MeiraGTx Holdings plc
For the period ended Sep 30, 2025. Filed Nov 13, 2025.
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Sep 30, 2025 · Filed Nov 13, 2025
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Notes to Condensed Consolidated Financial Statements
- Organization and Basis of Presentation
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 genetic medicines company with a broad pipeline of late-stage clinical programs, including Parkinson's disease, radiation-induced xerostomia and AIPL1-associated retinal dystrophy. MeiraGTx clinical programs use targeted local delivery of small doses of genetic medicines to treat both inherited and more common conditions with severe unmet need. The successful development of the clinical pipeline is supported by the Company’s internal end-to-end manufacturing capabilities. The Company has two viral vector production facilities for good manufacturing practices (“GMP”), internal plasmid production for GMP, as well as an in-house Quality Control hub for stability and release, all fit for IND through commercial supply. In addition, the Company has developed a proprietary manufacturing platform with leading yield and quality aspects and commercial readiness. The Company’s core capabilities in viral vector and capsid optimization allow increased potency, decreased dose and significantly reduced cost of goods for its genetic medicines. The Company has developed a transformative gene regulation platform using bespoke synthetic riboswitch technology invented in-house that allows for the precise, dose-responsive expression of any transgene under the control of oral small molecules. The Company is focusing the riboswitch platform onin vivodelivery of biologic therapeutics such as the metabolic peptides GLP-1, GIP, glucagon, amylin, PYY and leptin via oral small molecules, as well as cell therapy for oncology and autoimmune diseases, and long-term intractable pain. The Company has developed unique comprehensive technology capabilities 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 high.
Hologen Transactions
On March 9, 2025 (the “Signing Date”), the Company and its affiliates entered into a strategic collaboration with Hologen Limited, a non-cellular company limited by shares incorporated in Guernsey (“Hologen”), and its affiliates. Hologen is a leading developer of multi-modal generative AI foundation models of real-world clinical data for clinical medicine and pharmaceutical drug development. As part of the strategic collaboration, the Company and Hologen have entered into the Framework Agreements (as defined below), pursuant to which the Company and its affiliates will receive from Hologen an upfront cash payment of $200 million (the “Upfront Payment”) on the Closing Date (as defined below), and Hologen will provide additional funding of up to an additional $230 million as further described below. As part of the strategic collaboration, the Company also received 250,000 Class A shares of Hologen at a nominal price. Furthermore, the Company has the option to receive up to an additional 500,000 Class A shares of Hologen at a nominal price if certain funding obligations as described herein are not met by Hologen. During the nine month-period ended September 30, 2025, Hologen made a $28.0 million payment as part of its commitment toward the Upfront Payment which is included in other current liabilities, and made an additional $22.0 million payment to date during the fourth quarter 2025.
The closing of the transactions contemplated by the Framework Agreements (the “Closing Date”) is expected to occur in the fourth calendar quarter of 2025, subject to customary closing and funding conditions.
Neuro Framework Agreement
On the Signing Date, the Company, MeiraGTx Neuro UK Limited, a private company limited by shares incorporated in England and a wholly-owned subsidiary of the Company (“MeiraGTx Neuro UK”), Hologen Neuro AI Limited, a non-cellular company limited by shares incorporated in Guernsey and an affiliate of Hologen (“Hologen Neuro”), and Hologen, entered into that certain Framework Agreement (the “Neuro Framework Agreement”), pursuant to which, on the Closing Date, the Company, MeiraGTx Neuro UK, MeiraGTx Neuro I, LLC, a Delaware limited liability company and a wholly-owned subsidiary of the Company (“MeiraGTx Neuro US”), Hologen, Hologen Neuro and Hologen Neuro AI UK Limited, a private company limited by shares incorporated in England and an affiliate of Hologen (“Hologen Neuro UK”), shall enter into a Collaboration and License Agreement (the “Hologen Collaboration Agreement”) for the research, development, manufacture and commercialization of the Company’s (i) AAV-GAD investigational gene therapy for the treatment of Parkinson’s disease, AAV-BDNF investigational gene therapy for the treatment of genetic obesity disorders and other potential locally delivered genetic medicines to the central nervous system (the “Clinical Programs”) and (ii) proprietary device designed to effect the local delivery of a gene therapy product into the central nervous system or any topographic or subcutaneous tissue modification on the face and scalp, of humans or animals (the “Delivery Device”), in each case, in accordance with the terms and conditions of the Hologen Collaboration Agreement.
On the Closing Date, under the Hologen Collaboration Agreement, MeiraGTx Neuro US will receive the applicable portion of the Upfront Payment in consideration for granting to Hologen Neuro and Hologen Neuro UK, as of the Closing Date and subject to the license granted by Hologen Neuro and Hologen Neuro UK back to MeiraGTx Neuro UK, exclusive, worldwide, royalty-free, fully paid-up licenses to certain of the Company’s intellectual property rights for the research, development, manufacture and commercialization of the Clinical Programs and the Delivery Device. On the Closing Date, (a) MeiraGTx Neuro UK will receive Class A shares of Hologen Neuro representing a 30% ownership of the issued share capital of Hologen Neuro, in consideration for the provision of services to Hologen Neuro and Hologen Neuro UK as specified in the Hologen Collaboration Agreement, including services relating to the development of the Clinical Programs and the Delivery Device and certain other transition services, and (b) Hologen Guernsey will receive Class B shares of Hologen Neuro representing a 70% ownership of the issued share capital of Hologen Neuro, in consideration for paying the applicable portion of the Upfront Payment to MeiraGTx Neuro US, as well as a commitment to provide additional capital of up to $230 million to fund the development of the Clinical Programs and the Delivery Device. Additionally, Hologen will license to Hologen Neuro its proprietary multi-modal generative foundation models (LMMs), or large medicine models, pursuant to a license agreement mutually agreeable to the parties.
As of the Closing Date, Hologen Neuro shall be governed by a board of directors comprised of three representatives designated by Hologen and two representatives designated by MeiraGTx Neuro UK, and certain material business decisions (as further enumerated in the Neuro Framework Agreement) will require the approval of at least 70% of the directors then in office.
