant provisions of that literature. If the Company concludes that some or all aspects of the arrangement are within the scope of ASC 808 and do not represent a transaction with a customer, the Company recognizes its allocation of the shared costs incurred with respect to the jointly conducted activities pursuant to ASC 730, Research and Development Refer to the discussion in Note 11 for further information related to the accounting for the Collaboration Agreement. |
Revenue Recognition | Revenue Recognition The Company evaluates the promised goods or services to determine which promises, or group of promises, represent performance obligations in contracts with the Company’s. In contemplation of whether a promised good or service meets the criteria required of a performance obligation, the Company considers the stage of development of the underlying intellectual property, the capabilities and expertise of the customer relative to the underlying intellectual property, and whether the promised goods or services are integral to or dependent on other promises in the contract. When accounting for an arrangement that contains multiple performance obligations, the Company must develop judgmental assumptions, which may include market conditions, reimbursement rates for personnel costs, development timelines and probabilities of regulatory success to determine the stand-alone selling price for each performance obligation identified in the contract. When the Company concludes that a contract should be accounted for as a combined performance obligation and recognized over time, the Company must then determine the period over which revenue should be recognized and the method by which to measure revenue. The Company generally recognizes revenue using a cost-based input method. At inception, the Company determines whether contracts are within the scope of ASC 606 or other topics. For contracts that are determined to be within the scope of ASC 606, the Company recognizes revenue when its customer or collaborator obtains control of promised goods or services, in an amount that reflects the consideration which the Company expects to receive in exchange for those goods or services. To determine revenue recognition the Company performs the following five steps: i. identify the contract(s) with a customer; ii. identify the performance obligations in the contract; iii. determine the transaction price; iv. allocate the transaction price to the performance obligations within the contract; and v. recognize revenue when (or as) the entity satisfies a performance obligation. The Company only applies the five-step model to contracts when it determines that it is probable it will collect the consideration it is entitled to in exchange for the goods or services it transfers to the customer. At contract inception, the Company assesses the goods or services promised within the contract to determine whether each promised good or service is a performance obligation. The promised goods or services in the Company’s arrangements typically consist of a license to the Company’s intellectual property and research, development and manufacturing services. The Company may provide options to additional items in such arrangements, which are accounted for as separate contracts when the customer elects to exercise such options, unless the option provides a material right to the customer. Performance obligations are promises in a contract to transfer a distinct good or service to the customer that (i) the customer can benefit from on its own or together with other readily available resources, and (ii) is separately identifiable from other promises in the contract. Goods or services that are not individually distinct performance obligations are combined with other promised goods or services until such combined group of promises meet the requirements of a performance obligation. The Company determines transaction price based on the amount of consideration the Company expects to receive for transferring the promised goods or services in the contract. Consideration may be fixed, variable, or a combination of both. At contract inception for arrangements that include variable consideration, the Company estimates the probability and extent of consideration it expects to receive under the contract utilizing either the most likely amount method or expected amount method, whichever best estimates the amount expected to be received. The Company then considers any constraints on the variable consideration and includes in the transaction price variable consideration to the extent it is deemed probable that a significant reversal in the amount of cumulative revenue recognized will not occur when the uncertainty associated with the variable consideration is subsequently resolved. The Company then allocates the transaction price to each performance obligation based on the relative standalone selling price and recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) control is transferred to the customer and the performance obligation is satisfied. For performance obligations which consist of licenses and other promises, the Company utilizes judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time and, if over time, the appropriate method of measuring progress. The Company evaluates the measure of progress each reporting period and, if necessary, adjusts the measure of performance and related revenue recognition. If there are multiple performance obligations, the Company allocates the transaction price to each performance obligation based on their estimated standalone selling prices (“SSP”). The Company estimates the SSP for each performance obligation by considering information such as market conditions, entity-specific factors, and information about its customer that is reasonably available. The Company considers estimation approaches that allow it to maximize the use of observable inputs. These estimation approaches may include the adjusted market assessment approach, the expected cost plus a margin approach or the residual approach. The Company also considers whether to use a different estimation approach or a combination of approaches to estimate the SSP for each performance obligation. Developing certain assumptions (e.g., treatable patient population, expected market share, probability of success and product profitability, and discount rate based on weighted-average cost of capital) to estimate the SSP of a performance obligation requires significant judgment. The Company records amounts as accounts receivable when the right to consideration is deemed unconditional. When consideration is received, or such consideration is unconditionally due, from a customer prior to transferring goods or services to the customer under the terms of a contract, a contract liability is recorded as deferred revenue. Amounts received prior to satisfying the revenue recognition criteria are recognized as deferred revenue in the Company’s consolidated balance sheet. Amounts expected to be recognized as revenue within the 12 months following the balance sheet date are classified as deferred revenue – related party, current. Amounts not expected to be recognized as revenue within the 12 months following the balance sheet date are classified as deferred revenue – related party. The Company’s collaboration and revenue arrangements include the following: Up-front License Fees: If a license is determined to be distinct from the other performance obligations identified in the arrangement, the Company recognizes revenues from nonrefundable, up-front fees allocated to the license when the license is transferred to the licensee and the licensee is able to use and benefit from the license. For licenses that are bundled with other promises, the Company utilizes judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time and, if over time, the appropriate method of measuring progress for purposes of recognizing revenue from non-refundable, up-front fees. The Company evaluates the measure of progress each reporting period and, if necessary, adjusts the measure of performance and related revenue recognition. Milestone Payments: At the inception of an agreement that includes research and development milestone payments, the Company evaluates each milestone to determine when and how much of the milestone to include in the transaction price. The Company first estimates the amount of the milestone payment that the Company could receive using either the expected value or the most likely amount approach. The Company primarily uses the most likely amount approach as that approach is generally most predictive for milestone payments with a binary outcome. Then, the Company considers whether any portion of that estimated amount is subject to the variable consideration constraint (that is, whether it is probable that a significant reversal of cumulative revenue would not occur upon resolution of the uncertainty.) The Company updates the estimate of variable consideration included in the transaction price at each reporting date which includes updating the assessment of the likely amount of consideration and the application of the constraint to reflect current facts and circumstances. Royalties: For arrangements that include sales-based royalties, including milestone payments based on a level of sales, and the license is deemed to be the predominant item to which the royalties relate, the Company will recognize revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied). To date, the Company has not recognized any revenue related to