MeiraGTx Reports Third Quarter 2022 Financial and Operational Results and Receives $25 Million Investment from Johnson & Johnson Innovation - JJDC, Inc. -- Updated Positive Topline Data from the Phase 1/2 Trial of Botare

MeiraGTx Reports Third Quarter 2022 Financial and Operational Results and Receives $25 Million Investment from Johnson & Johnson Innovation - JJDC, Inc.

-- Updated Positive Topline Data from the Phase 1/2 Trial of Botaretigene Sparoparvovec (AAV-RPGR) for the Treatment of X-linked Retinitis Pigmentosa (XLRP)

-- Now Dosing Patients in Phase 1 Trial Evaluating AAV-GAD Gene Therapy for Parkinson's Disease

-- Received $25.0 Million Investment from Johnson & Johnson Innovation - JJDC, Inc. ("JJDC"), the Investment Arm of Johnson and Johnson

-- Company to Provide Clinical Program Update for the Phase 1 AQUAx Trial of AAV-hAQP1 for the Treatment of Grade 2/3 Radiation-Induced Xerostomia in December 2022

LONDON and NEW YORK, November 10, 2022 -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical-stage gene therapy company, today announced financial results for the third quarter ended September 30, 2022, and provided an update on recent progress.

"As we head into year-end, we continue to make progress in advancing our clinical and preclinical pipeline," said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. "This includes presenting data from our Phase 1/2 clinical study in XLRP at AAO, further demonstrating botaretigene sparoparvovec's potential to improve vision in patients with retinitis pigmentosa. We are currently dosing patients in the Phase 3 LUMEOS trial and remain on track for a BLA in 2024. We are also pleased with the $25 million investment from JJDC which will help us to further our programs and continue development of our internal capabilities."

"In addition, we are now dosing patients with adeno-associated virus (AAV) encoding glutamic acid decarboxylase (AAV-GAD), an investigational gene therapy product candidate for Parkinson's disease'" Dr. Forbes continued. "And in our xerostomia program, we look forward to presenting updated data from our Phase 1 AQUAx trial later this year."

"We are also very excited by the progress of our technology and manufacturing platforms", stated Dr. Forbes. "We published 15 abstracts at ESGCT in October this year, highlighting the robustness of our riboswitch gene regulation technology for gene and cell therapies, as well as the depth of our proprietary promoter and manufacturing and process development platforms."

Recent Highlights and Anticipated Milestones

Botaretigene Sparoparvovec for the Treatment of XLRP:

AAV-hAQP1 for the Treatment of Grade 2/3 Radiation-Induced Xerostomia:

AAV-GAD for the Treatment of Parkinson's Disease:

Riboswitch Gene Control Platform:

Gene Therapy Manufacturing:

In addition to the $25.0 million investment from JJDC, the Company had cash and cash equivalents of approximately $114.7 million as of September 30, 2022, as well as approximately $23.7 million in receivables due from Janssen from the third quarter of 2022. The Company believes it will have sufficient capital to fund operating expenses and capital expenditure requirements into the fourth quarter of 2024.

License revenue was $4.8 million for the three months ended September 30, 2022, compared to $6.9 million for the three months ended September 30, 2021. This decrease represents decreased amortization of the $100.0 million upfront payment from Janssen as well as amortization of the $30.0 million milestone payment received in connection with the Janssen Collaboration Agreement.

General and administrative expenses were $10.8 million for the three months ended September 30, 2022, compared to $7.9 million for the three months ended September 30, 2021. The increase of $2.9 million is primarily due to an increase of $0.9 million in consulting fees, $0.7 million in rent and facilities costs due to additional allocations to research and development, $0.6 million in legal and accounting fees, $0.6 million in share-based compensation, $0.3 million in payroll and payroll-related costs and $0.1 million in depreciation. These increases were partially offset by a decrease of $0.3 million in insurance costs.