Following the Closing Date and in accordance with the terms of the Hologen Collaboration Agreement, the parties shall negotiate in good faith and enter into clinical and commercial supply agreements, pursuant to which MeiraGTx Neuro UK (directly, or through affiliates or subcontractors) shall manufacture and supply AAV-GAD, AAV-BDNF and other potential locally delivered genetic medicines to the central nervous system.
Manufacturing Framework Agreement
On the Signing Date, MeiraGTx Manufacturing Limited, a private company limited by shares incorporated in England and a wholly-owned subsidiary of the Company (“MeiraGTx Manufacturing”), MeiraGTx Limited, a private company limited by shares incorporated in England and a wholly-owned subsidiary of the Company (“MeiraGTx Limited”), and Hologen, entered into that certain Framework Agreement (the “Manufacturing Framework Agreement” and, together with the Neuro Framework Agreement, the “Framework Agreements”), pursuant to which, on the Closing Date and in exchange for the applicable portion of the Upfront Payment, Hologen will acquire a minority interest in MeiraGTx Manufacturing, an entity that will comprise the Company’s flexible and scalable end-to-end genetic medicines manufacturing business, which is solely run by MeiraGTx Manufacturing and MeiraGTx Limited prior to the Closing Date. Hologen will also contribute a portion of the annual funding to MeiraGTx Manufacturing.
As of the Closing Date, MeiraGTx Manufacturing shall be governed by a board of directors comprised of three representatives designated by MeiraGTx Limited and two representatives designated by Hologen, and certain material business decisions (as further enumerated in the Manufacturing Framework Agreement) will require the approval of at least 70% of the directors then in office.
For a period of twelve months following the Closing Date, Hologen has an exclusive, irrevocable option to purchase additional shares in MeiraGTx Manufacturing at a specified price, such that following exercise of such option, Hologen shall own 40% of the issued share capital of MeiraGTx Manufacturing in the aggregate. In the event that Hologen does not exercise its option, MeiraGTx has an exclusive, irrevocable option to purchase all of the shares of MeiraGTx Manufacturing held by Hologen for the same price that Hologen paid for such shares. Such option shall be exercisable anytime beginning on the third anniversary of the Closing Date and ending three years thereafter.
Asset Purchase and Related Agreements with Johnson & Johnson Innovative Medicine
On January 30, 2019, the Company entered into a Collaboration, Option and License Agreement with Johnson & Johnson Innovative Medicine (formerly known as Janssen Pharmaceuticals, Inc.) (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 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 Johnson & Johnson Innovative Medicine pursuant to which the Company sold and assigned to Johnson & Johnson Innovative Medicine, and Johnson & Johnson Innovative Medicine 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 botaretigene sparoparvovec, or bota-vec (formerly referred to as AAV-RPGR), for the treatment of X-linked retinitis pigmentosa related to mutations in the retinitis pigmentosa GTPase regulator gene, or XLRP-RPGR (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 Johnson & Johnson Innovative Medicine terminating the Collaboration Agreement. The Company and Johnson & Johnson Innovative Medicine also entered into a Supply Agreement on December 20, 2023 (the “Supply Agreement”) pursuant to which the Company agreed to manufacture and supply the RPGR Product for Johnson & Johnson Innovative Medicine.
Under the Asset Purchase Agreement, Johnson & Johnson Innovative Medicine 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, Johnson & Johnson Innovative Medicine 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; (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 Johnson & Johnson Innovative Medicine; and (vii) $10.0 million upon regulatory approval of a Johnson & Johnson Innovative Medicine-selected manufacturing facility in each of the United States and European Union for commercial manufacture of the RPGR Product. During 2024, the Company received $60.0 million in milestone payments from Johnson & Johnson Innovative Medicine. Johnson & Johnson Innovative Medicine 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 condensed 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 Updates (“ASU”) of the Financial Accounting Standards Board (“FASB”). Certain reclassifications of prior period activities have been made to conform to current year presentation.
Interim Financial Statements
The accompanying condensed consolidated financial statements have been prepared in accordance with GAAP for interim financial information and with the instructions to Form 10-Q and Article 10 of Regulation S-X. Accordingly, they do not include all the information and footnotes required by GAAP for complete consolidated financial statements. In the opinion of management, the condensed consolidated financial statements include all adjustments (consisting of normal recurring adjustments) necessary in order to make the condensed consolidated financial statements not misleading. Operating results for the nine-month period ended September 30, 2025 are not necessarily indicative of the results that may be expected for the year ending December 31, 2025. These unaudited condensed consolidated financial statements should be read in conjunction with the audited consolidated financial statements and the notes thereto for the year ended December 31, 2024 included in the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2024 (the “Form 10-K”).
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 September 30, 2025 totaled $831.3 million, and management expects to incur substantial losses in future periods. The success of the Company is subject to certain risks and uncertainties, including, among others: uncertainty of product development; competition in the Company’s field of use; uncertainty of capital availability; uncertainty in the Company’s ability to enter into agreements and consummate transactions with collaborative partners; expanding and protecting the Company’s intellectual property portfolio; dependence on third parties; and dependence on key personnel. For the nine months ended September 30, 2025, the Company used $93.0 million in cash flows from operations and there are no assurances that the Company will generate positive cash flows in the future. Additionally, there are no assurances that the Company will be successful in obtaining an adequate level of financing for the development and commercialization of its product candidates.
As of September 30, 2025, the Company had cash, cash equivalents and restricted cash in the amount of $17.1 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, tax incentive receivable and accounts receivable – related party at September 30, 2025, together with the $75.0 million upfront payment from Eli Lilly and Company as described in Note 12, and the $22.0 million deposit received from Hologen to date during the fourth quarter 2025 and the remaining $150.0 million from the anticipated closing of the strategic collaboration with Hologen, will be sufficient to cover its expenses for at least the next twelve months from the date of issuance of these condensed 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 10. 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 Accounting Policies and Recent Accounting Pronouncements
Certain of the Company’s significant accounting policies are described below. All of the Company’s significant accounting policies are disclosed in the notes to the audited consolidated financial statements as of and for the year ended December 31, 2024 included in the Company’s Form 10-K.
