Meiragtx Holdings Plc Stock Price, News & Analysis

MeiraGTx (Nasdaq: MGTX) reported Q3 2025 results and operational updates on Nov 13, 2025. Key corporate actions include a broad ophthalmology collaboration with Eli Lilly (upfront $75.0M; >$400M in potential milestones; tiered royalties) and progress on a joint venture with Hologen (received $50.0M of $200.0M; MeiraGTx to hold 30% of JV).

Clinical/regulatory highlights: RMAT designations for AAV2-hAQP1 and AAV-GAD, AQUAx2 pivotal enrollment on track to finish end-2025 with potential BLA pathway in early 2027, and Specials program dosing for BBS10 (RPDD).

Cash: $17.1M at Sept 30, 2025; service revenue fell to $0.4M for Q3 2025 from $10.9M year-ago.

MeiraGTx (Nasdaq: MGTX) announced a broad strategic collaboration with Eli Lilly on Nov 10, 2025 to develop and commercialize genetic medicines in ophthalmology.

MeiraGTx granted Lilly worldwide exclusive rights to its AAV-AIPL1 program for LCA4 and exclusive access to in‑house intravitreal capsids, bespoke promoters (including AI‑generated promoters), and certain riboswitch technology for ocular gene editing. Clinical data reported that 11 children born legally blind gained vision after AAV-AIPL1, with additional developmental benefits described.

Financial terms include a $75 million upfront payment, eligibility for over $400 million in milestones, and tiered royalties on licensed products.

MeiraGTx (Nasdaq: MGTX) will present two posters at the ESGCT 2025 Annual Congress in Seville, Oct 7-10, 2025.

Poster P0089 reports directed evolution of novel AAV capsids from a ~1E7-variant library in non-human primate eyes, validated in human retinal organoids and iPSC-derived RPE. Selected capsids showed >2-fold higher transduction versus AAV2.7m8 and higher retinal expression in mouse intravitreal studies, supporting ocular gene therapy applications.

Poster P0170 describes scale-up of a perfusion-based AAV manufacturing process to 40L bioreactors. Bench optimization delivered up to +120% volumetric VG yield, ~50% lower plasmid use, and up to 2.2-fold lower cost-of-goods per dose; the work focuses on translating these parameters to 40L while preserving quality.

MeiraGTx (NASDAQ:MGTX), a clinical-stage genetic medicines company, reported significant progress across multiple programs in Q2 2025. The company achieved FDA alignment on its Phase 2 AQUAx2 study for radiation-induced xerostomia and received RMAT designation for AAV-GAD in Parkinson's disease treatment.

Key developments include plans to file for Marketing Authorization Approval with MHRA and BLA with FDA for LCA4 treatment in Q4 2025. The company's strategic collaboration with Hologen includes $200 million in cash consideration and formation of Hologen Neuro AI Ltd joint venture with up to $230 million additional funding.

Financial position shows $32.2 million in cash as of June 30, 2025, with additional receivables and expected proceeds sufficient to fund operations into 2027.

MeiraGTx (Nasdaq: MGTX) presented four posters at the ASGCT 2025 Annual Meeting showcasing their gene and cell therapy technology platforms. Key presentations included:

1. An ultra-low dose CNS gene therapy for pediatric obesity using optimized BDNF clinical construct, showing 40% weight loss in mice vs 12% with semaglutide

2. Novel CAG-based promoters that are shorter and more potent than traditional ones, with one variant (C178) showing superior performance in muscle, liver, heart, and brain while being 700 base pairs shorter

3. Research on transcription factor binding sites (TFBS) to enhance synthetic promoter activity, identifying optimal combinations for gene expression

4. Development of optimized AAV-hUPF1 gene therapy for ALS and FTD, showing improved efficacy in multiple disease models while reducing construct size by 1.5kb

MeiraGTx (MGTX) reported significant Q1 2025 developments, highlighted by a $200 million strategic collaboration with Hologen AI and formation of Hologen Neuro AI Ltd joint venture with $230 million additional committed capital. Key achievements include: FDA's RMAT designation for AAV-GAD Parkinson's treatment, positive Phase 2 data showing disease-modifying effects, and alignment with FDA on Phase 2 AQUAx2 study for radiation-induced xerostomia. The company reported breakthrough results in LCA4 treatment, with 11 out of 11 children showing meaningful responses, leading to plans for Marketing Authorization filing. The Phase 3 LUMEOS trial for X-linked Retinitis Pigmentosa showed positive secondary endpoints despite missing primary endpoint. MeiraGTx continues advancing its riboswitch technology platform and maintains strong manufacturing capabilities with renewed MHRA licenses.

MeiraGTx (NASDAQ: MGTX) has received FDA Regenerative Medicine Advanced Therapy (RMAT) designation for AAV-GAD, a one-time gene therapy treatment for Parkinson's disease. The designation was granted based on positive data from three clinical studies, including a Phase 1 dose-escalating study (n=12) and two double-blind, sham-controlled Phase 2 studies (n=45 and n=14). The therapy demonstrated significant clinical benefits in motor function (UPDRS Part 3) and, through AI analysis with partner Hologen, showed evidence of disease-modifying changes in brain circuitry. The RMAT designation provides benefits similar to Fast Track and Breakthrough Therapy designations, enabling expedited development, increased FDA interaction, and potential routes to accelerated approval and Priority Review.

MeiraGTx (NASDAQ: MGTX) announced significant developments in Q4 2024 and early 2025, highlighted by a strategic collaboration with Hologen AI including a $200 million upfront payment and formation of Hologen Neuro AI joint venture with $230 million additional committed capital.

Key achievements include: positive data from AAV-GAD Parkinson's disease clinical study showing significant UPDRS Part 3 improvements; FDA Regenerative Medicine Advanced Therapy designation for AAV2-hAQP1 xerostomia treatment; and exceptional clinical results for LCA4 gene therapy with 100% response rate in treated children.

The company ended 2024 with $103.7 million in cash and expects sufficient funding into 2027, including the Hologen collaboration proceeds. Additional potential revenue includes $285 million in milestone payments upon first commercial sales of bota-vec in US and EU.

MeiraGTx (MGTX) has announced a strategic collaboration with Hologen AI, receiving $200 million upfront and forming a joint venture called Hologen Neuro AI The venture will receive $230 million in committed capital from Hologen to fund AAV-GAD development for Parkinson's disease through commercialization.

The joint venture combines MeiraGTx's genetic medicines expertise with Hologen's AI capabilities. MeiraGTx will retain 30% ownership in the venture and lead clinical development and manufacturing. The company's AAV-GAD program is Phase 3 ready, with positive data reported in October 2024 showing an 18-point improvement in UPDRS Part 3 scores at 26 weeks.

The collaboration includes exclusive manufacturing agreements and Hologen taking a minority stake in MeiraGTx's manufacturing subsidiary. The deal is expected to close in Q2 2025, subject to regulatory approvals.