Entrada Therapeutics, Inc. · NASDAQ:TRDA
Entrada Therapeutics — Investor Presentation (August 2026)
50 slides from Entrada Therapeutics's investor presentation, August 2026
Slide 1: Corporate Presentation
Corporate Presentation August 2026
Slide 2: Disclaimer
Disclaimer This presentation has been prepared by Entrada Therapeutics, Inc. (“Entrada”) and shall not constitute an offer to sell or a solicitation of an offer to buy securities or an invitation or inducement to engage in investment activity nor shall there be any sale of securities in any jurisdic
Slide 3: Entrada is developing best-in-class genetic medicines to address diseases with a significant unmet need
Entrada is developing best-in-class genetic medicines to address diseases with a significant unmet need Proprietary Modalities Clinical Validation Expanding Pipeline Proprietary active moieties and Favorable patient safety and Advancing programs across novel delivery constructs functional improvemen
Slide 4: Advancing five fully owned programs and one partnered program
Advancing five fully owned programs and Preclinical Phase 1/2 Phase 2/3 one partnered program Duchenne Muscular Dystrophy (DMD) ENTR-601-44 Phase 1/2 data from ex-U.S. DMD studies to support ENTR-601-45 Accelerated Approval ENTR-601-50 regulatory filings in the U.S. ENTR-601-51 d Myotonic Dystrophy
Slide 5: Neuromuscular Programs
THERAPEUTICS Neuromuscular Programs
Slide 6: Entrada's neuromuscular strategy is to clinically de-risk a differentiated portfolio and launch best-in-class drugs
Entrada’s neuromuscular strategy is to clinically de-risk a differentiated portfolio and launch best-in-class drugs Multiple clinical catalysts, growing platform validation and a differentiated pipeline position Entrada for significant value creation Neuromuscular Strategy Clinical Differentiation N
Slide 7: The DMD challenge: Muscle damage and impaired repair
The DMD challenge: Muscle damage and impaired repair An ideal treatment is regenerative – replacing damaged, dystrophic muscle with healthy muscle; Requires satellite cell correction which enables asymmetric differentiation to develop healthy muscle Healthy • Dystrophin supports stabilizing and repa
Slide 8: ELEVATE-44-201 Cohort 1: Study Results Summary
ELEVATE-44-201 Cohort 1: Study Results Summary
Slide 9: Executive summary and key takeaways ELEVATE-44-201 Cohort 1 results
Executive summary and key takeaways ELEVATE-44-201 Cohort 1 results Cohort 1 achieved the objective of demonstrating safety/tolerability; Importantly, Cohort 1 achieved functional benefit at the lowest dose with dystrophin levels expected to increase in Cohorts 2 and 3 • Favorable safety and tolerab
Slide 10: ELEVATE-44-201 Cohort 1: Trial Design and Treatment Population
ELEVATE-44-201 Cohort 1: Trial Design and Treatment Population
Slide 11: ELEVATE-44-201 is designed to support U.S. Accelerated Approval and forms the basis of a global registrational program
ELEVATE-44-201 is designed to support U.S. Accelerated Approval and forms the basis of a global registrational program* ENTR-601-44 ELEVATE-44-201 is a global, two-part, Primary objective: Safety and tolerability of ENTR-601-44 randomized, double-blind placebo- Secondary objectives: Evaluation of ph
Slide 12: Demographics and baseline characteristics
Demographics and baseline characteristics ELEVATE-44-201 ENTR-601-44 Placebo Baseline Characteristics n=2 6 mg/kg n=6 Age, mean 13.5 9.3 Body mass index, mean, kg/m2 17.96 20.00 Corticosteroid use, n (%) 2 (100%) 6 (100%) Ambulatory, n (%) 2 (100%) 6 (100%) Baseline dystrophin 4.6% 4.0% Time to Rise
Slide 13: ELEVATE-44-201 Cohort 1: Safety Data
ELEVATE-44-201 Cohort 1: Safety Data
Slide 14: Safety was consistent and unremarkable at the 6 mg/kg dose
Safety was consistent and unremarkable at the 6 mg/kg dose ELEVATE-44-201 All TEAEs were mild to moderate ENTR-601-44 Placebo • Headache was the most common Participants with ≥1 TEAE, n (%) 6 mg/kg n=2 study drug–related TEAE, reported in n=6 50% of the treatment group and 50% Any TEAE 2 (100%) 6 (1
Slide 15: Renal markers were within normal range and comparable to placebo
Renal markers were within normal range and treated patient observations were comparable to placebo ELEVATE-44-201 eGFR Serum Cystatin C Renal marker results further de-risk current and future DMD programs and support Serum Magnesium Serum Creatinine dose escalation to drive a higher therapeutic inde