Consolidation
The accompanying condensed 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 UK Limited, a limited company incorporated under the laws of England and Wales (“Meira UK”);
MeiraGTx Belgium, a private company with limited liability incorporated under the laws of Belgium (“Meira Belgium”);
MeiraGTx Cell Therapies, a simplified joint stock company, incorporated under the laws of France;
BRI-Alzan, Inc., a Delaware corporation;
MeiraGTx Bio, Inc., a Delaware corporation;
MeiraGTx B.V., a private company with limited liability incorporated under the laws of the Netherlands (“Meira B.V.”);
MeiraGTx Netherlands, B.V., a private company with limited liability incorporated under the laws of the Netherlands (“Meira Netherlands”);
MeiraGTx Neurosciences, Inc., a Delaware corporation;
MeiraGTx Therapeutics, Inc., a Delaware corporation;
MeiraGTx Neuro I, LLC, a Delaware limited liability company;
MeiraGTx Neuro II, LLC, a Delaware limited liability company;
MeiraGTx Manufacturing Limited, a limited company incorporated under the laws of England and Wales;
MeiraGTx Ocular UK Limited, a limited company incorporated under the laws of England and Wales (“Meira Ocular”);
MeiraGTx Gene Regulation Limited, a limited company incorporated under the laws of England and Wales; and
MeiraGTx Neuro UK Limited, a limited company incorporated under the laws of England and Wales.
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 condensed 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 condensed consolidated financial statements, management used significant estimates in the following areas, among others: service revenue, fair value of nonfinancial assets, stand-alone selling price and material rights in connection with the Asset Purchase and 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.
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 condensed consolidated balance sheets to the total of the amount presented in the condensed consolidated statements of cash flows (in thousands):
| · · · · · · | ||||
|---|---|---|---|---|
| | | September 30, | | December 31, |
| | | 2025 | | 2024 |
| Cash and cash equivalents | $14,841 | $103,659 | ||
| Restricted cash | | 2,265 | | 2,009 |
| Total cash, cash equivalents and restricted cash in the condensed consolidated statement of cash flows | | $17,106 | | $105,668 |
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, or ASC 820, for application to financial assets and liabilities. In addition to defining fair value, the standard expands the disclosure requirements around fair value and establishes a fair value hierarchy for valuation inputs. The hierarchy prioritizes the inputs into 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 | ||||||||
| | | September 30, | | Observable Inputs | | Observable Inputs | | Unobservable |
| Description | | 2025 | | (Level 1) | | (Level 2) | | (Level 3) |
| Cash equivalents | | $9,975 | | $9,975 | | $— | | $— |
| Restricted cash | | $2,265 | | $2,265 | | $— | | $— |
| · · · · · · · · · · · · | ||||||||
|---|---|---|---|---|---|---|---|---|
| · · Fair Value Measurement Using: | ||||||||
| · · · Significant · Significant Other · Significant | ||||||||
| | | December 31, | | Observable Inputs | | Observable Inputs | | Unobservable |
| Description | | 2024 | | (Level 1) | | (Level 2) | | (Level 3) |
| Cash equivalents | | $80,930 | | $80,930 | | $— | | $— |
| Restricted cash | | $2,009 | | $2,009 | | $— | | $— |
At September 30, 2025, the Company's financial instruments included cash and cash equivalents, restricted cash, accounts receivable – related party, and accounts payable. The carrying amounts reported in the Company's condensed consolidated financial statements for these instruments approximate their respective fair values because of the short-term nature of these instruments. In addition, at September 30, 2025, the Company believes the carrying value of the Tranche 1 Notes (as defined in Note 10) approximates fair value as the interest rate is reflective of the rate the Company could obtain on debt with similar terms and conditions.
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 condensed 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 condensed consolidated statements of operations and comprehensive loss.
During the first quarter of 2025, the Company determined that it was reasonably certain to exercise its early termination option on an operating lease for laboratory and office space, for which the Company had an associated asset retirement obligation liability in the amount of $1.7 million and a corresponding leasehold improvement with a net book value of $0.3 million as of March 31, 2025. At that time, the landlord indicated that it would no longer require the Company to make any changes to the premises prior to the lease termination and the Company only expected to incur $0.1 million related to decontamination costs. Therefore, a change in estimate of the asset retirement obligation was recorded for the three months ended March 31, 2025 resulting in a gain on termination of lease liabilities of $1.3 million which is included in general and administrative expenses for the nine months ended September 30, 2025 in the accompanying condensed consolidated statements of operations. The lease was terminated as of August 31, 2025 and the Company did not incur any costs applicable to the asset retirement obligation. The remaining asset retirement obligation of $0.1 million was derecognized and reflected as an additional gain on termination of lease liabilities.
The change in asset retirement obligations is as follows (in thousands):
| | | | | | | |
|---|---|---|---|---|---|---|
| · · For the Nine-Month Periods Ended September 30, | ||||||
| · · 2025 · · 2024 | ||||||
| Balance at beginning of period | $2,821 | $2,401 | ||||
| Change in estimate | | | (1,476) | | | 370 |
| Amortization of interest | | 126 | | 146 | ||
| Effects of exchange rate changes | | (106) | | (37) | ||
| Balance at end of period | | $1,365 | | $2,880 | ||
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 condensed consolidated balance sheet. All expenditures were required to be completed by December 31, 2024, and the agreement terminates on the later offive years from the date of the last payment from
the grant orfive 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 $0.6 million for the employment grant and $0.4 million for the capital grant. The Company received its second drawdown under the grant in 2024, which was comprised of $0.5 million for the employment grant and $0.5 million for the capital grant. The Company received its third and final drawdown under the grant in 2025, which was comprised of a de minimis amount for the employment grant and $0.1 million for the capital grant. During the nine-months ended September 30, 2025 and 2024, the Company recognized de minimis grant income as a reduction of research and development expenses in the accompanying condensed consolidated statements of operations and comprehensive loss.
