Pepgen Inc. · NASDAQ:PEPG
Pepgen — Investor Presentation (October 2025)
21 slides from Pepgen's investor presentation, Oct 1, 2025
Slide 1: October 2025 Company Presentation
October 2025 Company Presentation
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by words such as “aims,” “anticipates,” “believes,” “could,” “estimates,” “expects,” “forecasts,
Slide 3: Leveraging EDO Platform to Drive Meaningful Impact for Patients
Leveraging EDO Platform to Drive Meaningful Impact for Patients PGN-EDODM1: Myotonic Dystrophy Type 1 Best-in-class potential; selectively targets only pathogenic DMPK RNA Favorable emerging safety profile and highest splicing correction ever reported in patients Orphan Drug & Fast Track Designation
Slide 4: The Challenge of Oligonucleotides
The Challenge of Oligonucleotides PGN-PMODM1 (Naked Oligo at 20 µM) Red = oligo Naked oligonucleotides do not efficiently penetrate muscle cells and nucleus Immortalized myoblasts from a healthy individual or a DM1 patient with 2600 CTG repeats were cultured then differentiated for 4 days into myotu
Slide 5: PepGen's EDO Platform Has Been Designed to Solve this Decades Long Problem
PGN-EDODM1 (10 µM) Red = oligo PepGen’s EDO Platform Has Been Designed and Developed to Solve this Decades Long Problem EDO platform results in nuclear delivery of oligonucleotide therapeutics Immortalized myoblasts from a healthy individual or a DM1 patient with 2600 CTG repeats were cultured then
Slide 6: EDO Technology Can Improve Endosomal Escape and Increase Nuclear Uptake up to 98-Fold
15x 20x 19x 22x 55x 98x 73x PMO Delivery in DM1 Cells EDO Technology can Improve Endosomal Escape and Has Been Shown to Increase Nuclear Uptake up to 98-Fold CELLULAR UPTAKE NUCLEAR UPTAKE Immortalized myoblasts from a healthy individual or a DM1 patient with 2600 CTG repeats were cultured then diff
Slide 7: PGN-EDODM1 – Myotonic Dystrophy Type 1 (DM1)
PGN-EDODM1 – Myotonic Dystrophy Type 1 (DM1)
Slide 8: Myotonic Dystrophy Type 1 Overview and Unmet Medical Need
Myotonic Dystrophy Type 1 Overview and Unmet Medical Need Sources: Neuroepidemiology (2022) 56 (3): 163–173., Neurology 2021 Feb 16;96(7):e1045-e1053 CUG: cytosine-uracil-guanine; DMPK: dystrophia myotonica protein kinase Overview Market Opportunity CUG expansion in the DMPK gene Onset of symptoms v
Slide 9: PGN-EDODM1 Blocking Approach Targets the Pathogenic CUGexp Repeats DMPK RNA
PGN-EDODM1 Blocking Approach Targets the Pathogenic CUGexp Repeats DMPK RNA Wojciechowska, et al., Quantitative Methods to Monitor RNA Biomarkers in Myotonic Dystrophy, Nature, April 12, 2018 DM1 is caused by pathogenic DMPK transcripts PGN-EDODM1 binds selectively to the pathogenic DMPK transcript
Slide 10: PGN-EDODM1 Reduced Pathogenic Nuclear Foci, Liberated MBNL1 and Corrected Mis-Splicing
MBNL1 Liberation Foci Reduction PGN-EDODM1 Reduced Pathogenic Nuclear Foci, Liberated MBNL1 and Corrected Mis-Splicing in Patient Cells with Long CUG Repeats Immortalized myoblasts from healthy individual or DM1 patient with 2600 CTG repeats were cultured then differentiated for 4 days into myotubes
Slide 11: Multiple Doses of PGN-EDODM1 Led to Greater Improvement vs Single Dose in Preclinical Studies
Multiple Doses of PGN-EDODM1 Led to Greater Improvement in Splicing Correction and Myotonia vs Single Dose in Preclinical Studies Protocol: HSALR mice received 1 or 4 doses of PGN-EDODM1, with 4-week intervals between doses. Skeletal muscle tissues were collected 4 weeks post-final dose. Skeletal mu
