LONDON and NEW YORK, March 13, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage genetic medicines company, today announced financial and operational results for the fourth quarter and full-year ended December 31, 2024, and provided a corporate update.
“MeiraGTx demonstrated excellent execution in 2024, marked by significant advancements across each of our late stage clinical programs as well as our end-to-end manufacturing capabilities, achieving multiple positive clinical and regulatory milestones,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. “This extraordinary progress has continued in 2025 with today’s announcement of a strategic collaboration with Hologen which includes a $200 million cash upfront payment to MeiraGTx, and the formation of a joint venture, Hologen Neuro AI Ltd, with an additional $230 million committed capital from Hologen. The joint venture will initially be focused on increasing the robustness, efficiency and probability of success of the AAV-GAD Phase 3 clinical study. Hologen is a world-leading developer of multi-modal generative AI foundation models using large real-world clinical and research data sets from multiple modalities for clinical medicine and pharmaceutical drug development. The use of Hologen’s AI technology applied to MeiraGTx’s Phase 2 clinical data sets in Parkinson’s disease has already significantly de-risked the AAV-GAD program and identified disease modifying changes in the physiology of the brain in response to AAV-GAD treatment.”
Dr. Forbes continued, “In forming this joint venture, MeiraGTx and Hologen have created the first neuro-AI clinical drug development company in which pioneering technologies from both companies will be deployed to transform the discovery and development of therapies targeting CNS circuitry to advance the development of drugs with meaningful impact on neurodegenerative and neuropsychiatric diseases which have been largely intractable to effective treatment to date.”
“We have also achieved excellent progress with our pivotal stage AAV2-hAQP1 treatment for radiation-induced xerostomia (RIX),” stated Dr. Forbes. “AAV2-hAQP1 was granted Regenerative Medicine Advanced Therapy (RMAT) designation for Grade 2/3 RIX in December 2024 by the U.S. Food and Drug Administration (FDA), reinforcing the strength of our clinical data and its potential to significantly improve the lives of xerostomia patients. We anticipate data from the ongoing pivotal study, using material manufactured in-house at MeiraGTx, to support a potential BLA filing in 2026.”
“Turning to ophthalmology, we are incredibly excited to see the transformative effect of treatment with rAAV8.hRKp.AIPL1 in 100% of the young children who received this genetic medicine,” said Dr. Forbes. “LCA4 is one of the most severe forms of inherited blindness and the results from these 11 young children are truly remarkable and unrivalled in treatment benefit. With these truly exceptional clinical results in hand, along with our in-house manufacturing process, we anticipate filing with the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) for approval under exceptional circumstances, and taking a parallel path with the FDA and other global regulators this year in order to expedite access to this truly transformative medicine to LCA4 babies globally. We also received multiple regulatory designations granted to four different IRD programs in 2024, including rAAV8.hRKp.AIPL1 for LCA4.”
Dr. Forbes continued, “We accomplished a great deal in 2024, and we have started 2025 in a very strong position with a continuing commitment to drive innovation in genetic medicine, address the severe unmet needs of our patients with both rare and common diseases, and create value for our shareholders.”
Recent Development Highlights
Strategic Collaboration with Hologen AI:
AAV-GAD for the Treatment of Parkinson’s Disease:
AAV2-hAQP1 for the Treatment of Xerostomia:
AAV-AIPL1 Specials License in the UK:
An RPDD may be granted by the FDA to drugs and biologics intended to treat certain orphan diseases affecting fewer than 200,000 patients in the U.S., the serious or life-threatening manifestations of which primarily affect individuals aged 18 years or younger. Under the FDA’s Rare Pediatric Disease Priority Review Voucher (PRV) program, a sponsor that receives approval for a biologics license application for a rare pediatric disease may be eligible to receive a voucher for a priority review of a subsequent marketing application for a different product. PRVs may be used by the sponsor or sold to another sponsor for their use and have recently been sold for between $100 million to $158 million.
Botaretigene Sparoparvovec for the Treatment of XLRP:
Riboswitch Gene Regulation Technology Platform for in vivo Delivery:
Manufacturing:
United Kingdom (MeiraGTx UK II Ltd.)
MeiraGTx’s UK manufacturing facility holds two authorizations issued by the MHRA:
The UK facility was inspected in May 2024, and the licences were successfully renewed. The outcome of this inspection confirmed that the site was found to be in compliance with GMP requirements for Investigational Medicinal Products (IMPs) and was operating at the required compliance level to support an application for a commercial MIA licence. MeiraGTx plans to submit this application in the second quarter of 2025.
