Metagenomi Therapeutics, Inc. · NASDAQ:MGX
Metagenomi Therapeutics — Corporate Update (November 2025)
17 slides from Metagenomi Therapeutics's corporate update, Nov 11, 2025
Slide 1: MGX-001 Program and Business Update
jenomi pa '~ a, ul" Metag MGX-001 Program and Business Update November 11, 2025
Slide 2: Forward-looking statements
Forward-looking statements This presentation includes forward-looking statements, including forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of historical facts contained in this presentation are forward looki
Slide 3: New data supports MGX-001 advancing toward the clinic
New data supports MGX-001 advancing toward the clinic Demonstrated curative Factor Vill activity with best-in-class treatment potential Potential competitive advantages of MGX-001 * Dose-dependent efficacy of both AAV and LNP * Therapeutically relevant FVIII activity in each animal treated in all bu
Slide 4: Strategic evolution focuses resources for advancement
Strategic evolution focuses resources for advancement Prioritizing high impact programs * Advancing wholly-owned MGX-001 hemophilia A program toward IND/CTA by Q4'26 * Advancing additional secreted protein deficiency indications * Focusing on cardiometabolic collaboration with lonis Streamlined orga
Slide 5: Glenn F. Pierce, MD, PhD
Glenn F. Pierce, MD, PhD was born with severe hemophilia A was cured in 2008 via liver transplant -— Career highlights: Biogen: senior vice president of hematology, cell and gene therapie. Ambys Medicines: co-founder and CMO Voyager Therapeutics: voyager Interim CSO. National Bleeding Disorders Foun
Slide 6: Hemophilia A: a validated target waiting for a durable cure
Hemophilia A: a validated target waiting for a durable cure TE. \ y Current SOC: ww x? ‘ J ¥ Factor Vill replacement therapy FVIII * IV typically dosed 1 - 3 times/week * Significant adherence challenges * Risk of breakthrough bleeding * Chronic treatment, non-curative >» Bi-specific antibody "mimet
Slide 7: MGX-001 mechanism leverages natural promoter
MGX-001 mechanism leverages natural promoter MGX-001 components: AAV delivers promoterless FVIII gene. AAV enters hepatocytes and the donor FVIll cassette localizes to the nucleus. of" naspaa: Mex LNP delivers MG29-1 nuclease mRNA + guide targeting albumin promoter. The mRNA is released inthe cytopl
Slide 8: NHP study designs
NHP study designs Step1 Step2 } T be t AAV LNP Baseline Y) . r Durability study. ~ Dose range finding study ~ N=3 NHP FVIII N=24. Human FVII Study duration: 19 months ‘Oo +4 @® Study duration 3 weeks. ‘Oo +4 Q)) - FVIII evaluation: day 5, 8, 11 , AAV dose LNP dose AAV dose LNP dose (va/ka) (ma/ka) (
Slide 9: Dose dependent FVIII activity in NHP
Dose dependent FVIII activity in NHP: 5c, Development minimally efficacious and clinically relevant doses identified AAV dose response identified LNP dose response identified 0.2 mpk the optimal efficacious dose as the minimally efficacious dose of 5x10” vg/kg: and 0.6 mg/kg as optimal dose: thrombo
Slide 10: Optimal dose group demonstrated encouraging safety profile supporting curative potential
Optimal dose group demonstrated encouraging safety profile supporting curative potential Mild and transient ALT changes: Mild and transient AST changes: 4005 400 3604 350 300-4 300 2 2 os04 5 250 200 2007 rE z 2 150 1604 100 100-4 so 50 ; : : o4 ; : : ; ° 10 20 20 40 ° » 20 30 40 Days post-AAV infus
Slide 11: Durable, therapeutic levels of FVIII achieved in NHP
Durable, therapeutic levels of FVIII achieved in NHP NHP durability study: 1257 100-4 Zz = 1001 2 754 2 i < 50+ 2 40% s 1003 25 sx 1002 o 0 50 100 150 200 250 300 350 400 450 500 550 600 Days post LNP dosing Fill activity values are the mean and standard deviation of at least 3 independant assay run
Slide 12: Treatment well tolerated over month duration of the study
Treatment well tolerated over 19 month duration of the study Alanine transaminase (ALT) levels post LNP infusion: Aspartate transaminase (AST) levels post LNP infusion: 230 tee 300 + hams “ 3 3 100 b T . med © + -“ . 1 a ee ee ee — r °F re tt o2e es ® 2 4 © & ae Be 120 180 200 260 ZAP 20 360 400 449
Slide 13: No genotoxicity observed with MGX-001
No genotoxicity observed with MGX-001 Discovery of potential off-target sites 1. In silico off-target 2. Biochemical off-target discovery: No potential off-targets were discovered in cell-based assays. 3. In cell off-target discovery: No potential off-targets were found. af!" naspaa: Mex No validate
Slide 14: De-risked and clear opportunity for genome editing in Hemophilia A
De-risked and clear opportunity for genome editing in Hemophilia A Hemophilia A is an ideal entry indication for genome editing approach: MGX-001 is uniquely suited for patients of all ages: Technology: Durability: Monogenic and well-characterized biology proprietary Clear biomarker-disease link Reg
Slide 15: Focused on generating clinical data in 2027
Focused on generating clinical data in 2027 Demonstrated curative FVIII activity in NHPs Built upon approximately 19 month durability study data 2025 2026 2027 ——————————————————— ee File IND and CTA; tet. . . iT te Phy 1 clinical trial C Prepare for 2027 clinical entry ) C nitiate Phase t clinical
Slide 16: From novel genome editing systems to curative therapies
From novel genome editing systems to curative therapies al! Metagenomi Metagenomiis an in vivo genome editing company capitalizing on its proprietary technologies to precisely correct a wide range of genetic mutations across the human genome. The company is focused on wholly owned programs in Hemoph
Slide 17: Q&A
Q&A afl! Naspaa: Mex
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