sales-based royalties or milestone payments based on the level of sales. Research and Development Services: The Company is incurring research and development costs, with Janssen responsible for up to 100% of the costs, depending on the type of research and development services being performed. The Company records costs associated with the development activities as research and development expenses in the consolidated statement of operations and comprehensive loss consistent with ASC 730, Research and Development Manufacturing Supply Services: Arrangements that include a promise for future supply of drug substance or drug product for either clinical development or commercial supply at the customer’s discretion are generally considered options. The Company assesses if these options provide a material right to the licensee and if so, they are accounted for as separate performance obligations at the outset of the arrangement. Customer Options: Customer options are evaluated at contract inception to determine whether those options provide a material right (i.e., an optional good or service offered for free or at a discount) to the customer. If the customer options represent a material right, the material right is treated as a separate performance obligation at the outset of the arrangement. The Company allocates the transaction price to material rights based on the standalone selling price. As a practical alternative to estimating the standalone selling price of a material right when the underlying goods or services are both (i) similar to the original goods or services in the contract and (ii) provided in accordance with the terms of the original contract, the Company allocates the total amount of consideration expected to be received from the customer to the total goods or services expected to be provided to the customer. Amounts allocated to any material right are recognized as revenue when or as the related future goods or services are transferred or when the option expires. |
Research and Development | Research and Development Research and development costs are charged to expense as incurred. These costs include, but are not limited to, employee-related expenses, including salaries, benefits and travel of the Company’s research and development personnel; expenses incurred under agreements with contract research organizations and investigative sites that conduct clinical and preclinical studies and for the drug product for the clinical studies and preclinical activities; facilities; supplies; rent, insurance, certain legal fees, share-based compensation, depreciation, other costs associated with clinical and preclinical activities and regulatory operations and acquisition of in process research and development write-offs. Research funding under collaboration agreements and refundable research and development credits / tax credits are recorded as an offset to these costs. Costs for certain development activities, such as Company funded outside research programs, are recognized based on an evaluation of the progress to completion of specific tasks with respect to their actual costs incurred. Payments for these activities are based on the terms of the individual arrangements, which may differ from the pattern of costs incurred, and are reflected in the consolidated financial statements as prepaid or accrued research and development expenses, as the case may be. |
Foreign Currencies | Foreign Currencies The Company’s consolidated financial statements are presented in U.S. dollars, the reporting currency of the Company. The financial position and results of operations of Meira UK II, Meira Ireland, Meira Netherlands, Meira Belgium and Meira B.V. are measured using the foreign subsidiaries’ local currency as the functional currency. These entities’ cash accounts holding U.S. dollars and intercompany payables and receivables are remeasured based upon the exchange rate at the date of remeasurement with the resulting gain or loss included in the consolidated statements of operations and comprehensive loss. Expenses of such subsidiaries have been translated into U.S. dollars at average exchange rates prevailing during the period. Assets and liabilities have been translated at the rates of exchange on the consolidated balance sheet dates. The resulting translation gain and loss adjustments are recorded directly as a separate component of shareholders’ equity and as other comprehensive (loss) income on the consolidated statements of operations and comprehensive loss. |
Income Taxes | Income Taxes Income taxes are recorded in accordance with ASC Topic 740, Income Taxes The Company accounts for uncertain tax positions in accordance with the provisions of ASC 740. When uncertain tax positions exist, the Company recognizes the tax benefit of tax positions to the extent that the benefit will more likely than not be realized. The determination as to whether the tax benefit will more likely than not be realized is based upon the technical merits of the tax position as well as consideration of the available facts and circumstances. As of December 31, 2023 and 2022, the Company recorded unrecognized tax positions of $2.0 and $0.9 million, respectively. The Company is required to estimate income taxes in each of the jurisdictions in which it operates. |
Net Loss per Ordinary Share | Net Loss per Ordinary Share Basic net loss per ordinary share is computed by dividing net loss by the weighted average number of shares of the Company’s ordinary shares outstanding during the period of computation. Diluted net loss per ordinary share is computed similar to basic net loss per share except that the denominator is increased to include the number of additional ordinary shares that would have been outstanding if the ordinary share equivalents had been issued at the beginning of the year and if the additional ordinary shares were dilutive (treasury stock method) or the two-class method, whichever is more dilutive. For all periods presented, basic and diluted net loss per ordinary share are the same as any additional ordinary share equivalents would be anti-dilutive. The following securities are considered to be ordinary share equivalents, but were not included in the computation of diluted net loss per ordinary share because to do so would have been anti-dilutive: December 31, December 31, 2023 2022 Share options 8,226,707 6,858,409 Restricted share units 2,661,250 2,182,500 Deferred share units 185,000 110,000 Warrants 700,000 700,000 Restricted ordinary shares subject to forfeiture — 14,049 11,772,957 9,864,958 |
Other Comprehensive Income (Loss) | Other Comprehensive Income (Loss) Other comprehensive income (loss) is defined as the change in equity of a business enterprise during a period from transactions and other events and circumstances from non-owner sources. The only component of other comprehensive income (loss) impacting the Company is foreign currency translation. |
Segment Information | Segment Information Management has concluded it has a single reporting segment for purposes of reporting financial condition and results of operations. The Company’s license revenue, research funding and deferred revenue from the Collaboration Agreement were generated in the United Kingdom. The following table summarizes long-lived assets by geographical area (in thousands): December 31, December 31, 2023 2022 United States $ 11,071 $ 14,382 United Kingdom 33,798 36,775 European Union 112,487 105,013 $ 157,356 $ 156,170 |
Recent Accounting Pronouncements Adopted | Recent Accounting Pronouncements Adopted In June 2016, the FASB issued ASU 2016-13, Financial Instruments – Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments |
Recent Accounting Pronouncements Not Yet Adopted | Recent Accounting Pronouncements Not Yet Adopted In October 2023, the FASB issued ASU 2023-06, Disclosure Improvements: Codification Amendments in Response to the SEC’s Disclosure Update and Simplification Initiative In November 2023, the FASB issued ASU 2023-07, Segment Reporting - Improvements to Reportable Segment Disclosures , which updates reportable segment disclosure requirements, primarily through enhanced disclosures about significant segment expenses and information used to assess segment performance. The amendments in this update are effective for fiscal years beginning after December 15, 2023, and interim periods within fiscal years beginning after December 15, 2024. Early adoption is permitted. The Company is still in the process of determining the effect this ASU will have on the consolidated financial statements. In December 2023, the FASB issued ASU 2023-09, Improvements to Income Tax Disclosures |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Summary of Significant Accounting Policies | |
Schedule of Restricted Cash | December 31, December 31, 2023 2022 Cash and cash equivalents $ 129,566 $ 115,516 Restricted cash 1,083 — Total cash, cash equivalents and restricted cash in the condensed consolidated statement of cash flows $ 130,649 $ 115,516 |
Schedule of Financial Assets and Liabilities Measured at Fair Value | Fair Value Measurement Using: Significant Significant Other Significant December 31, Observable Inputs Observable Inputs Unobservable Description 2023 (Level 1) (Level 2) (Level 3) Cash equivalents $ 46,868 $ 46,868 $ — $ — Restricted cash $ 1,083 $ 1,083 $ — $ — Fair Value Measurement Using: Significant Significant Other Significant December 31, Observable Inputs Observable Inputs Unobservable Description 2022 (Level 1) (Level 2) (Level 3) Cash equivalents $ 57,336 $ 57,336 $ — $ — Other long-term liabilities $ 262 $ 262 $ — $ — |
