Pepgen Inc. · NASDAQ:PEPG
Pepgen — Investor Presentation (March 2026)
20 slides from Pepgen's investor presentation, Mar 4, 2026
Slide 1: March 2026 Company Presentation
March 2026 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 Regulatory clearance received in Sou
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: A Differentiated Drug with Best-in-Class Potential
1. FREEDOM 15 mg/kg data readout when compared with prior published data. Differentiated Delivery Technology Receptor-independent EDO peptide delivery Designed to escape the endosome – unlike TfnR targeting 1 Differentiated Target Selectively targets pathogenic RNA (CUG repeat in DMPK) Demonstrated
Slide 8: PGN-EDODM1 – Myotonic Dystrophy Type 1 (DM1)
PGN-EDODM1 – Myotonic Dystrophy Type 1 (DM1)
Slide 9: 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 10: 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 11: 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 12: Multiple Doses Led to Greater Improvement in Splicing Correction and Myotonia vs Single Dose
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 13: FREEDOM: Phase 1 PGN-EDODM1 Single-Ascending Dose Study Design
FREEDOM: Phase 1 PGN-EDODM1 Single-Ascending Dose Study Design DSMB: data safety monitoring board; IV: intravenous; PBO: placebo; SAD: single-ascending dose; PK: pharmacokinetics PGN-EDODM1 dose FREEDOM Phase 1 Study Overview Multinational, randomized, double-blind, placebo-controlled SAD study in p
Slide 14: PGN-EDODM1 Produced Dose-Dependent Best-in-Class Splicing Correction Following Single Dose
PGN-EDODM1 Produced Dose-Dependent Best-in-Class Splicing Correction Following Single Dose Splicing Index Changes: 22-Gene Panel* at D28 Improvement -53.7% -29.1% 3.3% -12.3% 87.5% of participants across all doses showed improved splicing % Mean change from baseline Placebo1 (n=5) 5 mg/kg (n=6) 10 m
Slide 15: Greater Than Dose-Proportional Increase in Muscle Tissue Concentration Following Single Dose
1.Missing samples due to unavailability of biopsy tissue 2.One subject at 15mg/kg received 77% of the dose and was still included in the muscle tissue concentration analysis for the cohort * Robust, Greater Than Dose-Proportional Increase in Muscle Tissue Concentration Following Single Dose 5 mg/kg
Slide 16: PGN-EDODM1 Was Generally Well Tolerated, with TEAEs Primarily Mild to Moderate
PGN-EDODM1 Was Generally Well Tolerated, with TEAEs Primarily Mild to Moderate Across Dose Cohorts *As of database lock on December 23, 2025. Unblinded FREEDOM safety data TEAE: treatment-emergent adverse event, SAE: serious adverse event, DLT: Dose limiting toxicity, eGFR(cys): estimated glomerular
Slide 17: 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 * Dose dependent on recommendations of DSMB **The U.S. FDA recently place
Slide 18: Summary: PGN-EDODM1 Designed to Address the Underlying Cause of DM1
Summary: PGN-EDODM1 Designed to Address the Underlying Cause of DM1 87.5% of participants across all doses showed improved splicing More than dose-proportionate increases in splicing correction observed across doses at Day 28 Unprecedented splicing provides a window of pharmacodynamically active dos
Slide 19: Summary of PGN-EDODM1 and FREEDOM Program
Summary of PGN-EDODM1 and FREEDOM Program Favorable emerging safety profile Unprecedented splicing correction achieved with single dose FREEDOM STUDY : EXPLORATORY: PD (SPLICING) PRIMARY: SAFETY Fully enrolled 5 mg/kg cohort; All patients have completed dosing Dosing patients in 10 mg/kg cohort; 50%
Slide 20: Thank you
@ Thank you