Metagenomi Therapeutics, Inc. · NASDAQ:MGX
Metagenomi Therapeutics — Investor Presentation (May 2024)
23 slides from Metagenomi Therapeutics's investor presentation, May 14, 2024
Slide 1: Investor Overview Q2 2024
Nasdaq listed (MGX) Non-Confidential Investor Overview Q2 2024 Unlocking 4 Billion Years of Microbial Evolution to Create Curative Genetic Medicines Exhibit 99.2
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: Building the leading genetics medicine company
Building the leading genetics medicine company 3 Non-Confidential Investor Overview Therapeutic translation $175M Series B1 2022 $100M Series B2 2023 $75M Series A 2021 $94M IPO 2024 2025 2026 Anticipated Milestones by 2026 2 IND filings At least 2 additional DCs In vivo proof of concept for large g
Slide 4: A highly differentiated genetic medicines platform
Wholly-Owned IP Diverse enzymes with novel IP & unique characteristics Proprietary library A highly differentiated genetic medicines platform 4 Broad Pipeline Key NHP PoC Durable editing in disease model Saturating in vivo editing Genome Editing Toolbox High efficiency & precision Leader in large ge
Slide 5: Metagenomics powers our discovery platform
AI-enabled, High- throughput screening AI-based cloud computing Proprietary algorithms Robotics & automation Metagenomi Library Highly active & precise editing systems Modular engineering Metagenomics powers our discovery platform Proprietary sampling Diverse climates & geographies Uncovering previo
Slide 6: Metagenomi toolbox designed for any desired gene correction
6 Programmable Nucleases, including ultra-small systems (SMART) (Knockdown, exon skipping) Base Editors, including ultra-small systems (SMART) (Single nucleotide changes) Big RIGS (>100 base pair integrations) Little RIGS (prime editing) (1-100 base pair replacement, insertion, or deletion) G A T C
Slide 7: Highly efficient nucleases designed for any target in the human genome
Efficiency: MG nucleases are selected for their native high efficiency against controls Precision: MG nuclease library expands targeting options within genes of interest, potentially increasing precision Broad targetability: MG nucleases have the estimated potential to target every codon in the huma
Slide 8: Ultra small (SMART) systems expand in vivo delivery
Ultra small (SMART) systems expand in vivo delivery Our SMART systems are ultra small CRISPR nucleases and base editors that create potential advantages for safety, delivery, manufacturing and dosing Published in Nature Communications, December 2022* Size of Small SMART base editors vs Cas9 ABE Cas9
Slide 9: MGX Base Editors greatly expand genome targetability
MGX Base Editors greatly expand genome targetability MGX base editors increased the genome targetability by 5x compared to SpCas9 base editors MGX base editors have broadened the targetability of base editors without losing efficiency or specificity Achieved by swapping the PAM interacting domains o
Slide 10: Multiple options for large, targeted genome integrations
Multiple options for large, targeted genome integrations CAST: DNA-Mediated Integration Systems Big RIGS: RNA-Mediated Integration Systems DNA Template Transposase Reverse transcriptase RNA Template Cargo Target DONOR PAM Junction between genomic target and donor template Junction between genomic ta
Slide 11: Flexibility in delivery expands targetable organs and disease areas
Flexibility in delivery expands targetable organs and disease areas MGX Toolbox designed to have broad compatibility with viral and nonviral delivery technologies Accomplished by a variety of nuclease and gRNA structures, which range in terms of their size and biochemistry Small guides for some type
Slide 12: The ability to edit anywhere in the human genome, with high precision
The ability to edit anywhere in the human genome, with high precision, tailored for each therapeutic indication Non-Confidential Investor Overview
Slide 13: Therapeutic translation pipeline
Delivery Target Editing Approach Indication Discovery Lead Optimization IND-Enabling Clinical Partner Knock in HEMOPHILIA A Knock down PRIMARY HYPEROXALURIA TYPE 1 Knock down TRANSTHYRETIN AMYLOIDOSIS Knock down CARDIOVASCULAR DISEASE (AGT) RIGS/Base Editing ALPHA 1 ANTITRYPSIN DEFICIENCY RIGS WILSO
Slide 14: Hemophilia A: A life-altering bleeding disease with possibility of cure
AAV delivers Factor VIII gene (donor DNA) Genome editing offers the potential for a lifelong cure for hemophilia A morbidity and mortality Estimated prevalence of nearly 30,000 hemophilia A patients in US* Genome editing approach MG nuclease creates highly efficient cut at safe harbor locus in album
Slide 15: A potentially curative gene editing approach for Hemophilia A
Expression at therapeutic levels in all 3 animals Durable FVIII activity demonstrated out to 4.5 months DC nomination planned for mid 2024 12 month FVIII durability data to be presented in 2H 2024 Animal ID INDELS in liver (d7) FVIII gene integration frequency # (copies per 100 genomes) Mean FVIII a
Slide 16: Established platform for large gene integrations
AAV delivers donor DNA template Leveraging Heme A experience to deliver and integrate a variety of target genes MG nuclease creates highly efficient cut at safe harbor locus The LNP component stays fixed and serves as a platform New programs can move quickly by simply substituting the donor DNA carg
Slide 17: Primary Hyperoxaluria Type 1: durable knockdown of HAO1
17 *GO = glycolate oxidase Primary Hyperoxaluria, Type 1 (PH1)—a durable knockdown of HAO1 for substrate reduction therapy Genome Editing Strategy for Targeting HAO1: Non-Confidential Investor Overview Goal: durably knock down HAO1 resulting in stable and permanent reduction of oxalate levels to eff
Slide 18: Dose dependent editing and knockdown of HAO1 in normal mice
Dose Dependent Editing, mRNA Knockdown and Protein Knockdown of HAO1 in Normal Mice after a Single Administration of the Lead Nuclease mRNA and Lead Guide Encapsulated in a LNP with Tropism to the Liver ~70% editing in whole liver = ~100% editing in hepatocytes Mouse PoC shows 90% reduction of targe
Slide 19: Accelerating therapeutic translation
Accelerating therapeutic translation Liver Targets Knock-in & knock-down: Hemophilia A Primary Hyperoxaluria Type I Transthyretin Amyloidosis Cardiovascular Indications (AGT) Gene corrections & integrations: A1AT Deficiency Wilson’s Disease Neuromuscular + CNS Targets Ultra small systems for Knock d
Slide 20: Early investment in automation & manufacturing
Early investment in automation & manufacturing We aim to develop and characterize complex human gene editing components that are essential to pursue a successful regulatory pathway for genetic medicine development by investing in: 1. Integrated computational & high-throughput automated workstreams 2
Slide 21: Management team
Brian Thomas, PhD CEO & Founder Prior to co-founding the company, Dr. Thomas spent more than 20 years in academic research helping to pioneer the field of metagenomics. Dr. Thomas has been cited over 16,000 times and listed as an inventor in 28 patent families. Luis Borges, PhD CSO Spent over 27 yea
Slide 22: Strong Cash Position
22 Strong Cash Position $327.4 million as of March 31, 2024 Cash runway into 2027 Partners Non-Confidential Investor Overview Join us on our journey to transform patients’ lives Anticipated Milestones by 2026 2 IND filings At least 2 additional DCs In vivo proof of concept for large gene integration
Slide 23: Thank you
Thank you 23 Non-Confidential Investor Overview Investor Contact: Simon Harnest, MSc CIO & SVP of Investor Relations simon@metagenomi.co