Schedule of Estimated Useful Lives of Assets | Asset Category Useful Lives Computer and office equipment 3 years Laboratory equipment 5 years Manufacturing equipment 7 years Furniture and fixtures 5 years Leasehold improvements lesser of useful life or remaining term of lease |
Schedule of Change in Asset Retirement Obligations | For the Years Ended December 31, 2023 2022 Balance at beginning of period $ 2,179 $ 2,081 Additional asset retirement obligations incurred during the period — 9 Amortization of interest 177 168 Effects of exchange rate changes 45 (79) Balance at end of period $ 2,401 $ 2,179 |
Schedule of Securities Excluded from Computation of Diluted Net Loss per Share | December 31, December 31, 2023 2022 Share options 8,226,707 6,858,409 Restricted share units 2,661,250 2,182,500 Deferred share units 185,000 110,000 Warrants 700,000 700,000 Restricted ordinary shares subject to forfeiture — 14,049 11,772,957 9,864,958 |
Schedule of Long-lived Assets by Geographical Area | December 31, December 31, 2023 2022 United States $ 11,071 $ 14,382 United Kingdom 33,798 36,775 European Union 112,487 105,013 $ 157,356 $ 156,170 |
Equity Method and Other Inves_2
Equity Method and Other Investments (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Equity Method and Other Investments | |
Schedule of equity method and other investments | December 31, 2023 Investee Investment Type Ownership Percentage Carrying Value Cost Basis Visiogene LLC Equity Method Investment 25 % $ 5,150 $ 5,165 Other Equity Investment 1.6 % 1,616 1,500 Total equity method and other investments $ 6,766 $ 6,665 |
Prepaid Expenses (Tables)
Prepaid Expenses (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Prepaid Expenses | |
Summary of Prepaid Expenses | December 31, December 31, 2023 2022 Clinical trial costs $ 1,530 $ 3,411 Dues and license fees 1,439 909 Insurance 861 1,485 Research and development 685 1,220 Facilities costs 630 539 Consulting 243 — Manufacturing costs 38 347 Other 199 222 $ 5,625 $ 8,133 |
Property, Plant and Equipment_2
Property, Plant and Equipment, net (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Property, Plant and Equipment, net | |
Schedule of Property, Plant and Equipment, net | December 31, December 31, 2023 2022 Leasehold improvements $ 103,082 $ 91,053 Manufacturing equipment 22,646 17,373 Laboratory equipment 15,389 13,804 Computer and office equipment 7,370 6,787 Furniture and fixtures 734 642 149,221 129,659 Less: Accumulated depreciation and amortization (33,325) (20,393) $ 115,896 $ 109,266 |
Intangible Assets (Tables)
Intangible Assets (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Intangible Assets | |
Schedule of Intangible Assets | December 31, December 31, 2023 2022 Licensed Technology $ 2,000 $ 1,900 Less: Accumulated amortization (882) (565) $ 1,118 $ 1,335 |
Schedule of Amortization Expense | Amortization Expense 2024 $ 285 2025 285 2026 285 2027 263 Total amortization $ 1,118 |
Accrued Expenses (Tables)
Accrued Expenses (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Accrued Expenses | |
Schedule of Accrued Expenses | December 31, December 31, 2023 2022 Compensation and benefits $ 12,129 $ 9,600 Clinical trial costs 8,713 13,041 Professional fees 6,499 732 Research and development 5,834 7,400 Interest on Tranche 1 Notes 2,936 — Manufacturing costs 2,634 4,326 Consulting 2,104 694 Fixed assets 1,472 3,093 Rent and facilities costs 142 — Other 176 932 $ 42,639 $ 39,818 |
Share-Based Compensation (Table
Share-Based Compensation (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Schedule of Share Option Activity | Weighted- Weighted- Average Average Remaining Number of Exercise Contractual Options Price Term (years) Outstanding at December 31, 2022 6,858,409 $ 14.03 6.86 years Granted 1,546,840 $ 8.49 Exercised (3,864) $ 2.64 Forfeited (174,678) $ 14.95 Outstanding at December 31, 2023 8,226,707 $ 12.96 6.35 years Options exercisable at December 31, 2023 5,709,190 $ 13.30 5.40 years Options vested and expected to vest at December 31, 2023 8,226,707 $ 12.96 6.35 years Aggregate intrinsic value of options outstanding as of December 31, 2023 $ 2,262 Aggregate intrinsic value of options exercisable as of December 31, 2023 $ 2,228 |
Schedule of Inputs Used in Determining Fair Value of Share Options | 2023 2022 Risk-free interest rate 3.86 - 4.48% 1.56 - 4.23% Expected volatility 72% 80% Expected dividend yield 0% 0% Expected term (in years) 3.6 - 6.1 5.5 - 6.1 |
Schedule of Restricted Share Unit (RSU) Activity | Weighted- Number of Average Restricted Grant Date Share Units Fair Value Outstanding at December 31, 2022 2,182,500 $ 18.59 Granted 1,097,500 $ 8.52 Vested (618,750) $ 15.17 Outstanding at December 31, 2023 2,661,250 $ 15.24 |
Schedule of Share-Based Compensation Expense | Years Ended December 31, 2023 2022 Research and development $ 12,780 $ 14,165 General and administrative 14,936 14,458 Total share-based compensation $ 27,716 $ 28,623 |
Employee Stock Option | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Schedule of Share-Based Compensation Expense | Years Ended December 31, 2023 2022 Research and development $ 8,577 $ 10,431 General and administrative 5,054 5,654 Total share-based compensation $ 13,631 $ 16,085 |
Restricted Share Units | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Schedule of Share-Based Compensation Expense | Years Ended December 31, 2023 2022 Research and development $ 4,203 $ 3,734 General and administrative 9,882 8,804 Total share-based compensation $ 14,085 $ 12,538 |
Income Taxes (Tables)
Income Taxes (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Income Taxes | |
Summary of Net Operating Loss Carryforwards | Federal State/City United Kingdom $ 169,599 $ — United States $ 65,549 $ 14,741 Ireland $ 47,681 $ — Other $ 29,399 $ — |
Schedule of Company's Pre-Tax Earnings | December 31, 2023 December 31, 2022 United Kingdom $ 12,881 $ (54,636) United States (63,017) (54,513) Ireland (29,965) (16,661) Other (3,926) (3,805) $ (84,027) $ (129,615) |
Schedule of Reconciliation of Income Tax Benefit with Amounts at the U.K. Statutory Income Tax Rate | December 31, 2023 December 31, 2022 Statutory rate (19,746) 23.50 % (24,627) 19.00 % Permanent differences - other 2,391 (2.85) % 2,323 (1.79) % RTP and other adjustment (1,165) 1.39 % 1,244 (0.96) % State and local rate, net of federal tax (698) 0.83 % (5,660) 4.37 % U.K. tax credit (4,028) 4.79 % 2,296 (1.77) % U.S. tax credit (9,830) 11.70 % (2,436) 1.88 % Foreign tax rate differential 4,558 (5.42) % 195 (0.15) % UK rate change (25% & 19%, respectively) 239 (0.28) % (1,973) 1.52 % US state rate change 15,394 (18.32) % (3) 0.00 % Section 162(m) deferred adjustment 2,728 (3.25) % 1,386 (1.07) % Disallowed interest 2,812 (3.35) % — 0.00 % Change in valuation allowance 7,345 (8.74) % 27,255 (21.03) % Actual income tax benefit effective tax rate — 0.00 % — 0.00 % |
Schedule of Expense/(Benefit) for Income Taxes from Continuing Operations | December 31, 2023 December 31, 2022 Current Tax Expense/(Benefit) United Kingdom — — United States — — Other — — Total Current — — Deferred Tax Expense/(Benefit) United Kingdom 2,393 (8,708) United States (6,251) (17,466) Ireland (3,963) (1,498) Netherlands (1,840) 1,717 Total Deferred (9,661) (25,955) Change in Valuation Allowance 9,661 25,955 Total Income Tax Expense/(Benefit) — — |
Schedule of Deferred Tax Assets and Liabilities | December 31, 2023 December 31, 2022 Deferred Tax Assets: Net operating loss carryforwards $ 70,650 $ 74,350 Capitalized research and development 19,606 16,288 Share-based compensation 14,290 13,684 R&D credit 21,687 10,837 Lease liability 4,070 6,461 Other 1,121 3,745 Deferred tax assets 131,424 125,365 Deferred Tax Liabilities: Indefinite-lived intangibles and fixed assets — (186) Depreciation (1,763) (2,935) Right of use assets (3,777) (6,207) Less: valuation allowance (125,884) (116,223) Net deferred tax liability $ — $ (186) |
Schedule of Unrecognized Tax Benefits | December 31, 2023 December 31, 2022 Unrecognized tax benefits as of January 1 $ 937 $ 666 Gross increases/(decreases) related to current year 756 279 Gross increases/(decreases) related to prior years 336 (8) Foreign currency translation — — Unrecognized tax positions as of December 31 $ 2,029 $ 937 |
Related Party Transactions (Tab
Related Party Transactions (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Related Party Transactions | |
Schedule of Deferred Revenue Recognition | Deferred revenue at December 31, 2021 $ 64,866 Deferred revenue recognized as license revenue during the year ended December 31, 2022 (15,920) Effects of exchange rate changes (6,387) Deferred revenue at December 31, 2022 $ 42,559 Deferred revenue recognized as license revenue during the year ended December 31, 2023 (14,017) Effects of exchange rate changes 2,033 Remaining deferred revenue under the Collaboration Agreement 30,575 Standalone selling price allocated to performance obligations 7,469 Effects of exchange rate changes (1,101) Deferred revenue at December 31, 2023 $ 36,943 |
Leases (Tables)
Leases (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Leases | |
Schedule of Components of Lease Cost | Years Ended December 31, 2023 2022 Finance lease cost Amortization of right-of-use assets $ 1,124 $ 1,103 Interest on lease liabilities — 1 Total finance lease cost 1,124 1,104 Operating lease cost 5,473 5,307 Short-term lease cost 159 154 Total lease cost $ 6,756 $ 6,565 |
