MeiraGTx (MGTX) Corporate Presentation summary

Clinical pipeline and late-stage programs

• Four pivotal programs: Parkinson's disease, radiation-induced xerostomia, AIPL1 retinal dystrophy, and X-linked retinitis pigmentosa, with near-term BLA filings expected between 2025 and 2027.

• AAV-AIPL1 for LCA4 congenital blindness showed transformative visual improvements in children, with UK and FDA filings planned for 2025.

• AAV-AQP1 for xerostomia demonstrated strong Phase 1 data, significant patient-reported improvements, and a pivotal Phase 2 study is enrolling.

• AAV-GAD for Parkinson's disease is Phase 3 ready, with robust efficacy and safety data from multiple controlled studies.

• X-linked RP program acquired by Janssen, with MeiraGTx to manufacture commercial product and potential milestone payments up to $350M.

Manufacturing and technology platforms

• Fully integrated, end-to-end GMP manufacturing with two viral vector facilities, in-house plasmid production, and commercial QC labs.

• Commercial-grade manufacturing at IND stage accelerates development by 2–3 years and increases ROI.

• Proprietary vector engineering platforms optimize potency, safety, and manufacturability, including AI-driven promoter design.

• Riboswitch gene control platform enables precise, dose-responsive in vivo protein production using oral small molecules, with dynamic range >5000-fold.

• Riboswitch technology is modular, applicable to any transgene, and supports cell therapy, gene editing, and metabolic disease programs.

Key clinical data and patient impact

• AAV-AQP1 Phase 1: No treatment-related serious adverse events, 39.5% improvement in XQ score, 112.5% increase in saliva flow at 12 months.

• Bilateral treatment yielded greater improvements in xerostomia symptoms and quality of life.

• AAV-GAD Phase 2: Met primary endpoint with significant UPDRS motor score improvements vs. sham, reduced dyskinesia, and no worsening of Parkinson's in treated subjects.

• FDG-PET imaging identified a unique biomarker (GADRP) correlating with clinical improvement in Parkinson's disease.

• Riboswitch-regulated CAR-T cells showed superior potency, expansion, and safety profile compared to constitutive CAR-T in preclinical models.