MeiraGTx (MGTX) Goldman Sachs 45th Annual Global Healthcare Conference summary

Strategic Focus and Pipeline Overview

• Streamlined operations to focus on two late-stage clinical programs and advanced in-vivo delivery using riboswitch technology.

• Pipeline targets large, unmet needs with small, locally delivered doses, enabling low-cost manufacturing.

• Advancing three pivotal/Phase 3 programs: retinitis pigmentosa (dosing complete), radiation-induced xerostomia (pivotal), and Parkinson's disease (Phase 3 ready), with potential global filings in 2025–2027.

• Deep pre-IND pipeline includes ALS, MC4R obesity, and metabolic diseases targeting large patient populations.

• Proprietary vectorization and next-generation vectors improve potency, safety, and cost of goods, supporting affordable therapies.

Clinical Program Highlights

• Ophthalmology program partnered and sold to Johnson & Johnson/Janssen, with $115M–$130M received upfront and $285M in potential milestones; commercial manufacturing agreement in place.

• Xerostomia program showed unprecedented improvements in patient-reported outcomes and saliva production, supporting ongoing pivotal phase II/III studies.

• Parkinson's program demonstrated significant improvements in motor scores and disease modification, with Phase 3 readiness and global reach.

• AAV-AQP1 for radiation-induced xerostomia addresses a large, underserved patient population, with strong Phase 1 data and pivotal Phase 2 enrolling; global filings targeted for 2026.

• Cell engineering with riboswitch control enables precise, oral regulation of therapeutic protein production in engineered cells.

Market Expansion and Future Indications

• Xerostomia vector applicable to Sjögren's syndrome and prostate cancer-induced xerostomia, with IND-enabling studies for preventive use.

• Large addressable markets identified, with 170,000 target patients for radiation-induced xerostomia in the U.S. alone.

• AI-driven screening identified multiple BBB-penetrant small molecules, with lead candidates entering IND-enabling studies in 2024.