Schedule of Amounts Reported in the Consolidated Balance Sheets for Leases | December 31, December 31, 2023 2022 Operating leases Right-of-use asset $ 15,910 $ 20,109 Capitalized lease obligations $ 17,145 $ 21,215 Finance leases Right-of-use asset $ 24,432 $ 24,718 Capitalized lease obligations $ — $ — Weighted-average remaining lease term Operating leases 4.3 years 5.6 years Finance leases 174.8 years 175.8 years Weighted-average discount rate Operating leases 8.8 % 8.8 % Finance leases 8.0 % 8.0 % |
Schedule of Other Information Related to Leases | Years Ended December 31, 2023 2022 Cash paid for amounts included in the measurement of lease liabilities Operating cash flows from finance leases $ — $ 52 Operating cash flows from operating leases $ 5,662 $ 5,384 Financing cash flows from finance leases $ — $ — Right-of-use assets obtained in exchange for lease liabilities Operating leases $ — $ 1,793 |
Schedule of Future Minimum Payments Under Non-cancellable Leases | Operating Leases 2024 $ 5,565 2025 5,524 2026 5,623 2027 1,626 2028 1,335 Thereafter 706 Total undiscounted lease payments $ 20,379 Less: Imputed interest (3,234) Total lease liabilities $ 17,145 |
Principal Business Activity (De
Principal Business Activity (Details) - USD ($) $ in Thousands | 1 Months Ended | 3 Months Ended | 12 Months Ended | |||
Dec. 20, 2023 | Dec. 31, 2021 | Mar. 31, 2019 | Mar. 31, 2024 | Dec. 31, 2023 | Dec. 31, 2022 | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||
Accumulated deficit | $ (554,231) | $ (470,204) | ||||
Cash flows from operations | (105,365) | (73,098) | ||||
Cash, cash equivalents | $ 137,703 | $ 130,649 | $ 115,516 | |||
Janssen Pharmaceuticals Inc | Collaboration Agreement | ||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||
Initial funding | $ 100,000 | |||||
Milestone funding | $ 30,000 | |||||
Janssen Pharmaceuticals Inc | Asset Purchase Agreement | ||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||
Non-refundable upfront cash funding | $ 65,000 | |||||
Milestone funding for development of drug substances | 10,000 | |||||
Milestone funding for development of drug products | 5,000 | |||||
Milestone funding for sale of drug products in the US | 175,000 | |||||
Milestone funding for sale of drug products in the UK, France, Germany, Spain and Italy | 75,000 | |||||
Milestone funding for transfer of manufacturing technology | 25,000 | |||||
Milestone funding upon regulatory approval | 10,000 | |||||
Minimum | Janssen Pharmaceuticals Inc | Collaboration Agreement | ||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||
Offsetting expense, period | 12 months | |||||
Maximum | Janssen Pharmaceuticals Inc | Asset Purchase Agreement | ||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||
Contingent Funding Available | $ 350,000 | |||||
Subsequent Events | Janssen Pharmaceuticals Inc | Asset Purchase Agreement | ||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||
Milestone funding for extension of RPGR product clinical trials | $ 50,000 |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies - Tax Incentives (Details) € in Millions, $ in Millions | 12 Months Ended | ||
Dec. 31, 2023 EUR (€) | Dec. 31, 2023 USD ($) | Dec. 31, 2022 USD ($) | |
SME tax incentive program | Maximum | |||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |||
Number of employees | 500 | 500 | |
Annual aggregate revenue | € | € 100 | ||
Aggregate total assets | € | € 86 | ||
Other Current Assets | |||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |||
Research & Development tax incentive receivable | $ | $ 2.9 | $ 6.8 | |
VAT receivable | $ | $ 0.6 | $ 1.1 |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies - IDA Ireland Grant (Details) $ in Millions | 1 Months Ended | 12 Months Ended | |
Aug. 31, 2021 EUR (€) | Dec. 31, 2023 EUR (€) | Dec. 31, 2023 USD ($) | |
IDA Ireland Financial Assistance Grants | Research and Development Expenses | |||
Government Assistance [Line Items] | |||
Recognized grant income | $ | $ 1 | ||
IDA Ireland Financial Assistance Grants | Maximum | |||
Government Assistance [Line Items] | |||
Termination period (upon completion of capital investment) | 5 years | ||
IDA Ireland Financial Assistance Grants | Minimum | |||
Government Assistance [Line Items] | |||
Termination period (from last grant payment) | 5 years | ||
IDA Ireland Employment Grants | |||
Government Assistance [Line Items] | |||
Grant funding received | € 500,000 | 0.6 | |
IDA Ireland Employment Grants | Maximum | |||
Government Assistance [Line Items] | |||
Grant funding for each new job created | € 1,000,000 | ||
IDA Ireland Employment Grants | Minimum | |||
Government Assistance [Line Items] | |||
Grant funding for each new job created | € 10,000 | ||
Aggregate number of new jobs required to receive Grant benefits | 100 | ||
Vacant job duration prompting repayment of grant funding | 6 months | ||
IDA Ireland Capital Grants | |||
Government Assistance [Line Items] | |||
Grant funding received | € 400,000 | $ 0.4 | |
IDA Ireland Capital Grants | Maximum | |||
Government Assistance [Line Items] | |||
Grant amount for machinery and equipment | € 1,200,000 | ||
IDA Ireland Capital Grants | Minimum | |||
Government Assistance [Line Items] | |||
Grant amount for machinery and equipment (percent of actual expenditure) | 4% | ||
Aggregate number of new jobs required to receive Grant benefits | 100 | ||
Expenditure requirement on eligible machinery and equipment | € 30,000,000 |
Summary of Significant Accoun_6
Summary of Significant Accounting Policies - Revenue Recognition (Details) | 12 Months Ended |
Dec. 31, 2023 | |
Janssen Pharmaceuticals Inc | Collaboration Agreement | |
Revenue Recognition, Milestone Method [Line Items] | |
Percentage of incurred R&D costs to be reimbursed by third parties | 100 |
Summary of Significant Accoun_7
Summary of Significant Accounting Policies - Assets and Liabilities Measured at Fair Value (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |||
Cash and cash equivalents | $ 129,566 | $ 115,516 | |
Restricted cash | 1,083 | ||
Total cash, cash equivalents and restricted cash in the condensed consolidated statement of cash flows | $ 130,649 | 115,516 | $ 137,703 |
Maximum | |||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |||
Maturity period of highly liquid investments classified as cash and cash equivalents | 90 days | ||
Other long-term liabilities | |||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |||
Fair value, net asset (liability) | 262 | ||
Restricted Cash | |||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |||
Fair value, net asset (liability) | $ 1,083 | ||
Cash and cash equivalents | |||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |||
Fair value, net asset (liability) | 46,868 | 57,336 | |
Level 1 | Other long-term liabilities | |||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |||
Fair value, net asset (liability) | 262 | ||
Level 1 | Restricted Cash | |||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |||
Fair value, net asset (liability) | 1,083 | ||
Level 1 | Cash and cash equivalents | |||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |||
Fair value, net asset (liability) | $ 46,868 | $ 57,336 |
Summary of Significant Accoun_8
Summary of Significant Accounting Policies - Estimated Useful Lives of Assets (Details) | Dec. 31, 2023 |
Licensed Technology | |
Property, Plant and Equipment [Line Items] | |
Intangible assets amortization period | 7 years |
Computer and Office Equipment | |
Property, Plant and Equipment [Line Items] | |
Property and equipment estimated useful life | 3 years |
Laboratory Equipment | |
Property, Plant and Equipment [Line Items] | |
Property and equipment estimated useful life | 5 years |
Manufacturing Equipment | |
Property, Plant and Equipment [Line Items] | |
Property and equipment estimated useful life | 7 years |
Furniture and Fixtures | |
Property, Plant and Equipment [Line Items] | |
Property and equipment estimated useful life | 5 years |
Summary of Significant Accoun_9
Summary of Significant Accounting Policies - Asset Retirement Obligations (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Asset Retirement Obligation [Abstract] | ||
Asset retirement obligations at beginning of period | $ 2,179 | |
Additional asset retirement obligations incurred during the period | $ 9 | |
Amortization of interest on asset retirement obligations | 177 | 168 |
Effects of exchange rate on asset retirement obligations | 45 | (79) |
Asset retirement obligations at end of period | $ 2,401 | $ 2,179 |
Summary of Significant Accou_10
Summary of Significant Accounting Policies - Income Taxes (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 |
Summary of Significant Accounting Policies | |||
Unrecognized tax positions | $ 2,029 | $ 937 | $ 666 |
Unrecognized tax benefits, income tax penalties and interest accrued | $ 0 |
Summary of Significant Accou_11
Summary of Significant Accounting Policies - Anti-dilutive Securities Excluded from EPS (Details) - shares | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Anti-dilutive securities excluded from EPS | 11,772,957 | 9,864,958 |
Employee Stock Option | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Anti-dilutive securities excluded from EPS | 8,226,707 | 6,858,409 |
Restricted Share Units | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Anti-dilutive securities excluded from EPS | 2,661,250 | 2,182,500 |
Stock Appreciation Rights (SARs) | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Anti-dilutive securities excluded from EPS | 185,000 | 110,000 |
