Financial Performance - Total revenue for 2024 was $33,279,000, a significant increase from $14,017,000 in 2023, representing a growth of 137%[714] - Operating expenses increased to $197,491,000 in 2024 from $151,078,000 in 2023, marking a rise of 30.7%[714] - The net loss for 2024 was $147,791,000, compared to a net loss of $84,027,000 in 2023, indicating a 75.9% increase in losses[714] - Cash and cash equivalents decreased to $103,659,000 in 2024 from $129,566,000 in 2023, reflecting a decline of 20%[713] - Total current assets fell to $123,518,000 in 2024, down from $159,622,000 in 2023, a decrease of 22.6%[713] - Total liabilities increased to $201,924,000 in 2024 from $188,567,000 in 2023, an increase of 7.1%[713] - Shareholders' equity decreased significantly to $67,827,000 in 2024 from $138,177,000 in 2023, a decline of 50.9%[713] - The company reported a basic and diluted net loss per ordinary share of $2.12 for 2024, compared to $1.49 for 2023[714] - The segment net loss for 2024 was $147,791,000, compared to a net loss of $84,027,000 in 2023, indicating a worsening financial performance[814] Capital and Funding - The company requires additional capital to fund operations, which may not be available on acceptable terms[14] - The Company’s capital resources have been primarily funded through collaboration agreements and equity offerings[727] - The Company raised gross proceeds of $8.4 million through the sale of 1,508,517 ordinary shares under the "at-the-market" equity offering program during the year ended December 31, 2024[842] - The Company completed a public offering of 12,500,000 ordinary shares at a price of $4.00 per share, resulting in gross proceeds of $50.0 million on August 12, 2024[843] Research and Development - The company is heavily dependent on the success of its product candidates, which are still in development, and if none receive regulatory approval, the business may be harmed[14] - Clinical trials are expensive and time-consuming, with uncertain outcomes, and the company may encounter substantial delays[14] - The Company incurred research and development costs with Johnson & Johnson Innovative Medicine responsible for up to 100% of the costs, depending on the type of services performed[795] - Research and development costs are charged to expense as incurred, including employee-related expenses and costs associated with clinical studies[800] - The Company recorded reductions to research and development expenses of $5.5 million and $5.1 million for the years ended December 31, 2024 and 2023, respectively, related to tax incentive programs[736] Regulatory and Compliance - The company is subject to significant regulation regarding the manufacturing of its products, and its manufacturing facilities may not continue to meet regulatory requirements[14] - The Company has not recognized a provision for obsolete and excess inventory as of December 31, 2024[734] Competition and Market Environment - The company faces significant competition in a rapidly changing technological environment, which may affect its financial condition and ability to market its product candidates[21] - The company depends on proprietary technology licensed from others, and losing these licenses could hinder its ability to develop product candidates[21] Tax and Deferred Tax - The Company recorded unrecognized tax positions of $2.2 million and $2.0 million as of December 31, 2024 and 2023, respectively[805] - The Company has recorded a full valuation allowance against its deferred tax assets, indicating management's assessment that it is more likely than not that these assets will not be fully realized[852] - The Company recorded a deferred tax asset of $155.2 million as of December 31, 2024, compared to $130.3 million in 2023, primarily due to net operating loss carryforwards increasing from $70.7 million to $85.3 million[852] - The total deferred tax expense for the Company was $24.0 million in 2024, compared to $9.7 million in 2023, reflecting a significant increase in deferred tax liabilities[852] Collaboration Agreements - The Company received a non-refundable upfront cash payment of $65.0 million from Janssen in December 2023 under the Asset Purchase Agreement[720] - Janssen agreed to pay future contingent consideration of up to $350.0 million, including milestone payments of $50.0 million for initiating a Phase 3 clinical trial and $175.0 million upon the first commercial sale of the RPGR Product in the U.S.[720] - Under the Collaboration Agreement with Johnson & Johnson Innovative Medicine, the Company received a non-refundable upfront fee of $100.0 million and a milestone payment of $30.0 million in December 2021[856] Asset Management - Long-lived assets decreased from $157,356,000 in 2023 to $136,473,000 in 2024, with notable declines in assets located in the United States and the European Union[816] - The balance of asset retirement obligations increased from $2,401,000 in 2023 to $2,821,000 in 2024, reflecting a change in estimate of $345,000[763] Share-Based Compensation - The total share-based compensation expense for the year ended December 31, 2024, was $25.2 million, a decrease from $27.7 million in 2023[841] - The total compensation expense related to unvested options as of December 31, 2024, was $8.5 million, expected to be recognized over 3.2 years[836] - The total compensation expense related to unvested RSUs as of December 31, 2024, was $21.9 million, expected to be recognized over 3.3 years[839] - The Company recorded share-based compensation expense of $15.2 million for RSUs in 2024, compared to $14.1 million in 2023[838] Revenue Recognition - The Company recognizes revenue based on the control of promised goods or services, following ASC 606, and evaluates performance obligations to determine revenue recognition[782] - The Company recognizes revenues from nonrefundable, up-front fees allocated to licenses when the license is transferred and the licensee can benefit from it[792] - The Company’s collaboration revenue is recognized using the cost-to-cost input method under ASC 606, reflecting the progress towards completion of performance obligations[864] - The company recognized $33.3 million of service revenue during the year ended December 31, 2024, which included $14.0 million of deferred revenue recognized as service revenue[876]

