MeiraGTx Holdings Plc (MGTX:NASDAQ) has announced its financial and operational results for the fourth quarter and full year 2024, highlighting a year of significant progress across its clinical programs and manufacturing operations. The company also reported a strategic collaboration with Hologen AI, securing a US$200 million upfront payment and an additional US$230 million in committed capital through the joint venture Hologen Neuro AI Ltd. The partnership is set to accelerate the Phase 3 clinical development of AAV-GAD, a gene therapy candidate for Parkinson's disease.
The company posted cash and cash equivalents of approximately US$103.7 million as of December 31, 2024. Service revenue for the year reached US$33.3 million, attributed to process performance qualification services under an asset purchase agreement with Johnson & Johnson Innovative Medicine. Research and development expenses totaled US$119.5 million, reflecting increased manufacturing and preclinical costs. Net loss for the year was US$147.8 million, compared to US$84.0 million in 2023.
MeiraGTx has made substantial regulatory and clinical progress, as outlined in the announcement. The company received FDA Regenerative Medicine Advanced Therapy (RMAT) designation for AAV2-hAQP1, a treatment for radiation-induced xerostomia, and reported positive results from a randomized, sham-controlled bridging study of AAV-GAD for Parkinson's disease. The study showed statistically significant improvements in motor function and patient-reported outcomes. In ophthalmology, efficacy data for rAAV8.hRKp.AIPL1 in treating Leber congenital amaurosis (LCA4) were published in The Lancet, demonstrating benefits in all treated children. The company plans to submit a Marketing Authorization Application (MAA) in the UK and follow a parallel pathway for U.S. approval.
Manufacturing capabilities also expanded, with additional regulatory licensures secured for facilities in Ireland and the UK. The Shannon site in Ireland now holds authorizations for viral vector manufacturing, allowing for the production of materials used in clinical trials.
Gene Therapy Market Expands Amid Innovation and Cost Challenges
The gene therapy market has continued to expand as advancements in genetic medicine drive innovation in treating rare diseases, genetic disorders, and complex medical conditions. According to a February report from MarketsandMarkets, the global gene therapy market was valued at US$7.21 billion in 2023 and was projected to grow at a 19.4% compound annual growth rate (CAGR), reaching US$8.85 billion in 2024 and US$36.55 billion by 2032. The report noted that increasing regulatory approvals and investments in gene therapy research had played a key role in this expansion.
While the industry has seen significant progress, challenges remain. MarketsandMarkets further noted that high development costs and complex regulatory frameworks continue to limit broader adoption. A report by Evernorth Health in 2022 indicated that the average cost of developing a gene therapy could reach US$5 billion, making it significantly more expensive than traditional pharmaceutical drugs. Additionally, newly launched gene therapies have carried high price tags, with treatments like Beqvez for Hemophilia B costing US$3.5 million and Lenmeldy for Metachromatic Leukodystrophy priced at US$4.25 million. These costs have restricted patient access, particularly in lower-income markets.
According to a Yahoo! Finance January 20 report from Usman Kabir, billionaire investor Joseph Edelman included MeiraGTx Holdings plc. among his long-term stock picks.
A March 13 report from DataM Intelligence projected even higher growth, estimating that the global gene therapy market would reach US$51.22 billion by 2031, reflecting a 31.89% CAGR over the forecast period. The report highlighted that gene therapy has gained traction due to its ability to target genetic disorders and rare diseases through advanced delivery methods, including viral and non-viral vectors. It also pointed to recent FDA approvals of gene therapies for conditions like spinal muscular atrophy and inherited blindness, demonstrating the market's clinical progress.
According to the Business Research Company, on March 13, the demand for gene therapy was further fueled by the rise in rare genetic disorders and the growing adoption of precision medicine. The report cited improvements in genetic testing and early diagnoses as key factors contributing to the market's development. Additionally, the presence of major pharmaceutical companies and biotech firms in the sector has intensified research efforts, leading to increased clinical trials and regulatory approvals.
MeiraGTx Advances Gene Therapy Pipeline with AI, Regulatory Milestones, and Manufacturing Expansion
MeiraGTx's near-term growth prospects are supported by its expanding clinical pipeline and manufacturing capabilities, as outlined in its investor presentation. The company's joint venture with Hologen Neuro AI Ltd positions it at the forefront of AI-driven clinical drug development, with a focus on neurodegenerative and neuropsychiatric disorders. The AAV-GAD program, which has shown promising clinical data, is expected to enter Phase 3 trials in 2025.
The company's regulatory designations, including RMAT for AAV2-hAQP1 and Rare Pediatric Disease Designations for multiple inherited retinal disease therapies, provide pathways for accelerated approvals. The potential submission of an MAA in the UK for rAAV8.hRKp.AIPL1 marks a significant milestone in gene therapy for inherited blindness.
Manufacturing investments continue to strengthen MeiraGTx's position as a vertically integrated gene therapy company. Its proprietary riboswitch technology platform, aimed at gene regulation and in vivo protein expression, is advancing toward first-in-human studies in metabolic disease and oncology applications.
With multiple late-stage clinical programs, regulatory milestones, and strategic collaborations in place, MeiraGTx remains focused on advancing its genetic medicine pipeline while strengthening its financial position to support long-term growth.
Gene Therapy Innovator Surpasses Projections, Secures Billionaire Backing
According to a Yahoo! Finance January 20 report from Usman Kabir of Insider Monkey, billionaire investor Joseph Edelman included MeiraGTx Holdings plc. among his long-term stock picks. Edelman, the founder and CEO of Perceptive Advisors, has built a reputation as one of the most successful investors in the biotechnology sector, identifying high-growth companies with strong clinical pipelines. His fund held a US$6.3 million stake in MeiraGTx at the end of the third quarter of 2024, reflecting confidence in the company's trajectory.
The report highlighted MeiraGTx’s strong financial performance, noting that the company significantly surpassed analyst revenue estimates in the third quarter of 2024. It reported US$10.9 million in revenue, far exceeding the projected US$1.63 million, and held approximately US$122.9 million in cash and cash equivalents. The analysis pointed to the company's expanding pipeline of gene therapy programs as a key factor supporting its investment appeal. Notably, the company had initiated a Phase 2 trial for radiation-induced xerostomia (RIX), a condition associated with head and neck cancer treatments, alongside a program targeting Sjogren's syndrome, an autoimmune disease affecting salivary glands.
Edelman's long-term investment in MeiraGTx positioned it seventh among his top biotech stock picks, indicating strong backing from a prominent institutional investor known for early bets on breakthrough gene therapy technologies.
Streetwise Ownership Overview*While the report acknowledged the inherent volatility in the biotech sector, it underscored the company’s potential within the expanding gene therapy market.
Ownership and Share Structure
According to Refinitiv, about 4% of the company is held by insiders and management and 59% by institutions. Strategic entities hold 35.39%.
Top shareholders include Perceptive Advisors LLC with 15.95%, Sanofi SA with 14.58%, Johnson & Johnson Innovation with 8.42%, Adage Capital Management with 6.38%, and Prosight Capital with 5.57%.
It has about 78 million shares outstanding with a market cap of US$644.77 million and trades in a 52-week range of US$3.85 and US$7.60.
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- As of the date of this article, officers and/or employees of Streetwise Reports LLC (including members of their household) own securities of MeiraGTx Holdings Plc.
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