CONTENTS
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| PART I | 4 | |
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| Item 1. | Business | 4 |
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| Item 1A. | Risk Factors | 50 |
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| Item 1B. | Unresolved Staff Comments | 109 |
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| Item 1C. | Cybersecurity | 109 |
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| Item 2. | Properties | 111 |
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| Item 3. | Legal Proceedings | 111 |
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| Item 4. | Mine Safety Disclosures | 111 |
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| PART II | 112 | |
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| Item 5. | Market For Registrant’s Common Equity, Related Stockholder Matters And Issuer Purchases Of Equity Securities | 112 |
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| Item 6. | [Reserved] | 112 |
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| Item 7. | Management’s Discussion And Analysis Of Financial Condition And Results Of Operations | 112 |
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| Item 7A. | Quantitative And Qualitative Disclosures About Market Risk | 131 |
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| Item 8. | Financial Statements And Supplementary Data | F-1 |
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| Item 9. | Changes In And Disagreements With Accountants On Accounting And Financial Disclosure | 124 |
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| Item 9A. | Controls And Procedures | 124 |
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| Item 9B. | Other Information | 125 |
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| Item 9C. | Disclosure Regarding Foreign Jurisdictions that Prevent Inspections | 125 |
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| PART III | 126 | |
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| Item 10. | Directors, Executive Officers And Corporate Governance | 126 |
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| Item 11. | Executive Compensation | 126 |
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| Item 12. | Security Ownership Of Certain Beneficial Owners And Management And Related Stockholder Matters | 126 |
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| Item 13. | Certain Relationships And Related Transactions, And Director Independence | 127 |
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| Item 14. | Principal Accountant Fees And Services | 127 |
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| PART IV | 128 | |
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| Item 15. | Exhibits and Financial Statement Schedules | 128 |
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SPECIAL NOTE REGARDING FORWARD-LOOKING STATEMENTS
This Annual Report on Form 10-K (the “Form 10-K”) contains forward-looking statements that can involve substantial risks and uncertainties. All statements other than statements of historical facts contained in this Form 10-K, including, but not limited to, statements regarding our future results of operations and financial position, business strategy, financing, licensing and manufacturing arrangements, prospective products, development and timing of product candidates, timing of and expected success and efficacy of our product candidates, plans and objectives for future operations, expected future results of anticipated products and prospects, and plans and objectives of management are forward-looking statements. These statements involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements.
In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential,” “would” or “continue” or the negative of these terms or other similar expressions. The forward-looking statements in this Form 10-K are only predictions. We have based these forward-looking statements largely on our current expectations and projections about future events and financial trends that we believe may affect our business, financial condition and results of operations. These forward-looking statements speak only as of the date of this Form 10-K and are subject to a number of risks, uncertainties and assumptions described under the sections in this Form 10-K entitled “Item 1A. Risk Factors” and “Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations” and elsewhere in this Form 10-K. Because forward-looking statements are inherently subject to risks and uncertainties, some of which cannot be predicted or quantified and some of which are beyond our control, you should not rely on these forward-looking statements as predictions of future events. The events and circumstances reflected in our forward-looking statements may not be achieved or occur and actual results could differ materially from those projected in the forward-looking statements. Moreover, we operate in an evolving environment. New risk factors and uncertainties may emerge from time to time, and it is not possible for management to predict all risk factors and uncertainties. Except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. T**hus, one should not assume that our silence over time means that actual events are bearing out as expressed or implied in such forward-looking statements.
You should read this Form 10-K and the documents that we reference in this Form 10-K and have filed as exhibits to this Form 10-K, completely and with the understanding that our actual future results may be materially different from what we expect.
*In addition, statements that “we believe” and similar statements reflect our beliefs and opinions on the relevant subject. These statements are based upon information available to us as of the date of this Form 10-K, and while we believe such information forms a reasonable basis for such statements, such information may be limited or incomplete, and our statements should not be read to indicate that we have conducted an exhaustive inquiry into, or review of, all relevant information. These statements are inherently uncertain and investors are cautioned not to unduly rely upon these statements.*These statements should not be relied upon as representing our views as of any date subsequent to the date of this Form 10-K.
