MeiraGTx (MGTX) Corporate presentation summary

Pipeline and clinical programs

• Four pivotal, late-stage programs: radiation-induced xerostomia, X-linked retinitis pigmentosa, Parkinson's disease, and AIPL1 retinal dystrophy, with multiple BLA filings expected between 2026 and 2028.

• Broad preclinical pipeline includes ALS, neuropathic pain, obesity, diabetes, and major ophthalmology indications.

• Proprietary Riboswitch platform enables precise, oral small molecule control of therapeutic protein production in vivo, with first clinical candidate (leptin) entering clinic in 2026.

• End-to-end, in-house GMP manufacturing with two cGMP vector facilities, plasmid production, QC, and fill/finish capabilities, supporting rapid clinical and commercial supply.

• Commercial licenses in Ireland and UK for viral vector production and QC, expediting IND to commercial timelines by 2-3 years.

Key clinical data and market opportunities

• AAV-AQP1 for radiation-induced xerostomia shows transformative, durable improvements in both patient-reported and objective endpoints, with pivotal Phase 2 enrollment complete and BLA filing expected in 2027.

• Bota-vec for X-linked retinitis pigmentosa demonstrated statistically significant and clinically meaningful improvements in all domains of vision in Phase 3, with strong advocacy support and BLA/MAA filings underway.

• AAV-GAD for Parkinson's disease met primary endpoints in two sham-controlled trials, showing evidence of disease modification and is Phase 3 ready.

• AAV-AIPL1 for LCA4 congenital blindness restored vision in all treated children, with potential near-term marketing authorization under exceptional circumstances.

• Large commercial opportunities: peak global revenue for AAV-AQP1 estimated at $3.7B, Bota-vec at $1.7B, and significant market potential for Parkinson's and LCA4 programs.

Technology platforms and manufacturing

• Advanced vector engineering platforms include a >250k promoter library, AI-driven optimization, and proprietary capsids for improved potency, safety, and lower cost of goods.

• Riboswitch gene regulation platform enables precise, titratable in vivo production of a wide range of therapeutic proteins, peptides, antibodies, and gene editing tools via oral dosing.

• Demonstrated tight, dose-dependent control of protein expression in vitro and in vivo, with applications in metabolic disease, obesity, cell therapy, and gene editing.

• In vivo delivery of regulated peptides (e.g., GLP1-GIP, leptin) shows superior efficacy and safety compared to constitutive or synthetic peptide therapies.

• Manufacturing infrastructure supports rapid scale-up, commercial readiness, and global regulatory compliance.