Metagenomi Therapeutics (MGX) H.C. Wainwright 26th Annual Global Investment Conference 2024 summary

Strategic vision and technology platform

• Focused on developing curative genetic medicines using an AI-driven metagenomics platform for programmable genome editing.

• Proprietary toolbox includes nucleases, base editors, and RIGs for a range of genetic corrections.

• Compact and highly specific editing systems offer advantages over first-generation technologies.

• Partnerships with Ionis (in vivo gene editing) and Affini-T (ex vivo immuno-oncology) provide significant milestone and royalty opportunities.

• Actively seeking additional partnerships to accelerate clinical development and maintain fiscal responsibility.

Pipeline and program highlights

• Pipeline selection prioritizes compelling disease biology, available biomarkers, clear development paths, and unmet medical needs.

• Lead program MGX001 targets Hemophilia A, achieving 12-month durability in non-human primates.

• Ionis collaboration includes four programs, two publicly disclosed (transthyretin amyloidosis and refractory hypertension), all in lead optimization.

• Additional programs address alpha-1 antitrypsin, Wilson's disease, and extrahepatic indications.

• Achieved a milestone with Affini-T, supporting regulatory filings and supplying cGMP materials.

Hemophilia A program and clinical progress

• MGX001 uses a two-component system: LNP-delivered nuclease and AAV-delivered donor template for targeted gene integration.

• Preclinical studies in non-human primates showed stable, therapeutically relevant Factor VIII levels over 12 months, with favorable safety profile.

• Optimizations include use of type five nuclease for specificity, mRNA enhancements, and bioengineered Factor VIII variant.

• No off-target editing observed; delivery components optimized for safety and reduced dosing.

• IND-enabling manufacturing underway, with plans for IND submission in 2026 and ongoing regulatory engagement.