During thefive-year period ending on the termination of the grant agreement, Meira Ireland must maintain compliance with the terms of the grant. If the total number of jobs is less than 100 at the time of IDA Ireland’s annual review, the Company may have to repay a portion of the capital grant, and if a job for which the Company received employment grant funding remains vacant for a period in excess ofsix calendar months, the Company may have to repay the employment grant received for that job. As of September 30, 2025, the Company is in compliance with the terms of the grant.
Revenue Recognition
The Company evaluates the promised goods or services to determine which promises, or group of promises, represent performance obligations. 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 contract assets when the right to consideration is deemed unconditional. Contract assets are reclassified as accounts receivable once billed. 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 condensed 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 revenue arrangements include the following:
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.
Research and Development Services: Under the Asset Purchase Agreement, research and development services (PPQ services) are recorded as incurred under cost of service revenue – related party.
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 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 and other costs associated with clinical and preclinical activities and regulatory operations. 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 condensed consolidated financial statements as prepaid or accrued research and development expenses, as the case may be.
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 assumed to be 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 potential 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:
| | | | | |
|---|---|---|---|---|
| · September 30, · September 30, | ||||
| · 2025 · 2024 | ||||
| Share options | 7,964,365 | 8,273,270 | ||
| Restricted share units | | 6,182,250 | | 4,252,250 |
| Deferred share units | | — | | 272,500 |
| Warrants | | 700,000 | | 700,000 |
| | 14,846,615 | 13,498,020 | ||
Segment Information
Operating segments are defined as components of an enterprise about which separate discrete information is available for evaluation by the chief operating decision maker, or decision-making group, in deciding how to allocate resources in assessing performance. The Company hasone reportable andoperating segment, which is the development and manufacturing of genetic medicines, for purposes of reporting financial condition and results of operations. The Company’s chief operating decision maker (“CODM”) is the chief executive officer.
The accounting policies of its segment are the same as those described in the summary of significant accounting policies. The CODM allocates resources and assesses performance of the Company’s single reportable segment by regularly reviewing the segment net loss that also is reported on the condensed consolidated statement of operations and comprehensive loss as net loss.
The following table sets forth information about the Company’s single reportable segment and the significant expenses reviewed by the CODM, including a reconciliation to net loss (in thousands):
| | | | | | | | | | | | | |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| · · For the Three-Month Periods Ended September 30, · · For the Nine-Month Periods Ended September 30, | ||||||||||||
| · · 2025 · · 2024 · · 2025 · · 2024 | ||||||||||||
| Service revenue - related party | | $410 | $10,910 | $6,027 | $11,889 | |||||||
| Operating expenses: | | | | | | | | | | | | |
| Cost of service revenue - related party | | | 313 | | 11,985 | | | 4,367 | | | 11,985 | |
| General and administrative | | 9,751 | | 8,972 | | 24,699 | | 25,922 | ||||
| Clinical programs: | | | | | | | | | | | | |
| Botaretigene sparoparvovec | | | — | | | 204 | | | — | | | 1,339 |
| AAV-hAQP1 | | | 1,997 | | | 7,852 | | | 9,603 | | | 14,131 |
| AAV-CNGB3 / AAV-CNGA3 | | | — | | | (399) | | | — | | | (760) |
| AAV-GAD | | | 1,200 | | | 1,613 | | | 11,630 | | | 5,249 |
| Other ocular diseases | | | 2,915 | | | 1,470 | | | 4,380 | | | 1,764 |
| Manufacturing | | | 12,568 | | | 1,781 | | | 35,557 | | | 35,017 |
| Preclinical programs: | | | | | | | | | | | | |
| Gene regulation | | | 2,683 | | | 1,991 | | | 8,210 | | | 7,592 |
| Neurodegenerative diseases | | | 271 | | | 443 | | | 995 | | | 1,194 |
| Preclinical ocular diseases | | | 688 | | | 595 | | | 2,475 | | | 1,505 |
| Other research and development1 | | | 4,968 | | | 4,881 | | | 10,620 | | | 10,842 |
| Share-based compensation | | | 5,952 | | | 6,304 | | | 16,504 | | | 19,074 |
| Depreciation and amortization | | | 3,155 | | | 3,259 | | | 9,427 | | | 9,757 |
| Total operating expenses | | | 46,461 | | | 50,951 | | | 138,467 | | | 144,611 |
| Other segment items2 | | | (4,462) | | | 711 | | | 3,151 | | | 24,330 |
| Segment net loss | | $(50,513) | | | (39,330) | | $(129,289) | | | (108,392) | ||
| Net loss | | $(50,513) | | $(39,330) | | $(129,289) | | $(108,392) | ||||
1 Other research and development is comprised of all other costs including payroll and payroll related costs, travel, rent and facilities costs and other non-program specific expenses.
2 Other segment items is comprised of foreign currency gain (loss), interest income, interest expense and gain on sale of nonfinancial assets.
The Company’s service revenue and deferred revenue from the Asset Purchase Agreement and related agreements were generated in the United Kingdom.
The following table summarizes long-lived assets by geographical area (in thousands):
| | | | | | | |
|---|---|---|---|---|---|---|
| · September 30, · December 31, | ||||||
| · · 2025 · · 2024 | ||||||
| United States | | $9,160 | | $7,209 | ||
| United Kingdom | | 27,082 | | 28,419 | ||
| European Union | | | 108,930 | | | 100,845 |
| | | $145,172 | | $136,473 | ||
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. This update includes a number of amendments to clarify or improve disclosure and presentation requirements of a variety of topics in order to allow users to more easily compare entities subject to the SEC’s existing disclosures with those entities that were not previously subject to the requirements and to align the requirements in the FASB accounting standard codification with the SEC’s regulations. The effective date for each amendment will be the date on which the SEC’s removal of that related disclosure requirement from Regulation S-X or Regulation S-K becomes effective, with early adoption prohibited. The Company does not expect that the application of this standard will have an impact on its consolidated financial statements.