Warrants | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Anti-dilutive securities excluded from EPS | 700,000 | 700,000 |
Restricted Ordinary Shares Subject to Forfeiture | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Anti-dilutive securities excluded from EPS | 14,049 |
Summary of Significant Accou_12
Summary of Significant Accounting Policies - Non-Current Assets by Geographical Area (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Revenues from External Customers and Long-Lived Assets [Line Items] | ||
Non-current assets | $ 157,356 | $ 156,170 |
United States | ||
Revenues from External Customers and Long-Lived Assets [Line Items] | ||
Non-current assets | 11,071 | 14,382 |
United Kingdom | ||
Revenues from External Customers and Long-Lived Assets [Line Items] | ||
Non-current assets | 33,798 | 36,775 |
European Union | ||
Revenues from External Customers and Long-Lived Assets [Line Items] | ||
Non-current assets | $ 112,487 | $ 105,013 |
Equity Method and Other Inves_3
Equity Method and Other Investments - Summary of Investments (Details) $ in Thousands | Dec. 31, 2023 USD ($) |
Schedule of Equity Method Investments [Line Items] | |
Carrying Value | $ 6,766 |
Cost Basis | $ 6,665 |
Visiogene LLC | |
Schedule of Equity Method Investments [Line Items] | |
Ownership percentage | 25% |
Carrying Value | $ 5,150 |
Cost Basis | $ 5,165 |
Other Investments | |
Schedule of Equity Method Investments [Line Items] | |
Ownership percentage | 1.60% |
Carrying Value | $ 1,616 |
Cost Basis | $ 1,500 |
Equity Method and Other Inves_4
Equity Method and Other Investments - Additional Information (Details) - USD ($) $ in Millions | 12 Months Ended | ||
Jan. 04, 2021 | Dec. 31, 2023 | Dec. 31, 2022 | |
Visiogene LLC | |||
Schedule of Equity Method Investments [Line Items] | |||
Number of preferred units acquired | 3,000,000 | ||
Cash consideration | $ 5 | ||
Ordinary shares issued in connection with a license agreement (in shares) | 75,000 | ||
Value of shares issued | $ 1.2 | ||
Visiogene LLC | Visiogene License Agreement | |||
Schedule of Equity Method Investments [Line Items] | |||
Allocation of payments | 1 | ||
Investment allocated amount expensed | 1 | ||
Visiogene LLC | Visiogene Unit Agreement | |||
Schedule of Equity Method Investments [Line Items] | |||
Allocation of payments | $ 5.2 | ||
Other Investments | |||
Schedule of Equity Method Investments [Line Items] | |||
Impairment due to dilution in ownership percentage | $ 0.3 | ||
Impairment recognized as adjustment to fair value | $ 0.5 |
Prepaid Expenses - Summary of P
Prepaid Expenses - Summary of Prepaid Expenses (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Prepaid Expenses | ||
Clinical trial costs | $ 1,530 | $ 3,411 |
Dues and license fees | 1,439 | 909 |
Insurance | 861 | 1,485 |
Research and development | 685 | 1,220 |
Facilities costs | 630 | 539 |
Consulting | 243 | |
Manufacturing costs | 38 | 347 |
Other | 199 | 222 |
Total prepaid expenses | $ 5,625 | $ 8,133 |
Property, Plant and Equipment_3
Property, Plant and Equipment, net (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Property, Plant and Equipment [Line Items] | ||
Property, plant and equipment, gross | $ 149,221 | $ 129,659 |
Less: Accumulated depreciation | (33,325) | (20,393) |
Property, plant and equipment, net | 115,896 | 109,266 |
Depreciation and amortization expense | 12,300 | 7,300 |
Leasehold Improvements | ||
Property, Plant and Equipment [Line Items] | ||
Property, plant and equipment, gross | 103,082 | 91,053 |
Manufacturing Equipment | ||
Property, Plant and Equipment [Line Items] | ||
Property, plant and equipment, gross | 22,646 | 17,373 |
Laboratory Equipment | ||
Property, Plant and Equipment [Line Items] | ||
Property, plant and equipment, gross | 15,389 | 13,804 |
Computer and Office Equipment | ||
Property, Plant and Equipment [Line Items] | ||
Property, plant and equipment, gross | 7,370 | 6,787 |
Furniture and Fixtures | ||
Property, Plant and Equipment [Line Items] | ||
Property, plant and equipment, gross | $ 734 | $ 642 |
Property, Plant and Equipment_4
Property, Plant and Equipment, net - Additional Information (Details) - USD ($) $ in Thousands | 12 Months Ended | |||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2020 | Jan. 01, 2019 | |
Property, Plant and Equipment [Line Items] | ||||
Asset retirement obligations | $ 2,401 | $ 2,179 | $ 2,081 | |
Operating Sublease | ||||
Property, Plant and Equipment [Line Items] | ||||
Asset retirement obligations, discounted rate | 8% | |||
Asset retirement obligations | $ 1,800 | $ 3,900 |
Intangible Assets (Details)
Intangible Assets (Details) - USD ($) $ in Thousands | 1 Months Ended | 12 Months Ended | |
Nov. 30, 2020 | Dec. 31, 2023 | Dec. 31, 2022 | |
Finite-Lived Intangible Assets [Line Items] | |||
Total | $ 1,118 | ||
Licensed Technology | |||
Finite-Lived Intangible Assets [Line Items] | |||
Gross Carrying Amount | 2,000 | $ 1,900 | |
Less: Accumulated Amortization | (882) | (565) | |
Total | $ 1,118 | 1,335 | |
Upfront payment for intangible assets | $ 2,100 | ||
Intangible assets amortization period | 7 years | ||
Intangible asset amortization | $ 300 | $ 200 |
Intangible Assets - Amortizatio
Intangible Assets - Amortization Expense (Details) $ in Thousands | Dec. 31, 2023 USD ($) |
Amortization expense | |
2024 | $ 285 |
2025 | 285 |
2026 | 285 |
2027 | 263 |
Total | $ 1,118 |
Accrued Expenses - Schedule of
Accrued Expenses - Schedule of Accrued Expenses (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Accrued Expenses | ||
Compensation and benefits | $ 2,634 | $ 4,326 |
Clinical trial costs | 12,129 | 9,600 |
Professional fees | 2,936 | |
Research and development | 8,713 | 13,041 |
Interest on Tranche 1 Notes | 6,499 | 732 |
Manufacturing costs | 5,834 | 7,400 |
Consulting | 1,472 | 3,093 |
Fixed assets | 2,104 | 694 |
Rent and facilities costs | 142 | |
Other | 176 | 932 |
Accrued expenses | $ 42,639 | $ 39,818 |
Share-Based Compensation - Summ
Share-Based Compensation - Summary of Company's Share Option Activity (Details) - USD ($) | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Number of Options | ||
Number of options, Beginning balance | 6,858,409 | |
Number of options, Granted | 1,546,840 | |
Number of options, Exercised | (3,864) | |
Number of options, Forfeited | (174,678) | |
Number of options, Ending balance | 8,226,707 | 6,858,409 |
Weighted-Average Exercise Price | ||
Weighted-average exercise price, Beginning balance | $ 14.03 | |
Weighted-average exercise price, Granted | 8.49 | |
Weighted-average exercise price, Exercised | 2.64 | |
Weighted-average exercise price, Forfeited | 14.95 | |
Weighted-average exercise price, Ending balance | $ 12.96 | $ 14.03 |
Options additional disclosures | ||
Number of options, Options exercisable | 5,709,190 | |
Weighted-average exercise price, Options exercisable | $ 13.30 | |
Weighted-average remaining contractual life of options, outstanding | 6 years 4 months 6 days | 6 years 10 months 9 days |
Weighted-average remaining contractual life of options, exercisable | 5 years 4 months 24 days | |
Number of options, Options vested and expected to vest | 8,226,707 | |
Weighted-average exercise price, Options vested and expected to vest | $ 12.96 | |
Weighted-average remaining contractual life, Options vested and expected to vest | 6 years 4 months 6 days | |
Aggregate intrinsic value, options outstanding | $ 2,262 | |
Aggregate intrinsic value, options exercisable | $ 2,228 |
Share-Based Compensation - Sche
Share-Based Compensation - Schedule of Grant Date Fair Values of the Share Options Granted Black-Scholes Valuation Model (Details) - Employee Stock Option | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Risk-free interest rate, minimum | 3.86% | 1.56% |
Risk-free interest rate, maximum | 4.48% | 4.23% |
Expected volatility | 72% | 80% |
Expected dividend yield | 0% | 0% |
Minimum | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Expected term (in years) | 3 years 7 months 6 days | 5 years 6 months |
Maximum | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Expected term (in years) | 6 years 1 month 6 days | 6 years 1 month 6 days |
Share-Based Compensation - Su_2
Share-Based Compensation - Summary of Restricted Share Units (Details) - Restricted Share Units | 12 Months Ended |
Dec. 31, 2023 $ / shares shares | |
Number of restricted share units | |
Number of restricted share units, Beginning balance | shares | 2,182,500 |
Number of restricted share units, Granted | shares | 1,097,500 |
Number of restricted share units, Vested | shares | (618,750) |
Number of restricted share units, Ending balance | shares | 2,661,250 |
Weighted-average grant date fair value | |
Weighted-average grant date fair value, Beginning balance | $ / shares | $ 18.59 |
Weighted-average grant date fair value, Granted | $ / shares | 8.52 |
Weighted-average grant date fair value, Vested | $ / shares | 15.17 |
Weighted-average grant date fair value, Ending balance | $ / shares | $ 15.24 |
Share-Based Compensation - Sc_2
Share-Based Compensation - Schedule of Share-Based Compensation Expense (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Share Based Compensation Expense [Line Items] | ||
Total share-based compensation | $ 27,716 | $ 28,623 |
Research and Development Expenses | ||
Share Based Compensation Expense [Line Items] | ||
Total share-based compensation | 12,780 | 14,165 |
General and Administrative Expenses | ||
Share Based Compensation Expense [Line Items] | ||
Total share-based compensation | $ 14,936 | $ 14,458 |
Share-Based Compensation - Addi
