Metagenomi Therapeutics (MGX) TD Cowen 46th Annual Health Care Conference summary

Strategic focus and company evolution

• Recently rebranded to reflect transition into a clinical-stage company focused on in vivo genome editing for curative genetic medicines.

• Prioritized capital allocation to advance lead program MGX-001 for hemophilia A and expand related programs.

• Extended cash runway into Q4 2027 after strategic capital reallocation, with $184 million in cash at last quarter's end.

• Engaged in strategic partnerships, notably with Ionis, to leverage gene-editing platform and accelerate development.

Lead program MGX-001 for hemophilia A

• Achieved proof-of-concept with normalized Factor VIII activity in non-human primate studies, showing dose-dependent efficacy and no thrombosis risk.

• Durability study demonstrated stable Factor VIII activity up to 19 months, even with less optimized delivery systems.

• IND submission targeted for Q4 this year, with a global first-in-human Phase I/II study planned to establish safety and optimal dosing.

• Initial clinical trials to start with adults, with rapid expansion to pediatric patients based on strong KOL support.

Safety, regulatory, and manufacturing considerations

• Off-target editing risk assessed using three orthogonal methods; no detectable off-target effects or translocations observed.

• Manufacturing process already scaled for pivotal stage, supporting both clinical and future commercial needs.

• Durability data to be included in registrational studies, with ongoing FDA discussions.