MeiraGTx (MGTX) RBC Capital Markets Virtual Ophthalmology Conference summary

Company overview and late-stage pipeline

• Three late-stage programs: two in ophthalmology for inherited retinal diseases (partnered with J&J and Lilly, both awaiting global regulatory filings), and two outside ophthalmology (xerostomia and Parkinson's, with Parkinson's phase III starting this year).

• Internal manufacturing capabilities enable cost efficiency and rapid regulatory response, with advanced vector optimization technologies reducing required doses.

• Platform enables one-time DNA delivery and daily oral pills for a wide range of protein and peptide therapeutics.

• All programs use locally delivered small doses of AAV, enhancing safety and reducing costs.

Ophthalmology and inherited retinal disease (IRD) programs

• IRDs are ideal for gene therapy due to their genetic basis and the eye's suitability for local delivery and detailed functional assessment.

• Deep relationships with leading global centers (e.g., Moorfields Eye Hospital) and KOLs have been crucial for clinical and commercial success.

• The IRD field is highly specialized, with a small number of experts seeing the majority of patients worldwide.

• Synergies exist across IRD programs, leveraging the same clinical sites and physician expertise for multiple indications.

Key program updates and partnerships

• XLRP program (partnered with J&J) showed promising phase III results; awaiting J&J's commercialization strategy.

• Learnings from XLRP pivotal study (surgical techniques, steroid regimens, surgeon training) are being applied across other IRD programs.

• LCA4 program (AIPL1 mutation, partnered with Lilly): 11 children treated under specials license gained sight; Lilly paid $75M upfront plus milestones and is preparing global filings.

• BBS10 program initiated via patient advocacy, using the U.K. specials license to treat children with no other options; similar approach may be used for other rare IRDs.