In December 2023, the FASB issued ASU 2023-09, Improvements to Income Tax Disclosures, which requires that an entity disclose specific categories in the effective tax rate reconciliation as well as provide additional information for reconciling items that meet a quantitative threshold. Further, the ASU requires certain disclosures of state versus federal income tax expense and taxes paid. This ASU is effective for annual periods beginning after December 15, 2024. The Company does not expect the adoption of this ASU to have a material impact on its consolidated financial statements.
In November 2024, the FASB issued ASU No. 2024-03, Income Statement: Reporting Comprehensive Income: Expense Disaggregation Disclosures (Subtopic 220-40), which requires public business entities to disclose additional information about specific expense categories in the notes to financial statements at interim and annual reporting periods. In January 2025, the FASB issued ASU No. 2025-01, Clarifying the Effective Date, which revised the effective date of ASU No. 2024-03 for interim periods. The guidance is effective for annual periods beginning after December 15, 2026, and interim periods within fiscal years beginning after December 15, 2027. The Company is currently assessing the impact of ASU 2024-03 and ASU 2025-01 on its consolidated financial statements.
In July 2025, the FASB issued ASU 2025-05, Financial Instruments - Credit Losses (Topic 326): Measurement of Credit Losses for Accounts Receivable and Contract Assets. This amendment introduces a practical expedient for the application of the current expected credit loss model to current accounts receivable and contract assets. The guidance is effective for annual periods beginning after December 15, 2025, and interim periods within those annual reporting periods. Early adoption is permitted. The Company is currently evaluating the timing of the adoption and the impact of this ASU on its consolidated financial statements and related disclosures.
- Equity Method and Other Investments
The Company’s investments consist of the following (in thousands):
| · · · · · · · · · · · | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|
| · · · · September 30, 2025 | ||||||||||
| Investee · Investment Type · Ownership Percentage · Carrying Value · Cost Basis | ||||||||||
| Visiogene LLC | | Equity Method Investment | | 25.0% | | $5,133 | | $5,165 | ||
| Other | | Equity Investment | | 0.9% | | | 1,616 | | | 1,500 |
| Total equity method and other investments | | | | | | | $6,749 | | $6,665 | |
- Accrued Expenses
Accrued expenses for the periods presented are comprised of the following (in thousands):
| | | | | | | |
|---|---|---|---|---|---|---|
| · September 30, · December 31, | ||||||
| · · 2025 · · 2024 | ||||||
| Professional fees | | $7,235 | | $6,326 | ||
| Research and development | | 3,793 | | 2,234 | ||
| Manufacturing costs | | 3,691 | | 1,540 | ||
| Interest on Tranche 1 Notes | | | 2,747 | | | — |
| Compensation and benefits | | | 2,708 | | | 11,197 |
| Clinical trial costs | | | 1,295 | | | 3,864 |
| Consulting | | 1,221 | | 1,530 | ||
| Rent and facilities costs | | 236 | | 257 | ||
| Fixed assets | | 212 | | 326 | ||
| Other | | | 734 | | 140 | |
| | | $23,872 | | $27,414 | ||
- 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 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.
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 December 31, 2024 and for the nine-month period ended September 30, 2025 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, 2024 | | 8,232,587 | | $12.57 | | 5.35 | years | ||
| Granted | | 222,050 | | $6.07 | | | | ||
| Exercised | | (8,464) | | $5.48 | | | | ||
| Forfeited | | (481,808) | | $15.01 | | | | ||
| Outstanding at September 30, 2025 | | 7,964,365 | | $12.26 | | | 4.86 | years | |
| Options exercisable at September 30, 2025 | | 7,040,004 | | $12.76 | | 4.47 | years | ||
| Options vested and expected to vest at September 30, 2025 | | | 7,964,365 | | $12.26 | | | 4.86 | years |
| Aggregate intrinsic value of options outstanding as of September 30, 2025 | | $4,647 | | | | ||||
| Aggregate intrinsic value of options exercisable as of September 30, 2025 | | $3,779 | | | | ||||
Options granted under the Plans have a maximum contractual term often 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 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 three-month and nine-month periods ended September 30, 2025 and 2024 (in thousands):
| | | | | | | |
|---|---|---|---|---|---|---|
| · · Three-Month Periods Ended September 30, | ||||||
| · 2025 · 2024 | ||||||
| Research and development | | $857 | | $1,450 | ||
| General and administrative | | | 472 | | | 971 |
| Total share-based compensation | | $1,329 | | $2,421 | ||
| · · · · · · | |||||
|---|---|---|---|---|---|
| · · Nine-Month Periods Ended September 30, | |||||
| · 2025 · 2024 | |||||
| Research and development | | $2,639 | | $4,665 | |
| General and administrative | | 1,569 | | | 3,048 |
| Total share-based compensation | | $4,208 | | $7,713 | |
The total fair value of options vested during the three-month periods ended September 30, 2025 and 2024 was $1.3 million and $2.3 million, respectively.
The total fair value of options vested during the nine-month periods ended September 30, 2025 and 2024 was $4.7 million and $9.8 million, respectively.
The weighted-average grant date fair value of options granted during the nine-month period ended September 30, 2025 and 2024 was $3.85 and $3.91 per share, 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:
| · · · · | ||
|---|---|---|
| | 2025 | 2024 |
| Risk-free interest rate | 3.81 - 4.13% | 4.04 - 4.17% |
| Expected volatility | 67% | 67% |
| Expected dividend yield | 0% | 0% |
| Expected term (in years) | 6.01 | 3.6 - 6.1 |
As of September 30, 2025, the total compensation expense relating to unvested options granted that had not yet been recognized was $4.7 million, which is expected to be realized 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 as of December 31, 2024 and for the nine-month period ended September 30, 2025 is as follows:
| · · · · · · | |||||
|---|---|---|---|---|---|
| · · · Weighted- | |||||
| · · Number of · · Average | |||||
| · · Restricted · · Grant Date | |||||
| · · Share Units · · Fair Value | |||||
| Outstanding at December 31, 2024 | | 4,252,250 | | $8.91 | |
| Granted | | 3,174,500 | | $6.17 | |
| Vested | | | (1,222,500) | | $11.52 |
| Forfeited | | | (22,000) | | $6.15 |
| Outstanding at September 30, 2025 | | 6,182,250 | | $7.00 | |
RSUs granted generally vest 50% on the second anniversary of the date of grant and 25% on the third andfourth anniversaries of the date of grant. Annual RSUs granted to directors generally vest in a single installment on the earliest to occur of the first anniversary of the grant date or the day immediately prior to the date of the next annual meeting of the Company’s shareholders occurring after the date of grant. The RSUs granted to the directors in June 2021 will be paid on or within 30 days after the date a director ceases to serve on the board. For RSUs granted in June 2022 and future years, the directors may annually elect whether to defer the payment of their annual RSU awards under the Deferred Compensation Plan for Non-Employee Directors, which was adopted by the board on December 17, 2021. During the nine months ended September 30, 2025,225,000 RSUs granted to Directors vested and payment of shares was deferred. As of September 30, 2025, there were 497,500 vested shares that have been deferred and are excluded from ordinary shares outstanding. The related share-based compensation expense, which is recognized ratably over the requisite service period, is included in general and administrative and research and development expenses, as applicable, in the condensed consolidated statements of operations and comprehensive loss.
