Slide 1: RNA Therapeutics to Deliver a Cancer-Free Future
RNA Therapeutics to Deliver a Cancer-Free Future NASDAQ: RNAZ October 28, 2024
Slide 2: Forward Looking Statements
Forward Looking Statements Before you invest in the securities of TransCode Therapeutics, Inc. (“TransCode” or the “Company”), you should read TransCode’s filings with the U.S. Securities and Exchange Commission (“SEC”) for more complete information about the Company. You can obtain these documents
Slide 3: TransCode's Innovative Solution to Metastatic Cancer Using RNA
TransCode’s Innovative Solution to Metastatic Cancer Using RNA Unique Approach Lead Candidate • Proprietary nanoparticle delivery platform • TTX-MC138 targets miR-10b, an important invented at Harvard Medical School 01 oncogene in metastatic cancer designed to overcome decades of RNA delivery challe
Slide 4: Critical Need for an Effective Therapy Against Metastatic Cancer
CriticalNeed Critical Needfor forAn AnEffective Effective TherapyAgainst Therapy AgainstMetastatic MetastaticCancer Cancer Five-Year Survival Metastatic cancer is essentially incurable Of the 10 million cancer deaths annually worldwide, ~90% are due to metastasis ~$137B global market by 2032 4
Slide 5: Delivery Challenges / Opportunities
Delivery Challenges / Opportunities About 80% of currently known oncology targets are undruggable using monoclonal antibodies (mAbs) and/or small molecules Engaging these targets using RNA/DNA is possible, provided that the RNA/DNA molecules can be delivered to cancer cells in humans TransCode’s pro
Slide 6: TransCode's Tunable TTX Delivery Platform
TransCode’s Tunable TTX Delivery Platform Tunable Nanoparticle Platform • Size customizable according to Customizable Coating desired pharmacokinetics • Stabilizes nanoparticles • Long circulation half-life • Protects cargo from degradation • Avoids early kidney and liver • Promotes tumor uptake and
Slide 7: Multiple First-in-Class RNA Therapeutic Candidates
Multiple First-in-Class RNA Therapeutic Candidates Candidate Strategic Modality Disease Indication Preclinical IND Enabling Phase 0 Phase 1 Phase 2 Phase 3 Partner Metastatic Cancer TTX- MC138 Internal Antisense *Pancreatic Cancer TTX - siPDL1 Internal RNAi *Pancreatic Cancer TTX - RIGA Internal PRR
Slide 8: Lead Candidate: TTX-MC138
Lead Candidate: Critical Need for AnTTX-MC138 Effective Therapy Against Metastatic Cancer First-in-Class Therapeutic Candidate Targeting Metastatic Cancer Target: miRNA-10b, an RNA critical in metastatic cancer miRNA-10b: • linked to metastatic disease in >200 clinical studies in cancer patients • s
Slide 9: Phase 0 Preliminary Results - Scans
Phase 0 Preliminary Results - Scans PET-MRI To Determine Drug Delivery FDG PET-MRI 64Cu-TTX-MC138 PET-MRI • Female, Stage IV, metastatic breast cancer. Metastatic sites: bone, liver, lungs • Before dosing with TTX-MC138, FDG PET-MRI was used to locate metastatic lesions (red arrows in larger image)
Slide 10: Phase 0 Preliminary Results - Blood
Phase 0 Preliminary Results - Blood qRT-PCR To Determine Drug Functionality TTX-MC138 Preliminary Results • Accumulation in the metastatic lesions (red arrows in scans) • Drug functionality/target engagement (patient blood) • No safety issues and absence of any allergic hypersensitivity related adve
Slide 11: TTX-MC138 Phase I/II Trial Design
TTX-MC138 Phase I/II Trial Design TTX-MC138 Phase I/II clinical trial designed to: - Assess safety - Determine recommended Phase 2 study dose - Early tumor activity - Results in different types of metastatic tumors Screening Phase 1a Phase 1b Escalating Dose Levels Dose Expansion Advanced Solid Tumo
Slide 12: Enrolling TTX-MC138 Phase I/II Trial
Enrolling TTX-MC138 Phase I/II Trial Design: Open-label, multicenter, dose-escalation Primary Objectives: Evaluate safety and tolerability Determine maximum tolerated dose (MTD) Select recommended Phase 2 dose Secondary objectives: Characterize pharmacokinetics and pharmacodynamics Exploratory Objec
Slide 13: TTX-MC138 Clinical Program Overview
TTX-MC138 Clinical Program Overview 2024 2025 2026 2027 2028 2029 Q1-4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Metabolite Studies Phase 2 Rat and Dog Tox Non-Clin. $100,000 $2,000,000 Clinical Phase 1A Phase 1B $3,000,000 $3,000,000 Phase 2 $22,000,000 Regulatory IND 166264 End P
Slide 14: Recent and Expected Milestones
Recent and Expected Milestones 2024 2025 ✓TTX-MC138 IND • TTX-MC138 Phase 1a completion ✓TTX-MC138 Phase 0 • TTX-MC138 Phase 1b initiation completion • TTX-MC138 Phase 0 publication ✓TTX-MC138 Phase 1a • TTX-MC138 Partnership initiation • TTX Partnership(s) ✓Second NIH Grant ($2M) • Grants ✓Debiopha
Slide 15: Capitalization
Capitalization Source of Capital Amount NASDAQ: RNAZ Sept 30, 2024 Seed Capital (Angel investors) $2,240,000 Common Stock 17,265,658 SBIR Grants 4,309,000 Warrants (WAEP/Sh $2.49) 12,231,491 IPO (Net Proceeds) 25,400,000 Options (WAEP/Sh $2.70) 1,935,813 Equity Financings (2023, 2024) (Net Proceeds)
Slide 16: Team of Experts
Critical Need Teamfor of An Effective Experts Therapy Against Metastatic Cancer TransCode’s senior leadership combines decades of oncology drug discovery and development expertise, bringing both scientific insight and valuable strategic perspective Executive Team Independent Directors Key Advisors P
Slide 17: Back-up Slides
TRANSCODE THERAPEUTICS™
Slide 18: Value-Generating Strategic Collaborations
Value-Generating Strategic Collaborations Candidate Partner Program Targeted TTX-RNA Debiopharm Testing in vitro delivery of RNA inside tumor cells TTX-mRNA Undisclosed mRNA delivery to tumors Development of cell-killing G-dase E, a Class 2 CRISPR TTX-CRISPR (BEC) Akribion Genomics nuclease, with Tr
Slide 19: Patents and Applications
Patents and Applications Cover both composition of matter and methods claims Technology Geography Expiration Patents/Applications Notes TTX for Payload Delivery US, EU, CA, CN, KR 2039 WO2021/113829 Payload delivery Polynucleotide and polypeptide Nanosensor 75% of World 2043 US10,086,093; EP 2 961 3