MeiraGTx (MGTX) RBC Inaugural Virtual Ophthalmology Conference summary

Company overview and pipeline

• Focuses on local delivery gene therapy with a diverse pipeline targeting ophthalmology, xerostomia, and Parkinson's disease.

• Internal manufacturing capabilities enable rapid clinical advancement and regulatory flexibility.

• AI-driven optimization enhances vector potency, reduces dose, and improves safety and cost.

• Recent partnership with HalogenAI supports manufacturing and clinical development.

Regulatory environment and agency interactions

• Maintains strong, positive relationships with the FDA and global regulators, with no negative impact from recent leadership changes.

• Recent communications with the FDA have been extremely positive, supporting ongoing pivotal programs.

• Regulatory agencies value transformative clinical data, especially for severe unmet needs.

AAV-AIPL1 (LCA4) program highlights

• Treated 11 blind children with AAV-AIPL1, all gained meaningful vision, with data published and shared with regulators.

• UK MHRA supports approval under exceptional circumstances based on unprecedented clinical data.

• Engaged in positive discussions with the FDA and other agencies for expedited review.

• Rare pediatric disease designation could yield a priority review voucher if approved.

• Newborn testing could enable early identification and treatment of eligible patients.