MeiraGTx (MGTX) Corporate presentation summary

Late-stage clinical pipeline and technology platforms

• Four pivotal and BLA-ready programs target radiation-induced xerostomia, X-linked retinitis pigmentosa, Parkinson's disease, and AIPL1 retinal dystrophy, with additional preclinical assets in ALS, neuropathic pain, obesity, diabetes, and ophthalmology indications.

• Proprietary Riboswitch platform enables precise, oral small molecule-controlled in vivo production of therapeutic proteins, applicable across gene therapy, cell therapy, and gene editing.

• In-house, end-to-end GMP manufacturing includes two cGMP viral vector facilities, plasmid production, QC, fill & finish, and commercial licenses in the UK and Ireland.

• Next-generation vector optimization leverages a >250k promoter library and AI-driven design for improved potency, safety, and reduced cost of goods.

Clinical program highlights

• AAV-AQP1 for radiation-induced xerostomia shows transformative, durable improvements in patient-reported outcomes and saliva flow, with pivotal Phase 2 enrolling and BLA filing expected in 2027.

• Bota-vec for X-linked retinitis pigmentosa completed Phase 3 with robust efficacy across all vision domains, strong safety, and is preparing for BLA/MAA filings.

• AAV-GAD for Parkinson's disease is Phase 3 ready, with evidence of disease modification and positive results from multiple randomized, sham-controlled trials.

• AAV-AIPL1 for LCA4 congenital blindness demonstrated vision restoration in all treated children, with near-term regulatory filings advised by both MHRA and FDA.

Market opportunity and commercial strategy

• Persistent radiation-induced xerostomia represents a large, unmet need with a US prevalence of 165k and global prevalence of 435k; peak global revenue estimated at $3.7B.

• Bota-vec targets a concentrated IRD market with projected peak sales of ~$1.7B and strong KOL and patient advocacy support.

• Commercial launches are supported by in-house manufacturing, ensuring rapid supply and scalability.

• Strategic partnerships with Eli Lilly, Sanofi, and Hologen AI provide significant upfront payments, milestone opportunities, and funding for pipeline advancement.