Exhibit 99.2 MeiraGTx Reports Fourth Quarter and Full Year 2024 Financial and Operational Results and Recent Business Updates 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 advanc ...

Core Viewpoint - MeiraGTx announced a strategic collaboration with Hologen AI, involving a $200 million upfront payment and the formation of a joint venture, Hologen Neuro AI Ltd, aimed at advancing the development of therapies for neurodegenerative diseases, particularly Parkinson's disease [1][2] Recent Developments - The joint venture will focus on the Phase 3 clinical development of AAV-GAD for Parkinson's disease, with an additional $230 million committed by Hologen [1][2] - MeiraGTx reported positive results from a clinical study of AAV-GAD, showing significant improvements in the Unified Parkinson's Disease Rating Scale (UPDRS) and the Parkinson's Disease Questionnaire (PDQ-39) [1][8] - The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation for AAV2-hAQP1 for treating Grade 2/3 radiation-induced xerostomia (RIX) [1][8] Clinical and Regulatory Milestones - MeiraGTx achieved FDA Rare Pediatric Disease Designations for four therapies targeting rare inherited retinal diseases, including rAAV8.hRKp.AIPL1 for LCA4 [3] - The company plans to submit a Marketing Authorization Application (MAA) for rAAV8.hRKp.AIPL1 based on exceptional clinical results observed in children [4][14] Financial Overview - As of December 31, 2024, MeiraGTx reported cash and cash equivalents of approximately $103.7 million, with expectations to fund operations into 2027 [19] - The company experienced a net loss of $147.8 million for the year ended December 31, 2024, compared to a net loss of $84.0 million in 2023 [32][39] - Service revenue for 2024 was $33.3 million, a significant increase from zero in 2023, attributed to process performance qualification services [24][26] Manufacturing and Regulatory Compliance - MeiraGTx's UK manufacturing facility received successful GMP compliance renewal, supporting its application for a commercial MIA license [16] - The Shannon facility in Ireland obtained additional licenses for GMP viral vector manufacturing, marking a significant milestone for gene therapy in the region [18] Strategic Partnerships - The collaboration with Hologen AI is expected to enhance the efficiency and success probability of MeiraGTx's clinical programs through the application of advanced AI technologies [2][8] - Sanofi's strategic investment of $30 million in MeiraGTx provides additional financial backing and a right of first negotiation for certain therapeutic targets [23]