Slide 16: ELEVATE-44-201 Cohort 1: Functional Efficacy Data
ELEVATE-44-201 Cohort 1: Functional Efficacy Data
Slide 17: ELEVATE-44-201 Cohort 1 functional benefit summary
ELEVATE-44-201 Cohort 1 functional benefit summary Cohort 1 achieved functional benefit via statistically significant measurements in both TTR and TTRV; 10MWR trending favorably with additional metrics to be shared by year-end 2026 • Improvement in TTR and in TTRV was seen 0.1 across the majority of
Slide 18: ENTR-601-44 delivered a differentiated impact on TTRV
ENTR-601-44 delivered a differentiated impact on TTRV TTRV is a validated registrational endpoint ENTR-601-44’s observed Cohort 1 (n=6) TTRV versus placebo is superior to reported numbers from steroids, gene therapy and second-generation exon skipping therapies 0.1 Reported TTRV Competitive Comparis
Slide 19: ELEVATE-44-201: Pharmacokinetics and Biomarkers
ELEVATE-44-201: Pharmacokinetics and Biomarkers
Slide 20: Pediatric plasma exposures demonstrated target engagement at the lowest dose tested
Pediatric plasma exposures demonstrated target engagement at the lowest dose tested (6 mg/kg) In Cohort 1 dosing, Cmax and AUC observed in pediatric participants (versus adults) have not yet crossed the critical threshold needed to demonstrate high levels of dystrophin Percent Exon Skipping Mean Per
Slide 21: Higher dystrophin levels expected in Cohorts 2 and 3 based on juvenile NHP data
Higher dystrophin levels expected in Cohorts 2 and 3 based on juvenile NHP data Juvenile NHP data suggest an increase of AUC should result in substantially higher dystrophin levels and increases in muscle function Juvenile NHP Plasma Concentration and Exon Skipping • Juvenile NHP data suggest a righ
Slide 22: ENTR-601-44: Mechanistic Rationale
ENTR-601-44: Mechanistic Rationale
Slide 23: EEV-enabled stem cell uptake implies the potential for the return of healthy muscle and function
EEV-enabled stem cell uptake implies the potential for the return of healthy muscle and function Enhanced satellite cell activity would result in the regeneration of healthy fibers and the stabilization of the overall muscle; In turn, this may convert into greater strength, as measured by TTRV Entra
Slide 24: ELEVATE-45-201
THERAPEUTICS BLEVATE-45-204 . = # > F
Slide 25: ELEVATE-45-201: Patient population, competitive treatment and data catalysts
ELEVATE-45-201: Patient population, competitive treatment and data catalysts ELEVATE-45-201 Cohort 1 is enrolled; Dosing completed and clinical data readout in October 2026; Cohort 2 is enrolling and actively dosing with clinical data readout expected H1 2027 Exon 45 Patient Population ELEVATE-45-20
Slide 26: ELEVATE-45-201 is designed to support U.S. Accelerated Approval and forms the basis of a global registrational program
ELEVATE-45-201 is designed to support U.S. Accelerated Approval and forms the basis of a global registrational program* ENTR-601-45 ELEVATE-45-201 is a global, two-part, Primary objective: Safety and tolerability of ENTR-601-45 randomized, double-blind placebo- Secondary objectives: Evaluation of ph
Slide 27: Myotonic Dystrophy Type 1 (DM1)
Myotonic Dystrophy Type 1 (DM1)
Slide 28: Vertex DM1 partnership: Phase 1/2 readout expected in second half of 2026
Vertex DM1 partnership: Phase 1/2 readout expected in second half of 2026 No disease-modifying treatments are currently available for the more than 110,000 people in the U.S. and Europe living with DM1 Vertex completing a placebo-controlled Phase 1/2 clinical program at over 25 global sites • MAD po
Slide 29: Inherited Retinal Diseases (IRDs)
Inherited Retinal Diseases (IRDs)
Slide 30: Addressing areas of high unmet need in IRDs with new oligonucleotide-based therapeutics
Addressing areas of high unmet need in IRDs with new oligonucleotide-based therapeutics Entrada’s first IRD clinical candidate, ENTR-801, will target Usher syndrome type 2A (USH2A); Addressable population is ~15,000 patients* in the U.S. and Europe with no approved therapeutics Usher Syndrome Type 2
Slide 31: 2026 Inflection Points
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Slide 32: Multiple 2026 near-term value drivers anticipated across expanding pipeline of intracellular therapeutics