Share-Based Compensation - Additional Information (Details) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | 12 Months Ended | 24 Months Ended | ||||
Feb. 28, 2023 | Jan. 31, 2023 | Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2022 | Dec. 31, 2020 | Jan. 01, 2019 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Weighted-average remaining contractual life of options, outstanding | 6 years 4 months 6 days | 6 years 10 months 9 days | |||||
Fair value of options, Vested | $ 15,300 | $ 16,200 | |||||
Weighted-average grant date fair value of options granted | $ 5.67 | $ 12.81 | |||||
Period expected to realize unrecognized compensation expense | 3 years 8 months 12 days | ||||||
Total share-based compensation | $ 27,716 | $ 28,623 | |||||
Excess tax benefits in cash flows from operations | $ 0 | ||||||
Excess tax benefits in cash flows from financing activities | $ 0 | ||||||
General and Administrative Expenses | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Total share-based compensation | 14,936 | 14,458 | |||||
Research and Development Expenses | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Total share-based compensation | $ 12,780 | 14,165 | |||||
2018 Incentive Award Plan | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Annual percent increase in available shares under equity incentive plan | 4% | ||||||
Shares reserved for issuance | 11,110,748 | 3,054,996 | |||||
Additional shares reserved for issuance | 2,544,040 | ||||||
Shares available for future issuance | 310,488 | ||||||
Restricted Share Units | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Period expected to realize unrecognized compensation expense | 3 years 4 months 24 days | ||||||
Total compensation expense not yet recognized relating to unvested RSUs | $ 25,300 | ||||||
Total share-based compensation | $ 14,085 | $ 12,538 | |||||
Withheld ordinary shares | 237,859 | 128,812 | |||||
Taxes withheld on issuance of share-based awards | $ 1,500 | $ 2,800 | |||||
Restricted Share Units | General and Administrative Expenses | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Total share-based compensation | 9,882 | 8,804 | |||||
Restricted Share Units | Research and Development Expenses | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Total share-based compensation | 4,203 | 3,734 | |||||
Employee Stock Option | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Total unvested options compensation expense not yet recognized | 18,300 | ||||||
Total share-based compensation | 13,631 | 16,085 | |||||
Employee Stock Option | General and Administrative Expenses | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Total share-based compensation | 5,054 | 5,654 | |||||
Employee Stock Option | Research and Development Expenses | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Total share-based compensation | $ 8,577 | 10,431 | |||||
Employee Stock Option | 2018 Incentive Award Plan | Maximum | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Weighted-average remaining contractual life of options, outstanding | 10 years | ||||||
Directors | Employee Stock Option | 2018 Incentive Award Plan | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Vesting period | 36 months | ||||||
Certain executives, employees and consultants | Restricted Share Units | 2018 Incentive Award Plan | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Additional shares reserved for issuance | 2,675,000 | ||||||
Three Participants | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Modifications to share-based compensation | 300 | ||||||
Three Participants | General and Administrative Expenses | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Modifications to share-based compensation | 100 | ||||||
Three Participants | Research and Development Expenses | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Modifications to share-based compensation | $ 200 | ||||||
Six Participants | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Modifications to share-based compensation | $ 300 | ||||||
Six Participants | General and Administrative Expenses | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Modifications to share-based compensation | 200 | ||||||
Six Participants | Research and Development Expenses | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Modifications to share-based compensation | $ 100 | ||||||
First Anniversary | Employee Stock Option | 2018 Incentive Award Plan | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Vesting percentage | 25% | ||||||
Second Anniversary | Restricted Share Units | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Vesting percentage | 50% | ||||||
Third Anniversary | Restricted Share Units | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Vesting percentage | 25% | ||||||
Fourth Anniversary | Restricted Share Units | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Vesting percentage | 25% | ||||||
Over Three Years | Employee Stock Option | 2018 Incentive Award Plan | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Vesting period | 36 months |
Ordinary Shares (Details)
Ordinary Shares (Details) - USD ($) | 12 Months Ended | |||||
Oct. 30, 2023 | May 05, 2023 | May 03, 2023 | Nov. 09, 2022 | Dec. 31, 2023 | Dec. 31, 2022 | |
Class of Stock [Line Items] | ||||||
Common stock, shares issued | 63,601,015 | 48,477,209 | ||||
Private Placement | ||||||
Class of Stock [Line Items] | ||||||
Stock issuance costs | $ 6,418,000 | |||||
Accredited Investors | Private Placement | ||||||
Class of Stock [Line Items] | ||||||
Common stock, shares issued | 10,773,913 | |||||
Aggregate Offer Price | $ 5.75 | |||||
Proceeds from Issuance of Private Placement | $ 62,000,000 | |||||
Stock issuance costs | $ 4,100,000 | |||||
Sanofi Foreign Participations B.V. | Private Placement | ||||||
Class of Stock [Line Items] | ||||||
Common stock, shares issued | 4,000,000 | |||||
Aggregate Offer Price | $ 7.50 | |||||
Proceeds from Issuance of Private Placement | 30,000,000 | |||||
Stock issuance costs | $ 2,300,000 | |||||
JJDC | Private Placement | ||||||
Class of Stock [Line Items] | ||||||
Common stock, shares issued | 3,742,514 | |||||
Aggregate Offer Price | $ 6.68 | |||||
Proceeds from Issuance of Private Placement | $ 25,000,000 |
Income Taxes - Summary of Net O
Income Taxes - Summary of Net Operating Loss Carryforwards (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Operating Loss Carryforwards [Line Items] | ||
Loss before income taxes | $ (84,027) | $ (129,615) |
United Kingdom | ||
Operating Loss Carryforwards [Line Items] | ||
Loss before income taxes | 12,881 | (54,636) |
United States | ||
Operating Loss Carryforwards [Line Items] | ||
Loss before income taxes | (63,017) | (54,513) |
Ireland | ||
Operating Loss Carryforwards [Line Items] | ||
Loss before income taxes | (29,965) | (16,661) |
Other | ||
Operating Loss Carryforwards [Line Items] | ||
Loss before income taxes | (3,926) | (3,805) |
Federal | Research and Development Tax Credits | ||
Operating Loss Carryforwards [Line Items] | ||
Tax credit carryforwards | 20,300 | |
Federal | United Kingdom | ||
Operating Loss Carryforwards [Line Items] | ||
NOL carryforwards | 169,599 | |
Federal | United States | ||
Operating Loss Carryforwards [Line Items] | ||
NOL carryforwards | 65,549 | |
Federal | Ireland | ||
Operating Loss Carryforwards [Line Items] | ||
NOL carryforwards | 47,681 | |
Federal | Other | ||
Operating Loss Carryforwards [Line Items] | ||
NOL carryforwards | 29,399 | |
State/City | United States | ||
Operating Loss Carryforwards [Line Items] | ||
NOL carryforwards | $ 14,741 | |
Increase (decrease) in NOL carryforwards | (114,000) | |
State/City | Netherlands | ||
Operating Loss Carryforwards [Line Items] | ||
Increase (decrease) in NOL carryforwards | 3,700 | |
U.K. | Research and Development Tax Credits | ||
Operating Loss Carryforwards [Line Items] | ||
Tax credit carryforwards | $ 3,400 |
Income Taxes - Schedule of Expe
Income Taxes - Schedule of Expense/(Benefit) for Income Taxes from Continuing Operations (Details) - USD ($) | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Deferred Income Tax Expense (Benefit), Continuing Operations [Abstract] | ||
Total Deferred | $ (9,661,000) | $ (25,955,000) |
Change in Valuation Allowance | 9,661,000 | 25,955,000 |
United Kingdom | ||
Deferred Income Tax Expense (Benefit), Continuing Operations [Abstract] | ||
Foreign | 2,393,000 | (8,708,000) |
United States | ||
Deferred Income Tax Expense (Benefit), Continuing Operations [Abstract] | ||
United States | (6,251,000) | (17,466,000) |
Netherlands | ||
Deferred Income Tax Expense (Benefit), Continuing Operations [Abstract] | ||
Foreign | (1,840,000) | 1,717,000 |
Federal | ||
Deferred Income Tax Expense (Benefit), Continuing Operations [Abstract] | ||
Total Income Tax Expense (Benefit) | $ 0 | $ 0 |
Income Taxes - Summary of Recon
Income Taxes - Summary of Reconciliation of Income Tax Benefit with Amounts at the U.K. Statutory Income Tax Rate (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Change in valuation allowance | $ 9,661 | $ 25,955 |
U.K. | ||
Actual income tax benefit effective tax rate | 25% | 19% |
U.K. | HMRC | ||
Statutory rate | $ (19,746) | $ (24,627) |
Permanent differences - other | 2,391 | 2,323 |
RTP and other adjustments | (1,165) | 1,244 |
State and local rate, net of federal tax | (698) | (5,660) |
U.K. tax credit | (4,028) | 2,296 |
U.S. tax credit | (9,830) | (2,436) |
Foreign tax rate differential | 4,558 | 195 |
UK rate change (19% & 25% at expected DTA turn) | 239 | (1,973) |
US state rate change | 15,394 | (3) |
Section 162(m) deferred adjustment | 2,728 | 1,386 |
