Entrada Therapeutics, Inc. · NASDAQ:TRDA
Entrada Therapeutics — 2025 J.P. Morgan Healthcare Conference
25 slides from Entrada Therapeutics's conference presentation, Jan 15, 2025
Slide 1: 2025 J.P. Morgan Healthcare Conference
2025 J.P. Morgan Healthcare Conference NASDAQ: TRDA
Slide 2: Disclaimer
Disclaimer This presentation has been prepared by Entrada Therapeutics, Inc. (the “Company”) and shall not constitute an offer to sell o r 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 jur
Slide 3: Entrada is entering 2025 with momentum
d Entrada is entering 2025 with momentum January 2025 3 Entrada enters 2025 with significant momentum January 2025 3 Entrada expects to have four Phase 1/2 MAD patient studies in DMD and DM1 by the end of 2025* Actively planning the initiation of two global Phase 1/2 MAD studies in H1 2025 and one i
Slide 4: Our Mission: To Treat Devastating Diseases With Intracellular Therapeutics
OUR MISSION: To Treat Devastating Diseases With Intracellular Therapeutics January 2025 4
Slide 5: Breakthrough approach to intracellular therapeutics
Breakthrough approach to intracellular therapeutics January 2025 5 75% of disease - causing targets are located inside cells 1 Increasing cellular uptake and improving endosomal escape Potential for best - in - class therapeutics These targets are largely considered to be inaccessible and undruggabl
Slide 6: Endosomal Escape Vehicle (EEV)-based therapies
Endosomal Escape Vehicle (EEV ) - based therapies January 2025 6 High Intracellular Uptake ~90% Uptake Efficient Endosomal Escape ~50% Escape vs. ~2% Standard Unique Budding Mechanism Conserves Endosomal Integrity Unique chemistry Improved uptake and endosomal escape Cyclic structure Extended half -
Slide 7: An Expanding Pipeline of Intracellular Therapeutics
d Entrada’s pipeline includes a diverse array of high potential and high value assets Each target disease has a substantial patient population with a significant unmet medical need An Expanding Pipeline of Intracellular Therapeutics January 2025 7 DM1 DMD ENTR - 601 - 50 VX - 670 ENTR - 601 - 44 ENT
Slide 8: EEV therapies have potential for a best-in-class approach in neuromuscular diseases
EEV therapies have the potential for a best - in - class approach in neuromuscular diseases January 2025 8 Delivered positive Phase 1 data in DMD (ENTR - 601 - 44) • Robust clinical validation in healthy volunteers • No treatment - related AEs • Potential best - in - class target exposure and target
Slide 9: Positive Phase 1 data support initiation of Phase 1/2 MAD study in patients
(n=6:2) (n=6:2) (n=6:2) (n=6:2) Positive ENTR - 601 - 44 Phase 1 data support the initiation of a Phase 1/2 MAD clinical study in patients January 2025 *Data presented at 2024 World Muscle Society conference; **One participant enrolled and randomized into Cohort 3 was removed pri or to dosing. 9 Coh
Slide 10: 44-101: Safety
ENTR - 601 - 44 - 101: Safety Safety and tolerability were assessed at each study visit following a single IV dose of ENTR - 601 - 44 or placebo. One participant enrolled and randomized into Cohort 3 was removed prior to dosing. Renal biomarkers assessed using FNIH and the C - Path . Kidney Safety C
Slide 11: Plasma concentration and urinary excretion of PMO-44 metabolite
Plasma Concentration of ENTR - 601 - 44 Dose - Dependent Increases In Urinary Excretion of Final PMO - 44 Metabolite ENTR - 601 - 44 Dose (mg/kg) Plasma ENTR - 601 - 44 AUC last ( hr *ng/mL) 0 3 0.75 1.5 6 0 20,000 40,000 60,000 11 For every doubling of dose, there is a more than doubling of metabol
Slide 12: 44-101: Target Exposure and Engagement
Muscle concentrations and exon skipping were assessed using a needle muscle biopsy taken from biceps brachii 72 hours ( ± 4 hours) post - dose of ENTR - 601 - 44. Box and whisker plot illustration: the boxes represent the IQR and median. Whiskers show the smallest and largest values within 1.5 times
