Entrada Therapeutics, Inc. · NASDAQ:TRDA
Entrada Therapeutics — Investor Presentation (May 2026)
51 slides from Entrada Therapeutics's investor presentation, May 19, 2026
Slide 1: Meet Andrew and JJ, living with Duchenne muscular dystrophy
Meet Andrew and JJ, living with Duchenne muscular dystrophy Corporate Presentation May 2026
Slide 2: Disclaimer
Disclaimer May 2026 2 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 a
Slide 3: 2026 is a transformational year for Entrada
2026 is a transformational year for Entrada May 2026 Entrada is a clinical-stage biopharma company developing proprietary genetic medicines to deliver best-in-class outcomes in high unmet need diseases *ELEVATE-44-201 Cohort 1, DMD: Duchenne muscular dystrophy; DM1: Myotonic dystrophy type 1; Cash r
Slide 4: Program updates and milestones
May 2026 *All references in this presentation regarding planned regulatory filings and clinical study designs are subject to ongoing discussion with regulatory authorities; DMD: Duchenne muscular dystrophy; DM1: Myotonic dystrophy type 1; SAD: Single ascending dose; MAD: Multiple ascending dose. ENT
Slide 5: Pipeline and Platform Expansion
d Each target disease has a substantial patient population with a significant unmet medical need May 2026 Phase 1/2 ENTR-801: USH2A Undisclosed Undisclosed Duchenne Muscular Dystrophy (DMD) Inherited Retinal Diseases Myotonic Dystrophy Type 1 (DM1) – Partnered with ENTR-601-44 ENTR-601-45 ENTR-601-5
Slide 6: Duchenne Muscular Dystrophy (DMD)
Duchenne Muscular Dystrophy (DMD)
Slide 7: Entrada's DMD strategy is to de-risk a differentiated portfolio and commercially launch best-in-class drugs
Entrada’s DMD strategy is to de-risk a differentiated portfolio and commercially launch best-in-class drugs May 2026 7 • Novel EEV and PMO sequences • Best-in-class non-clinical data translated into compelling safety and functional data • Progenitor cell uptake supports regenerative potential, speed
Slide 8: Entrada is developing genetic medicines to correct the mechanism of disease
Entrada is developing genetic medicines to correct the mechanism of disease and slow, halt or reverse progression May 2026 8 ~8% ~9% ~4% ~15% The portfolio covers ~35% of the total population totaling ~11,500 patients in the U.S. and EU* DMD Mutations and Tissues of Focus in the Entrada Portfolio PM
Slide 9: U.S. Annual Market Opportunity
U.S. Annual Market Opportunity $ Millions $0 $1,000 $2,000 $3,000 $4,000 $5,000 $6,000 May 2026 9 Entrada’s DMD franchise is positioned to address ~1/3 of all U.S. DMD patients, representing a potential ~$5B annual market opportunity in the U.S. alone Entrada’s DMD franchise unlocks a potential $5B
Slide 10: ELEVATE-44-201 Cohort 1: Study Results Summary
ELEVATE-44-201 Cohort 1: Study Results Summary
Slide 11: Executive summary and key takeaways
Executive summary and key takeaways ELEVATE-44-201 Cohort 1 results May 2026 • Favorable safety and tolerability profile at 6 mg/kg; All AEs were mild to moderate ▪ No reported SAEs nor AEs leading to discontinuation ▪ Renal markers within normal range and comparable to placebo • Functional benefit
Slide 12: ELEVATE-44-201 Cohort 1: Trial Design and Treatment Population
ELEVATE-44-201 Cohort 1: Trial Design and Treatment Population
Slide 13: Trial design
May 2026 13 *Expansion cohort and potential for Accelerated Approval is subject to regulatory feedback; Global registration and approval will require Phase 3 studies to follow the Phase 1/2 studies; Phase 3 studies will likely include functional measures as the primary endpoint (U.S. confirmatory st
Slide 14: Demographics and baseline characteristics
Demographics and baseline characteristics May 2026 ELEVATE-44-201 Placebo n=2 ENTR-601-44 6 mg/kg n=6 Age, mean 13.5 9.3 Body mass index, mean, kg/m2 17.96 20.00 Age at disease onset, mean, years 1.0 2.2 Corticosteroid use, n (%) 2 (100%) 6 (100%) Ambulatory, n (%) 2 (100%) 6 (100%) Baseline dystrop
Slide 15: ELEVATE-44-201 Cohort 1: Safety Data
ELEVATE-44-201 Cohort 1: Safety Data
Slide 16: Safety was consistent and unremarkable at the mg/kg dose
Safety was consistent and unremarkable at the 6 mg/kg dose May 2026 ELEVATE-44-201 TEAE: Treatment emergent adverse event. 16 Participants with ≥1 TEAE, n (%) Placebo n=2 ENTR-601-44 6 mg/kg n=6 Any TEAE 2 (100%) 6 (100%) TEAEs related to study drug 1 (50%) 5 (83%) Serious TEAEs 0 0 TEAEs leading to