Slide 12: FREEDOM: Phase 1 PGN-EDODM1 Single-Ascending Dose Study Design
FREEDOM: Phase 1 PGN-EDODM1 Single-Ascending Dose Study Design *Data collection ongoing for 15 mg/kg cohort; data current through Aug 5, 2025 1.15 mg/kg cohort added to expand pharmacokinetic and pharmacodynamic understanding DSMB: data safety monitoring board; IV: intravenous; PBO: placebo; SAD: si
Slide 13: PGN-EDODM1 Produced Mean 53.7% Splicing Correction Following Single 15 mg/kg Dose
PGN-EDODM1 Produced Mean 53.7% Splicing Correction Following Single 15 mg/kg Dose Placebo n=5 as biopsies were not obtained from one of the cohort 3 placebo patients due to biopsy associated complications on day 0. One subject at 10 mg/kg biopsy was not collected at day 28 due to pseudoaneurysm in c
Slide 14: Greater Than Dose-Proportional Increase in Muscle Tissue Concentration After Single Dose
1. One subject at 10 mg/kg did not have biopsy collected at day 28 and was excluded from tissue quant analysis *One subject at 15 mg/kg received 77% of the full dose. Due to limited amount of biopsy, one subject at 15 mg/kg was excluded from tissue quant analysis * Robust, Greater Than Dose-Proporti
Slide 15: Favorable Emerging Safety Profile of PGN-EDODM1
Favorable Emerging Safety Profile of PGN-EDODM1 Summary of Treatment Emergent Adverse Events (TEAEs) 5 mg/kg (n=8)* n(%) 10 mg/kg (n=8)*1 n(%) 15 mg/kg (n=8)* n(%) Any related TEAE 1 (13) 3 (38) 4 (50) Mild/Moderate Severe 1 (13) 0 2 (25) 1 (13) 4 (50) 0 (0) Any Serious Adverse Ev
Slide 16: PGN-EDODM1 Continues to Demonstrate Favorable Safety Profile
PGN-EDODM1 Continues to Demonstrate Favorable Safety Profile PGN-EDODM1 Serum Creatinine in FREEDOM* *Data collection ongoing for 15 mg/kg cohort; data current through Aug 5, 2025 BL: baseline; ULN: upper limit of normal; M: male; F: female All kidney AEs were transient and mild or moderate No inter
Slide 17: Functional Outcomes After Single Dose
Functional Outcomes After Single Dose vHOT: video hand opening time 10-Meter Walk Run Test (10MWRT) at D28 Myotonia (vHOT) at D28 Baseline Day 28 Mean CFB in 10MWRT (sec) Improvement Improvement Baseline Day 28 Mean Middle Finger vHOT (sec)
Slide 18: Summary: PGN-EDODM1 Designed to Address the Underlying Cause of DM1
Summary: PGN-EDODM1 Designed to Address the Underlying Cause of DM1 100% of patients responded to PGN-EDODM1 in 15 mg/kg cohort More than dose-proportionate increases in splicing correction observed across doses at Day 28 Unprecedented splicing creates potential optionality for clinical dosing Highe
Slide 19: FREEDOM2 Phase 2 MAD Study Underway
FREEDOM2 Phase 2 MAD Study Underway DSMB: data safety monitoring board; FU: follow-up; IV: intravenous; MAD: multiple-ascending dose; PBO: placebo; PK: pharmacokinetics; vHOT: video hand opening test; OLE: open label extension FREEDOM2 Study Overview Multinational, randomized, double-blind, placebo-
Slide 20: FREEDOM Study Exceeded Splicing Correction Objectives, Positioning FREEDOM2 for 2026 Readouts
FREEDOM Study Exceeded Splicing Correction Objectives, Positioning FREEDOM2 for Important Readouts in 2026 Favorable emerging safety profile Unprecedented splicing correction achieved with single dose FREEDOM STUDY GOALS: EXPLORATORY: PD (SPLICING) PRIMARY: SAFETY Currently dosing 5 mg/kg cohort 10
Slide 21: Thank you
@ Thank you