Metagenomi Therapeutics, Inc. · NASDAQ:MGX
Metagenomi Therapeutics — 43rd Annual J.P. Morgan Healthcare Conference (January 2025)
15 slides from Metagenomi Therapeutics's conference presentation, Jan 16, 2025
Slide 1: Unlocking 4 Billion Years of Microbial Evolution to Create Curative Genetic Medicines
43rd Annual J.P. Morgan Healthcare Conference Nasdaq: MGX January 2025 Unlocking 4 Billion Years of Microbial Evolution to Create Curative Genetic Medicines
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: Our Vision: Harness the power of our metagenomics platform
3 Our Vision: Harness the power of our metagenomics platform to create curative genetic medicines for patients Programmable Genome Editing Tools Precise Human Gene Modification Curative Genetic Medicines AI-Driven Metagenomics Platform
Slide 4: The metagenomics platform is the foundation of our gene editing toolbox
Proprietary Sampling Exploring diverse microbe- rich ecosystems to extract DNA from environmental samples AI-powered Screening Leveraging AI, ancestral reconstruction, proprietary algorithms, robotics, and automation to reveal novel cellular machinery Complete Genome Editing Capabilities Building a
Slide 5: Our proprietary toolbox enables precise edits of the human genome
Our proprietary toolbox enables precise edits of the human genome Programmable chassis with additional effector enzymes to cause single nucleotide changes Knockdown, knock-in, exon skipping Single nucleotide changes Proprietary library of highly precise and efficient nucleases, including ultra-small
Slide 6: Pipeline by Indication / Editing Target
Editing Platform Delivery Indication / Editing Target Discovery Lead Optimization IND-Enabling Clinical LIVER LNP + AAV Hemophilia A / ALB Knock-in Undisclosed secreted protein diseases Knockdown LNP Transthyretin Amyloidosis / TTR Refractory Hypertension / AGT Undisclosed cardiovascular disease Und
Slide 7: 2024 milestones and key publications
Hemophilia A Program Cardiometabolic Programs Other Therapeutic Programs / Technology Development Declared MGX-001, wholly-owned Development Candidate (DC) Factor VIII activity sustained in NHP study for more than 16 months presented at ASH 1 Initial regulatory engagement with FDA GxP Manufacturing
Slide 8: Hemophilia A Disease Background
Disease Background Most common X-linked inherited bleeding disorder; vast majority of patients are male Caused by large variety of mutations in the Factor VIII (FVIII) gene leading to loss of functional FVIII protein Intracranial bleeding is of greatest concern as this can lead to major morbidity an
Slide 9: Genome editing approach for hemophilia A
Genome editing approach MG nuclease creates efficient and specific cut at safe harbor locus in albumin gene Factor VIII donor DNA is inserted at cut site Strength of albumin promoter provides high level of FVIII expression even at low integration rates Mechanistically different from AAV gene therapy
Slide 10: Durable FVIII activity achieved in non-human primates
Mouse dose dependent FVIII activity Bioengineered FVIII construct used in MGX-001 has higher activity than wild-type FVIII Durable FVIII activity achieved in non-human primates (NHP) Wild-type FVIII activity levels sustained for 16.5 months (Data cutoff: 11-13-24) Days post LNP dosing cFVIII Activit
Slide 11: No off-target editing observed
Potential OT site discovery No off-target editing observed On target 1x - Control 2x - Control No off-target editing observed across multiple hepatocyte donors Hemophilia A Data Source: “Specific and efficient genome editing with metagenomics-derived tools for in vivo and ex vivo therapeutic applica
Slide 12: Addressing secreted protein diseases with large gene insertion
Target protein in mice can achieve normal circulating levels with multiple construct designs Addressing secreted protein diseases with large gene insertion Secreted Protein Deficiencies AAV delivers donor DNA LNP delivers nuclease mRNA and guide targeting albumin site mRNA splicing Tx Gene Tx Gene A
Slide 13: 95% protein knockdown in spontaneous hypertensive rats
95% protein knockdown in spontaneous hypertensive rats Dose dependent editing, mRNA and protein knockdown in primary human hepatocytes >85% editing, 90% mRNA and protein knockdown Refractory Hypertension Rodent proof-of-concept achieved Partnered with mRNA, gRNA, and LNP were optimized to increase p
Slide 14: Anticipated milestones allow advancement towards the clinic
2025 2026 Hemophilia A Program Complete ongoing NHP durability study Conduct Pre-IND and ex-US regulatory meetings IND / CTA filings Cardiometabolic Programs Other Therapeutic Programs / Technology Development Continue to advance early-stage pipeline for multiple future IND filings Nominate 1-2 DCs
Slide 15: Metagenomi: Pipeline advancing, positioned for success
Metagenomi: Pipeline advancing, positioned for success Well capitalized with cash runway into 2027 Harnessing the power of our metagenomics platform to create curative genetic medicines for patients Advancing cardiometabolic programs with Ionis Progressing additional wholly owned therapeutic candida
Metagenomi — 43rd Annual J.P. Morgan Healthcare Conference (January 2025) on 6ix