Slide 17: Renal markers were within normal range and comparable to placebo
May 2026 Data from ELEVATE-44-201; eGFR: Estimated glomerular filtration rate; LLN: Lower limit of normal; ULN: Upper limit of normal; Serum creatinine reference range shown is 27–106 μmol/L and is dependent on age of the participant enrolled in Cohort 1; Serum creatinine is below normal in particip
Slide 18: ELEVATE-44-201 Cohort 1: Functional Efficacy Data
ELEVATE-44-201 Cohort 1: Functional Efficacy Data
Slide 19: Functional benefit summary
Functional benefit summary May 2026 • Time to Rise and Time to Rise Velocity are the first functional measurements to ”move” • Functional benefit demonstrated via Time to Rise and Time to Rise Velocity measurements ▪ TTRV is a calculation based on TTR that minimizes any imputation, outliers and nois
Slide 20: The importance and meaning of Time to Rise velocity
Functional improvement The importance and meaning of Time to Rise velocity May 2026 20 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.Cicala et al., Itali
Slide 21: Functional cascade: TTR velocity is a robust indicator of treatment effect
Functional cascade: TTR velocity is a robust indicator of treatment effect and the earliest measurement to move May 2026 21 Proximal functional measures, including TTR and 4SC, detect changes before composite/distal measures like PUL and NSAA; TTR and TTRV were statistically significant versus place
Slide 22: Disease-modifying functional benefit seen at lowest dose
Disease-modifying functional benefit seen at lowest dose TTRV results May 2026 22 *Analysis on TTRV versus placebo shown rather than TTR in order to maintain the study blind, Wilcoxon rank-sum (one sided) test, nominal p-values, post hoc analysis; MCID: “The Minimal Clinical Important Difference (MC
Slide 23: ENTR-601-44 delivered a differentiated impact on TTRV
ENTR-601-44 delivered a differentiated impact on TTRV TTRV is a validated registrational endpoint May 2026 23 Sources: *VISION-DMD (JAMA 2022) registrational study; DELIVER (Dyne Company presentation); EMBARK (FDA Decisional Memo 2024); ENTR-601-44 (Cohort 1); No head-to-head trials have been conduc
Slide 24: ELEVATE-44-201: Pharmacokinetics and Biomarkers
ELEVATE-44-201: Pharmacokinetics and Biomarkers
Slide 25: Lower-than-expected plasma exposures resulted in lower exon skipping and dystrophin expression
May 2026 Lower-than-expected plasma exposures resulted in lower-than-expected exon skipping and dystrophin expression AUC: Area under the curve (proportions are approximate); 1.Healthy normal volunteer, single dose 6 mg/kg, 2.Pediatric DMD participants following third dose of 6 mg/kg every 6 weeks (
Slide 26: ENTR-601-44: Mechanistic Rationale
ENTR-601-44: Mechanistic Rationale
Slide 27: The DMD challenge: Muscle damage and impaired repair
May 2026 Source: Servais L, et al. ICNMD 2026. Healthy muscle The DMD challenge: Muscle damage and impaired repair An ideal treatment is regenerative – replacing damaged, dystrophic muscle with healthy muscle; This requires satellite cell correction which then promotes asymmetric differentiation DMD
Slide 28: Enhanced satellite cell activity would result in regeneration of healthy fibers
May 2026 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 D2-mdx: Dystrophic mouse model; D2-mdx mice were treated with 3 IV doses of EEV-PMO administe
Slide 29: Myotonic Dystrophy Type 1 (DM1)
Myotonic Dystrophy Type 1 (DM1)
Slide 30: DM1 partnership established with Vertex
May 2026 30 DM1 partnership established with Vertex in Q1 2023 • Entrada received an upfront payment of $224M and an equity investment of $26M upon initiation of the collaboration • Agreement provided for up to $485M for the achievement of milestones • Tiered royalties on future net sales VX-670 dif
Slide 31: Inherited Retinal Diseases (IRDs)
Inherited Retinal Diseases (IRDs)
Slide 32: Addressing high unmet need in IRDs with oligonucleotide-based therapeutics
Addressing areas of high unmet need in IRDs with new oligonucleotide-based therapeutics May 2026 Usher Syndrome Type 2A (USH2A) • An inherited eye condition caused by changes in the USH2A gene • In some patients, mutations in exon 13 prevent production of the usherin protein • Without usherin, photo
Slide 33: 2026 Inflection Points
> _ 5 ; pentrada > = = , 2026 Inflection Points = o )
