Metagenomi Therapeutics, Inc. · NASDAQ:MGX
Metagenomi Therapeutics — Investor Presentation (November 2025)
21 slides from Metagenomi Therapeutics's investor presentation, Nov 12, 2025
Slide 1: Company presentation November 2025
Company presentation November 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 1996. All statements other than statements of historical facts contained in this presentation are forward looki
Slide 3: From novel genome editing systems to curative therapies
From novel genome editing systems to curative therapies y sl" Metagenomi ~ Metagenonmi is 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
Slide 4: Strategic evolution focuses resources for advancement
Strategic evolution focuses resources for advancement Prioritizing high impact programs Transitioned to clinical development leadership * Advancing wholly-owned MGX-001 hemophilia A program toward IND/CTA by Q4'26 * Advancing additional secreted protein deficiency indications + Jian Irish, Ph.D., M.
Slide 5: Proprietary genome editing beyond CRISPR/Cas9
Proprietary genome editing beyond CRISPR/Cas9 20,000+ signature editing system from Metagenomi's database Nucleases Enabling gene knockdown, large deletions and large gene integration RIGS Enabling gene correction ofl! naspaa: Mex Base editors Enabling single base correction CAST systems Enabling la
Slide 6: Undisclosed cardiovascular disease
Undisclosed cardiovascular disease
Slide 7: Focused on generating clinical data in 2027
Focused on generating clinical data in 2027 2025 2026 2027 ioe A C Pr sor for 2027 cinical enti ) C intinte Phase 1 caaicon trial +) _— Conduct Pre-IND regulatory spare to vy meeting Disclose lead indication for secreted Secreted protein protein deficiency platform deficiencies Achieve NHP PoC to va
Slide 8: Expanding pipeline — precision genetic medicines
Expanding pipeline — precision genetic medicines Current partnering IONIS efforts include: * MGX's in vivo genome editing wus complements lonis leadership in cardiometabolic space. AGT + 4 targets: two co-development : and co-commercialization Undisclosed options. Undisclosed * Multi-billion dollar
Slide 9: MGX-001 Program
MGX-001 Program November 11, 2025
Slide 10: New data supports MGX-001 advancing toward the clinic
New data supports MGX-001 advancing toward the clinic Demonstrated curative Factor Vill activity Potential competitive with best-in-class treatment potential advantages of MGX-001 * Dose-dependent efficacy of both AAV and LNP * Therapeutically relevant FVIII activity in each animal treated in all bu
Slide 11: Hemophilia A: a validated target waiting for a durable cure
Hemophilia A: a validated target waiting for a durable cure Current SOC: Factor Villreplacement therapy + IV typically dosed 1 - 3 times/week * Significant adherence challenges Annual treatment cost®: ~$565K - $750K Fvill * Risk of breakthrough bleeding * Chronic treatment, non-curative Lifetime Bi-
Slide 12: 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 MGX-001 is uniquely suited for patients of all ages: for genome editing approach: Technology: Durability: Monogenic and well-characterized biology proprietary Clear biomarker-disease link Reg
Slide 13: MGX-001 mechanism leverages natural promoter
MGX-001 mechanism leverages natural promoter i . LNP delivers i i j AAV deli guide RNA to albumin : MGX-001 Fval one (jommmmnen{}— Promoteriess nuclease mRNA gene (sgRNA) i . FVill gene & sgRNA targetin: ; components: (donor DNA): ian nuclease mRNA (MG29-1) Fvili = \ protein DNA DNA DNA DNA DNA AAV
Slide 14: NHP study designs
NHP study designs Step1 be Oo, Qiaw une Baseline r Durability study. Dose range finding study ~ N=3 NHP FVIII N=24. Human FVIII Study duration: 19 months @) +4 @®) Study duration 3 weeks. @) 4+ (») , FVIII evaluation: day 5, 8, 11 , AAV dose LNP dose AAV dose LNP dose (va/kg) (ma/kg) (va/kg) (ma/kg)
Slide 15: Dose dependent FVIII activity in NHP
Dose dependent FVIII activity in NHP: 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: thrombosis risk 1505 15
Slide 16: 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 400 360+ 350 300-4 an 2 250 > 250-5 > 2 2 200 5 2007) rE = 2 150 160 a 4 * 100-4 50 8° : 7 7 ° T r r T ° 10 20 30 40 o bed 20 bad “0 Days post
Slide 17: Durable, therapeutic levels of FVIII achieved in NHP
Durable, therapeutic levels of FVIII achieved in NHP NHP durability study: 1284 1005 a 2 1001 2 754 i < 50: = sox < 6 1003 26 16% 1002 ° 0 50 100 150 200 250 300 350 400 450 500 550 600 Days post LNP dosing ofl" naspaa: Mex # at least 3 independent assay oted to the treatment Plasma FVIII activity l
Slide 18: 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: 10 200 _, ed oF 3 t00 t t x io tithe ttt; a to © 2 4 6 8 & 80 120-160 200 248 290 320 200 408 440 480 520 500 «00 4 90 130 990 250
Slide 19: No genotoxicity observed with MGX-001 development candidate
No genotoxicity observed with MGX-001 i candidate Discovery of potential No validated off target off-target sites sites observed 1. In silico off-target 3 independent primary human hepatocyte donors: = — se} e High genome s | P, integrity maintained a as observed 3 in off-target editing, 2. Biochemi
Slide 20: APPENDIX
APPENDIX fl" Metagenomi
Slide 21: Team
Team Executive Leadership: Board of Directors: Jian Irish, PhD, MBA Alan Brooks, PhD Willard H. Dere, MD s President and CEO SVP, Research Professor Emeritus, 5 Internal Medicine, University of Utah AMGEN ~WKite eo) (3) CASEBIA Eric Bjerkholt, MBA CFO, Mirum Pharmaceuticals, Inc. Pamela Wapnick, MBA