The Company recorded the following share-based compensation expense in connection with the RSUs for the three-month and nine-month periods ended September 30, 2025 and 2024 (in thousands):
| | | | | | | |
|---|---|---|---|---|---|---|
| · · Three-Month Periods Ended September 30, | ||||||
| · 2025 · 2024 | ||||||
| Research and development | | $1,367 | | $1,292 | ||
| General and administrative | | | 3,256 | | | 2,591 |
| Total share-based compensation | | $4,623 | | $3,883 | ||
| · · · · · · | |||||
|---|---|---|---|---|---|
| · · Nine-Month Periods Ended September 30, | |||||
| · 2025 · 2024 | |||||
| Research and development | | $3,723 | | $3,785 | |
| General and administrative | | 8,573 | | | 7,576 |
| Total share-based compensation | | $12,296 | | $11,361 | |
As of September 30, 2025, the total compensation expense relating to unvested RSUs granted that had not yet been recognized was $29.1 million, which is expected to be realized over a period of 3.5 years.
To satisfy employee minimum statutory tax withholding requirements for restricted share units that vest, the Company withholds a portion of the vested ordinary shares. During the three months ended September 30, 2025 and 2024, the Company withheld 28,501 and 28,500 ordinary shares with a total value of $0.2 million and $0.1 million, respectively. During the nine months ended September 30, 2025 and 2024, the Company withheld 433,960 and 377,152 ordinary shares with a total value of approximately $3.0 million and $2.3 million, respectively. These
amounts are presented as a cash outflow from financing activities in the accompanying condensed consolidated statement of cash flows.
During the three-month and nine-month periods ended September 30, 2025 and 2024, the Company recognized total share-based compensation expense in the accompanying condensed consolidated statements of operations and comprehensive loss as follows (in thousands):
| · · · · · · | |||||
|---|---|---|---|---|---|
| · · Three-Month Periods Ended September 30, | |||||
| · 2025 · 2024 | |||||
| Research and development | | $2,224 | | $2,742 | |
| General and administrative | | 3,728 | | | 3,562 |
| Total share-based compensation | | $5,952 | | $6,304 | |
| · · · · · · | |||||
|---|---|---|---|---|---|
| · · Nine-Month Periods Ended September 30, | |||||
| · 2025 · 2024 | |||||
| Research and development | | $6,362 | | $8,450 | |
| General and administrative | | 10,142 | | | 10,624 |
| Total share-based compensation | | $16,504 | | $19,074 | |
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 toexcess tax benefits have been reported in cash flows from operations or cash flows from financing activities for the nine-month periods ended September 30, 2025 and 2024.
- Ordinary Shares
At-the-Market
In December 2023, the Company entered into an“at-the-market” sales agreement with BofA Securities, Inc., or BofA, pursuant to which the Company may sell from time to time, ordinary shares having an aggregate offering price of up to$100.0 million through BofA, acting as the Company’s agent. During the nine-month period ended September 30, 2025, the Company raised gross proceeds of$9.9 million through the sale of 1,510,300 ordinary shares pursuant to an “at-the-market” equity offering program. Under the“at-the-market” equity program which is currently effective and may remain available for the Company to use in the future, the Company may sell an additional$81.8 million of ordinary shares. Whether the Company chooses to affect future sales under the“at-the-market” equity offering program will depend on a number of factors, including, among others, market conditions and the trading price of the Company’s ordinary shares relative to other sources of capital.
- Income Taxes
The Company didnot record a provision for income taxes for the three-month and nine-month periods ended September 30, 2025 and 2024, as the Company has generated losses for all periods.
The Company periodically evaluates the realizability of its deferred tax assets based on all available evidence, both positive and negative. The realization of deferred tax assets is dependent on the Company’s ability to generate sufficient future taxable income during periods prior to the expiration of tax attributes to fully utilize these assets. The Company weighed both positive and negative evidence and determined that there is a continued need for a full valuation allowance on its deferred tax assets (after consideration of the reversal of the deferred tax liabilities for the ROU assets and fixed assets) in the United States, United Kingdom, Ireland and Netherlands as of September 30, 2025. Should the Company determine that it would be able to realize its remaining deferred tax assets in the foreseeable future, an adjustment to its remaining deferred tax assets would cause a material increase to income in the period such determination is made.
