Metagenomi Therapeutics (MGX) investor relations material

Strategic overview and pipeline updates

• Lead program MGX-001 for Hemophilia A is advancing toward an IND submission in Q4, with clinical entry targeted for 2027.

• Proprietary gene editing platform includes over 20,000 systems, enabling high specificity and multiplex editing.

• Pipeline expansion includes metabolic and neuromuscular indications, with ongoing business development for cell therapy applications.

• Collaboration with Ionis covers four cardiometabolic targets, with Ionis controlling data release.

• Cash position at Q2 end was $120.7 million, expected to fund operations through Q4 2027.

MGX-001 program details and differentiation

• MGX-001 uses site-specific gene integration at the albumin locus, aiming for lifelong benefit, especially in children.

• Preclinical studies in non-human primates showed dose-dependent, durable Factor VIII expression at significantly lower AAV doses than prior therapies.

• Durability study demonstrated stable Factor VIII activity for up to 19 months, correlating with gene integration.

• Approach addresses limitations of current therapies by aiming for curative, one-time treatment and reducing lifetime costs.

• Safety profile is enhanced by promoterless AAV cassette and low AAV dosing; preclinical safety data are favorable.

Clinical development and study design

• IND-enabling studies and GMP manufacturing are complete; dossier is being finalized.

• Phase I/II study will be global, single-arm, open-label, starting with adults and expanding to younger patients.

• Dose escalation will begin at biologically active, low doses, with rapid progression expected.

• Strong interest from investigators and treatment centers; patient community is knowledgeable and motivated.

• Data disclosure plans will be clarified post-IND clearance; early Factor VIII activity readouts are anticipated.