Ireland (MeiraGTx Ireland DAC)
MeiraGTx’s Shannon facility holds two authorizations issued by Ireland’s Health Products Regulatory Authority (HPRA):
The QC laboratory is actively undertaking release and stability testing on PPQ batches.
The latest HPRA inspection in February 2025 was highly successful—both QC licences were renewed, and viral vector manufacturing was added to the MIA(IMP) licence. This means the Shannon site can manufacture material for use in clinical trials, a first-of-its-kind licence for a gene therapy facility in Ireland.
Strategic Investment from Sanofi:
As of December 31, 2024, MeiraGTx had cash and cash equivalents of approximately $103.7 million, as well as $0.7 million in receivables due from Johnson & Johnson Innovative Medicine and $6.8 million in tax incentive receivables. The Company believes that with such funds, together with the proceeds from the anticipated closing of the strategic collaboration with Hologen, it will have sufficient capital to fund operating expenses and capital expenditure requirements into 2027 and to repay its debt obligation of $75.0 million to Perceptive Advisors (due in August 2026). This estimate does not include the $285.0 million in milestones the Company is eligible to receive under the asset purchase agreement upon first commercial sale of bota-vec in the United States and in at least one of the United Kingdom, France, Germany, Spain and Italy, for completion of the transfer of certain manufacturing technology to Janssen and upon regulatory approval of a Janssen-selected manufacturing facility in each of the United States and European Union for commercial manufacture of bota-vec.
For more information related to our clinical trials, please visit www.clinicaltrials.gov
Financial Results
Cash, cash equivalents and restricted cash were $105.7 million as of December 31, 2024, compared to $130.6 million as of December 31, 2023.
Service revenue was $33.3 million for the year ended December 31, 2024 due to the progress of process performance qualification services under the asset purchase agreement with Johnson & Johnson Innovative Medicine. There was no service revenue for the year ended December 31, 2023.
There was no license revenue for the year ended December 31, 2024, compared to $14.0 million for the year ended December 31, 2023. The decrease is due to the termination of the collaboration agreement concurrent with the execution of the asset purchase agreement with Johnson & Johnson Innovative Medicine.
Cost of service revenue was $23.8 million for the year ended December 31, 2024, due to progress of process performance qualification services under the asset purchase agreement with Johnson & Johnson Innovative Medicine. There was no cost of service revenue for the year ended December 31, 2023.
General and administrative expenses were $54.2 million for the year ended December 31, 2024, compared to $47.3 million for the year ended December 31, 2023. The increase of $6.9 million was primarily due to an increase in professional fees and various other general and administrative costs, none of which were individually significant.
Research and development expenses were $119.5 million for the year ended December 31, 2024, compared to $103.8 million for the year ended December 31, 2023. The increase of $15.7 million was primarily due to an increase in manufacturing costs, preclinical expenses and a reduction in reimbursements from Johnson & Johnson Innovative Medicine as the reimbursement for the year ended December 31, 2023 was in connection with research funding provided under the collaboration agreement, which was terminated on December 20, 2023. These increases were partially offset by a decrease in clinical trial expenses primarily related to bota-vec as Johnson & Johnson Innovative Medicine is now primarily funding the research and development related to this program as a result of the asset purchase agreement as well as a decrease in other research and development expenses.
Foreign currency loss was $2.9 million for the year ended December 31, 2024, compared to a gain of $9.3 million for the year ended December 31, 2023. The change of $12.2 million was primarily due to the restructuring and payment of certain intercompany receivables and payables during the year ended December 31, 2023. Foreign currency gains and losses subsequent to the restructuring are recorded as a part of accumulated other comprehensive income.
Interest income was $4.1 million for the year ended December 31, 2024, compared to $2.3 million for the year ended December 31, 2023. The increase was due to higher interest rates and cash balances during 2024.
Interest expense was $13.3 million for each of the years ended December 31, 2024 and 2023.
Gain on sale of nonfinancial assets was $28.4 million for the year ended December 31, 2024 compared to $54.2 million for the year ended December 31, 2023. This decrease was a result of a lower value of transaction consideration recognized in connection with the asset purchase agreement during the year ended December 31, 2024 compared to the year ended December 31, 2023.