- Related-Party Transactions
Asset Purchase and Related Agreements with Johnson & Johnson Innovative Medicine
On December 20, 2023, the Company entered into the Asset Purchase Agreement with Johnson & Johnson Innovative Medicine pursuant to which the Company sold and assigned to Johnson & Johnson Innovative Medicine, and Johnson & Johnson Innovative Medicine 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 Johnson & Johnson Innovative Medicine also entered into a Supply Agreement pursuant to which the Company agreed to manufacture and supply the RPGR Product for Johnson & Johnson Innovative Medicine. Under the Supply Agreement, MeiraGTx UK II, together with its affiliates, will manufacture commercial supply of the RPGR Product for Johnson & Johnson Innovative Medicine for an initial term offour years, with Johnson & Johnson Innovative Medicine having an option to extend the Supply Agreement for a fifth year upon written notification. Johnson & Johnson Innovative Medicine may terminate the Supply Agreement for convenience upon 90 days’ written notice with payment of a termination fee. Under the Asset Purchase Agreement, Johnson & Johnson Innovative Medicine 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; (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 Johnson & Johnson Innovative Medicine; and (vii) $10.0 million upon regulatory approval of a Johnson & Johnson Innovative Medicine-selected manufacturing facility in each of the United States and European Union for commercial manufacture of the RPGR Product. To date, the Company received $60.0 million in milestone payments from Johnson & Johnson Innovative Medicine. Johnson & Johnson Innovative Medicine 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 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 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 net of future billings, 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 Johnson & Johnson Innovative Medicine in the amount of $53.8 million.
Since the commencement of the Asset Purchase Agreement and related agreements, the Company received a $50.0 million milestone payment in connection with the achievement of the initiation of the extension study for the Phase 3 LUMEOS clinical trial for the RPGR Product. The milestone payment was allocated to the four performance
obligations on the same basis noted above increasing the value of each performance obligation as follows: PPQ services in the amount of $1.6 million, material rights representing the commercial supply of RPGR Product and an in-substance contract renewal option in the amount of $3.8 million, manufacturing technology transfer in the amount of $15.6 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 Johnson & Johnson Innovative Medicine in the amount of $29.0 million. Additionally, the Company received a $10.0 million milestone payment in connection with the completion of certain specified development services for the drug substance for the RPGR Product which was allocated to the PPQ services performance obligation. The Company also entered into additional agreements to provide additional services under the Asset Purchase Agreement and related agreements amounting to an aggregate of $5.8 million which was included in the transaction price and allocated to each of the respective performance obligations. Furthermore, the Company recorded certain changes in estimates related to the valuation of its performance obligations.
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 Johnson & Johnson Innovative Medicine exercises its option to purchase supply and the Company transfers control of such supply to Johnson & Johnson Innovative Medicine. The transaction price allocated to the in-substance renewal option (material right) will be recorded as deferred revenue until Johnson & Johnson Innovative Medicine exercises the option and the Company transfers control of the underlying goods or services to Johnson & Johnson Innovative Medicine. 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 nine-month period ended September 30, 2024, the Company recognized a gain of $28.4 million related to the sale of nonfinancial assets which is included in other income in the condensed consolidated statements of operations and comprehensive loss. There wasno gain on sale of nonfinancial assets recognized during the nine-month period ended September 30, 2025.
As of September 30, 2025, the aggregate transaction price allocated to unsatisfied performance obligations was $67.0 million which the Company expects to recognize over an estimated period of approximately 2.3 years.
A summary of the deferred revenue is as follows (in thousands):
| | | | |
|---|---|---|---|
| Deferred revenue at December 31, 2024 | | $62,403 | |
| Other amounts collected or invoiced | | | 1,765 |
| Deferred revenue recognized as service revenue during the nine-month period ended September 30, 2025 | | | (2,465) |
| Effects of exchange rate changes | | | 5,249 |
| Deferred revenue at September 30, 2025 | | $66,952 |
During the three-month periods ended September 30, 2025 and 2024, the Company recognized $0.2 million and $1.8 million, respectively, of deferred revenue – related party as service revenue in connection with PPQ services under the Asset Purchase Agreement and related agreements.
During the nine-month periods ended September 30, 2025 and 2024, the Company recognized $2.5 million and $2.8 million, respectively, of deferred revenue – related party as service revenue in connection with PPQ services under the Asset Purchase Agreement and related agreements.
During the three-month periods ended September 30, 2025 and 2024, the Company recognized $0.4 million and $10.9 million of service revenue, inclusive of the $0.2 million and $1.8 million, respectively, of deferred revenue – related party recognized as service revenue, based on cumulative progress of PPQ services under the Asset Purchase Agreement and related agreements.
During the nine-month periods ended September 30, 2025 and 2024, the Company recognized $6.0 million and $11.9 million of service revenue, inclusive of the $2.5 million and $2.8 million, respectively, of deferred revenue – related party recognized as service revenue, based on cumulative progress of PPQ services under the Asset Purchase Agreement and related agreements.
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 greater than 10% 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 10 for further information related to the accounting for the debt financing.
- 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 under these leases was $1.3 million and $1.4 million for the three-month periods ended September 30, 2025 and 2024, respectively.
Total rent expense under these leases was $3.7 million and $4.3 million for the nine-month periods ended September 30, 2025 and 2024, respectively.
The Company entered into a lease agreement for laboratory space in March 2025 with an initial lease term of 91 months. The Company also entered into a lease agreement for office space in July 2025 with an initial lease term of 39 months. The lease for the laboratory space contains provisions for tenant allowances and future rent increases. The Company took possession of both spaces in August 2025 and recognized initial operating lease right-of-use assets in the amount of $8.2 million and corresponding lease liabilities in the amount of $8.0 million, in connection with these leases, which are included in the right-of-use assets and lease obligations in the condensed consolidated balance sheets as of September 30, 2025. The lease liabilities have been measured using the incremental borrowing rate (“IBR”) rather than the rate implicit in the lease agreements, as the implicit rates could not be readily determined. The IBR applied to both leases reflects the parent company’s borrowing rate as the Company considers it to be a more appropriate reflection of the financing rate that would be available to the consolidated group, rather than the rates specific to the subsidiary that is the lessee under the agreements. There wereno leases recognized during the nine-month period ended September 30, 2024. During the nine months ended September 30, 2025, the Company determined that it was reasonably certain to exercise its early termination option on an operating lease for laboratory and office space, resulting in a remeasurement of the ROU asset and lease liability reflected in the accompanying condensed consolidated balance sheets. The lease was terminated August 31, 2025.
Four clinical trial site leases were assigned to Johnson & Johnson Innovative Medicine during the three-month period ended September 30, 2024 in connection with the Asset Purchase Agreement and related agreements.
