Slide 1: Pioneering Immuno-Oncology and RNA Therapeutics for Treatment of High Risk and Advanced Cancers
PIONEERING IMMUNO- ONCOLOGY AND RNA THERAPEUTICS FOR TREATMENT OF HIGH RISK AND ADVANCED CANCERS NASDAQ: RNAZ
Slide 2: Company Mission
Company Mission We are pioneering complementary approaches that redefine the boundaries of oncology innovation — from RNA-targeted therapeutics that precisely reach tumors to vaccine immunotherapies that mobilize the immune system to recognize and destroy cancer cells. 2
Slide 3: Forward Looking Statement
Company Forward Looking Statement 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 doc
Slide 4: Important Additional Information and Where to Find It
Company Important Additional Information and Where to Find It The Company, its directors and certain of its executive officers are deemed to be participants in the solicitation of proxies from the Company’s stockholders in connection with the Company’s expected special meeting seeking stockholder ap
Slide 5: Company Background
Company Background Founded in 2016 to develop treatments for advanced cancer Scientific Co-founders - Professors of Radiology at Harvard Medical School IPO 2021; net invested to date (financings; grants) ~$101.6 million Three major pathways for value creation: TTX-MC138 Seviprotimut-L TTX Acquired S
Slide 6: Experienced Management and Board of Directors
Experienced Management and Board of Directors Philippe P Calais, PhD Zdravka Medarova, PhD Michel Janicot, PhD CEO Founder and CSO Senior Development Internationally Officer Member of multiple Deep global recognized geneticist international scientific experience in the and non-coding RNA boards and
Slide 7: Leading Advisors with Oncology and RNA Expertise
Leading Advisors with Oncology and RNA Expertise Board of Directors Elizabeth Czerepak, MBA Magda Marquet, PhD Philippe P Calais, PhD Director Director Chairman CEO, Mirror Biologics Co-founder Acurion ad CEO, TransCode Therapeutics ALMA life Sciences Tom Fitzgerald ,MBA Erik Manting, PhD Director D
Slide 8: Critical Need for Effective Therapies Targeting Advanced Cancer
Problem Critical Need for Effective Therapies Targeting Advanced Cancer >90% Low Survival if Diagnosed at Advanced Stage of Cancer Deaths Due to Advanced Disease Advanced Cancer Reduces 5-year Survival Key Challenge to Treatment Dissemination to distant sites, immune evasion, resistance Global Marke
Slide 9: Pipeline
Multiple Modalities Pipeline 9
Slide 10: TTX Tunable Drug Design Engine
TTX Tunable Drug Design Engine TTX can be customized according to predesigned specifications Nucleic acids Peptides Radionuclides Small Molecules Payload diversity Nanometers Microns Size Non-cleavable pH sensitive Tissue-specific Time-sensitive Smart release linkers Polymers Biomolecules (e.g. gluc
Slide 11: TTX-MC138
Lead Candidate TTX-MC138 TTX-MC138 Lead Therapeutic Candidate: AntagomiR targeting microRNA-10b 1 1
Slide 12: TTX-MC138
Lead Candidate TTX-MC138 Type: Nucleic acid-based therapeutic designed to bind to a specific microRNA and block its function Target: microRNA-10b, a master regulator of metastatic cancer Mechanism: inhibiting microRNA-10b blocks multiple cancer pathways Indication: advanced solid tumors and micromet
Slide 13: Target microRNA-10b: A Driver of Metastasis
Target microRNA-10b: A Driver of Metastasis Select miR-10b Molecular Pathways TWIST1 Clinical evidence MICB demonstrated in >300 peer- reviewed publications over the PD-L1 TET2 miR-10b TTX-MC138 last ten years • Biomarker of metastasis, higher cancer risk and poor TBX5 HOXD10 NF1 survival outcomes •
Slide 14: TTX-MC138 Clinical Development Plan: Metastatic Disease
Lead Candidate TTX-MC138 Clinical Development Plan: Metastatic Disease Phase 1a Phase 2a Phase 2 Indications: Stage 1-3 colorectal Indications: Advanced solid tumors Indications: Pending Phase 2a adenocarcinoma, with molecular disease Design: Bayesian Optimal Interval results (ctDNA positive), follo
Slide 15: Radiolabeled TTX-MC138 Phase 0 Purpose & Results