Core Viewpoint - MeiraGTx Holdings plc has announced a strategic collaboration with Hologen Limited, which includes a $200 million upfront cash payment and the formation of a joint venture, Hologen Neuro AI Ltd, aimed at developing therapies for neurodegenerative diseases, particularly Parkinson's disease [1][2][10]. Financial Aspects - MeiraGTx will receive $200 million in upfront cash at closing [2][10] - Hologen will provide up to $230 million in committed capital to fund the development of AAV-GAD for Parkinson's disease and other CNS programs [2][6] - The collaboration is expected to extend MeiraGTx's cash runway and support its internal manufacturing capabilities [7] Joint Venture Details - The joint venture, Hologen Neuro AI Ltd, will be co-owned, with MeiraGTx retaining 30% ownership and leading clinical development and manufacturing [3][10] - Hologen will contribute its generative AI technology to optimize MeiraGTx's manufacturing processes [3][10] - The joint venture will focus on the development of AAV-GAD and other locally delivered CNS genetic medicines [6][10] Clinical Development - MeiraGTx's AAV-GAD program for Parkinson's disease is Phase 3 ready, with positive data reported from a clinical bridging study showing significant improvements in patient outcomes [4][10] - The collaboration aims to enhance the robustness and efficiency of the AAV-GAD Phase 3 clinical study using Hologen's AI technology [5][7] Company Background - MeiraGTx is a clinical-stage genetic medicines company with a broad pipeline, including four late-stage clinical programs targeting various diseases [9] - The company has developed advanced manufacturing capabilities and innovative technologies for gene therapy, including a novel riboswitch gene regulation technology [9]

Core Viewpoint - MeiraGTx Holdings plc has announced a strategic collaboration with Hologen Limited, which includes a $200 million upfront cash payment and the formation of a joint venture, Hologen Neuro AI Ltd, aimed at developing therapies for neurodegenerative diseases, particularly Parkinson's disease [1][2][10]. Financial Terms - MeiraGTx will receive $200 million in upfront cash at closing [2][10] - Hologen will provide up to $230 million in committed capital to fund the development of AAV-GAD for Parkinson's disease and other CNS programs [2][6][10] - MeiraGTx will retain 30% ownership in the joint venture and will lead all clinical development and manufacturing [3][10] - Hologen will own a minority stake in MeiraGTx's manufacturing subsidiary and contribute to its annual funding [3][10] Joint Venture Details - The joint venture, Hologen Neuro AI Ltd, will utilize Hologen's proprietary multi-modal generative foundation models for drug development [2][10] - The joint venture aims to transform the discovery and development of therapies targeting CNS circuitry in neurodegenerative and neuropsychiatric disorders [2][3] Clinical Development - MeiraGTx's AAV-GAD program for Parkinson's disease is Phase 3 ready, with ongoing commercial manufacturing [4][10] - Positive data from a randomized clinical study showed significant improvement in the Unified Parkinson's Disease Rating Scale (UPDRS) and quality of life measures [4][10] Strategic Importance - The collaboration is expected to enhance the robustness and efficiency of the AAV-GAD Phase 3 clinical study [5][7] - The partnership aims to leverage AI technology to elucidate brain circuitry and improve the probability of success in clinical trials [5][7] Company Overview - MeiraGTx is a clinical-stage genetic medicines company with a broad pipeline, including four late-stage clinical programs targeting various diseases [9] - The company has developed comprehensive manufacturing capabilities and innovative technologies for gene delivery and expression control [9]

MeiraGTx Holdings (MGTX) Update / Briefing February 21, 2025 08:00 AM ET Speaker0 Good morning, and welcome to the GTX Investor Event. At this time, all attendees are in a listen only mode. A question and answer session will follow the presentations. As a reminder, this call is being recorded and a replay will be made available on the Mirror GTX website following the conclusion of the event. I'd now like to turn the call over to Doctor. Sandy Forbes, President and Chief Executive Officer of Miura GTX. Pleas ...