The components of lease cost for the three-month and nine-month periods ended September 30, 2025 and 2024 are as follows (in thousands):
| · · · · · · | ||||
|---|---|---|---|---|
| · · Three-Month Periods Ended September 30, | ||||
| | | 2025 | | 2024 |
| Finance lease cost · · | ||||
| Amortization of right-of-use assets | | $304 | | $218 |
| Total finance lease cost | | 304 | | 218 |
| Operating lease cost | | 1,276 | | 1,428 |
| Short-term lease cost | | 103 | | 74 |
| Total lease cost | | $1,683 | | $1,720 |
| · · · · · · | ||||
|---|---|---|---|---|
| · · Nine-Month Periods Ended September 30, | ||||
| | | 2025 | | 2024 |
| Finance lease cost · · · | ||||
| Amortization of right-of-use assets | | $891 | | $778 |
| Total finance lease cost | | 891 | | 778 |
| Operating lease cost | | 3,673 | | 4,300 |
| Short-term lease cost | | 298 | | 162 |
| Total lease cost | | $4,862 | | $5,240 |
Amounts reported in the condensed consolidated balance sheets for leases where the Company is the lessee as of September 30, 2025 and December 31, 2024 were as follows (in thousands):
| · · · · · · · · | ||||||
|---|---|---|---|---|---|---|
| · · September 30, · December 31, | ||||||
| · · 2025 · · 2024 · | ||||||
| Operating leases · · · · · | ||||||
| Right-of-use assets | | $13,064 | | | $10,576 | |
| Capitalized lease obligations | | $14,006 | | | $11,576 | |
| Finance leases | | | | | ||
| Right-of-use assets | | $23,802 | | | $22,198 | |
| Weighted-average remaining lease term | | | | | ||
| Operating leases | | 6.6 | years | | 3.6 | years |
| Finance leases | | 173.2 | years | | 173.9 | years |
| Weighted-average discount rate | | | | | | |
| Operating leases | | 11.2% | | 8.8% | ||
| Finance leases | | 8.0% | | 8.0% | ||
Other information related to leases for the three-month and nine-month periods ended September 30, 2025 and 2024 are as follows (in thousands):
| · · · · · | |||
|---|---|---|---|
| | Three-Month Periods Ended September 30, | ||
| | 2025 | | 2024 |
| Cash paid for amounts included in the measurement of lease liabilities | |||
| Operating cash flows from operating leases | $955 | | $1,329 |
| · · · · · | |||
|---|---|---|---|
| | Nine-Month Periods Ended September 30, | ||
| | 2025 | | 2024 |
| Cash paid for amounts included in the measurement of lease liabilities | |||
| Operating cash flows from operating leases | $3,412 | | $4,180 |
Future minimum lease payments under non-cancellable leases as of September 30, 2025 are as follows (in thousands):
| | | | |
|---|---|---|---|
| · Operating Leases | |||
| 2025 | | $573 | |
| 2026 | | 4,211 | |
| 2027 | | 3,697 | |
| 2028 | | 3,479 | |
| 2029 | | | 2,395 |
| Thereafter | | 6,051 | |
| Total undiscounted lease payments | | $20,406 | |
| Less: Imputed interest | | (6,400) | |
| Total lease liabilities | | $14,006 | |
10. 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 “Second Consent and Amendment”).
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 was able to request in its sole discretion, and Perceptive 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 aone-month tenor, subject to a 1.00% floor. The annual interest rate was 14.33% at September 30, 2025. As of September 30, 2025, the outstanding balance of the Tranche 1 Notes was $75.0 million plus accrued interest of $2.7 million. During the three-month periods ended September 30, 2025 and 2024, the Company recorded interest expense of $2.7 million and $2.9 million, respectively. During the nine-month periods ended September 30, 2025 and 2024, the Company recorded interest expense of $8.2 million and $8.8 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. As of September 30, 2025, the Company is in compliance with all covenants.
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 $0.3 million of the discount to interest expense during each of the three-month periods ended September 30, 2025 and 2024. The Company amortized $0.8 million of the discount to interest expense during each of the nine-month periods ended September 30, 2025 and 2024, respectively.
11. Commitments and Contingencies
There wereno new material commitments or contingencies entered into during the nine-month period ended September 30, 2025.
12. Subsequent Event
On November 7, 2025 (the “Effective Date”), Meira Ocular, MeiraGTx Limited and Meira UK II (collectively, “Meira”), entered into a strategic collaboration and license agreement with Eli Lilly and Company (“Lilly”)(the “Lilly Collaboration Agreement”) for the research, development and commercialization of genetic medicines inand related tothe area of ophthalmology. Under the Lilly Collaboration Agreement, Meirahas granted Lilly exclusive, worldwide rights to research, develop and commercialize the Company’s product candidate AAV-AIPL1, which treats Leber congenital amaurosis 4, or LCA4, caused by mutations in theAIPL1 gene, as well as two other preclinical product candidates which are intended to treat other inherited retinal dystrophies. As of the Effective Date, Lilly has (i) an exclusive license to proprietary intravitreal capsids for use with up to five targets, relating to or useful in the field of ophthalmology, to be selected by Lilly, (ii) an exclusive license to proprietary pan-retinal or rod-specific promoters for use with up to five targets, relating to or useful in the field of ophthalmology, to be selected by Lilly and (iii) a right of first designation with respect to certain target-specific transactions that MeiraGTx Ocular or its affiliates may seek to pursue in the field of ophthalmology. Lilly also has a right of first negotiationfor use of the Company’s proprietary riboswitch technology in the field of ophthalmological gene editing.
Under the terms of the Lilly Collaboration Agreement, Meira will receive an upfront payment of $75.0 million after signing the Lilly Collaboration Agreement and will be eligible to receive up to over $400.0 million in total milestone payments, including up to $135.0 million in other potential near-term cash consideration upon the achievement of certain development and regulatory approval milestones. Lilly will have the right to research, develop and commercialize products under the Lilly Collaboration Agreement, at its cost. The Lilly Collaboration Agreement also provides for tiered royalties to be paid to MeiraGTx Ocular.
The text of this filing, as the company filed it with the SEC, without its images or exhibits. This is not investment advice.