RISK FACTOR SUMMARY
We are providing the following summary of the principal risk factors contained in this Form 10-K to enhance the readability and accessibility of our risk factor disclosures. We encourage you to carefully review in their entirety the full risk factors set forth in the section of this Form 10-K captioned “Item 1A. Risk Factors”* *for additional information regarding the material factors that make an investment in our ordinary shares speculative or risky. These risks and uncertainties include, among others, the following:
| ● | We have incurred significant losses since inception and anticipate that we will incur continued losses for the foreseeable future, and may never achieve or maintain profitability. |
| ● | There is no guarantee that we will receive in a timely fashion or at all the additional milestone payments contemplated under the Asset Purchase Agreement or the revenues associated with our manufacture of the commercial supply of the RPGR Product under the Supply Agreement. |
| ● | We will require additional capital to fund our operations, which may not be available on acceptable terms, if at all. |
| ● | We may not have sufficient cash flows or cash on hand to satisfy our debt obligations or covenants under our financing arrangements, or we may not be able to effectively manage our business in compliance with such covenants. |
| ● | Our review of potential strategic transactions may not result in an executed or consummated transaction or other strategic alternative and may not result in anticipated benefits to us or our shareholders, and the process of reviewing strategic transactions or its conclusion could be disruptive and distracting to our business operations and management. |
| ● | We are heavily dependent on the success of our product candidates, which are still in development, and if none of them receive regulatory approval or are successfully commercialized, our business may be harmed. |
| ● | It is difficult to predict the time and cost of product candidate development on our novel gene therapy platform. Very few gene therapies have been approved in the United States or in Europe. |
| ● | Because gene therapy is novel and the regulatory landscape that governs any product candidates we may develop is uncertain and may change, we cannot predict the time and cost of obtaining regulatory approval, if we receive it at all, for any product candidates we may develop. |
| ● | Clinical trials are expensive, time-consuming, difficult to design and implement, and involve an uncertain outcome. Further, we may encounter substantial delays in our clinical trials. |
| ● | The affected populations for our product candidates may be smaller than we or third parties currently project, which may affect the addressable markets for our product candidates. |
| ● | We and our contract manufacturers for plasmid are subject to significant regulation with respect to manufacturing our products. Our manufacturing facilities and the third-party manufacturing facilities which we rely on may not continue to meet regulatory requirements and have limited capacity. |
| ● | Enacted and future healthcare legislation may increase the difficulty and cost for us to obtain marketing approval of and commercialize our product candidates and may affect the prices we may set. |
| ● | We are subject to regulation and other legal obligations relating to data privacy and protection. Compliance with these requirements is complex and costly. The actual or perceived failure to comply with such obligations could materially harm our business, results of operations, and financial condition.. |
| ● | We face significant competition in an environment of rapid technological change, and there is a possibility that our competitors may achieve regulatory approval before us or develop therapies that are safer or more advanced or effective than ours, which may harm our financial condition and our ability to successfully market or commercialize any product candidates we may develop. |
| ● | We depend on proprietary technology licensed from others. If we lose our existing licenses or are unable to acquire or license additional proprietary rights from third parties, we may not be able to continue developing our product candidates. |
| ● | If we are unable to obtain and maintain patent protection for our technology and product candidates or if the scope of the patent protection obtained is not sufficiently broad, we may not be able to compete effectively in our markets. |
| ● | We may need to expand our organization, and we may experience difficulties in managing this growth, which could disrupt our operations. |
| ● | Our future success depends on our ability to retain our key personnel and to attract, retain and motivate qualified personnel. |
BASIS OF PRESENTATION
Unless the context otherwise requires, references in this Form 10-K to “Meira,” “MeiraGTx,” “we,” “us”, “our” or “the Company” refer to MeiraGTx Holdings plc and its subsidiaries.
We have proprietary rights to trademarks, trade names and service marks appearing in this Form 10-K that are important to our business. Solely for convenience, the trademarks, trade names and service marks may appear in this Form 10-K without the® and TM symbols, but any such references are not intended to indicate, in any way, that we forgo or will not assert, to the fullest extent under applicable law, our rights or the rights of the applicable licensors to these trademarks, trade names and service marks. All trademarks, trade names and service marks appearing in this Form 10-K are the property of their respective owners.
INDUSTRY AND OTHER DATA
We obtained the industry, market and competitive position data in this Form 10-K from our own internal estimates and research as well as from industry and general publications and research, surveys and studies conducted by third parties. Industry publications, studies and surveys generally state that they have been obtained from sources believed to be reliable, although they do not guarantee the accuracy or completeness of such information. While we believe that each of these studies and publications is reliable, we have not independently verified market and industry data from third-party sources. While we believe our internal company research as to such matters is reliable and the market definitions are appropriate, neither such research nor these definitions have been verified by any independent source.
