MeiraGTx (MGTX) investor relations material

Executive summary

• Clinical-stage genetic medicines company with late-stage programs in Parkinson's, xerostomia, and retinal dystrophy, and advanced pivotal Phase 2 and Phase 3 studies.

• Internal end-to-end manufacturing capabilities and proprietary gene regulation platform.

• Strategic collaborations with Hologen AI and Eli Lilly, including significant upfront and milestone payments, and joint ventures.

• Acquisition of Smart Immune assets to expand cell therapy pipeline.

• Completed optimization of riboswitch leptin program, with IND-enabling discussions ongoing.

Financial highlights

• Net loss of $50.5M for Q3 2025, up from $39.3M in Q3 2024; net loss of $129.3M for nine months ended September 30, 2025.

• Revenue of $0.4M for Q3 2025, down from $10.9M in Q3 2024; $6.0M for nine months, down from $11.9M year-over-year.

• Cash, cash equivalents, and restricted cash of $17.1M as of September 30, 2025, down from $105.7M at December 31, 2024.

• Operating expenses decreased to $46.5M in Q3 2025 from $51.0M in Q3 2024; $138.5M for nine months, down from $144.6M.

• R&D expenses increased to $32.5M in Q3 2025 from $26.2M in Q3 2024, mainly due to higher manufacturing and clinical trial material costs.

Outlook and guidance

• Anticipates sufficient funding into the second half of 2027, including repayment of $75M debt due August 2026, supported by upfront and milestone payments from Eli Lilly and Hologen AI.

• Expects pivotal data for AAV-hAQP1 in early 2027, with potential BLA filing and approval later that year.

• Expects continued operating losses and need for additional capital for product development and commercialization.

• Plans to initiate Phase 3 AAV-GAD study for Parkinson's disease in the coming months.

• Riboswitch leptin program expected to enter the clinic following IND-enabling discussions.