Phase 0 Radiolabeled TTX-MC138 Phase 0 Purpose & Results To derisk further clinical development by demonstrating delivery to Purpose metastases and to frame dose and dosing schedule based on pharmacokinetics and tissue distribution Delivery Successful Delivery to Metastatic Lesions - PET-MRI imaging
Slide 16: TTX-MC138 Accumulation in Metastatic Lesions Confirmed
Phase 0 TTX-MC138 Accumulation in Metastatic Lesions Confirmed FDG PET-MRI TTX-MC138 PET-MRI Phase 0 microdosing study with radiolabeled TTX-MC138 Stage IV, metastatic breast cancer. Metastatic sites: bone, liver, lungs PET/MRI at 2, 3, 6 and 24 hours post-dosing shows TTX-MC138 accumulation in the
Slide 17: TTX-MC138 Phase 1a Trial - Safety and PK
Lead Candidate TTX-MC138 Phase 1a Trial- Safet and PK TTX-MC138 was administered to 16 patients, 77 total doses to date. No DLTs or significant safety events across all doses tested- 0.8-4.8mg/kg. AEs included infusion related reactions, grade 1 and 2 PK analysis suggests that the three highest dose
Slide 18: TTX-MC138 Phase 1a Trial - Preliminary Clinical Activity
Lead Candidate TTX-MC138 Phase 1a Trial – Preliminary Clinical Activity Median PFS is 4 months with the longest to date over 12 months 44% of patients had durable responses Preliminary PK analysis demonstrates evidence of dose response One patient diagnosed with thyroid cancer and historic evidence
Slide 19: Seviprotimut-L
Lead Candidate Seviprotimut-L Seviprotimut-L Lead Therapeutic Candidate: Cancer Vaccine 19
Slide 20: Seviprotimut-L Polyvalent Shed Antigens Vaccine for Melanoma
Lead Candidate Seviprotimut-L Seviprotimut-L is a novel polyvalent shed antigens vaccine for the adjuvant treatment of Stage IIB and IIC melanoma patients 60 years and younger • FDA “Orphan Drug” status • Successful completion of adaptive Phase 3 clinical trial “MAVIS” • FDA “Fast Track” designation
Slide 21: Key MAVIS Study Design Elements
Phase 3 Seviprotimut-L: Clinical Trial Design Key MAVIS Study Design Elements Study Drug • 40 µg seviprotimut (0.8 mL suspension containing 0.05 mg/mL drug substance – shed antigens – plus 20 mg/mL alum in 0.9% saline. Placebo does not include shed antigens) Study Population • Ages 18 to 80, female
Slide 22: Seviprotimut-L: MAVIS Part B1, Efficacy Results
Phase 3 Seviprotimut-L: MAVIS Part B1, Efficacy Results The planned subgroup analysis of Stage IIB/IIC patients (n = 111) revealed trends to longer RFS (HR 0.65 [0.37,1.17]), and OS (HR 0.37[0.13, 1.06]) with seviprotimut-L. RFS, Stage IIB/IIC patients OS, Stage IIB/IIC patients 22
Slide 23: Seviprotimut-L: Competitive Assessment
Potential Seviprotimut-L: Competitive Assessment Two checkpoint inhibitors have shown efficacy for the adjuvant treatment of melanoma in Stage IIB and IIC patients at risk for disease recurrence: Keytruda (Merck) and Opdivo (BMS) have both been approved Advantages of Seviprotimut-L over checkpoint i
Slide 24: IP Portfolio
IP Portfolio Patents and applications cover both composition of matter and methods claims Technology Expiration Patents/Applications Notes Compositions and Methods for 2040 WO2021/113829 Delivery platform for drug Tunable Magnetic Nanoparticles design engine. Covers TTX- MC138 Template Directed Immu
Slide 25: TransCode Company Timeline: Achievements & Milestones
TransCode Company Timeline: Achievements & Milestones License obtained from eIND- FDA Study May Proceed IND- FDA Study May Achievements Massachusetts General with Phase 0 clinical trial with Proceed with Phase 1a Hospital radiolabeled TTX-MC138 with TTX-MC138 . 2016 2018 2021 2022 2023 2024 Company
Slide 26: The Next 18 Months
Timeline The Next 18 Months 2H:2025 2026 Clinical Goals Phase 1a (TTX-MC138) Dose Escalation Completion and data Phase 2a (TTX-MC138) study initiation analysis Phase 2a (TTX-MC138) Dose Expansion FDA Protocol Submission and IRB Review Potential News Flow Completion of Dose-Escalation Phase 1 Clinica
Slide 27: Publications
Publications • Anna Moore, N A. Savan, Paulo V. Saavedra, Alan Halim, Vilma Yuzbasiyan- • Yoo B, Fuchs BC and Medarova Z: New Directions in the Study and Treatment of Gurkan, Ping Wang, Byunghee Yoo, Matti Kiupel, Lorenzo Sempere, Zdravka Metastatic Cancer. Frontiers in Oncology Volume 8, Article 25
Slide 28: TransCode Therapeutics
TRANS CODE THERAPEUTIC S™