Multiple 2026 near-term value drivers anticipated across expanding pipeline of intracellular therapeutics ENTR-601-44 ENTR-601-45 ENTR-601-50 ENTR-601-51 Global Phase 1/2 MAD ENTR-601-45 Global Phase 1/2 MAD Received authorization for Clinical Trial Authorization Phase 1/2 MAD study (U.K.) (CTA)-ena
Slide 33: Appendix
Appendix
Slide 34: EEV Platform
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Slide 35: Understanding EEV endosomal escape advantages
Understanding EEV endosomal escape advantages Unique pH-dependent membrane binding affinity enhances EEV endosomal escape • EEVs have a higher affinity to phospholipids compared to traditional linear CPPs; The interaction of EEVs with phospholipids is pH-dependent • The specific interaction of EEVs
Slide 36: Entrada optimizes both the EEV delivery vehicle and the active conjugate to create best-in-class medicines
Entrada optimizes both the EEV delivery vehicle and the active conjugate to create best-in-class medicines In the below DMD example, Entrada has optimized the EEV and the PMO sequence; The data represents the superiority of each component against other approaches and sequences EEV Optimization PMO O
Slide 37: DMD Approach
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Slide 38: Entrada addresses the underlying cause of DMD and restores dystrophin in muscle fibers and muscle stem cells
Entrada addresses the underlying cause of DMD and restores dystrophin in muscle fibers and muscle stem cells The Duchenne Disease Cascade Current and Emerging Treatment Options 1. Disease-modifying agents address the root cause of morbidity and mortality DMD Gene Mutation • High-quality dystrophin p
Slide 39: Entrada is developing genetic medicines to correct the mechanism of disease and slow, halt or reverse progression
Entrada is developing genetic medicines to correct the mechanism of disease and slow, halt or reverse progression Entrada’s Approach: Modulate RNA Splicing and Produce DMD Mutations and Tissues Functional Protein to Preserve and Rebuild Muscle of Focus in the Entrada Portfolio Individuals with Duche
Slide 40: Competitive Comparison
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Slide 41: EEV therapies are expected to show superior dystrophin production and dose-dependent increases in therapeutic index
EEV therapies are expected to show superior dystrophin production and dose-dependent increases in therapeutic index A 25-50-fold improvement in endosomal escape, novel PMO sequences, non-linear increases in PK/PD, satellite cell uptake and lower whole drug requirements compared to antibody-based the
Slide 42: ELEVATE-44-201
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Slide 43: Functional Measures: Time to Rise and Time to Rise Velocity
Functional Measures: Time to Rise and Time to Rise Velocity
Slide 44: Functional improvement
Functional improvement The importance and meaning of Time to Rise velocity Time to Rise, a secondary endpoint in this study, is a robust, prognostic factor that predicts disease progression; Time to Rise velocity reduces the impact of imputation, outliers and noise 1 • TTR declines rapidly over time
Slide 45: Pharmacokinetic Data
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Slide 46: Predictive pharmacokinetics
Predictive pharmacokinetics Lower Cmax and AUC observed in juvenile humans and NHPs; A linear (or better) increase in exposure is expected in Cohort 2 at 12 mg/kg (double dose = double exposure) Human (HNVs and Patients)* Non-Human Primate** Proportional Difference (Juvenile = ~60% of Adult) ~50% Ad
Slide 47: DMD Functional Data: Preclinical Models
DMD Functional Data: Preclinical Models
Slide 48: Preclinical data support potential for best-in-class clinical profile for ENTR-601-45
Preclinical data support potential for best-in-class clinical profile for ENTR-601-45 ENTR-601-45 Dose-dependent increase in exon skipping and dystrophin expression correlates to complete functional correction Exon Skipping Dystrophin Restoration Functional Correction **** 100 100 100 **** 100 ****
Slide 49: Dose-dependent and durable improvements in muscle function observed in del44hDMD.mdx mice
Dose-dependent and durable improvements in muscle function observed in del44hDMD.mdx mice ENTR-601-45 Dose-dependent increase in percent force retention following 10 contractions; Increase maintained for at least 8 weeks after the third Q6W dose of ENTR-601-45 Skeletal Muscle Membrane Stability Stab
Slide 50: Learn more at EntradaTx.com
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