Net loss attributable to ordinary shareholders for the year ended December 31, 2024, was $147.8 million, or $2.12 basic and diluted net loss per ordinary share, compared to a net loss attributable to ordinary shareholders of $84.0 million, or $1.49 basic and diluted net loss per ordinary share for the year ended December 31, 2023.
About MeiraGTx
MeiraGTx (Nasdaq: MGTX) is a vertically integrated, clinical-stage genetic medicines company with a broad pipeline with four late-stage clinical programs. Each of these programs use local delivery of small doses resulting in disease modifying effects in both inherited and more common diseases, in the eye, Parkinson’s disease and radiation-induced xerostomia. MeiraGTx uses its innovative technology in optimization of capsids, promoters and novel translational control elements to develop best in class, potent, safe viral vectors. MeiraGTx’s broad pipeline is supported by end-to-end in-house manufacturing. MeiraGTx has built the most comprehensive manufacturing capabilities in the industry, with 5 facilities globally, including two that are licensed for GMP viral vector production and a GMP QC facility with clinical and commercial licensure. In addition, MeiraGTx has developed a proprietary manufacturing platform process over 9 years based on more than 20 different viral vectors with leading yield and quality aspects and commercial readiness. Uniquely, MeiraGTx has developed a novel technology for in vivo delivery of any biologic therapeutic using oral small molecules. This transformative riboswitch gene regulation technology allows precise, dose-responsive control of gene expression by oral small molecules. MeiraGTx is focusing the riboswitch platform on the regulated in vivo delivery of metabolic peptides, including GLP-1, GIP, Glucagon, Amylin, PYY and Leptin, as well as cell therapy, CAR-T for liquid and solid tumors and autoimmune diseases, and additionally PNS targets addressing long term intractable pain. MeiraGTx has developed the technology to apply genetic medicine to common diseases, increasing efficacy, addressing novel targets, and expanding access in some of the largest disease areas where the unmet need remains high.
For more information, please visit www.meiragtx.com
About Hologen
Hologen Limited is a world-leading developer of generative AI capabilities for clinical medicine and pharmaceutical drug development. Hologen builds the largest, most expressive, accurate, and equitable generative AI models in healthcare, using large real-world clinical and research data sets from multiple modalities. Hologen’s Large Medical Models learn the rich biological diversity of healthy and pathological variation in unprecedented breadth and detail. By capturing complex biological heterogeneity with high fidelity, as revealed by clinical data, Hologen's technology overcomes the insensitivity of interventional trials, enabling accurate quantification of therapeutic effects, and illuminates disease mechanisms opaque to conventional models, revealing new therapeutic and commercial opportunities. In Phase 2 and Phase 3 trials, the technology is used to increase trial success probabilities substantially and to gain much greater control over trial design and approvability. The company emerged as a spin-out from University College London and Kings College London. It is privately held.
For more information, please visit www.hologen.ai
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 milestones regarding our pre-clinical and clinical data, reporting of such data and the timing of results of data and regulatory matters, statements regarding the collaboration with Hologen, including the anticipated timing for its closing and funding thereunder, the success of the activities to be performed under the collaboration, the efficacy of Hologen’s AI technology, the development of our AAV-GAD and other CNS product candidates and the development of our manufacturing technology, 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 transactions or 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 or rare pediatric disease designations, retain key personnel or attract qualified employees, or incur expected levels of operating expenses; the impact of pandemics, epidemics or outbreaks of infectious diseases 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 Annual Report on Form 10-K for the year ended December 31, 2024, 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.
Contacts
Investors:
MeiraGTx
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Media:
Jason Braco, Ph.D.