Research and development expenses for the three months ended September 30, 2022, were $16.9 million, compared to $21.6 million for the three months ended September 30, 2021. The decrease of $4.7 million is primarily due to a decrease of $0.6 million in license fees, $0.4 million in rent and facilities costs, $0.9 million reduction of the estimated research and development tax credit refund and an increase of $7.1 million in research funding provided under the Collaboration Agreement with Janssen. These decreases were partially offset by an increase of $1.3 million in payroll and payroll-related costs, $1.0 million in share-based compensation, $1.0 million in costs related to the manufacture of material for the Company's clinical trials and $1.0 million in costs related to the Company's pre-clinical research and clinical trials.

Foreign currency loss was $12.8 million for the three months ended September 30, 2022, compared to a loss of $3.4 million for the three months ended September 30, 2021. The increase in the loss of $9.4 million was primarily due to an unrealized loss on the quarterly valuation of the Company's intercompany payables and receivables due to the strengthening of the U.S. dollar against the pound sterling and euro during the three months ended September 30, 2022.

Net loss attributable to ordinary shareholders for the quarter ended September 30, 2022, was $37.3 million, or $0.83 basic and diluted net loss per ordinary share, compared to a net loss attributable to ordinary shareholders of $25.9 million, or $0.59 basic and diluted net loss per ordinary share for the quarter ended September 30, 2021.

MeiraGTx (Nasdaq: MGTX) is a vertically integrated, clinical-stage gene therapy company with six programs in clinical development and a broad pipeline of preclinical and research programs. MeiraGTx has core capabilities in viral vector design and optimization and gene therapy manufacturing, and a transformative gene regulation platform technology that allows precise, dose responsive control of gene expression by oral small molecules with dynamic range that can exceed 5000-fold. Led by an experienced management team, MeiraGTx has taken a portfolio approach by licensing, acquiring, and developing technologies that give depth across both product candidates and indications. MeiraGTx's initial focus is on three distinct areas of unmet medical need: ocular diseases, including both inherited retinal diseases as well as large degenerative ocular diseases, neurodegenerative diseases and severe forms of xerostomia. Though initially focusing on the eye, central nervous system, and salivary gland, MeiraGTx plans to expand its focus to develop additional gene therapy treatments for patients suffering from a range of serious diseases.

For more information, please visit www.meiragtx.com

Forward Looking Statement

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding our product candidate development and anticipated 2022 milestones regarding our pre-clinical and clinical data and reporting of such data and the timing of results of data, including in light of the COVID-19 pandemic, as well as statements that include the words "expect," "will," "intend," "plan," "believe," "project," "forecast," "estimate," "may," "could," "should," "would," "continue," "anticipate" and similar statements of a future or forward-looking nature. These forward-looking statements are based on management's current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, our incurrence of significant losses; any inability to achieve or maintain profitability, raise additional capital, repay our debt obligations, identify additional and develop existing product candidates, successfully execute strategic priorities, bring product candidates to market, expansion of our manufacturing facilities and processes, successfully enroll patients in and complete clinical trials, accurately predict growth assumptions, recognize benefits of any orphan drug designations, retain key personnel or attract qualified employees, or incur expected levels of operating expenses; the impact of the COVID-19 pandemic on the status, enrollment, timing

and results of our clinical trials and on our business, results of operations and financial condition; failure of early data to predict eventual outcomes; failure to obtain FDA or other regulatory approval for product candidates within expected time frames or at all; the novel nature and impact of negative public opinion of gene therapy; failure to comply with ongoing regulatory obligations; contamination or shortage of raw materials or other manufacturing issues; changes in healthcare laws; risks associated with our international operations; significant competition in the pharmaceutical and biotechnology industries; dependence on third parties; risks related to intellectual property; changes in tax policy or treatment; our ability to utilize our loss and tax credit carryforwards; litigation risks; and the other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2022, as such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SEC's website at www.sec.gov. These and other important factors could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management's estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, unless required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Thus, 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. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release.

1 Michaelides, M et al. Ph1/2 AAV5-RPGR (Botaretigene Sparoparvovec) Gene Therapy Trial in RPGR-associated X-linked Retinitis Pigmentosa (XLRP). Abstract #30071754. Presented at the 2022 American Academy of Ophthalmology Annual Meeting.

LifeSci Communications

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