Slide 13: Rapidly expanding DMD clinical programs
Rapidly expanding DMD clinical programs January 2025 13 ENTR - 601 - 44’s Phase 1 results unlock DMD portfolio investment across multiple populations Regulatory Filings Under Review Accelerated Program Timelines ENTR - 601 - 44 ENTR - 601 - 45 ENTR - 601 - 51 IND Enabling ENTR - 601 - 50 IND Enablin
Slide 14: Clinical strategy designed for efficient regulatory path
Multiple Ascending Dose Clinical strategy is designed for efficient regulatory path January 2025 14 All ENTR - 601 - series programs will follow a similar clinical and regulatory approach Multiple Ascending Dose Phase 2b Accelerated Approval Regulatory Filings* Direct to Patient Studies Accelerated
Slide 15: Robust preclinical data support global direct-to-patient Phase 1/2 MAD studies
Robust preclinical data support global direct - to - patient Phase 1/2 MAD clinical studies across DMD franchise January 2025 15 ENTR - 601 - 50 ENTR - 601 - 45 • Robust dystrophin restoration in del44hDMD. mdx mouse model after just 3 doses, 6 weeks apart • Complete functional correction and mainte
Slide 16: Ongoing exploration of pipeline expansion opportunities
Gene Editing RNA Splicing RNA Editing RNA Silencing RNA Blocking Protein Replacement Protein Degradation Protein Inhibition Ongoing exploration of pipeline expansion opportunities Deliver CRISPR enzyme and repair gene function with guide RNA Deliver oligonucleotide therapeutics for RNA editing Modif
Slide 17: Multiple near and long-term value drivers
Multiple near and long - term value drivers Cash runway extended into Q2 2027 January 2025 17 • ENTR - 601 - 44: Discussions underway with several regulatory agencies • ENTR - 601 - 45: Global Phase 1/2 MAD regulatory filings ongoing • ENTR - 601 - 50: Global Phase 1/2 MAD regulatory filings expecte
Slide 18: Q&A
Q&A
Slide 19: Learn more at EntradaTx.com
Learn more at EntradaTx.com entrac THERAPEUTIC!
Slide 20: Consistent and durable efficacy across species
Consistent and durable efficacy across species ~40 - fold higher ENTR - 601 - 44 Exon 44 Skipping in Healthy and Patient Myoblasts Exon 44 Skipping (%) Healthy Cells; n=4 cells/dose Patient Cells (DMDD45); n=4 cells/dose Exon 44 Skipping (%) W e e k 1 W e e k 2 W e e k 4 W e e k 8 W e e k 1 2 0 20 4
Slide 21: Dose-dependent exon skipping and dystrophin
Dose - dependent exon skipping and dystrophin • Del45hDMD.mdx mice dosed with EEV - PMO - 44*** • n=4, gastrocnemius sample collection 2 weeks post - injection Dystrophin Restoration** 15 mg/kg 30 mg/kg 60 mg/kg 0 20 40 60 80 100 % D y s t r o p h i n R e s t o r a t i o n Dose Exon Skipping* 15 mg/
Slide 22: Preclinical data support potential for best-in-class clinical profile
Preclinical data support potential for best - in - class clinical profile 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 to ~15 mg/kg HED; JESS: a
Slide 23: Functional correction in del44hDMD.mdx mice
January 2025 23 del44hDMD. mdx mice were treated with three Q6W IV injections of ENTR - 601 - 45 or vehicle. ECC - induced muscle force loss generated by repeated ec centric force (ECC) contraction of the gastrocnemius muscle was assessed 5 weeks (left/center) or 4 and 8 weeks (right) after the thir
Slide 24: Repeated doses in hDMD show durable exon skipping
ENTR - 601 - 50 in hDMD show high levels of durable exon skipping • Repeated doses administered via IV injection (Q6W x 3 doses) with 1 or 6 - week washout; Exon skipping assessed by ddPCR (tibialis anterior muscle shown) Data are shown as mean ± SD; **p ≤ 0.01, ****p ≤ 0.0001 vs vehicle. d dPCR , d
Slide 25: Learn more at EntradaTx.com
Learn more at EntradaTx.com entrac THERAPEUTIC!
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