Transcode Therapeutics, Inc. · NASDAQ:RNAZ
Transcode Therapeutics — Investor Presentation (May 2025)
35 slides from Transcode Therapeutics's investor presentation, May 23, 2025
Slide 1: RNA Therapeutics to Deliver a Cancer-Free Future
RNA THERAPEUTICS TO DELIVER A CANCER-FREE FUTURE May 23, 2025
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: Critical Need for Therapy Targeting Advanced Cancer
Problem Critical Need for Therapy Targeting Advanced Cancer Low Survival if Diagnosed at Metastatic (Distant) Stage 90% of Cancer Deaths Due to Metastasis Metastatic Cancer Reduces 5-year Survival $136.9B global market by 2032 *Source: 2019 American Cancer Society, Inc., Surveillance Research; Inter
Slide 4: Our Solution Lead Candidate: TTX-MC138
Our Solution Lead Candidate: TTX-MC138 Type: RNA-based therapeutic (an antagomir) designed to bind to a specific microRNA and block its function. Target: microRNA-10b. A master regulator of metastatic cancer Mechanism: inhibits microRNA-10b, blocking multiple cancer pathways Indication: advanced sol
Slide 5: Untapped Opportunity to Target RNA in Oncology
RNA Solution Untapped Opportunity to Target RNA in Oncology Only 20% of known cancer-causing genes can be targeted using current approaches Using RNA, any gene can be targeted thus opening up every cancer-causing gene for therapeutic targeting TransCode has built a drug design engine that harnesses
Slide 6: RNA Landscape
RNA Landscape siRNA $1.7b Novartis acquisition of Regulus to advance novel microRNA inhibitor, with $800M upfront for Phase 3- ready kidney drug Bayer and NextRNA Therapeutics enter strategic IO ASO collaboration to develop small molecules targeting long non-coding RNAs (lncRNAs) in oncology Biorche
Slide 7: Drug Design Engine TTX
DRUG DESIGN ENGINE TTX
Slide 8: Tunable Drug Design Engine
TTX Tunable Drug Design Engine Our TTX carrier can be customized along a variety of dimensions, including payload, size and PK elements Nucleic acids Peptides Radionuclides Small Molecules Payload diversity Nanometers Microns Size Non-cleavable pH sensitive Tissue-specific Time-sensitive Smart relea
Slide 9: TTX-MC138
TTX-MC138 Lead Therapeutic Candidate: AntagomiR targeting microRNA-10b 9
Slide 10: microRNA-10b: A Driver of Metastasis
Validated Biomarker microRNA-10b: A Driver of Metastasis Clinical evidence demonstrated in >200 peer-reviewed publications over the last ten years • Biomarker of metastasis, higher cancer risk and poor survival outcomes • Linked to metastatic progression in multiple cancer indications including Brea
Slide 11: Pleiotropic Effects of miR-10b Driving Metastasis
Pleiotropic Effects of miR-10b Driving Metastasis TTX-MC138 Inhibits miR-10b Thus Blocking Metastasis Through Multiple Downstream Pathways 11
Slide 12: Clinical Validation of miR-10b as a Target
Clinical Validation of miR-10b as a Target: High Hazard Ratios (HR) Correlate with High miR-10b Expression Hazard Ratios for Overall Survival Based on High vs. Low miR-10b Expression 12
Slide 13: TTX-MC138: Designed to Inhibit miR-10b and Eliminate Metastasis
MOA TTX-MC138 : Designed to Inhibit miR-10b and Eliminate Metastasis TTX-MC138 binds to miR-10b target inside Metastatic tumor cells causing its inhibition Lesion TTX-MC138 TTX-MC138 microRNA-10b TTX-MC138 delivered to metastatic lesions infiltrating miR-10b inhibition has been shown miR-10b inactiv
Slide 14: Mechanism of Delivery to Tumors and Metastases
Mechanism of Delivery to Tumors and Metastases Hemodynamic targeting • TTX is long-circulating (24-30 hours) - distributes throughout the microcirculation of tumors and metastases • Small hydrodynamic size - easily extravasates from the vascular endothelium (the inner cellular lining of arteries, ve
Slide 15: TTX-MC138 - Evidence Of Durable Regressions (Murine Models)
TTX-MC138 - Evidence Of Durable Regressions (Murine Models) Human (Stage II/III) or mouse (Stage IV) triple negative breast cancer cells implanted orthotopically into mice Mice were treated with TTX- MC138 after formation of metastasis 100% (Stage II/III) and 65% (Stage IV) animals regressed disease
Slide 16: Pre-Clinical Efficacy of TTX-MC138 in Pancreatic Cancer Model
Pre-Clinical Efficacy of TTX-MC138 in Pancreatic Cancer Model • Human pancreatic cancer cells (BxPC3) implanted orthotopically into mice • Mice were treated with TTX-MC138 after tumor formation • Metastatic incidence was inhibited by 50% relative to standard-of-care chemotherapy • TTX-MC138 displaye
Slide 17: TTX-MC138 Efficacy in Spontaneous Feline Mammary Carcinoma
TTX-MC138 Efficacy in Spontaneous Feline Mammary Carcinoma TTX-MC138 accumulated in metastatic lesions TTX-MC138 remained in the tumor cells and demonstrated PD activity 3 months after injection Animal was scheduled for euthanasia the week after initial treatment, but after just one dose, gained wei
Slide 18: TTX-MC138 Prevention of Metastatic Breast Cancer
TTX-MC138 Prevention of Metastatic Breast Cancer Human breast cancer cells implanted orthotopically into immunocompromised mice One cohort then treated with MN-anti-miR10b (TTX-MC138) prior to formation of metastases None of the treated animals formed metastases By contrast, control animals treated