Slide 34: Open-label functional data, Cohort 2 and ELEVATE-45-201 data are 2026 catalysts
May 2026 ELEVATE-44-201 Cohort 1 open-label functional data, Cohort 2 dystrophin and functional data and ELEVATE-45-201 Cohort 1 data represent additional 2026 catalysts • All ELEVATE-44-201 Cohort 1 participants have rolled over into the open-label portion of the study ▪ Placebo participants crosse
Slide 35: Multiple 2026 near-term value drivers
Note: All references in this presentation regarding planned regulatory filings and clinical study designs are subject to ongoing discussion with regulatory authorities; *Based on May 2026 current operating plans and ~$255M in cash, cash equivalents and marketable securities as of March 31, 2026. 35
Slide 36: Appendix
2 w » > aga Appendix 5 >
Slide 37: EEV Platform
EEV™ Platform
Slide 38: Understanding EEV endosomal escape advantages
Understanding EEV endosomal escape advantages May 2026 38 Unique pH-dependent membrane binding affinity enhances EEV endosomal escape Sahni, A. et al. ACS Chem. Biol. 2021; CPP: Cell-penetrating peptide. • EEVs have a higher affinity to phospholipids compared to traditional linear CPPs; The interact
Slide 39: Entrada optimizes both the EEV delivery vehicle and the active conjugate
R6-PMO EEV-PMO 0 10 20 30 40 50 60 70 80 90 100 Exon Skipping (%) * • PMO sequences are the same, cell penetrating peptides are different (linear vs. cyclic) • EEV-PMO significantly improved exon skipping after 3 days in mdx mice as compared to competitive R6-PMO EEV Optimization PMO Optimization •
Slide 40: DMD Approach
DMD Approach
Slide 41: Entrada addresses the underlying cause of DMD and restores dystrophin
Inflammation Necrosis Fibrogenesis Atrophy Entrada addresses the underlying cause of DMD and restores dystrophin in muscle fibers and muscle stem cells May 2026 Adapted from Front. Cell Dev. Biol., 18 August 2021; Sec. Molecular and Cellular Pathology; Volume 9, 2021; 1.No other exon skippers nor ge
Slide 42: Competitive Comparison
Competitive Comparison
Slide 43: Competitive comparison of delivery approaches
Design Dose Uptake Target engagement Anti-TfR1-mAb-PMO (exon 44) Novartis/Avidity Anti-TfR1-Fab-PMO (exon 51) Dyne Full-length antibody ~150 kDa TfR1 specific deliver; Non-cleavable linker Fragment antibody ~50 kDa TfR1 specific delivery; Cleavable linker Large conjugate doses 5 mg/kg PMO (registrat
Slide 44: Pharmacokinetic Modeling
Pharmacokinetic Modeling
Slide 45: Predictive pharmacokinetics
May 2026 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) Predictive pharmacokinetics *Healthy normal volunteer, single dose 6mg/kg, pediatric DMD patients following third dose; **Ad
Slide 46: Juvenile NHP plasma concentration and exon skipping
May 2026 46 Juvenile NHP Plasma Concentration and Exon Skipping Expected AUC in Cohort 1 • Juvenile NHP data suggest a right-shifted double-digit dystrophin expectation starting at 12 mg/kg in Cohort 2 • As the exposure to exon skipping relationship has not changed, updated projections indicate sign
Slide 47: DMD Functional Data Preclinical Models
DMD Functional Data Preclinical Models
Slide 48: Preclinical data support best-in-class potential for ENTR-601-45
Preclinical data support potential for best-in-class clinical profile for ENTR-601-45 May 2026 48 Data are shown as mean ± SD; *p ≤ 0.05; **p ≤ 0.01; ***p ≤ 0.001; ****p ≤ 0.0001; 25 mg/kg correlates to ~5 mg/kg human equivalent dose (HED), 50 mg/kg correlates to ~10 mg/kg HED, 75 mg/kg correlates t
Slide 49: Dose-dependent and durable improvements in muscle function in del44hDMD.mdx mice
Dose-dependent and durable improvements in muscle function observed in del44hDMD.mdx mice May 2026 49 del44hDMD.mdx mice were treated with three Q6W IV injections of ENTR-601-45 or vehicle; ECC-induced muscle force loss generated by repeated eccentric force (ECC) contraction of the gastrocnemius mus
Slide 50: Efficacy of ENTR-601-50 in exon skipping amenable mice
Efficacy of ENTR-601-50 in exon 50 skipping amenable mice Single dose of ENTR-601-50 produced robust human DMD exon 50 skipping and dystrophin; Translated into improvements in muscle function within 2 weeks post-dose ENTR-601-50 Exon Skipping and Dystrophin in the Gastrocnemius Percent Force After 1
Slide 51: Learn more at EntradaTx.com
Learn more at EntradaTx.com
Entrada Therapeutics — Investor Presentation (May 2026) on 6ix