Disallowed interest | 2,812 | |
Change in valuation allowance | $ 7,345 | $ 27,255 |
Tax rate | 23.50% | 19% |
Permanent differences - other | (2.85%) | (1.79%) |
RTP and other adjustments | 1.39% | (0.96%) |
State and local rate, net of federal tax | 0.83% | 4.37% |
U.K. tax credit | 4.79% | (1.77%) |
U.S. tax credit | 11.70% | 1.88% |
Foreign tax rate differential | (5.42%) | (0.15%) |
UK rate change (19% & 25% at expected DTA turn) | (0.28%) | 1.52% |
US state rate change | (18.32%) | 0% |
Section 162(m) deferred adjustment | (3.25%) | (1.07%) |
Disallowed interest | (3.35%) | 0% |
Change in valuation allowance | (8.74%) | (21.03%) |
Actual income tax benefit effective tax rate | 0% | 0% |
U.K. | HMRC | Tax Year 2022 | ||
Tax rate | 19% | |
U.K. | HMRC | Tax Year 2023 | ||
Tax rate | 25% |
Income Taxes - Schedule of Ex_2
Income Taxes - Schedule of Expense (Benefit) for Income Taxes from Continuing Operations (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Deferred Tax Expense/(Benefit) | ||
Total Deferred | $ (9,661) | $ (25,955) |
Change in Valuation Allowance | 9,661 | 25,955 |
United Kingdom | ||
Deferred Tax Expense/(Benefit) | ||
Foreign | 2,393 | (8,708) |
United States | ||
Deferred Tax Expense/(Benefit) | ||
United States | (6,251) | (17,466) |
Netherlands | ||
Deferred Tax Expense/(Benefit) | ||
Foreign | (1,840) | 1,717 |
Ireland | ||
Deferred Tax Expense/(Benefit) | ||
Foreign | $ (3,963) | $ (1,498) |
Income Taxes - Schedule of Defe
Income Taxes - Schedule of Deferred Tax Assets and Liabilities (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Deferred Tax Assets: | ||
Net operating loss carryforwards | $ 70,650 | $ 74,350 |
Capitalized research and development | 19,606 | 16,288 |
Share-based compensation | 14,290 | 13,684 |
R&D credit | 21,687 | 10,837 |
Lease liability | 4,070 | 6,461 |
Other | 1,121 | 3,745 |
Deferred tax assets | 131,424 | 125,365 |
Deferred Tax Liabilities: | ||
Indefinite-lived intangibles and fixed assets | (186) | |
Depreciation | (1,763) | (2,935) |
Right of use assets | (3,777) | (6,207) |
Less: valuation allowance | $ (125,884) | (116,223) |
Net deferred tax liability | $ (186) |
Income Taxes - Unrecognized Tax
Income Taxes - Unrecognized Tax Positions (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Income Taxes | ||
Unrecognized tax benefits, beginning balance | $ 937 | $ 666 |
Gross increases/(decreases) related to current year | 756 | 279 |
Gross increases/(decreases) related to prior years | 336 | |
Gross increases/(decreases) related to prior years | (8) | |
Unrecognized tax benefits, ending balance | $ 2,029 | $ 937 |
Income Taxes - Additional Infor
Income Taxes - Additional Information (Details) - USD ($) | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Income Tax Examination [Line Items] | |||
Unrecognized Tax Benefits | $ 2,029,000 | $ 937,000 | $ 666,000 |
Unrecognized tax benefits, income tax penalties and interest accrued | 0 | ||
Federal | |||
Income Tax Examination [Line Items] | |||
Income tax expense (benefit) | 0 | 0 | |
Unrecognized Tax Benefits | 2,000,000 | 900,000 | |
Unrecognized tax benefits, income tax penalties and interest accrued | $ 0 | $ 0 | |
U.K. | |||
Income Tax Examination [Line Items] | |||
Effective income tax rate | 25% | 19% |
Related Party Transactions - Co
Related Party Transactions - Collaboration and License Agreements (Details) - USD ($) $ in Thousands | 1 Months Ended | 3 Months Ended | 12 Months Ended | |||||
Dec. 20, 2023 | Dec. 31, 2023 | Dec. 31, 2021 | Mar. 31, 2019 | Jan. 30, 2019 | Mar. 31, 2024 | Dec. 31, 2023 | Dec. 31, 2022 | |
Related Party Transaction [Line Items] | ||||||||
Common stock, shares issued | 63,601,015 | 63,601,015 | 48,477,209 | |||||
Shares issued to related party | 63,601,015 | 63,601,015 | 48,477,209 | |||||
Gain on sale of nonfinancial assets | $ 54,208 | |||||||
Other Income | ||||||||
Related Party Transaction [Line Items] | ||||||||
Gain on sale of nonfinancial assets | 54,200 | |||||||
Private Placement | ||||||||
Related Party Transaction [Line Items] | ||||||||
Offering costs | 6,418 | |||||||
Clinical IRD Product Candidate development | ||||||||
Related Party Transaction [Line Items] | ||||||||
Milestone payments | 30,000 | |||||||
Clinical IRD Product Candidate development | Maximum | ||||||||
Related Party Transaction [Line Items] | ||||||||
Additional development and commercialization milestones | $ 340,000 | |||||||
Clinical IRD Product Candidate development | Collaboration Agreement | ||||||||
Related Party Transaction [Line Items] | ||||||||
Royalties receivable, as a percentage | 20% | |||||||
Janssen Pharmaceuticals Inc | Collaboration Agreement | ||||||||
Related Party Transaction [Line Items] | ||||||||
Deferred revenue recognized as license revenue during the period | (14,017) | $ (15,920) | ||||||
Reimbursement of research and development expenses | 70,400 | 73,300 | ||||||
Initial funding | $ 100,000 | |||||||
Milestone funding | $ 30,000 | |||||||
Janssen Pharmaceuticals Inc | Collaboration Agreement | License revenue - related party | ||||||||
Related Party Transaction [Line Items] | ||||||||
Deferred revenue - related party recognized | $ 14,000 | $ 15,900 | ||||||
Janssen Pharmaceuticals Inc | Asset Purchase Agreement | ||||||||
Related Party Transaction [Line Items] | ||||||||
Initial agreement term | 4 years | |||||||
Written days-notice to terminate agreement | 90 days | |||||||
Non-refundable upfront cash funding | $ 65,000 | |||||||
Contingent Funding Available | 350,000 | |||||||
Milestone funding for development of drug substances | 10,000 | |||||||
Milestone funding for development of drug products | 5,000 | |||||||
Milestone funding for sale of drug products in the US | 175,000 | |||||||
Milestone funding for sale of drug products in the UK, France, Germany, Spain and Italy | 75,000 | |||||||
Milestone funding for transfer of manufacturing technology | 25,000 | |||||||
Milestone funding upon regulatory approval | 10,000 | |||||||
Janssen Pharmaceuticals Inc | UCLB RPGR License Agreement | ||||||||
Related Party Transaction [Line Items] | ||||||||
Deferred revenue - related party included in contract price of license agreement | 30,600 | |||||||
Variable consideration for services included in contract price of license agreement | 1,800 | |||||||
Asset purchase agreement funding included in contract price of license agreement | 65,000 | |||||||
Contract price of license agreement | $ 92,300 | $ 92,300 | ||||||
Offsetting credit for transition services | 5,100 | |||||||
Performance obligation for process performance qualification services | 2,900 | |||||||
Performance obligation for commercial supply of RPGR product | 6,900 | |||||||
Performance obligation for manufacturing technology transfer | 28,700 | |||||||
Performance obligation for sale of nonfinancial assets | $ 53,800 | |||||||
Janssen Pharmaceuticals Inc | Research, development and commercialization of gene therapies | ||||||||
Related Party Transaction [Line Items] | ||||||||
Collaboration agreement upfront payment | $ 100,000 | |||||||
Janssen Pharmaceuticals Inc | Clinical IRD Product Candidate development | Collaboration Agreement | ||||||||
Related Party Transaction [Line Items] | ||||||||
Percentage of costs payable on annual net sales of licensed product statement | 100% | |||||||
Subsequent Events | Janssen Pharmaceuticals Inc | Asset Purchase Agreement | ||||||||
Related Party Transaction [Line Items] | ||||||||
Milestone funding for extension of RPGR product clinical trials | $ 50,000 |
Related Party Transactions - Pe
Related Party Transactions - Performance Obligation (Details) - UCLB RPGR License Agreement - Janssen Pharmaceuticals Inc - Revenue, Remaining Performance Obligation, Expected Timing of Satisfaction, Start Date [Axis]: 2024-01-01 $ in Millions | Dec. 31, 2023 USD ($) |
Related Party Transaction [Line Items] | |
Unsatisfied performance obligation, amount | $ 36.9 |
Unsatisfied performance obligation, expected recognition period | 4 years |
Related Party Transactions - Fi
Related Party Transactions - Financing Agreement (Details) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | |||||
May 03, 2023 | Nov. 09, 2022 | Dec. 31, 2023 | Dec. 31, 2022 | Aug. 02, 2022 | Feb. 27, 2019 | |
Related Party Transaction [Line Items] | ||||||
Common stock, shares issued | 63,601,015 | 48,477,209 | ||||
Private Placement | ||||||
Related Party Transaction [Line Items] | ||||||
Offering costs | $ 6,418 | |||||
Private Placement | Ordinary Shares | ||||||
Related Party Transaction [Line Items] | ||||||
Offering costs | $ 119 | |||||
JJDC | Private Placement | Ordinary Shares | ||||||
Related Party Transaction [Line Items] | ||||||
Common stock, shares issued | 2,898,550 | |||||
JJDC | Secured Financing Agreement | Private Placement | Ordinary Shares | ||||||
Related Party Transaction [Line Items] | ||||||
Common stock, shares issued | 3,742,514 | |||||
Common stock, price per share | $ 6.68 | |||||
Proceeds from Issuance of Private Placement | $ 25,000 | |||||
Perceptive Advisors, LLC | Ordinary Shares | ||||||
Related Party Transaction [Line Items] | ||||||
Common stock, shares issued | 4,347,826 | |||||
Perceptive Advisors, LLC | Secured Financing Agreement | ||||||
Related Party Transaction [Line Items] | ||||||
Equity ownership in outstanding ordinary shares | 18% | |||||
Adage Capital Partners, L.P. | Ordinary Shares | ||||||
Related Party Transaction [Line Items] | ||||||
Common stock, shares issued | 1,565,217 | |||||
Perceptive Advisors, LLC and Adage Capital Partners, L.P. | Ordinary Shares | Minimum | ||||||