Slide 19: TTX-MC138 is Effective in Multiple Cancer Types
TTX-MC138 is Effective in Multiple Cancer Types Sensitivity to TTX-MC138 Profile of response across cell lines was tested in 624 human from different tissues of origin. The cell lines representing response to TTX- metastatic and non- MC138 is shown as Emax (maximum metastatic cancers. effect observe
Slide 20: Pipeline
Multiple Modalities Pipeline TTX-RIGA - intended to activate the RIG-I signaling pathway in turn triggering an immune response that targets cancer. TTX-siPD-L1- incorporates a siRNA against PD-L1 as its functional component to inactivate PD-L1 at the post-transcriptional level, that is, it triggers
Slide 21: Phase 0 Completed 2023; Phase I Clinical Trial Ongoing
Phase 0 First-In-Human (FIH) Clinical Trial Completed 2023 Phase I Clinical Trial Ongoing 21
Slide 22: Phase 0: 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 No safety issues and absence of any allergic hypersensitivity-related adverse e
Slide 23: Phase 0: TTX-MC138 Proven Clinical Activity at Microdose Levels
Phase 0: TTX-MC138 Proven Clinical Activity at Microdose Levels Patient blood collected pre-dose and up to 30-hrs post- dose with 90 micrograms of TTX-MC138 qRT-PCR shows that the drug has remarkable pharmacodynamic activity in patient blood with target inhibition of 66% compared to pre-dose p.i. =
Slide 24: Phase 0 Results
Phase 0 Results Delivery Successful Delivery to Metastatic Lesions - PET-MRI imaging confirmed accumulation in metastatic tumors PK Pharmacokinetic Consistency - Drug circulation and tissue distribution similar to preclinical models Target Single microdose reduced miR-10b levels in blood by 66% Enga
Slide 25: TTX-MC138 Clinical Development Plan
TTX-MC138 Clinical Development Plan (Advanced Solid Tumors) Phase 0 Microdose * Phase 1/2 (All comers) Dose Escalation Phase 1/2 (3 Indications) Dose Expansion Phase 1/2 (First indication) Phase 2 Pivotal Simon 2 Stage (First Indication) Phase 2 Dose Optimization (First Indication) Add On Indication
Slide 26: Phase I/II Study Design
Phase 1 Phase I/II Study Design 1a: Dose Escalation BOIN Design: All comers 1b: Dose Expansion Dose 4 4.8mg/kg Expansion Cohort Dose 3 N = 20 3.2mg/kg Dose 2 Indications: 3 1.6mg/kg Dose 1 Backfill 0.8mg/kg Backfill 2a: Dose Expansion: Simon Two Stage (S2S) If # Responses > R then Reject Ho, # Respo
Slide 27: Phase 1a and 1b: Trial Design
Phase 1a and 1b: Trial Design INDICATION PHASE 1a PHASE 1b Dose Expansion Advanced Solid Tumors Escalating Dose Levels 1 Cohort; N = 20 Indication: All comers Indications: CRC, NSLC, BC; molecular Design: Bayesian Optimal Interval diagnosis, high recurrence risk (BOIN) N ≤ 18+14 Dose level pending P
Slide 28: TTX-MC138-002: Phase 1a Preliminary Trial Results
Phase 1 TTX-MC138-002: Phase 1a Preliminary Trial Results • No significant safety or dose limiting toxicities reported in Cohorts 1, 2, 3 or 4 dosed at 0.8, 1.6, 3.2 and 4.8 mg/kg every 28 days. • Out of 15 patients treated with TTX-MC138 in the first four cohorts, 10 remain on study for continued t
Slide 29: Company
Company 29
Slide 30: History
History Scientific Co-founders are Former Professors of Radiology at Harvard Medical School 12 years of R&D and optimization before company formation Two major discoveries during this period: 1. Identified RNA Molecule responsible for metastatic progression and survival of metastatic tumor cells in
Slide 31: Company Timeline
Overview Company Timeline ACHIEVEMENTS 2016 2018 2021 2022 2023 2024 Company License obtained IPO FDA approval to Phase 0 results showed Initial Dosing in Formation from proceed with delivery to metastases and Phase 1a trial Massachusetts Phase 0 clinical PD activity General Hospital trial with lead
Slide 32: Experienced Management Team and Board of Directors
Experienced Management Team and Board of Directors Tom Fitzgerald, MBA Zdravka Medarova, PhD Susan Duggan, RN, MBA Tania Montgomery, MBA Interim CEO; CFO; Director Founder and CSO Sr. VP of Operations VP Business Development 30+ years experience in Internationally recognized geneticist 28+ years of
Slide 33: Leading Advisors with both Oncology and RNA Expertise
Leading Advisors with both Oncology and RNA Expertise Keith Flaherty, MD Anna Moore, PhD Raghu Kalluri, MD, PhD George Calin, MD, PhD Lubo Nechev, PhD Professor of Medicine & TransCode Founder & Director Professor and Chairman Co-Director, The RNA Interference Chief CMC Officer Director of Clinical
Slide 34: Publications
Publications References by TransCode's scientific co-founders • Anna Moore, N A. Savan, Paulo V. Saavedra, Alan Halim, Vilma • Yoo B, Fuchs BC and Medarova Z: New Directions in the Study and Treatment of Yuzbasiyan-Gurkan, Ping Wang, Byunghee Yoo, Matti Kiupel, Lorenzo Metastatic Cancer. Frontiers i
Slide 35: TransCode Therapeutics
TRANS CODE THERAPEUTIC S™
TransCode Therapeutics — Investor Presentation (May 2025) on 6ix