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| MEIRAGTX HOLDINGS PLC AND SUBSIDIARIES CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (in thousands, except share and per share amounts) | ||||||||
| For the Years Ended December 31, | ||||||||
| 2024 | 2023 | |||||||
| Revenues: | ||||||||
| Service revenue - related party | $ | 33,279 | $ | — | ||||
| License revenue - related party | — | 14,017 | ||||||
| Total revenue | 33,279 | 14,017 | ||||||
| Operating expenses: | ||||||||
| Cost of service revenue - related party | 23,791 | — | ||||||
| General and administrative | 54,216 | 47,293 | ||||||
| Research and development | 119,484 | 103,785 | ||||||
| Total operating expenses | 197,491 | 151,078 | ||||||
| Loss from operations | (164,212 | ) | (137,061 | ) | ||||
| Other non-operating income (expense): | ||||||||
| Foreign currency (loss) gain | (2,886 | ) | 9,300 | |||||
| Interest income | 4,145 | 2,272 | ||||||
| Interest expense | (13,272 | ) | (13,245 | ) | ||||
| Gain on sale of nonfinancial assets | 28,434 | 54,208 | ||||||
| Fair value adjustment | — | 499 | ||||||
| Net loss | (147,791 | ) | (84,027 | ) | ||||
| Other comprehensive loss: | ||||||||
| Foreign currency translation loss | (2,284 | ) | (7,482 | ) | ||||
| Comprehensive loss | $ | (150,075 | ) | $ | (91,509 | ) | ||
| Net loss | $ | (147,791 | ) | $ | (84,027 | ) | ||
| Basic and diluted net loss per ordinary share | $ | (2.12 | ) | $ | (1.49 | ) | ||
| Weighted-average number of ordinary shares outstanding | 69,822,353 | 56,486,525 | ||||||
| MEIRAGTX HOLDINGS PLC AND SUBSIDIARIES CONSOLIDATED BALANCE SHEETS (in thousands, except share and per share amounts) | ||||||||
| December 31, | December 31, | |||||||
| 2024 | 2023 | |||||||
| ASSETS | ||||||||
| CURRENT ASSETS: | ||||||||
| Cash and cash equivalents | $ | 103,659 | $ | 129,566 | ||||
| Accounts receivable - related party | 707 | 10,138 | ||||||
| Contract assets - related party | 950 | — | ||||||
| Inventory | 385 | — | ||||||
| Prepaid expenses | 6,828 | 5,625 | ||||||
| Tax incentive receivable | 8,971 | 13,277 | ||||||
| Other current assets | 2,018 | 1,016 | ||||||
| Total Current Assets | 123,518 | 159,622 | ||||||
| Property, plant and equipment, net | 102,878 | 115,896 | ||||||
| Intangible assets, net | 821 | 1,118 | ||||||
| Restricted cash | 2,009 | 1,083 | ||||||
| Other assets | 1,002 | 1,917 | ||||||
| Equity method and other investments | 6,749 | 6,766 | ||||||
| Right-of-use assets - operating leases, net | 10,576 | 15,910 | ||||||
| Right-of-use assets - finance leases, net | 22,198 | 24,432 | ||||||
| TOTAL ASSETS | $ | 269,751 | $ | 326,744 | ||||
| LIABILITIES AND SHAREHOLDERS' EQUITY | ||||||||
| CURRENT LIABILITIES: | ||||||||
| Accounts payable | $ | 23,586 | $ | 16,042 | ||||
| Accrued expenses | 27,414 | 42,639 | ||||||
| Lease obligations, current | 4,053 | 4,193 | ||||||
| Deferred revenue - related party, current | 4,827 | 2,926 | ||||||
| Other current liabilities | 903 | 1,278 | ||||||
| Total Current Liabilities | 60,783 | 67,078 | ||||||
| Deferred revenue - related party | 57,576 | 34,017 | ||||||
| Lease obligations | 7,523 | 12,952 | ||||||
| Asset retirement obligations | 2,821 | 2,401 | ||||||
| Note payable, net | 73,221 | 72,119 | ||||||
| TOTAL LIABILITIES | 201,924 | 188,567 | ||||||
| COMMITMENTS AND CONTINGENCIES (Note 14) | ||||||||
| SHAREHOLDERS' EQUITY: | ||||||||
| Ordinary Shares, $0.00003881 par value, 1,288,327,750 authorized, 78,397,380 and 63,601,015 shares issued and outstanding at December 31, 2024 and December 31, 2023, respectively | 3 | 2 | ||||||
| Capital in excess of par value | 773,565 | 693,841 | ||||||
| Accumulated other comprehensive loss | (3,719 | ) | (1,435 | ) | ||||
| Accumulated deficit | (702,022 | ) | (554,231 | ) | ||||
| Total Shareholders' Equity | 67,827 | 138,177 | ||||||
| TOTAL LIABILITIES AND SHAREHOLDERS' EQUITY | $ | 269,751 | $ | 326,744 | ||||
| Last Trade: | US$8.25 |
| Daily Change: | 1.84 28.71 |
| Daily Volume: | 6,282,058 |
| Market Cap: | US$644.740M |
December 09, 2024 November 13, 2024 | |
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