Related Party Transaction [Line Items] | ||||||
Third-party equity ownership in outstanding ordinary shares | 5% | |||||
Perceptive Advisors, LLC and Adage Capital Partners, L.P. | Private Placement | ||||||
Related Party Transaction [Line Items] | ||||||
Common stock, shares issued | 10,773,913 | |||||
Proceeds from Issuance of Private Placement | $ 62,000 | |||||
Offering costs | $ 4,100 |
Related Party Transactions - Le
Related Party Transactions - Leases (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Related Party Transaction [Line Items] | ||
Operating lease rent expenses | $ 5,500 | $ 5,300 |
Other current liabilities | 1,278 | 6,631 |
Cash paid | $ 5,662 | $ 5,384 |
Related Party Transactions - Re
Related Party Transactions - Revenue Recognition (Details) - Janssen Pharmaceuticals Inc - Collaboration Agreement - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Related Party Transaction [Line Items] | ||
Deferred revenue at the beginning | $ 42,559 | $ 64,866 |
Deferred revenue recognized as license revenue during the period | (14,017) | (15,920) |
Remaining deferred revenue under the Collaboration Agreement | 30,575 | |
Standalone selling price allocated to performance obligations | 7,469 | |
Effects of exchange rate changes | (1,101) | (6,387) |
Effects of exchange rate changes | 2,033 | |
Deferred revenue at the end | $ 36,943 | $ 42,559 |
Leases - Schedule of Components
Leases - Schedule of Components of Lease Cost (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Leases | ||
Amortization of right-of-use assets | $ 1,124 | $ 1,103 |
Interest on lease liabilities | 1 | |
Total finance lease cost | 1,124 | 1,104 |
Operating lease cost | 5,473 | 5,307 |
Short-term lease cost | 159 | 154 |
Total lease cost | $ 6,756 | $ 6,565 |
Leases - Schedule of Consolidat
Leases - Schedule of Consolidated Balance Sheets for Leases (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Operating leases | ||
Right-of-use asset | $ 15,910 | $ 20,109 |
Capitalized lease obligations | 17,145 | 21,215 |
Finance leases | ||
Right-of-use asset | $ 24,432 | $ 24,718 |
Weighted-average remaining lease term | ||
Operating leases | 4 years 3 months 18 days | 5 years 7 months 6 days |
Finance leases | 174 years 9 months 18 days | 175 years 9 months 18 days |
Weighted-average discount rate | ||
Operating leases | 8.80% | 8.80% |
Finance leases | 8% | 8% |
Leases - Schedule of Other Info
Leases - Schedule of Other Information Related to Leases (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Cash paid for amounts included in the measurement of lease liabilities | ||
Operating cash flows from finance leases | $ 52 | |
Operating cash flows from operating leases | $ 5,662 | 5,384 |
Right-of-use assets obtained in exchange for lease liabilities | ||
Right-of-use assets obtained in exchange for lease liabilities | $ 1,793 |
Leases - Schedule of Future Min
Leases - Schedule of Future Minimum Lease Payments Under Non-Cancellable Leases (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Operating Leases | ||
2024 | $ 5,565 | |
2025 | 5,524 | |
2026 | 5,623 | |
2027 | 1,626 | |
2028 | 1,335 | |
Thereafter | 706 | |
Total undiscounted lease payments | 20,379 | |
Less: Imputed interest | (3,234) | |
Operating lease liability | $ 17,145 | $ 21,215 |
Leases - Additional Information
Leases - Additional Information (Details) € in Millions | 12 Months Ended | |||||
Aug. 04, 2020 USD ($) contract property | Aug. 04, 2020 EUR (€) contract property | Dec. 31, 2023 USD ($) | Dec. 31, 2023 EUR (€) | Dec. 31, 2022 USD ($) | Dec. 31, 2022 EUR (€) | |
Lessee, Lease, Description [Line Items] | ||||||
Operating lease rent expenses | $ 5,500,000 | $ 5,300,000 | ||||
Right-of-use asset | 15,910,000 | 20,109,000 | ||||
Operating lease liability | $ 17,145,000 | 21,215,000 | ||||
Janssen Pharmaceuticals Inc | ||||||
Lessee, Lease, Description [Line Items] | ||||||
Number of operating leases | 3 | |||||
Right-of-use asset | $ 1,800,000 | |||||
Operating lease liability | 1,800,000 | |||||
Meira Ireland [Member] | Shannon Free Zone [Member] | ||||||
Lessee, Lease, Description [Line Items] | ||||||
Number of agreements | contract | 2 | 2 | ||||
Number of properties | property | 2 | 2 | ||||
Purchase price consideration | $ 21,200,000 | € 18 | ||||
Annual lease cost and annual maintenance fees | 400,000 | € 0.3 | ||||
Operating lease liability | $ 0 | |||||
Meira Ireland [Member] | Shannon Free Zone [Member] | Building one | ||||||
Lessee, Lease, Description [Line Items] | ||||||
Capitalized building costs | 13,800,000 | € 11.9 | ||||
Meira Ireland [Member] | Shannon Free Zone [Member] | Building two | ||||||
Lessee, Lease, Description [Line Items] | ||||||
Capitalized building costs | $ 8,900,000 | € 7.5 | ||||
Minimum | ||||||
Lessee, Lease, Description [Line Items] | ||||||
Lease term | 3 years | 3 years | ||||
Minimum | Janssen Pharmaceuticals Inc | ||||||
Lessee, Lease, Description [Line Items] | ||||||
Operating lease, term of contract | 3 years | |||||
Maximum | ||||||
Lessee, Lease, Description [Line Items] | ||||||
Lease term | 191 years | 191 years | ||||
Maximum | Janssen Pharmaceuticals Inc | ||||||
Lessee, Lease, Description [Line Items] | ||||||
Operating lease, term of contract | 9 years | |||||
Maximum | Meira Ireland [Member] | Shannon Free Zone [Member] | ||||||
Lessee, Lease, Description [Line Items] | ||||||
Lease term | 191 years | 191 years |
Debt Financing (Details)
Debt Financing (Details) - USD ($) | 12 Months Ended | ||
Aug. 02, 2022 | Dec. 31, 2023 | Dec. 31, 2022 | |
Line of Credit Facility [Line Items] | |||
Common stock, shares issued | 63,601,015 | 48,477,209 | |
Perceptive Credit Holdings | Note Purchase Agreement | Term Loan | |||
Line of Credit Facility [Line Items] | |||
Cash liquidity requirement | $ 3,000,000 | ||
Shares available through warrants | $ 400,000 | ||
Warrant exercise price | $ 15 | ||
Common stock, shares issued | 300,000 | ||
Aggregate Offer Price | $ 20 | ||
Unamortized debt discount, warrants | 2,300,000 | ||
Unamortized debt discount, legal costs | 2,100,000 | ||
Unamortized debt discount, gross | 4,400,000 | ||
Amortization of debt discount | 1,100,000 | $ 400,000 | |
Remaining unamortized discount on debt, total | 2,900,000 | ||
Perceptive Credit Holdings | Note Purchase Agreement | Term Loan - Tranche I | |||
Line of Credit Facility [Line Items] | |||
Initial face amount | 75,000,000 | ||
Unused borrowing capacity | $ 25,000,000 | ||
Term loan outstanding amount | $ 75,000,000 | ||
SOFR adjustment term | 1 month | ||
Interest rate at period-end | 15.32% | ||
Increase in accrued interest | $ 3,000,000 | ||
Interest expense on debt | 11,300,000 | $ 4,000,000 | |
Obligations secured by UK and Ireland subsidiaries | $ 3,000,000 | ||
Perceptive Credit Holdings | Note Purchase Agreement | Term Loan - Tranche I | Base variable rate | |||
Line of Credit Facility [Line Items] | |||
Stated interest rate | 10% | ||
Perceptive Credit Holdings | Note Purchase Agreement | Term Loan - Tranche I | SOFR | |||
Line of Credit Facility [Line Items] | |||
Floor percentage | 1% |
Commitments and Contingencies (
Commitments and Contingencies (Details) | Dec. 31, 2023 |
Commitments and Contingencies | |
Number of new material commitments | 0 |
Employee Benefit Plans - Additi
Employee Benefit Plans - Additional Information (Details) - USD ($) $ in Millions | 12 Months Ended | |||
Jan. 01, 2017 | Aug. 01, 2016 | Dec. 31, 2023 | Dec. 31, 2022 | |
Compensation And Retirement Disclosure [Line Items] | ||||
Defined benefit plan, plan assets, contributions by employer | $ 2.5 | $ 2 | ||
United States | ||||
Compensation And Retirement Disclosure [Line Items] | ||||
Defined contribution plan, annual contributions per employee, percent | 6% | |||
United States | Minimum | ||||
Compensation And Retirement Disclosure [Line Items] | ||||
Defined contribution plan, employee eligibility age | 21 years | |||
United Kingdom | Minimum | ||||
Compensation And Retirement Disclosure [Line Items] | ||||
Defined contribution plan, annual contributions per employee, percent | 0.50% | |||
Defined Benefit plan, employer matching contribution, percent | 7.50% | |||
United Kingdom | Maximum | ||||
Compensation And Retirement Disclosure [Line Items] | ||||
Defined contribution plan, annual contributions per employee, percent | 8% | |||
Defined Benefit plan, employer matching contribution, percent | 10% | |||
Country_IE | ||||
Compensation And Retirement Disclosure [Line Items] | ||||
Defined contribution plan, employer contribution exclusive of employee match, percent | 4.50% | |||
Belgium | ||||
Compensation And Retirement Disclosure [Line Items] | ||||
Defined contribution plan, employer contribution exclusive of employee match, percent | 8% |
Subsequent Event - (Details)
Subsequent Event - (Details) $ in Millions | 3 Months Ended |
Mar. 31, 2024 USD ($) | |
Subsequent Events | Janssen Pharmaceuticals Inc | Asset Purchase Agreement | |
Subsequent Event [Line Items] | |
Milestone funding for extension of RPGR product clinical trials | $ 50 |
Pay vs Performance Disclosure
Pay vs Performance Disclosure - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Pay vs Performance Disclosure | ||
Net Income (Loss) | $ (84,027) | $ (129,615) |
Insider Trading Arrangements
Insider Trading Arrangements | 12 Months Ended |
Dec. 31, 2023 | |
Trading Arrangements, by Individual | |
Rule 10b5-1 Arrangement Adopted | false |
Non-Rule 10b5-1 Arrangement Adopted | false |
Rule 10b5-1 Arrangement Terminated | false |
Non-Rule 10b5-1 Arrangement Terminated | false |