Core Insights - MeiraGTx Holdings plc announced positive results from a first-in-human interventional study using rAAV8.hRKp.AIPL1 to treat children with AIPL1-associated severe retinal dystrophy, showing significant improvements in visual acuity and functional vision [1][2][5] Study Details - The study involved 11 children, with 4 treated unilaterally and 7 treated bilaterally, all of whom were legally blind at birth and showed improved visual acuity after treatment [1][5][6] - The treatment was administered via subretinal injection, and the outcomes were measured using various methods including visual acuity tests and retinal imaging [3][4] Results - Prior to treatment, children's visual acuities were limited to light perception, but post-treatment, the mean visual acuity improved to 0.9 LogMAR from an equivalent of 2.7 LogMAR [4] - All treated children demonstrated enhanced visual function and structural preservation of the retina, with no serious adverse effects reported [2][4][6] Regulatory Pathway - Following the positive results, MeiraGTx is working with global regulators for expedited approval of rAAV8.hRKp.AIPL1, intending to submit a Marketing Authorization Application (MAA) based on the current data without the need for further clinical trials [7][8] Company Overview - MeiraGTx is a clinical-stage genetic medicines company with a focus on developing treatments for severe genetic conditions, including AIPL1-associated retinal dystrophy, and has capabilities in viral vector production and gene therapy technologies [10]

Core Insights - The article discusses the Biotech Analysis Central service, which provides in-depth analysis of pharmaceutical companies and investment opportunities in the biotech sector [1][2]. Group 1: Service Offerings - Biotech Analysis Central offers a library of over 600 biotech investing articles, a model portfolio of more than 10 small and mid-cap stocks, and live chat features for investors [2]. - The service is priced at $49 per month, with a discounted annual plan available at $399, representing a 33.50% savings for subscribers [1]. Group 2: Analyst Background - The author of the article is associated with Biotech Analysis Central and emphasizes the importance of informed decision-making for healthcare investors [2]. - The article does not indicate any personal investment positions in the companies discussed, ensuring an unbiased perspective [3].

Core Viewpoint - MeiraGTx has received Rare Pediatric Disease Designation from the FDA for four inherited retinal diseases, highlighting the potential of its proprietary technology platforms in treating severe childhood blinding conditions [1][2][3] Group 1: Rare Pediatric Disease Designation - The FDA granted Rare Pediatric Disease Designation to MeiraGTx's AAV8-RK-RetGC program for treating Leber congenital amaurosis due to GUCY2D mutations, marking the fourth designation received in three months [1] - The other three designations include AAV8-RK-AIPL1 for LCA4 retinal dystrophy, AAV8-RK-BBS10 for Bardet-Biedl syndrome, and AAV5-RDH12 for RDH12 associated retinal dystrophy [1][3] Group 2: Company Strategy and Regulatory Milestones - The receipt of the designation is viewed as a regulatory milestone that underscores the therapeutic potential of the company's technology [2] - The company plans to leverage its manufacturing infrastructure and clinical expertise to expedite the delivery of treatments to affected children [3] Group 3: Overview of Genetic Medicines - AAV8-RK-RetGC targets GUCY2D mutations, which account for 88% of LCA1 cases, leading to early blindness [4] - AAV8-RK-AIPL1 is designed to treat severe forms of LCA through a one-time subretinal injection [5] - AAV8-RK-BBS10 addresses Bardet-Biedl syndrome, affecting approximately 1 in 250,000 people globally, with visual impairment as a primary symptom [6] - AAV5-RDH12 targets RDH12 associated retinal dystrophy, which accounts for 3-10% of LCA cases [7][8] Group 4: Company Profile - MeiraGTx is a vertically integrated, clinical-stage genetic medicines company with a broad pipeline and end-to-end manufacturing capabilities [9] - The company has developed a proprietary manufacturing platform and focuses on applying genetic medicine to address common diseases with high unmet needs [9]

Group 1 - The biotech forum has seen significant discussions around profitable buy-write or covered call strategies on selected biotech stocks in recent months [1] - MeiraGTx Holdings plc has experienced a stock price increase of over 40% following the release of Parkinson's disease data on October 15, 2024, which is expected to facilitate a pivotal trial [2] - The investing group, The Biotech Forum, offers a model portfolio featuring 12-20 high upside biotech stocks, along with live chat for trade discussions and weekly market commentary [2]